Cancer Policy Monitor - Dec. 11, 2018​

AACR Mourns the Loss of George H.W. Bush, the 41st President of the United States

The AACR issued a statement mourning the loss of former President George H.W. Bush, who passed away on Nov. 30 at the age of 94. President Bush and his wife, Barbara, were actively involved in many efforts to support cancer research. Read the AACR’s statement.

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Mid-term Elections Bring Big Changes to Congress

By Mary Lee Watts, MPH, RD

The results of the November mid-term elections will bring significant changes to Congress in January. When the 116th Congress is sworn in Jan. 3, 2019, the Democrats will control the House of Representatives, and there will be many new faces on both sides of the "aisle." This year's elections bring a record number of women to Congress, who now represent about 20 percent in both chambers. Congress also will be the most diverse it has ever been in terms of racial, ethnic and other minority group representation.

Following the November elections, which resulted in the Democrats winning a majority and thus control of the House of Representatives, there are multiple committees relevant to the NIH and FDA that will have new leadership, in addition to new members. In the Senate, the Republicans will remain in the majority and, therefore, we can anticipate less change in Committee leadership. 

The committees with the greatest influence over cancer research funding and other issues of importance to the AACR are the House and Senate Appropriations Committees, the House Energy and Commerce Committee, and the Senate Health, Education, Labor and Pensions (HELP) Committee. In the House, Rep. Nita Lowey (D-NY) is expected to chair the House Appropriations Committee, with Republican Rep. Kay Granger (R-TX) becoming the Ranking Member for the minority. On the House Energy and Commerce Committee, which has authorizing jurisdiction over the NIH and the FDA, Rep. Frank Pallone (D-NJ) will be the chair, and Rep. Greg Walden (R-OR) will likely serve as ranking member.

Rep. Rosa DeLauro (D-CT) is expected to become chair of the House Appropriations Subcommittee on Labor, Health and Human Services (HHS),Education, and Rep. Sanford Bishop (D-GA) will serve as chair of the House Appropriations Subcommittee on Agriculture, Rural Development and the Food and Drug Administration (FDA). While DeLauro has been a staunch supporter of NIH for many years, we can expect that there will be a broader range of health, education and labor priorities under Democrats than we saw under Republican leadership.

In the Senate, things are expected to stay the same with Sen. Roy Blunt (R-MO) and his Democratic counterpart Sen. Patty Murray (D-WA) continuing in their roles as chair and ranking member of the Senate Appropriations Subcommittee on Labor, HHS, Education. Both members have been stalwart champions of NIH funding and we expect that strong, bipartisan support to continue. Likewise, Sen. Lamar Alexander (R-TN) will continue to chair the Senate HELP Committee, with Sen. Murray remaining as ranking member on that committee.

Although the bipartisan support for cancer research is expected to continue in 2019, there are challenges ahead. Strict caps on defense and nondefense spending required by the Budget Control Act will go back into effect in fiscal year 2020, unless Congress reaches another bipartisan budget agreement to lift them. A budget agreement followed by a larger allocation for appropriations bills, including the Labor, HHS and Education bill, will be required if Congress wants to continue to increase funding for priority areas like the NIH next year.  Further complicating matters will be pressure to limit overall federal expenditures and address the federal deficit, which the Congressional Budget Office has reported has dramatically increased over the past year.

As the 116th Congress gets underway, the AACR will urge Congress and the White House to reach a bipartisan budget agreement. We also will continue to make the case to Congress that robust, sustained, and predictable funding increases for the NIH are essential to the health and economic security of our country.

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FDA Moves to Restrict Youth Access to Flavored Tobacco Products

By Nicole Boschi, PhD

On Nov. 17, 2018, U.S. Food and Drug (FDA) Commissioner, Scott Gottlieb, MD, announced new actions aimed at addressing the alarming rise in youth nicotine use. This comprehensive plan seeks to employ a multipronged approach to ameliorate this growing epidemic. The FDA's comprehensive plan will involve: 1) imposing sales restrictions on flavored e-cigarettes, 2) banning menthol cigarettes, and 3) banning flavored cigars. The FDA's plan was announced the same day that the FDA and U.S. Centers for Disease Control and Prevention (CDC) released the 2018 National Youth Tobacco Survey.

According to data from the 2018 National Youth Tobacco Survey, youth use of e-cigarettes has surged to epidemic proportions in the U.S. The number of high school students who use e-cigarettes has risen 78 percent last year to about 3.6 million, or 20 percent of high school students. Additionally, use among middle school students has increased about 48 percent. Current data show that youth who use e-cigarettes are more likely to try combustible cigarettes later. Tobacco use is the leading preventable cause of death, disease, and disability in the U.S., and, at a time when U.S. use of combustible tobacco products is decreasing, we cannot afford for a new generation to become addicted to these dangerous products.

