August 28, 2023
The National Center for Health Research (NCHR) appreciates the opportunity to express our views regarding the Centers for Medicare and Medicaid Services (CMS) Notice on Transitional Coverage for Emerging Technologies (TCET).
NCHR is a nonprofit think tank that conducts, analyzes, and scrutinizes research on a range of health issues, with a particular focus on which prevention strategies and treatments are most effective for which patients and consumers. We do not accept funding from companies that make products that are the subject of our work, so we have no conflicts of interest.
We support the overall goals of the TCET program, but we see an enormous disconnect between the types of evidence that FDA requires for breakthrough devices and the CED standards that CMS requires for coverage. CDRH and the industry it regulates is more focused on getting new devices to market but is not focused on scientific evidence of safety or effectiveness. This is especially true for 510(k) devices, but is unfortunately true for all devices.1 According to the FDA’s own analysis, the agency gave breakthrough designation to nearly 800 devices as of March 2023, with 67 of these devices receiving marketing authorization.2 However, research indicates that most devices that were authorized provided limited clinical evidence, with some completely lacking effectiveness data.3 Of even greater concern, higher recall rates are seen among devices given priority review.4 Innovation should be defined to require that a device is proven to be better, not just newer. We urge CMS to work with industry to make it clear that CMS evidence standards of clinical benefit are very different from the standards required by CDRH. Therefore, we urge CMS to require companies to provide the evidence needed to be worthy of CMS coverage.
It is important to recognize that the approval standards for devices by the FDA does not include proof of safety or effectiveness, but rather the “reasonable assurance of safety and effectiveness.” Often, that “reasonable assurance” is not so reassuring. There is a lack of clinical trial data for the vast majority of medical devices. Even when clinical data are provided to the FDA, many if not most types of Medicare beneficiaries are under-represented. This includes older patients, people of color, and women. Too many device studies have results that are not generalizable to Medicare beneficiaries because of the small number of patients in these groups; this is especially true for people over 65, frail elderly, people with disabilities, and people of color.5 As is often the case, research typically focuses on the youngest, healthiest sick patients, in order to reduce the confounding impact of co-morbidities and maximize the chances that the results will indicate benefits rather than harm. To ensure that medical interventions will benefit Medicare beneficiaries, data need to include enough patients to conduct subgroup analyses to determine if the benefits outweigh the risks for the subgroups of patients that together comprise the majority of Medicare beneficiaries. To be consistent with CMS requirements, manufacturers utilizing the TCET pathway should be required to demonstrate that they will recruit and retain diverse participant populations, across a wide range of patient characteristics.
Another major concern with the TCET program is the lack of transparency. Data collected with Medicare resources funded by the public should be available to the public. According to Part H of the TCET guidance related to the Evidence Preview, there will be no publicly posted tracking sheet and no public notification that an Evidence Preview was completed. However, CMS will share these data with Medicare Administrative Contractors (MACs), as “the development of an Evidence Preview represents a substantial investment of public resources in a thorough evidence review for pre-market devices.” Because of the importance of this program, this information should also be shared with the public.
The TCET program is expected to have significant costs, although the FDA Breakthrough device criteria allows for even more uncertainty than the usual CDRH pathways. These devices may or may not meet FDA’s definition of safety and effectiveness, and so it seems unlikely that they would meet Medicare’s criteria of “reasonable and necessary. While there may be appropriate times for coverage with evidence development for a small number of breakthrough devices, coverage with evidence development shifts costs from the device company to the taxpayers. Instead of device companies paying for clinical trials to show a device is reasonable and necessary for the Medicare population, taxpayers must deal with this substantial financial burden, while patients are exposed to devices that may have no benefit to Medicare patients and could potentially cause irreparable harm. The use of device registries is even more problematic, since private device registries limit public access to data and rarely include a control group to scientifically determine risks or benefits. CMS previously projected that covering the first 14 Breakthrough devices would increase Medicare spending by $2.04 billion between 2022 and 2025.6 This is an extremely high cost for access to products that are not proven to have benefits that outweigh the harms for Medicare beneficiaries.
We want to thank CMS for their effort to improve access to safe and effective medical products, but urge them to hold firm to their standard for coverage based on scientific evidence that all medical products are proven to be reasonable and necessary for Medicare patients.
[1] Zuckerman, D., Brown, P., & Das, A. (2014). Lack of publicly available scientific evidence on the safety and effectiveness of implanted medical devices. JAMA internal medicine, 174(11), 1781–1787. https://doi.org/10.1001/jamainternmed.2014.4193
[2] Orr, E. (2023). FDA Breakthrough Device Program Nears 800 Designations. MedTech Insight. https://medtech.pharmaintelligence.informa.com/MT148092/FDA-Breakthrough-Device-Program-Nears-800-Designations
[3] Johnston, J., Dhruva, S., Ross J., Rathi V. (2020). Early experience with the FDA’s Breakthrough devices program. Nature Biotechnology.;38:933-938. https://www.nature.com/articles/s41587–020–0636–7.
[4] Ong, C., Ly, V., Redberg, R. (2020). Comparison of Priority vs Standard US Food and Drug Administration Premarket Approval Review for High-Risk Medical Devices. JAMA Intern Med. 180(5):801–803. doi:10.1001/jamainternmed.2020.0297
[5] Fox-Rawlings, S. R., Gottschalk, L. B., Doamekpor, L. A., & Zuckerman, D. M. (2018). Diversity in Medical Device Clinical Trials: Do We Know What Works for Which Patients?. The Milbank quarterly, 96(3), 499–529. https://doi.org/10.1111/1468-0009.12344
[6] The Federal Register. (2021). Medicare program; Medicare coverage of innovative technology (MCIT) and definition of “reasonable and necessary”. https://www.federalregister.gov/documents/2021/11/15/202124916/medicare–program–medicare–coverage–of–innovative–technology–mcit–and–definitionof–reasonable–and.