[Congressional Record (Bound Edition), Volume 147 (2001), Part 14]
[Senate]
[Pages 20292-20293]
[From the U.S. Government Publishing Office, www.gpo.gov]



                 BEST PHARMACEUTICALS FOR CHILDREN ACT

  Mr. DeWINE. One of the things that passed today was a bill that 
Senator Dodd and I have been working on for some time. Senator Dodd 
talked a little bit about it on the Senate floor earlier today. This 
bill is S. 838, the Best Pharmaceuticals for Children Act.
  This is reauthorization legislation which Senator Dodd and I wrote to 
ensure that more medicines are tested for children and that useful 
prescribing and dosing information appears on labels.
  Let me take a moment on a personal note to congratulate my friend, 
Senator Dodd, and his wife Jackie on the recent birth of their daughter 
Grace. I had the opportunity a couple of days ago when Senator Dodd and 
his wife Jackie brought baby Grace into the Capitol to see baby Grace, 
a beautiful child--a great joy. So our congratulations go to both of 
them.
  It is appropriate that the first piece of legislation that Senator 
Dodd passed after the birth of his little girl was a bill that will 
help children, a bill that will make sure that good pharmaceuticals are 
available for children and that doctors, specifically pediatricians, 
and parents will know what the dosage for each medicine should be for 
their particular child, for the age of that child.
  Four years ago, Senator Dodd and I first learned that the vast 
majority of drugs in this country that came on the market every week--
in fact over 80 percent--had never been formally tested or approved for 
pediatric use and therefore lacked even the most basic labeling 
information regarding dosing recommendations for children. When we 
found that out, we began writing what is now referred to as the 
pediatric exclusivity law. That bill passed. In the 3 years since that 
law went into effect, the FDA has issued about 200 written requests for 
pediatric studies.
  Companies have undertaken over 400 pediatric studies, of which over 
58 studies have been completed, for a wide range of critical diseases, 
including juvenile diabetes, the problem of pain, asthma, and 
hypertension.
  Mr. President, 37 drugs have been granted pediatric exclusivity. Some 
studies generated by this incentive have led to essential dosing 
information; for example, Luvox. Luvox is a drug prescribed to treat 
obsessive-compulsive disorder. Pediatric studies performed pursuant to 
our law have shown inadequate dosing for adolescents, which resulted in 
ineffective treatment. The studies also have shown that some girls 
between the ages of 8 and 11 were potentially overdosed, with levels up 
to 2 to 3 times that which was really needed.
  Since our law has been in effect, the private sector has increased 
its investment in pediatric training and developing a infrastructure to 
support and expand pediatric research. The FDA stated in a January 2001 
report:

       The pediatric exclusivity provision has done more to 
     generate clinical studies and useful prescribing information 
     for the pediatric population than any other regulatory or 
     legislative process to date.

  The bill this Senate and House passed 3 years ago has done a great 
deal of good. We are seeing more drugs for children on the market that 
have a label that tells how they can be used, and more basic 
information for pediatricians. So when they look at that little child 
and they know the age of that child and they know the weight of that 
child, they can look it up and see exactly what the prescription should 
be, what the dosage should be, what the indicators are. They can do 
that because we have given the pharmaceutical companies an incentive to 
do that research, research they were doing prior to passage of this 
bill in only 20 percent of the cases.
  A great deal of progress has been made, but we have further to go. 
That is what we were about today with the passage of the bill that I am 
now describing. Senator Dodd and I and the other cosponsors knew that 
the law we passed 3 years ago could be improved. We knew that it had 
some holes in it. We set out to improve that, to fill the gaps, and 
address the outstanding issues, such as the testing of off-patent 
drugs, which the original law was never designed to include. It is 
understandable why the original law wasn't designed to include off-
patent drugs. The original law extended the patent by 6 months. They 
extend it for 6 months if and only if they tested these drugs for 
children.
  If a drug is not on-patent, if it is off-patent, the patent has 
basically expired, obviously that incentive doesn't do any good. What 
we tried to do with this bill that we passed today was to change that 
and therefore expand it and expand the purpose of this bill to include 
off-patent drugs as well.
  For some products and some age groups, the existing market incentives 
are simply inadequate to encourage new pediatric research. In the bill 
we passed several hours ago, we have built upon the existing law's 
basic incentive structure to further ensure that these essential 
products, and young age groups, are included within the scope of the 
program.
  To make perfectly clear the need for additional legislation, I would 
like to quote a significant passage from the FDA's January 2001 report, 
which stated the following:

       A majority of marketed drugs are not labeled for use in 
     pediatric patients, or are labeled for use only in specific 
     pediatric age groups . . . And many of the drugs most widely 
     used in pediatric patients carry disclaimers in their 
     labeling stating that safety and effectiveness in pediatric 
     patients have not been established. The absence of pediatric 
     labeling information poses significant risks for children. 
     Inadequate dosing information exposes pediatric patients to 
     the risk of adverse reactions, usually age-specific adverse 
     reactions that could be avoided if such information were 
     provided in product labeling. The absence of pediatric 
     testing and labeling may also expose pediatric patients to 
     ineffective treatment through underdosing, or may deny 
     pediatric patients therapeutic advances because physicians 
     choose to prescribe existing, less effective medications in 
     the face of insufficient pediatric information about a new 
     medication.

