Program meant to promote research for neglected diseases seriously flawed
On November 17, 2015, Doctors Without Borders/Médecins Sans Frontières (MSF), the American Thoracic Society, the Drugs for Neglected Diseases initiative (DNDi), the Global Alliance for TB Drug Development, the IDSA Center for Global Health Policy, the Sabin Vaccine Institute, and the Treatment Action Group sent a letter to the US Senate Committee on Health, Education, Labor, and Pensions (HELP) leadership asking for amendments to fix critical flaws in the US Food and Drug Administration’s Priority Review Voucher (PRV) program for neglected diseases.
The US FDA PRV is supposed to reward innovation for companies that invest in research for neglected diseases. Under the program, companies that successfully register a product to treat certain neglected diseases are rewarded with a voucher that allows them to fast-track any of its products or medicines through the crucial FDA review process.
This voucher can be used or sold to another entity, offering an accelerated FDA review with a targeted decision time of six months. To date, three PRVs for neglected diseases and five PRVs for rare pediatric diseases have been issued. Four of these vouchers have been sold, increasing in value, with the most recent PRV sold for $350 million in August 2015.
In spite of this clear and growing value of the incentive, the program has so far failed to ensure that neglected disease medical products are appropriately incentivized and are accessible to the patients and health care providers who urgently need them.
The Honorable Lamar Alexander, Chairman
The Honorable Patty Murray, Ranking Member
Committee on Health, Education, Labor and Pensions
United States Senate
Dear Chairman Alexander and Ranking Member Murray:
We are writing to ask for your support, as the Chairman and Ranking Member of the Senate Committee on Health, Education, Labor and Pensions (HELP), to ensure that new research and development (R&D) for neglected diseases is effectively incentivized, and that any new products brought to market are made accessible and affordable to those who need them. We would specifically like to call your attention to the need for amendments to the Food and Drug Administration (FDA) Priority Review Voucher (PRV) program for neglected diseases.
As several of the most recent and ongoing global health emergencies have reminded the world, the need for wellfunctioning incentives for R&D for neglected diseases is today more urgent than ever. Yet, despite representing more than 10% of the global disease burden, only 4% of new drugs and vaccines approved across the world were indicated for neglected diseases between 2000 and 2011.
In July 2014, Médecins Sans Frontières/Doctors Without Borders (MSF), the Drugs for Neglected Diseases initiative (DNDi) and the Global Alliance for TB Drug Development (TB Alliance) sent a letter to all the members of the HELP Committee raising several concerns with the design of the FDA PRV program for neglected diseases and proposed legislative amendments.
Since then, the increasing monetary value of PRVs has been established through sales, with the most recent voucher being sold for $350 million in August 2015. Additionally, several of the concerns we raised in our 2014 letter have been addressed. We welcome amendments made in 2014 that lift limits on transfers of the PRV for neglected diseases, increasing the potential appeal and value to prospective PRV recipients. We also welcome the extension of the list of eligible neglected diseases, including for Ebola and Chagas disease.
However, the lack of requirements for a product to be novel or to be made available to and affordable for those whom the product is designed to treat or protect are two critical flaws in the design of the program that remain unaddressed. Now, as we understand, the Committee is preparing to release draft biomedical R&D legislation. We hope that the Committee will use this opportunity to fix the neglected disease FDA PRV program to ensure that neglected disease medical products, including treatments and vaccines, are appropriately incentivized and are accessible to the patients and health care providers who urgently need them.
There are two key amendments to the PRV program for neglected diseases that we strongly recommend:
- The PRV program should have a novelty requirement. A PRV for neglected diseases can still be awarded even when new 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.
- The PRV program should require an access strategy. The PRV program 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 program 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 program is intended to benefit.
There are a number of simple legislative amendments that could help to remedy the functioning of the PRV for neglected diseases, based on existing law for the rare pediatric disease PRV program or proposed legislation for PRV programs. We have discussed these proposals with many HELP Committee member offices and with HELP Committee staff, and we hope that solutions will be incorporated into the forthcoming Senate biomedical R&D legislation.
As organizations working to develop and provide access to neglected disease treatments and vaccines, we see every day the need for more effective strategies to incentivize needs-driven R&D for neglected diseases, including appropriate rewards for investments. Improvements to the PRV program will be one important step toward broader changes that are urgently needed to ensure the R&D system delivers appropriate and affordable health technologies for those who need them. We therefore hope that you will consider not only leading the Senate HELP Committee in amending the PRV program for neglected diseases, but also in considering the potential creation of additional mechanisms to ensure that R&D for neglected diseases is successfully and appropriately incentivized, and that all patients in need can benefit from the fruits of biomedical innovation.
Dr. Atul Malhotra
American Thoracic Society
Dr. Bernard Pécoul
Drugs for Neglected Diseases initiative
Rachel M. Cohen
Regional Executive Director
Drugs for Neglected Diseases initiative
Dr. Mel Spigelman
President and Chief Executive Officer
Global Alliance for TB Drug Development
Vice President for Global Health
IDSA Center for Global Health Policy
Executive Director USA
Médecins Sans Frontières/Doctors Without Borders
Dr. Manica Balasegaram
Access Campaign Médecins Sans Frontières
Dr. Peter J. Hotez
Sabin Vaccine Institute
Treatment Action Group
CC'ed to Members of the Senate HELP Committee and the US FDA Acting Commissioner of Food and Drugs