PhRMA Comes Out Against Additional Pediatric Trial Requirements
PhRMA has made clear it’s not in favor of any additional FDA requirements for pediatric trials of cancer drugs, at least for the time being, speaking out against policy changes during congressional testimony.
Stressing the importance of keeping incentives in place for developing rare disease drugs, Lucy Vereshchagina, vice president, science and regulatory advocacy for the industry trade group, called any changes or additions to pediatric cancer trial requirements “premature,” as relatively recently imposed requirements are assessed.
Specifically, the FDA Reauthorization Act of 2017 imposed new requirements that sponsors conduct pediatric trials of certain molecularly targeted cancer drugs if they’re evaluating them in adults and the drugs have the potential to address an unmet need in pediatric cancer patients. In conjunction, the legislation instructed the Government Accountability Office (GAO) to assess the effect of these requirements on drug development for pediatric cancer indications. With these provisions effective in August 2020, Vereshchagina contends there should be no new requirements until GAO issues its report in August 2023.
Vereshchagina’s comments could be in reference to legislation introduced by Reps. Michael McCaul (R-Tex.) and G.K. Butterfield (D-N.C.) that was discussed at a House Committee on Energy and Commerce health subcommittee hearing.
The legislation, which aims to help find treatments for relapsed pediatric cancers, would arm the FDA with the power to require trials that evaluate combinations of drugs. The lawmakers contend that although nearly all pediatric cancer trials involve children with relapsed cancer, the agency may currently only require pediatric cancer trials of single drugs, which rarely work for children whose cancers are advanced.