[Congressional Record Volume 149, Number 110 (Wednesday, July 23, 2003)]
[Senate]
[Pages S9811-S9819]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




                 PEDIATRIC RESEARCH EQUITY ACT OF 2003

  Mr. DeWINE. Mr. President, I ask unanimous consent the Senate proceed 
to the immediate consideration of calendar 183, S. 650.
  The PRESIDING OFFICER. The clerk will report the bill by title.
  The legislative clerk read as follows:

       A bill (S. 650) to amend the Federal Food, Drug, and 
     Cosmetic Act to authorize the Food and Drug Administration to 
     require certain research into drugs used in pediatric 
     patients.

  There being no objection, the Senate proceeded to consider the 
bill which had been reported from the Committee on Health, Education, 
Labor, and Pensions, with amendments, as follows:

       [Strike the part shown in black brackets and insert the 
     part shown in italic.]

                                 S. 650

       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 ``Pediatric Research Equity 
     Act of 2003''.

     SEC. 2. RESEARCH INTO PEDIATRIC USES FOR DRUGS AND BIOLOGICAL 
                   PRODUCTS.

       (a) In General.--Subchapter A of chapter V of the Federal 
     Food, Drug, and Cosmetic Act (21 U.S.C. 351 et seq.) is 
     amended by inserting after section 505A the following:

     ``SEC. 505B. RESEARCH INTO PEDIATRIC USES FOR DRUGS AND 
                   BIOLOGICAL PRODUCTS.

       ``(a) New Drugs and Biological Products.--
       ``(1) In general.--A person that submits an application (or 
     supplement to an application)--
       ``(A) under section 505 for a new active ingredient, new 
     indication, new dosage form, new dosing regimen, or new route 
     of administration; or
       ``(B) under section 351 of the Public Health Service Act 
     (42 U.S.C. 262) for a new active ingredient, new indication, 
     new dosage form, new dosing regimen, or new route of 
     administration;

     shall submit with the application the assessments described 
     in paragraph (2).
       ``(2) Assessments.--
       ``(A) In general.--The assessments referred to in paragraph 
     (1) shall contain data, gathered using appropriate 
     formulations for each age group for which the assessment is 
     required, that are adequate--
       ``(i) to assess the safety and effectiveness of the drug or 
     the biological product for the claimed indications in all 
     relevant pediatric subpopulations; and
       ``(ii) to support dosing and administration for each 
     pediatric subpopulation for which the drug or the biological 
     product is safe and effective.
       ``(B) Similar course of disease or similar effect of drug 
     or biological product.--
       ``(i) In general.--If the course of the disease and the 
     effects of the drug are sufficiently similar in adults and 
     pediatric patients, the Secretary may conclude that pediatric 
     effectiveness can be extrapolated from adequate and well-
     controlled studies in adults, usually supplemented with other 
     information obtained in pediatric patients, such as 
     pharmacokinetic studies.
       ``(ii) Extrapolation between age groups.--A study may not 
     be needed in each pediatric age group if data from 1 age 
     group can be extrapolated to another age group.
       ``(3) Deferral.--On the initiative of the Secretary or at 
     the request of the applicant, the Secretary may defer 
     submission of some or all assessments required under 
     paragraph (1) until a specified date after approval of the 
     drug or issuance of the license for a biological product if--
       ``(A) the Secretary finds that--
       ``(i) the drug or biological product is ready for approval 
     for use in adults before pediatric studies are complete;
       ``(ii) pediatric studies should be delayed until additional 
     safety or effectiveness data have been collected; or
       ``(iii) there is another appropriate reason for deferral; 
     and
       ``(B) the applicant submits to the Secretary--
       ``(i) certification of the grounds for deferring the 
     assessments;
       ``(ii) a description of the planned or ongoing studies; and
       ``(iii) evidence that the studies are being conducted or 
     will be conducted with due diligence and at the earliest 
     possible time.
       ``(4) Waivers.--
       ``(A) Full waiver.--On the initiative of the Secretary or 
     at the request of an applicant, the Secretary shall grant a 
     full waiver, as appropriate, of the requirement to submit 
     assessments for a drug or biological product under this 
     subsection if the applicant certifies and the Secretary finds 
     that--
       ``(i) necessary studies are impossible or highly 
     impracticable (because, for example, the number of patients 
     is so small or the patients are geographically dispersed);
       ``(ii) there is evidence strongly suggesting that the drug 
     or biological product would be ineffective or unsafe in all 
     pediatric age groups; or
       ``(iii) the drug or biological product--

       ``(I) does not represent a meaningful therapeutic benefit 
     over existing therapies for pediatric patients; and
       ``(II) is not likely to be used in a substantial number of 
     pediatric patients.

       ``(B) Partial waiver.--On the initiative of the Secretary 
     or at the request of an applicant, the Secretary shall grant 
     a partial waiver, as appropriate, of the requirement to 
     submit assessments for a drug or biological product under 
     this subsection with respect to a specific pediatric age 
     group if the applicant certifies and the Secretary finds 
     that--
       ``(i) necessary studies are impossible or highly 
     impracticable (because, for example, the number of patients 
     in that age group is so small or patients in that age group 
     are geographically dispersed);
       ``(ii) there is evidence strongly suggesting that the drug 
     or biological product would be ineffective or unsafe in that 
     age group;
       ``(iii) the drug or biological product--

       ``(I) does not represent a meaningful therapeutic benefit 
     over existing therapies for pediatric patients in that age 
     group; and
       ``(II) is not likely to be used by a substantial number of 
     pediatric patients in that age group; or

       ``(iv) the applicant can demonstrate that reasonable 
     attempts to produce a pediatric

[[Page S9812]]

     formulation necessary for that age group have failed.
       ``(C) Pediatric formulation not possible.--If a waiver is 
     granted on the ground that it is not possible to develop a 
     pediatric formulation, the waiver shall cover only the 
     pediatric groups requiring that formulation.
       ``(D) Labeling requirement.--If the Secretary grants a full 
     or partial waiver because there is evidence that a drug or 
     biological product would be ineffective or unsafe in 
     pediatric populations, the information shall be included in 
     the labeling for the drug or biological product.
       ``(b) Marketed Drugs and Biological Products.--
       ``(1) In general.--After providing notice in the form of a 
     letter and an opportunity for written response and a meeting, 
     which may include an advisory committee meeting, the 
     Secretary may (by order in the form of a letter) require the 
     holder of an approved application for a drug under section 
     505 or the holder of a license for a biological product under 
     section 351 of the Public Health Service Act (42 U.S.C. 262) 
     to submit by a specified date the assessments described in 
     subsection (a)(2) if the Secretary finds that--
       ``(A)(i) the drug or biological product is used for a 
     substantial number of pediatric patients for the labeled 
     indications; and
       ``(ii) the absence of adequate labeling could pose 
     significant risks to pediatric patients; or
       ``(B)(i) there is reason to believe that the drug or 
     biological product would represent a meaningful therapeutic 
     benefit over existing therapies for pediatric patients for 1 
     or more of the claimed indications; and
       ``(ii) the absence of adequate labeling could pose 
     significant risks to pediatric patients.
       ``(2) Waivers.--
       ``(A) Full waiver.--At the request of an applicant, the 
     Secretary shall grant a full waiver, as appropriate, of the 
     requirement to submit assessments under this subsection if 
     the applicant certifies and the Secretary finds that--
       ``(i) necessary studies are impossible or highly 
     impracticable (because, for example, the number of patients 
     in that age group is so small or patients in that age group 
     are geographically dispersed); or
       ``(ii) there is evidence strongly suggesting that the drug 
     or biological product would be ineffective or unsafe in all 
     pediatric age groups.
       ``(B) Partial waiver.--At the request of an applicant, the 
     Secretary shall grant a partial waiver, as appropriate, of 
     the requirement to submit assessments under this subsection 
     with respect to a specific pediatric age group if the 
     applicant certifies and the Secretary finds that--
       ``(i) necessary studies are impossible or highly 
     impracticable (because, for example, the number of patients 
     in that age group is so small or patients in that age group 
     are geographically dispersed);
       ``(ii) there is evidence strongly suggesting that the drug 
     or biological product would be ineffective or unsafe in that 
     age group;
       ``(iii)(I) the drug or biological product--

       ``(aa) does not represent a meaningful therapeutic benefit 
     over existing therapies for pediatric patients in that age 
     group; and
       ``(bb) is not likely to be used in a substantial number of 
     pediatric patients in that age group; and

