PRESS RELEASE (Pharma trade media only)
London, 1 November 2023
THE GUIDEBOOK HAS BEEN PUBLISHED IN NATURE REVIEWS DRUG DISCOVERY AND IS DESIGNED TO SUPPORT A RANGE OF STAKEHOLDERS WHO WANT TO START A REPURPOSING PROGRAMME FOR RARE DISEASE
AS PART OF THE IRDiRC TASK FORCE, PHARMANOVIA AND MEDICINES FOR EUROPE AIM TO BOOST THE POTENTIAL OF DRUG REPURPOSING AS A SOLUTION FOR RARE DISEASES THAT ARE IN URGENT NEED OF THERAPEUTIC OPTIONS
Pharmanovia, a global pharmaceutical company that revitalises, extends and expands the lifecycle of already established medicines, has today announced its involvement in the development of the International Rare Disease Research Consortium’s (IRDiRC) Drug Repurposing Guidebook, which has been published in Nature Reviews Drug Discovery Journal.
The guidebook is authored by the IRDiRC task force comprising 25 research and development experts, including Pharmanovia.. Together, this task force is committed to realising IRDiRC’s mission of supporting 1,000 new therapies for rare diseases to be approved, the majority of which will focus on diseases without any existing approved options, by 2027.
In line with this aim, the Drug Repurposing Guidebook has been created over the past 18 months to help a wide range of stakeholders navigate the rare disease landscape and identify specific tools and practices of relevance for repurposing drugs outside their original indication. This guidebook builds on the Orphan Drug Development Guidebook, published by the IRDiRC in 2020, which explores incentives, regulatory tools, initiatives, and development tools that exist or are missing for drug repurposing.
Currently, fewer than 5% of rare diseases are estimated to have at least one approved treatment – known as ‘orphan drugs.’i The high costs of drug development, combined with a limited understanding of rare diseases and regulatory challenges, have hampered therapeutic advancements and, critically, access to potentially life-extending medications for people living with these diseases. While drug repurposing has emerged as a potential solution for expanding treatment options for rare diseases, the absence of guidance and a poor understanding of the process has stifled its potential. This new guidebook aims to help address these challenges, and in turn, harness the potential of drug repurposing to provide hope for those seeking therapeutic options for rare diseases.
Nivedita Valentine, Associate Vice President, Product Innovation at Pharmanovia, who was a part of the International Rare Disease Research Consortium’s (IRDiRC) Drug Repurposing Guidebook experts panel and member of the Medicines for Europe VAM (Value added medicines) leadership committee commented: “At Pharmanovia, we have long recognised and championed the advantages of drug repurposing, so it’s an honour to be part of the IRDiRC task force, drawing upon our expertise to help pave a clear pathway for drug repurposing. This is a pivotal stride towards a brighter future for the over 300 million people living with rare diseases, many of whom have limited treatment options.ii”
IRDiRC Representatives Anneliene Jonker and Marjon Pasmooij added:
“This guidebook represents a major achievement for both the IRDiRC and the rare disease community, as we move closer towards our goal of the approval of 1,000 new therapies for rare diseases by 2027. Our collaboration with Pharmanovia, who share the same vision regarding drug repurposing, has added substantial value by bringing a commercial perspective to enhance the quality of care for individuals living with rare diseases.”
· 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 / VAM Sector Group, Medicines For Europe
Notes to editors. Please find the Nature Reviews Drug Discovery publication here (paywall) https://www.nature.com/articles/d41573-023-00168-9
Find more information on the IRDiRC Task Force here
For further information, please contact:
Pharmanovia is a global lifecycle management healthcare company. Their purpose is to make medicines fit for tomorrow, to improve the lives of patients globally.
They do this by enhancing established medicines by rediscovering, repurposing or re-engineering iconic brands to improve patient outcomes and experiences both through in-house development and strategic partnerships.
In addition to expertise in life cycle management, Pharmanovia has a well-established presence in clinical development and the management of clinical trials. This extends beyond well-known brand names to encompass new-clinical entities that may have clinical trial commitments in the future.
Their diverse and growing team operate in over 160 countries across the globe, delivering high-quality solutions, ethically and sustainably, across our four core therapeutic areas – Endocrinology, Neurology, Cardiovascular and Oncology. For more information about Pharmanovia, please visit www.pharmanovia.com
i Willmer, G. and Willmer, G. (2022) The building blocks to make rare disease treatments more common, Horizon Magazine. Available at: https://ec.europa.eu/research-and-innovation/en/horizon-magazine/building-blocks-make-rare-disease-treatments-more-common (Accessed: 26 October 2023).ii Life with a rare disease (2023) Rare Diseases International. Available at: https://www.rarediseasesinternational.org/living-with-a-rare-disease/#:~:text=Over%20300%20million%20persons%20live,5.9%25%20of%20the%20global%20population. (Accessed: 27 October 2023).