This multi-stakeholder workshop consisted of a series of presentation sessions and three parallel breakout discussions.

Presentations explored trends in regulatory and HTA approvals of orphan products and perspectives on incentives for rare disease treatments, fitness of today’s tools to address uncertainty, the potential or a life cycle approach for evidence generation, the role of multiple stakeholders in reimbursement decision making, the evolution of real world evidence and considerations or the next generation of rare disease treatments.

The breakout groups were asked to examine three topics when considering evidence generation to support financially sustainable access to rare disease products:

  • Incentives from a policy and practice perspective – Do current incentives need to evolve for the development, review and reimbursement of rare diseases?
  • Evidence development for regulators and health technology assessors for rare disease products – How best to address and align the needs of regulators and HTA agencies?
  • Life cycle approach for rare disease products to manage clinical uncertainties due to small patient populations – What are the post-approval considerations for HTA agencies, regulators and payers?

This workshop synopsis summarises key points from the presentation sessions and breakout discussions, including the recommendations put forward to improve regulatory/HTA frameworks for rare disease products.

CIRS April 2023 Workshop report front cover