[Congressional Record Volume 153, Number 62 (Wednesday, April 18, 2007)]
[Senate]
[Pages S4694-S4699]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

      By Mr. DODD (for himself, Mr. Kennedy, Mr. Harkin, Mr. Bingaman, 
        Mrs. Murray, Mrs. Clinton, and Mr. Brown):
  S. 1156. A bill to amend the Federal Food, Drug, and Cosmetic Act to 
reauthorize the Best Pharmaceuticals for Children program; to the 
Committee on Health, Education, Labor, and Pensions.
  Mr. DODD. Mr. President, I rise today to introduce the Best 
Pharmaceuticals for Children Amendments of 2007, which is a bill to 
reauthorize the Best Pharmaceuticals for Children Act--BPCA. If 
Congress doesn't act, this successful program will expire on October I, 
2007. I thank my colleagues Senators Kennedy, Harkin, Bingaman, Murray, 
Clinton and Brown who are joining me as original cosponsors of this 
important legislation.

[[Page S4695]]

  I am pleased that Senators Kennedy and Enzi, the distinguished 
chairman and ranking member of the Health Education Labor, and 
Pensions--HELP--Committee, have included this bill in the chairman's 
mark for S. 1082, which is expected to be voted on today in the HELP 
Committee.
  I would also like to recognize the contributions and leadership of 
former Senator Mike De Wine, a friend and colleague, who always fought 
to ensure children would not be treated as second-class citizens when 
it came to drug and device development. He was a champion of BPCA along 
with me even when it wasn't popular to hold that view.
  The story of the Best Pharmaceuticals for Children Act is one of huge 
success for children and their families. Children with a wide range of 
diseases such as HIV/AIDS, cancer, allergies, asthma, neurological and 
psychiatric disorders, and obesity can now lead healthier, more 
productive lives as a result of new information about the safety and 
efficacy of drugs they use to treat and manage their diseases where 
previously there was none.
  Children are not simply little adults and results of the drug studies 
conducted under the BPCA have shown us that they should not be treated 
as such. Pediatric drug studies conducted. under the BPCA showed that 
children may have been exposed to ineffective drugs, ineffective 
dosing, overdosing, or side effects that were previously unknown.
  Since the BPCA's passage in 1997 and its reauthorization in 2002, FDA 
has requested nearly 800 studies involving more than 45,000 children in 
clinical trials. Useful new pediatric information is now part of 
product labeling for 119 drugs. By comparison, in the 7 years prior to 
the BPCA's passage, only 11 studies of marketed drugs were completed. 
In the past 10 years, there has been a twentyfold increase in the 
number of drugs studied in infants, children, and adolescents since 
BPCA was enacted.
  Labeling changes resulting from clinical studies under the BPCA have 
informed physicians of the proper dosing in the examples of Viracept, a 
protease inhibitor used in a combination therapy for the treatment of 
HIV, and Neurontin, a pain relief medication used to treat children 
with chronic pain. For children with epilepsy, the BPCA studies 
informed physicians that the drugs Keppra and Trileptal could be used 
safely and effectively at an even earlier age than previously known. 
Studies of Imitrex as a result of the BPCA showed no better results 
than placebo for the treatment of migraine headaches in adolescents. 
These same studies also showed serious adverse events due to Imitrex in 
pediatric populations and therefore the drug is not recommended to 
treat migraines in anyone less than 18 years of age.
  Recent studies of the BPCA by the Government Accountability Office--
GAO--and by several authors from Duke University in an article which 
appeared in the Journal of the American Medical Association--JAMA--have 
demonstrated that the program is a success and have identified 
opportunities to strengthen the program. Authors of the recent JAMA 
article found that outside of the BPCA, the FDA is limited in the 
number and scope of studies for which it can require pediatric data for 
existing products on the market.
  Data from this article showed that only a minority of drugs studied 
under the BPC, about 20 percent, had more than $1 billion in annual 
sales. In fact, the median drug granted exclusivity was a small-market 
drug with annual sales of $180 million and 30 percent of drugs studied 
had sales less than $200 million. This article went on to say that a 
universal reduction in the length of pediatric exclusivity from 6 to 3 
months would mean that products with small profit margins may not be 
submitted for pediatric testing.
  The BPCA has always tried to strike the right balance between cost to 
consumers and benefits to children. I believe there is an ongoing need 
to evaluate the cost of the incentive as it relates to reaching the 
goal of having medications properly studied and labeled for children. 
In fact, that is why I strongly support a 5-year sunset of the BPCA.
  After 10 years, experience and data has shown us that for a small 
number of drugs, pediatric exclusivity has far exceeded the ``carrot'' 
it was intended to provide for manufacturers. As the authors of the 
recent JAMA article noted, ``our study shows that the Pediatric 
Exclusivity Program overcompensates blockbuster products for performing 
clinical trials in children, while other products have more modest 
returns on investment under this program.''
  The bill I am introducing today contains a reasonable, workable 
proposal to address cost concerns without jeopardizing the 
extraordinary success of BPCA. I have worked closely with the chairman 
and ranking member of the HELP Committee to craft this proposal into 
the form it appears in this legislation and in the bipartisan 
chairman's mark which is expected to be voted on in the HELP Committee 
today.
  On March 27, the HELP Committee held a hearing, which I chaired, 
entitled ``Ensuring Safe Medicines and Medical Devices for Children.'' 
We learned from pediatricians and a parent of five children, four of 
whom are HIV-positive, Mrs. Susan Belfiore, about the tremendous impact 
BPCA has had on the quality of life for countless numbers of children 
and their families. We received testimony with many suggestions for 
improvements to BPCA which I believe are reflected in this bill. I 
would also add that in the month since I circulated this bill as a 
draft, I received comments from several pharmaceutical companies. Some 
have been strongly supportive of this effort and many of their ideas 
and suggestions are incorporated in this bill.
  The success of the BPCA has transformed the drug development process 
for children. It is my hope that we will achieve similar success with 
another piece of legislation I recently introduced called the Pediatric 
Medical Device Safety and Improvement Act. It is also contained within 
the chairman's mark to S. 1082 and I thank Chairman Kennedy and Ranking 
Member Enzi for working with me to ensure that medical devices used in 
children are safe and are designed specifically for their use.
  The BPCA has had a long history of bipartisan support and it has been 
my longstanding hope that this initiative will continue to be 
bipartisan as the chairman's mark to S. 1082 moves to the Senate floor. 
The safety of our Nation's children is not a partisan issue.
  As the parent of two young children, I know that it is essential that 
products used in children's growing bodies, whether they be drugs or 
devices, are appropriately tested and designed specifically for their 
use. We must continue the tremendous success of BPCA and its 
complementary program, the Pediatric Research Improvement Act, of which 
I am an original cosponsor, by strengthening both programs through the 
reauthorization process this year. It is essential that we use the past 
experience of both programs to ensure they will continue to thrive in 
the future.
  I urge my colleagues to support this legislation.
  I ask unanimous consent that the text of the bill be printed in the 
Record.
  There being no objection, the text of the bill was ordered to be 
printed in the Record, as follows:

