Findacure: empowering patient groups for drug development

Abstract

There is a large unmet need for helping rare disease patient groups and Findacure aims to empower these groups to become effective campaigners for change. Through its scientific meetings, Findacure also aims to gain the support of the scientific community in recognizing the importance of ‘fundamental diseases’ (conditions that manifest themselves as extreme and rare genetic disorders that offer a unique opportunity to better understand other diseases, including many common ones) and help to create a receptive research environment. Since it may often be commercially unattractive to develop treatments for many fundamental diseases, Findacure helps facilitate patient groups to themselves drive the development of treatments for fundamental diseases, using multi-stakeholder collaborative models.

Keywords::

• fundamental disease
• orphan drug
• patient group
• rare disease

Acknowledgments

AK Hall is a medical doctor with 20 years of drug development experience in the pharmaceutical industry, the last 4 as a consultant exclusively in rare diseases (since August 2014, he is working as Medical Director at Prosensa, a company developing treatments for Duchenne muscular dystrophy). He speaks and chairs conferences on orphan drugs and publishes extensively on issues related to orphan drugs, from clinical development strategies to pricing and value issues. He provides this expertise pro bono to Findacure. N Sireau is Chairman and CEO of the AKU Society since 2005 and has raised more than £11 million in funding and taken a treatment into clinical development. He is also Co-founder and Chairman of Findacure. Previously, he founded the charity SolarAid in 2006, which he grew into a multi-million pound award-winning social enterprise. F Raffai is Project Manager at Findacure. As part of this role, she develops new projects, organizes patient group training and scientific conferences, runs Findacure’s online communications and leads on funding applications.