[Memo to Research Committee for the Presidential Advisory Council on HIV/AIDS from Alexandra M. Levine]

II. FUTURE RECOMMENDATION REGARDING ACCESS OF INFANTS AND CHILDREN
TO TREATMENT RESEARCH TRIALS, AND TO NEW ANTI-HIV AGENTS.
Art Ammann, the Chair for Research of the Pediatric AIDS Foundation, spoke to
the Committee members. First, he thanked the Committee for providing significant help in the
area of pediatric issues related to HIWAIDS. Thus, Dr. Amrnann was invited to the meeting with
VP Gore on 120/96, and stated that one of the issues which was laid out by Gore at the meeting
was the issue of pediatric AIDS, stating that the FDA must work with the pharmaceutical
companies to assure that children have access to the experimental and newly licensed drugs. Dr.
Ammann was also asked to testify before the Ritonavir meeting for consideration of licensure at
the FDA. A contingency for full licensure was placed on the drug, such that full approval would
not be granted until SAFETY AND PHARMACOKINETIC STUDIES in children be completed. ( A
contingency means that there is a "hold" on labelling claims, but does not hold up other
availability of the drug). Dr. Ammann also believes that the President's bold statements
regarding the potential to stop all new pediatric AIDS by 2000 was exceedingly helpful in
moving the pediatric HIV/AIDS agenda forward.
In terms of the language for a recommendation from our Committee in regards to
pediatric issues, Dr. Amman discussed two bills which are currently "in preparation". A bill
by Nancy Kassenbaum (R) is apparently going to be "inserted" into the FDA Reform Bill.
Although the particular legislation related to pediatric access to pharmaceuticals may be
satisfactory, the placement of this proposed legislation into the FDA Reform Bill is extremely
problematic, and may doom it to failure in the long run.
Another bill is currently being written by Tim Westmoreland for Congressman
Greenwood (0, Pennsylvania). The bill would be more general than pediatric access to drugs for
HIV/AIDS, and would Include language pertaining to all drugs in development, and tested in
adults. Incentive legislation is planned, in which a particular company would be given 3 to 6
months of additional exclusivity for the drug, IF safety and PK studies are performed in infants
and children, and if an oral suspension is made by the company for the drug in question. The
companies would be asked to present their pediatric plan for the drug in question.
The FDA (Kessler) has already stated that efficacy studies do NOT have to repeated
in children, if the drug has already proven efficacious in adults. Rather, specific safety and PK
data must be developed. At the current time, this latter area (safety and PK data) is NOT
mandated.
The potential opposition to the proposed legislation would come from those who
wish to keep medical costs down. Parenthetically, it is estimated that a 3-6 month additional
exclusivity for a drug such as Ritanovir would bring approximately 100 million dollars for the
company). Parenthetically as well, the "market exclusivity" does NOT contradict the GATT
accords, since the PATENT would not be extended, just the exclusivity, whioh would be handled
similar to the special consideratons given to orphan drugs.
Antoher potential issue related to the pediatric access legislation might be the
entire issue of mandated HIV testing of all pregnant women. At the moment, the govenrment is
requesting mandatory OFFERING OF THE TEST, with COUNSELING, and several states have
already passed this legislation (ex: California=Leslie Bill, which mandates OFFERING of the test