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​​​​Cancer Policy Monitor: Sept. 5, 2017      

Key Policy Updates from Capitol Hill     

Childhood Cancer STAR Act – Advocacy Opportunity!

The AACR is joining with our partners in the Alliance for Childhood Cancer to advocate for passage of the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act of 2017. This is the most comprehensive childhood cancer bill ever taken up by Congress. It would expand opportunities for childhood cancer research, improve efforts to identify and track childhood cancer incidences, and enhance the quality of life for childhood cancer survivors. Learn more about the STAR Act.  

The STAR Act has strong support in both the House and the Senate (and passed the House last session before time ran out for the Senate to take it up). However, more Senate co-sponsors are needed to keep up the momentum and push this legislation onto Congress' busy calendar this year. You can help!  You can find out if your senator has signed on as a co-sponsor for S. 292 here, and you can find out if your representative in the House is a co-sponsor of H.R. 820 here. If they're not listed, please consider reaching out to them via phone, email or letter. For tools and tips on communicating with legislators, please see this page. If you tweet at your member of Congress, please include the hashtags #STARAct, #StepUp and #ChildhoodCancer.

Congress Passes Key FDA User Fee Bill

On Aug. 3, the Senate passed the FDA Authorization Act of 2017. The House had passed an identical bill July 12. The legislation reauthorizes the FDA to collect fees from drug and device manufacturers in order to hire the staff needed for review and approval of new products. As the Senate continued to debate health care reform, many worried that delays on the user fee bill would lead to layoffs at FDA, causing serious disruption to the pipeline of new drugs and devices under review. However, the bill was passed in time to avoid any pink slips being issued. Despite some reservations on certain points of the bill, President Trump signed it into law Aug. 18. 

RACE for Children Act Included in FDA Legislation

The FDA user fee legislation passed by Congress incorporated the Research to Accelerate Cures and Equity for Children Act, known as the RACE Act. Passage of the RACE Act was a hard-fought victory for childhood cancer advocates around the country. Under the provisions approved by Congress, companies developing cancer drugs will test the drugs in children when applicable, and pediatric oncologists will be able to study new cancer drugs, with industry funding. This addresses a critical gap in cancer research: there are currently hundreds of therapies in the pipeline to treat adults with cancer, but very few for children. RACE is an update to the Pediatric Research Equity Act (PREA) of 2003. Under PREA, drug companies have been required to develop drugs to treat diseases for children as well as adults. This was not applied to cancer, however, because cancers develop in different organs for children and adults. Under RACE, companies developing a cancer treatment for adults would also undertake PREA studies in children when the molecular target of the drug is relevant to a children's cancer. 

Appropriations Update

Members of the House and Senate left for August recess knowing that they have a lot on their plate in September. They must raise the debt ceiling by October in order to avoid a U.S. default on debt obligations. Congress must also pass a spending bill to fund the government by the end of September, or risk a government shutdown this fall. Congress is constrained by the Budget Control Act of 2011, which imposes spending caps on both defense and non-defense spending. To avoid sequestration, or across-the-board cuts, Congress will need to reach a bicameral budget agreement for FY 2018 and beyond that lifts the caps. This is very important if the NIH and NCI are to receive the robust funding increases necessary for sustained investment in cancer research. Without such an agreement, Congress will likely pass a short-term Continuing Resolution to fund the government at current levels. 

On July 19, The House Appropriations Committee approved an FY 2018 funding bill on Labor, Health and Human Services (HHS), Education and Related Agencies that includes a $1.1 billion funding increase for the National Institutes of Health (NIH). The Senate did not act on appropriations bills before the August recess; however, it is expected that the Senate Appropriations Committee will consider a bill including NIH funding in the first two weeks of September. This will be a critical time for advocates of medical research to engage with their members of Congress about the importance of NIH funding. 

UPDATE: Following a meeting with President Trump and Congressional leaders form both parties, Congress quickly passed and the President signed a legislative package that includes:

  • A Continuing Resolution (CR) funding the government at FY 2017 levels through December 8, 2017;
  • More than $15 billion in disaster relief;
  • A provision to suspend the debt ceiling; 
  • Language preventing the Department of Health and Human Services (HHS) from changing the levels of support from NIH for facilities and administrative (F&A) expenses.

