Presentation (CTI)

OBJECTIVES: The Conditional Marketing Authorization (CMA) is a regulatory tool that enables early access to medicines intended for serious or rare conditions, even in the presence of limited clinical evidence at the time of approval. However, the paucity of available data may present challenges for national evaluation processes—such as those conducted by AIFA—potentially affecting pricing negotiations and value assessment timelines.
This study aims to determine whether, in Italy, medicines approved under CMA experience longer pricing and reimbursement (P&R) timelines compared to other medicines authorized during the same period.
METHODS: A sample of 30 CMA-approved medicines—targeting rare diseases and conditions with high unmet medical needs—was analyzed. Publicly available data were retrieved from the official websites of AIFA, EMA, and the Italian Ministry of Health.
Procedural time was calculated as the interval between the date of dossier submission to AIFA and the date of publication in the Italian Official Journal of the final pricing and reimbursement determination. Results were compared with those of all other EMA-approved and P&R-negotiated medicines in Italy during the same period.
RESULTS: CMA medicines showed an average procedural time of 556 days (range: 195-970 days), compared to an average of 467 days (range: 176-918 days) for other medicines.
The difference remained consistent across yearly stratified analyses, with CMA products requiring, on average, 29% longer timelines.
CONCLUSIONS: Medicines approved under CMA appear to face significantly longer timelines in Italian pricing and reimbursement procedures. This may reflect the complexities involved in evaluating medicines with initially limited clinical evidence.
Developing dedicated assessment tools capable of managing uncertainty—without delaying access—may enhance the efficiency of the process and facilitate more timely availability for patients.