Coverage with evidence development for Medicare beneficiaries: challenges and next steps.

Physicians and patients must routinely make important decisions using limited and imperfect evidence. This includes decisions about new drugs and devices, which require a regulatory determination by the US Food and Drug Administration (FDA) that they are safe and effective for their intended use. For drugs, FDA approval is typically based on rigorous randomized clinical trials; for devices, requirements for approval depend on the potential risks and similarity with existing versions of devices on the market. Unsurprisingly, such evidence is often not ideal for “real-world” decision-making. Premarket evidence has limited ability to resolve uncertainty regarding long-term outcomes, effectiveness in different practice settings, and benefits and risks in populations poorly represented in trials. These limitations are particularly important for personalized medicine, where information is needed about the effects of a new drug or device in specific subgroups of patients based on preferences, genomics, and other clinical factors. Payers also must make difficult and consequential decisions with limited evidence. For example, restricting coverage to the narrow populations and indications studied for approval can lead to conflicts with health care providers and patients who believe that coverage restrictions do not reflect all of the current evidence or reasonable beliefs based on that evidence. Alternatively, allowing broader coverage contributes to practice variations and concerns about “unnecessary” health care costs. With either strategy, the path to getting better evidence is unclear. The US health care system should address these dilemmas by supporting the collection of better evidence during routine patient care. Through coverage with evidence development, the Centers for Medicare & Medicaid Services (CMS) and private payers agree to provide broader coverage of promising technologies despite important evidence gaps, provided that the coverage facilitates the collection of additional clinical data. The CMS and other payers have justified the use of coverage with evidence development for various reasons: existing evidence does not generalize to the covered population (eg, seniors) or new settings (eg, communitybased care), evidence is lacking on important longterm outcomes, and evidence suggests that a covered technology may be associated with poor outcomes or safety issues. Through clinical trials and registries, coverage with evidence development has provided evidence relevant to Medicare beneficiaries that otherwise might not have been obtained. For example, in 2006, the CMS granted coverage for intracranial stenting and angioplasty for beneficiaries meeting certain criteria who were enrolled in FDA-approved trials.1 The Stenting and Aggressive Medical Management for Preventing Recurrent Stroke in Intracranial Stenosis trial, which used the Wingspan stent system, was one such trial.2 After early evidence demonstrating significantly greater risk of death or stroke in patients receiving the new procedure, enrollment was stopped. The FDA subsequently issued a safety communication and limited indications for the use of the Wingspan stent system, and the CMS has not expanded its original coverage determination.3 In May 2012, the CMS applied coverage with evidence development when it agreed to cover transcatheter aortic valve replacement in conjunction with data collection through a registry.4 Maintained by medical specialty groups with support from industry, these registries collect clinical data for most Medicare beneficiaries who undergo the procedures under study. Although coverage with evidence development has provided important evidence, there have been challenges. First, the approach has historically been used on a “one-off” basis in conjunction with Medicare’s national coverage decision process. Because the CMS typically issues only 10 to 15 national coverage determinations a year, the approach is used infrequently. Second, the agency has limited time to work out the details. Third, there are no well-defined funding sources for the substantial costs of conducting such studies, and a shared data and research infrastructure is lacking. Thus, each study for coverage with evidence development requires an individual business plan to pay for infrastructure, data collection, and analysis. Fourth, there are no clear and predictable criteria and methods for individual studies. In sum, the costs of applying coverage with evidence development and the potential access problems for Medicare beneficiaries may outweigh the potential benefits that the additional evidence can offer. In November 2011, the CMS solicited public comment about the future of coverage with evidence development. It received many responses, including comments from our center.5 In April 2012, the Obama Administration, as part of the National Bioeconomy Blueprint, stated that the CMS was ready to better define “the parameters and guidance for [coverage with evidence development] so it can be used more widely and effectively as a driver of innovation. ”6 In November 2012, the CMS released revised draft guidance.7 The guidance clarified that coverage with evidence development should be carried out via prospective studies and that arrangements will end when the studies are not approved or VIEWPOINT