OBJECTIVES: To investigate the impact of products with European conditional marketing authorisation (CMA) on the National Institute for Health and Care Excellence’s (NICE) recommendations.

METHODS: Oncology products containing a new active substance which received marketing authorisation by the European Medicines Agency (EMA) from 2016 to 2021 were identified and matched with corresponding NICE recommendations. Products with CMA and products with full marketing authorisation were compared to see if they differed by type of NICE recommendation.

RESULTS:

Of the 58 oncology products investigated, 23 products received CMA and 35 received full marketing authorisation. NICE reviewed 17 products (19 appraisals) with CMA and 26 products (32 appraisals) with full marketing authorisation. The odds of receiving a NICE recommendation for use within a managed access agreement (i.e., cancer drug fund [CDF]) were much higher for products with CMA (74%, N=14) compared to those products with full marketing authorisation (22%, N=7).

CONCLUSIONS:

To increase patient access to medicines, the EMA has implemented accelerated approval processes (e.g., CMA) in cases where the benefit of immediate availability of the medicine outweighs the risks. This allows manufacturers to enter the European market with less comprehensive clinical data than normally required. However, the uncertainties resulting from a lack of robust clinical data has a substantial impact on NICE recommendations, for example through frequently requiring managed access agreements (e.g., CDF) to mitigate efficacy, safety, and financial risks. Given the unpredictable nature of managed access agreements for patients and the healthcare system (e.g., uncertain long-term access), manufacturers need to carefully consider whether the benefits of accelerated regulatory access outweigh the risks they pose to HTA recommendations.