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# FDA/CMS Heads Discuss Changing Evidence Standards
- URL: https://www.fdaweb.com/fda-cms-heads-discuss-changing-evidence-standards/
- Published: 2016-11-07T12:00:00.000Z
- Updated: 2026-09-14T21:45:38.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5137276

FDA commissioner **Robert Califf** and CMS administrator **Andrew Slavitt** offer their perspectives on the two agencies’ changing approaches and evidence standards for admitting products to the market and Medicare paying for them. Writing in a new *JAMA* [viewpoint article](http://jamanetwork.com/journals/jama/fullarticle/2580955?utm%5Fsource=twitter&utm%5Fcampaign=content-shareicons&utm%5Fcontent=article%5Fengagement&utm%5Fmedium=social&utm%5Fterm=110716#.WCCsfsxOBL4.twitter), the two say that “changes in the organization of health care and in the larger information ecosystem should allow the FDA and CMS to move increasingly toward use of shared sources of evidence while still applying the most appropriate criteria to their decision making. Such a move should help reduce current gaps in evidence that create uncertainty surrounding the approval or clearance of new therapies and their subsequent use in practice. It should also enable greater efficiency in medical product development and provide the higher-quality evidence needed in the emerging era of precision medicine.”

When a product demonstrates promise in early clinical research, “developers should rapidly pivot toward evaluating that product in the clinical contexts for which it is intended,” Califf and Slavitt write. “In other words, the United States needs to develop high-quality evidence about the risks and benefits of tangible health outcomes in clinical settings and among patients representative of those who will actually use these products. This expansion of scope earlier in the product life cycle will help establish how a given product is likely to perform once it is marketed and how best to administer that product. Early involvement of health systems and payers will also help to understand and address the kinds of evidence needed to incorporate the new product into practice, place it on formulary or device inventory, and decide whether and how much to pay for its use, thereby facilitating implementation and adoption. Notably, the European Medicines Agency has recently come to the same conclusion in concert with its health technology assessment organizations.”

The regulators say that it is important to bridge the gap between evidence that is generated through trials performed primarily to obtain marketing approval for drugs and high-risk devices and the optimal kinds of evidence needed to support the intended uses of these products in practice. Therefore, to ensure that patients, clinicians, and payers have adequate evidence to guide their choices, FDA and CMS are focusing on the following approaches:

· The agencies are clarifying the need for including diverse populations and measuring relevant clinical outcomes within the “sphere of trials” conducted for regulatory approval and to inform labeling. This effort is particularly important with accelerated approvals based on biomarkers because the current regulatory framework requires postmarket trials to evaluate the effect of the therapy on the clinical outcome of interest, and such approvals are expected to increase with rapid progress in molecular medicine.

· FDA and CMS are collaborating with other federal agencies to build functional links across a range of systems developed to capitalize on existing digital information collected in the course of health care delivery, including electronic health records, insurance claims, and clinical registry data. These approaches should significantly improve the quality and reduce the cost of evidence generation and enable the inclusion of much more representative populations into clinical trials and other studies. This in turn will provide the evidence needed to better characterize the balance of risk and benefit and establish the appropriate use and value of therapies in clinical practice.

Califf and Slavitt believe that as electronic health records, claims databases, and clinical data quality registries continue to advance and become accessible by multiple stakeholders, a “tipping point” will occur that will enable more inclusive trials to be done on a wider scale at much lower cost than heretofore. This will allow FDA and CMS to measure health outcomes that are important to patients and health care consumers and define the appropriate use of medical products in a more straightforward, evidence-based manner. “As the United States enters the era of technology-enabled precision medicine, regulators and payers have a clear interest in and responsibility for ensuring that we have the evidence needed to support such approaches,” they write.