Additionally, on September 7 the Senate Appropriations Committee approved its FY 2018 Labor-HHS-Education bill with a bipartisan vote of 29-2. If enacted, this would mark the third consecutive year that NIH has received an increase of $2 billion. This $2 billion increase includes the $496 million made available by the 21st Century Cures Act (Cures) in FY 2018 for targeted initiatives at the NIH, $300 million of which would go to the Beau Biden Cancer Moonshot as authorized by Cures. The bill also proposes a total funding level for the NCI of $5.858 billion for FY 2018, which is $168.9 million above current levels.

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Join the Rally for Medical Research National Day of Action      

The 2017 Rally for Medical Research is just around the corner!

Held every September, this Capitol Hill Day event continues the momentum established in 2013, and includes more than 300 national organizations united in support for medical research. The purpose of the rally is to call on our nation's policy makers to make funding for the National Institutes of Health (NIH) a national priority and raise awareness about the importance of continued investment in medical research that leads to MORE PROGRESS, MORE HOPE and MORE LIVES SAVED. 

We are pleased to have two influential members of Congress and champions for medical research attending the Rally events:

  • Senator Roy Blunt (R-MO), chairman of the Senate Labor-Health and Human Services-Education Appropriations Subcommittee, will speak at the Rally for Medical Research Reception Sept. 13.
  • Congressman Tom Cole (R-OK), chairman of the House Labor-Health and Human Services-Education Appropriations Subcommittee, will speak at the Rally for Medical Research Hill Day breakfast Sept. 14.
  • Additional members of Congress have been invited to attend and speak at the Rally Hill Day reception.

Can't come to Washington, D.C., but still want to support medical research on Sept. 14? Then join the National Day of Action! Start by joining our social media Thunderclap campaign with your Facebook, Twitter and/or Tumblr at any time between today and Sept. 13. Adding your voice means that our message in support of NIH and medical research will reach a broader audience! Then, Sept. 14, engage with your members of Congress via letters, calls, emails, Facebook and Twitter. Learn more and sign up for the National Day of Action here.

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Cancer Progress Report 2017 Congressional Briefing      

Date: Sept. 13, 2017. Time: noon - 1:30 p.m.

Location: Russell Senate Office Building, Room 485

RSVP. Download the Briefing Flyer.

The annual AACR Cancer Progress Report to Congress and the American public, now in its seventh edition, is a cornerstone of the efforts of the AACR to educate the public about cancer and the importance of biomedical research, as well as to advocate for increased federal funding for the NIH, NCI, and FDA. This year’s report chronicles how federally funded research continues to improve lives, and it shows that our ability to fully capitalize on our ever-growing knowledge of cancer is dependent on robust, sustained, and predictable federal funding. The report highlights how recent advances across the clinical cancer care continuum, in particular immunotherapy and molecularly-targeted therapies, are helping cancer patients and their families. This year’s report also focuses on the continued challenges we face in addressing cancer health disparities.

The American Association for Cancer Research is pleased to host this briefing that will highlight some of the exciting developments and advances in cancer research and illustrate the hope that these advances are bringing to cancer patients.  

Program:

Welcome

  • Margaret Foti, PhD, MD (hc), Chief Executive Officer of the AACR

Opening Remarks

  • The Honorable Charlie Dent, U.S. Representative for Pennsylvania’s 15th Congressional District (Invited)
  • The Honorable Jamie Raskin, U.S. Representative for Maryland’s 8th Congressional District (Invited)

Panelists
  • Michael A. Caligiuri, MD, President of the AACR and Chair, AACR Cancer Progress Report 2017; CEO and Director, James Cancer Hospital and Solove Research Institute; Professor of Internal Medicine, The Ohio State University Comprehensive Cancer Center, Columbus, Ohio
  •  John D. Carpten, PhD, Chair, Dept. of Translational Genomics, Keck School of Medicine, University of Southern California, Los Angeles, California
  • Carrie Best, Merkel cell carcinoma survivor, Plain City, Ohio
  • Evan Freiberg, MD, PhD, Leiomyosarcoma survivor, Englishtown, New Jersey
  • Teri Woodhull, Ovarian cancer survivor, Minnetonka, Minnesota

With special thanks to Senator Chris Van Hollen for assistance with logistics.

