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Cancer Policy Monitor: April 13, 2021

Appropriations Update from Capitol Hill

-Marc Johnson, MPP

On March 11, 2021, President Biden signed into law the American Rescue Plan Act of 2021.  This $1.9 trillion relief package addressed unmet needs due to the COVID-19 pandemic, including financial support for small businesses, assistance for child care, housing and food assistance, and stimulus payments for the majority of American households.  In addition, the American Rescue Plan provided funding for vaccine distribution and administration, testing, and contact tracing, and included provisions on expanding health insurance coverage such as incentives for Medicaid expansion, increase in the FMAP, and no-cost coverage of COVID-19 vaccines and treatments for Medicaid and CHIP beneficiaries.  Click HERE to view a section by section summary of the bill.  Additional supplemental funding for the National Institutes of Health (NIH) was not directly included in the American Rescue Plan.

On April 9th, the Biden administration released its fiscal year (FY) 2022 discretionary funding request.  This top-line budget calls for a total of $131.7 billion in discretionary funding for the Department of Health and Human Services (HHS), a $25 billion increase from the FY 2021 enacted level.  This includes $51 billion for NIH, a $9 billion increase, which would include $6.5 billion for the establishment of an Advanced Research Projects Agency for Health (ARPA-H) within NIH.  Modeled after the Defense Advanced Research Projects Agency (DARPA), according to the Biden administration, ARPA-H, “…would drive transformational innovation in health research and speed application and implementation of health breakthroughs” related to cancer, diabetes, Alzheimer’s, and other diseases.  The Centers for Disease Control and Prevention (CDC) would receive $8.7 billion in discretionary funding, an increase of $1.6 billion. The Biden administration is expected to release its complete budget proposal later this spring. In the meantime, House and Senate appropriators are beginning to review the budget and appropriations request to prepare appropriations bills for consideration later this summer.

FDA-AACR Recommendations on Eliminating Racial Disparities in Multiple Myeloma

-Tod Guidry, PhD

In February 2020, the AACR collaborated with the U.S. Food and Drug Administration (FDA) to conduct the Workshop to Examine Under-representation of African Americans in Multiple Myeloma Clinical Trials. During the workshop, working groups consisting of researchers, physicians, patients, regulators, and others met to develop recommendations for improving the study and understanding of safety and efficacy of multiple myeloma therapies in the African American population. Upon receiving input from industry representatives and public workshop participants, those recommendations were recently published in an article in Blood Cancer Discovery.

Both incidence and mortality rates of multiple myeloma are more than twice as high among African Americans compared to whites. Despite comprising 13% of the United States population and 20% of individuals diagnosed with multiple myeloma, African Americans have historically comprised a mere 4.5% of participants enrolled in clinical trials for a new drug application or biological license application for multiple myeloma indications. Under-representation of African Americans in multiple myeloma clinical trials has serious implications for the applicability of safety and efficacy data to this subgroup, particularly given the fundamental differences in disease biology observed between African Americans and whites.

The published recommendations include those aiming to increase clinical trial representation, such as setting appropriate eligibility criteria, incorporation of diversity officers into study teams during trial design, and consideration of representation when selecting clinical trial sites. Working groups also produced recommendations toward eliminating racial disparities in the post-approval setting, as the safety and efficacy data for multiple myeloma therapies in African Americans generated in first-in-human and registrational trials are often insufficient. The working groups recommended the interrogation of racial differences in pharmacokinetics and pharmacodynamics through pilot studies and expansion cohorts, and pooling data across cooperative group studies to power safety and efficacy analyses among specific racial and ethnic groups.

Given the frequent under-representation of African Americans in trial cohorts, solutions to racial disparities in safety and efficacy data for multiple myeloma treatments may also lie outside the traditional clinical trial setting. Real-world studies utilizing data from electronic health records, disease registries, and claims data can also provide invaluable insight into product safety and efficacy by flagging potential differences in patient outcomes and side effects in African Americans.

The working groups also stressed the importance of stakeholder engagement to overcome barriers to clinical trial access. Engagement with patient advocate groups and outreach to specific social groups (e.g., churches) are critical to encouraging clinical trial participation and increasing enrollment. Stakeholder groups are also encouraged to discuss with Congress potential incentives that could be provided for conducting clinical trials prioritizing inclusion of underrepresented groups.

