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Cancer Policy Monitor: January 14, 2025

Appropriations Update

-Matt Gontarchick

Just prior to leaving for the holidays on December 20, Congress passed a continuing resolution (CR) that funds the federal government at Fiscal Year (FY) 2024 levels through March 14, 2025. Lawmakers approved the stopgap spending measure because they were unable to complete negotiations on the twelve individual appropriations bills for FY 2025 by the December 20 deadline.

As the week of December 16 began, Congress was poised to pass a much broader CR, which included numerous childhood cancer bills (AKACA, Creating Hope Reauthorization, Innovation in Pediatric Drugs, and Give Kids a Chance), as well as other legislative proposals to provide disaster relief funding and agricultural subsidies. However, this broader CR failed to pass after objections from President-Elect Trump and businessman and tech mogul Elon Musk.

President Joe Biden signed the CR into law, without the childhood cancer bills included, on Saturday, December 21. As lawmakers prepare to address the next government funding deadline in March, the AACR will be working with members of the 119th Congress to advocate for the conclusion of the FY 2025 budget with the highest possible levels of funding for biomedical research.

Looking Ahead to the 119th Congress

-Carly McCallie

House Committee on Energy and Commerce Leadership Announcements

On Monday, December 9, 2024, the House Republican Steering Committee selected Representative Brett Guthrie (R-Kentucky) to lead the Energy and Commerce Committee in the 119th Congress.

Representative Guthrie currently chairs the panel’s Health Subcommittee. He rises to the position of full committee chair after the current chair Cathy McMorris Rodgers (R-Washington) did not seek reelection after two decades.

As chair of the Energy and Commerce Committee, Guthrie will be a key part of executing and shaping President-Elect Donald Trump’s agenda, which could come together through a process called reconciliation. Reconciliation bars the Senate filibuster and allows Congress to pass legislation by a simple majority vote in each chamber.

In his pitch to colleagues in the contest for full committee chair, Representative Guthrie stated he would prioritize Inflation Reduction Act oversight, bolstering drug and device supply chains, boosting transparency to lower drug prices, and improving the opioid crisis response.

Given Republican calls for a broad overhaul of the National Institutes of Health (NIH), Representative Guthrie has said he supports more transparency at the agency but backs its role in basic research.

“The NIH can and should remain a primary vessel for fundamental scientific research. We can ultimately unleash the agency’s full potential without stifling future research if we effectively increase transparency on how these research dollars are spent.”

On Friday, December 20, incoming chair Guthrie announced Energy and Commerce subcommittee leaders for the next Congress, elevating Representative Buddy Carter (R-Georgia) to be the chair of the health subpanel.

Representative Carter, a pharmacist, beat out Representatives Morgan Griffith of Virginia and Gus Bilirakis of Florida for Health Subcommittee chair. Representative Diana DeGette (D-Colorado) is widely expected to be his counterpart on the Health Subcommittee for Democrats.

Cures 2.1

On Tuesday, December 24, 2024, Representative Diana DeGette (D-Colorado) and retiring Representative Larry Bucshon (R-Indiana) released a laundry list of ideas intended to supplement incomplete policies in their Cures 2.0 legislation.

The 21st Century Cures Act was approved by Congress in 2016 and signed into law by President Obama that same year. To build upon its progress, Representative DeGette and former Representative Fred Upton (R-Michigan) announced the development of Cures 2.0 in 2019, which would further revolutionize America’s biomedical research. Parts of Cures 2.0 have been signed into law, including the authorization of the Advanced Research Projects Agency for Health (ARPA-H), though additional provisions remain outstanding. 

Representative DeGette is expected to be the ranking member on the Energy and Commerce Health Subcommittee for Democrats. With Republican control of the White House and both chambers of Congress, DeGette will have to find bipartisan support for the policies detailed in the white paper. The list includes proposals to speed the delivery of new treatments to patients, expand access to telehealth and clinical trials, encourage data-sharing, and update the FDA’s approval pathways.

President-Elect Trump Transition

In a new report issued by leaders of the House Energy and Commerce Committee, current Chair Cathy McMorris Rodgers (R-Washington), incoming Chair Brett Guthrie (R-Kentucky), and Oversight and Investigations Subcommittee Chair Morgan Griffith (R-Virginia) outlined Department of Health and Human Services (HHS) Secretary Xavier Becerra’s failure to legally reappoint 14 key officials at the National Institutes of Health (NIH). The report states that 14 of 27 NIH center directors were not properly reappointed by HHS Secretary Xavier Becerra after their terms expired at the end of 2021. The report concludes that the incoming administration would have legal standing to dismiss the NIH directors.

