Box 1. Box 1 How OBA differs from evidence-based medicine

Many readers are no doubt familiar with the application of evidence-based medicine to inform treatment decisions. Although evidence-based medicine leverages strict clinical endpoint data from double-blind, placebo-controlled trials to develop treatment guidelines for physicians, OBA moves beyond these considerations, to encompass a broader range of potential drivers of treatment choice and thus provide guidelines that dictate payer coverage. In addition to clinical measures, OBA incorporates humanistic and economic considerations into the coverage decision-making process. Clinical outcomes are typically hard endpoints (e.g., an event like a stroke or death) or a clinical measurement (e.g., cholesterol level or tumor size). Humanistic measures are softer, including considerations such as improvements in patient symptoms, function and quality of life. Economic considerations include the level of savings accrued from avoiding costly adverse events (e.g., heart attacks or stroke) or costly surgeries and emergency room visits.

What's more, payers also want drug access decisions to be rooted in real-world outcomes, which often vary widely from strictly controlled clinical trials. And unlike previous market paradigms, OBA strips out any marketing-oriented advantages that do not result directly in improved outcomes but for which companies often justify price premiums, such as a new delivery system, a new method of action or combinations of otherwise available drugs. Although these 'advantages' play well in advertising, they do not always result in improvements in efficacy, safety or economics. And in such cases, payers are very likely to resist paying a higher price over the current standard of care (Fig. 2).