[House Hearing, 114 Congress]
[From the U.S. Government Publishing Office]


 EXAMINING THE REGULATION OF DIAGNOSTIC TESTS AND LABORATORY OPERATIONS

=======================================================================

                                 HEARING

                               BEFORE THE

                         SUBCOMMITTEE ON HEALTH

                                 OF THE

                    COMMITTEE ON ENERGY AND COMMERCE
                        HOUSE OF REPRESENTATIVES

                    ONE HUNDRED FOURTEENTH CONGRESS

                             FIRST SESSION

                               __________

                           NOVEMBER 17, 2015

                               __________

                           Serial No. 114-100
                           
                           
[GRAPHIC NOT AVAILABLE IN TIFF FORMAT]                           


      Printed for the use of the Committee on Energy and Commerce

                        energycommerce.house.gov
                        
                              ___________
                               
                               
                        U.S. GOVERNMENT PUBLISHING OFFICE
99-657 PDF                    WASHINGTON : 2016                        
  
________________________________________________________________________________________                   
For sale by the Superintendent of Documents, U.S. Government Publishing Office, 
http://bookstore.gpo.gov. For more information, contact the GPO Customer Contact Center,
U.S. Government Publishing Office. Phone 202-512-1800, or 866-512-1800 (toll-free). 
E-mail, [email protected].  

             
                   
                   
                   
                   
                   COMMITTEE ON ENERGY AND COMMERCE

                          FRED UPTON, Michigan
                                 Chairman

JOE BARTON, Texas                    FRANK PALLONE, Jr., New Jersey
  Chairman Emeritus                    Ranking Member
ED WHITFIELD, Kentucky               BOBBY L. RUSH, Illinois
JOHN SHIMKUS, Illinois               ANNA G. ESHOO, California
JOSEPH R. PITTS, Pennsylvania        ELIOT L. ENGEL, New York
GREG WALDEN, Oregon                  GENE GREEN, Texas
TIM MURPHY, Pennsylvania             DIANA DeGETTE, Colorado
MICHAEL C. BURGESS, Texas            LOIS CAPPS, California
MARSHA BLACKBURN, Tennessee          MICHAEL F. DOYLE, Pennsylvania
  Vice Chairman                      JANICE D. SCHAKOWSKY, Illinois
STEVE SCALISE, Louisiana             G.K. BUTTERFIELD, North Carolina
ROBERT E. LATTA, Ohio                DORIS O. MATSUI, California
CATHY McMORRIS RODGERS, Washington   KATHY CASTOR, Florida
GREGG HARPER, Mississippi            JOHN P. SARBANES, Maryland
LEONARD LANCE, New Jersey            JERRY McNERNEY, California
BRETT GUTHRIE, Kentucky              PETER WELCH, Vermont
PETE OLSON, Texas                    BEN RAY LUJAN, New Mexico
DAVID B. McKINLEY, West Virginia     PAUL TONKO, New York
MIKE POMPEO, Kansas                  JOHN A. YARMUTH, Kentucky
ADAM KINZINGER, Illinois             YVETTE D. CLARKE, New York
H. MORGAN GRIFFITH, Virginia         DAVID LOEBSACK, Iowa
GUS M. BILIRAKIS, Florida            KURT SCHRADER, Oregon
BILL JOHNSON, Ohio                   JOSEPH P. KENNEDY, III, 
BILLY LONG, Missouri                 Massachusetts
RENEE L. ELLMERS, North Carolina     TONY CARDENAS, California7
LARRY BUCSHON, Indiana
BILL FLORES, Texas
SUSAN W. BROOKS, Indiana
MARKWAYNE MULLIN, Oklahoma
RICHARD HUDSON, North Carolina
CHRIS COLLINS, New York
KEVIN CRAMER, North Dakota

                         Subcommittee on Health

                     JOSEPH R. PITTS, Pennsylvania
                                 Chairman
BRETT GUTHRIE, Kentucky              GENE GREEN, Texas
  Vice Chairman                        Ranking Member
ED WHITFIELD, Kentucky               ELIOT L. ENGEL, New York
JOHN SHIMKUS, Illinois               LOIS CAPPS, California
TIM MURPHY, Pennsylvania             JANICE D. SCHAKOWSKY, Illinois
MICHAEL C. BURGESS, Texas            G.K. BUTTERFIELD, North Carolina
MARSHA BLACKBURN, Tennessee          KATHY CASTOR, Florida
CATHY McMORRIS RODGERS, Washington   JOHN P. SARBANES, Maryland
LEONARD LANCE, New Jersey            DORIS O. MATSUI, California
H. MORGAN GRIFFITH, Virginia         BEN RAY LUJAN, New Mexico
GUS M. BILIRAKIS, Florida            KURT SCHRADER, Oregon
BILLY LONG, Missouri                 JOSEPH P. KENNEDY, III, 
RENEE L. ELLMERS, North Carolina         Massachusetts
LARRY BUCSHON, Indiana               TONY CARDENAS, California
SUSAN W. BROOKS, Indiana             FRANK PALLONE, Jr., New Jersey (ex 
CHRIS COLLINS, New York                  officio)
JOE BARTON, Texas
FRED UPTON, Michigan (ex officio)

                                  (ii)
                                  
                             C O N T E N T S

                              ----------                              
                                                                   Page
Hon. Joseph R. Pitts, a Representative in Congress from the 
  Commonwealth of Pennsylvania, opening statement................     1
    Prepared statement...........................................     3
Hon. Gene Green, a Representative in Congress from the State of 
  Texas, opening statement.......................................     4
    Prepared statement...........................................     5
Hon. Michael C. Burgess, a Representative in Congress from the 
  State of Texas, opening statement..............................     6
Hon. Frank Pallone, Jr., a Representative in Congress from the 
  State of New Jersey, opening statement.........................     8
    Prepared statement...........................................     9
Hon. Fred Upton, a Representative in Congress from the State of 
  Michigan, prepared statement...................................    65
Hon. Renee L. Ellmers, a Representative in Congress from the 
  State of North Carolina, prepared statement....................    66

                               Witnesses

Jeffrey Shuren, M.D., Director, Center for Devices and 
  Radiological Health, Food and Drug Administration, Department 
  of Health and Human Services...................................    10
    Prepared statement...........................................    12
    Answers to submitted questions \1\...........................    84
Patrick Conway, M.D., Acting Principal Deputy Administrator, 
  Deputy Administrator for Innovation and Quality, and Chief 
  Medical Officer, Centers for Medicare and Medicaid Services, 
  Department of Health and Human Services........................    24
    Prepared statement...........................................    26
    Answers to submitted questions...............................    85

                           Submitted Material

Discussion Draft, H.R. ___, submitted by Mr. Pitts\2\
Letter of November 16, 2015, from American Clinical Laboratory 
  Association, et al., to the Honorable Lamar Alexandar, et al., 
  submitted by Mr. Pitts.........................................    67
Letter of November 11, 2015, from Action to Cure Kidney Cancer, 
  et al., to Mr. Upton, et al., submitted by Mr. Pitts...........    70
Letter of November 16, 2015, from Dick Woodruff, Vice President, 
  Federal Relations, American Cancer Society Cancer Action 
  Network, to Mr. Upton and Mr. Pallone, submitted by Mr. Green..    72
Statement of the Association for Molecular Pathology, November 
  17, 2015, submitted by Mr. Burgess.............................    74
Letter of November 16, 2015, from Jan A. Nowak, Clinical Chief of 
  Molecular Pathology, Roswell Park Cancer Institute, to Mr. 
  Collins, submitted by Mr. Collins..............................    77

----------
\1\ Dr. Shuren did not answer submitted questions for the record by the 
time of printing.
\2\ The discussion draft has been retained in committee files and also 
is available at  http://docs.house.gov/meetings/IF/IF14/20151117/
104127/BILLS-114pih-HR--------.pdf.
Report by the Office of Public Health Strategy and Analysis, 
  Office of the Commissioner, Food and Drug Administration, ``The 
  Public Health Evidence for FDA Oversight of Laboratory 
  Developed Tests: 20 Case Studies,'' November 16, 2015, 
  submitted by Mr. Green \3\
Letter of November 17, 2015, from David D. Koch, President, 
  American Association of Clinical Chemistry, to Mr. Upton, et 
  al., submitted by Mr. Burgess..................................    79

----------
\3\ The report has been retained in committee files and also is 
available at  http://docs.house.gov/meetings/IF/IF14/20151117/104127/
HMTG-114-IF14-20151117-SD009.pdf.

 
 EXAMINING THE REGULATION OF DIAGNOSTIC TESTS AND LABORATORY OPERATIONS

                              ----------                              


                       TUESDAY, NOVEMBER 17, 2015

                  House of Representatives,
                            Subcommittee on Health,
                          Committee on Energy and Commerce,
                                                    Washington, DC.
    The subcommittee met, pursuant to call, at 10:03 a.m., in 
room 2322, Rayburn House Office Building, Hon. Joseph R. Pitts 
(chairman of the subcommittee) presiding.
    Members present: Representatives Pitts, Guthrie, Barton, 
Shimkus, Murphy, Burgess, Blackburn, Lance, Griffith, 
Bilirakis, Long, Ellmers, Bucshon, Brooks, Collins, Green, 
Capps, Butterfield, Castor, Schrader, Kennedy, Cardenas, and 
Pallone (ex officio).
    Staff present: Rebecca Card, Assistant Press Secretary; 
Carly McWilliams, Professional Staff Member, Health; Graham 
Pittman, Legislative Clerk; Heidi Stirrup, Health Policy 
Coordinator; John Stone, Counsel, Health; Christine Brennan, 
Democratic Press Secretary; Jeff Carroll, Democratic Staff 
Director; Tiffany Guarascio, Democratic Deputy Staff Director 
and Chief Health Advisor; Samantha Satchell, Democratic Policy 
Analyst; and Kimberlee Trzeciak, Democratic Health Policy 
Advisor.
    Mr. Pitts. The subcommittee will come to order. The Chair 
will recognize himself for an opening statement.

OPENING STATEMENT OF HON. JOSEPH R. PITTS, A REPRESENTATIVE IN 
         CONGRESS FROM THE COMMONWEALTH OF PENNSYLVANIA

    Throughout the 21st Century Cures initiative, biomarkers, 
precision medicine, and targeted therapies were a few of the 
most consistently uttered terms and concepts. In order to 
advance each of them, we must establish a regulatory 
environment that fosters the development of, and access to, 
innovative, accurate, and reliable diagnostic testing. Such 
tests are increasingly important not only in diagnosing the 
onset of a specific disease or condition, but in determining 
the right course of treatment or procedure.
    It goes without saying that tests providing information to 
a doctor or consumer are fundamentally different products than 
traditional medical devices, which actually deliver therapy to, 
or are implanted in, a patient. Nonetheless, while FDA has used 
its medical device authorities to review and oversee tests 
developed by outside entities that are then sold to 
laboratories, the agency has not actively regulated laboratory-
developed tests, or LDTs.
    Last year, a week after we held a roundtable downstairs 
that highlighted the importance of this very topic, FDA 
announced that it would no longer exercise such enforcement 
discretion and detailed how the agency proposes to apply its 
medical device authorities to LDTs.
    Today, I am far less interested in litigating the 
boundaries of current FDA or CMS legal authority, but in 
hearing from our witnesses how such authority could be 
clarified or improved, understanding the unique and evolving 
nature of what is being regulated and each agency's area of 
expertise.
    In response to a white paper the committee circulated at 
the end of last year asking these very questions, we heard from 
a number of labs and pathologists that FDA should only have a 
limited role, if any, in regulating a select set of tests as 
medical devices. The rest, in their opinion, should be overseen 
by CMS through an updated Clinical Laboratory Improvement 
Amendments program. This is despite the fact that CMS has 
stated that they do not have the resources, the expertise, or 
the willingness to take on what is being asked of them. I am 
eager to hear what Dr. Conway has to say on this matter.
    We also received comments from a number of manufacturers, 
as well as over 40 patient groups, that FDA, not CMS, needs to 
be in the driver's seat, and that tests that have the same 
impact on a patient should be held to the same standards, 
regardless of who does the development. This is despite the 
fact that laboratories are uniquely nimble environments where 
pathologists continually modify and improve tests in ways that 
manufacturers cannot.
    I am well aware that this has been at times a heated debate 
with passionate advocates on both sides. With such a backdrop, 
I want to particularly commend the manufacturers, the 
laboratories, and other healthcare institutions that have been 
willing to roll up their sleeves and find as much common ground 
as possible through constructive dialogue, a willingness to 
compromise, and a pragmatic understanding of what a viable, 
modern framework entails.
    I do not believe that imposing a new regulatory reality on 
an increasingly important component of our healthcare system 
via guidance is the best way to address these issues. These 
products warrant a regulatory system designed with them in 
mind. They should not be shoehorned into a system that was 
drafted in the 1970s.
    This committee has clearly shown that we are willing and 
able to move complicated, comprehensive, bipartisan 
legislation. The discussion draft the committee circulated, 
along with the hearing notice, is of course not perfect, but it 
is a serious document based on significant consensus, and I 
would ask that all of the stakeholders out there, including our 
two distinguished witnesses, help us improve it as the process 
continues.
    With that, I would like to thank Dr. Shuren, a frequent, 
always welcome visitor, as well as Dr. Conway, for their 
willingness to testify today, and I look forward to working 
with them on these issues going forward.
    [The discussion draft is available at  http://
docs.house.gov/meetings/IF/IF14/20151117/104127/BILLS-114pih-
HR--------.pdf.]
    [The prepared statement of Mr. Pitts follows:]

               Prepared statement of Hon. Joseph R. Pitts

    Throughout the 21st Century Cures initiative, 
``biomarkers,'' ``precision medicine,'' and ``targeted 
therapies'' were a few of the most consistently uttered terms 
and concepts. In order to advance each of them, we must 
establish a regulatory environment that fosters the development 
of and access to innovative, accurate and reliable diagnostic 
testing.
    Such tests are increasingly important, not only diagnosing 
the onset of a specific disease or condition, but in 
determining the right course of treatment or procedure. It goes 
without saying that tests providing information to a doctor or 
consumer are fundamentally different products than traditional 
medical devices which actually deliver therapy to or are 
implanted in a patient. Nonetheless, while FDA has used its 
medical device authorities to review and oversee tests 
developed by outside entities that are then sold to 
laboratories, the agency has not actively regulated laboratory 
developed tests, or LDTs.
    Last year, a week after we held a roundtable downstairs 
that highlighted the importance of this very topic, FDA 
announced that it would no longer exercise such enforcement 
discretion and detailed how the agency proposes to apply its 
medical device authorities to LDTs.
    Today, I am far less interested in litigating the 
boundaries of current FDA or CMS legal authority than in 
hearing from our witnesses about how such authority could be 
clarified or improved, understanding the unique and evolving 
nature of what it is being regulated and each agency's areas of 
expertise.
    In response to a white paper the committee circulated at 
the end of last year asking these very questions, we heard from 
a number of labs and pathologists that FDA should only have a 
limited role, if any, in regulating a select set of tests as 
medical devices. The rest, in their opinion, should be overseen 
by CMS, through an updated Clinical Laboratory Improvement 
Amendments program. This is despite the fact that CMS has 
stated that they do not have the resources, the expertise or 
the willingness to take on what is being asked of them. I am 
eager to hear what Dr. Conway has to say on the matter.
    We also received comments from a number of manufacturers, 
as well as over forty patient groups, that FDA-not CMS-needs to 
be in the driver's seat and that tests that have the same 
impact on a patient should be held to the same standards, 
regardless of who does the development. This is despite the 
fact that laboratories are uniquely nimble environments where 
pathologists continually modify and improve tests in ways that 
manufacturers cannot.
    I am well aware that this has been, at times, a heated 
debate with passionate advocates on both sides. With such a 
backdrop, I want to particularly commend the manufacturers, 
laboratories, and other healthcare institutions that have been 
willing to roll up their sleeves and find as much common ground 
as possible through constructive dialogue, a willingness to 
compromise, and a pragmatic understanding of what a viable, 
modern framework entails.
    I do not believe imposing a new regulatory reality on an 
increasingly important component of our healthcare system via 
guidance is the best way to address these issues. These 
products warrant a regulatory system designed with them in 
mind. They should not be shoehorned into a system that was 
drafted in the 1970s.
    This committee has clearly shown that we are willing and 
able to move complicated, comprehensive, bipartisan 
legislation. The discussion draft the committee circulated 
along with the hearing notice is of course not perfect, but it 
is a serious document based on significant consensus. I would 
ask that all of the stakeholders out there, including our two 
distinguished witnesses, help us improve it as this process 
continues.
    With that I would like to thank Dr. Shuren-a frequent and 
always welcome visitor-as well as Dr. Conway, for their 
willingness to testify today. I look forward to working with 
them on these issues going forward.

