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Cancer Policy Monitor: December 12, 2023

Congressional Update

-Benjamin Krinsky, PhD

On November 16, President Biden signed H.R. 6363, the Further Continuing Appropriations and Other Extensions Act, 2024 into law, averting a government shutdown. This temporary spending measure (called a continuing resolution or CR) keeps funding the federal government at fiscal year (FY) 2023 levels through early 2024.

In an unusual departure from previous CRs, the new law funds different portions of the federal government for different lengths of time. The CR provides funding through January 19, 2024, for the parts of the federal government corresponding to the following four bills: Agriculture, Rural Development, Food and Drug Administration (FDA); Energy and Water Development; Military Construction and Veterans Affairs; and Transportation, Housing, and Urban Development. The parts of the government encompassed by the remaining eight appropriations bills are funded through February 2, 2024, including the Labor, Health and Human Services, and Education bill, which funds the National Institutes of Health (NIH) and the National Cancer Institute (NCI). The CR was written by Republican leadership in the House of Representatives to extend funding into early 2024 to side-step a political fight over federal spending just prior to the December holiday season. This strategy of staggered budget deadlines is also an attempt to try and move congressional budget negotiations forward in a way that would avoid an outcome opposed by many Republican members, namely the passage of a single, large bill to fund the entire federal government (an omnibus).

The latest CR passed the House by a vote of 336-95 and the Senate by a vote of 87-11. Like the previous CR that funded the federal government through November 17, the measure passed the House with many Democratic votes. Speaker of the House Mike Johnson (R-LA) continues to face pressure from conservative Republican members who are pushing to cut spending below the levels his predecessor, Representative Kevin McCarthy (R-CA), negotiated with President Biden.

In the lead-up to the passage of the CR, both the House and Senate had been working to pass FY 2024 appropriations bills. However, large differences remain between the measures being considered by each chamber: the House has been advancing bills with lower levels of spending, and deep disagreements remain over so-called “poison pill” policy riders that have been attached to appropriations bills.

With the fate of the FY 2024 budget still uncertain, the American Association for Cancer Research will continue to advocate for a final federal budget that provides robust funding increases for NIH, NCI, and other research and health programs.

Biden Administration Appoints W. Kimryn Rathmell, MD, PhD, as Director of the National Cancer Institute

-Blake William Rostine

The American Association for Cancer Research (AACR), the world’s first and largest cancer research organization, congratulates W. Kimryn Rathmell, MD, PhD, on her appointment by President Biden to serve as the 17th director of the National Cancer Institute (NCI). Previously an AACR Team Science Award recipient and an AACR-supported research grant awardee, Rathmell is a renowned physician-scientist and expert in the understanding and treatment of kidney cancer. Her research uses methods including genetics and molecular and cell biology to reveal the causes of kidney cancers and to develop new ways to improve outcomes for patients.

Dr. Rathmell comes to NCI from Vanderbilt University Medical Center, where she served as the Hugh Jackson professor of medicine and chair of the Department of Medicine. At NCI, she succeeds Monica A. Bertagnolli, MD, who was recently confirmed as NIH Director.

“The entire cancer research and patient care community owes Dr. Bertagnolli a tremendous amount of gratitude for her extraordinary dedication and outstanding commitment to reducing cancer incidence, morbidity, and mortality,” as stated in an official AACR press release by Margaret Foti, PhD, MD (hc), chief executive officer of the AACR. “[The] appointment by President Biden of Dr. Rathmell as NCI’s next director ensures a continuity in leadership and management at the NCI that is especially needed during this historic era of unprecedented scientific opportunities that are before us. We are absolutely thrilled about Dr. Rathmell’s appointment, and the AACR truly looks forward to working with Dr. Rathmell in the months and years to come towards our shared vision of a world without cancer.”

Bertagnolli stated that “Dr. Rathmell is an ideal candidate to lead NIH’s efforts to end cancer as we know it. She has spent her career driving efforts to boost support and improve outcomes for those facing a cancer diagnosis, living the principles of President Biden’s Cancer Moonshot Initiative.”

