Too early to gauge effectiveness of FDA's pediatric voucher program, says GAO

It is too early to gauge whether the US Food and Drug Administration's pediatric voucher program has stimulated the development of drugs to treat or prevent rare pediatric diseases, according to a Government Accountability Office (GAO) report released this week.

Given that the typical drug development process often exceeds a decade, insufficient time has elapsed to determine whether the three year-old program has been effective. Any drug sponsors motivated by the program to attempt to develop a drug for a rare pediatric disease may be many years from submitting new drug applications - which contain scientific and clinical data about safety and effectiveness - to the FDA for review.

As of December 31, 2015, there have been 11 requests for a pediatric voucher. Of these, six have been awarded, two denied, and three remain under review. The six drugs for which vouchers were awarded were in development prior to the program's implementation and these drugs helped fulfill unmet medical needs. One drug is indicated to treat a rare pediatric cancer, and the other five drugs treat rare metabolic diseases affecting children. No other drugs had been previously approved by FDA for these conditions.