Objectives: To investigate the impact of the uncertainty stemming from products with European conditional marketing authorization (CMA) or authorization in exceptional circumstances (AEC) on the National Institute for Health and Care Excellence's (NICE) recommendations.
Methods: Products which received CMA/AEC by European Medicines Agency (EMA) up to 1 December 2016 were identified and matched with corresponding NICE decisions issued by August 2017, the status of which was then traced to August 2019. We assessed whether the conversion of CMA to full marketing authorization triggered a review of a NICE decision. The odds of a recommendation carrying a commercial arrangement for products with and without CMA/AEC were calculated.
Results: Fifty-four products were granted CMA/AEC by EMA. NICE conducted thirty evaluations of products with CMA/AEC. Twelve products were recommended by NICE by August 2017 and fourteen by August 2019. All recommendations had an associated commercial arrangement. The odds of carrying a commercial arrangement were higher for products with CMA/AEC compared to those with full authorization. Conversions from conditional to full authorization among products not recommended by NICE did not trigger an appraisal review.
Conclusions: Uncertainty, stemming from the lack of robust clinical data of products authorized with CMA/AEC, has a substantial impact on HTA recommendations, frequently requiring risk mitigation mechanisms such as commercial and data collection arrangements. Further analyses should be conducted to assess whether the benefits of early access strategies outweigh the risks for patients and the healthcare system.
Keywords: Biomedical; Drug approval; Reimbursement mechanisms; Technology assessment; Uncertainty.