Impact of Restricted EU Market Access Decisions on Patient Access To Medicines in Multiple Myeloma

OBJECTIVES: Approaches to health technology assessment (HTA) vary around Europe and include evaluation of cost-effectiveness (CE) and/or clinical effectiveness. Previous research indicated that countries relying primarily on CE have more access restrictions on oncology and rare disease medicines. This case study assessed the impact of HTA decisions and restrictions on patient access to innovations for multiple myeloma (MM).

METHODS: HTA decisions from England, France, Germany, Italy, Scotland, Spain, and Sweden were reviewed for MM treatments with EMA approval (2016-2021: Darzalex®, Empliciti®, Ninlaro®), yielding nine indications. Conditions related to the recommended population additional to the EMA indication were considered HTA restrictions. Their impact was estimated from epidemiology and treatment-pattern publications.

RESULTS: Of six Darzalex® EMA indications, patients had access to 0-5 indications by country after HTA review. Four markets (England, Italy, Scotland, Spain), applied further access restrictions beyond the label. Two restriction decisions limited the number of patients with access (SMC, 38% eligible patients; AEMPS, 75%).

Empliciti® had two EMA approvals, with country patient access to 0-2 indications; the two restricted reimbursement decisions did not limit access.

Ninlaro’s® one EMA approval, had two full access, plus three restricted HTA decisions, limiting patient access to 89% (NICE), 69% (TLV), and 11% (AIFA) of the EMA label population.

Beyond decisions aligning patient access with trial criteria, HTA restrictions impacting the highest proportions of patients related to treatment line, use in combination therapy, or disease characteristics. Cumulatively across indications, a lower proportion of patients can access MM treatments in CE markets, given published HTA decisions and restrictions.

CONCLUSIONS: HTA agencies reached distinct conclusions and applied different restrictions beyond the regulatory label. Overall, patients had less access to MM medicines in CE than non-CE markets. Importantly, not all access restrictions meaningfully limited patient access. Further research is needed to better understand the impact of HTA restrictions on patient access.