                                S. 1156

       Be it enacted by the Senate and House of Representatives of 
     the United States of America in Congress assembled,

     SECTION 1. SHORT TITLE.

       This Act may be cited as the ``Best Pharmaceuticals for 
     Children Amendments of 2007''.

     SEC. 2. PEDIATRIC STUDIES OF DRUGS.

       (a) In General.--Section 505A of the Federal Food, Drug, 
     and Cosmetic Act (21 U.S.C. 355a) is amended--
       (1) in subsection (a), by inserting before the period at 
     the end the following: ``, and, at the discretion of the 
     Secretary, may include preclinical studies'';
       (2) in subsection (b)--
       (A) in paragraph (1)(A)(i), by striking ``(D)'' both places 
     it appears and inserting ``(E)'';
       (B) in paragraph (1)(A)(ii), by striking ``(D)'' and 
     inserting ``(E)'';
       (C) by striking ``(1)(A)(i)'' and inserting ``(A)(i)(I)'';
       (D) by striking ``(ii) the'' and inserting ``(II) the'';
       (E) by striking ``(B) if the drug is designated'' and 
     inserting ``(ii) if the drug is designated'';
       (F) by striking ``(2)(A)'' and inserting ``(B)(i)'';
       (G) by striking ``(i) a listed patent'' and inserting ``(I) 
     a listed patent'';
       (H) by striking ``(ii) a listed patent'' and inserting 
     ``(II) a listed patent'';

[[Page S4696]]

