Improvements to the US FDA priority review program for neglected diseases

The neglected tropical diseases world is benefiting from this prescient legislation to address the market failure in neglected tropical diseases. However, in reviewing the proposed legislation, there are some unexpected and potentially undesirable consequences to the proposed changes.

Medicines Development for Global Health received an investment from the Global Health Investment Fund to complete the development and registration of moxidectin, a new treatment for river blindness. The potential value of the PRV was the critical factor for GHIF’s investment in us. We believe we are the first non-profit and, indeed, the first company in the world to receive an investment on a potential voucher for a drug at the pre-registration stages. Should we successfully register moxidectin, our Priority Review Voucher proceeds will be the first to be dedicated to global health rather than shareholders. Also, our funding terms with the GHIF obliged access/pricing commitments post-approval and this serves as an example of hos the PRV program was intended to work.We suggest that the proposed changes to the legislation should include a requirement for access to ensure patients, governments and health care providers will have affordable and appropriate access to products for which a PRV has been awarded.

In reviewing the proposed legislation (Section 704 Tropical Disease Product Application), the requirement that ‘one or more new clinical investigations (other than bioavailability studies) are essential to the approval of the application and conducted or sponsored by the sponsor of such application’ is an over simplification. A clinical trial is only a part of the intellectual property added to the development of a new product and should not be the focus of this legislation. Important new medicines may not require new clinical trials, and resolving intractable manufacturing is no less significant a contribution to a new medicine – this is not recognized by the proposed legislation. The requirement as written may result in unnecessary clinical trials being conducted simply to comply, without necessarily ensuring that important new medicines are registered. In addition, identifying and rewarding new medicines for neglected tropical diseases that are novel is desirable but unrealistic. The current value of the PRV means that this alone is insufficient to support full development of a novel product. It should also be considered that most new medicines for neglected tropical diseases will have a tortured development history, including a number of sponsors over time, and/or approvals in non-“competent” regulatory authorities. These factors do not alter the potential benefit of a new medicine being registered by the FDA and being made available to the people of the United States.

The Priority Review Voucher program needs to effectively accomplish its goal of incentivizing registration of neglected disease products whilst also ensuring access to those in need. Medicines Development for Global Health will continue to work on neglected diseases indefinitely, regardless of what changes are made to the Priority Review Program, but we are eager to see the program optimized in a way that is appropriate for current and future regulatory science. Addressing access requirements will result in meaningful improvements to the program

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