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Cancer Policy Monitor: August 2019


Register Today: Rally for Medical Research 2019

Registration is now open for the seventh annual Rally for Medical Research, which will be held Sept. 18-19, 2019, in Washington, D.C. Make plans to join us for this exciting event that brings together medical research advocates from across the country in support of NIH funding! For those who cannot participate in person, the online Rally National Day of Action will take place Sept. 19. Stay tuned to the Rally website for more info on the National Day of Action.

Learn more about sponsorship opportunities for the Rally for Medical Research.

Appropriations Update from Capitol Hill

-Brandon Leonard, MA

After months of negotiation, congressional leaders and administration officials reached a bipartisan, two-year deal to raise strict budget caps and lift the debt ceiling. The resulting legislation, the Bipartisan Budget Act of 2019, was passed by the House July 25, and by the Senate Aug. 1. It was subsequently signed into law by President Trump.

Under the budget agreement, the non-defense funding cap will be raised by $27 billion over current levels for fiscal year 2020, and the defense spending limit will increase by $22 billion. Each of those limits would be increased by an additional $2.5 billion for fiscal year 2021.

The budget deal marked a critical step in the process to fund the government for the coming fiscal year, which starts Oct. 1. Without an agreement, across-the-board cuts referred to as sequestration could have led to a 10 percent reduction in funds for the National Institutes of Health (NIH) and other agencies.

Congress will be in recess throughout August and the first week of September, but much work remains to complete the appropriations process upon their return. Based on the overall spending caps agreed to in the deal, Senate appropriations leaders will allocate funding to 12 subcommittees, who will in turn determine how funds are distributed among the various agencies in their jurisdiction.

Earlier this year, the House passed most of its spending bills, including the Labor-Health and Human Services-Education bill, which provided a $2 billion increase for the NIH. The House was basing its allocations on overall spending levels that it “deemed” prior to the budget deal, however, and must ultimately conform to the levels in that agreement. Once the Senate advances its appropriations bills, the House and Senate will send members to a conference committee to work out the differences in their bills.

This is a critical time for advocates of medical research to make their voices heard and urge their members of Congress to continue to make the NIH a top funding priority. The AACR joins the broader medical research advocacy community in urging Congress to adopt an increase of $2.5 billion for the NIH in FY2020, for a total funding level of $41.6 billion.

We encourage medical research advocates from across the country to join us at the 2019 Rally for Medical Research in Washington, D.C. Learn more information and register.

Public Health Groups Support Raising the Minimum Purchase Age of Tobacco Product to 21

-Nicole Boschi, PhD

Nearly all tobacco use begins in youth and young adulthood and 95 percent of adult smokers begin smoking before they turn 21. To prevent a lifelong addiction to nicotine, it is important that tobacco control policies are in place to make it harder for youth and young adults to access these harmful products. In 2015, the National Institute of Science, Engineering and Medicine concluded that raising the minimum purchase age of tobacco products in the United States, particularly from age 18 to 21 or 25, would likely lead to a substantial reduction in smoking prevalence. As public health officials have called the significant rise in the popularity of youth e-cigarette use an epidemic, the issue of raising the legal purchase age of tobacco products (including e-cigarettes) has attracted the attention of Congress.

Two major bipartisan Tobacco 21 bills were introduced in 2019. In April 2019, the Tobacco to 21 Act was introduced in both houses of Congress by Senators Brian Schatz (D-HI), Todd Young (R-IN), Dick Durbin (D-IL) and Mitt Romney (R-UT) and Reps. Diana DeGette (D-CO) and Chris Stewart (R-UT). In May 2019, the Tobacco-Free Youth Act was introduced in the Senate by Majority Leader Mitch McConnell (R-KY) and Sen. Tim Kaine (D-VA). These bills would raise the Federal tobacco purchase age to 21 and require states to file annual reports on enforcement efforts, such as inspections, or risk losing up to 10 percent of their federal grants to fight substance abuse and addiction.

