Rare disease by definition implies the patient numbers are very small, and therefore the return on investment has historically been challenging. Whilst advocacy groups have always been active in drawing attention to the plight of rare disease patients, a number of scientific and technology advances are changing the economics of advances in rare disease research.
In addition commercial organisations now appear more driven to make social investments that offer even more hope for those suffering from rare disease. In this chaitime chaired by Prashant shah, we explore the latest science, the economics and the role of patients in the fight against rare disease.
Michael Binks is a VP of Rare Disease research at Pfizer and is a scientifically focused drug discovery and early clinical development professional. He is a clinical rheumatologist with 20 years’ experience in immuno-inflammation & rare diseases involving small molecules, small protein, monoclonal antibody & AAV gene therapy modalities.
Nicola Miller is the editor in Chief of Rare Revolution Magazine which is a voice and a platform for patients’ groups, industry, professionals, clinical & healthcare providers, and charities. She is also a trustee of the teddington trust which offers support & guidance to those affected by Xeroderma Pigmentosum.
Guilliams is the CEO of Healx and is passionate about using big data & artificial intelligence to accelerate treatments for rare diseases. He obtained his PhD at the University of Cambridge in the field of Biophysics & Neuroscience.