2. Early access
4. Named patient basis access
Named-patient basis access is the supply of unauthorised medicines (or authorised medicines from a different country, see below) for individual patients in response to requests by doctors on behalf of specific, or “named”, patients and is limited to the requested named patient or patients only. Unlike CUPs, NPs are entirely initiated by doctors, addressing their request directly to the company to treat an individual patient under the doctor’s direct responsibility.
The Guideline on good pharmacovigilance practices (GVP) – Annex I Definitions (Rev 4) EMA/876333/2011 defines ‘Named patient use’ as: The Supply of a medicinal product which is excluded by a Member State from the provisions of Directive 2001/83/EC, in accordance with legislation in force and to fulfil special needs, in response to a bona fide unsolicited order, formulated in accordance with the specifications of an authorised healthcare professional and for use by an individual patient under his direct personal responsibility [based on DIR 2001/83/EC Art 5(1)].
Article 5(1) 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 an individual patient under his direct personal responsibility”
and Article 5(2) of Directive 2001/83/EC:
“A EU member State may temporarily authorise the distribution of an unauthorised medicinal product in response to the suspected or confirmed spread of pathogenic agents, toxins, chemical agents or nuclear radiation, any of which could cause harm”
Some elements are common with CUPs. For example, accordingly with this definition, “special needs” may be considered equivalent to “patient with a chronically or seriously debilitating disease, or a life-threatening disease, and who cannot be treated satisfactorily by an authorised medicine”. 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”, would not apply. Thus, this term would cover any access to a non-authorised medicine at any time of its development provided it is not made available under a cohort approach. The decision is made on a case-by-case basis for any case (of NP). This article may also apply to the use of unauthorised medicines but not to off-label use of authorised medicines.
However, Named-patient basis access can also refer to a medicine for a specific patient or patients that is not (yet) authorised and available to them in their own country. Such medicine should or must (depending on legislation) be authorised in at least one country, from which it can be imported into the patient’s country under a Named-patient access programme (NPP).
These may be medicines that are:
- Authorised but not yet available to be prescribed in the patient’s country
- Authorised and available in one country but not authorised and available in the patient’s country
- Discontinued in the patient’s country but not in another country
- In shortage in the patient’s country but not in another country
An example for how Named-patient use is handled in the EU is given below.
Individual requests are initiated by treating doctors and may be submitted by pharmacists or local wholesalers. These requests are then evaluated by the Dutch Health Inspectorate (IGJ). Cohort-based programs are initiated by pharmaceutical companies and evaluated by the Dutch Medicines Evaluation Board (CBG).
Upon approval, the treating physicians can apply directly to the pharmaceutical companies for the supply of the medicine for individual patients or cohorts of patients. In case of named patient application, the investigational drug does not need to be registered elsewhere, and there is no strict requirement regarding the phase of development. In exceptional cases, individual requests may get approved for medicines in phase 1 clinical trials (for example in case of cancer, where often patients rather than healthy volunteers are included in phase 1 trials)
Article 5(1), (2) of Directive 2001/83/EC
The Guideline on good pharmacovigilance practices (GVP) – Annex I Definitions (Rev 4)