       (I) by striking ``(B) if the drug is the subject'' and 
     inserting ``(ii) if the drug is the subject'';
       (J) by striking ``If'' and all that follows through 
     ``subsection (d)(3)'' and inserting the following:
       ``(1) In general.--Except as provided in paragraph (2), if, 
     prior to approval of an application that is submitted under 
     section 505(b)(1), the Secretary determines that information 
     relating to the use of a new drug in the pediatric population 
     may produce health benefits in that population, the Secretary 
     makes a written request for pediatric studies (which shall 
     include a timeframe for completing such studies), the 
     applicant agrees to the request, such studies are completed 
     using appropriate formulations for each age group for which 
     the study is requested within any such timeframe and the 
     reports thereof are submitted and accepted in accordance with 
     subsection (d)(3), and if the Secretary determines that 
     labeling changes are appropriate, such changes are made 
     within the timeframe requested by the Secretary--''; and
       (K) by adding at the end the following:
       ``(2) Exception.--The Secretary shall not extend the period 
     referred to in paragraph (1)(A) or in paragraph (1)(B) later 
     than 9 months prior to the expiration of such period.'';
       (3) in subsection (c)--
       (A) in paragraph (1)(A)(i), by striking ``(D)'' both places 
     it appears and inserting ``(E)'';
       (B) in paragraph (1)(A)(ii), by striking ``(D)'' and 
     inserting ``(E)'';
       (C) by striking ``(1)(A)(i)'' and inserting ``(A)(i)(I)'';
       (D) by striking ``(ii) the'' and inserting ``(II) the'';
       (E) by striking ``(B) if the drug is designated'' and 
     inserting ``(ii) if the drug is designated'';
       (F) by striking ``(2)(A)'' and inserting ``(B)(i)'';
       (G) by striking ``(i) a listed patent'' and inserting ``(I) 
     a listed patent'';
       (H) by striking ``(ii) a listed patent'' and inserting 
     ``(II) a listed patent'';
       (I) by striking ``(B) if the drug is the subject'' and 
     inserting ``(ii) if the drug is the subject'';
       (J) by striking ``If'' and all that follows through 
     ``subsection (d)(3)'' and inserting the following:
       ``(1) In general.--Except as provided in paragraph (2), if 
     the Secretary determines that information relating to the use 
     of an approved drug in the pediatric population may produce 
     health benefits in that population and makes a written 
     request to the holder of an approved application under 
     section 505(b)(1) for pediatric studies (which shall include 
     a timeframe for completing such studies), the holder agrees 
     to the request, such studies are completed using appropriate 
     formulations for each age group for which the study is 
     requested within any such timeframe and the reports thereof 
     are submitted and accepted in accordance with subsection 
     (d)(3), and if the Secretary determines that labeling changes 
     are appropriate, such changes are made within the timeframe 
     requested by the Secretary--''; and
       (K) by adding at the end the following:
       ``(2) Exception.--The Secretary shall not extend the period 
     referred to in paragraph (1)(A) or in paragraph (1)(B) later 
     than 9 months prior to the expiration of such period.'';
       (4) by striking subsection (d) and inserting the following:
       ``(d) Conduct of Pediatric Studies.--
       ``(1) Request for studies.--
       ``(A) In general.--The Secretary may, after consultation 
     with the sponsor of an application for an investigational new 
     drug under section 505(i), the sponsor of an application for 
     a new drug under section 505(b)(1), or the holder of an 
     approved application for a drug under section 505(b)(1), 
     issue to the sponsor or holder a written request for the 
     conduct of pediatric studies for such drug. In issuing such 
     request, the Secretary shall take into account adequate 
     representation of children of ethnic and racial minorities. 
     Such request to conduct pediatric studies shall be in writing 
     and shall include a timeframe for such studies and a request 
     to the sponsor or holder to propose pediatric labeling 
     resulting from such studies.
       ``(B) Single written request.--A single written request--
       ``(i) may relate to more than 1 use of a drug; and
       ``(ii) may include uses that are both approved and 
     unapproved.
       ``(2) Written request for pediatric studies.--
       ``(A) Request and response.--
       ``(i) In general.--If the Secretary makes a written request 
     for pediatric studies (including neonates, as appropriate) 
     under subsection (b) or (c), the applicant or holder, not 
     later than 180 days after receiving the written request, 
     shall respond to the Secretary as to the intention of the 
     applicant or holder to act on the request by--

       ``(I) indicating when the pediatric studies will be 
     initiated, if the applicant or holder agrees to the request; 
     or
       ``(II) indicating that the applicant or holder does not 
     agree to the request and the reasons for declining the 
     request.

