OBJECTIVES : Orphan medicines are often associated with diseases of high unmet need with limited treatment options, thereby warranting rapid patient access. However, time to access for orphan medicines is highly variable, with months to years before an access and pricing decision. This research investigates the impact of disease unmet need, strength of supporting evidence, and price on the time to access for orphan medicines in England and France.

METHODS : 14 therapies with orphan designation and EMA approval (2014-2018) in non-oncology indications were selected. Following a structured literature review they were qualitatively assessed on their unmet need based on available treatment options, and the robustness of supporting clinical and economic evidence. HTA reports and published prices in both countries were reviewed to understand the impact of unmet need, strength of evidence, and price on time to access. Time to HTA decision (EMA approval to draft NICE recommendation/ TC decision) and price negotiation length (draft NICE to final NICE recommendations/ TC to CEPS decision) were considered.

RESULTS : Time to HTA decision was <1–26 months in England, and 4–23 months in France. Price negotiations took 3–46+ months in England, and 4–41+ months in France. Strength of evidence had the largest impact; evidence package uncertainty caused extended HTA timelines but had no identifiable impact on length of price negotiation. No relationship between unmet need, or price, on decision timelines were identified.

CONCLUSIONS : This research shows a trend between strength of evidence and time to HTA decision, but no trend for unmet need or price. None of the three factors had an observable impact on price negotiation timelines. Based on insights from former HTA and pricing committee members, factors such as early engagement, flexibility, and cooperation between the orphan medicine manufacturer and the Health Authority are important for driving faster negotiations. However, this needs validation through future research.