In part 1 of this series, we considered the complementary but distinct regulatory standards, objectives, and evidentiary requirements for medical products currently undergoing evaluation by the Food & Drug Administration (FDA) and the Centers for Medicare & Medicaid Services (CMS). The recent National Coverage Determination (NCD) on monoclonal antibodies for Alzheimer’s Disease (AD) serves as an example of how the 2 agencies use their discretion in different ways to make decisions jurisdiction. To appreciate why this discretion may differ, it is useful to understand the distinctions in each agency’s charge, borne out of the needs of the American public during unique periods of history (see Table 1).

cGMP: Current Good Manufacturing Practice; CMS: Centers for Medicare and Medicaid Services; FDA: Food and Drug Administration; FFDA: Federal Food and Drugs Act; FFDCA: Federal Food, Drug, and Cosmetic Act; PDUFA: Prescription Drug User Fee Act; MCCA: Medicare Catastrophic Coverage Act; MMA: The Medicare Prescription Drug Improvement and Modernization Act SSA: Social Security Act;​

Each agency’s jurisdiction and programs have adapted to meet the needs of the US healthcare system over time. Despite the bureaucratic proximity that we see today, opportunities for true collaboration between FDA and CMS have been both rare and relatively recent. Meaningful collaboration between the agencies has the potential to maximize value and expedite patient access to safe and effective medical products.

What Has Past Collaboration Looked Like?

CMS and FDA have worked together in the past, particularly as it relates to the applicability of innovative medical device technologies falling within CMS’s defined benefit categories. Thus far, these attempts at meaningful collaboration have yet to instill long-lasting cultural shifts to improve the regulatory and coverage approval processes.

In 2011, the agencies shared a pilot parallel review program for FDA-designated breakthrough medical devices. In this program, manufacturers could request a simultaneous FDA review for regulatory approval and CMS review for coverage in order to decrease time to patient access for these devices. Even after the pilot was officially adopted in 2016, the program is only designed to accept a maximum of 5 candidates per year.

In January 2021, CMS finalized the set to take effect in December 2021. The rule would have:

• Granted expedited Medicare coverage for certain FDA-designated breakthrough devices for up to 4 years
• Circumvented CMS’s NCD with Coverage with Evidence Development (CED) paradigm for products that may have unconfirmed benefit in a Medicare population

In September 2021, before the rule could take effect, CMS repealed the MCIT-RN rule.. CMS cited safety concerns and lack of evidence generation specific to its Medicare population as a direct rationale for withdrawal of the rule.

The 2022 NCD with CED for AD has led to criticism from various stakeholders, including members of Congress, for its implications to patient access. This is the first time CMS has proposed a CED requirement on an entire class of therapeutics. Despite FDA approval, this restrictive coverage determination has received criticism and raised concern for a lack of harmony between the agencies’ decisions. Key to this layered decision making is the jurisdictional discretion and legislative mandates which guide each agency, which are discussed in part I of this series.

What Are the Implications for Future Collaboration?

Given the FDA and CMS responsibilities in defining access to medical products, strong support exists for establishing meaningful collaboration that does not interfere with each agency’s mission. For instance, the November 2021 Cures 2.0 draft bill, which builds on the legacy of 2016’s successful Cures Act, proposes a legislative approach to clarifying the communication between FDA and CMS. This text proposes heightened communication specifically for products that have tailored data sets and may use surrogate endpoints or have small clinical trial populations. Though brief, the Cures 2.0 Sec. 305 language would codify heightened communication with CMS regarding approval and coverage decisions for products receiving FDA breakthrough, fast track, or accelerated approval designations. While statutory clarifications to the accelerated approval pathway may occur with FDA-focused legislation, such as in the user fee package, the clarification on how to perceive evidence for the purpose of clinical benefit to Medicare patients will require a broader scope of legislative intent, like that seen in Cures 2.0.

Avalere has performed extensive work in both coverage strategy and early evidence development planning that intersects with FDA and CMS’s responsibilities. A host of policy, FDA regulatory, value, and evidence experts continue to discuss specific impacts that CMS and FDA decisions have on patient access.

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