Accelerating Market Access in Rare Diseases


08 October 2020


Registration Deadline


  • Why market access challenges for OMPs and ATMPs, including autologous and allogeneic gene therapies, tissue engineered products and somatic cell therapies, are different from other drugs.
  • How to collaborate with patients and caregivers in rare conditions to generate patient-relevant outcomes.
  • Leveraging applications of RWE throughout the OMP and ATMP value chain.
  • 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 full-spectrum lifecycle evidence generation plan that maximises 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.

Additional Benefits

  • You will receive an up-to-date collection of demonstrator cases and literature focused on rare patient engagement, PROs, patient-centric RWE applications in rare conditions, value demonstration and market access.
  • You will be able to share your unique OMP- or ATMP-related challenges and to discuss practical solutions with the expert and delegates.