In their statement, the FDA announced that it will limit most e-cigarette sales to age-restricted, in-person locations. Additionally, the FDA plans to require heightened age-verification measures for online sales to try to ensure that minors are not able to buy flavored e-cigarette pods. Although this announcement will affect most e-cigarette flavors, mint and menthol flavors will be excluded from the new enforcement rules. The FDA has stated the agency's announcement will not affect the sale of mint and menthol flavors because data suggests that these flavors are more popular with adults than children and that these flavors may be important to adult smokers seeking to transition away from combustible cigarettes. However, Commissioner Gottlieb has stated that if evidence shows that youth use of mint or menthol e-cigarettes is not declining, he will revisit this policy.

The second prong of the FDA's plan is to ban menthol from combustible cigarettes. Menthol cigarette use is particularly high in youth smokers and is especially troubling in the African-American population where 7 out of ten youth African-American smokers use menthol cigarettes.  Informed by comments to the agency's 2018 Advanced Notice of Proposed Rulemaking on this issue, the FDA will issue a Notice of Proposed Rulemaking, the next step in the rulemaking process, that would seek to ban menthol in cigarettes and cigars.

The last part of the FDA's plan is to examine their regulatory approach to flavored cigars. Research has shown that relative to adult cigar users, youth cigar users are more likely to use flavored cigars. Data has shown that eliminating flavors from cigars is likely to help prevent cigar initiation in youth populations.

Concern over the rising epidemic of youth e-cigarette use has attracted the attention of Congress as well. On Aug. 1, 2018, Senators Dick Durbin (D-IL) and Lisa Murkowski (R-AK) introduced the Stopping Appealing Flavors in E-Cigarettes for Kids Act (SAFE Act), which would immediately ban the use of flavorings in cigars and would give tobacco companies one year to prove that e-cigarette flavorings: (1) aid in adult smoking cessation; (2) do not attract children to tobacco products; (3) and do not harm the user.  

The AACR has been very active on this issue. On July 18, 2018, the AACR hosted a Congressional briefing titled "E-cigarettes: Striking a Balance Between Preventing Youth Nicotine Addiction and Helping Current Smokers Quit Combustible Cigarettes." The panelists presented the latest available science pertaining to the effects and content of e-cigarettes and suggested approaches to halting the concerning rise in youth vaping and nicotine addiction. The AACR's Tobacco and Cancer Subcommittee has also commented on the FDA's 2018 Advanced Notice of Proposed Rulemaking on flavorings and will continue to work with the Congress and the FDA to keep harmful tobacco products out of the hands of children.

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Partners in Progress 2018: Cancer Patient Advocates and FDA

The FDA Oncology Center of Excellence (OCE) continues to highlight the important role patient advocates serve in the regulatory space. On Nov. 27, the OCE held the second annual educational workshop for new cancer patient advocates, "Partners in Progress: Cancer Patient Advocates and FDA" at their White Oak Campus. Cancer survivors and advocates from around the country converged (in person and online) to learn about the regulatory aspects of oncology product development and the multiple opportunities for patients to engage with the FDA. OCE emphasized their commitment to work with patients to make clinical trials faster, more inclusive, and more responsive; and to increase access to investigational drugs. 

The FDA has a long history of working with patient advocates dating back to 1988 and the first HIV/AID patient group. The Patient Representative Program (PRP) soon followed in 1991, when patients began to serve as consultants during the review cycle. Today the PRP has over 200 participants who provide FDA with the unique perspectives of patients and family members who are affected by a serious or life-threatening disease.  

Most recently, as part of FDA's Patient Focused Drug Development (PFDD) efforts in accordance with the 21st Century Cures Act and The Food and Drug Administration Reauthorization Act of 2017 Title I, the FDA is developing a series of four methodological guidance documents to address how stakeholders can collect and submit patient experience data from patients and caregivers for medical product development and regulatory decision making. Developing these important tools, called Clinical Outcome Assessments (COA), will allow the patient perspective to be integrated throughout the drug development continuum. If interested in learning more about COAs in cancer clinical trials, OCE will be hosting a public workshop July 12, 2019. AACR will share more information as it becomes available.

For more information on the 2018 Partners in Progress Workshop

For more information on FDA Patient Engagement

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FDA Proposes New Regulations for Prescription Drug-Linked Apps

By Elizabeth Barksdale, PhD

Continuing its efforts to promote development and innovation in digital health, on Nov. 19, 2018, the U.S. Food and Drug Administration (FDA) released a proposed regulatory framework for prescription drug-use-related software. Speaking at an event the same day, FDA Commissioner Scott Gottlieb, MD, said, “The new approach…would consider the circumstances when software disseminated by drug sponsors to be used with a prescription drug would require approval by the FDA. The FDA expects these circumstances to be limited.”

