Abstract
Results More orphan drugs were developed in the US during the first ten years of the US Orphan Drug Act (1983-1992, N=73) and during the first ten years of the EU Regulation on Orphan Medicinal products (2000-2009, N=112) than in the EU (2000-2009, N=59). Orphan drug approval was strongly associated with previous experiences 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).
Highlights
To encourage the development of drugs for rare diseases, an orphan drug legislation has been introduced in the USA (1983) and in the EU (2000)
More orphan drugs were developed in the US during the first ten years of the US Orphan Drug Act (1983-1992, N=73) and during the first ten years of the EU Regulation on Orphan Medicinal products (2000-2009, N=112) than in the EU (2000-2009, N=59)
Orphan drug approval was strongly associated with previous experiences of the sponsor in obtaining approval for another orphan drug (OR=17.3, 95% CI=5.6-53.1)
Summary
To encourage the development of drugs for rare diseases, an orphan drug legislation has been introduced in the USA (1983) and in the EU (2000). Recent literature provides criticism on the slow development of orphan drugs in the EU. This study aims at identifying predictors for successful marketing authorisation of potential orphan drugs in the EU and the US. Companies or institutions wishing to develop an orphan drug should seek experienced assistance and engage in dialogue with the regulatory authorities
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