The Drugs for Neglected Diseases initiative (DNDi) has joined Médecins Sans Frontières/Doctors Without Borders (MSF), the Global Alliance for TB Drug Development (TB Alliance), and other not-for-profit public health organizations in calling for amendments to the Food and Drug Administration (FDA) Priority Review Voucher (PRV) programme for neglected diseases.
In an open letter sent to US Senators Lamar Alexander and Patty Murray, DNDi and the other signatories strongly recommend two amendments to the PRV programme. Senator Alexander is the Chairman of the Senate Committee on Health, Education, Labor and Pensions (HELP) and Senator Murray is the Ranking Member of the HELP Committee, which has jurisdiction over the FDA.
1.) The PRV programme should have a novelty requirement. A PRV for neglected diseases can still be awarded even when new research and development (R&D) investments have not been made by the entity receiving the award or the medical product awarded a PRV for neglected diseases is not new.
The PRV rewards successful FDA registration of drugs for select neglected diseases that have not been registered in the U.S., even if that drug has already been in use in other countries for years. Two of the three FDA PRVs for neglected diseases, awarded to Knight Therapeutics and Novartis for products for treatment of leishmaniasis (miltefosine) and malaria (artemether-lumefantrine) respectively, were for drugs already in use for a long time in other countries. This has resulted in the granting of PRVs, but not in new investment in R&D. A PRV should only be awarded to products that are truly new, or that are registered with the FDA in a timely manner after initial registration in disease-endemic countries.
2.) The PRV programme should require an access strategy. The PRV programme for neglected diseases does not include any mechanism to ensure patients, governments and health care providers will have affordable and appropriate access to products for which a PRV has been awarded.
Critically, the PRV programme for neglected diseases does not ensure that the qualifying products will be accessible and affordable to patients in need. PRV recipients are not even required to market a product that earns a PRV. Additionally, products that are marketed do not need to be priced affordably. For example, in the case of miltefosine, health care providers like MSF, R&D organizations like DNDi, governments and others are still struggling to access this product at an affordable price – or in some cases to access it at all. A PRV should only be awarded to companies who commit to serious efforts to make the PRV-earning neglected disease product available and accessible to patients in disease-endemic countries, whom the PRV programme is intended to benefit.
The signatories:
Dr. Atul Malhotra, President American Thoracic Society
Dr. Bernard Pécoul, Executive Director, DNDi
Rachel M. Cohen, Regional Executive Director, DNDi North America
Dr. Mel Spigelman, President and Chief Executive Officer Global Alliance for TB Drug Development
Christine Lubinski, Vice President for Global Health, IDSA Center for Global Health Policy
Jason Cone, Executive Director, USA, MSF
Dr. Manica Balasegaram, Access Campaign, MSF
Dr. Peter J. Hotez,President, Sabin Vaccine Institute
Mark Harrington, Executive Director, Treatment Action Group
