E-DRUG: The US FDA Priority Review Voucher and neglected diseases
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The US FDA Priority Review Voucher -
The right mechanism to develop drugs for neglected diseases - or corporate welfare in disguise ?
A new mechanism to boost research and development (R&D) into neglected
diseases becomes operational in the US. But are priority review vouchers
really the way to go?
The need for more medical innovation for neglected diseases is now widely
recognised and getting this onto the agenda has been a success for activists and NGOs such as MSF. It is welcome that new incentives to stimulate R&D are being set up. But with the way things are going, it increasingly seems that too little attention is paid to the question of how far patients will actually be able to benefit from and access the medicines - so much so that those who will gain most from this renewed interest in neglected diseases, are in fact large pharmaceutical companies.
How it works
The priority review voucher is intended to act as an incentive for
companies to invest in researching and developing new treatments,
diagnostics or vaccines for diseases that are currently neglected, such as
tuberculosis, malaria or sleeping sickness.
For a company to be able to market a drug in the US, it has to be
approved by the US Food and Drug Administration (FDA). A company that
obtains US FDA approval for a treatment targeting a neglected tropical
disease is awarded a 'voucher'. The voucher then entitles the company to
benefit from priority review for another drug, diagnostic or vaccine. In
other words, the US FDA will fast track the approval process for an
unrelated medicine, allowing the company to sell the drug up to a year
sooner, shortening the time it takes for a drug to reach the market and
generate profits.
Controversial test case
Proponents of the priority review mechanism point to it being a way of
harnessing market forces in order to reward R&D for neglected tropical
diseases, and to accelerate access to new medicines that may otherwise not
have been developed for patients in the developing world.
Looking at the first possible use of the priority review mechanism to judge
its potential impact does not bode well. Swiss pharmaceutical company Novartis is currently seeking approval for a treatment against malaria combining the drugs artemether/lumefantrine (marketed under the name Coartem or Riamet) - surely this is not because of sudden outbreaks of malaria in the US.
If the US FDA approves Coartem (which is expected), Novartis would
receive a priority review voucher. Novartis has been quoted saying that the
company believes it is eligible for the voucher.
Yet artemether/lumefantrine was added to the WHO Essential Medicines List
in 2002 and has been available in the developing world for years. There is
therefore no benefit whatsoever to patients in the developing world from a
U.S. registration - and thus no reason to reward the company. After all,
other companies could now come forward with even older drugs for tropical
diseases that they never bothered to register in the US. Far from the
original intention, to spur new R&D, this scheme therefore rewards existing
drugs and is an incentive for companies to simply scour their existing drug
portfolios to benefit from the windfall of a tradable voucher, rather than
focus on new research to meet the needs of patients.
Did anyone stop to think about access?
Worse, there is no guarantee that the priority review mechanism actually
does anything to boost access to medicines for developing country patients.
It does nothing to require that any product developed for tropical diseases
is either priced affordably, or even made available in developing
countries.
A company could simply neglect to register a product in developing
countries, or maintain its price out of reach of people who need it or
could not market it at all. In that case, the mechanism would be giving
pharmaceutical companies a windfall, and patients would be getting nothing
in return.
And in the end, this is all based on the accelerated approval of medicines
for use in the U.S. More and more evidence is available to show that these
are of increasingly limited therapeutic value. According to the US FDA only
24% of approved drugs during a 12 year period were found to provide
significant clinical improvement (NIHCM Foundation, 2002). Thus,
accelerated marketing of medicines is in general not in the interest of
patients, except where it concerns a product for a disease for which
adequate treatment does not exist.
Should commercial factors play a role in regulatory procedures?
Crucially, the voucher would grant an expedited review not on public health
ground for the specific drug but as a commercial incentive. It raises
serious questions if activities of regulatory agencies are no longer
strictly based on public health ground alone. Drug regulation should be
about ensuring efficacy and safety of the product on the market.
Relying on premium prices paid in the West
The priority review mechanism is a form of incentive that relies 'like the
patent system it is designed to complement' on selling drugs at premium
prices. Such high prices mean many in the US, as in the developing
world, simply go without treatments they cannot afford and thus might not
be sustainable in the long-term.
Estimates put the size of the windfall companies could receive for each
voucher at more than US$ 300 million per product. Another way of putting it
is that American sick people are asked to pay this. Is this really the way
to go?
Other incentives can be more effective. Prize funds, for example, can give
a substantial financial incentive to invest in innovation for neglected
tropical diseases, in a way that ensures a clearly identified need will be
met and include terms to ensure low-cost access. In that way both
innovation and access needs are met. And those paying for the R&D are not
the sick consumers who often have a hard time to pay their drug bill to
begin with.
Leyla Pope
Communications Officer
Medecins Sans Frontieres
Campaign for Access to Essential Medicines
Tel. + 41(0) 22 849 89 88
Leyla.POPE@geneva.msf.org