       ``(II) the absence of adequate labeling could not pose 
     significant risks to pediatric patients; or
       ``(iv) the applicant can demonstrate that reasonable 
     attempts to produce a pediatric formulation necessary for 
     that age group have failed.
       ``(C) Pediatric formulation not possible.--If a waiver is 
     granted on the ground that it is not possible to develop a 
     pediatric formulation, the waiver shall cover only the 
     pediatric groups requiring that formulation.
       ``(D) Labeling requirement.--If the Secretary grants a full 
     or partial waiver because there is evidence that a drug or 
     biological product would be ineffective or unsafe in 
     pediatric populations, the information shall be included in 
     the labeling for the drug or biological product.
       ``(3) Relationship to other pediatric provisions.--
       ``(A) No assessment without written request.--No assessment 
     may be required under paragraph (1) for a drug subject to an 
     approved application under section 505 unless--
       ``(i) the Secretary has issued a written request for a 
     related pediatric study under section 505A(c) of this Act or 
     section 409I of the Public Health Service Act (42 U.S.C. 
     284m);
       ``(ii)(I) if the request was made under section 505A(c)--

       ``(aa) the recipient of the written request does not agree 
     to the request; or
       ``(bb) the Secretary does not receive a response as 
     specified under section 505A(d)(4)(A); or

       ``(II) if the request was made under section 409I of the 
     Public Health Service Act (42 U.S.C. 284m)--

       ``(aa) the recipient of the written request does not agree 
     to the request; or
       ``(bb) the Secretary does not receive a response as 
     specified under section 409I(c)(2) of that Act; and

       ``(iii)(I) the Secretary certifies under subparagraph (B) 
     that there are insufficient funds under sections 409I and 499 
     of the Public Health Service Act (42 U.S.C. 284m, 290b) to 
     conduct the study; or
       ``(II) the Secretary publishes in the Federal Register a 
     certification that certifies that--

       ``(aa) no contract or grant has been awarded under section 
     409I or 499 of the Public Health Service Act (42 U.S.C. 284m, 
     290b); and
       ``(bb) not less than 270 days have passed since the date of 
     a certification under subparagraph (B) that there are 
     sufficient funds to conduct the study.

       ``(B) No agreement to request.--Not later than 60 days 
     after determining that no holder will agree to the written 
     request (including a determination that the Secretary has not 
     received a response specified under section 505A(d) of this 
     Act or section 409I of the Public Health Service Act (42 
     U.S.C. 284m), the Secretary shall certify whether the 
     Secretary has sufficient funds to conduct the study under 
     section 409I or 499 of the Public Health Service Act (42 
     U.S.C. 284m, 290b), taking into account the prioritization 
     under section 409I.
       ``(c) Meaningful Therapeutic Benefit.--For the purposes of 
     paragraph (4)(A)(iii)(I) and (4)(B)(iii)(I) of subsection (a) 
     and paragraphs (1)(B)(i) and (2)(B)(iii)(I)(aa) of subsection 
     (b), a drug or biological product shall be considered to 
     represent a meaningful therapeutic benefit over existing 
     therapies if the Secretary estimates that--
       ``(1) if approved, the drug or biological product would 
     represent a significant improvement in the treatment, 
     diagnosis, or prevention of a disease, compared with marketed 
     products adequately labeled for that use in the relevant 
     pediatric population; or
       ``(2) the drug or biological product is in a class of 
     products or for an indication for which there is a need for 
     additional options.
       ``(d) Submission of Assessments.--If a person fails to 
     submit an assessment described in subsection (a)(2), or a 
     request for approval of a pediatric formulation described in 
     subsection (a) or (b), in accordance with applicable 
     provisions of subsections (a) and (b)--
       ``(1) the drug or biological product that is the subject of 
     the assessment or request may be considered misbranded and 
     subject to relevant enforcement action (except that the drug 
     or biological product shall not be subject to action under 
     section 303); but
       ``(2) the failure to submit the assessment or request shall 
     not be the basis for a proceeding--
       ``(A) to withdraw approval for a drug under section 505(e); 
     or
       ``(B) to revoke the license for a biological product under 
     section 351 of the Public Health Service Act (42 U.S.C. 262).
       ``(e) Meetings.--Before and during the investigational 
     process for a new drug or biological product, the Secretary 
     shall meet at appropriate times with the sponsor of the new 
     drug or biological product to discuss--
       ``(1) information that the sponsor submits on plans and 
     timelines for pediatric studies; or
       ``(2) any planned request by the sponsor for waiver or 
     deferral of pediatric studies.
       ``(f) Scope of Authority.--Nothing in this section provides 
     to the Secretary any authority to require a pediatric 
     assessment of any drug or biological product, or any 
     assessment regarding other populations or uses of a drug or 
     biological product, other than the pediatric assessments 
     described in this section.
       ``(g) Orphan Drugs.--Unless the Secretary requires 
     otherwise by regulation, this section does not apply to any 
     drug for an indication for which orphan designation has been 
     granted under section [526.''.] 526.
       ``(h) Integration With Other Pediatric Studies.--The 
     authority under this section shall remain in effect so long 
     as an application subject to this section may be accepted for 
     filing by the Secretary on or before the date specified in 
     section 505A(n).''.
       (b) Conforming Amendments.--
       (1) Section 505(b)(1) of the Federal Food, Drug, and 
     Cosmetic Act (21 U.S.C. 355(b)(1)) is amended in the second 
     sentence--
       (A) by striking ``and (F)'' and inserting ``(F)''; and
       (B) by striking the period at the end and inserting ``, and 
     (G) any assessments required under section 505B.''.
       (2) Section 505A(h) of the Federal Food, Drug, and Cosmetic 
     Act (21 U.S.C. 355a(h)) is amended--
       (A) in the subsection heading, by striking ``Regulations'' 
     and inserting ``Pediatric Research Requirements''; and
       (B) by striking ``pursuant to regulations promulgated by 
     the Secretary'' and inserting ``by a provision of law 
     (including a regulation) other than this section''.
       (3) Section 351(a)(2) of the Public Health Service Act (42 
     U.S.C. 262(a)(2)) is amended--
       (A) by redesignating subparagraph (B) as subparagraph (C); 
     and
       (B) by inserting after subparagraph (A) the following:
       ``(B) Pediatric studies.--A person that submits an 
     application for a license under this paragraph shall submit 
     to the Secretary as part of the application any assessments 
     required under section 505B of the Federal Food, Drug, and 
     Cosmetic Act.''.

     SEC. 3. TECHNICAL AND CONFORMING AMENDMENTS.

       (a) Abbreviated New Drug Application.--Section 505A of the 
     Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is 
     amended in subparagraphs (A) and (B) of subsection (b)(2) and 
     subparagraphs (A) and (B) of subsection (c)(2) by striking 
     ``505(j)(4)(B)'' and inserting ``505(j)(5)(B)''.
       (b) Pediatric Advisory Committee.--
       (1) Section 505A(i)(2) of the Federal Food, Drug, and 
     Cosmetic Act (21 U.S.C. 355a(i)(2)) is amended by striking 
     ``Advisory Subcommittee of the Anti-Infective Drugs'' each 
     place it appears.

[[Page S9813]]

       (2) Section 14 of the Best Pharmaceuticals for Children Act 
     (42 U.S.C. 284m note; Public Law 107-109) is amended--
       (A) in the section heading, by striking ``PHARMACOLOGY'';
       (B) in subsection (a), by striking ``(42 U.S.C. 217a),'' 
     and inserting (42 U.S.C. 217a) or other appropriate 
     authority,'';
       (C) in subsection (b)--
       (i) in paragraph (1), by striking ``and in consultation 
     with the Director of the National Institutes of Health''; and
       (ii) in paragraph (2), by striking ``and 505A'' and 
     inserting ``505A, and 505B''; and
       (D) by striking ``pharmacology'' each place it appears and 
     inserting ``therapeutics''.
       (3) Section 15(a)(2)(A) of the Best Pharmaceuticals for 
     Children Act (115 Stat. 1419) is amended by striking 
     ``Pharmacology''.
       (4) Section 16(1)(C) of the Best Pharmaceuticals for 
     Children Act (21 U.S.C. 355a note; Public Law 107-109) is 
     amended by striking ``Advisory Subcommittee of the Anti-
     Infective Drugs''.
       (5) Section 17(b)(1) of the Best Pharmaceuticals for 
     Children Act (21 U.S.C. 355b(b)(1)) is amended in the second 
     sentence by striking ``Advisory Subcommittee of the Anti-
     Infective Drugs''.
       (6) Paragraphs (8), (9), and (11) of section 409I(c) of the 
     Public Health Service Act (42 U.S.C. 284m(c)) are amended by 
     striking ``Advisory Subcommittee of the Anti-Infective 
     Drugs'' each place it appears.

