Accelerating Commercialization of Rare Disease Treatments

In Market Access, Orphan Drug Congress by Paul Gilbertson

On 15 November Mapi will be running a workshop titled “Overcoming Challenges in Rare Disease Health Research & Orphan Drug Program Commercialization

Methodologies in developing Real World Data: Engaging stakeholders in developing evidence strategies

  • How is expanded access used to provide treatment for patients with rare diseases?
  • Compassionate Use Programs in the window between submission and commercialization
  • What are the benefits of conducting clinical studies and registries for rare diseases?
  • What are the types of studies conducted during different phases of orphan drug development?
  • How are patient registries used to generate real-world data for rare diseases and orphan drugs?
  • Ethical Patient Warehousing – working with Advocacy to identify populations in advance of research
  • What are the unique challenges of conducting rare disease/orphan drug registries?
  • How can proactive study management strategies successfully address these challenges?

Market Access Challenges for Rare Diseases: Recent Developments in HTA and Reimbursement Decision-Making Considerations for Orphan Drugs in Europe

  • Opportunities for early engagement with stakeholders, including clinical and economic evidence generation advice
  • The current reimbursement environment in Europe
  • Country-specific orphan drug programs and information requirements
  • Existing challenges to reimbursement
  • Demonstrating cost-effectiveness/budget impact
  • Innovative schemes for reimbursement of orphan drugs

Global regulatory strategies: The emerging global Regulatory Pathway for Rare Disease treatments

  • Regulatory strategies for successful orphan drug R&D
  • Have regulations increased or shifted making designation or approval more difficult?
  • Perspectives form Europe and US priority review vouchers

Further details and registration can be seen here

Mapi is the market leader in all the disciplines needed to commercialize Orphan Drugs and manage Rare Disease programs