Drug program for rare pediatric diseases gets reprieve, but does it work?

It was a small headline late last week that was easy to miss: President Obama signed a bill that extends by a few months a voucher program that seeks to encourage drug-makers to develop more treatments for rare pediatric diseases.

That means the FDA’s pediatric voucher program, set to end on October 1, now has until the end of the year to find new life as part of the larger 21st Century Cures Act, which has yet to secure Congressional approval.

On its surface, a program that aims to develop new drugs for kids with rare diseases seems like an obvious winner, in any political climate. The fact that it does so by using economic incentives to spur research and development in an area the market might otherwise overlook seems like smart, market-savvy policy. Despite those advantages, a few inconvenient facts have emerged this year: Three years in, it’s still not known whether the voucher program actually does what it sets out to do. Worse, the agency tasked with carrying out the program seems to think it’s an ill-conceived mistake. The program’s uncertain fate reads like a small instance of a larger truism: Translating health policy into real-world results is never as simple as it would seem.

Congress authorized the pediatric voucher program in 2012 as a way of encouraging pharmaceutical companies to develop more drugs for rare childhood conditions. If a company wins FDA approval for such a drug, it’s then eligible for a voucher that allows it to get a fast-track review on another drug of its choice in the future — six months instead of the usual 10-month FDA review. Since time is money when bringing a new drug to market, a speedier review holds obvious appeal to drug makers. Alternatively, recipients can sell the voucher to another company.

That would seem like a rather juicy carrot to coax forth innovation in pediatric drugs. But is it working? The Government Accountability Office asked that same question in March and concluded it was too soon to tell. Over a three-year period, drug makers filed 11 applications for vouchers with the FDA, with six receiving the prize. Five of the six approved drugs treat rare metabolic disorders, while the other one treats a rare pediatric cancer called high-risk neuroblastoma. That might sound like encouraging evidence in the program’s favor, except that all six of these drugs were in development before the program got underway, according to the GAO. The longer time horizons involved in developing the drugs mean it could be years before we really know how well the program works to encourage the development of new drugs.

“Given that the typical drug development process often exceeds a decade, insufficient time has elapsed to determine whether the 3-year-old program has been effective,” the GAO report states. “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… to FDA for review.”

The FDA, meanwhile, is a bit peeved about these priority reviews at the heart of the program. Despite the program’s good intentions, the agency says it actually works against public health since it requires the FDA to devote staff time and resources for priority reviews of drugs that wouldn’t otherwise receive such expedited consideration. As the GAO puts it:

FDA explained that, in effect, the program allows sponsors to ‘purchase’ a priority review at the expense of other important public health work in FDA’s portfolio, which undermines FDA’s public health mission and the morale of its professional review staff.

The agency hasn’t been shy in voicing frustration. An FDA source told STAT’s Ed Silverman earlier this year:

The program was not negotiated with FDA, and we have opposed the program strongly… from the time it was first proposed. But in the legislative process, FDA does not get a true seat at the table. So, well-meaning academics, advocates and legislators ‘sold’ FDA to the highest bidder in setting up this program.

In addition to creating a strain on its workforce, the FDA says there’s no evidence the program has encouraged more development of drugs for rare pediatric diseases. Drug companies and patient advocacy groups strongly support the program, however, while physician and insurer groups agreed with the GAO conclusion that it’s too early to judge the program’s effectiveness.

It’s the kind of conundrum with which policy makers often have to wrestle: Powerful stakeholders and uncertain evidence. On one side, the curious alliance of drug makers excited by a powerful profit incentive and parents of children ill with rare diseases desperately seeking new treatments. On the other, a hostile FDA and an agnostic GAO, which both point out the evidence on vouchers isn’t there yet.

Will the 21st Century Cures Act win passage in the coming lame-duck session? And will the pediatric voucher program find a home in that legislation? Stay tuned.

[Graphic by Alan O'Rourke via Flickr.]