Introduction
In recent years, new business models (e.g. patient-led R&D efforts) – alternative to the predominant pharmaceutical and academic R&D models – have emerged to address remaining patient unmet need, specifically in under-researched and/or low prevalence rare conditions. The main aim of the project is to identify key take-aways and potential recommendations to IRDiRC stakeholders regarding the suitability and key recurring elements on sustainable economic models for development and commercialization of orphan drugs. In particular, the Task Force intends to:
Objectives
- Review successful case of academic and industrial drug repurposing;
- Identify their characteristics and similarities;
- Characterize the specificity of their economic models and their long-term outcomes;
- Develop recommendations to IRDiRC stakeholders and the RD community.
Plans and timeline
- Formation of the task force group (Q3 2020)
- Identification of use cases in drug repurposing (Q4 2020)
- Develop a questionnaire and conduct semi-structed interviews (Q2 2021)
- Disseminate the findings and the recommendations (Q4 2021)
Members (7)
- Diego Ardigo – Chiesi, Italy
- Florence Guillot – Agence Nationale de la Recherche, France
- Virginie Hivert – Eurordis, France (Co-Chair)
- Oxana Iliach – Canadian Organization for Rare Disorders, Canada
- Hervé Nabarette – AFM-Telethon, France
- Daniel O’Connor – Medicines and Healthcare Products Regulatory Agency, UK (Co-Chair)
- Anne Pariser – National Center for Advancing Translational Sciences at NIH, USA