    Mr. Pitts. And I now recognize the ranking member, Mr. 
Green, 5 minutes for his opening statement.

   OPENING STATEMENT OF HON. GENE GREEN, A REPRESENTATIVE IN 
                CONGRESS FROM THE STATE OF TEXAS

    Mr. Green. Thank you, Mr. Chairman, and thank you for 
calling this hearing today, and I want to welcome our witnesses 
from the FDA and the CMS.
    The role of diagnostic tests in our healthcare system has 
changed dramatically since Congress passed the medical device 
amendments in 1976 and added in vitro diagnostics to the device 
definition. It has been almost 4 decades, and the evolution of 
modern medicine and the advancement of science has surpassed 
what everyone could imagine at the time. The enthusiasm around 
precision medicine is high, and the potential of diagnostics to 
further transform the treatment of disease is limitless.
    When the FDA first began regulating medical devices, 
applicable regulatory requirements for lab-developed tests, or 
LDTs, were not enforced because they were relatively simple 
tests, generally combined the local labs, and frequently used 
for rare conditions.
    Today, LDTs have increased in complexity and availability. 
They are often used to diagnose serious medical conditions, and 
many have major impact on patient care. Not only have LDTs 
become sophisticated, the role that these tests play in 
delivery of health care has expanded.
     The Centers for Disease Control and Prevention estimated 
that approximately 6.8 billion laboratory tests are 
administered each year. An analysis found that results from the 
clinical laboratory tests influence about 70 percent of 
healthcare decisions.
    The clinical laboratory amendments of 1988 created minimum 
standards of quality for all clinical labs in the country. The 
Centers for Medicare and Medicaid Services, CMS, has 
jurisdiction over the program, and CLIA has successfully 
improved the quality of the clinical labs in accuracy of 
testing for nearly 25 years.
    However, under CLIA CMS does not confirm the clinical 
validation of LDTs, meaning that they do not look as to whether 
it is a particular test accurately that identifies, measures, 
or predicts the absence or the presence of a clinical 
condition. These known gaps in oversight have been a source of 
concern to this committee and to the healthcare community at 
large.
    Yesterday, the Food and Drug Administration released a 
report that included 20 case studies of problematic tests from 
labs that were following the minimum requirements of CLIA but 
proposed real risk to patients. In an area of so much promise 
and significance to patient care, the accuracy, reliability, 
and clinical meaningfulness of all diagnostic tests, regardless 
of where they are created, must be a top priority for 
healthcare providers, test developers, regulators, and 
lawmakers.
    Last year, the FDA issued a draft regulatory framework to 
phase in enforcement regulatory requirements, including 
premarket review, adverse event reporting for LDTs that pose 
greater risk to patients if their results are not accurate and 
reliable. And I appreciate FDA's efforts to ensure that tests 
are supported by rigorous evidence and that patients and 
healthcare providers can have confidence in their results.
    That said, I share the opinion of my colleagues that 
legislation is both appropriate and necessary to modernize 
clinical laboratory diagnostic oversight. The legislative 
solution is surely the surest way to establish a framework that 
will be embraced by stakeholders, avoid litigation, extended 
uncertainty, and foster innovation of new clinical diagnostic 
tests.
    The FDA's approach to this draft guidance let to a number 
of important questions, but the guidance documents also spurred 
a larger conversation about the overarching need to modernize 
oversight of these unique and increasingly important tests.
    During the 21st Century Cures initiative, as part of the 
broad effort to close the gap between science of cures and how 
we regulate medical products, the committee hosted a roundtable 
on precision medicine and advances in diagnostic testing. The 
committee also released a white paper on diagnostic test 
regulation and received outpouring of feedback from 
stakeholders.
    While all parties did not agree on all the principles, much 
less specifics, it was abundantly clear that any regulatory 
framework for diagnostic tests must prioritize patient benefit 
and allow for continued innovation and investment through 
regulatory certainty and appropriate regulatory controls.
    There is urgent need to establish clear and logical lines 
separating the practice of medicine and the actual conduct of 
the diagnostic tests and the development and manufacturing of 
diagnostic tests so that the promise of 21st century medicine 
can be fully realized.
    Today, we will hear from FDA and CMS about each agency's 
respective role in the oversight and regulation of clinical 
laboratory tests. Members of the committee will have questions 
about the appropriate role of each agency and any updated 
framework, and how Congress can best promote robust investment 
and innovation while protecting patient safety.
    Mr. Chairman, I look forward to hearing from our witnesses, 
and I yield back.
    [The prepared statement of Mr. Green follows:]

                 Prepared statement of Hon. Gene Green

    Good morning, and thank you all for being here today.
    The role of diagnostic tests in our healthcare system has 
changed dramatically since Congress passed the Medical Device 
Amendments in 1976 and added in-vitro diagnostics to the device 
definition.
    It has been almost four decades, and the evolution of 
modern medicine and advancement of science has surpassed what 
anyone could have imagined at that time.
    The enthusiasm around precision medicine is high, and the 
potential of diagnostics to further transform the treatment of 
disease is limitless.
    When FDA first began regulating medical devices, applicable 
regulatory requirements for lab-developed tests or ``LDTs'' 
were not enforced because they were relatively simple tests, 
generally confined to local labs, and frequently used for rare 
conditions.
    Today, LDTs have increased in complexity and availability. 
They are often used to diagnose serious medical conditions, and 
many have a major impact on patient care.
    Not only have LDTs become more sophisticated, the role 
these tests play in the delivery of health care has expanded.
    The Centers for Disease Control and Prevention estimated 
that approximately 6.8 billion laboratory tests are 
administered each year.
    Another analysis found that results from clinical 
laboratory tests influence around 70 percent of healthcare 
decisions.
    The Clinical Laboratory Amendments of 1988 created minimum 
standards of quality for all clinical labs in the country.
    The Centers for Medicare and Medicaid Services (CMS) has 
jurisdiction over the program, and CLIA has successfully 
improved the quality of clinical labs and accuracy of testing 
for nearly 25 years.
    However, under CLIA, CMS does not confirm the clinical 
validity of LDTs, meaning they do not look at whether a 
particular test accurately identifies, measures, or predicts 
the absence or presence of a clinical condition.
    These known gaps in oversight have been a source of concern 
to this committee, and to the healthcare community at large.
    Yesterday, the Food and Drug Administration (FDA) released 
a report that included 20 case studies of problematic tests 
from labs that were following the minimum requirements of CLIA, 
but posed real risk to patients.
    In an of area so such promise and significance to patient 
care, the accuracy, reliability and clinical meaningfulness of 
all diagnostic tests--regardless of where they are created--
must be a top priority of healthcare providers, test 
developers, regulators, and lawmakers.
    Last year, the FDA issued a draft regulatory framework to 
phase in enforcement of regulatory requirements, including 
premarket review and adverse event reporting, for LDTs that 
pose greater risk to patients if their results are not accurate 
and reliable.
    I appreciate the FDA's efforts to ensure that tests are 
supported by rigorous evidence, and that patients and 
healthcare providers can have confidence in their results.
    That said, I share the opinion of my colleagues that 
legislation is both appropriate and necessary to modernize 
clinical laboratory diagnostics oversight.
    A legislative solution is the surest way to establish a 
framework that will be embraced by stakeholders, avoid 
litigation and extended uncertainty, and foster innovation of 
new clinical diagnostic tests.
    The FDA's approach in its draft guidance led to a number of 
important questions, but the guidance documents also spurred a 
larger conversation about the overarching need to modernize 
oversight of these unique and increasingly important tests.
    During the 21st Century Cures Initiative, as part of the 
broad effort to close the gap between the science of cures and 
how we regulate medical products, the committee hosted a 
roundtable on precision medicine and advances in diagnostic 
testing.
    The committee also released a white paper on diagnostic 
test regulation, and received an outpouring of feedback from 
stakeholders.
    While all parties did not agree on all principles, much 
less specifics, it was abundantly clear that any regulatory 
framework for diagnostic tests must prioritize patient benefit, 
and allow for continued innovation and investment through 
regulatory certainty and appropriate regulatory controls.
    There is an urgent need to establish clear and logical 
lines separating the practice of medicine, the actual 
conducting of a diagnostic test and the development and 
manufacturing of such tests, so that the promise of 21st 
century medicine can be fully realized.
    Today, we will hear from FDA and CMS about each agency's 
respective role in the oversight and regulation of clinical 
laboratory testing.
    Members of the committee will have questions about the 
appropriate role of each agency in any updated framework, and 
how Congress can best promote robust investment and innovation, 
while protecting patient safety.
    I look forward to hearing from our witnesses and I yield 
back the balance of my time.

    Mr. Pitts. The Chair thanks the gentleman, now recognizes 
Dr. Burgess in lieu of Chairman Upton, 5 minutes.


OPENING STATEMENT OF HON. MICHAEL C. BURGESS, A REPRESENTATIVE 
              IN CONGRESS FROM THE STATE OF TEXAS

    Mr. Burgess. Thank you, Mr. Chairman.
    I appreciate the opportunity that we have before us with 
this hearing, but I do want to say at the outset, with 
everything else that is going on, this may be one of the most 
important and at the same time the most frightening concepts 
that is before the Congress right now. We are talking about a 
proposal that may not just stifle but eliminate medical 
innovation, something which this country has excelled for 
decades, and we are also opening the door for the first Federal 
regulation of the practice of medicine, not the needles and IV 
solutions, the actual diagnostic thought processes that go in 
to practicing medicine.
    Let me just say at the outset I do strongly believe in the 
potential of genomic medicine. I understand how important it is 
to really understand illness at a molecular level, quickly 
diagnose it, and get the treatment that is appropriate for the 
patient with a minimal amount of side effects.
    A year and a half ago, when the President talked about 
precision medicine during his State of the Union Address, I 
thought that was a very positive development. There are not 
many places where the White House and I agree on anything, but 
here was some common ground, and I took it to heart.
    Laboratory testing produces the informational building 
blocks that are at the heart of precision medicine. As former 
Administrator Mark McClellan at CMS said, we have got to get 
the right treatment at the right time to the right patient.
    We are not talking about test kits that are put in a box 
and shipped across State lines but medical procedures that are 
carried out by highly trained and qualified health 
professionals engaged in the practice of medicine.
    As we discuss the oversight of laboratory-developed tests, 
it is crucial that we do not slow innovation or create 
unnecessary regulatory hurdles. We have got to ask ourselves 
first, what is the problem that we are trying to solve, and is 
our response appropriate, and are there unintended consequences 
that could result?
    Requiring premarket review by the FDA will impose new and 
arguably unnecessary requirements and costs on clinical 
laboratories, hospitals, and doctors. Although an additional 
review of certain tests may be warranted, I actually have a 
greater confidence in a CLIA-centric approach, but there are 
others--and certainly people on this committee--who suggested a 
different track. But it remains unclear to me how we can 
separate the practice of medicine from these laboratory 
processes, and if we cannot, are we effectively opening the 
door to the Federal regulation of the practice of medicine? I 
reject that notion and believe by segmenting this process out 
has to be the fundamental first step of any proposal.
    Let me just reiterate I do want to be involved in this 
discussion. There is no question in my mind that CLIA can be 
improved. I was not a fan when CLIA came to my medical practice 
in 1988. I was not a fan of having to become a CLIA-certified 
location. I was not a fan of having to apply for a CLIA waiver. 
But since that time, I think arguably you can make the case 
that CLIA has been a useful enterprise.
    Look, we want doctors and patients to benefit from 
clinically valid tests, and the current FDA proposal, as such, 
creates regulatory uncertainty that will not be a catalyst for 
innovation.
    We talk a lot about the Administrative Procedures Act, we 
talk a lot about notice of proposed rulemaking. This is not 
coming through the normal regulatory process. It is coming as a 
guidance. My understanding is to be issued at the end of this 
year, and like it or not, there you have it.
    But, you know, it is hard. On this committee I still retain 
that romantic notion that our Government exists with the 
consent of the governed. In my mind that would not include 
issuing guidances, fiats that are expected to be followed, but 
rather, you go through the normal administrative procedures, 
hear people out, and make the best decision based on the 
information.
    Thank you, Mr. Chairman. I yield back the balance of my 
time.
    Mr. Pitts. The Chair thanks the gentleman.
    I now recognize the ranking member of the full committee, 
Mr. Pallone, 5 minutes for an opening statement.

OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE 
            IN CONGRESS FROM THE STATE OF NEW JERSEY

    Mr. Pallone. Thank you, Mr. Chairman. I want to also thank 
Dr. Shuren and Dr. Conway for being here today to discuss the 
regulation of lab-developed tests.
    There has been a lot of discussion over how to 
appropriately oversee lab-developed tests, and it is important 
that, as the committee considers this issue, we have a better 
understanding of the strengths and limitations of both FDA and 
CMS's authority in this area.
    Congress gave FDA authority over lab-developed tests under 
the Medical Device Amendments in 1976, and at that time, most 
LDTs were relatively simple tests used more often for rare 
conditions. Since then, advances in technology and medicine 
have resulted in LDTs that are increasingly more complex, more 
readily available to physicians and patients, and used to 
diagnose and treat a wider range of diseases, including breast 
cancer and heart disease. LDTs are also increasingly used to 
provide personalized treatment such as through genetic tests 
that help physicians to detect the risk of certain diseases 
earlier or to choose more targeted therapies.
    Unfortunately, many of these tests have not been reviewed 
or cleared by FDA prior to coming to the market to confirm that 
these tests are accurate, reliable, or provide clinically 
accurate results. This can result in patients going undiagnosed 
with certain medical conditions or undergoing treatment that is 
not medically necessary.
    For example, tests have been developed to identify certain 
gene sequences that can help determine appropriate treatment 
for ovarian cancer. I am sure many Members here are familiar 
with the example of OvaSure, which claimed to detect early-
stage ovarian cancer in high-risk women. This test, though, was 
not properly validated and was found to provide high numbers of 
false positive and false negative results, and this means many 
women who received a false positive result may have undergone 
unnecessary surgery to remove healthy ovaries, or some women 
may have gone undiagnosed after receiving a false-negative 
result.
    Patients deserve to know that the test results they are 
relying on to diagnose or treat a condition is accurate, a 
comfort that they do not always have today. And as we have 
heard from many organizations, patients and their physicians 
should be able to trust the results of their tests, regardless 
of how or where a test is developed or performed. It does not 
make sense to regulate tests differently based on who develops 
them.
    I also believe that we can provide patients and providers 
with this certainty without endangering or inhibiting the 
medical innovation that is occurring today. Scientific progress 
has been made to help facilitate the development and use of 
personalized medicine, which you all agree is the future of 
medicine, but this development can only be successful if we 
know that these complex, sophisticated tests are clinically 
valid.
    So I am glad that today we will have the opportunity to 
better understand FDA and CMS's authority in this area and hear 
their perspective on what regulatory changes, if any, are 
needed to address the future development of lab-developed 
tests. And I hope moving forward that both agencies will work 
with the committee on the discussion draft circulated today to 
ensure that any legislation that moves forward will ensure that 
LDTs are accurate, reliable, and safe for patients.
    I yield back.
    [The prepared statement of Mr. Pallone follows:]

             Prepared statement of Hon. Frank Pallone, Jr.

