OBJECTIVES

: Conditional marketing authorisation (CMA) facilitates early approval for medicines intended for seriously debilitating or life-threatening diseases whose benefit of immediate availability outweighs the risk of less mature clinical data. While CMA has the potential to accelerate patient access, the perceived value at the regulatory level may not translate to value at the health technology assessment (HTA) level. This research explores whether CMA can accelerate time to patient access while maintaining optimal reimbursement outcomes.

METHODS

: HTA outcomes of medicines with CMA from 2018-2020 were analysed, followed by 30-minute, qualitative, in-depth interviews with payer advisors from France and Germany to predict the evolution of HTA frameworks to accommodate future CMA candidates.

RESULTS

22 medicines received CMA from the European Medicines Agency (EMA) from 2018-2020. Despite substantial unmet medical need across indications, CMA translated to poor HTA outcomes. Of 18 available HTAs in France, 8 medicines received a negative reimbursement recommendation of SMR ‘Insufficient’ (in primary indication or subgroup), while the remaining outcomes were split between ‘Important’, ‘Moderate’ and ‘Low’. Most of the reimbursed medicines demonstrated poor quality of evidence, and received ASMR V. Of 13 available HTAs in Germany, only one medicine achieved an added benefit rating, while most demonstrated ‘no’ or ‘non-quantifiable’ added benefit. Payers expect future CMA candidates will face similar reimbursement hurdles if market-specific HTA requirements are not met.

CONCLUSIONS

: While CMA accelerates regulatory approval, limited data packages create huge uncertainty in the appraisal of medicines. This leads to significant risk of unfavourable reimbursement outcomes and delays in patient access. While HTA frameworks continue to evolve over time, they are unlikely to reward immature data submissions particularly as payer bodies face ever-increasing funding constraints likely exacerbated by the COVID-19 pandemic. Manufacturers should ensure their drug development programs meet both regulatory and payer requirements to support accelerated patient access.