Does Orphan Drug Designation Lead to Reimbursement in EU5?

OBJECTIVES: Orphan drug designation (ODD) is given to drugs intended for the treatment of rare conditions. The evaluation of the benefit these drugs can bring has been riddled with controversy. Fundamental principles and methods have been questioned, and ethical issues have been raised. Our objective was to ascertain the likelihood of reimbursement for drugs with an ODD compared with non-orphan drugs in EU5.

METHODS: We studied the statistical association between reimbursement status (Y/N) and ODD (Y/N). Data were retrieved for France, Germany, Italy, Spain, and the UK for 2011 to February 2024. Scope was restricted to branded drugs administered as monotherapy and approved by each country's centralized marketing authorization. We employed the GlobalData PharmOnline International (POLI) database.

RESULTS: Overall, 7968 reimbursement appraisals were included in the analysis. Of these, approvals had been granted for 1040 orphan drugs and 6008 non-orphan drugs. Similarly, there were negative reimbursements for 79 orphan drugs and 841 non-orphan drugs. These results indicate that, overall, an ODD has led to positive reimbursement across the included countries (odds ratio [OR], 1.84 [95% CI, 1.25–2.72]).

Individual countries offer mixed results. An ODD was positively associated with reimbursement in Germany (OR, 3.89 [95% CI, 1.52–9.96]) and Italy (2.22 [1.28–3.86]). In contrast, there was no evidence of an association in France (1.69 [0.95–2.99]), Spain (1.77 [0.86–3.62]), or the UK (1.27 [0.75–2.15]).

CONCLUSIONS: Available evidence suggests that an ODD can have a positive impact on obtaining reimbursement in EU5. However, these findings should be interpreted with caution for each market.