News: National Children's Study Postponed; Priority Review Vouchers Defined
Today, we bring you two recent news items of significance for children with cancer -- one from the National Institutes of Health and the other from the Food and Drug Administration: National Children’s Study Postponed Indefinitely
Advisors to the National Institutes of Health recently cancelled the National Children’s Study, after a pilot and a 14-year debate about its design and feasibility. The longitudinal documentation of environmental factors, chemical exposures and psychosocial influences, among other factors, was planned for a sample of 100,000 US children. The study could have yielded much-needed data about whether there are reliable environmental causes of childhood cancer. Dr. Francis Collins, Director of the NIH, is dissembling the NCS study office and reallocating its $165 million allocation. A report from the IOM in June, chaired by Dr. Philip Pizzo, Stanford-based childhood cancer expert, reaffirmed the aims of the NCS and called for a new design, new recruitment strategy and a smaller budget.
FDA Clarifies How Voucher Program Works
A Draft Guidance was recently released defining how and when drug companies can apply for Rare Pediatric Disease Priority Review Vouchers. FDA’s definition of a rare pediatric disease requires that more than 50% of the prevalence of the disease occurs in children (birth to age 18). FDA’s detailed FAQs allow sponsors to include adolescents and young adults with a disease if the overall prevalence is less than 200,000 -- FDA’s general definition of ‘rare.’ This provision is especially important for the potential treatment of AYAs with pediatric cancers. Drugs that qualify also must be brand new compounds whose use will not be extended to adults. The voucher program, originally called the Creating Hope Act, was enacted in 2012 as part of FDASIA (Food and Drug Administration Safety and Innovation Act).