Does Orphan Drug market exclusivity incentive sustains drug development for rare diseases ?

Objectives: To stimulate drug R&D, regulatory agencies in the United States (US) and Europe (EU) provide market exclusivity (ME) extensions for patented drugs, to compensate for patent life that is lost during the lengthy drug R&D and to allow the return of investment for the industry. An important contribution made in drug regulation has been to incentivize the development of drugs for rare diseases through the Orphan Drug (OD) legislation. In the US, ME is granted by the Food & Drug Administration (FDA) for 7 years upon market approval (MA), while in the EU, the European Medicines Agency (EMA) provide a 10 years' extension. One may wonder about the effects of such public policy as far as the incentives are not contingent to an ultimate drug R&D success. Case studies have supported the opinion that the OD legislation has played an important role in drug R&D for rare diseases, but little empirical research has been performed on the subject. Especially, no existing research has analyzed the link between the characteristics of OD ME and these downstream effects. The objective of this analysis is to evaluate the effective patent and ME life of OD approved in the US and EU. Methods: To explore these questions, OD market authorized products list was extracted with MA date and ME end date as well trade name, generic name, and company sponsor name, from the FDA and the EMA, since 1983 or 2000. For all drugs, patent number and patent expiry date, were obtained from the Orange Book. Data were cross-checked with USPTO and Espacenet patent databases for patent priority date, INPADOC family and patent legal status. Results: The effective market exclusivity was calculated with the OD ME expiration date and the patent monopoly end date. Preliminary results indicate that the OD ME provision extends slightly the effective ME life supported by patent monopoly for only a small majority of drugs. Details regarding how ME periods have been changing over time, and what variables affect ME period (designation, therapeutic class, firms, competition …) will be presented. Discussion: The limited gain in effective market monopoly calls into the question the impact of OD legislation and its ME provision on drug innovation. Moreover, our result challenge the common idea that OD R&D is longer than normal drugs. Therefore, OD market exclusivity might not be the incentive that push pharma-biotech firms to rush for OD market. This analysis can also give insights into what the distribution of ME period is likely to be for OD experiencing generic competition in coming years. The author declares no conflict of interest. This work is supported by the French National Institute of Cancer.