Rathmell will lead the NCI and the NIH’s efforts to support scientific discoveries that will advance cancer prevention and treatment, improve the lives of patients, survivors, and their families, and train the next generation of the cancer research workforce. The AACR applauds her appointment and looks forward to working with Dr. Rathmell in the months and years to come towards a shared vision of a world without cancer.

Register Now! FDA-AACR Workshop on Optimizing Dosages for Oncology Drug Products

–Nicholas Warren, PhD 

The U.S. Food and Drug Administration (FDA), Office of Clinical Pharmacology and the American Association for Cancer Research (AACR) are collaborating on a day-and-a-half long hybrid workshop titled “Optimizing Dosages for Oncology Drug Products: Quantitative Approaches to Select Dosages for Clinical Trials”, which will take place February 15-16 at Grand Hyatt in Washington D.C. (1000 H St NW). This follows previous FDA-AACR workshops in 2015, 2016, and 2017 on dosage optimization. Free registration is now available to attend the new workshop in-person or virtually.

Historically, dose-finding trials for oncology drugs have been primarily designed to determine the “Maximum Tolerated Dose” (MTD), which is determined by gradually increasing the dose in a small number of patients at each dose for short periods of time until it is too toxic for patients. This strategy was developed for cytotoxic chemotherapy because efficacy because of the small therapeutic window for that class of drugs. However, this approach may lead to poorly tolerated dosages for modern oncology drugs with novel mechanisms of action, including targeted therapies and immunotherapies. Continued reliance on the MTD may lead to unnecessary side effects without added benefits for patients.

FDA and AACR strongly encourage  a more holistic approach that leverages all available nonclinical and clinical data. In January 2023, FDA published new draft guidance intended to help sponsors identify an optimized dosage for oncology products. The February workshop aims to: discuss best practices and methods for evaluating all nonclinical and clinical data; incorporating modeling and simulation to identify optimized dosages; consider innovative clinical trial designs; and highlight ongoing efforts in academia, industry, and regulatory agencies. These changes may require a culture change in drug development and, in some cases, additional investments earlier in drug development. However, strategies to improve dosage selection hold great promise for improved  long-term patient outcomes.

We hope you can join us on February 15-16, please visit the workshop website for more information.

NEW Project Livin’ Label Episode 11 – fam-Trastuzumab Deruxtecan-nxki Available for Streaming

-Nicholas Warren, PhD

Project Livin’ Label is a collaboration between the U.S. Food and Drug Administration (FDA) and the American Association for Cancer Research (AACR) to help educate the cancer community about recently approved cancer therapies, and in particular, to help familiarize both providers and patients with complex product labels. Episode 11 – Tastuzumab Deruxtecan is now available. The episode details the science and the story behind developing the novel HER2-directed antibody and topoisomerase 1 inhibitor conjugate approved for treating metastatic HER2-positive and HER2-low breast cancer. All 11 Project Livin’ Label episodes are available for free on-demand streaming, provide 1.5 CME credits, and include perspectives from FDA, industry, academia, and patients.

FDA Finalizes Guidance to Optimize Regulatory Review of Cancer Therapies

-Rukiya Umoja, PharmD

On November 7, 2023, the U.S. Food and Drug Administration (FDA) finalized the Real Time Oncology Review (RTOR) Guidance for Industry. FDA’s Oncology Center of Excellence (OCE) in collaboration with the Office of Oncologic Diseases launched the RTOR pilot program in 2018, which allows FDA to evaluate individual sections of oncology drug applications that are most relevant to assessing a product’s safety and efficacy. The guidance is meant to streamline the review of cancer drugs in development that demonstrate substantial promise over current therapies and have simple study designs with easily interpretable endpoints.

