NO ORPHAN LEFT BEHIND: A NOVEL APPROACH TO THE ORPHAN DRUG ACT INCENTIVE SCHEME.

Since its enactment in 1983, the Orphan Drug Act ("ODA") has incentivized the development of more than 600 drugs used to treat rare diseases, or "orphan drugs." But today, most of the 7,000 identified rare diseases lack an FDA-approved therapy. The ODA uses a combination of mechanisms to stimulate orphan drug development, including a seven-year exclusivity period, user fee waivers, and tax credits. However, the Eleventh Circuit decision in Catalyst Pharmaceuticals, Inc. v. Becerra dampened the incentive by narrowing the FDA's authority to grant drug manufacturers seven years of marketing exclusivity. Rather than allowing multiple companies to earn exclusivity for an orphan drug approved to treat the same rare disease or condition, the Catalyst decision altered the FDA's authority so that the first company to gain approval for an orphan drug will have exclusivity for the entire class of rare disease. In effect, this decision eliminates the exclusivity incentive for companies to develop an orphan drug that could be approved to treat a different indication of the same rare disease. In the response, Congress considered the Retaining Access or Restoring Exclusivity ("RARE") Act to restore the previous incentive scheme. But is this the most ethical solution to effectuate the spirit of the ODA? This paper examines the pre-Catalyst incentive structure of the ODA and proposes key revisions to the exclusivity provision, introducing "proportional exclusivity," and suggesting an additional user-fee credit incentive to continue to enhance industry production of orphan drugs to treat our most vulnerable populations. [ABSTRACT FROM AUTHOR]