Cancer Policy Monitor: February 11, 2025
- President Donald Trump’s Second Administration Begins with Bold Policy Moves
- Confirmation Hearings for Robert F. Kennedy, Jr., To Lead the U.S. Department of Health and Human Services
- Appropriations Update
- Recording Available: FDA-AACR Workshop To Test or Not To Test – That Is The Question: DPD Deficiency And Weighing Potential Harms
- FDA Guidances Outline New Accelerated Approval and Artificial Intelligence Policies
- The FDA Center for Tobacco Products’ Big Week: Zyn Marketing Authorization and Proposed Nicotine Reduction Rule
- Save The Date: 2025 AACR-AACI Hill Day—Thursday, May 22
- Watch Now: AACR Patient Advocate Forum Exploring A New Generation of Patient-Centric Cancer Clinical Trials
- Oncology Approval Recap
President Donald Trump’s Second Administration Begins with Bold Policy Moves
-Carly McCallie
Early actions by the Trump Administration, including freezing federal funding, canceling grant review panels, and the nomination of vaccine skeptic RFK Jr. as Department of Health and Human Services (HHS) Secretary, have sparked concerns about disruptions to biomedical research and public health. The implications of many of these measures are unclear and will likely depend on how they are interpreted and applied, both by agencies and, potentially, the courts.
Administration Directs Federal Health Agencies to Pause Communications, Hiring, and Travel
In the weeks since his inauguration on Monday, January 21, the Trump Administration has taken numerous actions that have caused significant disruptions across various agencies within the HHS, particularly the National Institutes of Health (NIH), Food and Drug Administration (FDA), and Centers for Disease Control and Prevention (CDC). The president’s numerous executive orders and temporary suspensions of key research-related activities at these agencies have raised considerable concern among stakeholders in the medical research community.
On Tuesday, January 21, Acting Secretary of the HHS Dorothy Fink told agency staff leaders in a memo to temporarily stop public messaging, pending a review. The memo advises leaders of the CDC, the NIH, and the FDA to stop the publication of regulations, guidance documents, grant announcements, social media posts, press releases, and other “communications,” and to cancel speaking engagements. Any exceptions must be applied for and approved by the President’s appointees. These restrictions also apply to committees subject to the Federal Advisory Committee Act (FACA), which includes NIH study sections.
In an email sent on Wednesday, January 22, NIH travel chief Glenda Conroy notified senior agency officials of an “immediate and indefinite” suspension of all travel throughout HHS with few exceptions, such as currently traveling employees returning home. Researchers who planned to present their work at meetings must cancel their trips, as must NIH officials promoting agency programs off site or visiting distant branches of the agency. “Future travel requests for any reason are not authorized and should not be approved,” the memo said.
The hiring freeze applies governmentwide, whereas the pause on communications and travel appears to be limited to HHS. Such pauses are not unprecedented when a new administration comes in, but some of these measures, including pulling job ads and rescinding offers, are more extreme than any previously.
The agency expects “additional guidance” on Saturday, February 1.
Administration Orders Widespread Pause of Federal Loans and Grants
On Monday, January 27, the Office of Management and Budget (OMB) released a directive to federal agencies issuing a “temporary pause [on] all activities related to obligation or disbursement of all Federal financial assistance, and other relevant agency activities that may be implicated by the executive orders … .” During the “temporary pause,” agencies are directed to conduct a comprehensive review of their programs to identify any that may be affected by President Trump’s executive orders. Agencies are required to report their findings to OMB by Monday, February 10.
On Tuesday, January 28, OMB released a question and answer document to clarify the scope of the directive, stressing that any federal program not implicated by the president’s recent executive orders is not subject to the freeze.
The executive orders listed in the guidance are:
- Protecting the American People Against Invasion
- Reevaluating and Realigning United States Foreign Aid
- Putting America First in International Environmental Agreements
- Unleashing American Energy
- Ending Radical and Wasteful Government DEI Programs and Preferencing
- Defending Women From Gender Ideology Extremism and Restoring Biological Truth to the Federal Government
- Enforcing the Hyde Amendment
The freeze was to be effective Tuesday, January 28, at 5 p.m., but before it could take effect, U.S. District Judge Loren L. AliKhan issued an administrative stay temporarily blocking the federal funding freeze until Monday, February 3. This order was in response to a lawsuit filed in the District of Columbia by a coalition of nonprofits, public health organizations, and small businesses. The nonprofit organizations had alleged in their filing that the memo “fails to explain the source of OMB’s purported legal authority to gut every grant program in the federal government.” The groups also said the memo failed to consider the interests of grant recipients, “including those to whom money had already been promised.”
