OBJECTIVES

Orphan designation (OD) status was introduced to encourage development of therapies for rare disease. Products that fulfil the OD criteria benefit from a 10-12 year market exclusivity period from market authorisation (MA), which can extend protection from generic competition beyond patent expiry. In this study we explore the impact of OD market exclusivity on the likelihood of generic and biosimilar entry. Factors which may influence the attractiveness of generic entry are also analysed.

METHODS

A database containing all products with an OD which were granted MA from 2000-2020 was compiled by supplementing data from the European Medicines Agency (EMA) community register. The database was curated to distinguish paediatric vs. non-paediatric and oncologic vs. non-oncologic orphan medicinal products (OMPs). Patent expiry dates were collected using GlobalData, Evaluate Pharma and other publicly available sources. An EMA database was used to identify the earliest date of centralised MA for generics and biosimilars, and the numbers of these competitors that were approved.

RESULTS

This study provides an overview of the exclusivity status of 173 OMPs with 213 OD indications granted MA from 2000-2020. The majority of OMPs are still patent protected, whilst a small proportion (~10%) benefit from OD market exclusivity after loss of patent exclusivity. Approximately one fifth of OMPs have no exclusivity protection and generic entry has occurred in around half of these OMPs. There is a small trend for higher likelihood of generic entry for oncology and non-oncology paediatric OD-products. Analysis shows that non-OD products are more likely to experience generic entry than OMPs, with a higher number of generics approved also.

CONCLUSIONS

This study shows that OD status protects OMPs from generic competition beyond the 10-12 year market exclusivity period, with generic entry less likely after the lapse of market exclusivity than observed with non-OD products.