OBJECTIVES: Orphan designation (OD) is meant to facilitate development of orphan drugs in Europe. However not all drugs indicated in rare diseases have OD. We investigated if OD impacts HTA opinions for drugs indicated in rare diseases.

METHODS: Drugs approved after 2015 indicated in rare diseases with and without OD were identified through last Orphanet report. HTA opinions of HAS, G-BA, NICE and SMC of these drugs were classified as positive, positive with restrictions and negative. Statistical descriptive analysis was performed to compare opinions in the two groups (OD vs. non OD).

RESULTS: CONCLUSIONS: Overall OD had a positive impact on market access of drugs indicated in rare diseases, especially in Germany, where added benefit is recognized by law for drugs with OD (with annual revenue of up to 50 million Euros), and in Scotland, where HTA framework for orphan drugs exists. Differences were less pronounced in England (HTA framework only for ultra-orphan drugs). In France no positive impact of OD could be seen (no HTA framework for orphan drugs).