Director of Federal Affairs
National Committee for Quality Assurance
Thank you for the opportunity to comment on coverage with evidence development (CED). CED provides conditional payment while generating data on the effectiveness of promising treatments. It can be a practical way to obtain evidence and speed collection of much-needed data on risks and benefits in specific population groups. CED also can advance development of valid and reliable quality measures, which requires a strong evidence base that all too often is lacking.
NCQA is particularly interested in CED’s potential to develop more evidence on what works or does not. In developing quality measures, one of our biggest constraints is the lack of good evidence – both for services that are underused and overused. We also need more evidence to support quality analysis across broad populations, which CED could help provide.
However, there are questions about statutory authority and resources for CED, and no clear criteria for when and how to appropriately conduct CED. That has contributed to inconsistent policies and results, as well as inadequate protections against conflicts of interest.
Some CED initiatives, like the original lung volume reduction surgery for emphysema initiative, yielded important results and valuable lessons for criteria going forward.
• Beneficiary Focus. It evaluated a new treatment that held promise for a life-threatening condition with no other good treatment options for which rapid evidence development was literally a matter of life and death.
• Strong Study Design. Medicare covered the treatment only in a randomized trial at centers that agreed to use a strict research protocol.
• Objective Outside Expertise. It included partnership with the National Institutes of Health, which has substantial expertise in conducting clinical trials.
The finding of minimal benefit and increased risk promptly stopped use of the procedure and protected desperate and vulnerable beneficiaries from a treatment that did more harm than good.
In subsequent initiatives, CMS has sometimes applied less rigorous criteria and consequently has obtained less clear benefits. There have been concerns about the role of providers or manufacturers with financial interests at stake in some initiatives.
There also has been use of observational studies that produce less definitive results.
We therefore offer the following suggestions for a more coherent, consistent and reliable approach to CED efforts.
Establish Clear, Beneficiary-Focused Criteria. Build consensus with stakeholders, especially beneficiary advocates, on clear, transparent criteria and thresholds for CED. They should:
• Stipulate that beneficiary need, not provider or manufacturer interests, drives CED.
• Give priority to public health, unmet medical needs, and treatment in populations excluded in earlier trials like the elderly or other specific groups.
• Consider if therapies expose beneficiaries to excess risk of harm or insufficient benefit.
• Focus on the need for rapid evidence development on risks and benefits of the treatment in question because of its potential clinical importance, and before it is widely diffused.
• Not be influenced by arguments that other covered treatments also lack optimal evidence.
Require Strong Study Design. Develop a clear, transparent process for independent expert review to ensure CED produces valid data to support informed decisions. This should:
• Require use of well-designed randomized, controlled trials whenever feasible, and clear, transparent justifications for use of less rigorous methodologies.
• Ensure studies provide near-term answers to specific, clearly defined questions that will inform coverage determinations within specific time frames.
• Ensure testing in a variety of settings where beneficiaries get care.
• Include patient-reported outcomes such as on pain and quality of life.
Partner with Appropriate Experts. Include strong partnerships that build on the expertise and resources of independent, non-governmental entities like the Center for Medical Technology Policy.
• Include disease-based advocacy groups and research foundations.
• Do not have manufacturers or provider groups with a financial stake as sole or primary partners. Establish strong conflict-of-interest policies and an arms-length relationship in any partnerships with them. Balance their participation with more objective entities.
• Consult with the new Patient-Centered Outcomes Research Institute to determine when and where their work and CED can align and inform each other.
• Use the Medicare Coverage Advisory Committee, or another body with substantial beneficiary involvement and research expertise under Federal Advisory Committee Act procedures that ensures transparency, to recommend when CED is appropriate.
• Share CED results broadly with consumer groups, other insurers, and developers of shared decision-making tools and other patient decision aids.
Statutory Authority. Work with Congress, beneficiary advocates and other stakeholders to establish explicit statutory authority and resources for beneficiary-focused CED initiatives.
Thank you again for the opportunity to comment. Please contact our Vice President of Public Policy and Communications, Sarah Thomas, at Thomas@ncqa.org or at 202-955-1705 with any questions.
Margaret E. O’Kane