The progress in molecular biology, coupled with progress on pharmaceutical aspects, has enabled an increasing development of effective medicines, for a (sometimes highly) selected patient population with tumors defined based on so-called biomarkers. Commonly these are molecular features of a malignant disease that can be identified with an appropriate test.
The downside of this, is that a patient populations become increasingly fragmented, ultimately turning every malignant disease into a rare disease.
This creates challenges for drug development in general, and for the trials designed to obtain marketing approval in small populations specifically, and for the debate on drug-related use, -costs and -reimbursement after marketing approval.
The workshop will offer a neutral and collaborative platform for evaluation and discussion of innovation in these areas with active participants from all relevant stakeholders. Informative lectures and panel discussions open to all attendees will alternate.
The main objective of the workshop will be to formulate how biomarker use could best be implemented in drug development based on available insights from all relevant stakeholders.
CDDF intends to publish the outcome.
Learning outcomes / Meeting objective
- How to define a biomarker, and related populations within the health systems
- To align the conditions for the involved biomarker assessment in the EU
- Regulatory requirements for marketing authorizations
- Regulatory requirements for reimbursement approval after marketing authorization
The target is a multidisciplinary audience of academia representatives, EU and US regulatory bodies (EMA, FDA, National Agencies), the pharmaceutical industry, HTAs, and patient advocates.