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Waiting for traditional randomized controlled trials may delay the implementation of pharmacogenomic testing, necessitating alternative study designs and real-world evidence to demonstrate clinical utility.
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This editorial argues for a paradigm shift in evidence requirements for pharmacogenomic testing, suggesting that real-world studies and comparative effectiveness research may be more practical than waiting for large randomized controlled trials.
Copyright: © 2012 Francis Lam YW. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. The levels and strength of evidence expected for demonstration of clinical utility (defined as ability to predict clinical outcome and the added value over standard care) of pharmacogenomics-guided drug therapy continue to be a significant barrier for implementation of pharmacogenomic testing in clinical practice. Given the scope of the current healthcare expenditure and evidence-based driven clinical environment, any clinical trial to validate clinical utility is expected to not only be hypothesis-driven but also extensive in terms of time and sample size. As an example, the ongoing multi-site, randomized controlled Pharmacogenomics of Anti-platelet Intervention-2 (clinicaltrials.gov NCT01452152) trial compares pharmacogenomicguided antiplatelet therapy versus standard care on cardiovascular events in 7,200 patients undergoing percutaneous coronary intervention, but the results likely will not be available until 2015. In the meantime, clinicians continue to debate and disagree on the quantity and quality of data that are scientifically appropriate but at the same time realistically achievable, [1,2] and based on the lack of consensus, payers continue to decline reimbursement for pharmacogenomic biomarker tests, [3] even though at times the tests are much less expensive than the drugs that are paid for.
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Y. W. Francis Lam (2012) conducted an editorial in Pharmacogenomic testing. Pharmacogenomic testing vs. Standard care was evaluated. Waiting for traditional randomized controlled trials may delay the implementation of pharmacogenomic testing, necessitating alternative study designs and real-world evidence to demonstrate clinical utility.
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