OBJECTIVES: To assess the challenges of orphan drug pricing and reimbursement in European Union Five (EU5) countries (France, Germany, Italy, Spain, and the United Kingdom).

 

METHODS: The focus was on four orphan drugs (plerixafor, eculizumab, ofatumumab, and nelarabine) in the area of hematology/oncology. The information was retrieved from different sources, including the websites of health technology assessment (HTA) agencies, Cochrane Library reviews, and specialist European orphan disease resources. We reviewed the evaluation of the orphan drugs made by different HTA agencies including National Institute for Health and Clinical Excellence (NICE), Scottish Medicines Consortium (SMC), Transparency Commission France (TC), L’ Unità di Valutazione dell’Efficacia del Farmaco / Efficacy (UVEF), Italian Medicines Agency (AIFA), Institute of Health Carlos III-Spain (ISCIII), and the Federal Joint Committee (Gemeinsamer Bundesausschuss) (G-BA).

 

RESULTS: Uncertainties have been strongly associated with negative HTA feedback. These uncertainties include uncertainty around net therapeutic benefit, safety and adverse events offsetting therapeutic benefit, high cost relative to therapeutic benefit, high budget impact, and uncertainty around the incremental cost-effectiveness ratio (ICER). The clinical-effectiveness issues identified were related to the small numbers of patients that made efficacy assessment difficult. On the other hand, the economic evaluation issues include the study design, the health economics modeling techniques, in addition to the health-related quality of life and utility data that were not measured in the trials.

 

CONCLUSIONS: Payers are asked to reimburse high priced orphan drugs with limited clinical data (i.e., true long-term therapeutic and safety profile are unknown). Huge unmet medical needs force the high demand for access to orphan drugs, but the healthcare budget is limited in many countries. To limit the impact of coverage decisions on healthcare budgets, HTA agencies in EU5 biggest markets have restricted conditions to reimburse costly new drugs, which affected patient access to orphan drugs.