[Ip-health] MSF: Ebola To Be Added to List of Neglected Diseases Eligible for US government Research & Development Incentive

Joanna Keenan-Siciliano joanna.l.keenan at gmail.com
Thu Dec 4 02:43:34 PST 2014

Ebola To Be Added to List of Neglected Diseases Eligible for US government
Research & Development Incentive

*New legislation may miss key change to reform flawed FDA Priority Review
 *New York, December 3, 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 U.S. 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, U.S. 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 incentiv*e, *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” (available here: *
and  BMJ 2014;349:g4665 - US incentive scheme for neglected diseases: a
good idea gone wrong? **http://www.bmj.com/content/349/bmj.g4665*

Joanna Keenan
Press Officer
Médecins Sans Frontières - Access Campaign
P: +41 22 849 87 45
M: +41 79 203 13 02
E: joanna.keenan[at]geneva.msf.org
T: @joanna_keenan


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