       ``(ii) Disagree with request.--If, on or after the date of 
     enactment of the Best Pharmaceuticals for Children Amendments 
     of 2007, the applicant or holder does not agree to the 
     request on the grounds that it is not possible to develop the 
     appropriate pediatric formulation, the applicant or holder 
     shall submit to the Secretary the reasons such pediatric 
     formulation cannot be developed.
       ``(B) Adverse event reports.--An applicant or holder that, 
     on or after the date of enactment of the Best Pharmaceuticals 
     for Children Amendments of 2007, agrees to the request for 
     such studies shall provide the Secretary, at the same time as 
     submission of the reports of such studies, with all 
     postmarket adverse event reports regarding the drug that is 
     the subject of such studies and are available prior to 
     submission of such reports.
       ``(3) Meeting the studies requirement.--Not later than 180 
     days after the submission of the reports of the studies, the 
     Secretary shall accept or reject such reports and so notify 
     the sponsor or holder. The Secretary's only responsibility in 
     accepting or rejecting the reports shall be to determine, 
     within the 180 days, whether the studies fairly respond to 
     the written request, have been conducted in accordance with 
     commonly accepted scientific principles and protocols, and 
     have been reported in accordance with the requirements of the 
     Secretary for filing.
       ``(4) Effect of subsection.--Nothing in this subsection 
     alters or amends section 301(j) of this Act or section 552 of 
     title 5 or section 1905 of title 18, United States Code.'';
       (5) by striking subsections (e) and (f) and inserting the 
     following:
       ``(e) Notice of Determinations on Studies Requirement.--
       ``(1) In general.--The Secretary shall publish a notice of 
     any determination, made on or after the date of enactment of 
     the Best Pharmaceuticals for Children Amendments of 2007, 
     that the requirements of subsection (d) have been met and 
     that submissions and approvals under subsection (b)(2) or (j) 
     of section 505 for a drug will be subject to the provisions 
     of this section. Such notice shall be published not later 
     than 30 days after the date of the Secretary's determination 
     regarding market exclusivity and shall include a copy of the 
     written request made under subsection (b) or (c).
       ``(2) Identification of certain drugs.--The Secretary shall 
     publish a notice identifying any drug for which, on or after 
     the date of enactment of the Best Pharmaceuticals for 
     Children Amendments of 2007, a pediatric formulation was 
     developed, studied, and found to be safe and effective in the 
     pediatric population (or specified subpopulation) if the 
     pediatric formulation for such drug is not introduced onto 
     the market within 1 year of the date that the Secretary 
     publishes the notice described in paragraph (1). Such notice 
     identifying such drug shall be published not later than 30 
     days after the date of the expiration of such 1 year period.
       ``(f) Internal Review of Written Requests and Pediatric 
     Studies.--
       ``(1) Internal review.--
       ``(A) In general.--The Secretary shall create an internal 
     review committee to review all written requests issued and 
     all reports submitted on or after the date of enactment of 
     the Best Pharmaceuticals for Children Amendments of 2007, in 
     accordance with paragraphs (2) and (3).
       ``(B) Members.--The committee under subparagraph (A) shall 
     include individuals, each of whom is an employee of the Food 
     and Drug Administration, with the following expertise:
       ``(i) Pediatrics.
       ``(ii) Biopharmacology.
       ``(iii) Statistics.
       ``(iv) Drugs and drug formulations.
       ``(v) Legal issues.
       ``(vi) Appropriate expertise pertaining to the pediatric 
     product under review.
       ``(vii) One or more experts from the Office of Pediatric 
     Therapeutics, including an expert in pediatric ethics.
       ``(viii) Other individuals as designated by the Secretary.
       ``(2) Review of written requests.--All written requests 
     under this section shall be reviewed and approved by the 
     committee established under paragraph (1) prior to being 
     issued.
       ``(3) Review of pediatric studies.--The committee 
     established under paragraph (1) shall review all studies 
     conducted pursuant to this section to determine whether to 
     accept or reject such reports under subsection (d)(3).
       ``(4) Tracking pediatric studies and labeling changes.--The 
     committee established under paragraph (1) shall be 
     responsible for tracking and making available to the public, 
     in an easily accessible manner, including through posting on 
     the website of the Food and Drug Administration--
       ``(A) the number of studies conducted under this section;
       ``(B) the specific drugs and drug uses, including labeled 
     and off-labeled indications, studied under this section;
       ``(C) the types of studies conducted under this section, 
     including trial design, the number of pediatric patients 
     studied, and the number of centers and countries involved;
       ``(D) the number of pediatric formulations developed and 
     the number of pediatric formulations not developed and the 
     reasons such formulations were not developed;
       ``(E) the labeling changes made as a result of studies 
     conducted under this section;
       ``(F) an annual summary of labeling changes made as a 
     result of studies conducted under this section for 
     distribution pursuant to subsection (k)(2); and
       ``(G) information regarding reports submitted on or after 
     the date of enactment of the Best Pharmaceuticals for 
     Children Amendments of 2007.'';
       (6) in subsection (g)--

[[Page S4697]]

       (A) in paragraph (1)--
       (i) by striking ``(c)(1)(A)(ii)'' and inserting 
     ``(c)(1)(A)(i)(II)''; and
       (ii) by striking ``(c)(2)'' and inserting ``(c)(1)(B)'';
       (B) in paragraph (2), by striking ``(c)(1)(B)'' and 
     inserting ``(c)(1)(A)(ii)'';
       (C) by redesignating paragraphs (1) and (2) as 
     subparagraphs (A) and (B), respectively;
       (D) by striking ``Limitations.--A drug'' and inserting 
     ``Limitations.--
       ``(1) In general.--Notwithstanding subsection (c)(2), a 
     drug''; and
       (E) by adding at the end the following:
       ``(2) Exclusivity adjustment.--
       ``(A) Adjustment.--
       ``(i) In general.--With respect to any drug, if the 
     organization designated under subparagraph (B) notifies the 
     Secretary that the combined annual gross sales for all drugs 
     with the same active moiety exceeded $1,000,000,000 in any 
     calendar year prior to the time the sponsor or holder agrees 
     to the initial written request pursuant to subsection (d)(2), 
     then each period of market exclusivity deemed or extended 
     under subsection (b) or (c) shall be reduced by 3 months for 
     such drug.
       ``(ii) Determination.--The determination under clause (i) 
     of the combined annual gross sales shall be determined--

       ``(I) taking into account only those sales within the 
     United States; and
       ``(II) taking into account only the sales of all drugs with 
     the same active moiety of the sponsor or holder and its 
     affiliates.

