Pharmacogenomics, evidence, and the role of payers.

Initial enthusiasm for the potential of pharmacogenomics (PGx) to transform medical practice has been tempered by the reality that the process of biomarker discovery, validation, and clinical qualification has been disappointingly slow, with a limited number of PGx tests entering the marketplace since the initial publication of the human genome sequence. Reasons for the delays include the complexity of the underlying science as well as clinical, economic, and organizational barriers to the effective delivery of personalized health care. Nevertheless, payers are interested in using PGx services to ensure that drug use is safer and more effective, particularly in the settings of medications that are widely used, have significant risks of serious adverse events, have poor or highly variable drug response, or are very expensive. However, public and private payers have specific evidence requirements for new health care technologies that must be met prior to obtaining favorable coverage and reimbursement status. These evaluation criteria are frequently more rigorous than the current level of evidence required for regulatory approval of new PGx tests or PGx-related drug labeling. To support payer decision-making, researchers will need to measure the impact of PGx testing on clinical and economic outcomes and demonstrate the net benefit of PGx testing as compared to usual care. By linking payer information needs with the current PGx research agenda, there is the opportunity to develop the data required for informed decision-making. This strategy will increase the likelihood that PGx services will be both reimbursed and used appropriately in clinical practice.