Weekly Spotlight
Edwards EVOQUE Receives FDA Approval as First Transcatheter Therapy for Treatment of Tricuspid Regurgitation
Edwards Lifesciences Corporation (NYSE: EW) today announced the company’s EVOQUE tricuspid valve replacement system is the first transcatheter therapy to receive U.S. Food and Drug Administration (FDA) approval for the treatment of tricuspid regurgitation (TR). The EVOQUE system is indicated for the improvement of health status in patients with symptomatic severe TR despite optimal medical therapy (OMT), for whom tricuspid valve replacement is deemed appropriate by a heart team.
“Edwards has a long history of leading innovation and pioneering new therapies to address the unmet needs of patients with structural heart disease,” said Daveen Chopra, Edwards’ corporate vice president, transcatheter mitral and tricuspid therapies. “We are grateful for the strong collaboration with clinicians all over the world who contributed to the EVOQUE system now being available through FDA’s Breakthrough Pathway to provide a treatment option to the many patients in the US suffering with tricuspid valve disease.”
The EVOQUE system is comprised of a nitinol self-expanding frame, intra-annular sealing skirt and tissue leaflets made from the company’s proven bovine pericardial tissue. The EVOQUE valve will be available in three sizes, all delivered through the same low-profile transfemoral 28F system.
“Patients suffering with tricuspid regurgitation endure life-impairing symptoms and, until today, had no approved transcatheter treatment options,” said Susheel Kodali, MD, director, Structural Heart and Valve Center at Columbia University Irving Medical Center/New York-Presbyterian Hospital and TRISCEND II Study Principal Investigator. “The EVOQUE system is able to replace the native tricuspid valve, virtually eliminating tricuspid regurgitation in a wide range of patients. We see significant improvements in patients’ symptoms and quality-of-life, including not feeling short of breath and being able to care for themselves, which ranked highest on a patient preference survey conducted at baseline with TRISCEND II pivotal trial patients.”
Successful six-month results from the randomized controlled pivotal trial, TRISCEND II, were presented at TCT 2023 and reported favorable safety and effectiveness outcomes, demonstrating superiority to OMT alone and meeting all primary endpoints. Key findings in the trial included significant reduction or elimination of tricuspid regurgitation and significant and sustained quality of life improvement, while demonstrating a favorable balance between risk and benefit.
In addition to the six-month cohort, 318 of the total 392 randomized patients completed a 1-year visit. The results showed favorable trends in the device group compared to the control group in the primary composite endpoints, including all-cause mortality, tricuspid intervention, heart failure hospitalization, KCCQ, NYHA and 6MWD. Edwards expects to present the full cohort of 392 TRISCEND II pivotal trial patients at TCT 2024.
The EVOQUE system received CE Mark approval in October 2023, making it the world’s first transcatheter valve replacement therapy to receive regulatory approval to treat TR.
All Three Branches of Government Set Their Sights on Drug Prices
As three drug companies prepare to testify before Sen. Bernie Sanders (D-VT) and the Senate Health, Education, Labor, and Pensions (HELP) Committee, the Centers for Medicare and Medicaid Services (CMS) launched the opening salvo in the first ever Medicare drug price negotiations and prepared to defend the negotiation process in the first of a series of lawsuits from drug industry complainants.
- Thursday, February 8th: Johnson & Johnson CEO Joaquin Duato and Merck CEO Robert Davis will join Bristol Myers Squibb CEO Chris Boerner at a Senate HELP Committee hearing on prescription drug prices in the United States.
- On Wednesday, January 31, AstraZenica kicked off the first of a slew of legal actions by the drug industry seeking to stop Medicare drug price negotiations. Opening day in a Delaware federal district court came just one day before CMS was set to proffer its initial offer of a “maximum fair price” on the 10 drugs selected for negotiations in 2024.
- Also in a Delaware federal district court this week, the Department of Justice rebuked a claim by Novo Nordisk that CMS exceeded its mandate to negotiate prices on 10 Part D Drugs this year by including six separate diabetes treatments as one drug in their negotiations.