New Leadership at the FDA: What to Expect

-Brad Davidson, PhD

Confirmation hearings for Robert F. Kennedy Jr., prospective nominee to head the Department of Health and Human Services (HHS), and Marty Makary, MD, prospective nominee to lead the Food and Drug Administration (FDA), are likely to take place in late Q1 or early Q2 of 2025. While their policy impact on the FDA, which the HHS oversees, is only speculation at this time, their influence will be significant if confirmed.

RFK Jr. has expressed views on two topics of special relevance to those across the cancer research and treatment ecosystem: vaccines and user fees. Many types of cancer treatment can lead to weakened immune systems, and as seen during the COVID-19 pandemic, can lead patients to be more vulnerable to infection. Additionally, some viruses with vaccines against them are known to cause cancer. While RFK Jr. has indicated that he wouldn’t “take away anybody’s vaccines,” he and organizations he has overseen have expressed doubt about their safety. There are many policy actions he could take as leader of HHS to change the vaccine regulatory environment, including issuing new or revoking old guidance documents and instituting label changes, among other options.

Individuals and organizations in RFK Jr.’s orbit have also been critical of user fees, or payments made by pharmaceutical and medical device companies to the FDA to ensure timely review of their products. User fees fund nearly half of the FDA’s budget, and agreements between the FDA and companies are renegotiated every five years. With negotiations set to begin in 2025 for the next round of agreements, a new approach could shake up the regulatory environment for various drug products and diagnostics.

Makary is self-proclaimed pro-vaccine but has repeatedly criticized the FDA, including on COVID-19 vaccine regulation, remote work, and advisory committee policies. His other major known FDA-related view involves perceived misuse of the orphan drug designation, citing that pharmaceutical companies are inappropriately submitting drugs intended for use in non-rare disease populations as orphan drugs in order to receive lucrative benefits. As FDA commissioner, Makary would have broad ability to institute changes to these policies.

Others involved in the incoming administration, such as Department of Government Efficiency Colead Vivek Ramaswamy, have also been critical of the FDA. This speculation leaves aspects of the FDA’s future unclear, although generally politicians across both sides of the aisle are cognizant of and invested in American successes in cancer research and drug development. 

A Look Ahead for Planned FDA Policies in 2025: Uncertainty Prevails

-Brad Davidson, PhD

Many of FDA’s plans for 2025 are taking shape or are already in mid-stream. However, the incoming Trump administration could change the course of some of the FDA’s projected goals. Here are some of the FDA’s planned regulatory policy actions for 2025 that could impact cancer research and drug development, and potential interactions they might have with the incoming administration:

  •  In June 2024, the FDA released a draft guidance, required by the Consolidated Appropriations Act of 2023 to be finalized by June 2025, that would require all clinical trials to submit Diversity Action Plans outlining how the study will seek to enroll diverse patient populations that represent the American populace. It is expected by regulatory policy insiders that this guidance will undergo change based on the Trump administration’s goals: potentially being rebranded as putting American populations first, facing more significant alterations, and/or being delayed. There is no outlined penalty for missing the June 2025 deadline for finalization.
  • According to final guidance released in 2024, the FDA plans to begin phasing-in increased regulations in 2025 for lab-developed tests (LDTs), an important class of diagnostic test that is sometimes used in the clinical research stage to aid the development of cancer drug products and is used for many pharmacogenetic tests. It is unclear whether this will move forward as planned given pushback from industry partners and the robust diagnostic testing policy section in Project 2025, an extensive white paper written in part by individuals involved in the Trump administration.
  • As of this writing, FDA’s Center for Devices and Radiologic Health is the only FDA Center that has released a specific guidance agenda for 2025, with most of it concerning digital health and lab developed tests. Plans for other centers will likely be continuations of 2024 agendas, accessible online.
  • When asked about important actions he hoped to get done before the end of his time as FDA Commissioner at the 2024 Friends of Cancer Research Annual Meeting, Robert Califf, MD, stated that certain regulation of tobacco products would be key to protecting the public health. He specifically called out the finalization of the FDA’s proposed rule to prohibit menthol as a characterizing flavor in cigarettes and the proposal of a rule to reduce nicotine levels in cigarettes. Whether these actions will be completed prior to Califf’s resignation, or if they will be continued by Trump administration, is unclear.

REGISTER NOW for the FDA-AACR Workshop: To Test or Not to Test—That is The Question: DPD Deficiency and Weighing the Potential Harms

The U.S. Food and Drug Administration (FDA), the Oncology Center of Excellence (OCE), and the American Association for Cancer Research (AACR) are hosting a hybrid workshop titled “To Test or Not to Test—That Is the Question: DPD Deficiency and Weighing Potential Harms” on January 16, 2025, at the Bethesda Marriott Pooks Hill Hotel in Bethesda, Maryland. This workshop will explore the clinical, pharmacologic, genomic, device, and policy considerations for whether patients should be required to undergo DPD deficiency testing before initiating chemotherapy with fluoropyrimidines. Registration is open for in-person and virtual attendance.