     SEC. 4. EFFECTIVE DATE.

       (a) In General.--This Act and the amendments made by this 
     Act take effect October 17, 2002.
       (b) No Limitation of Authority.--Neither the lack of 
     guidance or regulations to implement this Act or the 
     amendments made by this Act nor the pendency of the process 
     for issuing guidance or regulations shall limit the authority 
     of the Secretary of Health and Human Services under, or defer 
     any requirement under, this Act or those amendments.

  Mr. DeWINE. Mr. President, I rise this evening in support of the 
passage of this bill, the pediatric rule. Passage of this bill will be 
a very important step in protecting the health of our children. This 
bill will help keep the pediatric rule in place to help ensure the 
drugs we give our children when they are sick are actually tested for 
use by our children. The tragic reality is there are medicines on the 
market today that are being used by and prescribed for our Nation's 
children that are oftentimes not being tested for their use. It has 
been that way for years and years.
  For many years, doctors have had to take a chance when prescribing 
medicines for our kids. Doctors have literally had to tell parents to 
cut the pill in half or in quarters to be given to a child. The doctors 
have used the best information they have to literally guess how much 
medicine to give a child. That is all they could do with the medicines; 
they have had to guess.
  Quite frankly, these medicines have been overprescribed, 
underprescribed, or maybe not prescribed at all when they should have 
been prescribed. For example, recently the drug Paxil, which is an 
antidepressant, has been prescribed without being tested in children at 
all. Many people have heard of this drug. Many people have heard of the 
beneficial effects for adults with anxiety and panic disorders. What 
people did not know, what doctors did not know, was what we have 
recently found out. Recently the British Government has warned doctors 
to stop prescribing this drug for children, warning that the medicine 
increased the risk of suicide or suicidal thinking among children with 
depression. This action, in turn, spurred the FDA to conduct its own 
investigation into the safety of this drug for younger patients, 
resulting in a similar warning to physicians here in the United States: 
Don't prescribe this drug for children.

  That is just one example. We have page after page of examples of 
drugs that have been prescribed to children in the past and once we 
then tested them, once the protocols were done, the testing was done, 
lo and behold, we found they were more effective for children than we 
thought. Sometimes they were not effective, sometimes the 
prescriptions, the amount, the dosage that had been used was too much, 
sometimes not enough.
  The facts are these. As we all know, children are not just miniature 
adults. You can't just take the weight and just reduce the dosage. Kids 
react differently. That is why it is so important to have the testing 
done. Yet when Senator Chris Dodd and I first started on this cause, 5 
or 6 years ago, 80 percent of the drugs that came on the market had 
never been tested for children at all.
  It has been over a year now since this Senate passed and the 
President signed into law the Best Pharmaceuticals for Children Act. 
The Best Act was a bill that followed the Better Pharmaceuticals for 
Children Act, which we passed a few years before that. That law, the 
Best Pharmaceuticals for Children Act, was part of the solution, just 
part of the solution to address the problem of getting medicines tested 
for use by children.
  That law provides, as its predecessor bill did, a 6-month patent 
extension to pharmaceutical companies in exchange for the testing of 
medicines in children. That was a voluntary law and it has worked 
pretty well. For as long as the bill has been law--its predecessor was 
law--the Food and Drug Administration reported success in ensuring that 
more medicines are tested for use in children. With this economic 
incentive by this Best Pharmaceutical and Better Pharmaceutical bill in 
place, companies are seeing the value of studying their drugs in 
children and are applying for the patent extension, and children are 
benefiting.
  But the Best Pharmaceuticals incentive cannot work alone. It was 
never intended to work alone to ensure that medicines for children are 
properly tested for their use. In order to ensure that no medicines 
needed to treat children, including vaccines or other biologics, would 
go untested, the FDA, in 1997, proposed what is known as the Pediatric 
Rule, a companion rule. The Pediatric Rule allowed the FDA to require 
that drugs deemed important for children be tested for their safety, 
for their effectiveness, and that they be properly, then, labeled for 
children.
  Unfortunately--and this is what brings us to the Senate floor tonight 
to consider this bill--the Pediatric Rule came under legal challenge 
and was, in fact, overturned in court in October 2002, last year, by a 
district court. That court ruled that the FDA lacked the statutory 
authority to require pediatric studies.
  What the court said was it was incumbent upon Congress to fix it. 
That is why we are here tonight. This was a troubling step backward for 
children's health, considering that today 75 percent of the medicines 
on the market still, even with the Better Pharmaceutical bill and the 
Best Pharmaceutical bill, still 75 percent of the medicines on the 
market today are not tested and labeled for pediatric use.

  Without the Pediatric Rule in place, without the necessary authority 
provided to the FDA, new medicines and biologics coming onto the market 
are not required to be tested for use in kids. Since that court 
decision on October 17, 2002, the FDA has indicated that over 300 
medicines either have applications pending or incomplete studies 
pending, and that unless the Pediatric Rule stays in place these will 
all be lost. Many more, hundreds more will be lost in the future. 
Pediatricians will not know how to prescribe these drugs in the future 
or whether to prescribe them at all.
  That is why Senator Clinton, Senator Dodd, and myself introduced the 
bill that we hope to pass tonight. It is a bill that would codify a 
significant piece of the Pediatric Rule to assure that it stays in 
place and ensures that children will remain on safe footing when it 
comes to the testing of the medications that they use.
  Furthermore, we need to keep the Pediatric Rule in place right now 
because the Pediatric Rule and incentives work together to ensure that 
drugs are tested for use in children.
  The Best Pharmaceuticals for Children Act, as I said already, was 
never intended to be a substitute for the rule but, rather, to 
reinforce and work with the rule. For example, the Pediatric Rule may 
be invoked in instances where pediatric information is essential but 
the patent exclusivity incentive is no longer available.
  The Pediatric Rule also applies to biologics, whereas the Best 
Pharmaceutical bill does not. A significant portion of therapeutics 
used in children, including many cancer treatments or biological 
products--by that, of course, we mean products that include a live 
agent. Because the Best Pharmaceutical law does not apply to biologics, 
the Pediatric Rule is the only way to ensure proper and effective 
pediatric labeling.
  Finally, the Best Pharmaceutical Act is voluntary. For any number of 
reasons, including insufficient sales, a manufacturer simply may choose 
to

[[Page S9814]]

not conduct the necessary testing to receive additional exclusivity 
under the ``Best'' law, and when that happens and the drug is not 
tested for kids, children are the losers. But just because a drug 
manufacturer chooses not to study the drug in children does not mean 
that the drug is not critical to the proper care of your children and 
my children or grandchildren. Without the Pediatric Rule that is in 
front of us today, there is no way to guarantee that a drug that is 
used in the pediatric population is tested for children's use.
  With the establishment of the Pediatric Rule and the financial 
incentives of the Best Pharmaceutical law, which will go with this, 
there has been a dramatic increase in the number of studies that have 
been undertaken. Let me quote from the Government's Response to 
Plaintiff's Notice of Reauthorization of FDA Modernization Act. This is 
the document the Government filed to defend the lawsuit against the 
rule.

       These two options--Best Pharmaceuticals for Children Act 
     and the Pediatric Rule--have resulted in a number of drugs 
     being labeled for use in pediatric applications. As of March 
     31, 2001, 94 applications containing complete or partial 
     pediatric use and information have been submitted to the 
     agency. Of these 94 applications, 45 are attributable to the 
     statutory exclusivity provision. FDA attributes 48 of the 94 
     applications to the authority of the pediatric rule alone.

