Scientific advances in biotechnology are ushering in a new era of medicine: targeted therapies and personalised medicine are coming of age. Traditional medicine manages a disease through standard treatments, without taking account of existing variability between individuals or groups of population. Personalised medicine, in contrast, adapts treatments to the profile of each individual. This revolution in biomedicine is due, in large part, to the discovery of biomarkers.
A biomarker is a biological indicator which can be used to monitor the presence or absence of a disease; the progression of a disease; the effect of a treatment; and the toxicity of a drug. Biomarkers are important because they will help deliver safer and more effective drugs to patients and they will be a part of the solution to the pharmaceutical sector’s R&D productivity problems.
At a workshop organised by the OECD Biotechnology Division in October 2008 in the UK (click here for agenda, presentations and documentation), experts discussed how to improve the development and use of biomarkers in health care. Topics included:
Workshop participants made several policy recommendations. First, they emphasised that the policy environment for biomarker development in precompetitive and proof of concept R&D phases functions well. However there are significant problems surrounding the clinical evaluation of biomarkers which impact both the clinical phases of development and biomarker use in clinical practice.
Second, experts agreed there is a need to create an evidence base for the clinical evaluation of diagnostic biomarkers. The paucity of relevant data, knowledge and studies needed for evaluation has multiple sources (e.g. a lack of biomarker data sharing, a lack of investment in larger scale clinical evaluation studies, and the difficulty of accessing health outcomes information). However, the costs of building the evidence base and of evaluating and disseminating results will have to be shared by industry and government. There is little consensus as yet on how to do so.
Third, experts noted significant business challenges to bringing diagnostic biomarkers to market . Diagnostics are considered low value added by health care payers in comparison with therapeutics. Moreover, evaluating the health and economic benefits of novel diagnostic tests has proven difficult. Firms are thus unwilling to invest heavily in the development of diagnostic biomarkers unless they are associated with the prescription of a particular drug which captures value for the test, as is the case in pharmacogenomics or theranostics. If there is a push toward requiring the generation of data for the clinical evaluation of diagnostic tests, the experts predict that will entail a shift the diagnostic business model towards one closer to that of therapeutics: fewer high quality products make it to market but will need to be reimbursed at rates that better reflect their value in health care.
Looking forward, experts noted that the linkage of multiple markers through complex algorithms will in the near future deliver individual risk profiles as either predictive and diagnostic health data. Such tools will be even harder to evaluate than the present generation of molecular diagnostic tests and will severely challenge health practitioners, patients and government policy, which calls into question the future of personalized healthcare. Education and training of clinicians as well as better point of care information will be required. They also noted that direct to consumer diagnostic tests and services were on the rise and that there was no consensus internationally on this issue.
Finally, workshop participants called for better dialogue amongst stakeholders. Governments have a role to play in creating a platform for dialogue in order to build consensus around the method of evaluation and ultimately the value placed on biomarkers.
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Last updated 29 November 2010.