    Mr. Chairman, thank you for holding today's hearing. I also 
want to thank both Dr. Shuren and Dr. Conway for being here 
today to discuss the regulation of lab developed tests.
    There has been much discussion over how to appropriately 
oversee lab developed tests and it is important that as the 
Committee considers this issue, we have a better understanding 
of the strengths and limitations of both FDA and CMS's 
authority in this area.
    Congress gave FDA authority over lab-developed tests under 
the Medical Device Amendments in 1976. At that time, most LDTs 
were relatively simple tests used most often for rare 
conditions. Since then advances in technology and medicine have 
resulted in LDTs that are increasingly more complex, more 
readily available to physicians and patients, and used to 
diagnose and treat a wider range of diseases, including breast 
cancer and heart disease. LDTs are also increasingly used to 
provide personalized treatments, such as through genetic tests 
that help physicians to detect the risk of certain diseases 
earlier or to choose more targeted therapies.
    Unfortunately, many of these tests have not been reviewed 
or cleared by FDA prior to coming to the market to confirm that 
these tests are accurate, reliable, or provide clinically 
accurate results. This can result in patients going undiagnosed 
with certain medical conditions, or undergoing treatment that 
is not medically necessary.
    For example, tests have been developed to identify certain 
gene sequences that can help determine appropriate treatment 
for ovarian cancer. I am sure many Members here are familiar 
with the example of OvaSure, which claimed to detect early 
stage ovarian cancer in high-risk women. This test though was 
not properly validated and was found to provide high numbers of 
false-positive and false-negative results. This means many 
women who received a false positive result may have undergone 
unnecessary surgery to remove healthy ovaries, or some women 
may have gone undiagnosed after receiving a false negative 
result.
    Patients deserve to know that the test results they are 
relying on to diagnose or treat a condition is accurate, a 
comfort that they do not always have today. As we have heard 
from many organizations, patients and their physicians should 
be able to trust the results of their tests regardless of how 
or where a test is developed or performed. It does not make 
sense to regulate tests differently based on who develops them.
    I also believe that we can provide patients and providers 
with this certainty without endangering or inhibiting the 
medical innovation that is occurring today. Scientific progress 
has been made to help facilitate the development and use of 
personalized medicine, which we all agree is the future of 
medicine. But this development can only be successful if we 
know that these complex, sophisticated tests are clinically 
valid.
    I am glad that today we will have the opportunity to better 
understand FDA and CMS's authority in this area and hear their 
perspective on what regulatory changes, if any, are needed to 
address the future development of lab-developed tests. I hope 
moving forward that both agencies will work with the Committee 
on the discussion draft circulated today to ensure that any 
legislation that moves forward will ensure that LDTs are 
accurate, reliable, and safe for patient use.
    I yield back.

    Mr. Pitts. The Chair thanks the gentleman.
    As usual, all the written opening statements of the Members 
will be made part of the record.
    That concludes the opening statements.
    I would like to submit under U.C. request the following 
documents for the record: a November 16 letter from a number of 
organizations and laboratory directors, and a November 11 
letter from organizations representing patients, advocates, 
caregivers, and healthcare professionals.
    Without objection, so ordered.
    [The information appears at the conclusion of the hearing.]
    Mr. Pitts. On our panel today we have two witnesses, and I 
welcome them, thank them for coming. First, Dr. Jeffrey Shuren, 
Director, Centers for Devices and Radiological Health, Food and 
Drug Administration, Department of Health and Human Services; 
and Dr. Patrick Conway, Deputy Administrator for Innovation and 
Quality, and Chief Medical Officer, Office of the 
Administrator, Centers for Medicare and Medicaid Services, 
Department of Health and Human Services.
    Thank you for coming. Your written testimony will be made 
part of the record. You will each be given 5 minutes to 
summarize.
    Dr. Shuren, you are recognized for 5 minutes for a summary.

   STATEMENTS OF JEFFREY SHUREN, M.D., DIRECTOR, CENTER FOR 
DEVICES AND RADIOLOGICAL HEALTH, FOOD AND DRUG ADMINISTRATION, 
 DEPARTMENT OF HEALTH AND HUMAN SERVICES, AND PATRICK CONWAY, 
      M.D, ACTING PRINCIPAL DEPUTY ADMINISTRATOR, DEPUTY 
  ADMINISTRATOR FOR INNOVATION AND QUALITY, AND CHIEF MEDICAL 
OFFICER, CENTERS FOR MEDICARE AND MEDICAID SERVICES, DEPARTMENT 
                  OF HEALTH AND HUMAN SERVICES

                  STATEMENT OF JEFFREY SHUREN

    Dr. Shuren. Well, thank you, Chairman Pitts, Ranking Member 
Green, members of the subcommittee. Thank you for the 
opportunity to testify today.
    We are excited about scientific developments in genomics 
and molecular biology that are leading to advances in health 
care, particularly in precision medicine. Getting the right 
treatment to the right patient at the right time, though, 
depends upon having accurate, reliable, and clinically valid 
tests. If not, we give the wrong treatment or we give no 
treatment, and patients get hurt.
    FDA has been regulating in vitro diagnostics for almost 4 
decades, and when such a test is made by a laboratory, we call 
it a laboratory-developed test, or LDT. And the law doesn't 
distinguish on who makes it. We regulate the test regardless of 
who makes that test. And we ensure that those tests are 
analytically and clinically valid.
    Now, when we first started regulating IVDs, as a matter of 
policy, we decided not to actively enforce existing 
requirements on LDTs because at the time they were generally 
simple, low-risk tests used on uncommon conditions in often a 
local setting, typically in a hospital for patients in that 
hospital. But over time they have come increasingly more 
complex, higher risk, they are used on common conditions like 
heart disease, and they may be offered on a national basis. In 
addition, we have been coming across increasing examples of 
problematic LDTs. We put out examples of 20 of them just 
yesterday, and there are others.
    As a result of this, the problems we have seen and the 
increasing complexity, there have been calls on the FDA to 
actively enforce existing requirements that started in the 
1990s, NIH and the Department of Energy. In the 2000s two 
advisory committees to the Secretary of Health and Human 
Services called on us to regulate. The Institute of Medicine 
has asked us to regulate.
    So in 2007 we put out draft policy to begin to actively 
regulate a subset of LDTs, and what the lab community said is 
don't pick off tests one by one. Please put in place an 
overarching framework. So in 2010 we had a public meeting to 
get input, and we were told put in place a risk-based phased-in 
approach.
    And then in response in October of last year we did just 
that. We put out draft policy to now put in place that 
framework. And what we heard from the lab community then, oh, 
no, there are no problems with LDTs. We don't need FDA 
oversight of anything, maybe a little beefing up on CLIA, but 
that is it.
    And now, just a few months ago, we started to see several 
proposals come out from the lab community that now, for the 
first time, acknowledge that LDTs must demonstrate that they 
are analytically valid and clinically valid, that they should 
be subject to premarket review, at least moderate- and high-
risk tests, the some modifications need to be subject to 
premarket review, that certain problems need to be reported to 
the Government, and they need to be under a risk-based approach 
with a three-tier risk classification system. None of those are 
currently enforced on them today. They all exist under an FDA 
framework.
    But what most of these proposals except one would do is it 
would create a duplicative program under CMS and a bifurcated 
system, leading to more inefficiencies, higher costs, and still 
putting patients unnecessarily at risk. For example, you can 
have a conventional manufacturer who makes an IVD we regulated. 
Now, a laboratory makes a big enough change to it, which 
laboratories do, and it is regulated by CLIA. Then the original 
manufacturer makes a change to that test and it bounces back to 
the FDA. So we will be stuck in a game of regulatory ping-pong, 
and the real loser here is patients.
    Doctors and patients don't care about who makes a test. 
They do care that their tests are accurate, reliable, and 
clinically valid.
    Now, some labs have already been working with us, and we 
congratulate them for crossing that picket line. But our 
message in our invitation to the rest of the lab community is 
to put down the swords, that for the sake of our patients it is 
time to end the saber-rattling and instead partner with us 
moving forward.
    Thank you.
    [The prepared statement of Dr. Shuren follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT] 
    
    Mr. Pitts. The Chair thanks the gentleman, now recognizes 
Dr. Conway, 5 minutes for his summary.

                  STATEMENT OF PATRICK CONWAY

    Dr. Conway. Thank you, Chairman Pitts, Ranking Member 
Green, and members of the committee. Thank you for the 
opportunity to talk about our work at the Centers for Medicare 
and Medicaid Services related to ensuring accurate and reliable 
laboratory testing.
    The Clinical Laboratory Improvement Amendments of 1988, 
commonly referred to as CLIA, of which CMS has primary 
jurisdiction, created minimum standards of quality for all 
clinical laboratories in the United States. CLIA successfully 
worked for approximately 25 years and has contributed to major 
improvements in the quality of clinical laboratories, promoted 
accurate testing, and improved patient safety.
    As of July of 2015 there were roughly 250,000 laboratories 
that have registered with CMS and held CLIA certificates. CLIA 
responsibilities are divided between three agencies: CMS; the 
Centers for Disease Control, or CDC; and the Food and Drug 
Administration. CMS conducts laboratory inspections to make 
sure that laboratories have appropriate controls, expertise, 
training, and procedures to ensure that tests are accurate and 
reliable. CMS also approves accreditation organizations and 
manages the laboratory certification process. CDC conducts 
laboratory quality improvement studies that guide policy 
determination and development of laboratory practice 
guidelines.
    FDA's primary responsibility under CLIA is to classify 
clinical tests into one of three categories--waived, moderate 
complexity, and high complexity--based on their level of 
complexity and risk to patients. FDA also has a critical role 
in determining clinical validity of tests and premarket 
evaluation. Standards that laboratories must meet under CLIA 
are based on the complexity of the tests they perform. 
Laboratories that perform more complex must meet higher 
standards.
    Laboratories that perform moderate- and high-complexity 
tests must meet requirements on quality assessment, quality 
control, personnel qualifications and education, general 
laboratory systems, and proficiency testing, among others. 
Laboratories that only perform waived tests, simpler tests that 
pose a low risk to patients, are exempt from most CLIA 
requirements. In addition, laboratories performing the same 
tests must meet the same standards, whether located in a 
hospital, doctor's office, or other site.
    This framework is designed to reduce the risk of potential 
harm and ensure patients receive the same high-quality clinical 
laboratory testing no matter where the test is performed.
    CLIA's provisions apply to all laboratories in the U.S., 
not just those that receive Medicare payment in order to ensure 
uniform quality across all laboratories.
    CMS enforces CLIA standards by requiring laboratories to 
obtain certificates in order to operate. CMS conducts onsite 
surveys prior to issuing a certificate to a lab that performs 
high- or moderately complex tests. Labs are resurveyed every 2 
years, and the surveys also assist laboratories in improving 
patient care through education.
    Laboratories may also receive CLIA certification by 
obtaining accreditation from one of the seven private nonprofit 
accreditation organizations approved by CMS. To receive CMS 
approval, the accreditation organization requirements must meet 
or exceed CLIA's requirements.
    Moving forward, we believe CLIA and our implementing 
regulations create the necessary framework to effectively 
oversee laboratories day-to-day operations and into the future, 
including those operations that pertain to the use of 
laboratory-developed tests and other high-complexity tests. We 
have several principles that have helped guide our work in 
CLIA, which may also be useful when informing future efforts of 
this committee.
    First, we aim to prevent duplicative oversight efforts 
across agencies. CLIA requires coordination across CMS, FDA, 
and CDC. We have worked to ensure our oversight efforts are 
consistent and complementary and not duplicative. In doing so, 
we have ensured that we take advantage of the unique expertise 
of each agency and its staff.
    Second, we focus on our agency's oversight strengths. When 
CLIA was implemented in the early 1990s, the responsibility to 
conduct certifications of laboratories was a natural fit for 
CMS because of our survey and certification experience. On the 
other hand, CMS does not have scientific staff capable of 
reviewing complex medical and scientific literature in 
determining clinical validity. This expertise resides within 
the FDA, which assesses clinical validity in the context of 
premarket reviews and other activities aligned with their 
regulatory efforts under the Food, Drug, and Cosmetic Act.
    Third, we value our relationship with our private 
accreditor organizations and State-based partners. These 
organizations play an important role in evaluating and 
certifying laboratories.
    Fourth, we take targeted, risk-based approaches to 
oversight to improve patient safety without creating burdensome 
administrative requirements. We believe the current approach in 
which laboratories must meet higher standards if they are to 
perform more complex tests has paid dividends in improving the 
quality of the testing process.
    Finally, as a practicing physician who works clinically on 
weekends, I know the importance of tests being assessed for 
clinical validity, as well as the need for assessment for 
laboratory standards. FDA and CMS can work together utilizing 
their respective authorities and strengths to assess premarket 
clinical validity and laboratory standards respectively.
    Thank you again for the opportunity to discuss CMS's work 
related to ensuring accurate and reliable laboratory testing. I 
look forward to your questions. Thank you.
    [The prepared statement of Dr. Conway follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT] 
    