  So you can see how the two must work together, how important the rule 
is. Our legislation is a step toward assuring this progress that we 
have made so far will not erode. Our bill, as amended, provides that 
the FDA may only impose the pediatric study requirement for already-
marketed drugs when the pediatric exclusivity incentive provisions fail 
to yield necessary pediatric information. This means that for already-
marketed drugs, drugs that the FDA has already approved and are already 
on the drugstore shelf, before FDA can require a company to study the 
drug for use in children, the incentive provisions of the Best 
Pharmaceuticals law have to be used first. So the drug manufacturer has 
to choose to use the incentive provisions first, before FDA can invoke 
the pediatric study requirement.
  Our bill also preserves the waiver and deferral process so that drug 
companies can get waivers or deferrals for a range of legitimate 
reasons. Waivers are a simple concept.
  Drugs, such as those used to treat Alzheimer's disease--those drugs 
that would not be used in children at all--obviously should not be 
tested for use in children. Those drug manufacturers would be allowed 
to waive the pediatric drug study requirement.
  Deferrals are similar. For drug manufacturers who require additional 
time to complete the drug study or need to get additional information 
in the adult population before beginning to study the drug in children 
can, in consultation with the FDA, defer the pediatric drug studies 
until a later date.
  Again, I am very pleased that my colleagues have agreed to pass our 
bill. It is a vital step toward ensuring that children are no longer a 
therapeutic afterthought.
  Our bill puts children on a level playing field with adults for the 
first time.
  Before I yield the floor, I would like to take this opportunity to 
thank the many people who have worked diligently to draft this bill and 
to help get it passed. I would like to thank Majority Leader Frist and 
Senators Clinton, Dodd, Gregg, Kennedy, and Murray for their leadership 
on this issue. Without their support, this bill would not be a reality.
  I would also like to thank Abby Kral of my staff for her dedication 
and hard work on this issue--she spent an unbelievable amount of time 
on it--as well as Christina Ho from Senator Clinton's Staff, Ben 
Berwick with Senator Dodd, Vince Ventimiglia with Senator Gregg's 
Staff, and David Dorsey with Senator Kennedy.
  Finally, I would like to recognize two groups that provided my staff 
and the staff of the HELP Committee with invaluable comments and 
insights--the American Academy of Pediatrics and the Elizabeth Glaser 
Pediatric AIDS Foundation.
  Thank you all for your efforts and commitment to protecting our 
children's health and safety.
  Mr. KENNEDY. Mr. President, this important bill guarantees that drugs 
and biological products used for children are tested and labeled for 
children. It helps assure that the miracle cures of today can be 
administered to our children in safe and effective ways.
  I commend Senators Gregg, Clinton, DeWine, and Dodd for their 
effective and tireless leadership to see this important legislation 
through the Senate. And it is endorsed by the American Academy of 
Pediatrics, the Elizabeth Glaser Pediatric AIDS Foundation, the March 
of Dimes, and many other organizations dedicated to children's health.
  Under this legislation, drug companies will be required to prove that 
their drugs and biological products are safe and effective for their 
intended use in children. For too long, drug companies have tested 
their products only in adults. For years, companies only rarely tested 
their drugs in children, unless the drug's use was for a juvenile 
disease. For other drugs, the label simply said that the product had 
not been shown to be safe and effective in children. To use such drugs 
on our children was a medical gamble.
  Fortunately, that practice began to change 6 years ago. In 1997, 
Congress authorized 6 months of ``pediatric exclusivity''--6 months of 
additional life of a drug patent if the company had studied the drug in 
children. The extra patent protection was a valuable economic incentive 
for drug companies to study their drugs on children, and it has been 
very successful in achieving that goal.
  In 1998, FDA issued its Pediatric Rule, which allowed the agency to 
require a drug company to test and label certain drugs for children.
  The patent exclusivity can be used once to study a drug. But the FDA 
rule can be used more than once, if needed, such as when the studies 
requested under exclusivity do not include studies in infants or 
newborns. In some cases, studies in older children are needed before 
studies can even be designed for younger children and newborn infants.
  The rule can be used to require testing for biological products, 
which are not eligible for the extra patent exclusivity. The rule can 
also be used when a drug company decides not to seek extra patent 
exclusivity and does not study a drug in children.
  Unfortunately, a Federal district court held that FDA does not have 
the statutory authority to issue the Pediatric Rule. Although the 
American Academy of Pediatrics and the Elizabeth Glaser Pediatric AIDS 
Foundation are appealing this decision, and we hope for their quick 
success, the Senate has now passed this legislation to correct the 
situation.
  With this legislation, the essential protections of the rule will be 
codified in law: There will be a presumption that newly approved 
applications for new active ingredients, new indications, new dosage 
forms, new dosing regimens, or new routes of administration for drugs 
and biological products will include assessments of safety and 
effectiveness for all relevant pediatric subpopulations. These 
assessments will support dosing and administration using a pediatric 
formulation for all pediatric subpopulations in which the product is 
safe and effective. This will be a huge step forward for children, and 
will put them on an equal footing with adults.
  In addition, many products already on the market have meaningful 
therapeutic benefit to children or may be used for a substantial number 
of children. However, the absence of adequate labeling in these 
products poses significant risks to pediatric patients. This 
legislation will allow FDA to require such products to be studied in 
children for its approved indication. The bill requires that FDA must 
first provide an opportunity for these studies to be conducted under 
the provisions of the Best Pharmaceuticals for Children Act. However, 
if a product's manufacturer does not agree promptly to perform such 
studies voluntarily, and if funds are not sufficient so that the NIH or 
the Foundation for the NIH does not contract or issue a grant for 
conduct of the studies within a set period of time, FDA may invoke the 
authority in this legislation to require the studies. Although FDA 
never used this authority under its Pediatric Rule, we expect FDA to 
use it as necessary to ensure that drugs and biological products that 
are already approved are studied in children when other mechanisms to 
get them studied fail.

[[Page S9815]]

  This legislation, with the managers' amendment, provides FDA with 
clear enforcement authority to bring a seizure or injunction action 
when a company fails to submit a required pediatric assessment. That 
failure alone will make the drug or biological product misbranded.
  This legislation, with the managers' amendment, clarifies that 
assessments required under FDA's Pediatric Rule that have not yet been 
submitted to FDA, whether deferred until after approval or not, are 
assessments required under this legislation. The legislation therefore 
ensures that hundreds of assessments that FDA required under its rule 
will be completed for the benefit of the Nation's children.
  Although this legislation is a giant step forward for children, I 
can't help but express my disappointment that its requirements are tied 
to the pediatric exclusivity provision that sunsets in 2007. Adults are 
guaranteed that new drugs will be reviewed for safety and effectiveness 
for them before they are approved by the FDA. Our Nation's children 
deserve no less. They should not have to come back in 4 years to plead 
for the right to safe and effective medicines.
  Again, I commend my colleagues for reaching bipartisan agreement on 
this important initiative for children. I urge the House to act 
promptly to pass this bill so that children may quickly be protected by 
this legislation.
  Mr. DeWINE. Mr. President, I ask unanimous consent that the committee 
amendment be agreed to, the Gregg amendment be agreed to, that the 
bill, as amended, be read a third time and passed, the motion to 
reconsider be laid upon the table, and that any statements and 
colloquies relating to the bill be printed in the Record.
  The PRESIDING OFFICER. Without objection, it is so ordered.
  The committee amendment was agreed to.
  The amendment (No. 1360) was agreed to, as follows:


                           amendment no. 1360

       On page 14, line 18, after ``misbranded'', insert ``solely 
     because of that failure''.
       On page 19, strike lines 5 and 6 and insert the following:
       (a) In General.--Subject to subsection (b), this Act and 
     the amendments made by this Act take effect on the date of 
     enactment of this Act.
       (b) Applicability to New Drugs and Biological Products.--
       (1) In general.--Subsection (a) of section 505B of the 
     Federal Food, Drug, and Cosmetic Act (as added by section 2) 
     shall apply to an application described in paragraph (1) of 
     that subsection submitted to the Secretary of Health and 
     Human Services on or after April 1, 1999.
       (2) Waivers and deferrals.--
       (A) Waiver or deferral granted.--If, with respect to an 
     application submitted to the Secretary of Health and Human 
     Services between April 1, 1999, and the date of enactment of 
     this Act, a waiver or deferral of pediatric assessments was 
     granted under regulations of the Secretary then in effect, 
     the waiver or deferral shall be a waiver or deferral under 
     subsection (a) of section 505B of the Federal Food, Drug, and 
     Cosmetic Act, except that any date specified in such a 
     deferral shall be extended by the number of days that is 
     equal to the number of days between October 17, 2002, and the 
     date of enactment of this Act.
       (B) Waiver and deferral not granted.--If, with respect to 
     an application submitted to the Secretary of Health and Human 
     Services between April 1, 1999, and the date of enactment of 
     this Act, neither a waiver nor deferral of pediatric 
     assessments was granted under regulations of the Secretary 
     then in effect, the person that submitted the application 
     shall be required to submit assessments under subsection 
     (a)(2) of section 505B of the Federal Food, Drug, and 
     Cosmetic Act on the date that is the later of--
       (i) the date that is 1 year after the date of enactment of 
     this Act; or
       (ii) such date as the Secretary may specify under 
     subsection (a)(3) of that section;

     unless the Secretary grants a waiver under subsection (a)(4) 
     of that section.
       On page 19, line 7, strike ``(b)'' and insert ``(c)''.


