This workshop was a follow-on from a collaborative forum held in Utrecht in 2018 entitled What new research can enable a joint approach by regulatory and HTA agencies to manage uncertainties for products using early access pathways?

Workshop objectives

  • Discuss the types of uncertainties that can be identified and, in theory, resolved during clinical development as well as those for which data will be unavailable for early-access medicines
  • Identify potentially resolvable uncertainties as perceived by companies, HTA bodies, payers, patients and regulatory agencies and how to better manage these for early-access medicines
  • Recommend appropriate approaches that build management or resolution of regulatory or HTA uncertainties for early-access medicines into the development space