OBJECTIVES: To research how orphan drugs are evaluated by HTA agencies in England, Scotland, France, Germany, Sweden, Australia, Canada, and US, and to compare decisions taken on orphan drugs across three key European HTA agencies.
METHODS: Publicly available information on each HTA agency’s procedures for orphan drug evaluation were identified, categorized, and compared. Drugs with an EMA orphan designation that had been assessed by NICE between June 2021 and June 2022 were identified, and HTA outcomes for the identified drugs were analyzed and compared across three European HTA agencies that each used a different category of HTA procedure.
RESULTS: Three categories of HTA procedures were identified— standard: procedures for orphan and nonorphan drugs are identical; standard with flexibilities for orphan drugs: procedures are the same as standard but with some flexibilities allowed that may benefit orphan drugs; specialized: different procedures that may benefit orphan drugs compared with standard. In total, 29 orphan drugs were identified; 15/29 had HTA decisions published by all 3 HTA agencies (terminated, in progress, unclear decisions, and drugs not assessed by all 3 agencies were excluded). The number of drugs that achieved the best possible HTA outcome (e.g., full recommendation/reimbursement) for each agency was 0 (HAS; standard procedure), 0 (NICE; flexible procedure), and 9 (G-BA; specialized procedure). The remaining drugs achieved restricted HTA outcomes that negatively impact reimbursement. HTA outcomes were considered consistent across all 3 agencies for only 3 (20%) orphan drugs.
CONCLUSIONS: HTA procedures and outcomes for orphan drug HTA vary widely. Drug benefits are considered differently by agencies, and drugs considered beneficial by one agency may not be considered the same in another. This creates a complex market access environment for companies to navigate. Analysis of 3 agencies indicated there was 20% agreement in decisions made. This creates an unpredictable market that ultimately affects patient access.
