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Cancer Policy Monitor: October 2019

Cancer Policy Monitor Oct. 8, 2019

Appropriations Update from Capitol Hill

-J. Tod Guidry, PhD

With Congress unable to complete the appropriations process for Fiscal Year (FY) 2020 by the Sept. 30 deadline, legislators have passed a continuing resolution (CR) to maintain funding of federal agencies at FY 2019 levels. The CR, passed by Congress and signed by President Trump on Sept. 27, will fund the government through Nov. 21.

After funding amounts known as 302(b) allocations were set by the Senate Appropriations Committee on Sept. 12, the chamber’s 12 subcommittees were set to produce individual bills allocating funds to various government agencies. Senator Roy Blunt (R-MO), chairman of the Labor-HHS-Education Subcommittee, announced his intention to provide a $3 billion increase to funding for the National Institutes of Health (NIH), which would amount to a $42.1 billion total budget for FY 2020.

However, the appropriations process has been hindered by what some legislators are calling ‘poison pills,’ partisan policy riders slipped into appropriations bills. The Labor-HHS-Education bill markup originally scheduled for Sept. 12 was postponed over Republicans’ objection to a family planning-related amendment introduced by the subcommittee’s Ranking Member, Patty Murray (D-WA). Murray refutes the ‘poison pill’ claim, citing strong bipartisan support on the measure in the past.

Meanwhile, legislators in the House of Representatives are adjusting the appropriations bills they passed this summer to fit the new spending caps set in the Bipartisan Budget Act of 2019. The fate of the House-passed $2 billion increase to NIH funding is yet unknown, though House Appropriations Labor-HHS-Education Subcommittee Chairwoman Rosa DeLauro (D-CT) and Ranking Member Tom Cole (R-OK) have repeatedly expressed their strong support for an increase of at least this amount.

The appropriations process could further be hampered by the recently announced impeachment inquiry in the House, which Senate Appropriations Chairman Richard Shelby (R-AL) has said could result in additional short-term CRs.

While a CR prevents a government shutdown, it disrupts the enterprise of medical research by introducing uncertainty over funding levels. The AACR, along with many others in the broader medical research advocacy community, urges Congress to adopt an increase of at least $2.5 billion for the NIH in FY 2020 in an expedient manner.

On Sept. 19, the AACR coordinated the seventh annual Rally for Medical Research in partnership with more than 350 other organizations across the spectrum of medical research. Advocates including patients, caregivers, researchers, and health professionals met with nearly 200 congressional offices to urge continued robust funding increases for the NIH.

The AACR Supports the Trump Administration’s Move to Remove Flavored E-cigarettes from the Market

-J. Tod Guidry, PhD

In response to steady increased usage of e-cigarettes by youth, as well as an outbreak of vaping-related lung illnesses and deaths, the Trump administration in September announced its intention to remove non-tobacco-flavored e-cigarette products from the market until such products are approved by the U.S. Food and Drug Administration (FDA). The AACR has expressed strong support for the move.

The AACR has the responsibility to take a strong stand against the marketing and use of e-cigarettes, especially among youth and young adults,” said AACR President Elaine Mardis, PhD, co-executive director of the Institute for Genomic Medicine at Nationwide Children’s Hospital. The AACR also joined other public health organizations in sending letters to President and Mrs. Trump supporting the announcement.

The number of children using e-cigarettes has drastically increased over the past several years. From 2011 to 2015, the number of middle schoolers and high schoolers vaping rose by 900%. Preliminary data from the federal government’s 2019 National Youth Tobacco Survey (NYTS) show that 27.5% of high school students were users of e-cigarettes, compared to 20.8% in 2018. Public health officials attest that flavors are a primary attractant for teenagers to use e-cigarettes. According to the 2019 NYTS, two-thirds of high school consumers used fruit-flavored e-cigarettes and two-thirds used mint and menthol flavored e-cigarettes.

