Real-World Evidence in Rare Diseases: Accelerating Market Access

Summary

This unique training focuses on leveraging patient-centric Real-World Evidence (RWE) to accelerate the patient and market access of innovative and potentially transformative Orphan Medicinal Products (OMPs). Delegates will address the challenge of early market access based on fewer and less mature data, with the creation of an Early Value Proposition and a Patient-Centric Evidence Generation Plan that comprehensively support value demonstration, product positioning, and that meet stakeholders’ requirements.

Learning Objectives

• Why are market access opportunities and challenges for OMPs different from other drugs?
• How to collaborate with patients & caregivers in rare conditions to generate patient-meaningful outcomes?
• Leveraging applications of RWE for pricing and access success and throughout the lifecycle
• To mitigate payer uncertainty by addressing the challenge of early market access with accelerated approvals and fewer clinical data
• How to develop a next-generation market access strategy with a life-cycle full spectrum evidence generation plan that maximizes product value from launch to loss of exclusivity?
• To differentiate and succeed in the increasingly competitive rare disease space with a ‘fast to patient’ approach

Key Topics

• Opportunities and Challenges in Orphan Medicinal Products (OMPs)
• Rare Patient Empowerment & Involvement
• Real-World Evidence (RWE) Throughout the Value Chain
• Real-World Case Examples
• Early Value Proposition and Evidence Generation Planning
• Next-Generation OMP Market Access Strategies

Who should attend?

C-level executives, Senior business development strategists, Portfolio executives, Pricing and market access executives, Medical affairs executives, Marketing and commercial executives, Patient advocates, Epidemiologists, Outcomes researchers and health economists, Big data and RWE researchers, Clinical and regulatory executives interested in incorporating value and access early in development

Past participants

• IVS Head, AstraZeneca
• Global Clinical Leader, Bayer
• Head of Market Access & Pricing, Merck Group
• Rare Diseases Lead Adriatic, Pfizer
• Patient Value & Acces Head, Takeda

Our online training experience includes

• Our client zone – a single source for all training materials as well as pre and post-training communication
• Live interactive format via the Zoom platform
• Direct interaction with the trainer
• Q&As, case studies, polls
• Revisit recorded sessions for 30 days
• Digital and LinkedIn certificates

Follow-up Session

This half-day follow-up workshop on October 10th aims to enhance the RWE knowledge gained by participants in the main course with a highly interactive session of practical solutions based on current demonstrator cases in rare conditions (successes and failures). The Follow-up session is only available and suitable for participants, who took the main course.