Earlier this week, Gilead ($GILD) was able to jump ahead on an FDA approval for its new HIV cocktail Odefsey after buying a priority review voucher from Knight Therapeutics for $125 million, underscoring yet again just how popular these regulatory short cuts are for top players in the biopharma industry. But a GAO review of a related voucher program underscores just how fed up the FDA has become with it.
The GAO concluded that it's still to early to tell if the pediatric voucher program, designed to encourage developers to jump into rare pediatric diseases by awarding a voucher that automatically shaves four months off the drug review process, is working as planned. Six have been awarded, and four have been sold on for as much as $350 million.
But the FDA says it's been a bust, forcing regulators to prioritize drugs that neither are focused on a key health issue nor offer all that much in terms of added safety or efficacy. It's also a chore to keep up with the mandate.
"FDA officials stated that, while they strongly support the goal of incentivizing drug development for rare pediatric diseases, they have seen no evidence that the program is effective," states the report. "The program's authorization, as amended, is set to terminate October 1, 2016, and FDA officials said they do not support the program's continuation. They expressed concern that the program adversely affects the agency's ability to set its public health priorities by requiring FDA to provide priority reviews of new drug applications that would not otherwise qualify if they do not treat a serious condition or provide a significant improvement in safety or effectiveness. Additionally, FDA officials said that the additional workload from the program strains the agency's resources."
But there's also some praise to consider.
"However, other stakeholders provided generally positive feedback on the program. For example, drug sponsors that sold these vouchers said they plan to reinvest portions of the proceeds they received into additional research on rare pediatric diseases, although there is no requirement to do so. Patient advocacy groups told GAO that the program could lead to the development of needed drugs."
AbbVie ($ABBV) plunked down $350 million for its review voucher back in the spring of 2015. And there's plenty of evidence that the industry sees significant value in the vouchers, looking for four added months of marketing for their top prospects.
Now Congress gets to decide where it all goes from here. -- John Carroll, editor-in-chief (email | Twitter)