Health affairs
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Under Food and Drug Administration (FDA) policy, communications by prescription drug manufacturers must be backed by "substantial evidence" from "adequate and well-controlled investigations." But numerous exceptions permit manufacturer promotion based on data other than randomized trials. The observational research presented in the Hemikrane hypothetical case in this month's Health Affairs is methodologically flawed and also does not meet any of these exceptions. ⋯ Further work is required to define the characteristics of high-quality observational research. However, as this field matures, higher-quality observational studies could meet the legal standard of an "adequate and well-controlled investigation." At that point, the FDA will need to issue formal guidance to minimize confusion on what kinds of observational research can meet its evidentiary standards.
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What role can rigorous observational comparative effectiveness studies play in guiding clinical decision making? What criteria should be used in determining whether the results of such studies should be communicated to clinicians and to patients? We address these questions by considering two hypothetical observational studies in patients with migraine against the backdrop of the review criteria drawn up by the Patient-Centered Outcomes Research Institute (PCORI). These criteria emphasize that patient-centered comparative effectiveness research should exhibit relevance to patients, have great potential to affect practice and improve outcomes, and be conducted using rigorous analytic methods. We conclude that these hypothetical studies would be unlikely to have been funded or communicated by PCORI, and we offer suggestions for improving their relevance and analytic approaches. We also conclude that high-quality observational studies can effectively complement findings from randomized trials, and that communicating their results to patients and clinicians is warranted.
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The Affordable Care Act created the Patient-Centered Outcomes Research Institute (PCORI) to help patients, clinicians, and policy makers make well-informed decisions about health care. Because its funding expires in 2019, the institute has little time in which to produce timely, practice-changing results that will build public support for comparative effectiveness research. ⋯ In January 2012 PCORI's first research agenda described broad research priorities rather than specific clinical questions. The institute must drive the burgeoning discipline of comparative effectiveness research forward, starting with a research project agenda that conveys a sense of urgency and strategic direction.
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Using observation units in hospitals to provide care to certain patients can be more efficient than admitting them to the hospital and can result in shorter lengths-of-stay and lower costs. However, such units are present in only about one-third of US hospitals. ⋯ Using a systematic literature review, national survey data, and a simulation model, we estimated that if hospitals without observation units had them in place, the average cost savings per patient would be $1,572, annual hospital savings would be $4.6 million, and national cost savings would be $3.1 billion. Future policies intended to increase the cost-efficiency of hospital care should include support for observation unit care as an alternative to short-stay inpatient admission.
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The private health plans that administer the Medicare drug benefit use various tools to encourage the use of generic drugs in order to lower total drug spending. Higher generic drug use also appears to encourage consumers to continue taking their medications. This study examines how different drug plan benefit and formulary designs influence the selection of generic drugs to treat high cholesterol among Medicare beneficiaries. ⋯ In this drug class, where generics can be readily substituted for brand-name drugs for most people, adoption of the policies most effective in encouraging generic use could lead to considerable savings for the plans, Medicare, and enrollees. We estimate that every 10 percent increase in the use of generic, rather than brand-name, statins would reduce Medicare costs by about $1 billion annually. Plans could apply the lessons from this analysis and consider a zero copay for use of generic drugs, and Medicare might consider further incentives for plans to use benefit designs that increase such drugs' use.