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Key Updates from the Food and Drug Administration    

FDA Oversight of Tobacco Products

In the previous issue of the Cancer Policy Monitor, we informed our readers about the U.S. Food and Drug Administration's (FDA) plans for a comprehensive regulatory plan for tobacco and nicotine regulation through a cohesive, agency-wide approach to nicotine. Since then, the New England Journal of Medicine published a perspective on A Nicotine-Focused Framework for Public Health by FDA Commissioner Scott Gottlieb, MD, and Center for Tobacco Products Director Mitchell Zeller, JD, outlining this plan and the effort to lower nicotine levels in combustible cigarettes to non-addictive levels.

In the article, Commissioner Gottlieb and Director Zeller discuss the potential for e-cigarettes to serve as smoking cessation devices, saying that "there are already products, such as electronic nicotine delivery systems, that could conceivably deliver nicotine without posing the dangers associated with tobacco combustion." They did acknowledge that there are currently opposing views on potential risks and benefits of e-cigarettes, adding that "we must continue to build on our understanding of the potential benefits for addicted cigarette smokers, in a properly regulated marketplace, of products capable of delivering nicotine without having to set tobacco on fire."

Last year, the AACR released a webinar titled "E-cigarettes: Research, Public Health Concerns, Opportunities, and Regulations," which you can view here.

FDA Approves First Gene Therapy in the U.S.

In other news, the FDA approved the first gene therapy in the United States, Kymriah (tisagenlecleucel), for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Elizabeth M. Jaffee, MD, president-elect of the AACR and deputy director​ of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University, was quoted in the Washington Post and Baltimore Sun stories about the FDA's approval of Kymriah.

Below is an excerpt from the Cancer Research Catalyst, the AACR's official blog, about this revolutionary treatment:

The CAR T-cell therapy in question is tisagenlecleucel (Kymriah). Known through most of its development as CTL019, tisagenlecleucel was approved for treating certain pediatric and young adult patients with acute lymphoblastic leukemia (ALL). Specifically, it was approved for treating children and young adults up to the age of 25 with B-cell ALL that is refractory, meaning that it has not responded to standard treatments, or has relapsed at least twice.

Tisagenlecleucel is a cell-based therapy that targets CD19-positive cells. Each patient receives a customized dose of tisagenlecleucel that is created using immune cells called T cells harvested from his or her blood. Once the T cells have been harvested, they are genetically modified to have a new gene that encodes a protein called a chimeric antigen receptor (CAR). After the T cells are modified, they are expanded in number and then infused back into the patient. The CAR directs the infused modified T cells to CD19-positive B cell ALL cells and triggers them to attack once they get there.

The approval of tisagenlecleucel was based on results from the phase II ELIANA clinical trial. According to the FDA statement, 83 percent of the 63 children and young adults who were treated with tisagenlecleucel had remission within three months of receiving the CAR T-cell therapy. Previously published data from earlier, smaller clinical trials suggest that for some patients, remission following tisagenlecleucel treatment is durable, but further follow-up is needed to determine long-term overall survival rates.

It is important to note, however, that treatment with tisagenlecleucel can cause severe adverse effects. One of the most concerning of these is cytokine-release syndrome, which is a systemic response to the activation and proliferation of the CAR T cells that causes high fever and flu-like symptoms. It can also cause life-threatening neurological events. Thus, the approval comes with a boxed warning for cytokine-release syndrome and neurological toxicities. In addition, the FDA expanded the approval of a medication called tocilizumab (Actemra) to include the treatment of CAR T cell–induced severe or life-threatening cytokine-release syndrome in patients ages 2 and older. This expanded approval was based on clinical trial data showing that 69 percent of patients treated with CAR T–cells had complete resolution of cytokine-release syndrome within two weeks following one or two doses of tocilizumab.

To further reduce risk to patients receiving tisagenlecleucel, the FDA has put in place a risk evaluation and mitigation strategy that requires that health care facilities using the new treatment be specially certified. As part of that certification, the health care facilities must have onsite, immediate access to tocilizumab and the staff involved in the prescribing, dispensing, or administering of tisagenlecleucel must be trained to recognize and manage cytokine-release syndrome and neurologic events.