Biogen Announces It Will Pull Alzheimer’s Disease Drug, Aduhelm, From the Market
In a statement published January 31, 2024, Biogen announced it would “reprioritize its resources in Alzheimer’s disease (AD), a strategic therapeutic area expected to drive near and long-term growth. The company will continue to advance LEQEMBI® (lecanemab-irmb), the first anti-amyloid beta treatment with FDA traditional approval in the United States, and will accelerate development of potential new treatment modalities, including its ASO targeting tau (BIIB080) and an oral small molecule inhibitor of tau aggregation (BIIB113). The company will discontinue the development and commercialization of ADUHELM® (aducanumab-avwa) 100 mg/mL injection for intravenous use and will terminate the ENVISION clinical study.
Aduhelm was the topic of considerable debate when it was approved for coverage with evidence development by CMS. After public outcry over an initial coverage decision that restricted access to only those patients who qualified for the premarket clinical trial, CMS finalized a national coverage determination provide access to monoclonal antibodies that target amyloid (or plaque) for the treatment of Alzheimer’s disease that receive traditional approval from the Food and Drug Administration (FDA) under coverage with evidence development (CED). CMS, as a part of this decision, provides enhanced access and coverage for people with Medicare participating in CMS-approved studies, such as a data collection through routine clinical practice or registries. CMS also expanded access to PET scans for patients participating in these trials.
Critics, including Energy and Commerce Committee Chair, Brett Guthrie blamed CMS’s restrictive coverage policy for the company’s economic decision. He echoed statements from Biogen’s press release stating that “it wasn’t because it wasn’t safe or effective” during an event hosted by Politico on Tuesday.
Other Regulatory News
Centers for Medicare and Medicaid Services
Medicare Advantage Remains in the Spotlight – CMS Releases Proposed Payment Updates for 2025 Medicare Advantage and Part D Programs
Medicare Advantage plans could see a pay bump of around 3.7% for 2025 — a slight increase from the 3.32 they received for 2024, and higher than the projected 1.03% increase included in the 2024 advance notice. This amounts to around $16 billion in expected MA payments, net of a 2.45% revenue cut tied to proposed MA risk model revisions and a 0.15% cut tied to lower star ratings. CMS is also proposing to continue phasing in its MA risk adjustment model through 2026. For more information, see MA Press Release, Fact Sheet, and CY 2025 Advance Notice (select “2025 Advance Notice”). Comments will be accepted through 6 p.m. on March 1 before the final rate announcement is published. See also: Part D Fact Sheet and Instructions.
Ahead of the MA plan update publication, a number of stakeholders weighed in with CMS, encouraging them to preserve the MA program. This included a bipartisan majority of Senators encouraging the agency to “protect and strengthen” the MA Program. Sen. Elizabeth Warren (D-MA) joined House Progressive Caucus Chair Pramila Jayapal (D-WA) in urging CMS to “address perverse incentives in MA’s payment system. Senate Finance lawmakers Mark Warner (D-VA) and Marsha Blackburn (R-TN) introduced legislation to require Medicare Advantage plans report enrollee-level data on supplemental benefits to the Centers for Medicare & Medicaid Services (CMS), as well as report eligibility for benefits, the types of benefit categories offered, and data on utilization of and payments for such benefits.
CMS Launches Value-Based Care Website
A new CMS website aims to enhance understanding of value-based care (VBC) information for both the public and health care providers. In value-based care, doctors and other health care providers work together to manage a person’s overall health, while considering an individual’s personal health goals. The “value” in value-based care refers to what an individual values most. Although VBC has been a popular topic for years, the success of various VBC programs at CMS has been muted. Many expect CMS to require provider participation in a VBC model in the near future. In fact, CMS announced increased participation in Accountable Care Organizations in 2024. However, the National Association of Accountable Care Organizations said “more work must be done to extend value-based care and reach CMS’s goal of having all patients in an accountable care relationship by 2030.”