                     pediatrics research authority

  Mr. GREGG. Mr. President, I rise to speak to a managers' amendment to 
S. 650, the Pediatric Research Equity Act. This amendment makes 
improvements to the legislation as reported out of the Committee on 
Health, Education, Labor and Pensions in June. Because these 
improvements were made after the committee report was filed, this 
statement is intended to serve as the committee's views on the amended 
legislation. This statement was shared with the other committee members 
and has their concurrence.
  Mr. KENNEDY. Mr. President, the Democratic sponsors of the bill and I 
concur with this statement.
  Mr. GREGG. Mr. President, the purpose of this legislation is to 
provide FDA with statutory authority to require pediatric studies in 
specified circumstances. In October 2002, a Federal district court held 
that existing law did not provide FDA the authority to issue a 
regulation requiring pediatric studies for drugs marketed to adults but 
important to children. Although this decision is being appealed, this 
legislation will provide the agency with definitive statutory authority 
to require pediatric studies of new and already marketed drugs and 
biologics in the circumstances specified in the legislation and to 
enforce any violations of those requirements in Federal court. This has 
always been the intent of S. 650. After the legislation was marked up 
in committee, the managers of the bill agreed to amend the language in 
section 505B(d) to make this intent even clearer.
  The enforcement mechanism in section 505B(d) provides that if a 
person fails to submit an assessment described in subsection (a)(2) or 
a request for approval of a pediatric formulation described in 
subsection (a) or (b) under the new law, ``the drug or biological 
product that is the subject of the assessment or request may be 
considered misbranded solely because of such failure.'' This language 
confers on the Secretary authority to bring a misbranding action where 
a violation has occurred.
  The committee has used the language ``may be considered'' 
(misbranded) rather than the traditional ``shall be deemed to be'' 
(misbranded) that is used in other provisions of the Federal Food, Drug 
and Cosmetic Act in order to emphasized that the Secretary may exercise 
traditional enforcement discretion in deciding whether to bring such an 
action. The Committee recognizes that the Secretary retains that 
discretion under other provisions of current law that use the ``shall'' 
formulation. Nevertheless, the Committee intends for this authority to 
be interpreted by the courts and to be implemented by FDA in a manner 
consistent with the agency's enforcement authorities in current law 
that use the ``shall'' formulation.
  As is true with other provisions of current law, once the Secretary 
decides to initiate an enforcement action under section 505B(d), no 
formal finding or other proceeding is required. Moreover, it is not 
necessary for the Secretary to identify any other misbranding authority 
in the act. The new authority conferred by section 505B(d) 
is sufficient. For example, the failure of a sponsor to submit 
pediatric studies in accordance with the requirements of the 
legislation alone would be a sufficient basis to prosecute an action in 
federal district court.

  The managers of the bill have agreed to the extraordinary retroactive 
application of the provisions of the new research authority in order to 
avoid even greater potential harm to children through the loss of 
research and agency resources should assessments, waivers, and 
deferrals under the Pediatric Rule be considered invalid following the 
recent district court decision invalidating the rule. This application 
should not be considered approval of the agency's interpretation of its 
authority nor disagreement of the court's ruling. In the extraordinary 
situation at hand, the managers' amendment modifies the effective date 
provision of the legislation to ensure a seamless transition of the 
pediatric study requirement from the Pediatric rule to this 
legislation. The intent is that waivers and deferrals of the study 
requirement previously granted under the rule be deemed to be in effect 
under the legislation. A sponsor that received a deferral under the 
rule would have the original deferral date extended by the number of 
days between October 17, 2002, and the date of enactment of this 
legislation.
  A sponsor that submitted an application in the time period between 
April 1, 1999, and the date of enactment of this legislation that was 
not granted a waiver or deferral under the rule would be required to 
submit pediatric assessments unless granted a waiver by FDA. However, 
no submission by a sponsor would be due until 12 months after the date 
of enactment of this legislation or until a date specified by FDA under 
section 505B(a)(3), whichever is later.
  Mr. KENNEDY. Mr. President, Although I and the Democratic sponsors

[[Page S9816]]

of the bill disagree with the chairman's view that the agency lacked 
the authority to promulgate the Pediatric Rule and his view that the 
Federal district court ruling invalidating the rule was correct, we do 
agree with the chairman's statements regarding the need to apply the 
requirements of this legislation retroactively to ensure that no 
pediatric studies are lost in the transition from the rule to this 
legislation.
  Mr. GREGG. Mr. President, S. 650 provides FDA the statutory authority 
to require that new and already marketed drugs and biological products 
be studied in children in specified circumstances. This authority is 
intended to work in a complementary fashion with pediatric exclusivity. 
With regard to already marketed products, S. 650 provides that FDA 
require pediatric testing only after pediatric exclusivity and the 
National Institutes of Health grant and contract provisions contained 
in sections 409I and 499 of the Public Health service Act have failed 
to produce the necessary studies. However, nothing in S. 650 requires 
FDA to wait until the voluntary mechanisms have failed or been 
exhausted before invoking the pediatric studies requirement for new 
drug applications under section 505 of the Federal Food, drug and 
Cosmetic Act or biological license applications under section 351 of 
the Public Health service Act. On the contrary, S. 650 creates the 
presumption that new drugs and biologics will be studied before 
approval unless a waiver or deferral is granted.
  Mr. KENNEDY. Mr. President, I agree with the Senator. Does he agree 
as well, that, in accordance with the plain language of the 
legislation, FDA shall grant a waiver of the requirement to submit 
pediatric assessments only if the applicant certifies and the Secretary 
finds that the conditions specified in 505B(a)(4) and 505B(b)(2) exist? 
By using the word ``including'' before listing the circumstances under 
which FDA shall grant a full or partial waiver in the committee report 
for S. 650, the committee does not intend that any conditions or 
circumstances other than those specifically stated in 505B(a)(4) and 
505B(b)(2) serve as the basis for FDA granting a full or partial waiver 
of the requirements of the legislation.
  Mr. GREGG. Mr. President, I do, and I thank the Senator for his work 
on this bill and the report.
  The bill (S. 650), as amended, was read the third time and passed as 
follows:

                                 S. 650

       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 ``Pediatric Research Equity 
     Act of 2003''.

     SEC. 2. RESEARCH INTO PEDIATRIC USES FOR DRUGS AND BIOLOGICAL 
                   PRODUCTS.

       (a) In General.--Subchapter A of chapter V of the Federal 
     Food, Drug, and Cosmetic Act (21 U.S.C. 351 et seq.) is 
     amended by inserting after section 505A the following:

     ``SEC. 505B. RESEARCH INTO PEDIATRIC USES FOR DRUGS AND 
                   BIOLOGICAL PRODUCTS.

