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Page 4 of 15 Zanello et al. Rare Dis Orphan Drugs J 2023;2:9 https://dx.doi.org/10.20517/rdodj.2023.04

Figure 1. Selection of the Medicinal Products. The graphics represents the methods used by the Task Force group to select cases of drug
repurposing in rare diseases. The medicinal products initially approved for common or rare indications were all repurposed into a new
rare disease. Approvals for the original and new indication were granted by the same regulatory authority. Medicinal products with an
extension of the same rare disease indication were excluded from the analysis.

considered.

● Cases were selected based on the ability of the Task Force members to identify and contact a
representative from each organization involved in the repurposing of the medicinal products.

Out of the ten selected repurposed drugs, targeted invitations were sent to six identified organization
representatives and six positive responses were received. The questionnaire was sent to the respondents and
interviews were conducted with the relevant representatives. The six drugs had initial indications for the
treatment of common or rare disorders and were all subject of a repurposed new indication for rare
diseases. The selection of medicinal products is presented in Figure 1.

Case identification of ongoing development programs
Through personal knowledge of the field, the Task Force identified cases of ongoing development programs
and contacted four organizations experienced in the management of drug repurposing projects (see
acknowledgments section) to perform structured interviews and collect additional information on both the
challenges and the key strategies implemented to increase the chance of success of a repurposing approach
in rare diseases.

Information from the questionnaires and interviews was integrated into Task Force discussions and the key
factors that should be considered by those embarking on repurposing projects were identified and agreed
upon through consensus.

KEY FACTORS FOR CREATING A SUSTAINABLE REPURPOSING PROJECT
Alongside expert Task Force opinion, the objective of the questionnaire and interview analysis was to draw
out common themes not yet considered, and to understand better the challenges and opportunities
associated with different models for drug repurposing. The Task Force Group identified ten core success
factors that are presented below and summarized in Figure 2.