Medicare Contractors Announce New Billing Instructions for Category III CPT Codes
Last week, two Medicare Administrative Contractors (MACs), First Coast and Novitas, announced new documentation requirements to be billed with the initial claim submission for providers billing Category III CPT Codes. The American Medical Association (AMA) develops temporary Current Procedural Terminology (CPT) Category III codes to track the utilization of emerging technologies, services, and procedures. The CATEGORY III CPT Code description does not establish a service or procedure as safe, effective or applicable to the clinical practice of medicine. According to their website, First Coast requests the following documentation be submitted with the initial claim submission for these T codes (First Coast / Novitas).
• History and physical examination
• Lab/Diagnostic test results, if applicable
• Progress or office notes for the service performed
• Operative or procedure report, if applicable
• Full text peer reviewed articles
• Society guidelines
• Any additional documentation that supports the need for the service
Effective February 26, 2024, when records are not submitted to support the code billed, the service will be rejected. The claim must then be resubmitted with the appropriate information.
Food and Drug Administration
FDA Publishes Draft Guidance for Industry on Collection of Race and Ethnicity Data in Clinical Trials and Clinical Studies
FDA published draft guidance to articulate the agency’s expectations for, and recommendations on, use of a standardized approach for collecting and reporting race and ethnicity data in submissions including
information collected and reported from clinical studies and clinical trials for FDA-regulated medical products. Using standard terminology for race and ethnicity helps ensure that data are collected and reported consistently in submissions to FDA. This draft guidance revises the final guidance for industry and FDA staff entitled “Collection of Race and Ethnicity Data in Clinical Trials” issued on October 26, 2016. However, new draft guidance expressly does not address diversity action plans required under Food and Drug Omnibus Reform Act; initial guidance on these plans was due in December 2023 but has yet to be released. The guidance not only has implications for ongoing efforts to improve diversity in clinical trials but also contributes to a growing discussion of the use of certain demographic data in quality assessments, risk adjustments, and provider decision support tools. (AHRQ, Ways and Means Democrats)
FDA Issues Final Rule on Quality System Regulations for Medical Devices
FDA issued its long-awaited final rule on Quality Management System Regulation (QMSR) for medical devices. The regulation aligns US practices with the International Standards Organization (ISO) device quality standard which was adopted in the European Union in 2021. FDA has agreed to a two-year phase-in period, meaning full compliance won’t be required until February 2026.
Upcoming Meetings
FDA Medical Device Sterilization Town Hall: Discussion of Premarket Submission Expectations and Additional Considerations for Sterility Review
On February 7, 2024, the FDA will host the third in a series of medical device sterilization town halls to:
- Understand FDA expectations for initial premarket submissions based on the 510(k) Sterility Guidance, including recent guidance changes, sterilization modality categories, and what to include in a submission.
- Understand additional device and submission considerations for sterility that impact FDA review.
CDER and Duke-Margolis Center for Health Policy Public Meeting: Enhancing Adoption of Innovative Clinical Trial Approaches
The U.S. Food and Drug Administration and the Duke-Margolis Center for Health Policy will convene a hybrid public workshop on March 19 and 20, 2024, to discuss efforts to advance innovation of clinical trial design and conduct. CDER’s portfolio of clinical trial innovation activities are wide ranging and span across drug development programs, therapeutic areas, and scientific disciplines.
Notable Notes
NMQF and CEO Roundtable on Cancer Join Forces on Cancer Stage Shifting Initiative
The mission of the Cancer Stage Shifting Initiative is to move from late stage to early- stage diagnosis and treatment of cancer, improving cancer care and reducing cancer deaths for all, with a particular focus on equity and underrepresented populations.
BPC Urges FDA, CMS Collaboration To Streamline Coverage, Improve RWE
The Bipartisan Policy Center (BPC) offers a new report on opportunities to further strengthen collaboration between FDA and CMS.
