Endocrine Abstracts

A range of innovative treatment approaches present ‘difficult development situations’ for drug developers and decision makers including medicines regulators. Treatment concepts where conventional development pathways and evidence standards may be difficult to apply include some cell therapies, gene therapies, personalised treatments, personalised treatment combinations and theranostics.

The recently elaborated concept of adaptive pathways (AP) may be useful to facilitate the development and licensing of treatments that represent difficult development situations. AP is based on stepwise learning under conditions of acknowledged uncertainty, with iterative phases of data gathering and regulatory evaluation. This approach allows approval to align more closely with patient needs for timely access to new technologies and for data to inform medical decisions.

With AP, evidence will be based on a diverse family of data sources and methodologies complementing randomised controlled trials.

This session will discuss difficult development situations, reflect on the associated methodological challenges for evidence generation, and outline the basic concept of AP.