OBJECTIVES: Since implementing its new innovativeness algorithm, AIFA have designated 12 drugs as innovative. This study analysed key characteristics of these 12 drugs while also reviewing the French HAS’s decisions for the drugs to identify similarities and differences between the two HTA bodies’ decisions.

METHODS: Assessment reports for drugs designated as innovative between September 2017 and May 2019 were retrieved from AIFA’s website. EMA reports and HAS decisions for the same drugs were downloaded from each organisation’s website. Data relating to orphan designation, pivotal trials, AIFA and HAS ratings, and the rationale for AIFA and HAS ratings were systematically collected using an extraction protocol, before detailed analysis and interpretation.

RESULTS: Half of drugs rated innovative had orphan designation. AIFA rated most (10/12) innovative drugs as offering ‘important’ therapeutic value (2/12 ‘moderate’ therapeutic value). Most drugs were considered to address ’moderate’ (5/12) or ‘important’ (5/12) unmet needs; only 2 were rated as addressing ‘maximum’ unmet needs. All drugs’ submissions were supported by ≥1 RCT; 10/12 submissions included ≥1 RCT powered for superiority and 7/12 included ≥1 RCT using an active comparator. Three of the five drugs lacking an active comparator RCT had orphan designation. AIFA only rated 3/12 drugs’ clinical trial data as being of ‘high’ quality (3/12 ‘low'; 6/12 ‘moderate'); critique related to comparators, generalisability, randomisation and missing data.

Of drugs designated innovative by AIFA, 11/12 were awarded an ‘important’ clinical benefit (SMR) rating by France’s HAS. In 9 cases, HAS awarded a ‘minor’ added therapeutic value (ASMR IV) rating.

CONCLUSIONS: AIFA innovativeness designation is mainly driven by unmet needs and therapeutic value of drugs; when the unmet need is high, clinical data of lesser quality are sometimes accepted. There is high overlap in AIFA’s and HAS’s assessment of drugs’ intrinsic value.