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Zanello et al. Rare Dis Orphan Drugs J 2023;2:9 https://dx.doi.org/10.20517/rdodj.2023.04 Page 3 of 15
sustainable repurposing models in rare diseases. Using the collective knowledge of the Task Force, we
created a questionnaire that could be used to capture the major steps of each of the repurposing approaches.
Creation of the questionnaire
A targeted questionnaire addressing the following [Supplementary File 1]:
● Description of the company/organization
● Description of the drug repurposing approach and strategy
● Identification of the stakeholders
● Required evidence generation and repurposing of research studies
● Sources and mechanisms of funding
● Sustainability of the model chosen
● Patent status and exclusivity periods
● Identification of the barriers and challenges
● Measure of progress
● Result of the drug repurposing process
● Recommendations for a successful drug repurposing process
Selection of medicinal products
The Task Force extracted a list of repurposed medicinal products that have been approved for a rare
indication in the United States [10,11] and/or the European Union . Both oncology and non-oncology drugs
[12]
were included in the analysis. The Task Force group used a set of five criteria to refine the case selection and
create a list of ten repurposed drugs.
● For each selected case, the approval for the original and the repurposed indications must have been
granted by the same regulatory body, which is either the United States Food and Drug Administration
(FDA) or the European Medicines Agency (EMA).
● Cases related to a variation of the same indication (age, line of treatment) were excluded from the analysis.
● Cases were chosen to avoid the bias of selecting drugs approved for a second indication without the
initiation of repurposing studies. This criterion was applied to maximize the probability of identifying cases
no longer covered by the initial patent families and/or regulatory exclusivity.
● Diverse economic models (private funds, public funds, social bonds, and venture philanthropy) and drug
developers (big pharma, SMEs, academics, patient-led groups, and public-private collaborations) were
