August 29, 2012

What does the private sector really think about priority review vouchers?

Manager of Research and Policy
BIO Ventures for Global Health

In this guest post, Rianna Stefanakis—manager of Research and Policy at BIO Ventures for Global Health (BVGH)—writes about a new BVGH study this week in PLoS Neglected Tropical Diseases. This post originally appeared on the BVGH blog.

Here at BVGH, we spend a lot of time working on incentives to mobilize companies toward pursuit of research and development (R&D) projects that tackle neglected tropical diseases. In this quest, we’ve focused a lot of our attention on the Priority Review Voucher (PRV) program—administered by the US Food & Drug Administration (FDA)—because it’s a rare instance of an innovative, cost-neutral program targeted specifically at incentivizing R&D for neglected diseases, and has actually been implemented. Because there is only one example of a voucher being awarded and used since the program was instituted in 2007, there’s a healthy debate around the impact of the PRV and its effectiveness in actually stimulating private sector investment in new neglected disease product development. One aspect of that debate is that pharmaceutical and biotech company executives often cite uncertainty around the value of a voucher as a reason for questioning the PRV’s merit.

But regardless of the uncertainty surrounding its value, the PRV program has had some impact on decision-making around neglected disease product development in the private sector, as seen in recent statements by companies such as PaxVax. Investors, CEOs, and other industry executives are familiar with the program, but there has been no in-depth analysis to better understand and document the perceptions of these stakeholders, including their estimates of the PRV’s value. To address this evidence gap, BVGH recently conducted a survey of companies with active drug or vaccine programs in one of the 16 PRV-eligible neglected tropical diseases. Our results are fresh off the online journal presses this week in PLoS Neglected Tropical Diseases.

Our study focused on understanding three areas:

We received responses from 12 companies, representing 27 unique neglected tropical disease drug or vaccine programs. Through the survey and follow-up interviews, we found that the majority of respondents did consider the PRV incentive when deciding whether or not to pursue neglected tropical disease R&D programs. In fact, two respondents specifically said the PRV was the controlling factor in the initiation or continuation of their respective neglected tropical disease programs. When compared to other possible motivating factors, however, the PRV ranked lower than other identified incentives, such as potential market value in the developing world or emerging markets. This result was not surprising. It is clear that the PRV is not a panacea, but one of a number of incentives meant to encourage biopharmaceutical companies to engage in neglected disease R&D.

In addition to the impact of the PRV on portfolio prioritization, we also compiled data on the perceived monetary value of the voucher. Responses spanned a wide range, suggesting there is no industry consensus on the value of the PRV. This finding is not surprising since a PRV has not actually been sold, nor has there been a dollar amount attached to a voucher’s use.

The bottom line is that without a better understanding of the actual market for a voucher, a PRV’s value is uncertain and difficult to assess. This was clear in our follow-up interviews with survey respondents. The respondents underscored, first and foremost, the need for a demonstrated sale of a voucher, with the purchase price disclosed. They also cited a need for the FDA to demonstrate support of the program. Finally, developers cited the need for revisions to the implementation rules governing the PRV program—changes that would have to be made by the FDA and Congress. These revisions varied among respondents and included relaxing the requirement that companies issue a one-year advanced notice prior to redeeming a PRV, relaxing the limit on voucher transferability, and providing greater clarity into the revision process for the list of PRV-eligible diseases.

We hope these constructive suggestions will be considered by the FDA and other industry stakeholders. These documented suggestions should also be examined in light of the recently-passed Creating Hope Act legislation, which creates a parallel three-voucher pilot PRV program for rare pediatric diseases.

Despite these uncertainties, we remain convinced that the PRV represents a valuable incentive for neglected disease research. The PRV program will ultimately generate a number of vouchers—as more PRV-eligible drug candidates are approved—which will reinforce the utility as well as the value of the PRV program. Those approved drugs will have the potential to make a real difference in the fight against neglected tropical diseases.

The full paper can be found here.

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