       ``(a) New Drugs and Biological Products.--
       ``(1) In general.--A person that submits an application (or 
     supplement to an application)--
       ``(A) under section 505 for a new active ingredient, new 
     indication, new dosage form, new dosing regimen, or new route 
     of administration; or
       ``(B) under section 351 of the Public Health Service Act 
     (42 U.S.C. 262) for a new active ingredient, new indication, 
     new dosage form, new dosing regimen, or new route of 
     administration;
     shall submit with the application the assessments described 
     in paragraph (2).
       ``(2) Assessments.--
       ``(A) In general.--The assessments referred to in paragraph 
     (1) shall contain data, gathered using appropriate 
     formulations for each age group for which the assessment is 
     required, that are adequate--
       ``(i) to assess the safety and effectiveness of the drug or 
     the biological product for the claimed indications in all 
     relevant pediatric subpopulations; and
       ``(ii) to support dosing and administration for each 
     pediatric subpopulation for which the drug or the biological 
     product is safe and effective.
       ``(B) Similar course of disease or similar effect of drug 
     or biological product.--
       ``(i) In general.--If the course of the disease and the 
     effects of the drug are sufficiently similar in adults and 
     pediatric patients, the Secretary may conclude that pediatric 
     effectiveness can be extrapolated from adequate and well-
     controlled studies in adults, usually supplemented with other 
     information obtained in pediatric patients, such as 
     pharmacokinetic studies.
       ``(ii) Extrapolation between age groups.--A study may not 
     be needed in each pediatric age group if data from 1 age 
     group can be extrapolated to another age group.
       ``(3) Deferral.--On the initiative of the Secretary or at 
     the request of the applicant, the Secretary may defer 
     submission of some or all assessments required under 
     paragraph (1) until a specified date after approval of the 
     drug or issuance of the license for a biological product if--
       ``(A) the Secretary finds that--
       ``(i) the drug or biological product is ready for approval 
     for use in adults before pediatric studies are complete;
       ``(ii) pediatric studies should be delayed until additional 
     safety or effectiveness data have been collected; or
       ``(iii) there is another appropriate reason for deferral; 
     and
       ``(B) the applicant submits to the Secretary--
       ``(i) certification of the grounds for deferring the 
     assessments;
       ``(ii) a description of the planned or ongoing studies; and
       ``(iii) evidence that the studies are being conducted or 
     will be conducted with due diligence and at the earliest 
     possible time.
       ``(4) Waivers.--
       ``(A) Full waiver.--On the initiative of the Secretary or 
     at the request of an applicant, the Secretary shall grant a 
     full waiver, as appropriate, of the requirement to submit 
     assessments for a drug or biological product under this 
     subsection if the applicant certifies and the Secretary finds 
     that--
       ``(i) necessary studies are impossible or highly 
     impracticable (because, for example, the number of patients 
     is so small or the patients are geographically dispersed);
       ``(ii) there is evidence strongly suggesting that the drug 
     or biological product would be ineffective or unsafe in all 
     pediatric age groups; or
       ``(iii) the drug or biological product--

       ``(I) does not represent a meaningful therapeutic benefit 
     over existing therapies for pediatric patients; and
       ``(II) is not likely to be used in a substantial number of 
     pediatric patients.

       ``(B) Partial waiver.--On the initiative of the Secretary 
     or at the request of an applicant, the Secretary shall grant 
     a partial waiver, as appropriate, of the requirement to 
     submit assessments for a drug or biological product under 
     this subsection with respect to a specific pediatric age 
     group if the applicant certifies and the Secretary finds 
     that--
       ``(i) necessary studies are impossible or highly 
     impracticable (because, for example, the number of patients 
     in that age group is so small or patients in that age group 
     are geographically dispersed);
       ``(ii) there is evidence strongly suggesting that the drug 
     or biological product would be ineffective or unsafe in that 
     age group;
       ``(iii) the drug or biological product--

       ``(I) does not represent a meaningful therapeutic benefit 
     over existing therapies for pediatric patients in that age 
     group; and
       ``(II) is not likely to be used by a substantial number of 
     pediatric patients in that age group; or

       ``(iv) the applicant can demonstrate that reasonable 
     attempts to produce a pediatric formulation necessary for 
     that age group have failed.
       ``(C) Pediatric formulation not possible.--If a waiver is 
     granted on the ground that it is not possible to develop a 
     pediatric formulation, the waiver shall cover only the 
     pediatric groups requiring that formulation.
       ``(D) Labeling requirement.--If the Secretary grants a full 
     or partial waiver because there is evidence that a drug or 
     biological product would be ineffective or unsafe in 
     pediatric populations, the information shall be included in 
     the labeling for the drug or biological product.
       ``(b) Marketed Drugs and Biological Products.--
       ``(1) In general.--After providing notice in the form of a 
     letter and an opportunity for written response and a meeting, 
     which may include an advisory committee meeting, the 
     Secretary may (by order in the form of a letter) require the 
     holder of an approved application for a drug under section 
     505 or the holder of a license for a biological product under 
     section 351 of the Public Health Service Act (42 U.S.C. 262) 
     to submit by a specified date the assessments described in 
     subsection (a)(2) if the Secretary finds that--
       ``(A)(i) the drug or biological product is used for a 
     substantial number of pediatric patients for the labeled 
     indications; and
       ``(ii) the absence of adequate labeling could pose 
     significant risks to pediatric patients; or
       ``(B)(i) there is reason to believe that the drug or 
     biological product would represent a meaningful therapeutic 
     benefit over existing therapies for pediatric patients for 1 
     or more of the claimed indications; and
       ``(ii) the absence of adequate labeling could pose 
     significant risks to pediatric patients.
       ``(2) Waivers.--
       ``(A) Full waiver.--At the request of an applicant, the 
     Secretary shall grant a full waiver, as appropriate, of the 
     requirement to submit assessments under this subsection if 
     the applicant certifies and the Secretary finds that--
       ``(i) necessary studies are impossible or highly 
     impracticable (because, for example, the number of patients 
     in that age group is so small or patients in that age group 
     are geographically dispersed); or
       ``(ii) there is evidence strongly suggesting that the drug 
     or biological product would be

[[Page S9817]]

     ineffective or unsafe in all pediatric age groups.
       ``(B) Partial waiver.--At the request of an applicant, the 
     Secretary shall grant a partial waiver, as appropriate, of 
     the requirement to submit assessments under this subsection 
     with respect to a specific pediatric age group if the 
     applicant certifies and the Secretary finds that--
       ``(i) necessary studies are impossible or highly 
     impracticable (because, for example, the number of patients 
     in that age group is so small or patients in that age group 
     are geographically dispersed);
       ``(ii) there is evidence strongly suggesting that the drug 
     or biological product would be ineffective or unsafe in that 
     age group;
       ``(iii)(I) the drug or biological product--

       ``(aa) does not represent a meaningful therapeutic benefit 
     over existing therapies for pediatric patients in that age 
     group; and
       ``(bb) is not likely to be used in a substantial number of 
     pediatric patients in that age group; and

       ``(II) the absence of adequate labeling could not pose 
     significant risks to pediatric patients; or
       ``(iv) the applicant can demonstrate that reasonable 
     attempts to produce a pediatric formulation necessary for 
     that age group have failed.
       ``(C) Pediatric formulation not possible.--If a waiver is 
     granted on the ground that it is not possible to develop a 
     pediatric formulation, the waiver shall cover only the 
     pediatric groups requiring that formulation.
       ``(D) Labeling requirement.--If the Secretary grants a full 
     or partial waiver because there is evidence that a drug or 
     biological product would be ineffective or unsafe in 
     pediatric populations, the information shall be included in 
     the labeling for the drug or biological product.
       ``(3) Relationship to other pediatric provisions.--
       ``(A) No assessment without written request.--No assessment 
     may be required under paragraph (1) for a drug subject to an 
     approved application under section 505 unless--
       ``(i) the Secretary has issued a written request for a 
     related pediatric study under section 505A(c) of this Act or 
     section 409I of the Public Health Service Act (42 U.S.C. 
     284m);
       ``(ii)(I) if the request was made under section 505A(c)--

       ``(aa) the recipient of the written request does not agree 
     to the request; or
       ``(bb) the Secretary does not receive a response as 
     specified under section 505A(d)(4)(A); or

       ``(II) if the request was made under section 409I of the 
     Public Health Service Act (42 U.S.C. 284m)--

       ``(aa) the recipient of the written request does not agree 
     to the request; or
       ``(bb) the Secretary does not receive a response as 
     specified under section 409I(c)(2) of that Act; and

       ``(iii)(I) the Secretary certifies under subparagraph (B) 
     that there are insufficient funds under sections 409I and 499 
     of the Public Health Service Act (42 U.S.C. 284m, 290b) to 
     conduct the study; or
       ``(II) the Secretary publishes in the Federal Register a 
     certification that certifies that--

       ``(aa) no contract or grant has been awarded under section 
     409I or 499 of the Public Health Service Act (42 U.S.C. 284m, 
     290b); and
       ``(bb) not less than 270 days have passed since the date of 
     a certification under subparagraph (B) that there are 
     sufficient funds to conduct the study.

