On Tuesday 12 December 2023, the Senate, in the Zuccari Room of Palazzo Giustiniani, presented the VII Ossfor Annual Reportcarried out by Omar - Rare Disease Observatory, and Crea Sanità - Centre for Applied Economic Research in Health, in collaboration with leading experts in the field.
The report offers an up-to-date overview of the world of rare diseases and orphan drugs, contains an in-depth look at the legislation regulating the sector, illustrates the processes and timelines of the authorisation pathways for orphan drugs, takes an in-depth look at consumption and regional expenditure, and provides a snapshot of ongoing trials around the world with a dedicated focus on Italy.
The Report contains 19 contributions that can be grouped according to four perspectives: epidemiological analyses, access and research in the field of orphan drugs, NHS expenditure, and governance aspects.
In continuity with previous years, trends in the sector were analysed, which showed that the situation is unchanged; while recognising the improvements in terms of new therapeutic opportunities reaching the market and the access times achieved in the medium term, some criticalities persisted, which were also highlighted in the care responses and, in particular, in the reports from patients and their Associations.
Again, OSSFOR points to a transitional phase, waiting for the effects of Law No. 175 of 2021 and the Tariff Decree to take effect at the national level, and at the European level to complete the process of reforming the regulatory framework for orphan and paediatric medicines in the EU, for which the European Commission has deemed it necessary to initiate a review (Inception Impact Assessment - Revision of the EU legislation on medicines for children and rare diseases, 2020).
At the presentation event, a number of topics of great interest were addressed, such as the governance of rare diseases, the reorganisation of networks, and the revision of regulations for orphan drugs. With the help of the associations of the Rare Disease Alliance, the PNMR and the regulation of the sector were also discussed, in order to understand what problems patients and families are facing and identify possible solutions. Finally, the implementation of the European regulation for orphan drugs was discussed, which will have a wide-ranging impact on the entire sector.
Also last year, Pharma Value participated in the presentation of the OSSFOR Annual Report, which included the article "Proposed regulatory reform of 648/96 for 'timely access' of innovative drugs" by Valeria Viola, Ginevra Mastroianni (Pharma Value) and Cinzia Timpano (University of Eastern Piedmont).