Shortly after the decision by Judge AliKhan on Tuesday, a group of attorneys general from 22 states and the District of Columbia filed a separate challenge in federal court in Rhode Island.
On Wednesday, January 29, the Administration rescinded the OMB memo freezing trillions in federal grants and loans. However, shortly after the OMB rescinded the memo, White House Press Secretary Karoline Leavitt posted on X, claiming, “This is NOT a rescission of the federal funding freeze. It is simply a rescission of the OMB memo. Why? To end any confusion created by the court’s injunction. The President’s EO’s on federal funding remain in full force and effect and will be rigorously implemented.”
On Friday, January 31, in the separate filing in Rhode Island, U.S. District Judge John McConnell ordered a temporary pause to the federal funding freeze, citing Leavitt’s social media post as evidence the case should continue. The Justice Department had argued the issue was moot because the White House memo was rescinded, but McConnell found the evidence showed “the alleged rescission of the OMB Directive was in name-only.”
“Based on the Press Secretary’s unequivocal statement and the continued actions of Executive agencies, the Court finds that the policies in the OMB Directive that the States challenge here are still in full force and effect and thus the issues presented in the States’ TRO motion are not moot,” he wrote.
Unlike Judge AliKhan’s short-term order, Judge McConnell’s order prevents the administration from adopting the OMB directive or cutting off the states’ federal grant funding. The block will last until the judge can consider the states’ motion for a longer injunction, which has not yet been filed.
In the coming days and weeks, the AACR expects the White House and federal agencies to offer updated guidance on what kinds of activities are impacted.
Matthew J. Memoli, MD, MS, to Serve as Acting NIH Director
The National Institutes of Health (NIH) announced Matthew J. Memoli, MD, MS, will be serving as Acting NIH Director as the nomination of Jay Bhattacharya, MD, PhD, awaits confirmation by the Senate. Memoli previously served as Director of the Laboratory of Infectious Diseases (LID) Clinical Studies Unit within the National Institute of Allergy and Infectious Diseases (NIAID). Memoli’s research focused on areas including human influenza pathogenesis; respiratory viruses; influenza transmission and correlates of protection; influenza and other viral human challenge models; impact of respiratory viruses on at risk populations; broadly protective vaccines; and emerging infectious diseases. Memoli received his master’s degree in microbiology from Thomas Jefferson University, and his MD from St George’s University School of Medicine. Before serving at NIH, Memoli completed residency in internal medicine at the Washington Hospital Center Georgetown University Internal Medicine Program in Washington, D.C.
President Trump Withdraws Proposed Menthol Ban
-Blake William Rostine
A Biden Administration proposal which would have banned menthol flavoring in tobacco products has been withdrawn by the Trump Administration. The U.S. Food and Drug Administration (FDA) proposed product standard would have prohibited menthol flavoring in cigarettes in order to combat the addictive nature of the flavor. Former Health and Human Services Secretary Xavier Becerra previously said the “proposed rules would help prevent children from becoming the next generation of smokers and help adult smokers quit,” and they represented “an important step to advance health equity by significantly reducing tobacco-related health disparities.”
However, a Tuesday, January 21, filing by the Office of Information and Regulatory Affairs shows the proposal is now “withdrawn.” The previously discussed pause in public communications for federal health agencies will not allow the FDA to comment on this issue.
President Trump Issues Executive Order on PCAST
On Thursday, January 23, the White House announced the formation of the President’s Council of Advisors on Science and Technology (PCAST). The council will consist of 24 members from academia, industry, and government aimed at harnessing the full potential of American innovation to chart a path forward for leadership in science and technology. PCAST will advise the President on matters related to science, technology, education, and innovation policy for two years from January 23, unless extended by the President.
President Trump’s executive order positions this iteration of PCAST as a body that will refocus science and technology policy to “emphasize results-driven excellence and merit-based achievement,” according to the fact sheet.