Importantly, the article highlights the fact that multiple myeloma is but one example of a disease that disproportionately impacts an underrepresented and underserved population. Together the published recommendations may not only serve to reduce racial disparities in multiple myeloma, but could also be generalized and applied to other groups and diseases, to improve understanding of a medical product’s performance in the populations in which it will be used.

Expanded Lung Cancer Screening Recommendation Will Help Find More Cancers Early

-Nicholas Warren, PhD

Lung cancer kills 135,000 Americans every year, more than any other cancer. Detecting lung cancer before it has spread throughout the body is critical for helping patients beat the disease; only 5.8 percent of patients diagnosed with late stage lung cancer survive more than 5 years, compared to 59 percent for early- stage lung cancer diagnoses. This is because lung cancer is easiest to treat at early stages, and in certain patients can be completely removed with surgery alone. Unfortunately, more than half of lung cancers are diagnosed at advanced stages, after they produce symptoms.

This March, the U.S. Preventive Services Task Force (USPSTF) expanded its recommendation for which Americans should be screened for lung cancer. Now, smokers as young as 50 years old and those who smoked a pack a day for 20 years are recommended to be screened with low-dose computed tomography annually. The previous recommendation only included current and former smokers aged 55-80 years old who smoked a pack a day for 30 years. The recommendation means insurance plans covered under the Affordable Care Act should now cover lung cancer screening for almost double the number of smokers.

When considering recommendations, the USPSTF balances the benefits of detecting aggressive cancers early with the risks of false positives, associated stress, and complications. Clinical trial results from 2020 found that screening reduced lung cancer deaths by 24 percent after 10 years among smokers in the expanded eligibility, thus providing rationale for the USPSTF’s recommendation. The expanded criteria especially benefit female and African American smokers. Women and African Americans smoke fewer cigarettes on average compared to White men, but appear to be at greater risk of lung cancer with lower smoking histories.

The AACR advocates for policies and organizes events to help address the public health burdens of tobacco. In case you missed it, be sure to check out the 2021 Tobacco Products and Cancer Policy Subcommittee Annual Meeting session “Issues Important to Tobacco Control.” The Annual Meeting content will remain available on-demand through mid-June. If you or a loved one need help quitting smoking, has free tobacco cessation resources.

Confidence in COVID-19 Vaccines is Growing, but There is More Work to Do

-Nicholas Warren, PhD

About one third of U.S. adults have received at least one dose of a COVID-19 vaccine, and millions more are vaccinated every day. While minor side effects are fairly common, particularly after a second dose, severe side effects seem to be exceedingly rare. The U.S. Centers for Disease Control and Prevention (CDC) reports there are fewer than 5 severe allergic reaction events per million doses (or less than 0.0005 percent), and there is no evidence of any deaths caused by COVID-19 vaccines in the U.S. Furthermore, the extremely high efficacy rates of vaccines have contributed to major declines in new COVID-19 cases since the January peak and bring hope of finally beating the pandemic. These are spectacular developments that have beaten even the most optimistic expert projections from 2020.

So far, demand for vaccines has far exceeded supply and left millions scrambling to figure out their priority level and navigate complicated registration processes. That may soon change. President Biden has called on states to open eligibility to all U.S adults by May 1 due to increasing supplies of vaccines. This shift may mean there will soon be more vaccine doses available than people who are willing to get vaccinated.

While confidence in vaccines has improved in the past few months, there remain millions of Americans who are skeptical. According to a February survey by the Kaiser Family Foundation, 55 percent of U.S. adults have either already been vaccinated or say they will get vaccinated as soon as possible, an increase from 47 percent in January and 34 percent in December. In comparison, 22 percent of respondents said they would “wait and see” how the vaccine works, and another 22 percent are opposed to being vaccinated. A separate survey by the Leukemia and Lymphoma Society found close to one third of patients with blood cancers are unlikely or unsure about getting vaccinated, despite a high risk of death from COVID-19.

