[Ip-health] The Impact of the US Priority Review Voucher on Private-Sector Investment in Global Health Research and Development

Sophie Mayer sophie.emma.mayer at gmail.com
Mon Sep 17 09:59:22 PDT 2012


As an incentive for initiating or continuing NTD research, the US FDA's
priority review voucher ranked consistently lower than developing/emerging
markets as a consideration for initiating and continuing research, behind
corporate social responsibility and employee morale at large companies, and
behind developed markets for small companies.

Read full article and survey results:
http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001>
The Impact of the US Priority Review Voucher on Private-Sector Investment
in Global Health Research and Development

Andrew S. Robertson, Rianna
Stefanakis*<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#cor1>,
Don Joseph, Melinda Moree

BIO Ventures for Global Health, San Francisco, California, United States of
America


Diseases such as trypanosomiasis, leishmaniasis, and lymphatic filariasis
impose substantial health burdens in developing countries
[1]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Trouiller1>,
[2]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-World1>.
These diseases are widely neglected because there is little financial
incentive for biopharmaceutical companies to invest in developing new
treatments, vaccines, and diagnostics. Most of the 1,000,000,000 people
affected by neglected diseases are poor and live in low-income
countries [3]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-World2>.
Although there is a significant need for new cost-effective drugs and
vaccines, from 1975–1999 less than 1%–2% of new chemical entities marketed
were for tropical diseases and tuberculosis
[1]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Trouiller1>,
[4]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Cohen1>
.

Market incentives play an important role in mobilizing companies toward
neglected tropical disease (NTD) research. Pharmaceutical companies have a
fiduciary responsibility to shareholders to maximize profits. Absent of
other incentives, companies will focus research and development (R&D) on
products and programs that possess a profitable market, have a sufficient
likelihood of technical success, and are likely to achieve a maximum return
on investment. In contrast, the global burden of neglected infectious
diseases is concentrated in developing countries with inadequate health
budgets and poor patients who can pay only low prices for drugs, if they
can afford to pay at all. Drugs and vaccines that target neglected diseases
thus typically cannot compete with potentially profitable products for
internal company or investor R&D dollars.

Governments and foundations have recognized the dearth of private-sector
incentives for investment in NTD research and have responded with “push”
funding (up-front funding for drug development) and “pull” mechanisms
(rewards for output) to promote successful development
[5]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Moran1>,
[6]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Towse1>.
The priority review voucher (PRV) program, currently administered by the US
Food and Drug Administration (FDA), was passed into United States law in
2007 as a pull mechanism to help promote R&D for new medicines targeting
NTDs, malaria, and tuberculosis
[7]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Priority1>.
Under this law, companies that receive FDA approval for a novel drug or
vaccine targeting one of 16 tropical diseases are awarded a transferable
voucher. This voucher can be sold to a second organization or can be
redeemed to grant the bearer priority six-month review for a future
medicine of their choosing
[8]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-US1>.
As average standard review periods can range between 10–16 months, the
voucher could potentially allow drugs to reach the market up to eight
months earlier. Economic models have predicted that this faster time to
market could be worth between US$50 million to US$300 million
[9]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Ridley1>,
[10]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Noor1>
.

However, the impact of the PRV incentive in developing new medicines for
NTDs has been questioned, in part due to uncertainty around the value of
the voucher among pharmaceutical and biotech companies
[11]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Gingery1>,
[12]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Kesselheim1>.
The PRV program as implemented carries restrictions that make earning or
using a voucher difficult or impractical, such as a requirement that
developers provide notice to the FDA at least one year prior to the use of
a voucher (often before clinical trials have concluded). Further, the
voucher has not resulted in demonstrated value for any company to date. At
writing, Novartis is the only company to have received a PRV, which they
used in 2011 to accelerate the review of a supplemental new drug
application (sNDA) for their gouty arthritis drug candidate Ilaris
(canakinumab) [13]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-The1>.
The FDA fulfilled their responsibilities under the PRV program to conduct a
six-month priority review, but ultimately denied approval of Ilaris citing
the need for further data to assess the overall safety profile
[14]<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001#pntd.0001750-Novartis1>.
It is possible that Novartis gained some benefit from the use of the PRV
for the review of Ilaris, but this value is difficult to quantify.
Understanding the Influence of the PRV

Despite the uncertainty surrounding its value, the PRV has had some
influence on the private sector. From March to July 2011, BIO Ventures for
Global Health conducted a survey of companies with active drug or vaccine
programs in one of the 16 PRV-eligible diseases. The purpose of the survey
was to investigate 1) the influence of the PRV in initiating and continuing
NTD product development, 2) the perceived monetary value of the PRV, and 3)
improvements needed for the PRV program. ...

Read full article and survey results:
http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750<http://www.plosntds.org/article/info:doi/10.1371/journal.pntd.0001750?imageURI=info:doi/10.1371/journal.pntd.0001750.t001>



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