Confirmation Hearings for Robert F. Kennedy, Jr., To Lead the U.S. Department of Health and Human Services
-Blake William Rostine
Two Senate committees held confirmation hearings during the week of January 27 for President Donald Trump’s pick for Secretary of Health and Human Services, Robert F. Kennedy, Jr.
Both the U.S. Senate committees on Health, Education, Labor, and Pensions (HELP) and Finance conducted their confirmation hearings for Robert F. Kennedy, Jr., Trump’s pick to become the Secretary of the Department of Health and Human Services (HHS), the nation’s top health agency.
Concerns revolve around Kennedy’s past statements about doubting the effectiveness of vaccines, as well as his disproven statements that link vaccinations and autism. During the Senate HELP Committee hearing, Kennedy stated that America should return to the “gold standard of science” and that he would support vaccines when data is shown.
In both hearings, lawmakers asked Kennedy to elaborate on his past comments to “clean house” at NIH by laying off 600 staff members. Kennedy minimized his previous calls to terminate NIH employees by pointing to the hiring and dismissal of similar numbers of political appointees that happens with each new administration. However, Kennedy pledged to not fire anyone who is doing their job.
Kennedy was questioned if he would continue to accept referral fees from a law firm representing patients alleging injuries from an HPV vaccine, which can help to prevent cervical cancer. He stated that should he be confirmed, he would not continue to collect these fees.
Kennedy will have the opportunity to speak with Senators until the committees vote on his nomination, which will be followed by the whole Senate’s consideration. Recordings of the HELP Committee hearing and the Finance Committee hearing are available online.
Appropriations Update
-Matt Gontarchick
The 119th Congress officially convened on January 3 with unfinished business from the previous Congress and a lengthy to-do list for the year ahead. The federal government is currently operating under a continuing resolution (CR) signed into law on December 21, 2024 that maintains Fiscal Year (FY) 2024 funding levels through March 14, 2025. In late January, Republican and Democratic leaders of the House and Senate Appropriations committees restarted negotiations on FY 2025 appropriations, with an eye on topline spending levels.
Concurrently, congressional Republicans are debating the best approach to use budget reconciliation to enact legislation on President Donald Trump’s priorities on energy, immigration, and taxes. House Republicans want to address all three issue areas in one bill, while Senate Republicans prefer to split the process into two bills, with the first concerning energy and immigration, and the second focused on taxes. Some health-related measures including an extension of telehealth flexibilities under Medicare as well as new rules for pharmacy benefit managers could be included in the budget reconciliation process. While President Donald Trump has expressed a preference for including all priorities in one comprehensive reconciliation bill, he has signaled an openness to a two-bill process. Speaker Mike Johnson (R-Louisiana) said he would like both the House and the Senate to approve a budget reconciliation framework by the end of February.
Adding to this mix is the debt ceiling, which must be raised within the next few months to avoid a default on the national debt. Some congressional Republicans have communicated an interest in tying debt ceiling legislation to California wildfire aid, although House Democratic Leader Hakeem Jefferies (D-New York) has openly rejected this proposal. Others have suggested including a debt ceiling hike in a reconciliation bill; however, this plan has drawn the ire of conservative Republicans. Additional lawmakers have put forth that a debt ceiling measure could be added to government funding legislation for FY 2025.
Regardless, members of Congress face tough decisions as competing priorities and deadlines unfold in the coming months. The AACR will continue to monitor the FY 2025 appropriations process and other legislative efforts and other as lawmakers work amid a busy schedule.
House Labor-HHS Appropriations Subcommittee Roster Announced
The House Labor-HHS Appropriations Subcommittee, which has jurisdiction over funding for the National Institutes of Health (NIH) and other federal health agencies, issued its roster for the 119th Congress. Representatives Robert Aderholt (R-Alabama) and Rosa DeLauro (D-Connecticut) will continue to serve as chair and ranking member respectively. Representative Julia Letlow (R-Louisiana) will serve as vice chair. The subcommittee has four new members including three new Republican members and one new Democratic member. New members include Representatives Jake Ellzey (R-Texas), Stephanie Bice (R-Oklahoma), Riley Moore (R-West Virginia), and Madeline Dean (D-Pennsylvania). Members cycling off the Labor-HHS subcommittee include Representatives Juan Ciscomani (R-Arizona) and Chuck Edwards (R-North Carolina).
