FDA’s innovative priority review voucher, intended to incentivize the development of new drugs for neglected diseases, may actually have created a hurdle in some cases.

It is the continued relatively unknown nature of the voucher that is causing the problem, according to Shing Chang, research and development director at the Drugs for Neglected Diseases initiative (DNDi), a nonprofit group that is developing treatments for diseases like Chagas and others that are ignored by mainstream manufacturers.

Chang said his group could develop a treatment and license it to a major manufacturer, but if a priority review voucher is in play, it complicates negotiations.

“It actually created a barrier for negotiation because now we have an unknown value of an asset and who’s going to own what if we form a partnership?” – Shing Chang, DNDi Research and Development Director

As such, Chang said DNDi has yet to see the benefit of the PRV and there hasn’t been much incentive among pharmaceutical companies to acquire them.

“I’m sorry to say it actually created a barrier for negotiation because now we have an unknown value of an asset and who’s going to own what if we form a partnership?” he said. “Unfortunately we’ve managed to chart this course and mostly delay the discussion. So we haven’t really seen a great incentive from the pharma side because of this.”

The priority review voucher program is three years old, yet to date only one has been issued. Novartis received one last year for its anti-malaria drug Coartem, but at the time FDA had not finalized the process for cashing in a voucher (Also see "FDA Awards First-Ever Priority Review Voucher To Novartis For Coartem Approval" - Pink Sheet, 8 April, 2009.).

Novartis has not redeemed the voucher yet, or sold it as allowed under statute. No market value has been determined yet, which is leaving several companies waiting to see how FDA will handle the first case.

Andrew Robertson, chief policy officer at BIO Ventures for Global Health, said experts estimate the potential value of a PRV at between $50 million and $500 million, a range that does not provide much clarity.

“One of the key barriers to these being a real powerful incentive is that there’s not very much accuracy about how much it’s worth,” Robertson said. “And so as such companies have trouble developing business plans, securing the investment and looking towards future reliability of getting a PRV.”

To determine the price of a voucher, a firm would need to estimate the extra sales a product would generate with a presumably faster review (see sidebar).

Kaitlin Christenson, coalition manager of the Global Health Technologies Coalition, said while the PRV holds great promise, stakeholders are waiting for the value of the first voucher to be determined.

PRV policy was a popular topic during a Sept. 23 public hearing on challenges to developing treatments for neglected tropical diseases. Testimony will help FDA formulate recommendations on studies, trial designs and regulatory approaches to make improvements; the final report is due in March 2011.

Unanswered Questions Scare Companies Away

Presenters identified the lack of clarity of how the priority review voucher program will work as a problem.

A voucher can cut four to 12 months off the typical review time, Robertson said. But the holder must notify FDA at least a year in advance of its plan to use it. Applicants also have to play a higher user fee.

Novartis and other potential voucher holders still are left with several questions, like whether the voucher is voided if the drug fails in development or if data come up short after they give the agency advanced notice (“FDA Priority Review Voucher Redemption Fee Set At $4.6 Million In Fiscal 2011,” “The Pink Sheet,” Sept. 14, 2010).

FDA also made no guarantee that its review would be completed in the prescribed six months. The agency in guidance only gave a goal to complete 90 percent of reviews in the timeframe. Pfizer said it wanted a guarantee and the voucher returned if reviewers fail to finish on time (Also see "Pfizer, Merck And Sanofi Show Interest In Priority Review Voucher Program" - Pink Sheet, 23 February, 2009.).

Limiting PRV transfers or sales also limits its desirability and use, Robertson said at the hearing. He argued unlimited transfers should be allowed.

A bill pending in the Senate would make adjustments to the program and address some of Robertson’s concerns. S. 3697, the Creating Hope Act of 2010, would allow unlimited voucher transfers before use and would cut the advance notice a sponsor must give FDA to 90 days.

Sen. Sam Brownback, R-Kan., who co-sponsored the original voucher legislation, introduced the bill Aug. 4 and it was assigned to the Senate Committee on Health, Education, Labor and Pensions. Brownback repeatedly has pressed FDA to be more aggressive in aiding the development of treatments for neglected diseases (Also see "Effectiveness Of Priority Review Voucher, Orphan Status Part Of Tropical Disease Hearing" - Pink Sheet, 20 July, 2010.).

PRV Should Be One Of Many Incentives

The PRV also could be modified to better ensure the drugs it speeds to market will make it into the countries that need it faster.

Emi MacLean, U.S. director of the Doctors Without Borders Access to Essential Medicines campaign, said PRV agreements should be written so companies cannot gain a quick approval and then sit on the drug or sell it for an unaffordable price.

MacLean also said any PRV should ensure companies license their drugs so they can be distributed in poor countries where the diseases are prevalent.

“An improved PRV in our view has the potential to increase innovation for neglected diseases,” she said. “It also needs to engage with the access considerations as well, and an array of complementary policies is also necessary to ensure effective metaphorical new product development for neglected diseases as the PRV, given all the barriers, is unlikely to be sufficient on its own.”

Robertson also wanted the list of rare diseases eligible for a PRV expanded, as well as a rulemaking process to add others as needed.

“Our belief is as this program matures and this becomes an incentive that companies would be able to comprehend in … more detail, we think it will become a more powerful incentive,” he said.

By Derrick Gingery

Priority Review Voucher's Uncertain Value May Hinder Development

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