In a new Op Ed online at NEJM, Neumann and Tunis ask if CMS can be an effective Health Technology Assessment organization (HTA). For example, the agency's "TCET" program for new technologies proposes to rely heavily on evidence evaluation and CED (coverage with evidence development.)
Neumann and Tunis note that there are several billion dollars allocated for implementation of IRA - drug price negotiation in Part B &D - and some of that may play out as HTA-like activities. If, like me, you haven't been closely following IRA drug news, here's a long report from Latham & Watkins.
Find the HTA essay here:
The article doesn't have an abstract, but here's a ChatGPT summary of it.
CMS and Health Technology Assessment: An In-depth Analysis
The Centers for Medicare and Medicaid Services (CMS), originating in the 1960s, started as the Health Care Financing Administration (HCFA) primarily to pay claims for public insurance. Congress intended HCFA to fund private medical facilities and professionals without intervening in medical practices to avoid suspicions of “socialized medicine.” However, a lingering challenge was to ensure the efficacy of prescribed and financed services and products.
While the Food and Drug Administration (FDA) endorsed the safety and efficacy of drugs and high-risk medical equipment, questions persisted about their application to Medicare patients, often older and sicker than those in FDA's clinical trials. Such trials often underrepresented women and non-White participants. Generally, CMS funded “accepted” medical procedures. Although authorized to reject funding for non-essential items or services, CMS seldom conducted its evaluations. Only controversial or expensive technologies were occasionally assessed against the “reasonable and necessary” standard. Moreover, CMS outsourced various local coverage decisions to regional Medicare Administrative Contractors.
Recognizing its broader mandate, HCFA rebranded as CMS in 2001. [*] The agency emphasized the need for evidence-based evaluations of new health technologies, seeking to ensure they beneficially impacted Medicare beneficiaries. Consequently, the “coverage with evidence development” (CED) option emerged, allowing payment for potential technologies while necessitating further studies to reinforce evidence validity.
Recent developments underscored the need for CMS to intensify its health technology assessment (HTA) role. In 2016, the FDA initiated breakthrough status for critical illness devices based on preliminary evidence, creating a dilemma for Medicare over their coverage amid substantial uncertainties. In 2021, the Trump administration proposed extending coverage for such breakthrough devices post-FDA approval. However, the Biden administration, apprehensive about potential inefficacies and risks, repealed this regulation. The subsequent proposal was geared towards engaging CMS more deeply in setting evidence-based standards for medical device coverage.
Another complication is the escalating number of high-cost drugs, prompting inquiries about Medicare's evaluation mechanisms. For instance, the potential expansion of Part D coverage for obesity medications, which could strain the program financially, demands meticulous assessment. Additionally, new cell and gene therapies, often lacking long-term data, pose a challenge. The debate surrounding Alzheimer’s drugs epitomizes the intricacies of evaluating new treatments that obtain FDA's accelerated approval. In 2022, CMS adopted a unique stance for Alzheimer’s treatments, endorsing coverage only within the context of randomized clinical trials. The episode revealed occasional disparities between Medicare’s and the FDA’s criteria, sometimes warranting CMS's independent evaluations.
The 2022 Inflation Reduction Act, which obligates Medicare to negotiate drug prices for selected therapies, further magnifies CMS's role in HTA. Mandating evaluations of a drug’s benefits, risks, and comparative effectiveness, this act allocates $3 billion over a decade for its implementation. With such substantial funding, CMS is poised to become a prominent HTA entity globally.
Nevertheless, challenges abound for CMS. The political quagmire concerning government intervention in medical practice, potential obstructions to innovation, and health care “rationing” concerns persist. Notably, though, CMS's evolution into a dominant HTA entity reflects the absence of such an organization at the federal level, given historical opposition. A few recommendations could bolster CMS’s mission:
- Resource Enhancement: Congress should allocate resources to CMS for recruiting experts in clinical research, trial design, epidemiology, and biostatistics.
- FDA Collaboration: CMS and the FDA must enhance their coordination, focusing on early discussions, manufacturer engagements, and addressing disparities between their respective requirements. While synchronization might complicate product approval and coverage, lessons from the FDA's extensive experience across medical domains can significantly benefit CMS.
- Strengthening CED: Although the existing CED framework is lengthy and often doesn’t lead to significant coverage policy modifications, leveraging advancements in databases and analytical techniques can significantly refine it.
In conclusion, as a responsible payer, Medicare's scrutiny of technology efficacy for its beneficiaries is prudent. Recent developments thrust Medicare into the HTA limelight, necessitating that it fully embrace this role and amplify its capabilities to align with these evolving responsibilities.
If you like topics like the rebranding of HCFA as CMS, see a fine 2015 essay on that kind of history, from Kinney, entry point here.