Most of the time when one mentions ‘compassionate use’ access to medicines, it is referred to patients’ access to medicines which have not obtained a marketing authorisation.
Nevertheless, this requires two important comments.
On the one hand, ‘compassionate use’ pathway is different than accessing to investigational medicines in the framework of clinical trials or to exempted ATMPs in the context of the hospital exemption.
On the other hand, ‘compassionate use’ is often used to refer to various existing early access pathway(s) regulated at National levels with a confusion between the terms used (sometimes interchangeably) and their linked procedures according to the country considered. In fact, these National pathways generally implement two distinct legislative pathways in EU law: what is considered legally as the ‘compassionate use pathway’ and as the ‘named-patients basis pathway’.
Although these two distinct pathways are indeed regulated at National levels, they do not come from the same EU legal provision, and thus, they are not linked to the same conditions.
Indeed, compassionate use pathways have been established at the National level in accordance with Article 83 of Regulation 726/2004, while named-patients basis pathway have been established at the National level in accordance with Article 5.1 of Directive 2001/83/EC.
One of the main differences that can be highlighted between these two different pathways is that the former relates to a group of patients, while the latter relates to an individual patient.
Table 1 below summarises the main differences established by EU law regarding these two different pathways.
| Compassionate use pathways | Named-patients basis pathways | |
|---|---|---|
| Legal Basis | Article 83 of Regulation 726/2004 | Article 5.1 of Directive 2001/83/EC |
| Definitions | Under strict condition for seriously ill groups of patients (life-threatening, long-lasting or seriously debilitating illnesses) | For a bona fide unsolicited order, with specifications of an authorised healthcare professional, & for use by an individual patient. |
| Recipients | A group of patients | An individual patient |
| Additional conditions | The medicine concerned must be: - the subject of a marketing authorisation application OR - The medicine concerned must be undergoing clinical trials | - |
| Who | Applicants (generally manufacturers or marketing authorisation applicants) to National competent authority | Applicants (generally doctors, clinicians) to National competent authority |
| Why | General derogation to the mandatory marketing authorisation of medicines for patients to access treatment | |
| How | National rules & procedures | |
| Additional provisions | National competent authorities can ask the EMA for a CHMP recommendation on how to use a medicine under compassionate use schemes across the EU | - |
Table 1: EU legal rules for “Compassionate use” and “named-patients basis” pathways
Member States may have established national rules for one or for both of these pathways, leading to various patients’ access to medicines through these schemes across Europe.
The Heads of Medicines Agencies have provided a list of national competent authorities that publish guidance regarding their national compassionate use programs. It is available here although it requires an update.
We are not aware of the existence of a similar list for existing national ‘named-patients basis’ programs.
It should also be highlighted that National competent authorities can ask the EMA for a CHMP recommendation on how to use a medicine under compassionate use schemes across the EU (Article 83.4 of Regulation 726/2004). This procedure has been used six times only regarding medicines for COVID-19, hepatitis C, and influenza virus as shown by the EMA’s webpage on compassionate use.
Patients may access ATMPs in accordance with “Compassionate use” or “named-patients basis” pathways as established in their country.