The right mechanism to develop drugs for neglected diseases…or corporate welfare in disguise ?
The U.S. Food and Drug Administration has just approved Coartem, an antimalarial treatment marketed by Novartis. In exchange, the Swiss pharmaceutical company receives the very first ‘priority review voucher’, a new U.S. mechanism intended to boost research and development (R&D) into neglected diseases. But are priority review vouchers really the way to go? The need for more medical innovation for neglected diseases is now widely recognised – 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 and access the medicines – so much so that those who will benefit 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 U.S., it has to be approved by the U.S. Food and Drug Administration (FDA). A company that obtains U.S. 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 U.S. 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 reward for Novartis
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 use of the priority review mechanism to judge its potential impact does not bode well. The U.S. FDA has just approved Novartis’ 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 U.S. As a result, Novartis now receives a priority review 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 U.S. 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 U.S. FDA only 24 percent 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 U.S., 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 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 consumers who often have a hard time to pay their drug bill to begin with.