At the June 12, 2019 AACR Congressional Briefing, E-Cigarettes and Nicotine Addiction: A Potential Public Health Crisis for Youth and Young Adults, co-sponsored by Senators Durbin and Kaine, Sen. Durbin referenced his legislation and stated that it is crucial to end the sale of tobacco products, including e-cigarettes, to anyone under 21 and to closely regulate these products. Similarly, Sen. Kaine remarked that although he and Majority Leader McConnell represent ideologies on opposite sides of the aisle, both Virginia and Kentucky have had long histories with tobacco use and anti-smoking measures are a bipartisan issue that brings both parties together.

The McConnell/Kaine bill was unanimously passed by the Senate Health, Education, Labor, Pensions (HELP) Committee as part of a larger health care package and will now go to the full Senate for a vote.

The AACR, along with other public health groups, strongly supports prohibiting the sale of tobacco products to individuals under the age of 21. However, these groups have cautioned that while raising the tobacco sale age to 21 is an important step, these bills should be a complement to, not a substitute for, other effective measures to reduce tobacco use. These measures include preventing youth access to flavored tobacco products that play a role in the initiation of tobacco use by youth.

Through the Tobacco Products and Cancer subcommittee, the AACR will continue to work with members of Congress and their staff and advocate for tobacco control policies that are based on the latest scientific evidence.

Patient Engagement in Cancer Research

-Carrie Treadwell, MBA

The value of patient engagement in medical research is no longer a philosophical ideology. There is now a collection of reports detailing process and system efficiencies that connect patient engagement to improved outcomes and lower costs.

Patient engagement is not a “one size fits all” activity or policy. It is a dynamic process, dependent on several factors, including the type of program, disease, institution, and desired outcome. How the concept of patient engagement translates into action is not always obvious, and what works for one study, program, PI, or patient may not work for another. Fortunately, there are a number of case studies emerging to help research and patient communities find a patient engagement practice or solution that works for them. Below is one such example.

AACR Scientist↔Survivor Program graduate, and breast cancer survivor, Robert Riter collaborated with Cornell University’s Department of Biomedical Sciences Professor, Robert Weiss to develop a training program for early-career cancer researchers that involved engaging with the patient community. They noted that Congressionally Mandated Medical Research Programs, the Patient-Centered Outcomes Research Institute (PCORI), and other funders require that scientists and patients serve together on research review panels. However, there is little training on how to communicate and work with the patient community. Anecdotal reports from both patients and researchers suggested that initial interactions with each other were occasionally awkward encounters.

Riter and Weiss proposed that it would be advantageous if patient-researcher interactions were moved upstream in the professional development of junior investigators. They created a curriculum that provides trainees with a broader understanding of cancer, beyond the fundamental biology and a deeper appreciation of the diversity of cancer diagnoses and patients with cancer. One trainee noted, Talking to patients changed my perspective about how we should pursue research and with what goals.? To learn more about Cornell’s new patient engagement program, read Cancer Research’s July 31, 2019, online article, Connecting Students with Patients and Survivors to Enhance Cancer Research Training. You can also learn more about Robert Riter’s cancer story in the winter 2018-2019 issue of Cancer Today.

For another example of patient engagement in research, tune in Aug. 14 at 4 p.m. ET, to hear thyroid cancer survivor, cancer scientist, and SSP alum, Dr. Aime Franco, in the first-of-its-kind virtual conference for cancer survivors: ShareTriumph.

AACR Announces Inaugural Science Policy Fellow

Tod Guidry, PhD, has been chosen as the inaugural AACR Science Policy Fellow. The fellowship is designed to provide early-career cancer researchers with experience drafting, analyzing, and implementing policy while rotating through various offices and agencies in the Washington, D.C., area.

Congratulations to AACR’s Fifth Cancer Health Disparities Scientist-Survivor Program Cohort

-Carrie Treadwell, MBA

We are pleased to congratulate and welcome the cancer survivors and advocates who have been selected to join AACR’s Fifth Scientist-Survivor Program (SSP) at the 12th AACR Conference on The Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved. Selected following a rigorous and competitive review process, the class of 20 cancer survivors will join community members and researchers from academia, government, and industry in San Francisco, California, Sept. 20-23, 2019, to address the disparities that represent a major public health problem in our country. The SSP 2019 Cancer Health Disparities cohort includes patient advocate leaders representing 12 cancer types, and 17 states in the U.S., as well as patient communities from Puerto Rico and Trinidad.

