It was ratified Monday, 24 May 2023in the State-Regions Conference, the approval of the National Plan for Rare Diseases 2023-2026, realised after 7 years of work, with the aim of giving concrete answers to people with rare diseases, some 2 million in Italy, and their families.

Pharma Value analysed the chapter on 'Pharmacological Treatments'.

The concise text is structured in: Foreword; Objectives; Actions; Instruments; Indicators.

The Logical Framework methodology was used to verify the feasibility of the PlanThis is a matrix tool, used in programmes promoted by the European Commission and other international bodies, which is useful for clearly and concisely describing the different elements into which a project idea or plan is articulated, both in the strategy development phase and in the monitoring and evaluation phases.

The analysis was based on the cause-effect relationship between the different project hubs, to verify the sequentiality between general and specific objectives, expected results, indicators and activities.

In general, we have noticed:

• a discrepancy and/or absence of actions and indicators for certain objectives,
• ineffective formulation of instruments which, in our opinion, if stated more clearly and consistently should be called 'outputs' or 'results'.

In project management terminology, tools are the techniques used to support the realisation of activities, while results are the end point, or product, of a series of activities, aimed at achieving the formulated objectives.

You can see how, even the use of appropriate terminology to represent the focal points of the National Plan for Rare Diseases can affectnot only in understanding the text, but also in the operational phases of its implementation.

Following our analysis several doubts remain:

• Without indicators and actions describing how the objectives of the plan will be implemented, how can the plan be implemented by the regions and autonomous provinces?
• Will the territorial gap with different actions region by region continue to widen?
• Doesn't the absence of certain actions and indicators constrain planning and could it accentuate the heterogeneity of access to drugs for rare diseases?

Our opinion in this regard is that The National Plan for Rare Diseases 2023-2026, as it is currently formulated, represents a missed opportunity for patients and patients with rare diseases who need the country to work as a system.

If you are interested in learning more, you can read the article on the summary of the Consolidated Text on Diseases rare approved on 3 November 2021.