Medicinal products that have not been given a marketing authorisation may be prescribed in some cases. This concerns a specific situation in which the MEB considers the availability of new medicinal products for multiple patients (cohort) to be essential prior to the granting of the definitive marketing authorisation.
Criteria for the prescription of medicinal products without a marketing authorisation
There are two scenarios where a medicinal product without a marketing authorisation may be prescribed:
- The provision of a medicinal product on the basis of a physician's statement. The physician's statement is prescribed to an individual patient and falls under the competence of the Health and Youth Care Inspectorate (IGJ).
- If the case involves a severe condition for which no alternative medicinal product is available on the market and the as-yet unauthorised medicinal product could be granted a marketing authorisation in the future, the manufacturer of the medicinal product can submit a request to the MEB to become eligible for the compassionate use programme.
The MEB can give permission for bringing a medicinal product on the market which has not (yet) been granted a marketing authorisation, known as a compassionate use programme. A company can make these medicinal products available to the patient group.
Request for compassionate use programme
A company should submit a request to the MEB for implementing a compassionate use program. This request must indicate how the cohort of patients will be defined and which patients fall under this definition.
The implementation of compassionate use program falls under national jurisdiction. In order to come under consideration, the company should submit the request and completed questionnaire (to be requested via BRI@cbg-meb.nl) to the MEB. The company itself defines a cohort and decides on the basis of this to submit a request for a compassionate use programme.
The arguments should, in any case, include:
- a clear aspect of compassion in case there is no registered alternative medicine
- that the previously mentioned criteria are fulfilled and there is thus a need to set up a compassionate use program
- an overview of the available (pre-) clinical data and, if necessary, quality data
- an overview of any studies still running and how the company guarantees that the compassionate use program will not interfere with this
- information about which phase the marketing authorisation process is in (there should be a successful authorisation in the near future) and whether the marketing authorisation dossier has been submitted to the Committee for Medicinal Products for Human Use (CHMP)
- whether the CHMP has given an opinion on a compassionate use programme.
The MEB will assess the request. If the MEB comes to a positive decision about the request for a compassionate use program, the company is notified. The Health and Youth Care Inspectorate (IGJ) is then also informed that a compassionate use program has been approved and the conditions under which this was done. If the MEB decides negatively, then both the company and the IGJ will be informed of this.
Duration and possibility of renewal
A compassionate use programme that has been approved is valid for 1 year, or when the medicinal product is available on the market following marketing authorisation. The approved compassionate use programmes are published on the MEB website.
In case the company wishes to apply for renewal, the request for renewal must be submitted to the MEB at least 6 weeks prior to the expiry date stated in the letter of approval.
Possible adverse events of a compassionate use programme must be reported, following the guidelines of Good Vigilance Practice Module VI. Reports in which a causal link with the medicine is identified (as assessed by the primary party reporting, or by the applicant or marketing authorisation holder) must be sent to EudraVigilance.
Article 83 of Regulation 726/2004/EC laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency, states the advisory role of the CHMP regarding the compassionate use programme.
Additional criteria indicate the scope (of Article 83 of the Regulation 76/2004/EG):
- It concerns a medicinal product for a severe condition (a disease with a fatal or progressive clinical course).
- It is intended for patients who cannot be sufficiently treated with existing authorised medicinal products (in short, there is no authorised medicinal product as an alternative).
- The compassionate use programme is intended for a group of patients (cohort).
- The medicinal product is currently in a clinical trial or a marketing authorisation has been requested.
The CHMP can advise the national competent authorities – the MEB in the Netherlands – to allow a compassionate use programme; the company must however submit an application to the MEB.
The execution and admission of compassionate use programmes is a national authorisation (the Member State must grant permission for a medicinal product to be made available without a marketing authorisation).
At the national level, the legal requirements have been implemented in the Medicines Act under article 40, section 3 (f) and the Ministerial Regulation article 3.18. The MEB is the competent authority for approving a compassionate use programme.