The Trump administration recently announced the FDA’s intention to formalize a compliance policy requiring removal of non-tobacco-flavored e-cigarettes from the market until manufacturers obtain premarket authorization from the agency. Until recently, the agency had exercised enforcement discretion, allowing e-cigarette products to remain on the market while giving manufacturers a shifting deadline to apply for FDA review. Though popularly called a “flavor ban,” Acting Commissioner of the FDA Ned Sharpless, MD, attests that the agency is simply enforcing regulatory action.

“The FDA is not banning flavors, as has been described-rather FDA is intending to enforce existing law against the marketing of such products,” he said during a recent hearing before the House Committee on Energy and Commerce. Manufacturers must submit applications for premarket review to the FDA no later than May 12, 2020. In the coming weeks, the FDA will release a guidance for implementation of its enforcement action on flavored e-cigarettes.

The rapid increase in the use of e-cigarettes by our nation’s youth and young adults has been an important issue for the AACR for some time now. On July 18, 2018, the AACR hosted a congressional briefing that examined the available science pertaining to the effects and content of e-cigarettes, while suggesting approaches to halting the concerning rise in youth vaping and nicotine addiction. More recently (on June 12, 2019), the AACR facilitated another congressional briefing around the concern that we are continuing to see such a rapid increase
in the use of e-cigarettes by our nation’s youth and young adults. This latest congressional briefing drew a standing room-only crowd, and speakers included leaders from the FDA, National Cancer Institute (NCI), U.S. Centers for Disease Control and Prevention (CDC), AACR, and Capitol Hill [Senators Dick Durbin (D-Illinois) and Tim Kaine (D-Virginia)]. Watch this special four-minute highlight video that was produced from the briefing.

The continued rise in vaping among American youth is now just one of two crises, as hundreds of vaping-related lung illnesses and several deaths have been reported since the spring of this year. Symptoms include nausea, vomiting, fatigue, fever, and difficult/painful breathing. The outbreak is particularly affecting young adults, with approximately half of all patients under the age of 25.

Three-quarters of patients afflicted by the mysterious lung illness have reported using e-cigarettes with tetrahydrocannabinol (THC), but some have reported vaping nicotine products alone. Neither nicotine nor THC has been ruled out as a causal factor in the reported lung illnesses. Some, but not all products linked to lung illness have also tested positive for pesticides and vitamin E oil. The chemicals in flavors may also be a contributing factor to the outbreak. A Harvard
published in 2016 confirmed that 47 out of 51 e-cigarette flavors tested contained chemicals previously associated with severe respiratory disease. According to the CDC, no specific product or substance has been linked to all cases.

While state and local health departments are working with the CDC and the FDA to investigate the outbreak of lung disease, state and local governments have taken actions to protect their populations from the dangers of e-cigarette use. The city of San Francisco led the way in June 2019, passing a city ordinance to ban sales of e-cigarettes. Early last month, Michigan was the first state to ban flavored e-cigarettes. More recently, Massachusetts governor Charlie Baker took the strictest action yet: effecting an immediate four-month ban on all vaping products, both in retail stores and online. More broadly, 12 states have raised the minimum age for purchase of tobacco products to 21 this past year alone, bringing the total to 18 states.

Bills aimed at raising the tobacco-purchasing age to 21 across all 50 states have been introduced in both chambers of Congress. This is a dynamic and rapidly changing area of government regulation and legislation, as local, state, and federal governments contend with the growing public health crisis created by e-cigarette products.

Stephen Hahn Likely to be Nominated to Lead FDA

-J Tod Guidry, PhD

On Oct. 1, it was reported that the Trump administration was set to nominate Stephen Hahn, MD, a radiation oncologist at MD Anderson Cancer Center, as commissioner of the U.S. Food and Drug Administration (FDA). The FDA has been under the leadership of Acting Commissioner Norman “Ned” Sharpless, MD, since April 5. But with federal law limiting acting officials in Senate-confirmed positions to a 210-day tenure, the administration is under pressure to choose someone to head the agency permanently by Nov. 1.

