This CIRS workshop brought together companies and agencies (HTA and Regulatory) to discuss the sources of uncertainty that are being built in, by the way medicines development has evolved and how these are being assessed, graded and viewed by both HTA and regulators. The meeting also discussed the key strategies that are being or can be employed to resolve uncertainties or mitigation strategies that are in place to provide confidence to decision makers and patients.

The workshop built on the outcomes of previous CIRS workshops conducted in the UK and the Netherlands between 2017 and 2019, where aspects of identification of regulatory and reimbursement uncertainty were discussed. These meetings identified a need to better understand the types of uncertainty facing different stakeholders so that they can be managed or mitigated, either during development or post approval and how best to meet different stakeholder needs.

It is important to have a clear understanding or map of the main drivers of uncertainty for each group of stakeholders, based on the medicine’s development review and reimbursement pathways. But once this understanding is obtained, the critical issue is development and application of better approaches to manage and mitigate uncertainty through medicines development. This should facilitate decision-making approaches and reduce expensive wasted investment by companies but hopefully enable more promising medicines to be brought to market with a greater consensus on how uncertainty will be managed.

Workshop objectives

  • Identify the source and drivers of clinical, regulatory and HTA uncertainties.
  • Identify what strategies, tools, criteria are utilised to assess and reduce uncertainties within drug development, review and reimbursement.
  • Recommend new approaches to manage uncertainty in regulatory and HTA decision making for innovative medicines and how to these can be managed or mitigated pre- or post- approval.