Read the complete blog post.

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FDA-AACR Joint Workshop: Liquid Biopsies in Oncology Drug and Device Development    

Date: Oct. 10, 2017. Time: 8:30 a.m. - 5 p.m.

Location: Renaissance Downtown Hotel, 999 9th St NW, Washington, DC 20001

Registration is now open!

Purpose: As a noninvasive method to detect genetic alterations in tumors, analysis of tumor-derived cell-free DNA (cfDNA) in plasma holds much promise for improving cancer diagnosis and monitoring as well as drug development. This technology is advancing quickly, being incorporated into numerous drug development programs, and likely to be rapidly incorporated into clinical care.

Theme: Technology has evolved to enable the development of tests that can detect signs of cancer in blood and other bio-fluids. Although liquid biopsies are an exciting development, this technology presents with a set of unique regulatory concerns, particularly in establishing analytic and clinical validity. This session will build upon a July 2016 U.S. Food and Drug Administration-AACR co-sponsored workshop on liquid biopsies and will examine the regulatory challenges in adopting this technology for early detection, disease monitoring, and the potential use as surrogate end point markers for drug development.

SESSION I: Cancer Liquid Biopsies: State of the Science

SESSION II: Liquid Biopsies for Early Detection

SESSION III: Liquid Biopsies in Cancer Drug Development and Clinical Use

SESSION IV: Liquid Biopsy Test Development

See the agenda.

Workshop Co-chairs:

  • AACR:
    • Carlos L. Arteaga, MD, AACR President, 2014-2015; Director, Harold C. Simmons Comprehensive Cancer Ctr., UT Southwestern Medical Ctr.
    • Pasi A. Jänne, MD, PhD, AACR Regulatory Science and Policy Subcommittee member; Director, Lowe Center for Thoracic Oncology; Scientific Director, Belfer Institute for Applied Cancer Science; Senior Physician, Dana-Farber Cancer Institute; and Professor of Medicine, Harvard Medical School
  • FDA:
    • Julia Beaver, MD, Director (acting), Division of Oncology Products 1, Office of Hematology and Oncology (OHOP), Center for Drug Evaluation and Research (CDER), FDA
    • Gideon Blumenthal, MD, Deputy Director (acting), Office of Hematology and Oncology Products (OHOP), Center for Drug Evaluation and Research (CDER), FDA
    • Reena Philip, PhD, director, Division of Molecular Genetics and Pathology (DMGP), Office of In Vitro Diagnostics and Radiological Health (OIR), Center for Devices and Radiological Health (CDRH), FDA

Registration is now open!​ 

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Funding is Essential for FDA Oncology Center of Excellence    

Benjamin Wolfson is a PhD candidate in molecular medicine at the University of Maryland, Baltimore, focusing on the role of noncoding RNAs in the breast cancer microenvironment. Find more of his writing here and follow him on Twitter.

In early 2017, the Oncology Center of Excellence (OCE) was established by the U.S. Food and Drug Administration (FDA) to modernize the cancer drug approval process by increasing intra-agency collaboration and promoting novel regulatory ideas and processes. Among critical, medical innovation initiatives, the 21st Century Cures Act (Cures) directed the agency to create inter-center institutes in major disease areas that would accelerate FDA review by increasing agency interoperability. With oncology products representing 30 percent of drugs approved by the FDA, the OCE was established as the first such inter-center institute. Led by Richard Pazdur, MD, often referred to by the media as the "Cancer Czar," the OCE will employ an integrated approach to coordinate clinical review of oncology products while fostering an environment that supports innovation for novel regulatory ideas and processes. However, specific, sustained funding for the OCE has not been elucidated which has forestalled its ability to undertake the essential mission of translating science into new, safe and effective therapies for cancer patients.

The formation of the OCE was a necessary step for the FDA to function at its highest capabilities and provide the best available therapeutics to cancer patients in a timely manner. However, while "Cures" authorized the creation of inter-center institutes at the FDA including the OCE, specific funding for the center has not yet been appropriated. This is complicated by the fact that the center was established concurrently with the initiation of the Beau Biden Cancer Moonshot Initiative as a key partner to the NCI in the effort, so the specific source of funding is still being sorted out between Congress and the administration.

