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Early Access Program (EAP)
Within the EU, in some cases, Member States (MS) can authorise the use of medicinal products that have not yet received a Marketing Authorisation (MA) but are undergoing clinical trials or have entered the MA application process.
Such cases are exceptional and usually only permissible when other treatment options for the patient have been exhausted, or are unavailable. The provision of unauthorised medicines in this manner can be made via different types of “Early Access Programs”, which require regulatory approval on a national basis. It should be noted that, in many cases, the EAP will be specific to the medicinal product in question and its provision may differ in each MS. Such EAPs are regulated on a national basis and commonly follow specific national registration procedures.
Compassionate Use Program (CUP)
Compassionate use allows the use of an unauthorised medicinal product under strict conditions for a group of patients who have a disease with no satisfactory authorised treatments and who are ineligible for clinical trials.
According to Article 83 of Regulation (EC) No 726/2004, medicinal products without a MA may be made available for compassionate reasons to a group of patients with a chronically or seriously debilitating disease, or whose disease is considered to be life-threatening, and who cannot be treated satisfactorily by an authorised medicinal product in the European Union.
The medicinal product concerned must either be the subject of an application for a centralised marketing authorisation in accordance with Article 6 of Regulation (EC) No 726/2004 or is undergoing clinical trials in the European Union or elsewhere.
Compassionate use programs fall under national jurisdiction and, in most Member States under the purview of National Competent Authorities (NCA). The NCA in the Member State decides if such a program fulfils an unmet medical need according to their clinical practices and available alternatives. Some Member States have a long tradition of early access programs, including compassionate use, while others have different provisions in their national legislation.
Compassionate use should not be confused with Named-Patient-Programs which are under the direct responsibility of the investigator and are conducted on an individual basis.
While compassionate use is a treatment option that allows the use of an unauthorised medicine for patients who have a disease with no satisfactory authorised therapies or who cannot enter a clinical trial, “Named-patient basis access” is the supply of unauthorised medicines in response to unsolicited requests from the treating physician for individual patients.
According to Article 5 of Directive 2001/83/EC, ‘a Member State may, in accordance with legislation in force and to fulfil special needs, exclude from the provisions of this Directive medicinal products supplied in response to a bona fide unsolicited order, formulated in accordance with the specifications of an authorised health-care professional and for use by his individual patients under his direct personal responsibility.’
Some elements are common with CUPs. For example, according to this definition, “special needs” may be interpreted as somewhat equivalent to “chronically or seriously debilitating disease, or a life threatening disease, and who cannot be treated satisfactorily by an authorised medicinal product” as outlined in Article 83 of Regulation (EC) No 726/2004.
However, the need to be “the subject of an application for a centralised marketing authorisation (...) or undergoing clinical trials” in the European Union or elsewhere, as a requirement for a CUP as explained above would not apply for named-patient use.
Thus, this term would cover any access to a non-authorised medicine at any stage of its development, provided it is not made available under a cohort approach. The decision is made on a case-by-case basis in any case. This article does not apply to off-label use of licensed medicines.