The Dutch Medicines Evaluation Board (MEB) has recently published a new policy for Compassionate Use Programmes (CUP).[1] To support the implementation of such programs, the MEB has published new policy guidelines in December 2024. These guidelines were developed in consultation with the pharmaceutical industry. The full policy is now in effect and contains a few key changes for the industry, with the aim to make CUPs better accessible for patients in need.[2]

Criteria for a CUP 

The MEB included in the policy the requirements upon which the CUP-applications will be assessed which of course also follow article 83 of Regulation 726/2004, and as such do not fundamentally differ from the requirements of the former policy:  

1. The medicinal product is intended to treat a group of patients with a chronic, health-deteriorating, or life-threatening disease.
    
2. The disease cannot be treated satisfactorily using an authorised medicinal product. 
   
   The MEB provides further clarification on this requirement, stating that there should be sufficient evidence that the benefit-risk balance of the medicinal product of the CUP is positive. If another medicinal product has been authorised for the same indication, but this is not yet available in the Netherlands, this should not hinder approval of the CUP
    
3. A marketing authorisation application for the medicinal product has been submitted, or supporting clinical trials are ongoing. If the studies are concluded but the marketing authorization application has not been submitted, a CUP application may still be submitted and will be assessed by the MEB.[3]

Key changes in the policy 

The new policy for CUP includes several key changes, with the most important one being the shift from temporary approval (valid for one year) to indefinite approval, until the product receives a marketing authorization or if a negative benefit-risk assessment requires early termination. This change eliminates the need for CUP renewal requests after one year, and as such will provide more security for patients and decrease the administrative burden on the pharmaceutical companies.[4]

This policy change is connected to the MEB’s current view that their former practice was lacking legal basis, as CUPs should only apply to medicinal products that have not yet received marketing authorization (regardless of whether they are already put on the market or not). The MEB came to the conclusion that under their former policy, extensions were requested and approved even though a market authorisation was already in place for a particular medicinal product, and were granted until the medicinal product came on the Dutch market and was reimbursed. After all, once a marketing authorization is granted, the medicinal product falls under a different regulatory framework (off label use).[5] To ensure continuity of the medicinal product for patients already enrolled in a CUP, pharmaceutical companies are now responsible for maintaining availability of the medicinal product until it becomes commercially available.[6]

Additionally, the new policy requires manufacturers to notify the MEB if a reassessment of the benefit-risk balance is necessary during an ongoing CUP.[7]

Back in March 2023, the MEB identified the need to implement EMA-imposed risk minimisation measures for medicinal products available under a CUP, which have received marketing authorisation, but are not yet available on the Dutch market, as a key issue.[8] The newly introduced policy now requires manufacturers to immediately implement additional Risk Minimisation Material (aRMM) right after marketing authorisation is granted, without waiting for the commercial launch of the product.[9]

Outlook

The MEB introduced the new policy to address recurring stakeholder inquiries, aiming to clarify the requirements and procedures for Dutch CUPs and to safeguard patient safety.[10] This policy ensures alignment with the regulatory status of the medicinal product and the legal framework around compassionate use programs, hopefully providing better access for patients and eliminating some of the administrative burden for pharmaceutical companies.  

[1] MEB 49: Compassionate Use Programma | Beleidsdocument | College ter Beoordeling van Geneesmiddelen.

[2] Policy document Compassionate Use Programme MEB 49 (December 2024) (link).

[3] Policy document Compassionate Use Programme MEB 49 (December 2024), p. 4-5 (link).

[4] Nieuw beleid voor Compassionate Use Programma’s | Nieuwsbericht | College ter Beoordeling van Geneesmiddelen. 

[5] Public notes of the 1023^e meeting of the MEB (4 May 2024), p. 22, paragraph 9.a (link, in Dutch).

[6] Policy document Compassionate Use Programme MEB 49 (December 2024), p. 7 (link).

[7] Policy document Compassionate Use Programme MEB 49 (December 2024), p. 7-8 (link).

[8] This is in line with Policy document National implementation of

additional risk minimisation measures MEB 45 (30 March 2022) (link).

[9] Policy document Compassionate Use Programme MEB 49 (December 2024), p. 7 (link).

[10] Public notes of the 1023^e meeting of the MEB (4 May 2024), p. 22, paragraph 9.a (link, in Dutch).