OBJECTIVES:

Managed access programs (MAPs) are a mechanism that allow patients with serious or life-threatening diseases with no treatment options or opportunity to enroll in clinical trials access to investigational medicines (IMs) that have not yet received regulatory approval. This study analyses the UK health technology assessment (HTA) outcomes of IMs that went through MAPs prior to European Medicines Agency (EMA) approval to determine the safety and value of their implementation.

METHODS:

The research was conducted on clinicaltrial.gov using MAP-related search terms, such as “compassionate use, early access, and expanded access”. Filtering criteria, including a European focus, were applied, and 10 completed MAPs were identified. Subsequent EMA approvals and HTAs in England (via NICE) and Scotland (via SMC) were then analysed.

RESULTS:

All ten IMs identified in this study subsequently received EMA approval. Only one IM was recommended by either NICE or SMC for the same patient population as the MAP, with no further restrictions following HTA assessment. Five IMs were recommended by either body with restrictions on patient population and/or previously agreed discounts via patient access schemes (PAS). Two IMs have not been recommended, whilst one received a restricted recommendation in Scotland but not in England. One IM did not go through HTA assessment.

CONCLUSIONS:

MAPs represent an opportunity for patients with high unmet medical need to gain early access to innovative medicines. All IMs in this study received EMA approval, illustrating that MAPs incorporate appropriate safety mechanisms, are not detrimental to the approval and reimbursement process and that, overall, the benefits outweigh the risks. A high proportion of the IMs also received HTA endorsement in the UK that validates the implementation of MAPs, though pharmaceutical companies should anticipate population restrictions and patient access schemes to gain market access.