Great news for rare disease patient groups announced by Nick Sireau and Findacure:
Dear friends,
This new book edited by rare disease experts Anthony Hall and Nicolas Sireau provides a collection of chapters by world leaders in the field of orphan diseases and drugs in order to help patient groups grow fast and sustainably. As a practical, hands-on guide for research and drug development, it provides tools and advice on topics such as:
– How to set up and manage a patient group: US and UK perspectives.
– How to build an international research consortium.
– How to promote basic research.
– How to engage with academia for drug discovery.
– How to engage with industry.
– How to set up a centre of excellence.
– New funding strategies for clinical research.
– Crowdfunding for rare diseases.
– Practical thinking to win bids.
– Orphan drug legislation and adaptive licensing.
– Early interaction with regulators and parallel scientific advice with health technology assessors.
– How to navigate the regulatory process.
– Empowering children to become involved in the research agenda.
– Natural history studies.
– Clinical efficacy measures and surrogate endpoints.
– Statistical considerations.
– Setting up and running a clinical trial.
– Patient registries.
– How to interact with government and lobby for change to the clinical trials system.
– Patent protection and ownership of patents.
– Engaging patients and carers online for clinical trials.
If you are involved in patient groups or rare diseases in any way, then this book will provide valuable reading.
—
Dr Nicolas Sireau
