2. Early access programmes (managed access)

 Early access programmes (managed access [1])

The EU legislative/regulatory framework offers additional possibilities for early access of (innovative) products at national level which can be subsumed under the term ‘Early access programmes’ (EAPs), such as:

  • compassionate use (CU),
  • named-patient basis access (NP),
  • expanded access (EU: options arising from participation in clinical trials).

Early access programmes offer ethical, compliant and controlled mechanisms of access to investigational medicinal products (IMP) outside of the clinical trial space and before marketing for patients with life‑threatening diseases having no treatment options available, who do not match clinical trial entry criteria, have no access to appropriate clinical trials or have completed a clinical trial and would benefit from receiving the investigational medicinal product after the trial before the authorisation is granted. EAPs can also be implemented when the medicine is approved in one country but not in another country where it is needed and requested by seriously ill patients. Different terms are used in various member States (MS) for Early access programmes, and so are different ways of implementing such programmes; there is no ‘one size fits all’. National laws differ from one country to another, and some countries have more significant barriers to EAPs than others. Most countries have named-patient programmes in place, but only a few have programmes based on Compassionate use to allow cohorts of patients rather than individuals, to benefit from early access.

EAPs have great potential in some European countries to benefit all stakeholders involved, from the patient who receives the medicine early, to the pharmaceutical company who provides it. Although EAPs can represent a significant undertaking (e.g., the degree of administrative effort required to initiate the programme, timing required to assess the application), companies who invest in them may see considerable benefit in terms of relationship building with key stakeholders, such as patients, advocacy groups, scientists and regulators, as well as launch preparedness.

A few examples [2]:

France: has the fewest barriers – it is the pioneer in Europe for EAPs, and the only country where systematic review and funding of EAPs exists, through the ‘autorisation d’accès précoce’ (AAP) system, which is social security-funded and covers CU-Programmes and Compassionate access authorisation (autorisation d’accès compassionnel (AAC) covering NP-programmes.

UK has the early access to medicine scheme (EAMS), a three-step process: (1) submission of the available (limited) data to regulators (Medicines and Healthcare products Regulatory Agency (MHRA)) and application for a promising innovative medicine (PIM) designation. 2) Upon receipt of the positive PIM designation, MHRA reviews the data. 3) Following an EAMS positive scientific opinion, commissioning of the medicine in the National Health Service (NHS). Fees are levied at each stage of the application and contrary to other countries such as the US and France, the early access provided by the company through EAMS is free of cost for the health system.

Other countries where early access to medicines is provided free of cost include, for instance, Austria, Germany, and Spain.

Conversely, countries such as Greece and Portugal do not have the infrastructure in place to support EAPs and thus the barriers to implementation are much higher.

Of note: EAPs in US vs EU differ in regulation and implementation but follow similar principles. The acronym EAP in the US, overseen by the FDA, stands for Expanded Access Programs whereas in the EU it signifies Early access programmes (the term ‘Expanded access’ is used differently, see below 2.5). In Europe Compassionate Use Programmes (CUP) and Named patient basis access (NP) differ in certain ways to typical Expanded Access Programs in the US. Compassionate Use is the most similar to the US Expanded Access Program.

[1] The term Managed access programmes (MAP), adopted early on by the pharmaceutical industry, is often used as an umbrella term to cover a variety of early access programmes across countries, e.g. all locally defined pre-approval access mechanisms and programmes such as “Compassionate Use”, “Expanded Access”, “Named Patient access”, “Special Access Schemes/Programs”, “Autorisations temporaires d’utilisation (ATU)” and others.

[2] Source:
- Yazdani Morteza, Boggio Francesca‑initiating early access programs in Europe: Five things to consider: Executive insights.

- Patil S. Early access programs: Benefits, challenges, and key considerations for successful implementation. Perspect Clin Res 2016; 7:4-8.