CMS Proposes Guidance on Use of Real World Data for Coverage with Evidence Development

Background

Medicare generally only covers or items that are “reasonable and necessary” for the diagnosis or treatment of illness or injury or to improve the functioning of a malformed body member. When CMS issues an NCD establishing whether an item or service is covered by Medicare, it must evaluate clinical, medical, technical, and scientific evidence to determine if the item or service satisfies this reasonable and necessary standard.

Certain NCDs only establish coverage for items or services under the “CED” framework. Under CED, Medicare will cover a product only when it is furnished in the context of a CMS approved clinical study or with the CMS approved collection of additional clinical data. CMS uses CED in situations where it does not believe there is sufficient evidence to warrant coverage under the ordinary reasonable and necessary standard, but the agency believes that limited coverage is still warranted in the context of qualifying clinical studies and/or data collection intended to develop additional evidence to support broader coverage. Accordingly, the CED framework allows time for additional evidence development while providing some degree of Medicare coverage. CED is also a key part of the newly established TCET pathway, which CMS developed to facilitate “more timely and predictable” access to certain FDA-designated Breakthrough Devices through expedited Medicare coverage.

While developing the CED framework and TCET pathway, CMS indicated it would allow a range of fit-for-purpose studies as part of permissible clinical evidence in support of coverage and planned to release additional guidance as to the use of RWD.1 In releasing this proposed guidance document, CMS acknowledged that past RWD research protocols varied significantly “in terms of the level of detail and content provided,” slowing down the study approval process. As such, CMS states that the purpose of its proposed RWD guidance is to provide clear and specific guidance to the public on the critical elements of a study protocol that relies on RWD sources for an NCD using the CED paradigm, including TCET and parallel review.

Proposed guidance

The proposed guidance document primarily consists of a proposed template to be used when submitting RWD study protocols to CMS, along with CMS’s rationale for each element of the template and certain sample materials. While CMS does not propose to require use of the template, CMS strongly encourages its use and notes that any departure from the template may lead to a delay in RWD study design approval. Notably absent from the proposed guidance is the interplay of what CMS proposes and the requirements specified in a particular CED NCD for approval of a clinical study or data collection.

The proposed template, “HARPER+,” is based on the existing HARPER but has been revised by CMS to better fit medical devices and Medicare coverage criteria, and includes new sections for study sponsors to provide information specific to CED study standards. CMS notes that it has cross-referenced HARPER+ with the FDA’s guidance for the use of RWE to support regulatory decision-making for medical devices and with the National Evaluation System for Health Technology Coordinating Center’s Research Methods Framework.

Some notable aspects of the proposed template, related to CED, TCET, parallel review, and unique challenges of using RWD include the following proposed elements:

• Information about data sources: CMS states that data credibility is higher when study sponsors can provide published citations demonstrating that “the data source has been successfully used in a similar context.” CMS believes that information about the data source aids it in assessing whether the RWD is fit-for-purpose to answer the research question posed by the study.
• Context and Rationale for Data Sources: CMS notes that there are a range of data types that can be used in RWD studies, such as electronic health records, insurance claims, pharmacy benefit claims, and patient registries. CMS states that understanding why researchers recommend and choose specific data sources is key to “success of the research proposal.”
• Quality Control: CMS requests that study sponsors state the steps taken to ensure data quality, including quality assurance and quality check procedures, double programming, source data verification, validation of endpoints, data transformation and linkages, and assessment of the data’s reliability.
• Applicable Federal Regulations: CMS requests that study sponsors confirm compliance with CED-specific requirements, along with other applicable federal privacy and patient protection regulations.

Authored by Beth Roberts, Stuart Langbein, Beth Halpern, Brian Carey, James Huang, Victoria Wallace, Erik Schulwolf, and Rianna Modi.