Pediatric
Oncology Subcommittee of the Oncologic Drugs Advisory Committee
Questions to
Subcommittee
Off-patent oncology drugs for which pediatric
studies are needed:
availability of information concerning the safe and effective use of the drugs
in the pediatric population; whether additional information is needed; and
whether new pediatric studies concerning the drugs may produce health benefits
in the pediatric population, as mandated by the Best Pharmaceuticals for Children
Act (BPCA)
The BPCA of 2002 provides a
mechanism to study off-patent medications in pediatric populations.
1.
What factors
should be considered in selecting off-patent drugs for study in children with
cancer (these may include use in only a pediatric population, use in particular
diseases, use in particular age groups, or toxicity questions of particular
concern)?
2.
Are there any
comments, on the proposed selections as discussed by the National Cancer
Institute, on the drugs actinomycin-D and vincristine as priority choices, and others to follow?
3.
Are there any
other off-patent oncology drugs that should be studied in children with cancer
that you would suggest? Please indicate
the rationale.
Age-appropriate formulation changes
to facilitate dosing of products used in the pediatric
oncology setting
1.
What factors
would be considered essential in the development of a formulation for children
with cancer? Please comment on any age-,
disease-, or pharmaceutical-specific considerations.
2.
What type of
testing or clinical trial design would you recommend for establishing the
efficacy and safety of a new formulation for an existing oncology drug that
already has efficacy and safety demonstrated in the same population?
3.
What type of
testing or clinical trial design would you recommend for establishing the
efficacy and safety of a new formulation for an existing oncology drug that
already has efficacy and safety demonstrated in a different population?