Hahn emerged as the frontrunner after it was announced in September that he and Alexa Kimball, MD, a professor of dermatology at Harvard Medical School, were under consideration along with Sharpless for the top job at the FDA. Hahn is chief of radiation oncology and chief medical executive at MD Anderson. Prior to joining MD Anderson in 2015, he chaired the Department of Radiation Oncology at the University of Pennsylvania School of Medicine for nine years and was also a senior investigator at the National Cancer Institute (NCI). As a member of the AACR, he has served on the AACR Radiation Science and Medicine Working Group Steering Committee and the Clinical and Translational Cancer Research Scientific Review Committee. Hahn was also cochair for the 2018 FDA-AACR-ASTRO Workshop on Clinical Development of Drug-Radiotherapy Combinations.

Sharpless’ short stint at the FDA has not been a quiet one, as he inherited the task of grappling with the youth vaping epidemic. Since taking the helm at the agency, an outbreak of vaping-related illnesses and deaths has increased pressure on Sharpless to enforce its regulatory authority over e-cigarette products. In early September, second ranking Senate Democrat Dick Durbin (D-IL) urged Sharpless to either take a stronger stance or step down. The FDA will formalize a policy in the coming weeks removing non-tobacco-flavored e-cigarettes from the market until manufacturers obtain premarket authorization from the FDA. The decision has been praised by public health advocates, though members of the House Committee on Energy and Commerce criticized Sharpless and the FDA for not acting sooner in a recent hearing.

Sharpless assumed the position of acting commissioner when former Commissioner Scott Gottlieb, MD, resigned. Prior to that he served as director of the NCI and was the director of the University of North Carolina Lineberger Comprehensive Cancer Center. He also previously served on the AACR Science Policy and Government Affairs Committee.

In addition to dealing with the public health crisis linked to e-cigarette use, the next FDA commissioner will also be responsible for spearheading ongoing initiatives to modernize the agency’s regulatory processes, leverage regulatory uses of real-world evidence and artificial intelligence, and better incorporate the patient experience into drug and product development.

Hundreds of Advocates Meet with Congress for the Rally for Medical Research Hill Day

-J. Tod Guidry, PhD

On Sept. 18-19, more than 350 organizations participated in the seventh Annual Rally for Medical Research in Washington, D.C. Advocates from 35 states as well as D.C. joined together to support robust, sustained, and predictable annual funding increases for the National Institutes of Health (NIH).

Advocates including patients, caregivers, researchers, and healthcare professionals attended an advocacy training event on Sept. 18 to learn about the appropriations process and effective ways of communicating with congressional offices. The training event was followed by a reception to celebrate medical research, with welcome remarks provided by AACR CEO Margaret Foti, PhD, MD (hc). Speakers at the reception included NIH Director Francis Collins and a number of congressional champions for medical research: House Labor-HHS-Education Appropriations Subcommittee Chairwoman Rosa DeLauro (D-CT), Congressman Jamie Raskin (D-MD), Congressman Peter King (R-NY), and Congresswoman Donna Shalala (D-FL).

On Sept. 19, Congressman Tom Cole (R-MO), ranking member of the Labor-HHS-Education Appropriations Subcommittee, provided remarks at a breakfast held for the rally participants. The breakfast also featured a tribute to Cokie Roberts, who passed away due to complications from breast cancer Sept. 17. Roberts served as a moderator during the first Annual Rally for Medical Research in 2013. The advocates then journeyed to Capitol Hill to meet with nearly 200 congressional offices. Rally participants asked members of Congress to support an increase of at least $2.5 billion for the NIH in Fiscal Year (FY) 2020, bringing the total NIH funding level to at least $41.6 billion.

In addition to all the advocates present on Capitol Hill, many more participated in the Rally National Day of Action, also held Sept. 19. Individuals and organizations across the country contacted their members of Congress via phone, email, and social media to urge their support for an increase of at least $2.5 billion for the NIH in FY 2020.

The Rally for Medical Research initiative was launched in April 2013 to bring together hundreds of national organizations to call on Congress to make the NIH a national priority. Through the annual Hill Day, the Rally for Medical Research continues to raise awareness about the critical need for an increased investment in the NIH to improve health, spur progress, inspire hope, and save more lives.

The brief video shown at the rally breakfast, along with photos from all
of the rally activities, can be found on the Rally website.

