Stratified medicine: methods for evaluation of predictive biomarkers

Malottki, Kinga (2017). Stratified medicine: methods for evaluation of predictive biomarkers. University of Birmingham. Ph.D.

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Abstract

Background: Stratified medicine was defined as the use of biomarkers to select patients more likely to respond to a treatment or experience an adverse event.
Alms: To investigate the hypothesis that there is a mismatch between the theoretical proposals and practice of predictive biomarker research, focusing on the clinical utility stage.
Methods: Methodological research was identified in a systematic review of frameworks for staged evaluation of predictive biomarkers. Actual research supporting 50 real cases identified in European Medicines Agency licensing was analysed. A case study of recent research into ERCC l in non-small cell lung cancer was undertaken. Existing discrepancies between the theory and practice were identified and possible reasons and consequences of these were discussed.
Findings: A mismatch between theory and practice was identified. It appeared to be a result of both the practice not following some theoretical requirements, and the underdevelopment of methodology for certain situations. Areas of clinical research with insufficient relevant methodology were identified.
Conclusions: The major research priorities identified in this thesis were development of a clear hierarchy of biomarker research designs and development of methodology related to the biomarker threshold.

Type of Work:

Thesis (Doctorates > Ph.D.)

Award Type:

Doctorates > Ph.D.

Supervisor(s):