In the meantime, the FDA has allocated $3.5 million from existing resources to get the center off the ground. The AACR and other stakeholders have asked Congress to ensure that the OCE is fully funded in FY 2018. The AACR will work beyond the FY 2018 appropriations process to ensure the OCE receives the resources it needs to continue the fast pace of cutting edge research, oncology product review, and therapeutic development necessary for the millions of cancer patients nationwide.

Pazdur's vision for the OCE includes ensuring that perspectives of patients and patient advocacy groups are considered in regulatory decision-making through such efforts as the Patient-Focused Drug Development program. Beyond simply engaging in dialogue with patients and other stakeholders, the OCE will work to advance metrics of patient experience across all aspects of development, testing, approval, and clinical use of new oncology products. Further, the OCE seeks to modernize the eligibility criteria of cancer clinical trials by enrolling patients that reflect the real-world population including allowing more older adults to enroll.

Other aims of the OCE include encouraging novel clinical trial designs such as large, simple trials that use easily measurable endpoints and common control trials that share a control arm, but may involve multiple drugs for the same indication. The discovery of early disease biomarkers has greatly increased the capability of clinicians to specifically select clinical trial participants, and has allowed for broadening of clinical trials to explore multiple populations and dosing regimens. With improved, biomarker-based patient selection and novel trial designs, the OCE is already delivering on its promise with the first FDA approval for a tissue-agnostic cancer therapeutic occurring earlier this year.  

Lastly, the OCE intends to strive for "excellence" through several defined goals. The OCE will organize meetings and collaborative workshops to bring new, external voices to the regulatory process. They will prioritize communication with international regulatory agencies to foster regulatory cohesiveness and outreach beyond U.S. borders. By restructuring the oncology products review process, the OCE is expected to increase recruitment and retention of bright minds in cancer biology and related fields to the agency. To fulfill its goals and do so with excellence, the OCE must have expert human capital within the FDA.

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Share Your Cancer Research or Advocacy Story    

We’re looking for inspiring stories from researchers and advocates about why they became interested in cancer research or advocacy and how they work in their own way on behalf of cancer patients and survivors. You can submit your story starting today for a chance to be featured on our Take Action website and future editions of the Cancer Policy Monitor.

You can be as creative as you like. Write your story in your own words, make an online mini-site, create a video, or submit a digital collage. Any way you think would best tell your story is acceptable. You can submit your story and/or links here. There is no deadline to submit your story. We will be accepting stories throughout the year.

Ken Dutton-Regester, a melanoma researcher who serves as the chairperson of the AACR Associate Member Council, the leadership body of the associate members of the AACR, shared the story of his work. Ivy Elkins, a lung cancer advocate in the AACR Scientist↔Survivor Program, shared the story of how she became a lung cancer advocate. Kerie Berkowitz shared the story of how she survived ovarian cancer and became an advocate for survivorship issues.

Read more stories here and submit yours today!

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Join the AACR Cancer Action Alliance    

Launched in January 2012, the alliance leverages the strength of AACR members and interested nonmembers to build nationwide public and congressional support for cancer research and scientific progress. Alliance members are kept regularly informed about the news and events that affect cancer research and are alerted to opportunities to contact Congress or participate in other forms of outreach. Alliance members receive action alerts, timely legislative updates and other resources to facilitate their efforts.

With the Trump administration proposing deep cuts to the NIH budget, it is imperative that everyone who cares about defeating cancer speak out now and urge lawmakers not to turn their backs on the incredible scientific opportunities that are before us. Senators and representatives need to hear directly from residents in their district and/or state to understand what is at stake for their constituency, in terms of economics, health and competitiveness, if cancer research is not made a priority.

It is free and simple to join and your contact information will be used only to contact you about the AACR’s advocacy efforts and opportunities to take action.

Sign up here to receive action alerts and learn about opportunities to join other scientists, patients, survivors, and concerned citizens in calling on Congress to provide critical funding increases for the lifesaving cancer and biomedical research supported by NIH and NCI.

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