    Mr. Pitts. The Chair thanks the gentleman, both witnesses 
for your opening statements. I will begin the questioning. I 
will recognize myself 5 minutes for that purpose.
    The discussion draft, which the committee circulated before 
the hearing, divides FDA and CMS responsibilities based upon 
the type of activity being conducted by the regulated entity. 
FDA would regulate test development activities in a risk-based 
manner, and CMS would regulate lab operations.
    Unlike the discussion draft, some alternative proposals 
being floated would divide regulatory oversight between CMS and 
FDA depending on the type of test.
    I would like each of you to respond. Dr. Shuren, would you 
comment on the implications of an approach that would divide 
oversight between CMS and FDA based on the type of test, as 
opposed to the type of activity?
    Dr. Shuren. So such a system is going to lead to 
inefficiencies. It is going to lead to inconsistent standards, 
treating the same kind of test differently depending upon who 
makes the test. And as a result, you can go to one institution, 
get a test, and it is regulated by FDA. You can get the same 
kind of test across the street and it is regulated by CMS. And 
the people who are put at risk, it is patients.
    If we are going to assure that tests work, we need one 
unified system that we are applying consistent standards and we 
are assuring that those tests are accurate, reliable, and 
clinically valid.
    Mr. Pitts. Dr. Conway, would you comment on that, the 
implications of an approach that divides oversight between CMS 
and FDA based on the type of test, as opposed to the type of 
activity?
    Dr. Conway. Yes, I agree with Dr. Shuren. The concern here 
is we want to reduce and avoid duplication and ensure 
coordination across agencies. You know, from a CLIA construct 
we really are focused on post-market review, laboratory by 
laboratory, and we are really focused on the things such as the 
protocols in place in the laboratory, the equipment and 
equipment maintenance, the training of staff and personnel. So 
CLIA's focus really is on that laboratory-by-laboratory 
assessment of quality standards.
    Mr. Pitts. And expand a little bit more on whether such an 
approach would create administrative duplication or any 
inconsistencies, Dr. Shuren?
    Dr. Shuren. That is correct. It will create inefficiencies 
and higher costs because essentially we have duplicative 
systems in FDA and CMS, and the real distinction is just simply 
who makes the test, which doesn't make sense. And we will have 
inconsistent standards. We can try to coordinate between 
ourselves, but quite frankly, that becomes much more 
challenging as tests also begin to bounce between FDA oversight 
and CMS oversight.
    Mr. Pitts. OK. Dr. Conway, you stated in your testimony 
that ``CMS does not have a scientific staff capable of 
determining whether a test is difficult to successfully carry 
out or likely to prove detrimental to a patient if carried out 
improperly. This expertise resides within the FDA.'' From your 
perspective at CMS, what would be the impact on patients if FDA 
were precluded from reviewing the clinical validity of most 
LDTs?
    Dr. Conway. Yes. So as Dr. Shuren mentioned, I think the 
challenge is if FDA is not reviewing the test in a premarket 
manner for clinical validity, then our surveyors in CLIA are 
not assessing clinical validity. They are assessing laboratory 
practices and the protocols and standards in those 
laboratories. So as a practicing physician, it is critical, as 
Dr. Shuren said, that we know that a test is clinically valid, 
meaning it is truly detecting the presence or absence of 
disease. Therefore, the premarket review by FDA is important.
    Mr. Pitts. Now, some stakeholders have said that CMS should 
be tasked with reviewing tests for clinical validity. What are 
your thoughts on that approach?
    Dr. Conway. So our survey staff are not trained to assess 
clinical validity, and then let me build on that. Our survey 
staff are trained in laboratory protocols, equipment, standards 
around those protocols, whereas--and Dr. Shuren can certainly 
speak directly to FDA's staff--is, you know, physicians, 
Ph.D.'s, biostatisticians who are trained in assessing the 
scientific literature in its entirety and assessing clinical 
validity.
    Mr. Pitts. Now, some stakeholders have suggested that CMS 
should regulate tests developed by labs. FDA should regulate 
tests developed by manufacturers. Some have proposed carving 
out a role for FDA only when a test developer chooses not to 
publicize their methodologies. Shouldn't the test's impact on 
the patient, regardless of who developed it, be the primary 
factor in developing a regulatory framework? Dr. Shuren and 
then Dr. Conway.
    Dr. Shuren. Well, we agree that this should be a risk-based 
framework. We also think that you should have one agency that 
is reviewing those tests to assure that they are accurate, 
reliable, and clinically valid. That assures consistency.
    But also, one of the things we have found is when someone 
makes a test, another lab or another entity makes a similar 
test, we learn from that, and we sometimes identify problems or 
common problems and we are able to feed that back to test 
developers. If you split it between two agencies, we are going 
to lose all that learning that ultimately benefits innovation 
and benefits patients.
    Mr. Pitts. Dr. Conway, do you want to comment?
    Dr. Conway. So I agree with Dr. Shuren. I believe one 
agency doing the premarket review, as Dr. Shuren said, and that 
agency being FDA, makes sense given the training and expertise. 
We also, as you have heard, have a principle of coordination 
and using each agency's expertise. CMS's focus and expertise is 
in the area of laboratory assessment, laboratory by laboratory, 
on protocols, equipment, et cetera.
    Mr. Pitts. My time is expired. The Chair recognizes the 
ranking member, Mr. Green, 5 minutes for questions.
    Mr. Green. Thank you, Mr. Chairman.
    I would like to ask unanimous consent to submit a letter 
from the American Cancer Society Cancer Action Network for the 
record.
    Mr. Pitts. Without objection, so ordered.
    [The information appears at the conclusion of the hearing.]
    Mr. Green. Dr. Shuren, with other medical devices, FDA has 
proposed regulating lab-developed tests based on the risk of 
the test to the patient and the public. Under proposed FDA 
would classify LDTs into three risk classes: low, moderate, and 
high. Can you explain how FDA proposes to finding the low, 
moderate, and high risk in the agency's framework describing 
how premarket or post-market requirements would vary among 
these risk classes?
    Dr. Shuren. So we look at risk based upon what the risk is 
to patients if that test provides a false result, an incorrect 
result. And we estimate that for low-risk tests we do not 
conduct premarket review because they are so low risk. We think 
about 50 percent of the tests out there--that has been our 
experience--are low risk. And then we conduct premarket review 
for high-risk and moderate-risk tests. High-risk tests are only 
about 1 to 2 percent of the tests out there, moderate risk 
about 48 percent.
    And the data needed to demonstrate analytical and clinical 
validity differs depending upon the risk of the disease. There 
is less burden involved when it is a less-riskier test rather 
that we are reviewing, and that is the risk-based approach that 
we apply.
    Mr. Green. FDA's approval standard for drugs and medical 
devices safe and effective, can you just please discuss the 
approval standard FDA has proposed using for the regulation of 
lab-developed tests?
    Dr. Shuren. So we would apply the same standard we would 
apply to in vitro diagnostic tests that are not made by a lab, 
that they are analytically valid, they are clinically valid, 
and they are safe to use under their conditions to use. That 
means they are accurate on what they measure, they are 
reliable, and they will identify they in fact do identify a 
disease.
    Mr. Green. Companion diagnostics is an area of great 
interest and enthusiasm. Can you talk about how the FDA views 
this category of tests, in particular the level of risk posed 
to patients and how they would be treated under the proposed 
guidance?
    Dr. Shuren. So companion diagnostics are increasingly 
playing a bigger role in health care. Essentially, companion 
diagnostic is a test where the safety and effectiveness of the 
therapeutic depends upon the diagnostic because the diagnostic 
informs whether or not that patient should receive a particular 
treatment. And that is why it is critically important that 
those tests truly work, because if not, then patients are not 
getting the right treatment or they may be getting no treatment 
at all.
    For example, we had a test for providing treatment for 
women with breast cancer and found that LDTs in the past were 
producing as much as 20 percent of them incorrect or inaccurate 
results. That means that women who should have gotten treated 
with the right treatment were not. And that is preventable.
    Mr. Green. OK. Thank you. Dr. Conway, I want to thank you 
also for participating. As you are aware, following the release 
of the FDA's guidance on enforcing requirements for lab-
developed tests, a number of stakeholders called for 
enhancement of CLIA as a more appropriate way to regulate the 
tests. And I appreciate your testimony on outlining the 
difference between FDA and CMS authority over the tests.
    One of the key differences is the fact that under CLIA CMS 
does not review a test for the clinical validity, that is, 
accuracy on which the test identifies measures or predicts the 
presence and absence of a clinical condition or predisposition 
to a patient. Rather, CMS reviews look at analytical validity. 
You noted that the experience and expertise in assessed 
clinical validity resides instead with the FDA.
    Despite CMS stating on more than one occasion that the 
agency does not have the experience or the scientific expertise 
to assess clinical validity in premarket review, many 
stakeholders continue to advocate for additional authority in 
that area for CMS. Can you please discuss further CMS 
capabilities in implementing regulations for overseeing LDTs, 
and can you also please comment on whether CMS would have the 
capability of conducting any type of premarket review or 
regulatory review of LDTs?
    Dr. Conway. Yes. So our framework that we believe is 
working well now is CLIA is focused on assessment of the 
protocols, the standards, the equipment, the training, and the 
personnel. Even in analytic validity, we are simply looking at, 
you know, does the lab test detect the analyte described? That 
is very different than clinical validity, which is assessing 
whether, you know, the test reliably and accurately detects the 
presence or absence of disease, as Dr. Shuren said.
    You know, the majority of our staff are--you know, we have 
got approximately 25 people in the central office running CLIA, 
a little over 100 surveyors across the States, all of the 
States. They are generally medical technologists, former 
laboratory personnel trained to assess laboratory by 
laboratory. They are not trained to assess premarket scientific 
literature and determine clinical validity.
    Mr. Green. OK. Thank you, Mr. Chairman.
    Mr. Pitts. The Chair thanks the gentleman and now 
recognizes the vice chair of the full committee, Mrs. 
Blackburn, 5 minutes for an opening statement.
    Mrs. Blackburn. Thank you, Mr. Chairman.
    Dr. Shuren, looking at the LDT guidance, do you plan to 
finalize that guidance that you issued last year? Do you plan 
to finalize that this year?
    Dr. Shuren. Yes, we do plan to finalize that.
     Mrs. Blackburn. OK. When?
    Dr. Shuren. In 2016.
    Mrs. Blackburn. OK. So basically you are going to put it 
off another year?
    Dr. Shuren. I don't get to determine when, but the plan is 
to put it out in 2016.
    Mrs. Blackburn. In 2016. Early or late?
    Dr. Shuren. Hopefully earlier than later.
    Mrs. Blackburn. OK.
    Dr. Shuren. I wish I could give you an answer. Again, it is 
so far above my pay grade. I don't even know the people who 
make the decisions.
    Mrs. Blackburn. Well, my goodness, we need to have a meet-
and-greet over at the FDA and see if we can't get some wheels 
turning over there. We should help with that.
    Let me ask you this. As you finalize that guidance, do you 
intend to use what I think is the outdated 1970s definition of 
a medical device in order to regulate the LDTs?
    Dr. Shuren. Well, so that definition also includes a 
distinct definition for in vitro diagnostics, which then 
incorporates laboratory-developed tests. It does not 
distinguish who makes the tests.
    Mrs. Blackburn. OK. Let me ask you this. Each improvement 
in an LDT technology or an upgrade or an update, will that need 
to go back through the medical device approval process?
    Dr. Shuren. No. Most modifications to tests are not 
reviewed by FDA. We only focus on those that have the really 
big impact, yes.
    Mrs. Blackburn. OK. Do you intend to add to the rapid 
growth of healthcare costs by taxing LDTs as medical devices in 
addition to charging the innovators the user fee?
    Dr. Shuren. So we are not responsible for administrating 
the device tax. That is IRS. We have nothing to do with it.
     That said, one of the reasons we put in place that 
laboratories could notify us about their tests as opposed to 
registering and listing was that it would not trigger the 
device tax.
    Mrs. Blackburn. I think your guidance informs the IRS, 
though, is that not correct?
    Dr. Shuren. No, the IRS would look separately to if that 
device has listed.
    Mrs. Blackburn. OK. I want to thank you for the report that 
you sent to the Congress last night. It was an interesting 
read. And what I found most interesting about it was what was 
left out and that you didn't discuss the FDA front-end process, 
which deserves some attention and some discussion, specifically 
the PREDICT program. Twenty-eleven this was put in place. It is 
a compliance program. It is an artificial intelligence program 
that is supposed to identify high-risk shipments at our ports 
of entry. And the problem with PREDICT is that it is 
significantly delaying the shipment of needed medicine and 
medical supplies. Medical shipments are often sent by express 
service to get them to patients in time for critical usage. And 
once a shipment is held up by PREDICT, almost all of them are 
subsequently released without any physical inspection.
    So when you look at it from the outside, Dr. Shuren, what 
it appears to be and the impression is that the bureaucracy of 
the FDA is keeping medicines and medical supplies from patients 
because of concerns that there may have been contamination in 
some cilantro that was in the very same shipment. And I would 
really like to see the FDA spend the effort to fix this before 
they try to regulate another area of commerce.
    I noticed in that report also that it is based on 20 case 
studies. And how often does the FDA use case studies as 
sufficient evidence to approve or deny a medical therapy?
    Dr. Shuren. So in terms of approval, we don't tend to rely 
on an example. We have used a series of case studies as part of 
support for valid scientific evidence as we have approved 
certain tests or other products.
    I will note one thing about the cases, too, we put out 
yesterday. One of the challenges is that we don't have post-
market surveillance in place for laboratory-developed tests as 
we do for tests made by other manufacturers. And as a result, 
it is very hard to identify when problems arise. And yet we 
know on the IVD side, when made by conventional manufacturers, 
we do detect problems, the manufacturers detect problems, and 
they fix them because that is in place.
    One of the other features in the FDA system is the post-
market surveillance to identify problems and to fix problems, 
and that is just as important as premarket review to prevent 
faulty tests from getting on the market in the first place.
    Mrs. Blackburn. Well, as I yield back my time, I hope that 
you will fix PREDICT. I yield back.
    Mr. Pitts. The Chair thanks the gentlelady and now 
recognizes the gentlelady from California, Mrs. Capps, 5 
minutes for questions.
    Mrs. Capps. Thank you both, each of you, for your 
testimony, and thank you, Mr. Chairman, for putting together 
this hearing. I appreciate the opportunity to further discuss 
the strengths and challenges, have a real conversation about 
laboratory-developed tests as they guide medical decision-
making by patients and providers. There are a lot of question 
marks and a lot of concern about where do we go from here, how 
do we dovetail these two agencies and giving the best outcome 
the patients.
    I recognize there are many perspectives in this space. I 
appreciate this conversation to try to illuminate some of the 
issues. Over the years, it goes without saying these tests have 
come more tailored and more elaborate, and this conversation is 
important to ensure that these tests do produce meaningful and 
reliable results for those who trust them.
    Dr. Conway, I will turn to you first. In your testimony you 
note that there are six key performance specification 
assessments for lab-developed tests: accuracy, precision, 
reference range, reportable range, analytic sensitivity, 
analytic specificity. This committee has heard a lot about how 
different research institutions are driving the future of 
medicine toward more personalized medicine. This has been 
particularly true in the field of cancer where the development 
of multi-gene panels is being used to identify important 
molecular characteristics of a tumor.
    And my question to you is whether the current CLIA 
regulations ensure these gene panels developed by different 
institutions or manufacturers will produce the same results? 
For example, if I am a patient and I am tested with hospital 
A's gene panel, how do I know I would get the same result if I 
am tested with hospital B's gene panel? Would each hospital 
reach the same treatment decision, and where does this lead us?
    Dr. Conway. Yes, you have highlighted--thank you for the 
question, and you have highlighted one of the challenges. And 
Dr. Shuren could certainly speak more.
    You know, our assessment of analytic validity is laboratory 
by laboratory where we are looking at the areas you described 
and the laboratory director's documentation, that they are 
following a protocol to detect the appropriate analyte.
    And you highlighted a great example, genetic testing. It is 
not assessing whether different genetic testing kits or 
combination of tests are detecting the disease with the same 
clinical validity and rigor. So you could in fact in the 
current framework, without premarket assessment of clinical 
validity, have different tests giving different answers to 
clinicians that could drive treatment that is inappropriate, 
which is why we think the assessment of premarket clinical 
validity in this area is critical and important.
    Mrs. Capps. So that leads me to focus now with you, Dr. 
Shuren. Many have argued that there is no need for greater FDA 
oversight of lab-developed tests, as we have not had the same 
types of problems with LDTs as we have had with drugs such as 
the outbreak of adverse events associated with use of 
contaminated heparin, for example, or adverse events associated 
with contaminated compounded drugs. They assert that if there 
are greater health risks associated with LDTs, we would have 
heard about them.
    I am not sure you agree, but it is clear to me from the 
report that FDA released yesterday that lab-developed tests do 
present real risks to patients. Can you please explain whether 
or not you agree with this criticism that came out? Would 
healthcare providers and patients necessarily know if tests 
were not giving good advice for clinical decisions?
    Dr. Shuren. I don't agree with that criticism. And doctors 
and patients would not know who made the device and whether it 
is one that was approved by FDA or it was one that was not 
approved by FDA. Quite frankly, the reason you don't see as 
many problems, you don't have the systems in place to identify 
them. So, for example, for IVDs we regulate, in 2014 we had 
over 300 recalls. It is not unusual.
    Things change, problems arise, but you need the systems to 
identify the problems and to fix them. And we have some labs 
who have submitted their tests to us, and we have approved or 
cleared some LDTs. And when they put the systems in place, 
these started to identify problems. One of them has already had 
eight recalls, but they only found the problems because they 
put in the systems that they should have in place.
    Mrs. Capps. Well, now, how can patients--I am just about 
out of time, but how can patients, providers, and payers be 
assured that the tests they are paying for are providing real 
value and enhancing the care of patients?
    Dr. Shuren. Well, that is why we would like to have a 
uniform, consistent approach to diagnostic tests, regardless of 
who makes them. That information will be made available to the 
public so they know what tests have been approved. There is 
information about what those tests are for. The makers have to 
put out information that explains its performance 
characteristics, its intended use, how to use it properly, and 
all that will provide necessary information to doctors and 
patients so they can use those tests appropriately.
    Mrs. Capps. Thank you.
    Dr. Shuren. Right now, they can't.
    Mrs. Capps. OK. It sounds like we need a follow-up. I yield 
back.
    Mr. Pitts. The Chair thanks the gentlelady and now 
recognizes the chair emeritus of the full committee, Mr. 
Barton, 5 minutes for questions.
    Mr. Barton. Thank you, Mr. Chairman. I want to thank both 
our witnesses for attending today. I appreciate the 
subcommittee chairman, the full committee chairman issuing the 
proposal as a discussion draft, which to me means that their 
minds are still open and that we can make some changes and 
things of this sort.
    I am one of those skeptics that Mrs. Capps just talked 
about. I am not sure that we need to get more Federal 
regulation. I don't necessarily think more Federal regulation 
is going to give us a safer, more efficacious result.
    So I guess my first question to either of you gentlemen 
would be what is the real problem? I mean why in the world 
would a laboratory develop a test that wasn't safe and 
accurate? My office is not being overrun with phone calls or 
emails from doctors, patients, hospitals, advocacy groups that 
there is some terrible laboratory diagnostic test out in the 
marketplace.
    Dr. Shuren. But those tests are out on the marketplace. So, 
for example, a test was developed for something called KIV 6. 
It was supposed to predict the risk of heart disease and 
response to statin treatment. And the lab came out with it, 
promoted it, said they had studies, but then subsequently good 
studies were performed and in fact found that there was no 
association between KIV 6 and those conditions. And by the time 
it came out, though, over 150,000 people had the tests 
performed. We estimated the cost to our healthcare system was 
over $2 billion. That is not money we can afford to waste on 
bad testing.
    Mr. Barton. And what happened----
    Dr. Shuren. So what happened----
    Mr. Barton. I assume that test was taken off the market and 
without FDA having to do anything.
    Dr. Shuren. It remained on the market and there was 
continued use for a while and then use started to dip down. But 
is that really the system we want, that we have bad tests, 
people can get hurt by it, and then afterwards if you find the 
problem and you get on top of it, then something happens to the 
test? The whole point of premarket review is and why we do that 
for the other tests----
    Mr. Barton. You are going to guarantee if we would let your 