       ``(B) Designation.--The Secretary shall designate an 
     organization other than the Food and Drug Administration to 
     evaluate whether the combined annual gross sales for all 
     drugs with the same active moiety exceeded $1,000,000,000 in 
     a calendar year as described in subparagraph (A). Prior to 
     designating such organization, the Secretary shall determine 
     that such organization is independent and is qualified to 
     evaluate the sales of pharmaceutical products. The Secretary 
     shall re-evaluate the designation of such organization once 
     every 3 years.
       ``(C) Notification.--Once a year at a time designated by 
     the Secretary, the organization designated under subparagraph 
     (B) shall notify the Food and Drug Administration of all 
     drugs with the same active moiety with combined annual gross 
     sales that exceed $1,000,000,000 during the previous calendar 
     year.''.
       (7) in subsection (i)--
       (A) in the heading, by striking ``Supplements'' and 
     inserting ``Changes'';
       (B) in paragraph (1)--
       (i) in the heading, by inserting ``applications and'' after 
     ``pediatric'';
       (ii) by inserting ``application or'' after ``Any'';
       (iii) by striking ``change pursuant to a report on a 
     pediatric study under'' and inserting ``change as a result of 
     any pediatric study conducted pursuant to''; and
       (iv) by inserting ``application or'' after ``to be a 
     priority''; and
       (C) in paragraph (2)(A), by--
       (i) striking ``If the Commissioner'' and inserting ``If, on 
     or after the date of enactment of the Best Pharmaceuticals 
     for Children Amendments of 2007, the Commissioner''; and
       (ii) striking ``an application with'' and all that follows 
     through ``on appropriate'' and inserting ``the sponsor and 
     the Commissioner have been unable to reach agreement on 
     appropriate'';
       (8) by striking subsection (m);
       (9) by redesignating subsections (j), (k), (l), and (n), as 
     subsections (k), (m), (o), and (p), respectively;
       (10) by inserting after subsection (i) the following:
       ``(j) Other Labeling Changes.--If, on or after the date of 
     enactment of the Best Pharmaceuticals for Children Amendments 
     of 2007, the Secretary determines that a pediatric study 
     conducted under this section does or does not demonstrate 
     that the drug that is the subject of the study is safe and 
     effective, including whether such study results are 
     inconclusive, in pediatric populations or subpopulations, the 
     Secretary shall order the labeling of such product to include 
     information about the results of the study and a statement of 
     the Secretary's determination.'';
       (11) in subsection (k), as redesignated by paragraph (9)--
       (A) in paragraph (1)--
       (i) by striking ``a summary of the medical and'' and 
     inserting ``the medical, statistical, and''; and
       (ii) by striking ``for the supplement'' and all that 
     follows through the period and inserting ``under subsection 
     (b) or (c).'';
       (B) by redesignating paragraph (2) as paragraph (3); and
       (C) by inserting after paragraph (1) the following:
       ``(2) Dissemination of information regarding labeling 
     changes.--Beginning on the date of enactment of the Best 
     Pharmaceuticals for Children Amendments of 2007, the 
     Secretary shall require that the sponsors of the studies that 
     result in labeling changes that are reflected in the annual 
     summary developed pursuant to subsection (f)(4)(F) 
     distribute, at least annually (or more frequently if the 
     Secretary determines that it would be beneficial to the 
     public health), such information to physicians and other 
     health care providers.'';
       (12) by inserting after subsection (k), as redesignated by 
     paragraph (9), the following:
       ``(l) Adverse Event Reporting.--
       ``(1) Reporting in year one.--Beginning on the date of 
     enactment of the Best Pharmaceuticals for Children Amendments 
     of 2007, during the 1-year period beginning on the date a 
     labeling change is made pursuant to subsection (i), the 
     Secretary shall ensure that all adverse event reports that 
     have been received for such drug (regardless of when such 
     report was received) are referred to the Office of Pediatric 
     Therapeutics established under section 6 of the Best 
     Pharmaceuticals for Children Act (Public Law 107-109). In 
     considering such reports, the Director of such Office shall 
     provide for the review of the report by the Pediatric 
     Advisory Committee, including obtaining any recommendations 
     of such Committee regarding whether the Secretary should take 
     action under this section in response to such reports.
       ``(2) Reporting in subsequent years.--Following the 1-year 
     period described in paragraph (1), the Secretary shall, as 
     appropriate, refer to the Office of Pediatric Therapeutics 
     all pediatric adverse event reports for a drug for which a 
     pediatric study was conducted under this section. In 
     considering such reports, the Director of such Office may 
     provide for the review of such reports by the Pediatric 
     Advisory Committee, including obtaining any recommendation of 
     such Committee regarding whether the Secretary should take 
     action in response to such reports.
       ``(3) Effect.--The requirements of this subsection shall 
     supplement, not supplant, other review of such adverse event 
     reports by the Secretary.'';
       (13) by inserting after subsection (m), as redesignated by 
     paragraph (9), the following:
       ``(n) Referral if Pediatric Studies Not Completed.--
       ``(1) In general.--Beginning on the date of enactment of 
     the Best Pharmaceuticals for Children Amendments of 2007, if 
     pediatric studies of a drug have not been completed under 
     subsection (d) and if the Secretary, through the committee 
     established under subsection (f), determines that there is a 
     continuing need for information relating to the use of the 
     drug in the pediatric population (including neonates, as 
     appropriate), the Secretary shall carry out the following:
       ``(A) For a drug for which a listed patent has not expired, 
     make a determination regarding whether an assessment shall be 
     required to be submitted under section 505B. Prior to making 
     such determination, the Secretary may take not more than 60 
     days to certify whether the Foundation for the National 
     Institutes of Health has sufficient funding at the time of 
     such certification to initiate 1 or more of the pediatric 
     studies of such drug referred to in the sentence preceding 
     this paragraph and fund 1 or more of such studies in their 
     entirety. Only if the Secretary makes such certification in 
     the affirmative, the Secretary shall refer such pediatric 
     study or studies to the Foundation for the National 
     Institutes of Health for the conduct of such study or 
     studies.
       ``(B) For a drug that has no listed patents or has 1 or 
     more listed patents that have expired, determine whether 
     there are funds available under section 736 to award a grant 
     to conduct the requested studies pursuant to paragraph (2).
       ``(2) Funding of studies.--If, pursuant to paragraph (1), 
     the Secretary determines that there are funds available under 
     section 736 to award a grant to conduct the requested 
     pediatric studies, then the Secretary shall issue a proposal 
     to award a grant to conduct the requested studies. If the 
     Secretary determines that funds are not available under 
     section 736, the Secretary shall refer the drug for inclusion 
     on the list established under section 409I of the Public 
     Health Service Act for the conduct of studies.
       ``(3) Public notice.--The Secretary shall give the public 
     notice of--
       ``(A) a decision under paragraph (1)(A) not to require an 
     assessment under section 505B and the basis for such 
     decision;
       ``(B) the name of any drug, its manufacturer, and the 
     indications to be studied pursuant to a grant made under 
     paragraph (2); and
       ``(C) any decision under paragraph (2) to refer a drug for 
     inclusion on the list established under section 409I of the 
     Public Health Service Act.
       ``(4) Effect of subsection.--Nothing in this subsection 
     alters or amends section 301(j) of this Act or section 552 of 
     title 5 or section 1905 of Title 18, United States Code.'';
       (14) in subsection (p), as redesignated by paragraph (9)--
       (A) striking ``6-month period'' and inserting ``3-month or 
     6-month period'';
       (B) by striking ``subsection (a)'' and inserting 
     ``subsection (b)''; and
       (C) by striking ``2007'' both places it appears and 
     inserting ``2012''.
       (b) Effective Date.--Except as otherwise provided in the 
     amendments made by subsection (a), such amendments shall 
     apply to written requests under section 505A of the Federal 
     Food, Drug, and Cosmetic Act (21 U.S.C. 355a) made after the 
     date of enactment of this Act.

