Rare Diseases and Orphan Drugs

Biography

Biography: Serge Braun

Abstract

Partnerships between industry, public institutions and charity organizations may lead to win-win situations and new models for advanced therapeutic medicinal products development. The example of AFMTelethon, a patient organization dedicated to rare diseases, will show why the caricatural vision of patient associations in an activism, awareness and emotional support holding pattern, should slip our mind. AFMTelethon has raised >$100M/year for 29 years, which helped to reframe the whole field, bringing it from a rather remote scientific and commercially unattractive area to a very active and innovative ground. A striking demonstration is the comprehensive genetic map of the human genome published in 1992 and 1996 by its non-for-profit biotech Genethon; a quantum leaps in the human genetics field. Not only patient organizations can serve as disease-specific experts, they more importantly work on reducing bottlenecks/barriers for all levels of the process of product development, initiate and drive research projects, provide tools (e.g., databases, registries, large animal facilities, high-throughput screening and even GMP biotherapeutics plateforms) and represent a voice for all aspects (ethical, regulatory, technical) of innovative drug developments. Through AFMTelethon initiatives, examples will be given of successful ecosystems, which also serve as models for frequent disorders. This includes shared scientific and financial risks in translational research by directly funding and supporting drug development programs in industry or in academia, putting in place its own research and development facilities, generating innovative drugs that are now either in the clinic or approved, founding start-up companies out of its own portfolio, taking equity shares in biotech companies, launching a venture capital fund and a new pharmaceutical entity together with the French Public Investment Bank and launching a new type of private company dedicated to cell and gene therapy. The different actions presented are also driven by the growing challenge of fair price of the therapeutics will be addressed.