Accelerating Commercialization of Rare Disease Treatments

Discussion Points

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

This Patient-Centered Research Workshop was presented on November 15 2016, at the World Oprhan Drug Congress in Brussels. Mapi and industry experts including William Maier, Patricia Anderson, and Céline Taveau shared their experience and expertise with attendees from across the industry. We would love to present this workshop for your team or at your tradeshow. To find out more, contact Mapi at

Download Summary PDF

To download a PDF summary of this workshop from a recent event, please complete the following form.

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

Real-World Evidence: Industry leaders in real world research to support regulatory requirements, commercialization evidence, and engage treatment populations
Patient Insights: Services to increase recruitment and retention, and ensure the patient’s voice is heard throughout the lifecycle of drug discovery, from protocol development to post marketing
Direct-To-Patient: A dedicated global team and call center enabling patient enrollment, retention, and support throughout the study
Patient-Reported Outcomes: World-renowned leadership with access to over 4,000 PRO measures and expertise in custom scale development
Language Services: Recognized leaders in PRO validation, patient support, and HCP materials
Strategic Regulatory Services: Offering consultation and experienced procurators for regulatory meetings
Pharmacovigilance & Risk Management: Experts in international pharmacovigilance and safety, with strategically placed Qualified PV contacts
Real World Strategy & Analytics: Global Health Economics team with expertise in Rare Disease research, including Meta Analysis, Cost Consequent Analysis, and Market Access to support payers and health technology assessments
Patient Advocacy: Deep relationships with both patient advocacy and patient community groups