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Cancer Policy Monitor: June 2019

Cancer Policy Monitor – June 11, 2019

RSVP Today for AACR June 12 E-cigarette Briefing

On June 12, 2019, the AACR will host a briefing, E-cigarettes and Nicotine Addiction: A Potential Public Health Crisis for Youth and Young Adults, to educate congressional staff and the public on the potential public health crisis of e-cigarette use and nicotine addiction in youth and young adults. Learn more.

The AACR, Moffitt Cancer Institute, and Biden Cancer Initiative will host a briefing titled Let’s End HPV-related Cancers,June 27, in partnership with American Cancer Society Cancer Action Network (ACS CAN), American Society of Clinical Oncology (ASCO), Association of American Cancer Institutes (AACI), Prevent Cancer Foundation, St. Jude Children’s Research Hospital, and the Union for International Cancer Control (UICC).

A panel of expert speakers will discuss efforts to eliminate cancers caused by the Human Papillomavirus (HPV) through vaccination, screening and treatment. With panelists representing a diverse range of experience in domestic and global health, the briefing will highlight current activities and provide a call to action for all stakeholders.

The briefing will take place from noon – 1:30 p.m. in Rayburn House Office Building, Room 2044, on Capitol Hill. Register for this briefing.

Gilbert S. Omenn, MD, PhD, Director, Center for Computational Medicine and Bioinformatics, University of Michigan; Chairman, Science Policy Subcommittee, American Association for Cancer Research

Keynote Speaker:
Anna R. Giuliano, PhD, Founding Director, Center for Immunization and Infection Research in Cancer, Moffitt Cancer Center

John T. Schiller, PhD, Deputy Chief, Laboratory of Cellular Oncology, Center for Cancer Research, National Cancer Institute

Melinda Wharton, MD, MPH, Director, Immunization Services Division, National Center for Immunization and Respiratory Diseases, Centers for Disease Control and Prevention

Vicki Benard, PhD, Chief, Cancer Surveillance Branch, Division of Cancer Prevention and Control, Centers for Disease Control and Prevention

Jason Mendelsohn, Patient Advocate,

June is National Cancer Survivor Month

Thanks to advances in cancer research and policy, more than 16.9 million people in the United States are cancer survivors. Join the American Association for Cancer Research (AACR) in support of this global awareness month celebrating survivors who have fought the disease and those currently in treatment.

Take Action

Appropriations Update from Capitol Hill

-Brandon Leonard, MA

House and Senate leaders are continuing negotiations with the White House to reach a budget deal that will raise strict caps on spending for both defense and non-defense agencies. A previous round of talks did not yield a result, but leaders from both parties remain optimistic that a deal can be agreed on soon.

At the same time, appropriators are moving ahead with fiscal year (FY) 2020 spending bills even in the absence of a budget deal. House Democrats have made plans to bring a package of spending bills to the House floor for a vote in June. This “minibus” is expected to bundle five bills that have passed the House Appropriations Committee, including the Labor-Health and Human Services (HHS)-Education bill. That measure includes a $2 billion increase for the National Institutes of Health (NIH) for FY 2020. Last year, both the House and Senate similarly packaged multiple appropriations bills together in an effort to garner more support from members of both parties.

Senate appropriators had hoped to wait until a budget deal was reached before writing their spending bills, but in the meantime, they may move forward as their House colleagues have done using assumed topline amounts that will ultimately need to be adjusted. The House and Senate would need to reconcile the amounts passed by each chamber.

The AACR continues to advocate for a prompt, bipartisan budget deal to lift the spending caps. We also join the broader medical research advocacy community in urging Congress to adopt an increase of $2.5 billion for the NIH in (FY) 2020, for a total funding level of $41.6 billion.

Public Health Groups Support Tobacco 21 Legislation but Raise Concerns on Potential Harmful Consequences

-Nicole Boschi, PhD

The AACR, along with other public health groups, strongly supports prohibiting the sale of tobacco products to individuals under the age of 21. Nearly all tobacco use begins in youth and young adulthood and 95 percent of adult smokers begin smoking before they turn 21. Tobacco companies spend $9.4 billion per year to market their products to this age group. In 2015, the National Academy of Medicine concluded that increasing the age of sale for tobacco products to 21 would, over time, significantly reduce the number of youth and young adults who start smoking and thereby decrease the number of smoking-related deaths.