     SEC. 3. PROGRAM FOR PEDIATRIC STUDIES OF DRUGS.

       Section 409I of the Public Health Service Act (42 U.S.C. 
     284m) is amended--
       (1) by striking subsections (a) and (b) and inserting the 
     following:
       ``(a) List of Priority Issues in Pediatric Therapeutics.--
       ``(1) In general.--Not later than 1 year after the date of 
     enactment of the Best Pharmaceuticals for Children Amendments 
     of 2007, the Secretary, acting through the Director of the 
     National Institutes of Health

[[Page S4698]]

     and in consultation with the Commissioner of Food and Drugs 
     and experts in pediatric research, shall develop and publish 
     a priority list of needs in pediatric therapeutics, including 
     drugs or indications that require study. The list shall be 
     revised every 3 years.
       ``(2) Consideration of available information.--In 
     developing and prioritizing the list under paragraph (1), the 
     Secretary shall consider--
       ``(A) therapeutic gaps in pediatrics that may include 
     developmental pharmacology, pharmacogenetic determinants of 
     drug response, metabolism of drugs and biologics in children, 
     and pediatric clinical trials;
       ``(B) particular pediatric diseases, disorders or 
     conditions where more complete knowledge and testing of 
     therapeutics, including drugs and biologics, may be 
     beneficial in pediatric populations; and
       ``(C) the adequacy of necessary infrastructure to conduct 
     pediatric pharmacological research, including research 
     networks and trained pediatric investigators.
       ``(b) Pediatric Studies and Research.--The Secretary, 
     acting through the National Institutes of Health, shall award 
     funds to entities that have the expertise to conduct 
     pediatric clinical trials or other research (including 
     qualified universities, hospitals, laboratories, contract 
     research organizations, practice groups, federally funded 
     programs such as pediatric pharmacology research units, other 
     public or private institutions, or individuals) to enable the 
     entities to conduct the drug studies or other research on the 
     issues described in subsection (a). The Secretary may use 
     contracts, grants, or other appropriate funding mechanisms to 
     award funds under this subsection.'';
       (2) in subsection (c)--
       (A) in the heading, by striking ``Contracts'' and inserting 
     ``Proposed Pediatric Study Requests'';
       (B) by striking paragraphs (4) and (12);
       (C) by redesignating paragraphs (1), (2), and (3), as 
     paragraphs (2), (3), and (4);
       (D) by inserting before paragraph (2), as redesignated by 
     subparagraph (C), the following:
       ``(1) Submission of proposed pediatric study request.--The 
     Director of the National Institutes of Health shall, as 
     appropriate, submit proposed pediatric study requests for 
     consideration by the Commissioner of Food and Drugs for 
     pediatric studies of a specific pediatric indication 
     identified under subsection (a). Such a proposed pediatric 
     study request shall be made in a manner equivalent to a 
     written request made under subsection (b) or (c) of section 
     505A of the Federal Food, Drug, and Cosmetic Act, including 
     with respect to the information provided on the pediatric 
     studies to be conducted pursuant to the request. The Director 
     of the National Institutes of Health may submit a proposed 
     pediatric study request for a drug for which--
       ``(A)(i) there is an approved application under section 
     505(j) of the Federal Food, Drug, and Cosmetic Act; or
       ``(ii) there is a submitted application that could be 
     approved under the criteria of section 505(j) of the Federal 
     Food, Drug, and Cosmetic Act; and
       ``(B) there is no patent protection or market exclusivity 
     protection for at least 1 form of the drug under the Federal 
     Food, Drug, and Cosmetic Act; and
       ``(C) additional studies are needed to assess the safety 
     and effectiveness of the use of the drug in the pediatric 
     population.'';
       (E) in paragraph (2), as redesignated by subparagraph (C)--
       (i) by inserting ``based on the proposed pediatric study 
     request for the indication or indications submitted pursuant 
     to paragraph (1)'' after ``issue a written request'';
       (ii) by striking ``in the list described in subsection 
     (a)(1)(A) (except clause (iv))'' and inserting ``under 
     subsection (a)''; and
       (iii) by inserting ``and using appropriate formulations for 
     each age group for which the study is requested'' before the 
     period at the end;
       (F) in paragraph (3), as redesignated by subparagraph (C)--
       (i) in the heading, by striking ``contracts'';
       (ii) by striking ``paragraph (1)'' and inserting 
     ``paragraph (2)'';
       (iii) by striking ``or if a referral described in 
     subsection (a)(1)(A)(iv) is made,'';
       (iv) by striking ``for contract proposals'' and inserting 
     ``for proposals''; and
       (v) by inserting ``in accordance with subsection (b)'' 
     before the period at the end;
       (G) in paragraph (4), as redesignated by subparagraph (C)--