Highlights will include:

  • A roundtable discussion between Paul Kluetz, MD, Deputy Director of the FDA’s OCE, Michael Pacanowski, PharmD, MPH, Director of the FDA’s Division of Translational and Precision Medicine, and patient advocates and community oncologists impacted by DPD deficiency. 
  • A presentation by Robert Diasio, MD, Director Emeritus of the Mayo Clinic Comprehensive Cancer Center and discoverer of DPD deficiency, to discuss the evidence for pharmacogenomic testing for DPD, including complex clinical cases.
  • A presentation by Alan Venook, MD, Madden Family distinguished professor in medical oncology and translational research at UCSF and member of the Board of Directors for the National Comprehensive Cancer Network (NCCN), to discuss the rationale behind current NCCN clinical guidelines for DPD testing.
  • A presentation by Christina Wu, MD, a professor and gastrointestinal oncologist at Mayo Clinic Arizona, to discuss DPD testing as it is currently practiced, including types of tests used and their associated timing and costs, as well as how guideline-driven or label-driven changes might affect current practice.

For additional information, please visit the workshop website.

Registration Open for the AACR Patient Advocate Forum on Clinical Trials

Join advocates and researchers from around the world on Tuesday, January 21 (1-2:30 p.m. ET), for the virtual American Association for Cancer Research (AACR) Patient Advocate Forum: Exploring a New Generation of Patient-Centric Cancer Clinical Trials. The forum features moderator AACR SSP Founder Dr. Anna Barker from the Ellison Institute of Technology and presentations by AACR President Dr. Patricia LoRusso from Yale University, AACR Past President Dr. David Tuveson from Cold Spring Harbor Laboratory, and winner of the SABCS William L. McGuire Memorial Lecture Award Dr. Laura van ’t Veer from UCSF.

More people are living through and beyond cancer thanks in large part to clinical trials which have given countless cancer patients access to lifesaving treatments. As scientific research has driven progress in cancer diagnosis and treatment, it has also led to significant changes in the way clinical trials are conducted. These renowned speakers will explore cutting-edge developments revolutionizing the process and paving the way for more efficient, inclusive, and patient-centric clinical trials.

The AACR offers this educational program to all members of the patient advocate and research community at no cost. If you can’t join the forum on January 21, register to access the recording.

Register online.

A Flurry of Guidance Documents from Oncology’s Center of Excellence to Close 2024

-Brad Davidson, PhD

The FDA Oncology Center of Excellence (OCE) released three guidance documents to round out 2024, including one final and two draft guidances. Guidances, while not describing legally enforceable responsibilities, generally outline the Food and Drug Administration’s (FDA) thoughts on how pharmaceutical companies should operate towards receiving approval for drug products. While final guidances are definitive documents outlining actual recommendations, draft documents offer a peek into the future and provide key interested parties an opportunity to give feedback.

A final guidance, based on draft guidance from 2022, was titled “Use of Circulating Tumor Deoxyribonucleic Acid for Early-Stage Solid Tumor Drug Development; Guidance for Industry,” and outlined recommendations for the use of circulating tumor DNA (ctDNA) as a biomarker in clinical development programs for early-stage solid tumors. The guidance particularly focuses on the use of ctDNA to detect minimal residual disease (MRD), the design of clinical trials around MRD tests, and the validation of various MRD ctDNA assay types. The guidance also highlighted the use of ctDNA as a response measure to support dosing optimization and outlined research that could facilitate the use of ctDNA as an early endpoint to support drug approval in the future. 

In late November 2024, FDA issued a draft guidance titled “Assessment of Ovarian Toxicity in Premenopausal Adults During Drug Development for Oncologic Products.” This document calls on sponsors of trials enrolling premenopausal patients with cancer to more thoroughly assess ovarian function, as some cancer treatments can lead to ovarian dysfunction and, as a result, infertility, osteoporosis, sexual dysfunction, and earlier onset of cardiovascular disease. Unfortunately, few trials in this population currently explore ovarian toxicity at depth. Herein, the FDA recommended best practices for measuring ovarian function throughout trials, including biomarkers to use and the frequency of testing, among other considerations. 