agency review all these diagnostic tests, the laboratory tests, 
that something like that will never happen again, that you all 
are perfect and all-knowing and you are going to do it in a 
cost-effective way and it will be peace and light from now 
until Judgment Day?
    Dr. Shuren. I will not promise you perfection, and I will 
leave it to God to decide if there will be peace on Earth, 
but----
    Mr. Barton. Well, I am glad to hear somebody use----
    Dr. Shuren. But that said, we have----
    Mr. Barton [continuing]. The Divinity's name in a positive 
way. That is----
    Dr. Shuren. Yes, well, they can fire me.
    Mr. Barton. That is a good thing.
    Dr. Shuren. But we have almost 40 years of experience of 
regulating in vitro diagnostic tests and assuring that those 
tests are accurate, reliable, and clinically----
    Mr. Barton. I mean, granting your point at least partially, 
wouldn't it be better to give FDA or some State regulatory 
agency--it doesn't necessarily have to be Federal--some sort of 
a penalty assessment that we can immediately put a stop if 
there is a bad test? Wouldn't that be a better use of your 
agency's resources? So to use your example, if that were to 
happen again, boom, we catch it, we stop it, we hit them with a 
big penalty and get that test off the market. I am not being a 
horse's rear on this, but, you know, if it is not broken, don't 
fix it, and it looks to me like we are just looking for ways to 
give the CMS and the FDA more authority. And it is obvious that 
Chairman Pitts and Chairman Upton and I assume Mr. Pallone and 
Mr. Green are concerned, too. But more regulation is not always 
the best answer.
    I guess my last question would be under the current system 
what role if any do the States play in looking at these tests?
    Dr. Shuren. So there are States--I can let Dr. Conway talk 
about it in terms of States that are involved in accreditation 
of laboratories, but they are not involved in premarket review 
for those tests with certain exceptions. New York State does do 
a review of tests. And quite frankly, under the proposal we 
have, we have the opportunity to leverage third parties. If New 
York State is meeting appropriate standards, we could leverage 
some of the work that they are doing.
    But I will tell you the problems are more prevalent than 
people want to recognize. You know, one of the medical centers 
at the University of Texas was concerned about this----
    Mr. Barton. I went to A&M so that doesn't scare me.
    Dr. Shuren. No, no, no--well--but I will tell you what they 
were finding is----
    Mr. Barton. I am going to hear it whether I want to or not.
    Dr. Shuren. That is right. Thank you for that. But there 
were inconsistencies in what they were seeing reported by labs 
for the same kind of tests, so what they did is they took 
results from 105 of their cancer patients and they had results 
from one laboratory, they sent it to a second laboratory, and 
of the 32 gene variants, they found 50 percent disagreement, 50 
percent. And so they even concluded that this suggests 
physician care would differ based on different interpretations 
of different companies. And this is not the only report out 
there, other ones reporting 27 percent finding of incorrect or 
inaccurate results. This is not uncommon. This goes on.
    It is fixable, and it shouldn't be fixed after the fact. 
Why should our people get hurt, and only when that happens--and 
if we can find it because we don't have the systems to do 
that--do we take action. Is that really the kind of health care 
we want to provide? Do we want to spend money on unnecessary 
care or do we want to spend it on innovation and assuring those 
tests work.
    Mr. Barton. Well, I am with you on the innovation part.
    Mr. Chairman, you know, the other subcommittee's got the 
FCC commissioners downstairs, so I am not going to be able to 
stay, but I appreciate you holding this hearing and thank you 
for the courtesy of the time.
    Mr. Pitts. The Chair thanks the gentleman and now 
recognizes the ranking member of the full committee, Mr. 
Pallone, 5 minutes for questions.
    Mr. Pallone. Thank you, Mr. Chairman.
    I wanted to ask Dr. Shuren. As you know, various 
stakeholders have been circulating legislative proposals 
regarding the regulation of lab-developed tests. Among these 
various legislative proposals, there seems to be a great deal 
of variance around moderate-risk tests. Some proposals have 
suggested that no premarket review is necessary for moderate-
risk tests, and one proposal would require premarket review of 
moderate-risk tests but would allow such tests to be deemed 
approved if FDA did not act in a specified time frame.
    So I wanted to ask you a couple questions about this. In 
your testimony you noted an example of a test that is moderate 
risk would blood glucose strips used by people with diabetes 
and tests to help doctors diagnose heart failure. Could you 
discuss what FDA considers to be moderate risk and provide some 
examples of tests that would be considered moderate risk?
    Dr. Shuren. So other moderate-risk tests would be for 
diagnosing cystic fibrosis, herpes, heart failure are all 
moderate-risk tests----
    Mr. Pallone. OK.
    Dr. Shuren [continuing]. That we currently regulate.
    Mr. Pallone. Does the FDA believe that premarket review of 
moderate-risk tests that there should be, and if so, can you 
describe when you believe that premarket review of a moderate-
risk test would be necessary?
    Dr. Shuren. So we do believe that most moderate-risk tests 
would be subject to premarket review. That is what we do now, 
but we do find that there are certain circumstances where we 
can put other mitigations in place that are good enough and you 
don't need premarket review. We just did that a little while 
back for next-generation sequencing platforms, when they are 
just making tool claims. We just did that for autosomal 
recessive carrier screening tests. And that is a natural course 
of action. As the science develops, technology evolves, we can 
actually change risk classification or what a test will have to 
do to come on the market. That is hallmarks of a risk-based 
approach.
    We are also very concerned about this deemed-approved 
approach. It essentially says if we don't make it decision in 
time, it is approved. So a test goes on the market that may be 
inaccurate simply because we didn't have enough time to finish 
up the review or, alternatively, we will not approve it to not 
let it go on the market. And yet if we had the time to work 
with the lab, we might get a good test on the market. Either 
way, bad tests on the market, good tests not going on a market, 
the loser is patients.
    Mr. Pallone. OK. I think you answered my next question, 
which would be, you know, the deemed approval if FDA does not 
act. But let me say, can you comment on whether or not--yes, I 
think you already talked about the deemed approved. So let me 
go to another question, Dr. Shuren.
    I understand that once the test is approved or cleared by 
FDA and enters the market, laboratories frequently modify the 
kits either to expand uses or to make improvements to the way 
the test is performed. And some stakeholders in the lab 
community have even suggested that manufacturers rely on 
laboratories to modify tests in order to expand the uses 
because it is too costly or burdensome to have a test approved 
for every use. So do you believe that modifications to LDTs 
should be subject to premarket requirements, and if so, what 
types of modifications would FDA want to look at before they 
are put in place?
    Dr. Shuren. So we think most changes that are made would 
not be subject to FDA review, and that is actually what occurs 
now for other IVDs. We would review those changes when there is 
a new intended use because it truly is a new test. Even CLIA 
have used that as a new test. Or if there is a big enough 
change that when a test is approved would determine what its 
performance specifications are, if now you make a change and it 
goes outside the performance specifications, we would review 
that as well.
    Mr. Pallone. OK.
    Dr. Shuren. But it is those kinds of changes.
    Mr. Pallone. Let me go back. There was other thing that I 
could have asked you about, these tests are deemed approved if 
FDA didn't act in a certain time frame. Can you explain whether 
or not you believe patients, physicians, or payers would know 
which tests were affirmatively cleared or approved by FDA 
versus tests that were deemed to be approved? Is there any way 
that they would know that?
    Dr. Shuren. No. I mean if the test is approved, the test is 
approved.
    Mr. Pallone. So that is one of the dangers if you will. In 
other words, you said before that you would be concerned that 
you might approve something that shouldn't be or not approve 
something that could be. But the secondary problem is that the 
user is not going to know.
    Dr. Shuren. That is exactly right.
    Mr. Pallone. All right. Thanks a lot.
    Mr. Guthrie [presiding]. Thank you. And the gentleman 
yields back. I now recognize myself for 5 minutes for 
questions.
    Dr. Shuren and Conway, what does premarket review mean in 
the context of lab-developed tests, or LDTs? And how important 
is it and who should be responsible for such reviews?
    Dr. Shuren. So our premarket review is to determine if the 
tests are analytically valid, clinically valid, and they are 
safe for use under their conditions for use. And it is 
important to conduct those reviews for moderate- and high-risk 
tests to make sure they work because doctors and patients rely 
on those tests to make well-informed healthcare decisions. If 
they get inaccurate results, they could make the wrong 
decisions and people get hurt as a result.
    Mr. Guthrie. Thank you. Dr. Conway?
    Dr. Conway. I agree with Dr. Shuren. And from the CLIA/CMS 
perspective, you know, we are really focused on laboratory-by-
laboratory post-market review of those laboratory protocols, 
equipment, training of personnel, et cetera.
    Mr. Guthrie. OK. Thank you. And I have heard that the same 
type of diagnostic test that is commonly available as both an 
LDT and a manufacturer kit that can be purchased by a lab, and 
tests for melanoma are one example. In other words, I could go 
to hospital A where they have a lab that developed their own 
test or, by chance, I could go to hospital B, whose lab 
purchased a test from a manufacturer. Can either of you walk me 
through this scenario from a regulatory perspective? In the 
case of hospital A, is it true that neither FDA nor CMS will 
have reviewed that test for clinical validity?
    Dr. Shuren. That is true. The test across the street would 
have been reviewed, and therefore, doctors and patients have 
the confidence to be able to rely on it, and you don't know in 
the case of the other one that has been offered.
    Mr. Guthrie. OK. Same----
    Dr. Conway. Yes, same. And CMS, once again, would not 
review clinical validity as part of the CLIA process.
    Mr. Guthrie. OK. Doing the premarket review of these types 
of tests, could each of you describe the general education and 
professional background and expertise of your reviewers?
    Dr. Shuren. So our review, particularly for more complex 
tests, tends to be performed by a team of experts. They may 
include physicians, Ph.D. scientists, and statisticians that do 
a deep dive into the scientific data. We review the science to 
see if in fact and not only that test works but what it is 
claiming to do matches up with the science.
    Mr. Guthrie. Thank you. Dr. Conway?
    Dr. Conway. Our CLIA team does not include any medical 
officers or other personnel trained in detailed biostatistics 
or Ph.D.-level training. Our CLIA team is really focused on, as 
I mentioned, laboratory assessment on an accreditation and 
quality and survey and certification framework in a post-market 
manner laboratory by laboratory.
    Mr. Guthrie. Thanks. And, Dr. Shuren, you previously 
testified about challenges you face in hiring and retaining 
sufficient medical expertise. How would your ability to do so 
be impacted if CMS were required to have the same types of 
expertise regarding test design and development?
    Dr. Shuren. Well, first off, I want to thank the committee 
for trying to take actions in 21st Century Cures to help us to 
better be able to attract and retain high quality talent. And 
that is where the answer lies.
    Mr. Guthrie. So the competition if CMS is doing the same 
would be----
    Dr. Shuren. Well, it makes no sense for competition, so all 
we are going to do is create a duplicative system in another 
agency. I mean it is interesting that people have raised 
concern about do not have duplicative regulatory frameworks in 
place, and yet some of the proposals we have seen now to put 
this under CLIA would do exactly that. It would create all this 
duplication the right now, as you have heard from both of us, 
doesn't exist.
    Mr. Guthrie. All right. Thank you. And I yield back the 
balance of my time.
    And I now recognize Ms. Castor from Florida.
    Ms. Castor. Thank you, Mr. Chairman. And thank you both for 
being here today.
    As we continue to develop a greater understanding of the 
genetics of individuals who have a wide variety of diseases and 
conditions, we are moving away from one-size-fits-all medicine 
to more targeted and effective prevention strategies and 
treatments and even cures. This is known as personalized 
medicine, and I believe it is fundamental to the vision of 21st 
Century Cures and holds great promise.
    This vision, though, will in large part be dependent upon 
accurate genetic tests, so it is imperative that these tests 
are scientifically credible. Dr. Shuren, can you provide some 
examples of the types of genetic tests that are being developed 
to help deliver personalized treatment? And describe in greater 
detail the role that these tests play in precision medicine.
    Dr. Shuren. So increasingly, we are seeing genetic tests 
being developed to help identify what the appropriate treatment 
may be for patients who have various conditions, including 
cancer. And it is critically important that those tests work, 
because if not, people are not getting the right treatment or 
they are not getting treatment when in fact they should get 
treatment.
    I will say that as we approach this, though, Government can 
be innovative. Increasingly, we are seeing next-generation 
sequencing tests being used, and last December, we put out a 
proposal for a new approach on next-generation sequencing that, 
rather than your standard model of maybe doing a clinical study 
is to leverage data in existing curated databases, which can 
allow for the clinical community to crowd-source the evidence, 
and as the science ultimately evolves to where it needs to be 
to be able to make claims about the use of that test. That way, 
the regulatory framework can stay step-in-step with the 
evolution of the science.
    In fact, we just held a two-day public meeting last week on 
this. There is a lot of support for moving forward with this 
approach. We have even relied on those curated databases to 
approve a test for cystic fibrosis.
    So that is where we need to focus our attention, and that 
is why we want the lab community at the table with us. Let's 
focus on the science. That is what we need to do. We have the 
regulatory tools. It is the science we have got to work 
together on. And we can do it if people are willing to work 
with us.
    Ms. Castor. Yes. And during our 21st Century Cures hearings 
and briefings, there was a lot of talk about data-sharing. What 
is going on--because we can't wait for Congress to act, 
frankly. What is going on with FDA and NIH and a lot of those 
research institutions across the country in being able to look 
at that data, share it, so we can develop the cures and 
treatments of the future?
    Dr. Shuren. Yes. So NIH has its own database of genetic 
variants. They do an assessment. We have other databases out 
there. We are now trying to work with these various groups on 
what the appropriate standards should be for the quality check 
for the curation and what should be the standards for clinical 
validity when you are evaluating that science.
    Also, we at the FDA have been developing a platform called 
precision FDA that would allow these test developers to 
essentially either share their genetic data to compare or 
providing analytical tools so they can test-drive some of these 
next-generation sequencing technologies to see if they are 
accurately sequencing the genome. We think this is a great 
role, if you will, to provide these common goods to all 
developers.
    Ms. Castor. Great. I do, too. Thank you. And I yield back 
my time.
    Mr. Guthrie. Thank you very much. The gentleman from 
Illinois, Mr. Shimkus, is recognized for 5 minutes.
    Mr. Shimkus. Thank you, Mr. Chairman. And welcome.
    Actually, I appreciate the comments from my colleague from 
Florida. That is kind of where I was just heading to a little 
bit, too, with the personalized medicine and the genetic 
testing and really being accurate on that test so then you can, 
as we talked about in the other piece of legislation, target 
based upon the genetic code or the individual patient. That is 
very exciting.
    And the other thing I think we have followed through the 
hearings and the 21st Century Cures is that then you just don't 
go down the route of prescribing remedial health action to 
someone without really full information, so the high cost of 
health care because you try this, didn't work, try this, didn't 
work, now you are trying this, and you can get more specific 
information. So it is very exciting times. And I think people 
were going around the same issues.
    But I wanted to ask this, and it is probably something I 
should know if I would have more thoroughly read my briefings, 
but when we talk about risk--basic, moderate, or high--so we 
are really focusing on moderate and high risk of the tests. 
What is the risk component? Is the risk component the risk of 
conducting it, the risk of not having accurate information, or 
the risk to the patient who hopes to get good information from 
a test because of the healthcare environment they presently 
find themselves in? So can you both talk on how do we define 
risk?
    Dr. Shuren. Yes. So the key consideration is the risk to 
the patient if they are getting an inaccurate result, they are 
getting a wrong result, and that is within the context of what 
would otherwise happen to that patient in clinical care. That 
is the way we look at it.
    Mr. Shimkus. So when you use the example of heart, you put 
that in a moderate--when you were giving the examples of--and I 
was kind of surprised. I mean, heart disease or heart issues, I 
think people would find it pretty risky if you have got heart 
disease, a higher risk than just in the moderate category. So 
there is some subjectivity to this or----
    Dr. Shuren. Well, so when we look at it, you put it within 
the clinical context. So in the case of heart failure, when you 
are making, you know, a diagnosis, there are other things that 
the clinician takes into account in making that determination. 
That is a little bit different, though, when I am dealing with 
something, let's say, like HIV where not only am I dealing with 
a high-risk condition, right, the risk to the patient is huge, 
secondly, I don't have another great means of truly determining 
is that HIV. And then there is also the risk of if I am wrong 
about this and that person goes out and doesn't know they have 
HIV, they may engage in activities that they will spread the 
disease. So we are really looking at it in the practical 
context of what in fact happens to the patient, not just simply 
the condition itself.
    Mr. Shimkus. Right. And I think this is a tough area for 
conservative Republicans who think Government is too big, costs 
too much, but there is obviously a position of we want to make 
sure that people are advertising and using tests, that they are 
given some stamp of approval, that they meet the requirements 
and the desires of what they are.
    So, Dr. Conway, real quick, you admit that the volume and 
complexity of these tests have kind of grown, I don't know, I 
would say exponentially almost. Would you agree with that?
    Dr. Conway. Yes. We don't have exact numbers for some of 
the reasons described, but it seems exponential.
    Mr. Shimkus. But you haven't asked for new authorities 
because of this growth, have you?
    Dr. Conway. So CMS has not put forward additional requests 
for statutory authority. As I mentioned, we think FDA can play 
a critical role in the premarket review, and we can play a 
critical role laboratory by laboratory, post-market.
    Mr. Shimkus. Yes, in your area do you require an individual 
review of the area that you have been involved with? Is there 
an independent review process of decisions that you are making, 
you know, in the CLIA process?
    Dr. Conway. Let me try to answer that. So I think we have a 
central office that has oversight of State surveyors, and 
therefore, oversight of the processes of those State surveyors. 
We also oversee accrediting organizations, of which there are 
seven. They have to meet or exceed CLIA standards, and we 
review that, including if any----
    Mr. Shimkus. But you are almost evaluating the 
organizations. The organizations aren't evaluating the 
independent decisions?
    Dr. Conway. We have bidirectional communication both with 
the States and their accrediting organizations like in any of 
our accrediting organizations, including at times in various 
programs accrediting organizations identify regulations or 
standards that need updating.
    Mr. Shimkus. Great. Thank you very much. I yield back my 
time.
    Mr. Guthrie. I thank the gentleman. The time is expired.
    And I recognize Dr. Schrader from Oregon for 5 minutes for 
questions.
    Mr. Schrader. Thank you, Mr. Chairman. And I would like to 
thank Dr. Shuren and Dr. Conway for being here. Interesting 
topic. I would hope that the chair or vice chair and ranking 
member would hopefully have us have an opportunity to talk to 
the stakeholders, including the physician groups, just to get a 
balanced perspective here. This is pretty darn important if we 
are going to go down this road, and I think the tender of the 
questions so far indicate that.
    And I appreciate the fact both of you testified in total 
agreement in pretty clear terms about how you guys have two 
different jobs in the different agencies. It is tough from a 
practitioner's standpoint, being part of the medical community, 
to really understand why that has to be. I understand it is 
right now, but I am not sure why it has to be. It seems odd to 
me that the Center for Medicare Services--medical services 
would not have some sort of health regulatory role or clinical 
analyzation capability.
    And it seems to me both of you are going to have to staff 
up, well, particularly FDA if you take on this new role of 
premarket approval. There is going to be a huge staffing 
increase. Why would that not also be possible for the folks in 
CLIA or somewhere in CMS to do the same thing? I ask both of 
you that question.
    Dr. Shuren. So we already have existing staff who do 
exactly these kinds of reviews, and we have years of experience 
on it. We have training programs for our people. And in terms 
of additional resources, one of the reasons that we put in 
place a phased-in approach would be also for tests that are out 
there, one, not disrupt the market; two, that we could try to 
accommodate what resources we have. But in addition, if we need 
additional resources, we have a user-fee program under which we 
work with the regulated community about appropriate funding for 
services that we then provide back like performance and 
premarket review. And that program, as you know, has been in 
place for a number of years.
    Mr. Schrader. So minimal staffing increase is what you are 