  These facts are very disturbing. Through our bill, we have sought to 
find a way to improve the labeling process. Since our law has not been 
implemented for very long, many labels are still in the process of 
being requested and negotiated by the FDA. In this new bill, the new 
timeframes established in the bill for labeling negotiations, together 
with the enforcement authority under the existing misbranding statute, 
will help to ensure that essential pediatric information generated from 
studies implemented under this law, will result in necessary and timely 
labeling changes.
  Our bill establishes timeframes for responding to written requests, 
timeframes and processes for negotiating label changes, and authorizes 
the federal government to deem a drug misbranded if the company refuses 
to relabel its drug. The government would then begin an enforcement 
action under its existing authority to seek a court order regarding the 
relabeling of the drug.
  Through the bill that we are about to pass today, we will ensure that 
priority drugs which lack patent or other market exclusivity will be 
tested for children. For example, the Ritalin label states the 
following:

       Precautions: Long-term effects of Ritalin in children have 
     not been well established. Warning: Ritalin should not be 
     used in children under six years since safety and 
     [effectiveness] in this age group has not been established.

  The point is that Ritalin is being prescribed off-label for children 
under six years of age, and yet we do not know the safety and 
effectiveness, since it has only been tested in children older than 
six, and we do not know long-term effects on children of any age.
  Our bill creates a mechanism to ``capture'' the off-patent drugs for

[[Page 20293]]

which the Secretary determines additional studies are needed to assess 
the safety and effectiveness of the drug's use in the pediatric 
population.
  In other words, our bill provides for the testing of some cases of 
these off-patent drugs.
  By expanding the mission of the existing NIH Foundation to include 
collecting and awarding grants for conducting certain pediatric 
studies, we have provided a funding mechanism for ensuring studies that 
are completed for both off-patent drugs and those marketed on-patent 
drugs that a company declines to study--and for which the Secretary 
determines there is a continuing need for information relating to the 
use of the drug in the pediatric population.
  That is the language in the bill. That is the correct area.
  By first seeking funding through the Foundation, we provide a 
mechanism for drug companies to contribute to the funding of mainly 
off-patent drugs and also to a narrow group of on-patent drugs, 
including those for neonates, for which companies have declined to 
accept the written request to pursue the six month market exclusivity 
extension.
  The Neonates, of course, are young children up to one-month of age.
  If the Foundation lacks the funds to study that prioritized drug, the 
Secretary may then issue a request for proposal--``RFP''--for a third 
party to study the commercially available drug using money from a 
Research Fund that we create in this bill. The Secretary may then 
publish the name of the company that declined to study the drug, the 
name of the drug, and the indication or use that is being requested to 
be studied. This would ensure that more data is collected and reported, 
so that we can better understand which drugs are not being studied.
  A condition of the RFP or contract with a third party is that all 
data and information generated from the pediatric study in the form of 
a report must be submitted to the NIH and the FDA. The FDA must then 
review the report and data and negotiate whatever labeling changes the 
FDA determines is appropriate.
  I thank Senator Bond for his determined focus on helping to further 
ensure that neonates also benefit from this pediatric testing law. I 
congratulate and thank him. We have included neonates in the definition 
of ``pediatric studies'' to which this pediatric exclusivity applies. 
Throughout the bill we have also encouraged the inclusion of neonates 
in written requests, when appropriate.
  To further ensure that the safety of children in clinical trials is 
protected, this bill requires that the Institute of Medicine--IOM--
conduct a review of federal regulations, reports, and research 
involving children and provide recommendations on best practices 
relating to research involving children. The IOM is to consider the 
results of the study by HHS that Senator Dodd and I included as part of 
the Children's Health act last year. I look forward to working with 
Senators Dodd, Frist, and Kennedy on the issue of human subject 
protections, especially in focusing on protections of children 
participating in clinical trials.
  I want to thank my friend, Senator Dodd for his relentless efforts in 
making this reauthorization a reality, and for his relentlessness in 
improving the bill. I look forward to working on many more pediatric 
initiatives with him in the future.
  Let me also thank Senators Kennedy and Clinton for their strong 
support of this bill and of children's health overall. Let me also 
thank Senator Collins for her support and for her work in regard to 
this bill.
  I want to acknowledge and thank Debra Barrett, Jeanne Ireland, 
Christie Onoda, David Dorsey, David Nexon, Paul Kim, Christina Ho, John 
Gilman, and Tim Trushel for their hard work in helping us reach 
agreement on such a well-crafted bill. I cannot think of a bill that 
took more hard work, more Members and staff than this bill.
  I also extend my appreciation to Elaine Holland Vining with the 
American Academy of Pediatrics for the tenacious effort, technical 
assistance, and expertise she brought to this bill. She is expecting 
her first child shortly, and I wish her and her husband, Paul, my very 
best wishes as they begin their family.
  I also appreciate the diligent work of Mark Isaac and Natasha 
Bilimoria with the Elizabeth Glaser Pediatric AIDS Foundation in 
helping us negotiate and pass this important reauthorization.
  Finally, I must say a very special thanks to a former member of my 
staff, Helen Rhee, who is now working for Senator Frist on the HELP 
Committee. She has been absolutely instrumental in seeing this 
legislation through from its inception to its passage. Without her 
tireless efforts, her dogged determination, and a work ethic that is 
just unsurpassed, we would not be at this point today, we would not 
have seen this bill pass. Literally, right up until the last moment, 
literally, before the bill passed, Helen was continuing her work. So I 
pay tribute to her. This bill is a real tribute to her dedication and 
to her efforts.
  So I thank Helen and all the members of the different staffs who have 
worked so hard on this bill.
  Let me also take a moment to thank Senator Hatch and his staff, Bruce 
Artim, for their work in drafting language to correct and clarify this 
bill, specifically to clarify that pediatric exclusivity law is not and 
was never intended to eliminate incentives granted to generic drug 
manufacturers that are awarded 180 days of exclusivity under the 1984 
Hatch-Waxman law for successfully challenging a patent.
  Mr. President, I yield the floor.
  The PRESIDING OFFICER (Mr. Dayton). The Senator from Vermont.

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