Nate Hughes, MPP, MPH
Johns Hopkins Alumnus
The global rare disease diagnostics market was valued at $45.18 Billion in 2017 and is anticipated to reach $86.15 Billion by 2025. Factors that contribute to this growth are as follows: high incidence and prevalence rate of rare diseases, R & D funding patterns allocated for rare disease research, rare disease registries, high diagnosis of rare disease in North America, and lastly, extensive investment in rare diseases therapeutic categories. According to Paradigm Global Events, the Asia-PAC region is expected to register the highest CAGR of 9.22% during the forecast period from 2018-2025. I wanted to share my thoughts about the most compelling rare disease event I have attended in my career thus far, the 10th Annual Orphan Drugs & Rare Disease – West Coast Meeting held at the Embassy Suites, South San Francisco from July 23-24, 2019 hosted by the one of leading conference organizers in biotech, Paradigm Global Events out of the UK. For example, CAR-T therapeutic developments alone has impressive statistics.
Regulatory authorities around the world are approving more therapies and receiving more INDs (investigational new drugs) in the cell and gene therapy (CGT) space than ever before. In 2018, the FDA Center for Biologics Evaluation and Research (CBER) received more than 150 INDs for gene therapy products, bringing the total number of active INDs up to nearly 800 applications. The Global Genes Project estimates some 300 million people worldwide are affected by a rare disease. The European Organization for Rare Diseases (EURORDIS) estimates that as many as 5,000 to 7,000 distinct rare diseases exist, and as much as 6% to 8% of the population of the European Union is affected by one. Only about 400 rare diseases have therapies and about 80% have a genetic component according to Rare Genomics Institute. Some of the topics of the two-day Summit included the following: the major market drivers in global rare disease, trends and clinical advancement in cell gene therapy, insurance reimbursement strategies, market access and affordability, accessibility, and acceptability, and lastly, the collaboration between patient advocacy organizations – such as Global Genes – and industry to lead to greater health outcomes and higher quality of life.
Sponsors included the following: Eiger Pharmaceuticals, INVITAE, DURBIN, Pulse Infoframe, and rareLife Solutions. This year had perhaps the most impressive speakers to date, including Lucas Kempf, Medical Officer for the Rare Disease OND at FDA, Serge Messerlian, Angela Ramirez Holmes at Cal Rare, Jim Shaffer, Chief Business Officer at Eiger BioPharmaceuticals, Mridula Shukla, Global Program Lead at Adverum Biotechnologies, David Liu, CSO at Protagonist Therapeutics, Javier San Martin, Senior VP and Head of Global Clinical Development at Ultragenyx, Fred Holdbrook, Director of Biostats at Amicus Therapeutics, Bruno Gagnon, Senior VP, Development Ops at Eidos Therapeutics, Eric Mueller, Associate Director, Cell Therapy Ops at Orchard Therapeutics, among several others.
The 11th Annual Orphan Drugs & Rare Diseases Global Congress 2019 of the Americas was held in Boston, MA. From September 9th-11th, 2019. For more info, please see: http://www.paradigmglobalevents.com/events/orphan-drugs-and-rare-diseases-2019-americas-east-coast/