Currently, there are two major bipartisan Tobacco 21 bills moving through Congress. On May 20, Senate Majority Leader McConnell (R-KY) and Senator Kaine (D-VA) introduced The Tobacco-Free Youth Act. Earlier, Senators Schatz (D-HI), Young (R-IN), Durbin (D-IL), and Romney (R-UT) introduced the Tobacco to 21 Act. The Tobacco to 21 Act also has a companion bill in the House of Representatives introduced by Representatives DeGette (D-CO), Stewart (R-UT), Cohen (D-TN), Herrera Beutler (R-WA), Payne (D-NJ), Flores (R-TX), and Wittman (R-VA).

While there is strong support for raising the sale age of tobacco products to 21, there is some concern regarding detrimental consequences of these bills. The bill introduced by Senators McConnell and Kaine contains provisions that would require each state to pass a law raising the tobacco purchase age to 21 or lose federal substance abuse grant funding. During the crafting of state legislation, it is possible that tobacco companies would lobby states to include special interest provisions, such as blocking localities from prohibiting the sale of flavored tobacco products or inserting weak enforcement actions into bills. For example, in Arkansas, a bill prevents cities and counties from enacting bans on e-cigarette flavors, tougher enforcement, and raising the tobacco purchase age higher than that of the state. A Virginia law, set to take effect in July 2019, did not provide funds for enforcement and would penalize youth buying cigarettes, some groups have argued that this could increase racial profiling of these youth.

Public health 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.

The AACR will continue to closely monitor these and similar bills and will work with Congress and other organizations to ensure that the public health goals of these bills are met while avoiding the influence of tobacco industry provisions that may weaken regulation of tobacco products.

The AACR is working with members of Congress to prevent youth uptake of tobacco products. On June 12, the AACR will host a briefing to educate congressional staff and the public on the potential public health crisis of e-cigarette use and nicotine addiction in youth and young adults.

Registration Now Open for FDA-AACR Real-world Evidence Workshop

-Elizabeth Barksdale, PhD

The American Association for Cancer Research (AACR) is partnering with the U.S. Food and Drug Administration (FDA) to present the Real-world Evidence Workshop, July 19, 2019, in Bethesda, Maryland. Real-world evidence (RWE) is clinical evidence generated from datasets including electronic health records, administrative claims data, patient-reported data, and genomics/biomarker data when collected in the routine provision of care. RWE has been lauded for its broad potential applications to improve clinical care, accelerate research, and support regulatory decision making.

Regulatory-grade RWE use cases span the drug development life cycle, including early clinical development, clinical trials, and post-marketing and commercialization. To fulfill the congressional mandate to further advance RWE, the FDA recently released the Framework for FDA’s Real-world Evidence Program.

This FDA-AACR workshop will discuss the agency’s work thus far on the implementation of that framework while exploring the current state of RWE in oncology. Speakers at the workshop will examine pre- and post- market use cases in which RWE was utilized to improve and accelerate treatment development and patient care and explore the potential of large genomic databases as real-world data sources. This event will also look to the future of RWE and consider how ongoing efforts will impact the oncology drug development landscape. See the preliminary agenda.

The FDA-AACR Real-world Evidence Workshop is being organized by:

  • Sean Khozin, MD, MPH, Associate Director, Oncology Center of Excellence, Director, Information Exchange and Data Transformation (INFORMED), FDA;
  • Pallavi Mishra-Kalyani, PhD, Team Leader, Division of Biometrics V, Office of Biostatistics, Center for Drug Evaluation and Research, FDA; and
  • Deborah Schrag, MD, MPH, Chief, Division of Population Sciences, Dana-Farber Cancer Institute.

Registration for this one-day workshop is free and open to the public.

Breast Cancer Approval Highlights Potential Role for Real-world Evidence in Drug Development

-Beth Oates

Beth Oates is a PhD candidate in the department of Microbiology, Immunology and Molecular Genetics at the University of Kentucky, College of Medicine, Lexington, Kentucky. Her research is focused on understanding bacterial competition and community interactions and the resulting effects on pathogenicity. Beth is also interested in the influence of disease on science and public health policy.

In December 2018, the U.S. Food and Drug Administration (FDA) published the Framework for FDA’s Real-World Evidence Program. The framework lays out how FDA is considering real-world evidence (RWE) to inform regulatory decision-making for drug and biologic development. The FDA’s RWE program evaluates the potential use of real-world data (RWD) collected in clinical practice to generate RWE to support changes in drug labeling such as expanding an indication to include a new population of patients or modifying instructions for administration. By utilizing RWE, the FDA may be able to expand the use of approved medicines to treat more individuals when appropriate.