suggesting?
    Dr. Shuren. It depends on the ultimate framework that goes 
into place as to what that workload would look like.
    Mr. Schrader. All right. Mr. Conway, if you can.
    Dr. Conway. On the CMS side, as I mentioned in the central 
office we have approximately 25 people in total overseeing 
CLIA. They are trained for their job, which they do well, which 
is oversight of laboratories, laboratory by laboratory. There 
are no medical officers, there are no Ph.D.'s, biostatisticians 
because we do not do premarket review.
    Mr. Schrader. I just get concerned still--sorry--because 
both your testimonies talk about accuracy, both of you. You 
both talk about reliability. And that sounds like overlap to 
me. So I am just concerned that we don't go down that road. 
Question on peer-review. I mean a lot of treatments and 
diagnoses are peer-reviewed in the literature and stuff. Has 
that occurred at all with laboratory tests? Is there any 
literature reviewing the efficacy of different laboratory 
tests?
    Dr. Shuren. In our review of tests we do look at published 
literature, and in some cases we have relied completely on 
published literature for certain tests like hemoglobin A1c for 
diabetes.
    This issue about accuracy, our look at it, though, for 
analytical validity is complementary but it is different. We 
truly look at is the test itself and what it measures, is it in 
fact accurate? CMS will look at is that test performed properly 
to get a result.
    Mr. Schrader. That is correct. So I guess my underlying 
concern as a medical professional listening to the testimony is 
that the consumer, as well as the physician or veterinarian, is 
not misled by having premarket review. There is going to be 
some certainty that that test is 100 percent appropriate for 
them in their situation.
    The reason I raised the question about the peer review, I 
mean, generally, the test from my standpoint is a secondary 
adjunct to helping establish the diagnosis. You got a lot on 
clinical sides, you got a lot on knowing your patient, got a 
lot to, you know, based on the environment they are living in. 
There are false positives all the time in every single test, 
false negatives in virtually every single test, whether it is a 
genetic test or, you know, a simple blood test for goodness 
sakes. I just don't want the consumer to be misled that by 
having FDA premarket approval, that that test is going to be 
100 percent. I think that is a mistake.
    At the end of the day I think it is up to the medical 
community, the physician to put that one small piece of the 
puzzle into the, you know, whole diagnostic scheme and come up 
with whether or not that is actually going to be a valid use of 
their patient.
    I am just very concerned the tone here is that we are going 
to put certainty into the art of medicine when there is not 
that much certainty, and the patient will be misled and frankly 
lead to greater lawsuits and customers frankly not 
understanding what medicine is really all about.
    Dr. Shuren. And so the accuracy of that test will also 
depend on what the use of the test is for. You know, when you 
deal with riskier conditions or where there are some tests you 
truly rely on the result of that test. Companion diagnostics, 
for example, it is the result of that test that will be telling 
you should they get Zelboraf, you know, for melanoma. And in 
those cases you want to have a higher accuracy.
    You are right, it is not 100 percent, but also what we 
assure you is that that information is made available to the 
practitioner like you, and you know that when you get that 
number for how accurate it is, the result, it is correct. It is 
the----
    Mr. Schrader. Well, laboratories have different--laboratory 
information from one lab to the other is going to be different. 
I can send the exact same blood sample in to a different 
laboratory. I can send the genetic code in. You testified a 
moment ago you are going to get different information back. So 
the idea that it is going to be dispositive, I would 
respectfully disagree. And I yield my time.
    Mr. Guthrie. Thank you. The gentleman's time is expired.
    I now recognize Dr. Burgess of Texas for 5 minutes.
    Mr. Burgess. Thank you, Mr. Chairman.
    Before I start my time, could I asking for a unanimous 
consent request?
    Mr. Guthrie. The gentleman is recognized.
    Mr. Burgess. Ask unanimous consent to enter the statement 
of the Association for Molecular Pathology into the record. And 
then a further unanimous consent request for a point of 
personal privilege, Mr. Chairman.
    For the past 7 years I have been joined at these committee 
hearings by Mr. Paluskiewicz, whose last name is so difficult 
to pronounce we all know him by J.P. And if I ever seem 
adequately prepared for these hearings, it is only because I 
have had J.P. advising me before we come into the hearing room. 
And so it is with great sadness that I announce that J.P. will 
be leaving my employment, but he will be joining the committee 
staff, so he will be here for all to participate and the 
wondrous things that he has to offer to any committee hearing.
    Thank you, Mr. Chairman. Now recognize for questions.
    Dr. Conway, so if Dr. Shuren puts his guidance out in 
January, are you no longer necessary?
    Dr. Conway. No, sir, and let me explain why. I think there 
will still be a role for CLIA to assess, and this is a 
critically important role, that laboratories have the proper 
equipment, training, protocols, and quality assurance 
procedures in place, and that laboratory-by-laboratory 
certification, which a few people have talked about, is a 
critical role for CLIA.
    Mr. Burgess. But, you know, we have heard several times the 
FDA is under-resourced, so why shouldn't the resources that are 
going to CMS just simply go to the FDA?
    Dr. Conway. So I will speak for CMS. You know, I think in 
the CLIA oversight framework we are efficiently using both 
central office resources and relying on States, which was a 
question earlier, and their State surveyors, obviously a user-
fee funded program based on user fees based on moderate, high 
complexity, et cetera, and volume. And then we also importantly 
have nonprofit accrediting organizations that are not 
Government organizations, that we--seven of them--approve that 
they meet or exceed CLIA standards. We are using people outside 
of the Federal Government as well to perform these important 
functions.
    Mr. Burgess. Dr. Shuren, this question has been posed to 
you several times in this subcommittee or oversight 
subcommittees about what is the problem that we are trying to 
solve? And last night at 7:00 p.m. you put out a report that 
detailed 20 times where perhaps there were problems with 
laboratory-developed tests, is that correct?
    Dr. Shuren. Yes.
    Mr. Burgess. And I am sure you would make the further 
statement that there are more than that, but we have also seen 
in testimony that what is the total universe of laboratory-
developed tests? It is in excess of 11,000, is that correct?
    Dr. Shuren. It is above 11,000.
    Mr. Burgess. So the rate at which you have detected 
problems would be, if my math is correct, .18 percent, which 
most things in medicine are hardly that reliable. Is that an 
unfair statement?
    Dr. Shuren. Yes. Reporting systems--first of all, there is 
no reporting system on LDTs. You are not monitoring for 
problems. And so you can't say what the rate is, quite frankly.
    Mr. Burgess. It took you 3 years to provide us with 20. 
When I asked you in hearings, when we were doing the FDA 
reauthorization, what is the problem we are trying to solve? So 
today, now, I have your report, 20 problems that we are trying 
to solve, and we have got a universe in excess of 11,000 tests.
    So let me just ask you this, since you think the risk is 
there from laboratory-developed tests, is there an FDA-approved 
kit that has ever had a failure?
    Dr. Shuren. Yes. And the point is the reason we can 
identify when there are problems and we can deal with it is 
because we have the systems in place and the maker of the test 
has implemented systems internally to identify those problems. 
That is critical. And the work that we are doing doesn't occur 
right now in CMS. It is not duplicative, and they don't go 
away.
    Remember, if you make a test, if you are a conventional 
manufacturer, that lab is going to get your test. They still 
have to perform that test properly, and that is what CMS is 
overseeing, are the laboratory operations conducted properly.
    Mr. Burgess. My time is limited. So do you envision any 
lack of access to testing because of the changes that you are 
proposing in the guidance or the committee is proposing in 
their legislation?
    Dr. Shuren. So we have tried to--a proposal was put in 
place so that we would not disrupt the marketplace. Our goal 
here is to try to assure we do have innovation. We think LDTs 
are important in health care. There is innovation, but----
    Mr. Burgess. I appreciate the recommendation----
    Dr. Shuren [continuing]. There is no value to patients if 
the tests in fact don't work. And one of the problems is 
because we haven't regulated, there has been a disincentive for 
innovation by conventional manufacturers. And we have heard 
from them, particularly the smaller companies are saying they 
are disadvantaged because they make a test and they go through 
and they have to demonstrate their test works. And then you can 
have a lab make the same kind of test go out the door----
    Mr. Burgess. I am going to have to interrupt you because my 
time is limited.
    Just as far as the labs themselves, who do you expect to be 
more greatly impacted, large labs and large hospitals or 
smaller rural labs? Is there likely to be a difference in the 
impact? It is a yes-or-no question.
    Dr. Shuren. The answer is you should regardless be 
developing the science that your test is validated, whether we 
review it or not.
    Mr. Burgess. If you don't know whether the answer is yes or 
no, why wouldn't we want to see an economic impact evaluation 
such as normally would be required in rulemaking but is not 
required in guidance?
    Dr. Shuren. Well, just to clarify again, whether we were 
overseeing them or not, a lab shouldn't be putting any test on 
the market that they haven't gotten the data to validate. What 
we are saying is you should have the data and we would look at 
it before the test went on the market to make sure that that 
test in fact worked.
    Mr. Burgess. And to the question----
    Dr. Shuren. The tests that wouldn't go on the market are 
the ones that in fact don't work.
    Mr. Burgess. And to the question of an economic impact 
statement, as would be required under normal rulemaking 
processes, why shouldn't the committee or the Congress expect 
that?
    Dr. Shuren. We are not under rulemaking because we are not 
imposing new requirements. These requirements already exist 
under the law. As a matter of policy, we have not actively 
enforced them. And in places where we put an enforcement 
discretion policy, we have withdrawn it. We have done that 
through guidance. It has been the practice all along.
    I would say in terms of economic analysis, too, we now have 
seen the lab community has come forward to say LDTs need to 
demonstrate analytical and clinical validity. Moderate- and 
high-risk LDTs need to be subject to premarket review. So those 
pieces, even the lab community has now said, you know what, 
that kind of a framework needs to be in place.
    Mr. Guthrie. The gentleman's time is expired. Would you 
again, Dr. Burgess, make--you had one unanimous consent request 
that we did not act on, then you went to a point of personal 
privilege. Can you make that once again before we move on?
    Mr. Burgess. Yes. It was to add to the record the statement 
from the Association of Molecular Pathology for the record.
    Mr. Guthrie. Without objection, so ordered.
    [The information appears at the conclusion of the hearing.]
    Mr. Guthrie. Thank you.
    Mr. Burgess. Thank you.
    Mr. Guthrie. The Chair now recognizes Mr. Sarbanes from 
Maryland for 5 minutes for questions.
    Mr. Sarbanes. Thank you, Mr. Chairman. I thank the two 
witnesses for being here.
    This is obviously a very important discussion, and I always 
try to look at these conversations through the eyes of sort of 
my constituents, people out there, and I have got to believe 
that if some of them are paying attention to this hearing, they 
would be scratching their head, the typical patient out there, 
and saying, you mean these kinds of protections and reviews and 
guidance and so forth are not already in place?
    And I understand that, you know, you start out in a 
different time period, and you are now trying to sort of update 
the framework that exists to protect patients out there, and I 
think the average person would think that this is a very 
reasonable undertaking on your part. So there is going to have 
to be some heightened degree of oversight and assurance in this 
arena.
    You have probably touched on all this before I came, but 
could you just take maybe two or three or four of the main 
categories of kind of constituencies out there and give me a 
shorthand on their perspective? What are physicians saying 
about this conversation? What are patient advocate groups 
saying? I think I can probably guess. You have alluded to the 
industry, the lab industry itself, but can you just--and in 
particular, I guess the physician perspective on it would be 
helpful to me, but if you can kind of shorthand those different 
lenses on this discussion.
    Dr. Shuren. We have heard mixed perspectives from the 
physician community. So oncologists have come out to say, yes, 
you need oversight, you need FDA oversight. Pathologists have 
felt that, no, we do not. FDA shouldn't be or should have 
little role in oversight of LDTs. The patient groups have been 
supportive of FDA. The consumer groups, payers have been--the 
medical device industry has been, the laboratory community has 
been split. Some of the labs have been working and promoting a 
proposal with FDA oversight, and the others have been proposing 
a system under CLIA.
    Mr. Sarbanes. The payers, that is interesting. Can you 
expand a little bit on that? Is that because they are seeing a 
lot of costs associated with faulty test results in the use of 
those?
    Dr. Conway. Maybe I will start since I am a large payer. It 
is a challenge in the payer aspect, so including in Medicare 
and similar and private payers. If the tests haven't gone 
through that FDA review, then we have a system of local 
contract medical directors in our national office also, you 
know, small numbers of people trying to review thousands of 
tests that either are identified to us or we identify that we 
need to assess reasonable and necessary for coverage. If there 
were an FDA review, you could potentially take a whole set of 
those that have been through FDA review and have those be 
covered and focus on the ones that are leftover. So this is an 
issue there.
    If you don't mind, on the practicing physician point, I am 
a practicing physician. I train residents and medical students 
on weekends as well. You know, you want an assurance as a 
physician that the test is clinically valid and that the report 
that says the patient has cancer or genetic disease X is 
correct. And the patient wants that assurance as well.
    Mr. Sarbanes. Right.
    Mr. Guthrie. Thank you. And the gentleman yields back his 
time.
    Mr. Lance of New Jersey is recognized for 5 minutes for 
questions.
    Mr. Lance. Thank you very much, Mr. Chairman. Good morning 
to you, gentlemen.
    Dr. Conway, it is my understanding that it is the Division 
of Laboratory Services within the Survey and Certification 
Group within the Center for Clinical Standards and Quality at 
CMS that has responsibility for administering the program. How 
many staff within the division are responsible for inspecting 
labs and reviewing the tests they performed?
    Dr. Conway. We have approximately 25 central office staff, 
and then we have approximately 110 surveyors across the Nation 
and all the States, so a small number per State.
    Mr. Lance. We have heard that there are tens of thousands 
of LDTs out there. Do you believe that the division is capable 
of reviewing all of these LDTs in a timely fashion for clinical 
validity?
    Dr. Conway. No, they are not, either in a timely fashion or 
with the current training of the staff that we have.
    Mr. Lance. And therefore, do you believe that new 
innovation would be effective, I presume, negatively because of 
the potential backlog?
    Dr. Conway. Yes, I would be very concerned about a 
potential backlog and the impact on innovation.
    Mr. Lance. Dr. Shuren, how would the FDA handle the 
workload and how would these submissions be based in line, on 
what priority if this were to be handled by the FDA?
    Dr. Shuren. To handle workload, it is one of the reasons we 
have put in a phased-in approach over a number of years, and 
review would occur--be prioritized based upon risk. What we 
proposed is we would start reviewing higher-risk devices before 
we would look at--high risk before moving to moderate risk.
    Mr. Lance. And to the best of your ability, how long do you 
believe it would take to review an LDT, your best estimate, 
Doctor?
    Dr. Shuren. So for the moderate risk LDTs now, the review 
times are--total times are a little over 100 days, thereabouts.
    Mr. Lance. Thank you. Thank you, Mr. Chairman. I yield back 
the balance of my time.
    Mr. Burgess. Would the gentleman yield to me?
    Mr. Lance. I yield to Dr. Burgess.
    Mr. Burgess. Thank you.
    Mr. Guthrie. Dr. Burgess is recognized.
    Mr. Burgess. On that point, could you state that figure 
again, the moderate-risk LDTs, 100 days? Is that right?
    Dr. Shuren. No, a little over 100 days.
    Mr. Burgess. So there are 11,000 and some laboratory-
developed tests. You said earlier that 50 percent are low risk, 
so presumably, that leaves 50 percent that are in the moderate- 
or high-risk category, is that correct?
    Dr. Shuren. Yes, that is correct.
    Mr. Burgess. So extrapolating out the number of days, 
assuming none of them are high risk, they are all low risk, and 
that is over 100 days of evaluation at the FDA, I mean that is 
a phenomenal amount of work that is ahead of you, is it not?
    Dr. Shuren. That is one of the reasons why we have looked 
at phased-in approach. We have gotten feedback, too, if we 
should consider any changes and take a different approach for 
some of the tests that are currently on the market, which we 
are doing. And we are also having those discussions about 
funding needs as part of user fee discussions, which are going 
on right now. They get authorized every 5 years. And that has 
been the natural course of business.
    We have those discussions with regulated industry--the 
laboratory community is that the table--to then talk about if 
people want to see a certain performance, what does that look 
like. We know in some of the proposals people have said for 
moderate-risk tests could that review time be 75 days? We can 
have a discussion about what it would take for review in 75 
days.
    Mr. Burgess. I will say some of the performance metrics 
that were introduced after the last FDA reauthorization in 
2012, I don't know that we ever got satisfactory answers back 
to this subcommittee or the Subcommittee on Oversight as to how 
the performance was on that, but there is a general unease that 
the FDA is able to perform its function in a timely fashion. 
During the time that we were doing the hearings for the FDA 
reauthorization, there was hardly a week that went by that 
there was not someone in my office with a tale of woe about a 
drug or device that just seemed to take forever in development 
and that the FDA would sometimes change the rules as that drug 
or device went through the development process. What assurance 
can we give to the laboratory-developed test community that 
they won't encounter similar problems with your agency going 
forward?
    Dr. Shuren. Well, our review times have been actually 
improving under MDUFA III. We are meeting our performance 
goals, as we committed to do. So we are seeing things move in 
the right direction. And I don't know what is happening to 
people coming into your office now. I have heard from other 
Members that they don't have the parade of people that were 
coming in several years ago.
    And you know when I took over the program several years 
ago, I was very upfront with this committee and others that 
there were challenges in the medical device program. We had 
seen roughly a decade of worsening performance, and we 
committed to turn that around. We committed to make changes 
regardless of what happened with MDUFA, and then MDUFA came 
along to give us additional resources. And we have continued to 
see improved performance, and we are going to continue to work 
on it, as we have been doing all along.
    Mr. Guthrie. Thank you.
    Mr. Burgess. I don't know that I share your enthusiasm. 
Thank you, Mr. Chairman.
    Mr. Guthrie. Thank you. The gentleman from New Jersey's 
time has expired, and recognizing Mr. Cardenas from California 
for 5 minutes for questions.
    Mr. Cardenas. Thank you very much, Mr. Chairman.
    My first question is to FDA and CMS. Today in your 
budgeting, are you being asked to do more with less?
    Dr. Shuren. Yes.
    Mr. Cardenas. I am not talking about prospectively. I am 
talking about in the cycle that you are currently in.
    Dr. Shuren. Yes, and that has been even the cycles before 
then. We are always asked to do more with less.
    Mr. Cardenas. OK.
    Dr. Conway. Yes. So it is also true for CMS, long history 
of doing more for less. Thank you for the more with less. Thank 
you for the question. I mean, I have managed in the delivery 
system. I have managed in CMS. I have never managed somewhere 
as hard as this. It is ridiculously harder than running a 
delivery system. And the reason is the amount of resources for 
the job. We are deploying lien and other operational techniques 
to increase our efficiency, learning from manufacturing and 
health systems, and that is working, but this is a major issue.
    Mr. Cardenas. OK. Thank you for clarifying that. I don't 
think the public understands how taxing it is for our agencies 
to continue to do more and more and more and try to protect the 
public and allow the American public to know or feel as though 
there are protections and the agencies are trying their best to 
look out for making sure that when they are engaged in 
something that is a--whether it is FDA-approved or it has gone 
through review of CMS, et cetera, that they can feel safe.
    So thank you for continuing to wade through the struggle of 
doing more with less and doing your best to keep up with that. 
I hope that we as the holders of the purse, Congress, will 
recognize that and realize that we are impeding on the safety 
of our American citizens when we just say no to a reasonable 
request of resources and we just say do with what you have and 
do a better job and just make it happen, easier said than done.
    And thank you for clarifying that in the environment that 
you have been and that this is probably the--I interpret that 
what you said is this is the most difficult environment for you 
to do justice to your efforts than any other environment you 
have been in.
    Dr. Conway. Yes. And I----
    Mr. Cardenas. And understand you are not coming across to 
me as complaining.
    Dr. Conway. No.
    Mr. Cardenas. I think it is important that you just be 
honest with us the way you have been. Thank you.
    Dr. Conway. Yes. No, do you mind if I----
    Mr. Cardenas. No, go ahead, please.
    Dr. Conway. This is the best job I have ever had and the 
most impactful, which is why I have stayed, but the challenge 
of managing in the CMS environment with the resources we have 
