13 December 2016 - Broad range of stakeholder questions addressed.

The EMA has hosted a workshop with stakeholders to discuss adaptive pathways, an approach to medicines development that aims to facilitate access to medicines that address patients’ unmet needs.

The adaptive pathways approach is not a new regulatory route but rather one that makes use of existing legislative and regulatory tools in a more efficient way.

• The adaptive pathways approach is intended only for medicines expected to have a significant clinical impact in patient populations with high unmet needs.
  
• It involves working with the full range of relevant stakeholders from very early in the development process to proactively plan the most appropriate ways of obtaining evidence.
  
• It identifies the appropriate tools to generate that evidence. This may mean making more use of observational (real world) data in addition to randomised controlled trials, especially where these trials are not adequate.
  
• It uses a stepwise approach, permitting approval of medicines in small, tightly defined populations until more data are available.
  
• The standards of regulatory approval remain unchanged.
  

Read EMA press release