Senate Labor-HHS Appropriations Subcommittee Roster Announced
The Senate Labor-HHS Appropriations Subcommittee, which has jurisdiction over funding for the National Institutes of Health (NIH) and other federal health agencies, issued its roster for the 119th Congress. Senators Susan Collins (R-Maine) and Patty Murray (D-Washington) will serve as chair and vice chair respectively.
Recording Available: FDA-AACR Workshop To Test or Not To Test—That Is The Question: DPD Deficiency And Weighing Potential Harms
The U.S. Food and Drug Administration (FDA) and the AACR hosted a hybrid workshop on January 16, 2025, in Bethesda, Maryland, to discuss the considerations around dihydropyrimidine dehydrogenase (DPD) deficiency testing for patients on fluoropyrimidine-based cancer treatments, such as fluorouracil (5-FU) and capecitabine. The workshop brought together over 700 people from around the world, underscoring a strong commitment to enhancing patient care and advancing the safety of cancer treatments.
A recording of the workshop along with the slide deck are now available on the workshop webpage.
FDA Guidances Outline New Accelerated Approval and Artificial Intelligence Policies
-Brad Davidson, PhD
In late 2024, U.S. Food and Drug Administration (FDA) released a draft guidance titled “Expedited Program for Serious Conditions—Accelerated Approval of Drugs and Biologics” as an update to the Accelerated Approval Program. Generally, accelerated approval (AA) provides faster access to drugs treating serious conditions with unmet medical needs by allowing review to hinge on early surrogate endpoints of clinical benefit instead of more traditional endpoints indicative of realized clinical benefit. Problematically, some drugs granted AA are later proven in confirmatory trials to provide no additional patient benefit or have difficulties completing the necessary confirmatory trial, especially in oncology. To speed these trials along, the late 2024 guidance stated a requirement for confirmatory trials to be “underway” before granting AA to a drug product but left unclear what “underway” meant. A new draft guidance titled “Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway” rectifies this, generally defining a confirmatory trial as underway if the trial has a reasonable target completion date, the sponsor outlines a satisfactory plan to complete the trial and will complete in a timely manner, and enrollment has been initiated. This definition is intended to be flexible as various diseases require trials that take different amounts of time, among other specific considerations. There are also carveouts where the FDA may not use this standard, including for certain rare diseases and viruses. On the other hand, the FDA indicated they may require enrollment to be complete upon AA if adequate trial enrollment or patient retention seems unlikely after AA. The FDA will be taking comments on this proposed guidance until March 10.
In a culmination of a multi-year effort encompassing the release of discussion papers and the holding of public workshops, the FDA recently released a draft guidance titled “Considerations for the Use of Artificial Intelligence To Support Regulatory Decision-Making for Drug and Biological Products.” This guidance addresses an uptick seen in the use of artificial intelligence (AI) in regulatory submissions to support the efficacy, safety, or quality of a product. Potential uses for AI in these submissions have included the prediction of clinical pharmacokinetics or exposure-response relationships, the prediction of patient outcomes, the processing and analyzing of large datasets, and more. However, the quality of AI models greatly varies, as does the relative importance of the decisions they are designed to make. In response, this guidance outlines a framework for the establishment of AI model credibility that responds to the amount of risk inherent to the specific context of use of the AI model. For example, in situations where the model is operating with low outside guidance and the decision being made is of clinical consequence, a higher standard would be applied to ensure the AI model is credible. The FDA also recently published a draft guidance surrounding AI-enabled medical devices; these efforts, in addition to the Trump Administration’s documented interest in AI policy, signal fast times ahead in this space. The FDA will be taking comments on this proposed guidance until April 7.
The FDA Center for Tobacco Products’ Big Week: Zyn Marketing Authorization and Proposed Nicotine Reduction Rule
-Brad Davidson, PhD
The U.S. Food and Drug Administration (FDA) Center for Tobacco Products (CTP) released two important decisions on back-to-back days that could have transformative effects on the way Americans interact with tobacco.