The National Cancer Institute (2018) defines cancer health disparities as adverse differences in cancer measures such as number of new cases, number of deaths, cancer-related health complications, survivorship and quality of life after cancer treatment, burden of cancer, or related health conditions, screening rates, and stage at diagnosis that exist among certain segments of the population.

There are many complex and interrelated factors that contribute to U.S. cancer health disparities, which makes it difficult to isolate and study the relative contribution of each. The factors may include, but are not limited to, differences and/or inequalities in: access and use of health care; genetics; physical and mental health; treatments received; social and economic status; exposure to environmental cancer risk factors; cultural beliefs; clinical trial participation; health literacy; and, lifestyle, including weight, diet, and physical activity (AACR Cancer Progress Report, 2018).

The focus of the 2019 SSP program is, “Implementing Change and Taking Action” with speakers highlighting programs designed and adopted to address a challenge that is contributing to a cancer health disparity at their institutions. In addition to educational and scientific sessions at the conference, SSP participants will have access to special sessions with the recipient of AACR’s Distinguished Lecture Award, presentations and panel discussions by researchers from the FDA, NCI, and academia; and, one-on-one mentoring with AACR scientists. Following the program, participating advocates are tasked with identifying, and proposing a way to operationalize, policy or programmatic solutions to a cancer health disparity challenge faced by their communities.

FDA and AACR Explore Uses of Real-world Evidence in Oncology

-Trevan Locke, PhD

On July 19, 2019, the U.S. Food and Drug Administration (FDA) and the AACR co-sponsored the FDA-AACR Real-world Evidence Workshop in Bethesda, Maryland. Including in-person and webcast, over 1,000 people joined in as experts from academia, industry, and FDA discussed the potential implications of real-world evidence (RWE) in oncology. The FDA-AACR Real-world Evidence Workshop was cochaired by Pallavi Mishra-Kalyani, PhD, team leader, Division of Biometrics V, Office of Biostatistics, Center for Drug Evaluation and Research, FDA; Deborah Schrag, MD, MPH, chief, Division of Population Sciences, Dana-Farber Cancer Institute; and Sean Khozin, MD, MPH, associate director, Oncology Center of Excellence; Director, Information Exchange and Data Transformation (INFORMED), FDA.

Workshop attendees examined pre- and post- market use cases in which RWE was utilized to improve and accelerate treatment development and patient care and considered the potential of large genomic databases as real-world data sources. Furthermore, presenters examined digital health initiatives and debated how these ongoing efforts to develop RWE could impact the oncology drug development landscape in the future.

A key and often-reiterated theme of the workshop was that real-world evidence should be considered a complement to, not a replacement for, traditional randomized clinical trials in regulatory decision-making. Materials from the workshop, including webcast recordings of each session, will be made available on the workshop webpage.

National Cancer Institute Convenes Experts and Seeks Stakeholder Input on the Childhood Cancer Data Initiative

-Audrey Jackson, PhD

In his 2019 State of the Union address, President Donald Trump pledged $500 million over 10 years to advance pediatric cancer research through a federal data-sharing initiative. In March 2019, Dr. Ned Sharpless, then director of the National Cancer Institute (NCI), announced the Childhood Cancer Data Initiative (CCDI), which focuses on the development of a framework to “collect, analyze, and share data to address the burden of cancer in children, adolescents, and young adults.” As NCI envisions and develops the CCDI, it has sought input through various avenues from pediatric cancer researchers, clinicians, data scientists, patients, families, advocates, and other funders. If you would like to share your ideas with NCI, you can submit through this website until Aug. 23, 2019.

From July 29-31, 2019, NCI convened 200 leaders in pediatric cancer and data-sharing for the CCDI Symposium. Experts defined the important clinical and research needs in pediatric cancer that can be overcome by data-sharing and considered the practical steps to creating a workable data-sharing framework.