One example of this is the recent approval of palbociclib (Ibrance). Palbociclib was initially approved in 2015 for postmenopausal women with estrogen receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative, metastatic breast cancer who have not received an endocrine-based therapy. Its indication was extended to include women with hormone receptor (HR)-positive, HER2-negative, advanced or metastatic breast cancer when used in combination with the hormone therapy fulvestrant or an aromatase inhibitor (in 2016 and 2017, respectively). These approvals were based on safety and efficacy data from nearly 1000 women in the PALOMA-2 and -3 clinical trials.

In April, FDA approved palbociclib in combination with fulvestrant or an aromatase inhibitor to treat men with HR-positive, HER2-negative, advanced or metastatic breast cancer. Unlike the previous approvals in women, this one was based on evidence gathered from electronic health records and post-marketing reports of male patients treated off-label with palbociclib.

Whereas breast cancer in women is the second most common type of cancer in the U.S., breast cancer in men is relatively rare. In 2019, it is estimated that there will be 2,670 new cases of breast cancer diagnosed in males in the U.S. and 500 deaths. These numbers represent about one percent of the total predicted breast cancer diagnoses and deaths in the U.S. Because of the relative rarity, most clinical trials of new breast cancer therapies, including the PALOMA trials, enroll few or no men resulting in limited clinical data to guide treatment decisions.

Without data from males in the PALOMA trials, Pfizer partnered with Flatiron Health to identify and analyze RWD used to support the claim that palbociclib was safe and effective in men with breast cancer. Drawing from de-identified electronic health records and postmarketing reports, they evaluated baseline characteristics, treatment patterns, and clinical outcomes for men with HR-positive, HER2-negative, advanced or metastatic breast cancer. Based on tumor responses and adverse event reporting, it was determined that the safety profile for men is consistent with that of women treated with palbociclib. FDA approved palbociclib for use in men based on RWE generated from these data.

“Men with breast cancer have limited treatment options, making access to medicines such as Ibrance critically important,” said Bret Miller, founder of the Male Breast Cancer Coalition. “We applaud the use of real-world data, a new approach to drug review, to make Ibrance available to certain men with metastatic breast cancer and help address an unmet need for these patients.”

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.

Apply Today: Scientist↔Survivor Program at the Science of Cancer Health Disparities Conference

The 12th AACR Conference on The Science of Cancer Health Disparities in Racial/Ethnic Minorities and the Medically Underserved, to be held Sept. 20-23, 2019, will address all levels of basic, population, clinical, and transdisciplinary research related to cancer.

The AACR Science of Cancer Health Disparities conferences advance the understanding of, and ultimately help to eliminate, the disparities that represent a major public health problem in our country. Reflecting this transdisciplinary field, professionals from academia, industry, government, and the community are brought together to promote the exchange of novel ideas, discuss the latest findings in the field, and stimulate the development of new research on cancer health disparities.

Patient advocates who participate in the Scientist↔Survivor Program at the Science of Cancer Health Disparities Conference will:

  • Discuss relevant and timely cancer topics with scientific mentors during small group meetings and roundtable discussions;
  • Attend scientific talks at the Cancer Health Disparities Conference;
  • Present at the poster session;
  • Communicate to scientists the key issues, questions, and concerns of the survivor and patient advocacy communities; and
  • Network with scientists and fellow advocates from local, national, and international cancer organizations.

Scientist↔Survivor Program participants are selected through a competitive process. Applications are due June 19, 2019. Patient advocates are encouraged to apply to the Science of Cancer Health Disparities Conference Scientist↔Survivor Program if they:

  • Actively support cancer research through advocacy and/or community outreach;
  • Have a constituency with which to share the knowledge they gain through the program;
  • Can participate in all components of the four-day program in San Francisco, California.

Learn more and apply here.