The primary reasons for vaccine hesitancy vary greatly between different demographics. Common concerns include: the history of systemic racism in health care and unethical experimentation; the potential for severe side effects; rapid development process; inconsistent messaging from political officials; uncertainties about vaccine efficacy in patients undergoing cancer treatment, and; concerns about clinical trials including patients who represent themselves. Therefore, it is critical to listen to the concerns of specific communities and engage trusted leaders to communicate clear, consistent messages in order to build confidence.

Learning more about the vaccine development process is helpful to answer honest questions people may have. While many focus on the rapid development COVID-19 vaccines, decades of basic research that enabled such rapid development often goes unnoticed. Additionally, tens of thousands of volunteers were studied in clinical trials for at least two months before vaccines were authorized. Peer-reviewed clinical trial safety and efficacy results for the Pfizer/BioNTech and Moderna vaccines have been made freely available for anyone to read; as of writing, the Johnson and Johnson results have not yet been published by a peer-reviewed source, but efficacy and safety data can be found in the documents submitted to the U.S. Food and Drug Administration for review. Additionally, the National Comprehensive Cancer Network has provided guidance on the timing of when different patients with cancer should get vaccinated.

The AACR has led cancer community advocacy efforts to ensure patients with cancer are prioritized for COVID-19 vaccines. Additionally, be sure to check out the Annual Meeting session on “Building Vaccine Confidence.” Annual Meeting sessions will be available for on-demand viewing through mid-June.

Mark Your Calendars for the May 4 AACR Virtual Patient Advocate Forum on Immuno-Oncology

The rapid development of immuno-oncology therapies is transforming the cancer treatment landscape and bolstering hope for many cancer patients.  But why doesn’t it create a durable response for all patients with cancer? Join us on May 4 for a free patient advocate forum to explore this question and many others.

Learn More

Dialogues: Cancer Equality

Patients with cancer provide invaluable insight on current issues impacting the cancer research community. The AACR Patient Advocacy Program developed the interview series, Dialogues to showcase unscripted discussions between patient advocates, cancer survivors, researchers, and physicians on pressing topics faced by patient advocacy communities.

Dialogues: Cancer Equality, is a discussion between advocates Dr. Aime Franco, director of the Pediatric Thyroid Translational Research Laboratory at Children’s Hospital of Philadelphia and thyroid cancer survivor and Jonny Imerman, co-founder of Imerman Angels and testicular cancer survivor. The Dialogue explores the challenges of living with, and advocating for cancers that are deemed “treatable.”

AACR Early-career Hill Day Participants Reflect on Advocacy Experience

Dr. Katie Campbell, a postdoctoral fellow at UCLA, and Bernat Navarro Serer, a PhD candidate at Johns Hopkins School of Medicine, participated in the AACR 2021 Early-career Hill Day on February 25. Following the Hill Day, they co-authored a blog post for the Cancer Research Catalyst discussing their advocacy experience. Read the full post: [link to come]

Cancer Policy Perspectives: What we can learn about cancer drug development from the COVID-19 approach

This column highlights policy articles in the public press that are written by AACR members. This month’s submission “What we can learn about cancer drug development from the COVID-19 approach” provides a personal and instructive perspective to pursuing an approach to the development of therapies for cancer by Ethan Dmitrovsky, MD. 

Oncology Approval Recap

Between February 22 and March 21, the U.S. Food and Drug Administration approved two novel oncology therapies and new indications for three oncology drugs.

  • Cemiplimab-rwlc was approved for the first-line treatment of patients with advanced non-small cell lung cancer (NSCLC) (locally advanced who are not candidates for surgical resection or definitive chemoradiation or metastatic) whose tumors have high PD-L1 expression as determined by an FDA-approved test, with no EGFR, ALK or ROS1 aberrations.
  • Melphalan flufenamide was granted accelerated approval in combination with dexamethasone for adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody.
  • Lorlatinib was granted regular approval for patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive, detected by an FDA-approved test
  • Axicabtagene ciloleucel was granted accelerated approval for adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.
  • Tivozanib was approved for adult patients with relapsed or refractory advanced renal cell carcinoma (RCC) following two or more prior systemic therapies.

Read more about FDA approvals on the AACR webpage and on the AACR Cancer Research Catalyst. To learn more about the approval of other cancer therapies, you can find more information on the FDA’s website and an AACR journal, Clinical Cancer Researchregularly publishes FDA approval summaries.