Predictors of orphan drug approval in the European Union

Abstract

Objective To encourage the development of drugs for rare diseases, orphan drug legislation has been introduced in the USA (1983) and in the EU (2000). Recent literature discusses factors that may influence the development of new orphan medicinal products in the EU. This study aims to identify predictors for successful marketing authorisation of potential orphan drugs in the EU. Methods A comparison between randomly selected authorised and a matched sample of not-yet-authorised orphan drug designations has been performed. Determinants in the study included characteristics of the indication, of the product and of the sponsor. Data were collected from the public domain only. Results Orphan drug approval was strongly associated with previous experience of the sponsor in obtaining approval for another orphan drug (OR=17.3, 95% CI=5.6-53.1). Furthermore, existing synthetic entities compared to biotechnology products tended to have a higher likelihood of reaching approval status (OR=3.9, 95% CI=0.9-16.6). ConclusionThis study showed that experience of a company in developing orphan drugs is an important predictor for subsequent authorisation of other orphan drugs. The same applies for existing (synthetic) molecules, for which much knowledge is available. Further research should be directed towards studying the quality of the clinical development program of those designated orphan medicinal products not reaching approval status.

Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency

“…Small companies have a larger proportion of medicinal products with OD relative to larger and mediumsized companies. Overall, our findings support the work of Heemstra et al [16] in showing that OD approval is strongly associated with the previous experience of the sponsor in obtaining approval for another OD.…”

Therapies for Inborn Errors of Metabolism: What Has the Orphan Drug Act Delivered?

“…A previous study in the European Union suggested that a company's previous success in developing orphan products might be an important factor in orphan product development. 8 Indeed, the clinical developments of most biological products approved for IEMs were shepherded by commercial manufacturers, which have better resources and/or experience to handle the complex issues involving biological manufacturing and clinical studies. In contrast, the development of small molecules (drugs) often was started by academic researchers before being taken over by pharmaceutical companies.…”

Industry Perspectives on Orphan Drug Development

“…Talele et al (2010) summarize the impact of those innovations on the development of therapies for rare inborn errors of metabolism in the United States over the preceding 26 years. Heemstra et al (2008Heemstra et al ( , 2009) provide a perspective on the effects of similar legislation in the European Union.…”