First, the FDA CTP announced a long-awaited proposed rule to restrict the amount of nicotine in most combusted tobacco products to “very low” levels that are 95% lower than current levels and are nearly incapable, if not incapable, of creating and sustaining addiction. Originally introduced in 2018 as an advanced notice of proposed rulemaking that elicited nearly 8,000 public comments, including a supportive comment from the AACR, this rule if finalized would overhaul tobacco use in the United States. In its current form, this rule would require an immediate drop in nicotine concentration, instead of a gradual phased drop, beginning two years after finalization for products including cigarettes, most cigars, and various forms of loose tobacco intended for smoking. According to FDA population health modeling, finalization of this rule would encourage 12.9 million adults to quit within just 1 year, and by the year 2060 would prevent 21.1 million youths from starting smoking as well as avert 1.8 million tobacco-related deaths. The FDA will be taking comments on this proposed rule until September 15.
Additionally, the FDA CTP authorized the marketing of 20 Zyn brand flavored nicotine pouch products. This is FDA’s first and only authorization to date of nicotine pouch products. Many other nicotine products, including most e-cigarettes and other brands of nicotine pouches, remain on the market illegally. While the FDA indicated that the newly authorized Zyn products are harmful to health, they met the standard of “appropriate for the protection of the public health,” which balances the benefits of a new tobacco product to benefit the cessation efforts of current cigarette smokers against the potential harm of new user initiation. Key datapoints that drove this authorization included the substantially lower presence of harmful cancer-causing constituents in Zyn than most other tobacco products, evidence that adults can completely switch from cigarettes and/or smokeless products to Zyn, and very low youth nicotine pouch uptake to date. While these products are flavored, which typically indicates a potential to appeal to youth, the 2024 National Youth Tobacco Survey showed that only 1.8% of U.S. middle and high school students report current use of nicotine pouch products. Critically, the authorization requires that these products are not marketed to youths and leaves the door open for revocation of authorization if marketing practices begin to cater to youths or if youth usage significantly increases.
Save The Date: 2025 AACR-AACI Hill Day—Thursday, May 22
The American Association for Cancer Research (AACR) and the Association of American Cancer Institutes (AACI) invite you to participate in a Capitol Hill Day to support cancer research on Thursday, May 22. This Hill Day will bring cancer center directors, researchers, physician-scientists, cancer survivors, and other advocates together to build support for a strong federal investment in biomedical research, and cancer research in particular, through the National Institutes of Health and the National Cancer Institute.
Participation is open to AACR and AACI members. Registration will open this spring; stay tuned for more information.
Watch Now: AACR Patient Advocate Forum Exploring A New Generation of Patient-Centric Cancer Clinical Trials
More people are living through and beyond cancer, thanks in large part to clinical trials that have provided countless cancer patients with 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. The AACR held a forum on Tuesday, January 21, to explore cutting-edge developments that are revolutionizing the process and paving the way for more efficient, inclusive, and patient-centric clinical trials.
Oncology Approval Recap
Between December 20 and January 24, the U.S. Food and Drug Administration (FDA) issued four new traditional approvals for two drugs which had previously received accelerated approval and two novel drugs.
- Acalabrutinib was granted traditional approval in two settings: as a single agent for adults with previously treated mantle cell lymphoma (MCL) and in combination with bendamustine and rituximab for adults with previously untreated MCL who are ineligible for autologous hematopoietic stem cell transplantation. This approval constitutes a conversion from a 2017 accelerated approval to traditional approval for the single agent indication. Review was conducted under Project Orbis in collaboration with the Australian Therapeutic Goods Administration, Health Canada, and Switzerland’s Swissmedic.
- Sotorasib was approved in combination with panitumumab for adults with KRAS G12C mutated metastatic colorectal cancer who have previously received prior triplet fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. This is the first traditional approval for sotorasib, which previously received accelerated approval in 2021 for use in adults with KRAS G12C mutant non-small cell lung cancer who have received at least one prior systemic therapy.
- Datopotamab deruxetecan-dlnk was approved for adults with unresectable or metastatic hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative breast cancer who have received prior endocrine therapy and chemotherapy for unresectable/metastatic disease. This is the first approval for this drug product in any setting.
- Treosulfan was approved in combination with fludarabine as a preparative regimen for allogenic hematopoietic stem cell transplantation in adult and pediatric patients 1 year of age or older with acute myeloid leukemia or myelodysplastic syndrome. This is the first approval for this drug product in any setting.