How to Teach an Old Medicine New Tricks: The Importance of Repurposing Medicines for Patients

Le résumé fourni par la source

Eurordis, the European Organisation for Rare Diseases, representing more than 1075 patient groups and around 2000 distinct rare diseases, exists since 1997. The first purpose of creating Eurordis by that time was to support the adoption of the EU Regulation on Orphan Medicine Products, and a large part of our activities consists in accompanying the R&D and evaluation of medicines towards their market authorisation and after.Yet, patients have always requested Eurordis’ support in situations where they had difficulties obtaining a medicine off-label and/or getting the reimbursement/coverage decision they expected. Eurordis conducted a survey along its members to detect possible off-label uses in rare diseases and engaged with Marketing Authorisation Holders and regulators to discuss how to make progress. As early as 2012, Eurordis proposed a specific scientific advice to discuss how to transform an off-label use into an on-label one, as well as Good Off-Label Use Practices (GOLUP). In 2018, Eurordis adopted a position on possible measures to facilitate neo-positioning of pharmaceuticals.Maybe the best example of neo-positioning of a medicine is thalidomide, from its first use, nausea during pregnancy with the consequences we all know, to treating leprosis and then multiple myeloma, as first suggested by the sister of a myeloma patient presented to Dr Bart Barlogie in Little Rock, Arkansas, in the late 1990s. Clinicians and patients test many different products, sometimes with a strong rational, sometimes less. There is a wealth of potential innovations in these ideas, and patients and organisations are eager to play a role in these developments, but realising the challenges ahead: <!-- named anchor --> How to protect the idea of the person who thought of the new use first? How to convince Marketing Authorisation Holders when the new use is in a rare disease? Sales won’t be impacted much. How to conduct trials, if the off-label use is well established? And why to do this effort as long as off-label use is possible? Even if a new indication is obtained, how to gain returns on investments at least to cover the costs (research and regulatory)? Do we ned to re-think the organisation of the market for new uses? What if the lowest price product is systematically substituted? Is there a risk of opportunistic price increases for the new use? From these experiences and questions, the second part will address possible solutions to move forward and how to transform these uses into evidence-based medicines? Apart from RePo4EU and Remedi4All, some actions in Member States will be explained, based on the analysis of real-world data and new analytic methods. Lastly, measures that can be taken to prevent abuse of dominant positions as a response to patients’ concerns when medicines are repurposed will be presented.

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