2. Early access programmes (managed access)

2.1. Access between regulatory approval and availability and patient access

Some national administrative bodies and/or health care professionals (HCP) may interpret that any action for accessing medicines through other than the usual channels should be considered at least together with any pre-approval programme[1].

According to the Heads of Medicines Agencies (HMA)[2] this should be included under the definition of early access programmes and be designated as “pre-reimbursement access” in order to avoid confusion with pre-approval access. In fact, it concerns authorised medicines that are used under the terms of the marketing authorisation in which the only hurdle for the usual distribution is the reimbursement or price decision.

To illustrate this: Following the marketing authorisation, there may be a considerable gap from the time of authorisation to the time of availability and to patient access. Even for centrally-authorised medicines (authorisation EU-wide) the decision on reimbursement or pricing is taken by individual member states (MS) according to local regulations/legislation.

The European Federation of Pharmaceutical Industries and Associations (EFPIA) regularly publishes a report from a large European study about the availability of innovative medicines,  known as the Patients W.A.I.T. (Waiting to Access Innovative Therapies) Indicator.. The time to availability (previously length of delay) is the days between marketing authorisation and the date of availability to patients in European countries (whether attributable to companies or authorities; for most this is the point at which products gain access to a reimbursement list). It was seen that the average time between market authorisation and patient access can vary by a factor greater than 7 across Europe, from as little as four months to 2.5 years (average time 504 days).

Definition of availability in the report: Inclusion of a centrally-approved medicine on the public reimbursement list in a country. In the report 2021 data from 2016 to 2020 on 34 countries (24 EU, and 10 non-EU) and on 152 innovative medicines are included.

[1] Example France: programme to ensure continued access to the product in AAP conditions after a marketing authorisation is granted, but before the decision on the reimbursement of the authorised product by the public health insurance system (post-marketing authorisation AAP)

[2] The Heads of Medicines Agencies (HMA) is a network of the heads of the National Competent Authorities (NCA) whose organisations are responsible for the regulation of medicinal products for human and veterinary use in the European Economic Area. The HMA co-operates with the European Medicines Agency (EMA) and the European Commission in the operation of the European medicines regulatory network and it is a unique model for cooperation and worksharing on statutory as well as voluntary regulatory activities.