Orphan Drugs & Priority Review Vouchers

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Rare illnesses and medical problems are often neglected in the pharmaceutical industry. This is because drug developers opt to focus on illnesses that are prevalent and their treatments more lucrative. The Federal Drug Administration (FDA) is addressing this problem with Priority Voucher Review Programs, whose recipients are awarded expedited FDA review for their future product. These vouchers have specific requirements in place making them applicable only to drugs being developed for overlooked afflictions, also known as “orphan drugs.” The main drug ingredient must not yet be approved by the FDA, which supports innovation while potentially decreasing overuse of antibiotics and subsequent resistance. Voucher programs are in place for treatments or preventatives in three areas: rare pediatric diseases, rare tropical diseases, and medical countermeasures for possible terroristic threats. Tropical diseases include Zika, malaria, filovirus, cholera, and leprosy and more; the list of accepted ailments is open to being added to. Medical countermeasures include treatments and preventatives for nuclear, chemical, biological, and radiological threats that the Department of Homeland Security finds significant.

These vouchers are attractive to drug developers for a variety of reasons. The development process often takes many years from conception to market placement, so early approval can provide a significant advantage over competitors, and shorter review time means money can be saved and directed toward other priorities. These vouchers are also able to be sold or traded to other companies, heightening their general value. These benefits are intended to be incentives to encourage companies to develop drugs in these underserved areas.

The program began a little more than 10 years ago, in September of 2007. The concept was designed by economic professors at Duke University and was intended to help with the long- standing problem of recognizing medical needs in developing countries. The first PRV was awarded to the antimalarial drug Coartem®, developed by Novartis Pharmaceuticals. Coartem was already a successful and popular drug in other countries, making it an easy first choice for expedited U.S. FDA review and approval. Since then, 14 vouchers have been awarded: six of which have been used for rare pediatric diseases, five for tropical diseases. Many vouchers remain unused, however, with companies waiting to put them toward a promising drug innovation or a lucrative trade.

There are specific conditions to the use of these vouchers. The voucher only benefits review time, not the approval itself, and the FDA has set a user fee that ranges from 2 million to 5 million dollars. This could be a worthless investment if the drug fails, so companies must be sure of their product’s effectiveness if they are to use a voucher. In addition, excluding pediatric priority review, vouchers can only be traded once.

These vouchers exist as a counterbalance to market forces. As with anything within the economic field, supply is consistent with demand. This works in most cases; however, sometimes this causes serious problems to be overlooked due to relative lack of profitability. Tools like that of Priority Review Vouchers aim to discourage stagnation of drug exploration in underserved areas and support finding life-saving or life-improving treatments for those in need.