Price and reimbursement for orphan medicines and managed entry agreements: does Italy need a framework?


  • Claudio Jommi Cergas SDA Bocconi School of Management, Milan - Italy
  • Antonio Addis Department of Epidemiology, Regional Health Service (Regione Lazio) Member of the Scientific Technical Committee, Italian Medicines Agency (AIFA), Rome - Italy
  • Nello Martini Fondazione ReS (Research and Health Foundation), Rome - Italy
  • Elena Nicod Dolon Ltd, London - UK and Cergas SDA Bocconi School of Management, Milan - Italy
  • Marcello Pani Agostino Gemelli IRCCS University Hospital Foundation, Rome - Italy
  • Annalisa Scopinaro UNIAMO (Italian Federation for Rare Diseases), Rome - Italy
  • Sabine Vogler WHO Collaborating Centre for Pharmaceutical Pricing and Reimbursement Policies, Austrian National Public Health Institute (GÖG), Vienna - Austria



Italy, Managed entry agreements, Orphan medicines, Value framework


This article illustrates a consensus opinion of an expert panel on the need and usefulness of a framework for price and reimbursement (P&R) process and managed entry agreements (MEAs) for orphan medicines in Italy.

This opinion was gathered in three rounds: an introductory document was sent to the panel and discussed during a recorded online meeting. A second document was sent to the panel for their review. In the third step the final document was validated. Members of the expert panel are the authors of the article.

The panel agreed that Italy does not need a specific value framework for orphan medicines, driving the P&R process. Rather, a more structured value framework for all medicines tailored to the specific drugs can be useful. For orphan drugs, the panel advocated for a multidisciplinary approach and the contribution of different stakeholders to value assessment, and acknowledged the importance of addressing, more than for other drugs, unmet needs, equity issues and societal value. The panel raised the need of increasing the importance of patient-reported outcomes. Experts, acknowledging the growing criticisms in implementation of outcome-based agreements in Italy, expressed their position against their abandonment in favour of discounts only and supported orphan medicines as natural candidates for these agreements.

Finally, the panel made some recommendations on the appraisal process for orphan medicines, including an early discussion on the uncertainty of the evidence generated and the adoption of a structured approach to identify the agreement, which better responds to the uncertainty.


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How to Cite

Jommi, C., Addis, A., Martini, N., Nicod, E., Pani, M., Scopinaro, A., & Vogler, S. (2021). Price and reimbursement for orphan medicines and managed entry agreements: does Italy need a framework?. Global and Regional Health Technology Assessment, 8(1), 114–119.



Point of View


Received 2021-05-14
Accepted 2021-07-26
Published 2021-08-05


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