AACR Releases Cancer Progress Report 2019

-Brandon Leonard, MA

The AACR released its Cancer Progress Report 2019 Sept 18 at a congressional briefing in Washington, D.C. The report highlights how research largely supported by federal investments in the National Institutes of Health (NIH) is spurring improvements in public health and innovative breakthroughs across the spectrum of cancer care.

AACR CEO Margaret Foti, PhD, MD (hc) provided welcome remarks at the briefing and presented a video featuring highlights of the report. Panelists included AACR President Elaine R. Mardis, PhD, of Nationwide Children’s Hospital in Columbus, Ohio; Tari A. King, MD, of Dana Farber Cancer Institute/Brigham and Women’s Hospital in Boston; and three cancer survivors whose stories were included in the report: Keith Taggart, Eva Joseph, and Tomma Hargraves. Justin Goldberger spoke on behalf of Congressman Donald McEachin (D-VA), whose cancer story was also featured in the report.

The report, now in its ninth edition, is a cornerstone of the efforts of the AACR to educate policymakers and the broader public about cancer. It highlights the remarkable progress that we are making against cancer, and the importance of robust, sustained, and predictable annual funding increases for the NIH, NCI, FDA, and CDC. Specifically, the report underscores how recent advances across the clinical cancer care continuum, in particular immunotherapy and molecularly-targeted therapies, are helping cancer patients and their loved ones. This year’s report also focuses on the ways we are addressing the needs of the rapidly expanding population of cancer survivors, as well as the disparities that continue to exist in certain populations.

To read more about the report, please see the article in Cancer Research Catalyst, the official blog of the AACR. View the full report, along with videos featuring the cancer survivors who shared their stories.

Cancer Health Disparities Scientist↔Survivor
Program: Implementing Change

-Carrie Treadwell, MBA

From Sept. 20 – 23, more than 850 researchers, patients, and health professionals gathered in San Francisco, California, for the 12th AACR Conference on the Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved. Among them was a record number of patient advocates, passionately calling for all cancer patients to receive equal access to information, health care, and treatment options.

Twenty of the patient advocates were members of the distinguished Cancer Health Disparities Scientist↔Survivor Program (SSP). An elite training program that empowers cancer survivors and patient advocates to be change agents and tackle the inequities that lead to cancer health disparities. The 2019 program had a clear agenda, to move from passively cataloging cancer health disparities, to identifying actionable solutions.

SSP presentations, posters, and strategic planning sessions keenly focused on community-based patient-research partnerships that promise to change the trajectory of cancer health disparities. Attendees heard lectures from Tuskegee University’s Clayton C. Thomas, a professor in the Department of Biology and the Center for Cancer Research, and Nicole Gormley, clinical team leader in the Food & Drug Administration’s Division of Hematology Products. Both shared insights into enrollment disparities in multiple myeloma, prostate, and breast cancer clinical trials, as well as potential opportunities to close the gap. AACR Distinguished Lectureship on the Science of Cancer Health Disparities recipient, Steven R. Patierno, deputy director of the Duke University Medical Center, continued to stimulate discussion and encourage action with a keynote that addressed access inequities and the biological basis of cancer health disparities.

Each year, a high-point of the Cancer Health Disparities Scientist↔Survivor Program is the one-to-one mentoring sessions between advocates and field leaders. This year was no different. More than 80 meetings were scheduled, affording great interactions between patients and researchers, to discuss ways to implement or expand programs to improve outcomes for their communities.

Outside of SSP, the theme of patient advocacy, partnership, and empowerment continued to be highlighted throughout the conference. Kicking off the conference, patient advocate, conference cochair, and associate director of research and science at Huntsman Cancer Institute, Phyllis Nassi invited members of SSP to introduce themselves, “it is only fitting that we begin this meeting hearing from the powerful partners in our community who are impacted most by cancer-our patient community.” Read more about the opening ceremony in the blog, “It Doesn’t Have to Be This Way”: Targeting Cancer Health Disparities.

