Page 66 - Read Online
P. 66

Zanello et al. Rare Dis Orphan Drugs J 2023;2:9  https://dx.doi.org/10.20517/rdodj.2023.04  Page 13 of 15

               Diseases; Castleman Disease Collaborative Network; University of Pennsylvania, Center for Cytokine Storm
               Treatment and Laboratory; Cell Therapies Research & Services Laboratory) who gave their time to fill in the
               questionnaire and/or attend an interview. We are grateful for these reflections which helped formulate the
               10 key factors.


               Authors Contribution
               Led the Task Force: Hivert V
               Initiated the desk search analysis to identify cases of repurposed medicinal products into new indication in
               rare diseases: Zanello G
               Initiated the redaction of the manuscript: Hivert V, Zanello G
               All the co-authors revised and approved the manuscript, and all the co-authors participated in the final
               selection of use cases, the development of the questionnaire, the interviews and the result analysis.


               Availability of data and materials
               The datasets analyzed as sources for the lists are available from the corresponding author upon request.


               Financial support and sponsorship
               The IRDiRC Scientific Secretariat is funded by the European Union through the European Joint
               Programme on Rare Disease under the European Union’s Horizon 2020 research and innovation
               programme Grant Agreement N°825575. The Scientific Secretariat is hosted at the French Institute of
               Health and Medical Research (INSERM) in Paris, France.


               Conflicts of interest
               All authors declared that there are no conflicts of interest. The findings and recommendations in this article
               are those of the contributors, who participated based on their individual expertise and are responsible for
               the contents, and do not necessarily represent the views of the members of the International Rare Diseases
               Research Consortium (IRDiRC) nor any employers of the contributors.


               Ethical approval and consent to participate
               Not applicable.

               Consent for publication
               Not applicable.

               Copyright
               © The Author(s) 2023.

               REFERENCES
               1.       Bellera CL, Llanos M, Gantner ME, et al. Can drug repurposing strategies be the solution to the COVID-19 crisis? Expert Opin Drug
                   Discov 2021;16:605-12.  DOI  PubMed
               2.       Venkatesan P. Repurposing drugs for treatment of COVID-19. Lancet Respir Med 2021;9:e63.  DOI  PubMed  PMC
               3.       Giannuzzi V, Conte R, Landi A, et al. Orphan medicinal products in Europe and United States to cover needs of patients with rare
                   diseases: an increased common effort is to be foreseen. Orphanet J Rare Dis 2017;12:64.  DOI  PubMed  PMC
               4.       Nguengang Wakap S, Lambert DM, Olry A, et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet
                   database. Eur J Hum Genet 2020;28:165-73.  DOI  PubMed  PMC
               5.       Southall NT, Natarajan M, Lau LPL, et al; IRDiRC Data Mining and Repurposing Task Force. The use or generation of biomedical
                   data and existing medicines to discover and establish new treatments for patients with rare diseases - recommendations of the IRDiRC
                   Data Mining and Repurposing Task Force. Orphanet J Rare Dis 2019;14:225.  DOI  PubMed  PMC
               6.       Breckenridge A, Jacob R. Overcoming the legal and regulatory barriers to drug repurposing. Nat Rev Drug Discov 2019;18:1-2.  DOI
                   PubMed
               7.       Begley CG, Ashton M, Baell J, et al. Drug repurposing: misconceptions, challenges, and opportunities for academic researchers. Sci
   61   62   63   64   65   66   67   68   69   70   71