In the final version, FDA clarified the eligibility and submission requirements, but did not expand RTOR to cover applications for cell and gene therapies. The agency also confirms that RTOR “does not alter the review performance goals and timelines associated with the applications, including as described in the Prescription Drug User Fee Amendments (PDUFA).” Although speedier review wasn’t a specific program goal, the average approval timeline for oncology products submitted under RTOR was 6 months vs. 6.5 months for other applications. As of November 2023, RTOR has supported the submission and review of 62 oncology drug approvals.

Given the challenges in cancer drug development, a flexible approach is often needed for drug approval. The finalized guidance is another signal to OCE’s commitment to enhancing the regulatory review of oncology therapies. ​As the RTOR pilot continues to evolve, it has the potential to expedite patient access to safe and effective cancer treatments, which continues to be an ongoing priority for the healthcare ecosystem.   

Decreased E-Cigarette Use Among Middle and High school Students Shows Positive Momentum Against Nicotine Addiction  

-Calais Prince, PhD 

Since 2014, e-cigarettes have been the most commonly used tobacco product among U.S. youth under the age of 18. Cooling flavors in e-cigarettes, synthetic cooling-fruit hybrids, and appealing packaging can potentially disrupt recent declines in e-cigarette use by young people. Although the 2023 National Youth Tobacco Use Survey indicated that current e-cigarette use among high school students decreased from 14 percent to 10 percent between 2022 and 2023, more than 89 percent of middle school and high school students who use e-cigarettes continue to use flavored types. Recent generations of e-cigarettes contain up to 60 mg/ml of nicotine in a form that increases nicotine delivery, similar to smoking cigarettes. This is concerning because nicotine is the main addictive component within tobacco; it is as reinforcing as heroin or cocaine; and young people who use e-cigarettes also smoke cigarettes. Under the Family Smoking Prevention and Tobacco Control Act of 2009, the U.S. Food and Drug Administration’s (FDA) Center for Tobacco Products (CTP) has the authority to regulate tobacco product manufacturing, marketing, and distribution. Tobacco product manufacturers cannot legally market or distribute new tobacco products, including e-cigarettes, unless the FDA evaluates Premarket Tobacco Product Applications (PMTAs) and conclude that the product is “appropriate for the protection of the public health.”   

As described in the AACR Cancer Progress Report 2023, the risk for nicotine addiction and tobacco product use is increased with e-cigarette use as it continues to be popular among populations 18 years old and younger. Additionally, the AACR and American Society of Clinical Oncology called for an immediate ban on all non-tobacco flavored electronic nicotine delivery systems that contain natural or synthetic nicotine in a joint policy statement. Decades of public health interventions that prevent tobacco related disease and death are at risk of being dismantled.  

The AACR will continue to support evidence-based regulatory and legislative efforts that decrease tobacco product use across all populations and demographics.  

White House Launches First-Ever Women’s Health Research Initiative

-Matthew Gontarchick

The Biden administration announced on November 13 the first-ever White House Initiative on Women’s Health Research, which will be led by First Lady Jill Biden and the White House Gender Policy Council. The initiative’s goal is to transform how the federal government approaches women’s health research and drive innovation.

Little is understood as to how different diseases and medical conditions disproportionately affect women. For example, women comprise two-thirds of Alzheimer’s disease and multiple sclerosis patients. Additionally, new research has found that pancreatic cancer rates are growing at a faster rate among younger women than among younger men.

This lack of understanding is likely attributable to years of underinvestment in women’s health research. While funding for the National Institutes of Health (NIH) has grown over the past eight consecutive years, just over 10 percent of this funding has been allocated to research into women’s health.

To address these disparities, the initiative will require the Biden administration to issue recommendations within 45 days on ways to improve women’s health care through prevention, diagnosis, and treatment. The initiative will also explore public-private partnerships and engage with external stakeholders on ways to drive advancements in women’s health research.

Available On-demand: 2023 AACR Virtual Patient Advocate Forums

The 2023 series of AACR Patient Advocate Forums are now available as on-demand recordings. Patient advocates and researchers from around the world join these free virtual events to learn the latest information on cancer research, new discoveries, and potential cancer treatments.