Fewer patients may be needed for some clinical trials, and other trials may be eliminated.
Priority review vouchers are awarded by FDA to drug companies that receive approval for a new drug (small-molecule or biologic) developed to treat a rare pediatric disease – a serious or life-threatening disease that mainly affects children from birth to 18 years old and is defined as a rare disease or condition under Section 529 of the Federal Food, Drug, and Cosmetic Act (FD&C Act). The vouchers can be used to receive priority review of a subsequent marketing application for a different product.
FDA has made a commitment to accelerating the development of treatments for rare pediatric diseases. To support that effort, the agency began developing a guidance document for sponsor in 2011 in collaboration with the European Medicines Agency (EMA). The draft guidance, Pediatric Rare Diseases: Collaborative Approach for Drug Development Using Gaucher Disease as Model was issued in early December 2017. The FDA’s expectation is that the principles outlined for Gaucher disease, a rare pediatric lysosomal storage disorder, can be to other rare pediatric diseases.
The draft guidance describes the use of controlled, multi-arm, multi-company clinical trials for the testing of multiple drug products in the same clinical trials, allowing for a reduction in the overall number of patients required to be treated with placebo. In addition, the use of available adult clinical data in combination with modeling and simulation to extrapolate and predict drug behaviors in children and adolescents is encouraged in the draft guidance. The approach allows for multiple products to be tested more efficiently.
Specifically, the guidance includes a description of the main inclusion criteria, relevant age groups, suggested efficacy endpoints, and study duration. It is stated in the draft: “Although such a program can be very challenging, the aim of the strategic plan is not only to facilitate agreement on individual applications, but also to address the feasibility of developing multiple drug products for a rare disease in a time-efficient manner.”
Other aspects covered by the guidance include the potential need to develop, validate and employ age-specific endpoints; the strong recommendation that clinical outcome assessments (COAs) be qualified and standardized, with sponsors first discussing selected COAs with FDA; and a description of the
"The FDA is committed to helping develop drugs for rare diseases – especially rare pediatric diseases where unmet needs exist,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “Working with our European regulatory colleagues at the EMA, the FDA has drafted an approach to pediatric rare disease drug development that could eliminate the need for certain clinical studies and, when pediatric clinical studies are needed, could reduce the total number of patients who would receive a placebo instead of a potentially helpful drug. This draft guidance aims to enhance the efficiency of drug development, while minimizing the number of patients required in the trials. Ultimately, we hope to promote new, creative approaches to drug development for Gaucher and other rare pediatric diseases."
