- Global Pharma News & Resources
19-Nov-2019 - 19-Nov-2019

Accelerating Market Access in Rare Diseases


  1. 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.
  2. How to collaborate with patients and caregivers in rare conditions to generate patient-relevant outcomes.
  3. Leveraging applications of RWE throughout the OMP and ATMP value chain.
  4. To mitigate payer uncertainty by addressing the challenge of early market access with accelerated approvals and fewer clinical data.
  5. 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.
  6. 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.

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