for the duty we have for the American people is by far the 
hardest job I have ever had. And I have led in the private 
sector, large groups, large budgets. It is nowhere close. This 
is the most challenging job I have ever had in my life.
    Mr. Cardenas. And again, I thank you for welcoming the 
challenge. It is too bad that we don't lessen that challenge by 
giving you the resources for you to be more effective without 
worrying about not being effective in your responsibilities.
    My family just got the news recently that my wife and I are 
going to be grandparents for the first time. And just the other 
day, we were invited to my daughter and my son-in-law's house, 
and they revealed to us it is going to be a boy. And the reason 
why they found is because my daughter underwent a test that 
went to a laboratory and the results came back. And one of the 
things that they could tell her--it wasn't the purpose of the 
test, but one of the things they could tell her is the gender 
of the fetus. And so it was a wonderful moment.
    However, what if the purpose of that test had been 
inadvertent, the results had been inadvertent? I think that is 
really what the focus of today is about. It is about safety of 
the public. It is about accuracy of what is going on out there. 
It is about whether or not they are being effective. And 
unfortunately, for those people who want less Government or no 
Government, there needs to be oversight from somewhere. I 
personally prefer that Government be involved in that oversight 
instead of just turning it completely over to private 
industries, which happens in some cases.
    But my question to you is, going forward, how do we as a 
country make sure that between CMS, between FDA, what your role 
can be in making sure that these critical tests, these 
laboratories are being accurate with the information to the 
patient, to the actual end-user?
    Dr. Shuren. Well, we have established a task force between 
FDA and CMS--NIH and CDC are also participating--to assure that 
we are not duplicating efforts. In fact, we have had 
conversations with certified laboratories, accrediting lab 
organizations under CLIA, two State licensure programs, and 
confirmed we are not duplicating efforts, but we do want to 
make sure we have good coordination moving forward. And we 
provide the education and information out for laboratories as 
well as we progress, so that work is happening in the task 
force right now.
    Dr. Conway. I agree.
    Mr. Cardenas. Thank you.
    Mr. Guthrie. They gentleman's time expired. And myself, and 
I think speaking for the entire subcommittee, we congratulate 
you on the good news and to your family, the next generation of 
your family, appreciate that very much.
    The Chair now recognizes Dr. Bucshon from Indiana for 5 
minutes for questions.
    Mr. Bucshon. Thank you, Mr. Chairman.
    Now, I have heard complaints that the FDA oversight of LDTs 
would interfere with the practice of medicine. I am a 
physician, cardiovascular surgeon, so I would like you both to 
comment as regulators but also as physicians on your view on 
that.
    Dr. Shuren. So we do not--and as a physician, we do not 
regulate the practice of medicine. Congress actually put in a 
statutory provision prohibiting us from regulating the practice 
of medicine in the medical device program. It is a unique 
provision that pertains to us. What we are regulating, talking 
about regulating, are the tests, the things that we regulate 
already today, reagents, the instruments, the protocol, 
instructions are used that go forward with it.
    In fact, a group of laboratories who were working with the 
device industry, the conventional IVD makers, when they sat 
down and went through it, they began to realize, too, you know 
what, there are parts here that is just like what happens in 
FDA: development, design, validation of tests. Then there are 
all these other activities that occur that are lab operations 
or the practice of medicine. They are not under our preview and 
we have never proposed to ever regulate those.
    Dr. Conway. And likewise, CLIA does not regulate the 
practice of medicine. It does regulate laboratories in terms of 
equipment, personnel, protocols, et cetera, which its focus is 
post-market laboratory by laboratory.
    Mr. Bucshon. OK. Great. And currently, CMS regulates how 
physicians operates a lab, as you described, and performs tests 
within it, but I really haven't heard any complaints about 
interfering with the practice of medicine myself from people 
that I know. Why is it that physicians feel differently maybe 
about the FDA oversight of these particular tests, and how can 
we address those concerns? Does that make sense?
    Dr. Shuren. It does. I think what we are dealing with are 
people who haven't dealt with us necessarily beforehand, and so 
it is new and they are assuming things that we don't believe to 
be the case. I hearken back to Dr. Burgess, who I respect very 
much, when he said when CLIA came in the door you were not a 
fan, but you began to realize the value of it. I would say the 
same thing here. As the lab community works with us--well, 
maybe you will allow it.
    Well, hopefully, we will see much the same here with the 
lab community in working with us. And it is our hope they will 
work with us to make sure that when we are doing things, it 
also best fits for their operations. Again, we both are 
committed and care about that those tests work.
    Mr. Bucshon. Yes. I mean I will just make a comment and I 
will--I mean, as a physician, obviously I want accuracy and 
patient safety to be at the top of the list, right? And again, 
I think Mr. Shimkus said I am not one that is generally for 
Government regulation, but I think in this area that, you know, 
this is a good discussion to be having on behalf of patients, 
and that I think the details and how things end up at the end 
are what are important. So I commend your hard work on trying 
to find the sweet spot as you go about your job.
    Thank you. I yield back.
    Mr. Burgess [presiding]. The Chair thanks the gentleman. 
The gentleman yields back.
    The Chair recognizes the gentleman from Florida, Mr. 
Bilirakis, 5 minutes for questions, please.
    Mr. Bilirakis. Thank you, Mr. Chairman. I appreciate it. 
And this question is for the panel.
    As we go forward with any new regulation--regulatory 
scheme--we need to balance the needs between consistency, 
accuracy, and innovation. In disease areas such as cancer, 
genetic testing is evolving rapidly, and I am sure you agree 
with that. Many major institutions today have developed their 
own gene panels that help diagnose or identify potential 
treatments for cancer patients.
    For example, in Tampa we have the Moffitt Cancer Center, 
the only NCI-designated Comprehensive Cancer Center in Florida. 
They have developed a TruSight Tumor Gene Set, which is used to 
identify lung and colon cancers that will benefit from targeted 
therapies. An advantage of the lab-developed tests is the 
ability to rapidly innovate current tests rather than the 
slower and expensive process of resubmitting to FDA for any 
changes. Centers such as Moffitt have the ability to innovate 
and rapidly improve their lab tests as fast as science evolves.
    Question: How can we resolve issues regarding consistency 
and accuracy and not stifle innovation in these labs and 
important healthcare institutions?
    Dr. Shuren. So one issue for consistency, certainly we 
don't--we were recommending not to have two duplicative systems 
out there, one under FDA, one under CMS, or we will have 
inconsistency. But then we have found that to assure 
consistency, we work with the community on trying to develop 
standards or in guidance so that, as we learn and the science 
evolves, we can have more of a common playing field of what 
performance should look like for certain kinds of tests. And 
that can help ensure consistency in terms of approach.
    Mr. Bilirakis. Thank you. Anyone else?
    Dr. Conway. I agree with Dr. Shuren, and I think, you know, 
from the CMS perspective, we think our strength is in that 
post-market review laboratory by laboratory on the 
qualifications, equipment, and personnel.
    Mr. Bilirakis. Thank you. Anyone else want to jump in? OK. 
Dr. Conway, since both the volume and complexity of lab-
developed tests on the market today have drastically increased 
in recent years, why hasn't CMS asked for these new 
authorities?
    Dr. Conway. So in terms of authorities we think FDA has a 
critical role in premarket review of clinical validity. We 
think CMS's role through CLIA really is and should be focused 
on laboratory-by-laboratory assessment, survey and 
certification and oversight of accrediting organizations, 
ensuring that the protocols, the equipment, and the standards 
are in place in individual laboratories in a post-market 
manner.
    Mr. Bilirakis. Dr. Conway, does CMS require definitive 
review of the clinical claims being made about the tests?
    Dr. Conway. And Dr. Shuren can certainly comment for FDA. 
On the CMS perspective, we do basic assessment of analytical 
validity so the analyte is the actual analyte in the test. We 
do not do assessments of clinical validity, meaning the test 
actually identifies the condition, the absence or presence of 
the condition it is supposed to identify.
    In our coverage process, we have occasionally looked at 
laboratory development tests for reasonable and necessary 
standard. There, we will look at the effect of the test on 
patients, but that is a very small number of LDTs we have 
looked at through that process.
    Mr. Bilirakis. Well, thank you very much, and I yield back, 
Mr. Chairman.
    Mr. Burgess. The Chair thanks the gentleman. The gentleman 
yields back.
    The Chair recognizes the gentleman from New York, Mr. 
Collins, 5 minutes for your questions, please.
    Mr. Collins. Thank you, Mr. Chairman.
    Before I start my questions, I would ask unanimous consent 
to enter into the record a letter from Roswell Park Cancer 
Institute.
    Mr. Burgess. Without objection.
    [The information appears at the conclusion of the hearing.]
    Mr. Collins. Thank you very much.
    In that letter they do mention the same we have talked 
about before, the Association of Molecular Pathology, or AMP, 
and their proposal, and I would certainly encourage the FDA, as 
we are in the discussion stage, to again take a look at this.
    So I guess one thing I am still trying to get clear on, you 
know, currently, a commercial test, something sold on the 
market which does have to go through FDA approval, premarket 
approval, and I assuming that is currently a 510(k)?
    Dr. Shuren. For premarket, most of them are 510(k). A very 
tiny number are PMA.
    Mr. Collins. So as a 510(k) currently, it is a medical 
device subject to the medical device tax, correct?
    Dr. Shuren. Yes.
    Mr. Collins. So if I understand what your guidance is now, 
you are going to move on IVDs out of that world, the medical 
device world, and have a different classification of class 1, 
2, or 3, or low, moderate, whatever. Just a question, does that 
mean on the good news side that IVDs will no longer be subject 
to the medical device tax since they are not going to be 
getting 510(k)s?
    Dr. Shuren. So the trigger for the device tax is 
registering at the listing with the FDA. What we have proposed 
for LDTs is that--and we use that to know what test is being 
made, who is making it, and that is a requirement by law. But 
we have worked through that instead they can give us a 
notification and not list with us, and particularly--and that 
is for starters. And for the tests that don't ultimately come 
in for premarket review, they also wouldn't end up registering 
and listing with us. And that would not trigger the device tax.
    Mr. Collins. So current tests would be still covered by the 
medical device tax even though there is not a 510(k) because 
they would be listed with the FDA?
    Dr. Shuren. They are registered and listed with that, and 
those are----
    Mr. Collins. OK. That clarification is important because I 
have heard that kind of going all over the board.
    Now, another, you know, concern has been, you know, 
accuracy of testing, and I think it is also important to make 
clear laboratory-developed tests are not sold to other 
facilities. They are used inside a facility such as Roswell 
Park, which is looking at very specific treatments for specific 
cancers and what we call personalized medicine. They are not 
then selling those tests to other folks or making claims, which 
is different than a commercialized test, which currently goes 
through FDA approvals.
    But, you know, Dr. Conway, it is my understanding that over 
97 percent of the CLIA laboratory test facilities have 
subjected themselves to outside third-party proficiency testing 
of their tests. Isn't that correct?
    Dr. Conway. So proficiency testing occurs in just 13 
specialty areas, occurs approximately three times per year. It 
has improved the accuracy over time. It will not assess for 
clinical validity of the test, so the premarket clinical 
validity, which Dr. Shuren spoke to, the proficiency testing 
does not analyze clinical validity.
    Mr. Collins. Well, it certainly analyzes whether you are 
properly getting--you know, you are identifying the antigen you 
are supposed to identify.
    Dr. Conway. So it will identify--if that laboratory-
developed test was within those 13 categories, which they are 
not all within those 13 areas, but for an LDT that was in one 
of those 13 areas--and Dr. Shuren may say more--it will detect 
that the analyte is the analyte, but that is not an assessment 
of the clinical usefulness or validity of the test.
    Dr. Shuren. And it goes to again, if you will, the accuracy 
of the performance of the test as opposed to the accuracy of 
the test itself, which is a different look, and that is what we 
look at for the test.
    Mr. Collins. Yes. I guess I would just say it is my belief 
anyway that the laboratory-developed tests, certainly in 
institutions like Roswell Park, are being done to get better 
treatment, quicker treatment to the patients. And a big concern 
all of us have, if this goes through, that a test might be used 
tomorrow to help a patient with cancer now is delayed 6 months 
as it goes through some kind of premarket review at FDA, which 
is a life-and-death situation for many of these cancers.
    And I think it goes back to--I think I go on to the same 
bandwagon as Mr. Barton and Dr. Burgess. This has not been a 
problem that I would identify, and putting any type of delay 
into this sphere of personalized medicine and treatment 
especially in the cancer and oncology world runs the risk 
frankly of causing people to die that don't need to die, 
treatments that could be given that would be delayed. And in 
the cancer world, delay is not a good thing.
    So personally, I would throw myself into the category I 
believe it is working now. We do have outside proficiency 
testing, third-party testing. And we have to remember these are 
laboratory-developed tests that are not being sold in the 
marketplace to other facilities, which is very different than 
what you are doing now.
    My time has expired, but if the Chair would like to hear a 
response, I would certainly yield a couple seconds.
    Mr. Burgess. Sure. He is recognized for a response.
    Dr. Shuren. I truly appreciate those comments because we do 
not want to stifle innovation in this place. We want to have 
patients get timely access to tests. And that is why even under 
an FDA mechanism if the test is being reviewed, it is being 
developed, it can still be made available to patients on an 
investigational basis or for compassionate use. But there at 
least you are telling the doctor and you are telling the 
patient we haven't validated this test yet. It is 
investigational. This may be----
    Mr. Collins. You know what, I appreciate that because my 
worry was they would develop a test, they couldn't use it until 
they had approval, but if on an investigative basis they know 
that--they think they have a good test they can use it, then 
you have actually helped me in a couple of ways there. Thank 
you for that response.
    I yield back.
    Mr. Burgess. The Chair thanks the gentleman. The gentleman 
yields back.
    The Chair recognizes the gentleman from Pennsylvania, Mr. 
Murphy, 5 questions for questions, please.
    Mr. Murphy. Thank you. Doctor, Doctor, it good to have you 
here, appreciate this.
    I want to pivot a little bit here to talk about piecing 
together post-market and premarket analysis to look at this, 
and in particular, a couple of devices used in women's health 
care, one is called a morcellator. Are you familiar with the 
morcellator, a device that is supposed to shred tumors, et 
cetera, but has been associated with complications in women in 
terms of actually spreading cancer for them?
    Now, it has been on the market for 20-plus years, and the 
FDA admitted for the first time it became aware of the safety 
issue with power morcellators was after December of 2013, 
correspondence from a physician citing the case of a family 
member. This is someone who had just recently had another 
surgery to remove another recurrence of cancer that was spread 
it by the morcellator.
    The manufacturer was apparently aware of the dangers of 
this device as early as 2006 based upon a report from Dr. 
Lamparter, a pathologist from central Pennsylvania, who cited 
about 1 out of 300 samples of morcellated tissue from his 
hospital had evidence of a hidden cancer, which is morcellated.
    So my question is did the FDA have any evidence of these 
dangers in 2006 or prior to that? Are you aware of this 
problem?
    Dr. Shuren. So in the past the thought was what risks of 
cancer there may be for a fibroma--for a fibroid actually to 
have cancer in there--were significantly less. And one of the 
things when we looked into it more recently we came to a 
different conclusion, that the likelihood of cancer is higher. 
There is still disagreement in the community because, as you 
know, the healthcare professional societies disagree. They 
think we have overestimated the risk of cancer.
    We said we have a different perspective, and that is why we 
went out and we put contraindications and warnings on the use 
of that device, that it should only be used in a more limited 
set or offered as an option in a limited set of women and think 
about primarily women who, in the absence of using the device, 
would no longer be able to bear children but they want to bear 
children. And we felt those cases the risk of the cancer is 
very low. They should have the opportunity to weigh in, but we 
scaled back dramatically how that should----
     Mr. Murphy. So this is a case where the science available, 
the premarket analysis has changed, and what is being used in 
the data, you have a mechanism to go forward on this and make 
some changes. Let me ask another question.
    Brigham and Women's Hospital was aware of the dangers in 
2012. A patient by the name of Mrs. Erica Katz was seriously 
injured in 2012 by the device and then died in 2013 according 
to reports. I wondered, do you know if that hospital reported 
that to the FDA? Would you know?
    Dr. Shuren. I am not aware that----
    Mr. Murphy. Is there a mechanism where the hospital is 
supposed to report that or the manufacturer is supposed to 
report that so you can do the analysis?
    Dr. Shuren. So user facilities have certain requirements 
for reporting. So do manufacturers if they become aware of 
certain events. And what I can tell you is we have been looking 
into those concerns that have been raised regarding reporting.
    Mr. Murphy. OK. In response to congressional inquiries 
about this, the FDA admitted that the 1-out-of-350 risk does 
not address other types of malignancies, which you would add to 
that risk, you said. They went on to say the FDA also 
identified studies showing that morcellated patients had worse 
outcomes than patients who had not undergone morcellation.
    So this is more than just the issue with just a fibroid or 
if it is cancerous. It is also a question of outcomes. Is this 
something that the FDA is reviewing also with regard to their 
stamp of approval on these things in terms of the outcome 
measures?
    Dr. Shuren. So in terms of the tests we have looked at, we 
think where we have constrained it right now is--for use is 
where the benefits outweigh the risks, but we are continuing to 
look at new data as it arises, and if so, we will act 
accordingly.
    Mr. Murphy. Thank you. There is another issue in women's 
health that was brought to my attention. It is a product called 
Essure. It is a permanent birth control device that went 
through FDA's rigorous premarket approval process. Yet despite 
getting the agency's approval, it has been linked to at least 
four deaths and deaths of five unborn children. Apparently, a 
total of 24,000 women have come forward claiming that they have 
been harmed by this device. And so the question is how it 
remains on the market with a potential for problems. And 
because this has the FDA stamp of approval, these women feel at 
this point they cannot take their cases forward, and they feel 
they don't have any recourse. Is the FDA also reviewing this 
issue as well as far as you know?
    Dr. Shuren. We are. In fact, we held an advisory committee 
meeting a few weeks ago at our behest to give an opportunity to 
put what new evidence is on the table to assure that people who 
wanted to raise concerns about it had an opportunity to provide 
those concerns. And we are now currently reviewing the feedback 
we received from the advisory committee, as well as what we 
have heard from other people, as well as the state of the 
evidence, and we will come out with our conclusions on that to 
the public.
    Mr. Murphy. Thank you. And as this goes through, since this 
hearing has been a lot about premarket analysis, what this 
comes down to is I just want to make sure that we are aware of 
what mechanism you have, because I understand the science of 
1996 is different from the science of 2015 and our knowledge 
base, but to have an ongoing mechanism for review and changes, 
devices and getting information there and looking at those 
things, I mean, I am glad you had some hearings on this, but I 
would certainly like to know that that is part of the system.
    I am out of time, but I look forward to hearing your 
comments on that in the future. Thank you.
    Mr. Burgess. The gentleman yields back. The Chair thanks 
the gentleman.
    The Chair recognizes the gentleman from Texas for a 
unanimous consent request.
    Mr. Green. Mr. Chairman, I ask unanimous consent to place 
in the record ``Public Health Evidence for FDA Oversight of 
Laboratory Developed Tests: 20 Case Studies.'' I ask unanimous 
consent to place that in the record.
    Mr. Burgess. Without objection, so ordered.\1\
---------------------------------------------------------------------------
    \1\ The information has been retained in committee files and also 
is available at  http://docs.house.gov/meetings/IF/IF14/20151117/
104127/HMTG-114-IF14-20151117-SD009.
---------------------------------------------------------------------------
    Mr. Burgess. And I recognize myself for an additional 
unanimous consent request to add the statement of the American 
Association of Clinical Chemistry to the record. Without 
objection, so ordered.
    [The information appears at the conclusion of the hearing.]
    Mr. Burgess. Seeing no further Members wishing to be 
recognized for questions, I do want to remind Members they have 
10 business days to submit questions for the record, and I ask 
the witnesses to respond to those questions promptly. Members 
should submit their questions by the close of business on 
December 2.
    With that, the subcommittee stands in adjournment.
    Dr. Shuren. Thank you.
    [Whereupon, at 12:07 p.m., the subcommittee was adjourned.]
    [Material submitted for inclusion in the record follows:]

