TAKE-HOME MESSAGES: CDDF Multi-Stakeholder Workshop – The Critical Role of Biomarkers in Delivering Drug Development-Related Precision Oncology

Read the take-home messages from the CDDF multi-stakeholder workshop on The critical role of Biomarkers in delivering drug development-related precision oncology:

 

SESSION 1: SETTING THE SCENE OF BIOMARKERS

Key takeaways:

1. Biomarkers are critical in the diagnosis of cancer, in defining prognosis and in prediction of response and potential toxicities to precision therapies
2. Timely precision genomic testing should be embedded within health systems to provide equal access to targeted treatment option for all patients
3. Biomarker driven research leads to faster, more effective and cost efficient drug development
4. Patients must be at the center of biomarker driven precision oncology

Next steps:

1. Promote patient education on importance of precision oncology and its impact on patient care
2. Promote collaboration between academic, industry and regulatory agencies to generate the data to support implementation of biomarker-driven precision care
3. Derive evidence to implement ctDNA approaches to enhance the cancer treatment pathway

 

SESSION 2: BIOMARKER BASED AUTHORISATION & REIMBURSEMENT

Key takeaways:

1. More data is required to better inform the selection of clinically relevant cut-off points.
2. Precision oncology currently accounts for more than a third of new FDA drug approvals but regulatory approval for CDx poses certain challenges
3. HTA bodies should incorporate the test-treatment combination within cost-effectiveness analyses.
4. In the Netherlands, a tripartite national committee, comprising health professionals, health insurance companies and patient advocates, helps selected appropriate testing and reimbursement.
5. Increased transparency regarding testing, has the potential to diminish practice variation and enhance access.

SESSION 3: THE COST OF NOT USING BIOMARKERS

Key takeaways:

1. Genomic testing rates are lower than anticipated in lung, colorectal and ovarian cancers, diseases for which there is clear data on the benefit of application of targeted therapy
2. Multisided strategies are needed to deliver precision medicine including mechanisms to streamline testing delivery, access and reimbursement.
3. Collaboration of patient advocacy, medical specialists, insurers, politicians and national health authority resulted in reimbursement of whole-genome sequencing for patients with cancer of unknown primary (CUP) in the Netherlands, and a network of CUP clinics to support testing.
4. Increasing access to broad biomarker sequencing panels and repeat testing can help address spatial and temporal heterogeneity across tumors.

SESSION 4: IVDR

Key takeaways: 

1. It is important to consider the real world setting  for Biomarker based treatment:  Multiple tests can be used in clinical care: CE marked assays and in house tests. We need to ensure that IVDs are constantly further developed and that the quality of tests is high.
2. From regulatory perspective the  IVDR adds complexity to clinical trials and to approval of drugs with CDx. We need to reduce complexity and come up with simplification. E.g. introduce a harmonized approval procedure for performance studies
3. In Europe the SmPC will describe the used CDx however the label will not refer to a specific approved test as in the US. This should allow broad access to testing in Europe. Clear labeling guidance and guidance related to the consultation procedure were presented.
4. Notified bodies are deeply involved in discussions with the EMA through pre-submission meetings and the consultation procedure.