Orphan Drug Congress Offers Pharmaceutical Companies Chance to Learn More About Regulatory Changes Affecting Mesothelioma and Rare Drug Development
Mesothelioma, an asbestos-related cancer, is one of the 7,000 rare, or “orphan,” diseases or conditions affecting over 30 million Americans. Later this month, pharmaceutical companies, dedicated to developing treatments for this often neglected population, will have the opportunity to network with other pharmaceutical representatives, patient advocates and government representatives at the 7th Annual Rare Disease and Orphan Drug Leadership Congress.
CBI, a provider of conferences for the pharmaceutical, biotechnology, medical device and healthcare industries, will sponsor its upcoming Leadership Congress on July 18-19 at the Loews Hotel in Philadelphia, PA. This year’s conference focuses on helping attendees gain an understanding of the proposed regulatory changes impacting orphan drug status, and providing key information to pharmaceutical companies regarding development of an orphan drug.
An orphan disease status is assigned to a disease or disorder if it affects fewer than 200,000 Americans at any given time. 3,000 Americans are diagnosed with mesothelioma each year. In addition, nearly one in 10 Americans is diagnosed with a rare disease. According to government statistics, between 85 and 90 percent of orphan diseases are serious or life-threatening, yet only about 200 of them currently have any effective treatments. Mesothelioma symptoms can be managed through chemotherapy and radiation, however, there is no cure for the disease.
The U.S. Food and Drug Administration (FDA) offers financial incentives to pharmaceutical companies willing to innovate for rare diseases. The FDA will cover vital information regarding its incentives and regulations at the Congress. James H. Reese, Ph.D., RAC, Reviewer, EMA Coordinator, Office of Orphan Products Development, FDA, will present the session: “Review Proposed Rulemaking Changes and EMA Harmonization Efforts by the FDA.” Reese will discuss the proposed changes to the Orphan Drug Act and the FDA-EMA collaboration effort.
Sessions for the Congress fall under the following primary categories:
- Understand the Goal and Impact of Policy Initiatives Promising Orphan Drug Advancement
- Understand the Rare Disease Business Model for Portfolio Prioritization
- Pricing, Reimbursement and Distribution Model for Orphan Products
- Collaborate with Foundations for Orphan Drug Development and Manage Access
- Leverage Social Media in the Rare Disease Community for Patient Recruitment and Awareness
To learn more about the conference, or to register, see CBI’s website.