FDA Seeks to Improve Expanded Access to Investigational Oncology Drugs with Project Facilitate

-Elizabeth Barksdale, PhD

On May 16, the U.S. Food and Drug Administration (FDA) Oncology Center of Excellence (OCE) hosted a public meeting with the Reagan-Udall Foundation to formally introduce Project Facilitate. Project Facilitate is a pilot project for the agency to streamline and increase access to the FDA’s Expanded Access (EA) program, specifically focused on oncology drugs and biologics. Under Project Facilitate, FDA establishes a single point of contact, a call center, through which physicians can initiate and/or get help completing single-patient investigational new drug (SPI) requests. (SPI is the mechanism by which physicians secure the FDA’s approval to treat individual patients with unapproved investigational drugs.) Trained FDA staff will guide callers through the request process, assisting with the necessary paperwork and identifying contacts at pharmaceutical companies and IRBs.

OCE Director Rick Pazdur, MD, came up with the idea for Project Facilitate after receiving questions from cancer center directors and National Cancer Institute officials about navigating EA and realizing that community oncologists must be even more confused. At the meeting, Pazdur described his vision of a “concierge service” for physicians wanting to file a SPI, and his hope is that this will open up the EA process to patients who may not normally receive investigational medicines thereby increasing equity of access.

Physicians must still complete all steps in the established EA process, including seeking a Letter of Authorization (LOA) from the drug sponsor. Because Project Facilitate staff will be copied on LOA requests, the FDA will, for the first time, be able to collect data on demand for investigational drugs. Currently, FDA only becomes aware of expanded access requests if they are approved by drug companies. Sponsors will still have discretion as to whether to provide requested drugs, but for requests initiated through Project Facilitate, they must respond in a specific amount of time and provide the reason(s) for denying access.

Another benefit expected of the program will be collection of outcomes data. Project Facilitate staff will follow up with physicians whose patients received experimental treatments to request summary reports of benefits and adverse events. However, FDA is wary of adding administrative burden to already-busy oncologists and is working to determine how much and how often data will be collected.

Feedback from the oncology community is welcome as FDA works to make Project Facilitate, and EA in general, more inclusive and useful. With a tentative launch date in late May 2019, the pilot program will evolve as stakeholders communicate with the agency about shortcomings and areas of strength.

ODAC Considers Two Therapies

-Trevan Locke, PhD

In May, the Oncologic Drugs Advisory Committee (ODAC) met to consider two investigational drugs, pexidartinib and quizartinib. Pexidartinib is a small tyrosine kinase inhibitor being investigated for the treatment of tenosynovial giant cell tumor (TCGT). Quizartinib is being investigated for the treatment of relapsed or refractory acute myeloid leukemia (AML). The U.S. Food and Drug Administration (FDA) had concerns about the benefit-risk profile for both of these therapies.

TCGT is a benign, nonfatal disease, but the tumors can have a dramatic impact on quality of life. Many patients with this disease can be treated with surgery, but a subset of patients with diffuse disease need a new option. Pexidartinib is intended to treat these patients, and the ENLIVEN trial showed a significant and large overall response rate as assessed by tumor size. However, assessments used to measure clinical outcomes in that trial had a large amount of uncollected data, leading to uncertainty around the clinical benefit of treatment. Also, high rate of liver toxicities raised concerns at the FDA. Considering the totality of the evidence, the ODAC voted 12-3 that pexidartinib’s demonstrated benefit outweighed the risks of the drug for the proposed indication.

Quizartinib showed a statistically significant increase in overall survival for patients with relapsed or refractory AML in a single trial. However, the OS increase was six weeks and secondary measures of survival did not confirm a significant effect. Additionally, the FDA had concerns about censoring and treatment imbalances between arms of the trial and toxicities from the drug, primarily cardiotoxicity. The imbalance called into question whether the survival advantage was real. While almost every committee member acknowledged that it provided some benefit in getting patients to blood marrow stem cell transplant, the committee voted 8-3 that the presented trial did not demonstrate that treatment with quizartinib provided a benefit that outweighed safety risks.

The FDA is not bound to follow the results of ODAC votes when making approval decisions, but often does. Decision deadlines for both of the therapies are scheduled for August 2019.

Oncology Approval Recap

Whenever the U.S. Food and Drug Administration approves new or expanded indications for oncology drugs or biologics, you can depend on Cancer Research Catalyst to cover it in detail. Cancer Research Catalyst is the official blog of the American Association for Cancer Research. This month Karen Honey, PhD, discussed the approvals of atezolizumab for certain patients with small-cell lung cancer, erdafitinib for certain patients with bladder cancer, and pembrolizumab for treating certain patients with kidney cancer. To learn more about these recent approvals, read her article, “FDA Approvals Provide New Advances Against Bladder, Lung, and Kidney Cancer.