       ``(B) No agreement to request.--Not later than 60 days 
     after determining that no holder will agree to the written 
     request (including a determination that the Secretary has not 
     received a response specified under section 505A(d) of this 
     Act or section 409I of the Public Health Service Act (42 
     U.S.C. 284m), the Secretary shall certify whether the 
     Secretary has sufficient funds to conduct the study under 
     section 409I or 499 of the Public Health Service Act (42 
     U.S.C. 284m, 290b), taking into account the prioritization 
     under section 409I.
       ``(c) Meaningful Therapeutic Benefit.--For the purposes of 
     paragraph (4)(A)(iii)(I) and (4)(B)(iii)(I) of subsection (a) 
     and paragraphs (1)(B)(i) and (2)(B)(iii)(I)(aa) of subsection 
     (b), a drug or biological product shall be considered to 
     represent a meaningful therapeutic benefit over existing 
     therapies if the Secretary estimates that--
       ``(1) if approved, the drug or biological product would 
     represent a significant improvement in the treatment, 
     diagnosis, or prevention of a disease, compared with marketed 
     products adequately labeled for that use in the relevant 
     pediatric population; or
       ``(2) the drug or biological product is in a class of 
     products or for an indication for which there is a need for 
     additional options.
       ``(d) Submission of Assessments.--If a person fails to 
     submit an assessment described in subsection (a)(2), or a 
     request for approval of a pediatric formulation described in 
     subsection (a) or (b), in accordance with applicable 
     provisions of subsections (a) and (b)--
       ``(1) the drug or biological product that is the subject of 
     the assessment or request may be considered misbranded solely 
     because of that failure and subject to relevant enforcement 
     action (except that the drug or biological product shall not 
     be subject to action under section 303); but
       ``(2) the failure to submit the assessment or request shall 
     not be the basis for a proceeding--
       ``(A) to withdraw approval for a drug under section 505(e); 
     or
       ``(B) to revoke the license for a biological product under 
     section 351 of the Public Health Service Act (42 U.S.C. 262).
       ``(e) Meetings.--Before and during the investigational 
     process for a new drug or biological product, the Secretary 
     shall meet at appropriate times with the sponsor of the new 
     drug or biological product to discuss--
       ``(1) information that the sponsor submits on plans and 
     timelines for pediatric studies; or
       ``(2) any planned request by the sponsor for waiver or 
     deferral of pediatric studies.
       ``(f) Scope of Authority.--Nothing in this section provides 
     to the Secretary any authority to require a pediatric 
     assessment of any drug or biological product, or any 
     assessment regarding other populations or uses of a drug or 
     biological product, other than the pediatric assessments 
     described in this section.
       ``(g) Orphan Drugs.--Unless the Secretary requires 
     otherwise by regulation, this section does not apply to any 
     drug for an indication for which orphan designation has been 
     granted under section 526.
       ``(h) Integration With Other Pediatric Studies.--The 
     authority under this section shall remain in effect so long 
     as an application subject to this section may be accepted for 
     filing by the Secretary on or before the date specified in 
     section 505A(n).''.
       (b) Conforming Amendments.--
       (1) Section 505(b)(1) of the Federal Food, Drug, and 
     Cosmetic Act (21 U.S.C. 355(b)(1)) is amended in the second 
     sentence--
       (A) by striking ``and (F)'' and inserting ``(F)''; and
       (B) by striking the period at the end and inserting ``, and 
     (G) any assessments required under section 505B.''.
       (2) Section 505A(h) of the Federal Food, Drug, and Cosmetic 
     Act (21 U.S.C. 355a(h)) is amended--
       (A) in the subsection heading, by striking ``Regulations'' 
     and inserting ``Pediatric Research Requirements''; and
       (B) by striking ``pursuant to regulations promulgated by 
     the Secretary'' and inserting ``by a provision of law 
     (including a regulation) other than this section''.
       (3) Section 351(a)(2) of the Public Health Service Act (42 
     U.S.C. 262(a)(2)) is amended--
       (A) by redesignating subparagraph (B) as subparagraph (C); 
     and
       (B) by inserting after subparagraph (A) the following:
       ``(B) Pediatric studies.--A person that submits an 
     application for a license under this paragraph shall submit 
     to the Secretary as part of the application any assessments 
     required under section 505B of the Federal Food, Drug, and 
     Cosmetic Act.''.

     SEC. 3. TECHNICAL AND CONFORMING AMENDMENTS.

       (a) Abbreviated New Drug Application.--Section 505A of the 
     Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355a) is 
     amended in subparagraphs (A) and (B) of subsection (b)(2) and 
     subparagraphs (A) and (B) of subsection (c)(2) by striking 
     ``505(j)(4)(B)'' and inserting ``505(j)(5)(B)''.
       (b) Pediatric Advisory Committee.--
       (1) Section 505A(i)(2) of the Federal Food, Drug, and 
     Cosmetic Act (21 U.S.C. 355a(i)(2)) is amended by striking 
     ``Advisory Subcommittee of the Anti-Infective Drugs'' each 
     place it appears.
       (2) Section 14 of the Best Pharmaceuticals for Children Act 
     (42 U.S.C. 284m note; Public Law 107-109) is amended--
       (A) in the section heading, by striking ``PHARMACOLOGY'';
       (B) in subsection (a), by striking ``(42 U.S.C. 217a),'' 
     and inserting (42 U.S.C. 217a) or other appropriate 
     authority,'';
       (C) in subsection (b)--
       (i) in paragraph (1), by striking ``and in consultation 
     with the Director of the National Institutes of Health''; and
       (ii) in paragraph (2), by striking ``and 505A'' and 
     inserting ``505A, and 505B''; and
       (D) by striking ``pharmacology'' each place it appears and 
     inserting ``therapeutics''.
       (3) Section 15(a)(2)(A) of the Best Pharmaceuticals for 
     Children Act (115 Stat. 1419) is amended by striking 
     ``Pharmacology''.
       (4) Section 16(1)(C) of the Best Pharmaceuticals for 
     Children Act (21 U.S.C. 355a note; Public Law 107-109) is 
     amended by striking ``Advisory Subcommittee of the Anti-
     Infective Drugs''.
       (5) Section 17(b)(1) of the Best Pharmaceuticals for 
     Children Act (21 U.S.C. 355b(b)(1)) is amended in the second 
     sentence by striking ``Advisory Subcommittee of the Anti-
     Infective Drugs''.
       (6) Paragraphs (8), (9), and (11) of section 409I(c) of the 
     Public Health Service Act (42 U.S.C. 284m(c)) are amended by 
     striking ``Advisory Subcommittee of the Anti-Infective 
     Drugs'' each place it appears.

     SEC. 4. EFFECTIVE DATE.

       (a) In General.--Subject to subsection (b), this Act and 
     the amendments made by this Act take effect on the date of 
     enactment of this Act.
       (b) Applicability to New Drugs and Biological Products.--
       (1) In general.--Subsection (a) of section 505B of the 
     Federal Food, Drug, and Cosmetic Act (as added by section 2) 
     shall apply to an application described in paragraph (1) of 
     that subsection submitted to the Secretary of Health and 
     Human Services on or after April 1, 1999.
       (2) Waivers and deferrals.--
       (A) Waiver or deferral granted.--If, with respect to an 
     application submitted to the Secretary of Health and Human 
     Services

[[Page S9818]]

