New legislation may miss key chance to reform flawed FDA Priority Review Voucher Program
New York, 1 December 2014 — A United States government program meant to reward pharmaceutical companies for investing in research and development (R&D) for neglected diseases, and which will soon include Ebola on its list of eligible diseases, is enriching drug companies without ensuring that patients, treatment providers, or governments have access to these lifesaving drugs at an affordable price, said the international medical humanitarian organization Doctors Without Borders/Médecins Sans Frontières (MSF) Monday.
The program, known as the Federal Drug Administration (FDA) Priority Review Voucher (PRV), provides a drugmaker that registers a pharmaceutical product for a qualifying neglected disease in the US for the first time with a voucher that essentially fast-tracks the approval process for any drug in its portfolio. The PRV recipient is also free to sell the voucher to other companies. The goal is to reward investments in R&D for new medicines and vaccines to treat or prevent diseases that have not historically garnered the interest of the pharmaceutical industry.
“The Senate recently proposed some much-welcome changes to the program by introducing legislation expanding the list of diseases to include filoviruses such as Ebola, making it easier to include other neglected diseases in the future and potentially increasing its economic value to pharmaceutical companies,” said Judit Rius Sanjuan, US Manager and Legal Policy Adviser at the MSF Access Campaign. “As a medical organization actively responding to the current outbreak, we welcome incentives to accelerate R&D efforts for Ebola. We are concerned that these changes still do not include any way to ensure that patients, governments and treatment providers like MSF, will have affordable and appropriate access to the potential resulting Ebola medicines and vaccines."
The FDA PRV program, in its current design, has critical flaws. For example, It does not ensure that only companies that have invested in new R&D receive the incentive and that patients, governments and treatment providers have affordable access to the medicines or vaccines developed, Rius said.
Some key problems with the FDA Priority Review Voucher program:
- A PRV can be awarded even if a drug is already on the market in other countries, which does nothing to fulfill the stated goal of the program— expansion of research and development for neglected diseases. Both Novartis and Knight Therapeutics have been awarded PRVs for drugs already in use in developing countries.
- A PRV can be awarded to companies even when they do not contribute in any significant way to the research or development of a drug. Knight Therapeutics was recently awarded a priority review voucher for registering the leishmaniasis drug miltefosine in the U.S, even though Knight made no significant investment in the research and development of this drug.
- A PRV does not ensure that the treatments will be available and affordable to the patients in need. PRV recipients are not even required to market a product that earns a PRV in the United States, let alone in the endemic developing countries. Miltefosine is again an example on how a drug that has received a FDA PRV is not available and affordable to patients and treatment providers like MSF.
MSF and partners outlined some of our concerns about the US FDA PRV mechanism in a letter to the US Congress in July this year and have called for improvements to its design and implementation. US lawmakers should take the opportunity to make changes now that the PRV is being amended to add ebola in the list of diseases.
Last month, Knight sold its priority review voucher for Miltefosine to Gilead for $125 million. The sale, at least, shows the vouchers have clear commercial value, but incentives and rewards should not go to companies who’ve played no significant part in researching and developing new medicines. And companies receiving these vouchers should have to ensure that these medicines are affordable and accessible to patients.
“We should not miss this opportunity to amend the FDA PRV mechanism to reward true innovation and ensure that benefits from this taxpayer-funded program reach the people most in need," Rius said. “The priority review voucher program has great potential as a meaningful incentive, but in its current form, it is pushing drug companies to the front of the line and leaving patients behind.”
For more information on the Miltefosine FDA PRV, please read DNDi/MSF press release on “Patient Access to Miltefosine in Developing Countries Not Secured Despite Award of US FDA Priority Review Voucher Sold for USD 125 Million” and BMJ 2014;349:g4665 - US incentive scheme for neglected diseases: a good idea gone wrong?