Drug Repurposing Guidebook


Developers in the rare diseases field, particularly those engaged in repurposing approaches, include diverse stakeholders ranging from big pharma, but increasingly and more often to clinicians and not for profit organisation including patients-led initiatives. Hence the whole Rare Disease Community and in particular the patients would benefit from a guide helping developers to navigate the regulatory & development tools and resources, efficiently and effectively repurposing medicines in new rare disease indications.


The objective of the Drug Repurposing Guidebook is to help developers (of all kinds) navigating the rare disease landscape and identifying specific tools and practices of relevance for repurposing projects. The creation of the Development Guidebook will focus on repurposing approaches, following the same successful methodology used for the Orphan Drug Development Guidebook, i.e. explore incentives, regulatory tools, initiatives, development tools (‘building blocks’) that exists or are missing for drug repurposing. 


Assembly of the Task Force Group (Q1, 2022) 


  • Anneliene Jonker – University of Twente, The Netherlands
  • Dan O’Connor – Medicines and Healthcare products Regulatory Agency, UK
  • Marjon Pasmooij – University Medical Center Groningen, The Netherlands
  • Ken Sakushima – Pharmaceuticals and Medical Devices Agency, Japan
  • Maria Cavaller  – EURORDIS – Rare Diseases Europe, France
  • Michela Gabaldo – Fondazione Telethon, Italy
  • Florence Guillot – Agence Nationale de la Recherche, France
  • Oxana Iliach – Canadian Organization for Rare Disorders, Canada
  • Virginie Hivert – EURORDIS – Rare Diseases Europe, France
  • Ramaiah Muthyala – India Organization for Rare Diseases, India
  • Christine Fetro – Fondation Maladies Rares, France
  • Sonsoles Hortelano Blanco – National Institute of Health Carlos III, Spain
  • Ania Korsunska – Castleman  Disease  Collaborative  Network, USA
  • Heather Stone – Food & Drug Administration, USA
  • Luca Falciola – Scibilis SRL, Belgium
  • Marco Schmidt – biotx, Germany
  • Maria Gogou – Evelina London Children’s Hospital, UK
  • Martin de Kort – EATRIS, The Netherlands
  • Peter Bram ’t Hoen – Radboud UMC, The Netherlands
  • Simon Day – Clinical Trials Consulting & Training Limited, UK
  • Vinciane Pirard – Senior HealthCare Advisor, Belgium
  • Nivedita Valentine​ – Global Associate Vice President, Product Innovation, Pharmanovia, UK