While one rare disease might impact a relatively small segment of the global population, added up the patients living with these conditions are a big deal. According to the National Institutes of Health (NIH), there are approximately 7,000 rare diseases affecting between 2m and 30m people in the US alone—about one in every 10 people in the country.
As a result, rare and orphan diseases are a big deal to the pharma industry. There exists a wealth of opportunities for drug companies and their research partners if they understand the particular needs of such patients and the conditions they face.
For Rare/orphan diseases, special patient population, the Outsourcing-Pharma team has put together an impressive group of experienced experts who have graciously agreed to take time out of their busy schedules to talk to our audience about key issues that impact rare/orphan disease research and drug development. Topics they plan to tackle include addressing recruitment and enrollment, ins and outs of the patient experience, and how sites and sponsors can best address their needs.
Dominique Pichard is the chief science officer of the International Rett Syndrome Foundation and the mother of a child living with the disease. She helps drive the organization in its mission to supporting families dealing with Rett syndrome, building awareness, and pursuing potential treatments.
Sanjeev Luther serves as president and CEO of Rafael Pharmaceuticals, a drug firm focused on developing treatments for rare cancers. Bringing more than 30 years of experience to the firm, he also has held positions with Bristol-Myers Squibb, Novartis, Bausch and Lomb, GE Healthcare, and Mallinckrodt.
Teresa Rubio is a health scientist administrator with the US Food and Drug Administration’s (FDA’s) Office of Orphan Products Development (OOPD), which provides a range of resources for professionals in the field. Her past positions include senior safety evaluator at the CDER Office of Surveillance and Epidemiology’s Division of Pharmacovigilance.
Contributing to the question and answer session after the trio of presentations is Lewis Fermaglich, acting senior clinical advisor at the OOPD. He brings to the table a range of experience with orphan drugs and rare pediatric disease designations.
Rare/orphan diseases, special patient population goes live on July 7. Register now and catch invaluable information from leading experts during this exclusive, free online event.