OBJECTIVES

There are several mechanisms other than HTA approvals and P&R decisions by which patients can access orphan drugs pre-launch within a local healthcare system. Early access programs (EAP) for orphan drugs differ among the EU Member States due to their national regulation based on which the EAPs are implemented. The objective of this analysis is to examine similarities and differences of EAPs in the EU-5 countries (Germany, France, Italy, Spain and UK).

METHODS

Data was retrieved by conducting targeted literature searches including relevant institutional websites, national laws and regulations. Additionally, market access expert interviews were executed.

RESULTS

Two types of EAPs exist in all five EU countries: (1) Compassionate use programmes [CUP; synonyms: “cohort Temporary Authorisation of Use” (ATU), France; Early Access to Medicines Scheme (EAMS), UK] which are put in place and requested/funded by marketing authorization holders for a group/cohort of patients of a hospital. (2) Individual patient funding requests [Named Patient Program, NPP; synonyms: “nominative ATU”, France; EAMS, UK] which are granted in response to individual requests by physicians on behalf of named patients. Products entering CPUs are not funded in Germany, UK and Spain as opposed to France and Italy, where the company (or the NHS: Italian’s L648) fixes the compensation until the price negotiation. In France, the company will have to repay the difference between the public compensation declared under ATU by the company and the negotiated price after obtaining the market authorization. Additionally, access to centrally licensed but not yet reimbursed drugs can be provided to patients in Italy, Spain and the UK.

CONCLUSIONS

There exist two types of EAPs across all five EU-5 countries. EAPs are country-specific, and products entering these programs are generally not paid for, except in France and Italy where dedicated payment can be granted.