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Page 12 of 16 Fetro. Rare Dis Orphan Drugs J 2023;2:7 https://dx.doi.org/10.20517/rdodj.2023.06

Medicines Agencies (HMA) launched a pilot project to support the repurposing of drugs. That project is a
follow-up to the European Commission’s Expert Group on Safe and Timely Access to Medicines for
Patients (STAMP) discussions. The objective is to support not-for-profit organizations, including academia,
to generate sufficient evidence on the use of a well-established off-patent drug in a new indication.
Academic researchers will be connected with regulatory agencies for early scientific advice and with
companies for regulatory application. These investments must also be recognized in pricing and
reimbursement policies to make access a reality for all patients [40,41] . Candidate projects were selected in June
2022.

Horizon Europe funding program for research and innovation
Being part of consortia involving teams from industry, academia, SMEs and patient associations may help
academic partners build lasting collaborations and networks. A call for proposal “Tackling diseases (2021)
(HORIZON-HLTH-2021-DISEASE-04)” was proposed under the Horizon Europe program and closed on
September 21, 2021. 13 proposals among 253 submitted were related to the topic “Building a European
innovation platform for the repurposing of medicinal products”. Among key outcomes, proposals had to
address a widened collaboration to set up a sustainable platform and an innovative repurposing model
integrating different components such as IP, methodological, scientific, regulatory, and financial aspects.
[42]
Particular attention was given to supporting academic-driven research .
IRDiRC Task Force on drug repurposing
As a brief overview, one could also mention the recent IRDiRC Task Force on DR based on the IRDiRC
Orphan Drug Development Guidebook . The DR Guidebook is aimed at facilitating DR for rare diseases
[43]
by identifying relevant available tools and practices, defining a list of Building Blocks (tools, incentives,
resources, and initiatives), and creating detailed factsheets for each Building Block. The factsheets will be
based on a systematic literature review and the expertise of the Task Force members. They will also provide
advice and recommendations. A web application and an article are expected by the end of 2023.

DR platforms are an example of tools and resources to be found in this DR Guidebook. The DR platform
established by the FDA is still available. It can be located now at cure.ncats.io. CURE ID (Infectious Disease)
is a collaboration between the FDA and the National Center for Advancing Translational Sciences
(NCATS), part of the National Institutes of Health (NIH). FDA and NCATS have made critical updates to
CURE ID to be a more effective tool in the COVID-19 public health emergency.

Recommendations from a recent pharmacology and translational research round table
A recent “Pharmacology and translational research” round-table of the Giens Workshops formulated and
[44]
[44]
published a series of recommendations to help overcome sticking points and obstacles to DR . The Giens
Workshops are held in Paris every year; they are a think tank whose objective is to advance thinking on
current issues in pharmacology and clinical research for therapeutic innovation and health technology
assessment. The meetings are organized into round tables, each covering pre-defined themes (translational
research; clinical research; health technologies including diagnostics, drugs, and medical devices; health and
economics; organizational and regulatory aspects, and topical issues). Each round table aims to bring
together experts from various backgrounds. In 2022 one of the round tables was dedicated to DR around the
following key points:

Optimizing access to and use of databases
Databases are a basis for DR methods and have, therefore, to be designed in a proper manner to achieve
relevant results. They can be used to predict drug-target interactions and discover new treatment benefits of
the existing drugs. Access has to be facilitated to pharmacological databases (chemical and molecule