FDA’s Center for Drug Evaluation and Research (CDER) released a draft guidance in collaboration with the Center for Biologics Evaluation and Research (CBER) and OCE titled “Expedited Program for Serious Conditions—Accelerated Approval of Drugs and Biologics” as an update to the Accelerated Approval Program. Congress passed legislation in 2023 updating accelerated approval and requiring FDA to issue guidance on the implementation of the changes; this guidance will be one of multiple that serve this purpose. Generally, accelerated approval allows faster review for drugs treating serious conditions with unmet medical needs by evaluating early surrogate endpoints of clinical benefit, instead of more traditional endpoints indicative of realized clinical benefit. Confirmatory trials then must be completed to verify benefit, but these trials sometimes fail or are not actually performed, especially in oncology. This guidance outlines, among other topics, the implementation of an expedited withdrawal process for drugs that do not show clinical benefit in confirmatory trials or for which confirmatory trials failed to be conducted after accelerated approval. It also previews a future guidance that will focus on the timeline for initiation of confirmatory trials with respect to accelerated approval. OCE has already issued draft guidance that recommends trials be nearly fully enrolled at the time of accelerated approval, but more details and whether the rest of the FDA will adopt this recommendation remain to be seen.  

ONCOLOGY APPROVAL RECAP

Between November 22nd and December 20th, the U.S. Food and Drug Administration (FDA) issued six new oncology drug approvals, including one accelerated approval for a novel agent and a previously approved agent, a new indication for a previously approved oncology drug, and three traditional approvals for novel therapies:

  • Zenocutuzumab-zbco was granted accelerated approval for adults with either non-small cell lung cancer or pancreatic adenocarcinoma that is advanced, unresectable, or metastatic and harbors a NRG1 gene fusion that has progressed on or after prior systemic therapy. This is the first approval of a systemic therapy product designed to target cancers with NRG1 gene fusions. This application was aided by priority review, breakthrough designation, and orphan drug designation.
  • Durvalumab was approved for adults with limited-stage small cell lung cancer whose disease has not progressed following concurrent platinum-based chemotherapy and radiation. This is the eighth indication for durvalumab, an immuno-oncology agent, and its fifth overall in lung cancer. This application received priority review and breakthrough designation.
  • Cosibelimab-ipdl was approved for adults with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or curative radiation. This is the first approval for cosibelimab-ipdl, an anti-PD-L1 immuno-oncology agent, in any indication.
  • Ensartinib was approved for adult patients with ALK-positive locally advanced or metastatic non-small cell lung cancer who have not previously received an ALK-inhibitor. This is the first approval of this ALK-targeting small molecule in any indication, expanding potential options for the first-line treatment of late-stage non-small cell lung cancer. Ensartinib is also under investigation for use in the adjuvant setting.  
  • Remestemcel-L-rknd, an allogenic bone marrow-derived mesenchymal stromal cell therapy, was approved for steroid-refractory acute graft versus host disease in pediatric patients 2 months of age and older. This is the first ever approval of a mesenchymal stromal cell therapy. This application received fast track, orphan drug, and priority review designations.  
  • Encorafenib was granted accelerated approval in combination with cetuximab and mFOLFOX6 for patients with metastatic colorectal cancer with BRAF V600E mutations. This moves encorafenib into the first line in this disease in accordance with Project FrontRunner, where it was already approved in combination with cetuximab alone for patients who had already received a prior line of therapy. Review was performed through Project Orbis in collaboration with Health Canada, utilized the Real-Time Oncology Review pilot program, and was granted priority review.

Apply NOW: AACR Annual Meeting 2025 Advocate Partners Pavilion

The American Association for Cancer Research (AACR) invites patient advocate exhibitors to participate during the AACR Annual Meeting 2025 in Chicago, Illinois (April 25-30, 2025) in the Advocacy Partners Pavilion. Eligible organizations will be accepted on a first-come, first-served basis. Please note that there are limited spots available. Submit an application online.

U.S. Surgeon General Releases New Advisory on the Link Between Alcohol and Cancer

-Blake William Rostine

A new report from the U.S. Surgeon General Dr. Vivek Murthy highlighted the risk factor of alcohol, notifying Americans about the direct link between alcohol consumption and cancer. As published in the 2024 AACR Cancer Progress Report, alcohol consumption increases the risk for at least seven types and cancer and is the third leading preventable cause of cancer in the U.S.

With alcohol contributing to approximately 100,000 cancer cases and 20,000 cancer deaths in the U.S. annually, communicating these links to the public is an important step towards reducing new cancer cases. Although the scientific community has been researching the link between cancer and alcohol for over four decades, less than half of Americans recognize alcohol as a risk factor.

The report also provides recommendations on how to increase public awareness of the link, including reassessment of the guidelines for alcohol consumption and updates to the current health warning label on alcoholic products. The American Association for Cancer Research will continue to work to promote research that can help save lives and looks forward to presenting up-to-date findings in the upcoming 2025 AACR Cancer Progress Report.

The U.S. Surgeon General’s report can be accessed online.