May 9, 2002
Dockets Management Branch (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Room 1061
Rockville, MD 20852
RE: ANPRM Obtaining Timely Pediatric Studies of and
Adequate Pediatric Labeling for Human Drugs
and Biologics (Docket No. 02N-0152, 4-24-02)
To Whom It May Concern:
The National Organization for Rare Disorders (NORD) firmly believes that FDA must continue to enforce its 1998 mandatory requirement for all new drugs that are expected to be used by children, specifying that they must be studied in the pediatric population before the drug is approved for marketing in the United States. We believe that pediatric exclusivity afforded under BPCA is not enough to ensure that all drugs used by children will be properly tested in children, and therefore the mandatory pediatric testing requirement for all new drugs is needed.
NORD is a non-profit voluntary health organization dedicated to the identification, treatment and cure of rare “orphan” diseases. Under the Orphan Drug Act of 1983, a rare disorder is defined as a health condition affecting fewer than 200,000 Americans. The National Institutes of Health (NIH) estimates there are approximately 6,000 of these “orphan diseases” cumulatively affecting more than 25 million Americans. Most rare diseases are genetic, and many affect children.
Except for a few prevalent health conditions such as common infections, asthma, allergies, and Attention Deficit Disorder, most other serious health conditions are rare in children. For example, cystic fibrosis affects 25,000 people in the United States (most of whom are children), Duchenne Muscular Dystrophy affects 10,000 boys, there are 200 different types of cancer and some of them affect children, Hemophilia affects only 15,000 males, etc.
Many serious health conditions in children are treated with drugs that were studied and approved for adults, but not for children. If sales of a drug are modest, companies do not want to invest in pediatric clinical trials because six months of added exclusivity for that drug will not earn enough profit. Thus financial incentives such as pediatric exclusivity, do not work unless there is reason to believe a drug is extraordinarily profitable. Since most serious diseases of childhood are rare, expanding the market of an existing drug to a small pediatric population is not a significant lone incentive.
Dockets Management Branch (HFA-305)
Food and Drug Administration
Docket No. 02N-0152, 4-24-02
May 9, 2002
Page Two
The Best Pharmaceuticals for Children Act (BPCA) offers six months of exclusivity to companies that study the use of a drug in children, but it does not require manufacturers to add pediatric information to the drug’s label. This means in too many cases pediatricians do not have appropriate data for the treatment of children, even though a manufacturer is awarded the extra exclusivity. Additionally, companies tend to wait as long as possible before they perform pediatric studies, and such studies tend to be done only for the most profitable drugs rather than the medicines most needed by seriously ill children (e.g., lifesaving cancer drugs). If FDA requires all new drugs to be studied in children, and if the information is added to a drug’s label before it is launched, the information that pediatricians need will reduce adverse events and save lives. If such testing is not mandatory for all new drugs, manufacturers will often wait until a patent is about to expire before they test the drug in children, and they may not agree to re-label the drug for pediatrics since they will gain exclusivity anyway.
For the purposes of BPCA, we believe FDA should consider biologics and antibiotics to be “drugs” because they have the same effect on the human body by controlling, reducing or curing disease symptoms. The critical question is whether FDA is empowered to add pediatric information to pharmaceutical labeling, even if the manufacturer objects. As far as we are concerned, BPCA is totally ineffective if it does not require pediatric labeling because children cannot benefit if pediatricians are unaware of appropriate dosing or side effects. We firmly believe FDA should mandate pediatric labeling when clinical trials in children are done. Pediatric exclusivity should not be awarded until the drug is properly labeled for use in children.
FDA’s responsibilities are to enhance and protect the public health. Children are a critical part of the public. Their special medical needs must be met, including requiring necessary formulation changes for pediatric medicines. This will greatly reduce adverse events especially when children are treated with a drug or biologic that was developed for adults with a different diagnosis. Special formulations should be required when a specified drug is known to cause serious adverse events in children if the drug is prescribed in the wrong dosage.
Very truly yours,
Abbey S. Meyers
President
ASM:aa
cc: Diane Dorman, Vice President for Public Policy (Via Fax)
Senator Christopher Dodd, Att: Deb Barrett (Via Fax)
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