Sunday morning’s advocate session, Addressing Advocacy at the Bench: Implementing Change was standing room only. Attendees delved into the reality (and reasons behind it) that populations at risk for health disparities are less likely than other groups to participate in research studies. Esteemed panelists included Candace Henley, Blue Hat Foundation, SSP; Phyllis Nassi, Huntsman Cancer Institute, SSP; Nynikka Palmer, UCSF; Jamie Brewer, FDA; and, Lola Fashoyin-Aje, FDA. With great participation from the audience, the session highlighted case studies of community-based initiatives, co-created and implemented by teams of patients and researchers, working together to eliminate research and health disparities. Read more about the Advocate Session in the blog, How Can We Encourage Minorities to Join Cancer Clinical Trials’

Thank you to all of the patient advocates who attended this very important conference and shared their perspectives and insights on how to improve cancer health disparities. And, thank you to all of the Scientist↔Survivor Program participants for making the 2019 program such a success.

For more information on the 12th AACR Conference on Cancer Health Disparities, please visit the following:

Oncology Center of Excellence Announces International Collaboration for Simultaneous Review

-Trevan Locke, PhD

In September, the U.S. Food and Drug Administration Oncology Center of Excellence (OCE) announced Project Orbis. Through Project Orbis, the FDA, the Australian Therapeutic Goods Administration (TGA), and Health Canada worked together to review applications for two cancer drugs, leading to simultaneous decisions in all three countries.

Differences in regulatory standards and standards of care across the globe have led to national differences in time to approval for new cancer therapies. With oncology clinical trials becoming predominantly global endeavors, collaboration between regulatory agencies may help ensure that patients receive the best care and allow patients to receive earlier access to some therapies.

“We are pleased to be working alongside our Australian and Canadian colleagues to help make potentially life-changing treatments available to patients as quickly as possible while still ensuring the FDA’s high standards of safety and effectiveness,” said Acting FDA Commissioner Ned Sharpless, MD. “As Project Orbis expands, we look forward to welcoming additional international partners to collaborate with us in this important initiative as we work to help further serve the global patient community.”

Through this initial Project Orbis action, the three agencies granted accelerated approved (FDA), provisional approval with conditions of registration (TGA), and conditional approval with conditions (Health Canada) to lenvatinib in combination with pembrolizumab for the treatment of patients with advanced endometrial carcinoma that is not microsatellite instability-high or mismatch repair deficient, and who have disease progression following prior systemic therapy but are not candidates for curative surgery or radiation. Due to the unique regulatory requirements of each country, minor differences exist in their respective drug labels and the approved indication in Canada differs slightly from the other two countries. These small differences should have little impact on how the products are used. The review process also took advantage of Real-Time Oncology Review and Assessment Aid.

Since 2004, the FDA’s Office of Hematology and Oncology Products has held regular, confidential teleconferences with regulators across the globe to discuss topics related to applications under review. More recently, the FDA and China’s recently reformed National Medical Products Administration have started holding quarterly meetings to discuss general regulatory challenges facing global development of new therapies. Project Orbis represents a new frontier of international collaboration from the FDA.

“For humanitarian reasons, we want people with cancer anywhere in the world to have access to safe and effective therapies,” explained Richard Pazdur, MD, director of the OCE. Cancer clinical trials are predominantly conducted internationally. Greater uniformity of new standards of treatment may lead to oncology trials that are more broadly applicable around the world, and thus, more efficient drug development and earlier access to therapies for people with cancer in the U.S. and worldwide.”

Oncology Approval Recap

In September, the U.S. Food and Drug Administration approved three expanded indications for oncology therapies.

  • Pembrolizumab and lenvatinib were granted accelerated approval for the treatment of patients with advanced endometrial carcinoma that is not microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR) and who have disease progression following prior systemic therapy but are not candidates for curative surgery or radiation.
  • Apalutamide was approved for patients with metastatic castration-sensitive prostate cancer (mCSPC).
  • Daratumumab was approved for adult patients with multiple myeloma in combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant (ASCT).

To learn more about the approval of specific cancer therapies, you can find more information on the FDA’s website, and an AACR journal, Clinical Cancer Research, regularly publishes FDA approval summaries.