Patient-Based Evidence in Mechanism-Based Drug Repurposing: Barriers and Facilitators

OBJECTIVES: Mechanism-based drug repurposing identifies new uses for existing drugs based on mechanism-based disease definitions. Patients’ perspectives, captured via patient-reported outcome measures (PROMs) and qualitative interviews, are essential in assessing a repurposed drug's impact on quality of life. However, patient-based evidence (PBE) has played a limited role in drug repurposing studies. This study identified barriers and facilitators in collecting and using PBE in the mechanism-based drug repurposing lifecycle.

METHODS: The evidence-based Measurement Instrument for Determinants of Innovations (MIDI) was distributed to international stakeholders with experience in drug repurposing, including pharmaceutical companies, funders, patient representatives, clinical investigators, and HTA bodies. The MIDI (21 items) assessed determinants associated with PBE, the user, the organization, and socio-political context, using statements with a 5-point scale (completely disagree to completely agree) and additional open-ended questions. Items to which ≥20% of participants responded with totally disagree/disagree and ≥80% with agree/totally agree were considered as barriers and facilitators, respectively.

RESULTS: Interim analysis revealed more barriers than facilitators for embedding PBE in the repurposing lifecycle. Participants believed PBE offers a comprehensive drug assessment, is relevant for decision-makers, and highlights the patient's role in value assessment. Barriers included lack of clear procedures, formal arrangements and resources for PBE collection and use, and especially for qualitative research a lack of knowledge and expected low acceptance by decision-makers. PROM collection should start early and continue throughout the repurposing lifecycle, while qualitative research is recommended during clinical trial follow-up. Importantly, because mechanism-based drugs do not target symptoms directly, available PROMs may not adequately capture treatment effect. Data collection will be concluded in August 2024.

CONCLUSIONS: Stakeholders agreed that PBE is essential to assess the value of a mechanism-based repurposed drug. The study found that awareness, education, organizational facilitation, and standardization of PBE collection are needed for its effective use in the drug repurposing lifecycle.