Definition of biomarkers and efficacy end-points
3. Challenges for Biomarkers in Medicines Development
3.1. Technical Challenges
used in clinical trials must be validated (‘qualified’): is there scientific evidencethat
the biomarker test is accurate, reliable, sensitive and specific enough?
Companies will have to collect all this data for each biomarker they develop.
must be valid measures: For example, if a biomarker is used to predict how
severe a disease may get, is there enough evidence of this ‘predictive
ability’? In other words, has it been shown to predict severity enough times
collection and processing methods must be consistent (e.g. for biopsies or
blood samples). Variations in the way samples are handled can affect the
results of biomarker tests. This could make the results of a trial invalid.
- The IT systems used for data management and data analysis must be robust and fast to cope with the amount of data generated. All biomarker measurements must be correctly linked with individual patients.
- When a companion diagnostic is needed to prescribe a new medicine , a new ‘platform’ or kit for testing patients in the clinic may need to be developed. The test kit will usually need to be ready for use during the large confirmatory trials of the medicine (Phase III). It is not ready until it has been validated and the validation results show that the diagnostic is accurate and clinically useful.