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Page 6 of 15 Zanello et al. Rare Dis Orphan Drugs J 2023;2:9 https://dx.doi.org/10.20517/rdodj.2023.04

Some of the approaches that this group, and other stakeholders, patient groups, for example, had classified
as repurposing were not labeled as such by pharmaceutical representatives. There is a thin border between
what some may call repurposing and the other one, extension of indication. This cannot be imputed only to
commercial sensitivities but also to a genuine lack of common understanding and terminology that we
should aim to overcome. One understanding of repurposing for companies involved in the development of
innovative medicines is that repurposing is considered only for medicinal products that are out of industrial
property (IP) and regulatory protection, while the extension of indication is a special type of variation of the
initial marketing authorization when new data are submitted and assessed to add a new indication to the
existing product.

In addition, repurposing in some quarters suffers from a “bad reputation” where well-known products have
seen their prices skyrocketing following a repurposing project [13,14] . This negative perception is not
connected to a unique category of stakeholders.

Furthermore, repurposing also brings a positive perception as it is sometimes the only way to provide a
therapeutic alternative to patients. Moreover, repurposing, in many cases, is an innovation in itself, where a
better understanding of the pharmacology of a medicinal product and the disease etiology reach a point of
convergence to stimulate a repurposing project [8,15] . This is increasingly the case where artificial intelligence,
[5]
data mining and in silico approaches are being used . In order to advance the field, we need to encourage
investment and continuous development in this field but also recognize that repurposing is a truly
innovative approach.

As for every therapeutic development, patients should be considered as equal partners
Within the sample of interviews, we observed a large variety of patient involvement, from patients and
patient organizations being the initiators and the drivers of the repurposing approach to patients only
considered at the time of recruitment for the clinical studies.

Amongst the various activities in which patients can take part, we have enlisted: identification of the
opportunity to repurpose a product, co-writing of the grant application, provision of the patient perspective
in the set-up of the objectives of the therapeutic development, in the design and the planning of the clinical
studies, patient identification, recruitment and retention for clinical studies, regulatory preparedness
(e.g., support to orphan drug designation), and dissemination to the broader patient community of the
outcomes of the development leading to better uptake of the product once on the market.

Often it appeared that the more the patients are involved as equal partners, the better, and that the positive
impact is correlated to the level of patient engagement. Interviewees that have reported no or few patients
involved in their development mention the identification of stakeholders as one of the main challenges in
the repurposing approach, leading to difficulties at the time of recruitment and consequences on the
business plan.

Respondents that have reported a high level of patient involvement, with all stakeholders (patients,
clinicians, and researchers) inputting in the design and planning of the studies, as well as regulatory
preparedness, mention a significant impact on the timeline of the development with faster clinical trial
recruitment and better uptake of the product in the market. This is comparable to previous studies that have
also emphasized the importance of patient involvement in the development and lifecycle of orphan
medicinal products [16-18] .