Repurposing of Medicines in the EU: Launch of a Pilot Framework

Abstract

Repurposing of authorised medicines has been under discussion for a long time. Drug repurposing is the process of identifying a new use for an existing medicine in an indication outside the scope of the original approved indication. Indeed, the COVID-19 health crisis has brought the concept to the frontline by proving the usefulness of this practise in favour of patients for an early access to treatment. Under the umbrella of the Pharmaceutical Committee and as a result of the discussions at the European Commission Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) a virtual Repurposing Observatory Group (RepOG) was set up in 2019 to define and test the practical aspects of a pilot project thought to provide support to “not-for-profit” stakeholders generating or gathering data for a new therapeutic use for an authorised medicine. The group's initial plan was impacted by the outbreak of the SARS-CoV-2 pandemic and the launch of the pilot needed to be postponed. This article describes the progress and the activities conducted by the group during this past and yet extraordinary 2020–2021 to keep the project alive and explores on the background of this topic together with the obvious opportunities this health crisis has brought up in terms of repurposing of medicines.

Repurposing empagliflozin in individuals with glycogen storage disease Ib: A value‐based healthcare approach and systematic benefit‐risk assessment

“…The role of the academia in drug repurposing for rare diseases is gaining attention and a framework for the repurposing of established medicines has been developed in the European Union. 22,23 Empagliflozin is wellestablished and safe in its current areas of usage. [24][25][26] According to an international questionnaire study, empagliflozin treatment was reimbursed in 78% of the cases, but mostly on an individual basis, depending on T A B L E 3 Budget impact analysis for 25 mg/day empagliflozin in persons with GSDIb in two nationwide scenarios.…”

“…The role of the academia in drug repurposing for rare diseases is gaining attention and a framework for the repurposing of established medicines has been developed in the European Union 22,23 . Empagliflozin is well‐established and safe in its current areas of usage 24–26 .…”

Identifying obstacles hindering the conduct of academic-sponsored trials for drug repurposing on rare-diseases: an analysis of six use cases

“…Some national competent authorities in Europe (for instance AEMPs, Spain) support informal consultations free of charge for investigator-initiated trials. More recently, in October 2021, a coordinated pilot program between EMA and HMA (Heads of Medicines Agencies) has been launched to support the repurposing of medicines [24]. The aim of this initiative is to support not-for-profit organizations and academia to gather or generate sufficient evidence on the use of an established medicine in a new indication with the view to have this new use formally authorized by a regulatory authority.…”

Sustainable approaches for drug repurposing in rare diseases: recommendations from the IRDiRC Task Force

“…However, the ODDG had not considered the aspects related specifically to repurposing and we aim to add a specific module to the existing materials. This represents a key IRDiRC activity in 2023 [24] .…”

“…The IRDiRC Task Force on Data Mining and Repurposing [5] initially addressed this topic by investigating the potential of biomedical data mining strategies to repurpose and accelerate the development of drugs for rare disease patients. More recently, IRDiRC launched the Drug Repurposing Guidebook Task Force [24] with the objective of describing the tools, incentives, resources and initiatives available to developers in the field. While the key principles of the Guidebook are applicable to the case of repurposing, no specific repurposing building blocks were created at the time and this will be the purpose of the creation of the "Drug repurposing Guidebook" module.…”