                 Prepared statement of Hon. Fred Upton

    The 21st Century Cures Act passed this committee 51-0 and 
was through the House in July with 344 votes. It was the 
product of over a year's worth of ideas Members received at 
hearings, roundtables in DC and across the country. Provisions 
were proposed and fleshed out with the help of a wide variety 
of stakeholders, in and out of Government, of all political 
stripes. It goes without saying that for any piece of 
comprehensive legislation to garner these vote totals, 
compromise is critical and the perfect can't become the enemy 
of the good. It also goes without saying that some important 
pieces of the puzzle didn't get included because the timing 
just wasn't right. Modernizing our regulatory framework for the 
review and oversight of diagnostics is one of those pieces.
    As I said at our first forum on this topic in July 2014, 
these increasingly important and complex tests are providing 
researchers and clinicians with valuable tools to match the 
right patients with the right treatments. We must ensure that 
our laws and regulations keep pace so that innovation in this 
space continues and patients benefit from accurate and reliable 
tests.
    I saw Cures as a unique opportunity to elicit feedback on 
what such a framework should look like and what role Congress 
could play in developing it. We issued a white paper asking 
targeted questions and were overwhelmed with the scope and 
thoroughness of the responses we received. We realized early on 
that the traditional medical device framework was not ideally 
suited for these unique tests, which provide clinicians with 
critical information but do not actually provide therapy to a 
patient.
    It was also apparent that there was quite a difference of 
opinion about what the roles and responsibilities of FDA and 
CMS should be. Developing legislative language with broad 
support on an abbreviated timeframe was not achievable. I told 
my staff to table these discussions until we got Cures through 
the House but to urge stakeholders to use the time to forge 
ahead and find as much common ground as possible.
    I was very encouraged to hear that a diverse group of 
stakeholders with different points of view came together and, 
in the spirit of finding consensus, developed a draft framework 
that answered a lot of our questions in a responsible, balanced 
manner. Of course there is room for improvement, but folks need 
to be realistic in their approach and pragmatic with their 
suggestions if the ultimate goal is a bill signed into law any 
time soon. We must get this right and we need everyone's help 
in order to do so.

              Prepared statement of Hon. Renee L. Ellmers

    Earlier this year, Democrats and Republicans on the House 
Energy and Commerce Committee worked together to develop the 
21st Century Cures legislation. I was proud to work with my 
colleagues on that landmark initiative in order to reduce 
regulation, inspire innovation, improve outcomes for patients 
and move our country towards precision medicine. Further, that 
legislation helped highlight the increased importance of 
diagnostics in modern health care.
    Today, diagnostics play a critical role in the rapid 
detection and diagnosis of diseases. Diagnostics help identify 
targeted, effective and often less invasive treatments-
ultimately leading to reduced costs to both patients and the 
Government.
    The Committee's current discussion draft legislation 
follows the work of 21st Century Cures and focuses on the 
future of diagnostics. It would advance innovation, protect 
patients, provide a predictable and timely path to market and 
avoid duplicative regulation. It does this by tailoring an 
appropriate role for the FDA (outside of the medical device 
framework) to oversee diagnostic test development activities, 
while modernizing CLIA oversight of separate and distinct 
laboratory operation activities.
    Without this legislation, I am concerned that the FDA would 
finalize guidance to regulate laboratory developed tests as 
medical devices, which could impact many stakeholders. This 
guidance may lead to costly litigation and uncertainty or could 
hamper innovation and patient access to critical diagnostic 
tests.
    Also, I am deeply concerned that this guidance would result 
in the medical device tax being imposed on laboratories. Dr. 
Shuren confirmed during the hearing in September 2014, that 
under the FDA's guidance, labs would ultimately be directly 
subject to this medical device tax. Labs already pay the tax 
indirectly when they purchase test kits from manufacturers, so 
under the guidance they would unfairly pay the same tax twice.
    I have many stakeholders in my district, so this discussion 
draft legislation represents a good compromise between the 
CLIA-centric approach and the medical device framework laid out 
in the FDA guidance. In addition, my district is home to many 
veterans and military families who rely on TRICARE for their 
health care, so ensuring market stability and access to these 
crucial tests directly affects my constituents.
    I am thankful for stakeholder engagement in finding a 
legislative solution that provides a feasible alternative to 
FDA's draft guidance. I stand prepared to work with the 
chairman and ranking member in order to accomplish this goal.

[GRAPHICS NOT AVAILABLE IN TIFF FORMAT] 

                                 [all]