       (i) by striking ``contract''; and
       (ii) by striking ``paragraph (2)'' and inserting 
     ``paragraph (3)'';
       (H) in paragraph (5)--
       (i) by striking the heading and inserting ``Contracts, 
     grants, or other funding mechanisms''; and
       (ii) by striking ``A contract'' and all that follows 
     through ``is submitted'' and inserting ``A contract, grant, 
     or other funding may be awarded under this section only if a 
     proposal is submitted'';
       (I) in paragraph (6)(A)--
       (i) by striking ``a contract awarded'' and inserting ``an 
     award''; and
       (ii) by inserting ``, including a written request if 
     issued'' after ``with the study''; and
       (3) by inserting after subsection (c) the following:
       ``(d) Dissemination of Pediatric Information.--Not later 
     than 1 year after the date of enactment of the Best 
     Pharmaceuticals for Children Amendments of 2007, the 
     Secretary, acting through the Director of the National 
     Institutes of Health, shall study the feasibility of 
     establishing a compilation of information on pediatric drug 
     use and report the findings to Congress.''
       ``(e) Authorization of Appropriations.--
       ``(1) In general.--There are authorized to be appropriated 
     to carry out this section--
       ``(A) $200,000,000 for fiscal year 2008; and
       ``(B) such sums as are necessary for each of the 4 
     succeeding fiscal years.
       ``(2) Availability.--Any amount appropriated under 
     paragraph (1) shall remain available to carry out this 
     section until expended.''.

     SEC. 4. REPORTS AND STUDIES.

       (a) GAO Report.--Not later than January 31, 2011, the 
     Comptroller General of the United States, in consultation 
     with the Secretary of Health and Human Services, shall submit 
     to Congress a report that addresses the effectiveness of 
     section 505A of the Federal Food, Drug, and Cosmetic Act (21 
     U.S.C. 355a) in ensuring that medicines used by children are 
     tested and properly labeled, including--
       (1) the number and importance of drugs for children that 
     are being tested as a result of the amendments made by this 
     Act and the importance for children, health care providers, 
     parents, and others of labeling changes made as a result of 
     such testing;
       (2) the number and importance of drugs for children that 
     are not being tested for their use notwithstanding the 
     provisions of this Act and the amendments made by this Act, 
     and possible reasons for the lack of testing, including 
     whether the number of written requests declined by sponsors 
     or holders of drugs subject to section 505A(g)(2) of the 
     Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a(g)(2)), 
     has increased or decreased as a result of the amendments made 
     by this Act;
       (3) the number of drugs for which testing is being done and 
     labeling changes required, including the date labeling 
     changes are made and which labeling changes required the use 
     of the dispute resolution process established pursuant to the 
     amendments made by this Act, together with a description of 
     the outcomes of such process, including a description of the 
     disputes and the recommendations of the Pediatric Advisory 
     Committee;
       (4) any recommendations for modifications to the programs 
     established under section 505A of the Federal Food, Drug and 
     Cosmetic Act (21 U.S.C. 355a) and section 409I of the Public 
     Health Service Act that the Secretary determines to be 
     appropriate, including a detailed rationale for each 
     recommendation; and
       (5)(A) the efforts made by the Secretary to increase the 
     number of studies conducted in the neonate population; and
       (B) the results of those efforts, including efforts made to 
     encourage the conduct of appropriate studies in neonates by 
     companies with products that have sufficient safety and other 
     information to make the conduct of the studies ethical and 
     safe.
       (b) IOM Study.--Not later than 3 years after the date of 
     enactment of this Act, the Secretary of Health and Human 
     Services shall enter into a contract with the Institute of 
     Medicine to conduct a study and report to Congress regarding 
     the written requests made and the studies conducted pursuant 
     to section 505A of the Federal Food, Drug, and Cosmetic Act. 
     The Institute of Medicine may devise an appropriate mechanism 
     to review a representative sample of requests made and 
     studies conducted pursuant to such section in order to 
     conduct such study. Such study shall--
       (1) review such representative written requests issued by 
     the Secretary since 1997 under subsections (b) and (c) of 
     such section 505A;
       (2) review and assess such representative pediatric studies 
     conducted under such subsections (b) and (c) since 1997 and 
     labeling changes made as a result of such studies; and
       (3) review the use of extrapolation for pediatric 
     subpopulations, the use of alternative endpoints for 
     pediatric populations, neonatal assessment tools, and ethical 
     issues in pediatric clinical trials.

