Rome, 16 October 2024 - In the context of rare diseases, orphan drugs play a key role because they treat conditions for which no alternative therapies exist. However, the current process of negotiating the price and reimbursement of these drugs presents critical issues, linked to the use of inappropriate comparators, such as off-label or patent-expired drugs.

This theme was the focus of an investigation conducted by Rarelab and Pharma Value, presented today during the 8th Orphan Drug Day, in Romeand included in the White Paper 'The Orphan Drug Comparator, Limits and the Right to Better Care' (pdf at the bottom of the page)

Valeria Viola, Founder & General Manager Pharma Value, explained that: "Orphan drugs are often compared with off-label, often patent-expired treatments, a practice that undermines the recognition of their therapeutic and economic value, investment in research, and negatively affects access times for rare disease patients. Therefore, there is a clear need to find replicable and more appropriate criteria for the identification of comparators'.

Francesco Macchia, Co-founder and CEO Rarelab, emphasised the need for an update in the evaluation criteria to recognise the uniqueness of orphan drugs and ensure timely access to innovative treatments.

The meeting was attended by pharmaceutical industry representatives, institutional representatives and patient associations: 𝐋𝐚𝐫𝐚𝐚 𝐍𝐢𝐜𝐨𝐥𝐞𝐭𝐭𝐚 𝐀𝐧𝐠𝐞𝐥𝐚 𝐆𝐢𝐭𝐭𝐨, President of the Scientific Economic Commission of AIFA; 𝐏𝐚𝐭𝐫𝐢𝐳𝐢𝐚 𝐏𝐨𝐩𝐨𝐥𝐢, Director of the National Centre for Drug Research and Evaluation of ISS; prof. Vincenzo Salvatore, Partner of Simmons&Simmons; Fulvia Filippini, Coordinator working group Orphan Drugs and Rare Diseases, Federchimica Assobiotec; Giorgia Tartaglia, Vice-President A.I.Vi.P.S ETS Associazione Italiana Vivere la Paraparesi Spastica, EUPATI expert patient.

All participants shared the need to find new and alternative mechanisms to ensure a fair and rapid evaluation of orphan drugs taking into account their particularities.