Data-sharing is especially critical for tackling pediatric cancers because they are rare, affecting a total of 16,000 patients a year in the United States. Multiple speakers emphasized the need to share data and information across research groups, hospital networks, and national borders in order to advance science and patient care. Dr. Elaine Mardis, AACR president, spoke on the opening panel of the CCDI Symposium to share her experience and ideas on “Envisioning a National Childhood Cancer Data Initiative.” Dr. Mardis noted that there are challenges with modifying the dosage of therapies approved for adults for use in children, and clinicians are uncertain about options for treating recurrent pediatric cancer. Diagnosis based only on tissue pathology is also challenging, but many difficulties can be overcome with clinical genomic assays. Dr. Mardis shared an example of a case in which gene expression analysis demonstrated that a patient’s central nervous system malignancy was a second malignancy and not a recurrence, which guided diagnosis, prognosis, and treatment planning for the patient. During her tenure as AACR president, Dr. Mardis plans to highlight pediatric, adolescent, and young adult cancers. She will continue to speak about and encourage more sharing of genomic and clinical data to advance diagnosis and treatment for these young patients.

Other important needs in pediatric cancer and data-sharing surfaced throughout the CCDI Symposium panel discussions and breakout groups. Several speakers emphasized the need to integrate multiple types of data through this initiative, including genomics, proteomics, imaging data, pathology, and clinical data including side effects and patient and caregiver reported outcomes. Ideally, data should be longitudinal, appropriately de-identified, and accessible to researchers and other users who are not data experts. There is a significant need to improve the inclusion of under-represented populations and address health disparities in cancer outcomes. Privacy and ethical issues regarding data-sharing will need to be considered as governance frameworks are developed. Finally, there is a growing need for a workforce with appropriate skills in data science to create the infrastructure, analyze the data, and bridge the divide between clinicians, researchers, health systems technologists, and others involved in this complicated endeavor.

The consensus from NCI leaders and CCDI Symposium speakers was that the most appropriate approach to creating a data-sharing initiative is through a federated database that connects existing and new data repositories, allowing users to submit queries, search multiple databases, and receive results. Creating an interoperable data-sharing framework will not be a trivial challenge, and some of the discussion identified technical challenges that must be surpassed, such as cloud-based systems that are unable to communicate with each other. NCI Acting Director Dr. Douglas Lowy communicated that the initiative would focus initially on American datasets and expand to countries that are currently in the Children’s Oncology Group, with the potential of expanding globally in the future. Funding is critical to advance the initiative. If Congress does not appropriate the $50 million in annual funding, NCI leaders expressed commitment to the initiative but cautioned that progress would be slower. NCI continues to gather external input on the initiative through this website until Aug. 23, 2019, and is planning a post-symposium webinar in September 2019.

Elizabeth M. Jaffee, MD, FAACR, Presents AACR Science Series at FDA

-J. Tod Guidry, PhD

AACR Immediate Past President Elizabeth M. Jaffee, MD, FAACR, visited the U.S. Food and Drug Administration Oncology Center of Excellence, July 26, 2019, to present AACR Science Series at the FDA. Jaffee discussed scientific highlights from the 2019 AACR Annual Meeting, centered around central themes of precision medicine, global health, cancer health disparities, and convergence of science and technology. The AACR Science Series at the FDA was started in 2012 to foster relationships between FDA scientific review staff and external experts in cancer research.

Oncology Approval Recap: August 2019

In July, the U.S. Food and Drug Administration approved two novel cancer therapies, an expanded indication, and a biosimilar.

  • Selinexor, an inhibitor of XPO1, was granted accelerated approval in combination with dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody.
  • Darolutamide, an androgen receptor inhibitor, was approved for the treatment of patients with non-metastatic castration-resistant prostate cancer.
  • Pembrolizumab was approved for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (Combined Positive Score [CPS] ≥10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy
  • Rituximab-pvvr, a biosimilar referencing Rituxan, was approved for the treatment of adult patients with CD20-positive B-cell Non-Hodgkin’s lymphoma (NHL) to be used as a single agent or in combination with chemotherapy, and CD20-positive Chronic Lymphocytic Leukemia (CLL) in combination with chemotherapy.

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