The regulatory framework outlines a risk-based approach to regulating software marketed by a drug sponsor specifically for use with one of the sponsor’s drugs. The new regulations would apply only to the output of the software, not the underlying code or the software itself. The software output, typically accessed through a smartphone app, would be regulated as labeling because it accompanies a specific drug. FDA recognizes two general types of drug labeling, FDA-required and promotional, and predicts that most prescription drug-use-related outputs would be considered promotional.

As promotional labeling, these apps would not require pre-market approval. Sponsors would submit screenshots or other representations of what the user would experience (i.e., see, hear, feel) when they first disseminate the apps, and the apps would then be subject to existing post-marketing requirements. Additionally, software updates would only require FDA approval if they change what the user experiences through the app; drug sponsors would not need to submit security patches and other minor tweaks to the FDA. Examples of software outputs that constitute promotional labeling include apps that enable patients to enter and track health symptoms related to the condition for which they are taking the drug; provide patients with prescription drug information that is also found in the FDA-required labeling; and allow patients to enter a drug regimen and then reminds them to take it if they don’t record doing so.

If use of prescription drug-use-related software effects a clinically meaningful outcome or is essential to the use of a product of which the software is a constituent part, then the software output (i.e., the app) is considered “FDA-required labeling.” In these cases, the sponsors must submit the output to FDA for review and approval as part of a new drug, abbreviated new drug, or biologics license application, and software updates would require FDA review.

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FDA App to Facilitate Real World Data Collection

By Trevan Locke, PhD

The U.S. Food and Drug Administration (FDA) has posted computer code and technical information for the FDA MyStudies App (see overview). This new app, which was developed in collaboration with private sector partners, is intended to allow patients to directly input their health data. This app represents the latest in the FDA's efforts to advance the use of real world data, which includes electronic health records (EHR), claims information, registries, and patient-reported outcomes. Using the app, the patient data can be linked to electronic health records and potentially support clinical trials, observational studies, and contribute to product or disease registries.

The MyStudies app is built to support iOS or Android development. By releasing the open source code and technical documentation to the public, the FDA is allowing other organizations to reconfigure the app, including in-app branding, for their specific clinical research efforts. The app features a secure data storage environment that complies with agency requirements for data management and supports the ability to run multi-site trials. It is configurable for different therapeutic areas and supports a wide variety of question types (e.g., yes/no, image selection, multiple choice, open text, etc.) for different health outcome measurements.

A pilot study following medication use and health care outcomes of pregnant women was led by Kaiser Permanente Washington Health Research Institute as a proof-of-concept for the app. For the pilot, the app was configured to collect data on participants' medical histories and determine how pregnancy affected their use of medication. Women in the study provided medical and non-medical reasons for discontinuing medication. The study also compared self-reported medication use via the app to EHR and found higher concordance between the app and EHR for prescription drugs than for over-the-counter medications. Sensitive information such as alcohol use during pregnancy was also reported. This study demonstrated that the app has the capacity to expand the amount and types of electronic health data collected and could be leveraged in a clinical research setting.

The agency expects the app to aid researchers and drug developers in collecting patient-reported data and promote efficiencies in drug development and drug safety monitoring.

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Apply Today: Scientist↔Survivor Program at the AACR Annual Meeting      

The AACR is now accepting applications for cancer survivors and patient advocates to enroll in the Scientist↔Survivor Program at the 2019 AACR Annual Meeting. This unique educational program provides an opportunity for survivors and patient advocates to learn and discuss the latest findings in cancer research, regulatory science, and health science policy. Travel, hotel, and registration expenses will be covered for participants accepted into the Scientist↔Survivor Program. 

The curriculum is designed to build enduring partnerships among the leaders of the scientific, survivor, regulatory, and patient advocacy communities. Participants have the opportunity to:

  • Attend special lectures about cancer research that are tailored to a lay audience;
  • Discuss relevant and timely cancer topics during small group meetings, roundtable discussions and meetings with scientific mentors;
  • Explore the scientific sessions of the annual meeting on their own or with scientific mentors or other advocates;
  • Communicate to scientists the key issues, questions, and concerns of the survivor and patient advocacy communities;
  • Promote their organizations' missions by participating in their own poster sessions; and
  • Network with scientists and fellow advocates from local, national, and international cancer organizations.

Participants are accepted through a competitive application process. Learn more and access application materials here.

Please share this announcement with survivors and patient advocates with an interest in cancer research and policy.

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