The US Food and Drug Administration (FDA) practically pleaded with Congress not to renew a 4-year-old program to give special voucher incentives to companies that develop drugs for rare pediatric diseases, saying it skews the agency’s work away from other priorities.
Editor's Note: This article was previously titled "Pediatric drug voucher program renewed."
The US Food and Drug Administration (FDA) practically pleaded with Congress not to renew a 4-year-old program to give special voucher incentives to companies that develop drugs for rare pediatric diseases, saying it skews the agencyâs work away from other priorities.
Within days after those pleas were published, the key US Senate Committee on Health, Education, Labor, and Pensions passed legislation to renew the program for another 6 years. Itâs part of a package of bills that are companion legislation to the 21st Century Cures Act (H.R.6) passed by the House of Representatives last year.
A similar provision on pediatric vouchers is in the House legislation, but it would reauthorize the program only through 2018. Both bills add a stipulation that the pediatric diseases the drugs target must be serious.
In 2012, Congress enacted the provision for pediatric drug vouchers as part of the much larger Food and Drug Administration Safety and Innovation Act (S.3187). It provides that a sponsor of a newly approved drug for a rare pediatric disease may be eligible for a voucher for faster FDA review for another drug, meaning the review would take about 6 months rather than the typical 10. However, the drug sponsor awarded the voucher may sell it to another sponsor.
The value of getting a drug on the market that much sooner can be substantial. Of the 6 pediatric vouchers the FDA has awarded so far, 4 have been sold for between $67.5 million and $350 million.
The programâs supporters say these incentives are critical because drug companies need be motivated to develop these drugs for which it is difficult to make a profit.
According to the Government Accountability Office (GAO), 7000 rare diseases, usually serious, affect more than 25 million people in this country, and half of those diseases affect children.
The FDA objected to the GAO, and the GAO reported to Congress: âThe program allows sponsors to âpurchaseâ a priority review at the expense of other important public health work in [the] FDAâs portfolio, which undermines [the] FDAâs public health mission and the morale of its professional review staff.â
The FDA said the vouchers might be used to get faster review for drug applications for more common conditions that already have treatments. The agency already has had to curtail or defer other important work when 1 voucher was redeemed, according to the agency. It said several kinds of work could be impacted, including monitoring postmarket safety, engaging with patient and stakeholder groups, and advising drug sponsors on development, including sponsors focused on pediatric drugs.
There are other FDA programs to encourage development for drugs for children, including those under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act.
Nevertheless, the Senate committee passed the bill, the Advancing Hope Act of 2016 (S.1878), along with the other bills, and sent it to the full Senate. Senator Robert Casey Jr. (D-PA), the billâs sponsor, said, âThis priority review voucher program is the only way to make sure that we make progress on these diseases.â
The new bill would give drugs that are designated as rare pediatric disease products by 2022, when this new legislation would sunset, an additional 5 years after that to earn a voucher, Casey said.
It is 1 of about 19 biomedical bills the Senate committee passed over several weeks, a package that committee chairman Lamar Alexander (R-TN) says may be the most important legislation Congress passes this year, after differences are hammered out with the House legislation.
The GAO, in its report that included the FDAâs pleas, noted that nearly all drug sponsors and patient advocacy groups it talked to said the program could potentially motivate drug development for these diseases.
Ms Foxhall is a freelance writer in the Washington, DC, area. She has nothing to disclose in regard to affiliations with or financial interests in any organizations that might have an interest in any part of this article.