On Wednesday, April 10, 2019, MME and LapidusData will host a workshop on Predicting and Influencing the Market Potential of Orphan Drugs and Gene Therapies as part of the World Orphan Drug Congress.

Market forecasting presents special challenges for orphan drugs and gene therapies. This workshop will provide real-world examples of how epidemiology, pricing, and early clinical decisions can inform your forecast, and more importantly, how you can influence these factors to achieve commercial success. Our unparalleled experience with rare diseases allows us to share cases of winning—and losing—strategies for market sizing, pricing/reimbursement, trial design, and even product formulation. Topics will include:

• Market potential: Defining the treatable population
• Key drivers: Assessing prevalence, diagnosis rates, and disease severity.  What resources can provide reliable information, and how can you generate new data when faced with unknowns?
• Predicting performance: Leveraging historical product analogs, prioritizing multiple indications, and understanding the value of early product formulation
• Market share: How to think about unmet need and competition for orphan drugs
• Price: How to assess and capture the value offered by your product.  What novel reimbursement mechanisms are viable?
• Q&A

The World Orphan Drug Congress will place April 10–12, 2019 at the Gaylord National Harbor Hotel in Oxon Hill, MD.