     between April 1, 1999, and the date of enactment of this Act, 
     a waiver or deferral of pediatric assessments was granted 
     under regulations of the Secretary then in effect, the waiver 
     or deferral shall be a waiver or deferral under subsection 
     (a) of section 505B of the Federal Food, Drug, and Cosmetic 
     Act, except that any date specified in such a deferral shall 
     be extended by the number of days that is equal to the number 
     of days between October 17, 2002, and the date of enactment 
     of this Act.
       (B) Waiver and deferral not granted.--If, with respect to 
     an application submitted to the Secretary of Health and Human 
     Services between April 1, 1999, and the date of enactment of 
     this Act, neither a waiver nor deferral of pediatric 
     assessments was granted under regulations of the Secretary 
     then in effect, the person that submitted the application 
     shall be required to submit assessments under subsection 
     (a)(2) of section 505B of the Federal Food, Drug, and 
     Cosmetic Act on the date that is the later of--
       (i) the date that is 1 year after the date of enactment of 
     this Act; or
       (ii) such date as the Secretary may specify under 
     subsection (a)(3) of that section;
     unless the Secretary grants a waiver under subsection (a)(4) 
     of that section.
       (c) No Limitation of Authority.--Neither the lack of 
     guidance or regulations to implement this Act or the 
     amendments made by this Act nor the pendency of the process 
     for issuing guidance or regulations shall limit the authority 
     of the Secretary of Health and Human Services under, or defer 
     any requirement under, this Act or those amendments.
  Mr. DODD. Mr. President, I rise today to applaud my colleagues for 
passing the Pediatric Research Equity Act of 2003, and to thank all of 
those who have worked so hard on this issue. This legislation 
represents a truly bipartisan compromise, and I deeply appreciate the 
commitment to this issue shown by Senators DeWine, Clinton, Gregg, and 
Kennedy. I also acknowledge the invaluable role played by the American 
Academy of Pediatrics and the Elizabeth Glaser Pediatric AIDS 
Foundation.
  Quite simply, this legislation will make our children safer. It will 
ensure that they have access to prescription drugs that have been 
properly evaluated for their use. It will remove the guesswork often 
done by pediatricians about what drugs are appropriate for a child, and 
in what dosages. And it will accomplish all of this by codifying into 
statutory language a tool that has already been shown to be effective: 
the Pediatric Rule.
  The Pediatric Rule went into effect in April of 1999 and was intended 
to work in conjunction with a voluntary incentives program that 
Congress passed in 1997 and was reauthorized last year. Both the 
incentives program and the rule were put into place to address an unmet 
need that had potentially serious consequences to the health of 
children.
  Children are not just small versions of adults when it comes to 
drugs. Some drugs that are completely safe for adults may be very 
harmful to children. In addition, some needed drugs are not available 
in a formulation that a child can take, such as a liquid or chewable 
tablet. Finally, the appropriate dosage for a child cannot be 
determined simply by extrapolating from adults. Yet, until the rule and 
the incentives program were enacted, this is exactly what pediatricians 
were forced to do. Roughly 75 percent of all prescription drugs on the 
market today have never been properly tested for safe use by children.
  As a result, children have suffered needlessly. For example, new 
tests on the epilepsy drug Neurontin have shown that higher dosages 
than expected are needed for children under 5. For years, pediatricians 
simply believed that Neurontin was a drug that was ineffective for 
children.
  In 1997, Congress enacted legislation, introduced by Senator DeWine 
and myself, to provide drug companies with an economic incentive to 
test their products to ensure their safety in children. This was 
followed by enactment of the Pediatric Rule in 1999, which worked with 
the incentive by giving the Food and Drug Administration (FDA) the 
authority to require that drugs and biologics important to children be 
tested and formulated for their use.
  Working as complements to each other, the rule and the incentive 
provided tremendous results. Between April 1999 and March 2002, 
research was completed on the safety and effectiveness in children of 
roughly 100 days. These medicines were for the treatment of, among 
other things, HIV/AIDS, diabetes, asthma pain and arthritis. In 
addition, studies of hundreds more drugs are in the pipeline.
  But continued success of this magnitude is dependent on the existence 
of both the rule and the incentive program. FDA has stated that 
approximately half of the completed studies were due to the authority 
provided by the Pediatric Rule.
  Unfortunately, in October of last year, the U.S. District Court for 
the District of Columbia ruled that FDA does not have the authority to 
enforce the rule. This decision represented a step backwards for 
children's health. We can hardly afford to do without the rule when we 
still do not have necessary information for kids for a majority of the 
medicines on the market.
  The legislation that we passed today will give the FDA clear 
authority to require that drugs be tested and formulated for children. 
Companies should continue to have access to voluntary incentives, but 
the rule must be in place to ensure that as many products as possible 
are studied for use in children.
  For example, the rule captures a class of products, specifically 
biologics, for which market exclusivity incentives do not apply. There 
are a number of biologic products used to treat cancer in children for 
which information about their specific use--safety and efficacy--in 
kids would be vital. Only the rule would apply here.
  The rule can also be applied as needed during the life of a drug as 
more information is required. For example, if a new use of a drug is 
discovered and safety or dosing information for that new use is needed. 
Exclusivity can only be applied once, even if an important new use for 
a product is found. Also, because the incentives are voluntary, for any 
number of reasons a manufacturer may choose not to conduct the 
necessary testing. Without the rule there is no way to guarantee that a 
drug that may be critically important to children's health is tested.
  I would be remiss if I did not mention one provision in this 
legislation with which I disagree. As a result of this provision, the 
authority that we clearly provide to FDA with this bill will sunset in 
2007. While I believe that FDA has the authority to enforce the rule 
even without this legislation, that has clearly been called into 
question given the District Court ruling. Therefore, it is imperative 
that we unequivocally and permanently provide the FDA with statutory 
authority to require pediatric testing. Unfortunately, as it now stands 
that critical authority will expire in 2007 unless reauthorized.
  It is my view that such a reauthorization should not be necessary. We 
take it for granted that studies will be done to assure that the drugs 
that adults use are safe and effective. Why should the assumption be 
any different for children? FDA should always have the authority to 
make sure that the drugs that kids use have been tested for their use. 
This is not something that Congress should have to reauthorize every 5 
years. Kids should not have to come back to Congress every 5 years to 
fight for the basic right to safe drugs.
  Despite my concern with the sunset provision, I strongly support this 
bill. The voluntary program has been a huge success, but its 
limitations can be addressed by passage of this legislation. Simply 
put, taking any tool off the table that promotes pediatric testing is 
at odds with our overarching goal of ensuring that medicines are safe 
and available for our children. That is why we must protect the rule 
and ensure that our efforts for kids will not be diminished. The 
Pediatric Research Equity Act of 2003 will do exactly that.
  I sincerely hope that the House will pass this bill as soon as 
possible, preferably without any changes so that we can send it to the 
President to be signed into law without delay.
  Mrs. CLINTON. Mr. President, I rise to mark the passage on the Senate 
floor of a bill, S. 650, that will assure the safety and efficacy of 
medicines for children, and address a problem that pediatricians, 
parents, and children's advocates have worked on for decades. A great 
deal of work went into this bill. So many hardworking, dedicated 
Senators made the effort on a bipartisan basis to come together around 
this important issue. In particular I want to thank Senators DeWine, 
Dodd, Gregg, and Kennedy. Senators DeWine and Dodd and I now have 
worked on pediatric research for many years, and we

[[Page S9819]]

will continue to be around to work on behalf of children, who, without 
dedicated advocates like Senators DeWine and Dodd, would not have a 
political voice.
  Last year this bill was passed out of committee but held up on the 
floor toward the end of session. Unfortunately, that meant no backstop 
was in place to assure the continuation of a minimum baseline 
protection for children when last October, a District Court judge 
struck down the 1998 FDA Pediatric Rule, based on his view that 
Congress did not intend to charge FDA with making sure our children are 
protected. Today, we pass legislation to clarify that FDA authority to 
assure safe, effective medicines for children is exactly what we 
intend.
  This bill was the product of compromise. We all worked hard and made 
concessions on all sides to craft the language the Senate was able to 
pass today. Some of us would have preferred a strong, permanent 
assurance for children, and not a sunset of these crucial protections 
in 2007. Indeed, because the purpose of this legislation was to address 
the uncertainty caused by the court-triggered lapse of pediatric 
studies, not codify such a lapse into statute, I cannot support the 
sunset provision.
  But others may have wished to change other aspects of the bill. So we 
were able to give on each side for the sake of moving forward on a 
central accomplishment providing FDA with undisputed, unencumbered 
authority to require and enforce studies of whether medicines important 
for children are also safe and effective for children. Our managers' 
amendment and the colloquy we submitted today reinforce that as the 
goal we all share here today in passing this language.
  I want to take a moment to bring special attention to the amount of 
work and cooperation that the chair and ranking member of Senate Health 
Education, Labor, and Pensions Committee have dedicated to this bill, 
both last Congress and this Congress. Senator Gregg and Senator 
Kennedy, and both their staffs, Vince Ventimiglia, and David Dorsey 
have lent their expertise and their time to this issue. Senator 
DeWine's staff, Abby Kral, and Senator Dodd's staff, Ben Berwick this 
year, Debra Barrett last year, have been more dedicated than anyone on 
this issue.
  I particularly want to acknowledge the outside experts who have 
devoted so much time to advocating on behalf of children and making 
this proposal a reality. The American Academy of Pediatricians, Elaine 
Vining here in DC and all the pediatricians across the country, have 
been championing this issue for so long. Also, Mark Isaac and Jeanne 
Ireland at the Elizabeth Glaser Pediatric AIDS Foundation have been 
tireless in their efforts. The children's hospitals, and so many others 
cannot be thanked enough. We would not be here today without their 
passionate advocacy. I also appreciate working with Phrma to get to 
this point and hope to continue to work with them in order to move this 
bill quickly into law.

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