     SEC. 5. TRAINING OF PEDIATRIC PHARMACOLOGISTS.

       (a) Investment in Tomorrow's Pediatric Researchers.--
     Section 452G(2) of the Public Health Service Act (42 U.S.C. 
     285g-10(2)) is amended by adding before the period at the end 
     the following: ``, including pediatric pharmacological 
     research''.
       (b) Pediatric Research Loan Repayment Program.--Section 
     487F(a)(1) of the Public Health Service Act (42 U.S.C. 288-
     6(a)(1)) is amended by inserting ``including pediatric 
     pharmacological research,'' after ``pediatric research,''.

     SEC. 6. FOUNDATION FOR THE NATIONAL INSTITUTES OF HEALTH.

       Section 499(c)(1)(C) of the Public Health Service Act (42 
     U.S.C. 290b(c)(1)(C)) is amended by striking ``and studies 
     listed by the Secretary pursuant to section 409I(a)(1)(A) of 
     the is Act and referred under section 505A(d)(4)(C) of the 
     Federal Food, Drug and Cosmetic Act (21 U.S.C. 
     355(a)(d)(4)(C)'' and inserting ``and studies for which the 
     Secretary issues a certification under section 505A(n)(1)(A) 
     of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
     355a(n)(1)(A))''.

     SEC. 7. CONTINUATION OF OPERATION OF COMMITTEE.

       Section 14 of the Best Pharmaceuticals for Children Act (42 
     U.S.C. 284m note) is amended by adding at the end the 
     following:
       ``(d) Continuation of Operation of Committee.--
     Notwithstanding section 14 of the

[[Page S4699]]

     Federal Advisory Committee Act (5 U.S.C. App.), the advisory 
     committee shall continue to operate during the 5-year period 
     beginning on the date of enactment of the Best 
     Pharmaceuticals for Children Amendments of 2007.''.

     SEC. 8. PEDIATRIC SUBCOMMITTEE OF THE ONCOLOGIC DRUGS 
                   ADVISORY COMMITTEE.

       Section 15 of the Best Pharmaceuticals for Children Act (42 
     U.S.C. 284m note) is amended--
       (1) in subsection (a)--
       (A) in paragraph (1)--
       (i) in subparagraph (B), by striking ``and'' after the 
     semicolon;
       (ii) in subparagraph (C), by striking the period at the end 
     and inserting ``; and''; and
       (iii) by adding at the end the following:
       ``(D) provide recommendations to the internal review 
     committee created under section 505A(f) of the Federal Food, 
     Drug, and Cosmetic Act (21 U.S.C. 355a(f)) regarding the 
     implementation of amendments to sections 505A and 505B of the 
     Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a and 
     355c) with respect to the treatment of pediatric cancers.''; 
     and
       (B) by adding at the end the following:
       ``(3) Continuation of operation of subcommittee.--
     Notwithstanding section 14 of the Federal Advisory Committee 
     Act (5 U.S.C. App.), the Subcommittee shall continue to 
     operate during the 5-year period beginning on the date of 
     enactment of the Best Pharmaceuticals for Children Amendments 
     of 2007.''; and
       (2) in subsection (d), by striking ``2003'' and inserting 
     ``2009''.

     SEC. 9. EFFECTIVE DATE AND LIMITATION FOR RULE RELATING TO 
                   TOLL-FREE NUMBER FOR ADVERSE EVENTS ON LABELING 
                   FOR HUMAN DRUG PRODUCTS.

       (a) In General.--Notwithstanding subchapter II of chapter 
     5, and chapter 7, of title 5, United States Code (commonly 
     known as the ``Administrative Procedure Act'') and any other 
     provision of law, the proposed rule issued by the 
     Commissioner of Food and Drugs entitled ``Toll-Free Number 
     for Reporting Adverse Events on Labeling for Human Drug 
     Products'', 69 Fed. Reg. 21778, (April 22, 2004) shall take 
     effect on January 1, 2008, unless such Commissioner issues 
     the final rule before such date.
       (b) Limitation.--The proposed rule that takes effect under 
     subsection (a), or the final rule described under subsection 
     (a), shall, notwithstanding section 17(a) of the Best 
     Pharmaceuticals for Children Act (21 U.S.C. 355b(a)), not 
     apply to a drug--
       (1) for which an application is approved under section 505 
     of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355);
       (2) that is not described under section 503(b)(1) of such 
     Act (21 U.S.C. 353(b)(1)); and
       (3) the packaging of which includes a toll-free number 
     through which consumers can report complaints to the 
     manufacturer or distributor of the drug.

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