Rapport oktober 2020 från EFPIA (europeiska branschorganisationen för läkemedelsindustrin) om betydelsen av EU:s regelverk från 2000 att stimulera innovation och utveckling även av särläkemedel (orphan drugs), och hur det har fallit ut.

The 2000 Regulation on orphan medicinal products (OMP) was introduced to mitigate the significant scientific and economic challenges inherent to the development of therapies for low-prevalence conditions with high unmet medical need and of medicines unlikely to attract investment. It established a set of incentives aimed at stimulating investment in rare diseases, by supporting development processes (through research grants and protocol assistance) and heightening potential economic returns (through orphan marketing exclusivity).
The strengths and weaknesses of the OMP Regulation are currently being evaluated by the European Commission. The innovation accrued as a result of the Regulation is an important element of this evaluation. The present study intends to contribute evidence on the current economic case for investment in OMP and on the impact of the Regulation, and thus hopes to contribute to a debate that ought to be evidence-based.