Advancing promising technologies and therapies towards clinical application is a complex undertaking. Additional challenges are faced when it comes to developing drugs for rare diseases such as the limited knowledge available for most diseases, the difficulties of generating adequate efficacy and safety data in small populations and the risk of financial unsustainability for both developers and health-care systems.
To address this issue, The International Rare Diseases Research Consortium (IRDiRC) Scientific Committee launched the Orphan Drug Development Guidebook. A patient focused guidebook that describes the available tools, incentives, resources and practices (building blocks) for developing traditional and innovative drugs/therapies for rare diseases and how to best use them.
The guidebook is based on the work done by 24 stakeholders and experts in rare disease drug development, representing academics, regulators, industry, health-care professionals, patients, public/non-profit research funders and consultancy firms and the mapping of a total of 110 building blocks available to orphan drug developers in Europe, USA and Japan. As a result, concise fact sheets for each of these supportive tools, were created including key information on their application and expert opinions on their advantages and disadvantages.
More information on the ODDG project can be found in the the commentary ‘Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook’ – authored by the IRDiRC ODDG Task Force led by Diego Ardigò and Virginie HIVERT and recently published in Nature Reviews – Drug Discovery.
The full guidebook and tutorial to understand more on the guidebook, are available on the IRDiRC website.
EATRIS mentoring service for the Rare Diseases research community can be found here