Working Together to Overcome Cancer Through Science-Based Public Policy That Is Patient Centered
In this section, you will learn:
Federal funding for biomedical research, most specifically through NIH and NCI, has a significant impact on our nation’s health and the United States economy.
Regulatory science initiatives at the FDA are vital to accelerating progress against cancer.
Policies and federally funded public health programs, many of which are supported by the CDC, ensure that individuals have access to preventive services, screening, and coverage for cancer treatment.
Tobacco control policies improve public health and significantly reduce cancer risk.
Newly passed legislation aims to improve outcomes for children and adolescents who are diagnosed with cancer.
There has never been a time of greater excitement in cancer research. The rapid pace of progress and the broadening scope of the advances made in recent years have been extraordinary. As detailed in Cancer in 2018, the hard work of individuals from all segments of the biomedical research community continues to transform the way we prevent, detect, diagnose, and treat cancer. Despite the progress, however, cancer remains a formidable challenge; it will claim an estimated 609,640 lives in the United States in 2018 (10).
Fortunately, scientific opportunities now exist that were previously unimaginable and that hold great promise for achieving our ultimate goal of preventing and curing all cancers.
To seize the opportunities that lie before us, and meet the challenges ahead, we need robust, sustained, and predictable funding for biomedical research through the NIH. For the research investments to yield dividends in the form of new medical products and community-based programs to improve public health, we also need strong federal investments in agencies such as the FDA and the CDC, and evidence-based policy making across and throughout the biomedical research ecosystem (see sidebar on Building Blocks to Overcome Cancer Through Science Policy).
Maintaining the Momentum with Continued Annual Funding Increases for Biomedical Research
Federal funding through the NIH is the lifeblood of biomedical research. It forms the foundation upon which most scientific discoveries in the United States are made. Faithfully investing federal dollars in biomedical research over many years can yield tremendous dividends. Many of the major medical breakthroughs made in the last 50 years can be traced in large part back to an NIH grant, including the extraordinary progress detailed in this report. Moreover, a recent study found that NIH funding contributed to the development of 210 new molecular entities approved by the FDA between 2010 and 2016 (24).
The AACR is deeply grateful to U.S. House and Senate leaders for making medical research a national priority in recent years and for working in a bipartisan way to set the NIH and NCI back on paths of annual growth above medical inflation (see Figure 19). Since fiscal year (FY) 2015, Congress has renewed its commitment to the promise of biomedical research and increased the NIH budget by $7 billion, a remarkable 23 percent increase. With this new trajectory of growth, the troubling trend of stagnant budgets that persisted for more than a decade has been halted. In addition, the NIH Innovation Fund, a multiyear, targeted funding stream created by the 21st Century Cures Act, is providing dedicated resources for the National Cancer Moonshot Initiative to further accelerate progress in priority areas where researchers are poised to make great strides in the next few years (see sidebar on The National Cancer Moonshot Initiative).
Funding for the NIH and NCI supports work across the spectrum of cancer research, from basic and population science to translational and clinical research, all of which are vital for making progress against cancer (see sidebar on Biomedical Research: What Is It and Who Conducts It?). Additionally, research on many cancers funded through the Department of Defense plays an important role and complements research projects funded by NIH (see sidebar on Department of Defense Congressionally Directed Medical Research Programs).
As noted by Congressman Ted Poe, supporting biomedical research is one of the most important investments that our government can make, as it reaches all areas of the United States. In fact, more than 80 percent of the funds appropriated by Congress to the NIH are distributed to scientists in all 50 states, the District of Columbia, and around the world to conduct research. Now, thanks to funding increases in recent years, the NIH is awarding more than 60,000 research and training grants annually, 10,000 more than in 2015. The impact of federal support for the NIH and NCI reaches well beyond the laboratory and the clinic. As the largest single public funder of biomedical research in the world, NIH extramural funding generated $68.8 billion in economic activity last year alone.
Congress must stay the current course and prevent a return to an era of stagnant annual budgets at the NIH. A continued commitment from our elected leaders to robust annual increases for the NIH is imperative if we are to continue to spur progress and yield results for all Americans and people around the world.
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A Strong, Diverse Research Workforce Depends on Predictable Funding
Many innovative research questions and fresh ideas come from scientists early in their careers. Ensuring the continued, rapid pace of progress against cancer requires that the next generation of cancer researchers be recruited, supported, and encouraged. A strong pipeline of talented researchers to whom current leaders in the field can pass the baton in the years to come will allow the work to continue in earnest to conquer this disease.
The current generation of early-career investigators has been privy to the exciting advancements that have been described in this report. At the same time, they came of age in a decade when research funds were scarce, federal research budgets declined by more than 20 percent, and the path forward was uncertain. As we recommit to biomedical research as a national priority, our country must continue to invest in education and training of scientists at all career levels, but especially to those who are in the early stages of their careers. The cancer workforce of tomorrow also must reflect the increasing diversity in our country, including disciplinary, gender, racial, ethnic, and geographic diversity. Robust, sustained, and predictable annual funding increases for the NIH, coupled with federal, state, and private sector-funded programs to assist early-career scientists, play an irreplaceable role in cultivating tomorrow’s scientific leaders.
Members of Congress and NIH officials have recognized the importance of supporting scientists early in their careers, and they have taken steps to assist these investigators through legislative provisions in the 21st Century Cures Act and new policies enacted through the NIH Office of Extramural Research, including the Next Generation Research Initiative (see sidebar on Fostering the Cancer Research Workforce of Tomorrow).
The NCI continues to identify ways to support early-career investigators. For example, in January 2018, the NCI announced the use of the Method to Extend Research in Time (MERIT)(R37) Award to provide longer term grant support to early-career investigators. By providing an opportunity for longer term support for these individuals, the NCI the NCI hopes this will provide them with more opportunities to take creative risks and allow them to have additional time to successfully establish their careers before having to submit renewal applications. NCI Director Norman E. “Ned” Sharpless, MD, also announced at the AACR Annual Meeting in April 2018 that the NCI is increasing the number of R01s awarded to early-career investigators by 25 percent this year. The NCI’s ability to take this step is a direct result of a greater federal investment in the NIH over the past three years.
Policies to Advance Regulatory Science at the FDA
The FDA is an indispensable federal partner to the NCI in the quest to conquer cancer. It is imperative that the FDA receive consistent, robust support from Congress through annual appropriations because this funding supports regulatory science initiatives not funded through user fees. These initiatives seek to develop new evidence-based regulatory policies that promote cutting-edge innovation and expedited approval.
In recent years, Congress has recognized that it must equip the FDA with the resources it needs to support these regulatory processes. The 21st Century Cures Act included a supplemental, dedicated stream of funding, the FDA Innovation Fund, to assist the Agency over the next decade with increased responsibilities related to the Act. The law also expands hiring authority at the agency, and it ensures the continued professional development of staff.
In January 2017, the FDA established the Oncology Center of Excellence (OCE), which brings together regulatory scientists and reviewers with oncology expertise to expedite the evaluation of drugs, biologics, and devices for the treatment of cancer. Because of the coordinated clinical review made possible by its silo-busting intercenter structure, in its first year of existence, the OCE played a critical role in the approval of breakthrough therapies such as the first tissue-agnostic approval of a cancer treatment based on a biomarker (pembrolizumab) and the approval of two CAR T-cell therapies that harness the power of the patient’s own immune system to fight cancer (tisagenlecleucel and axicabtagene ciloleucel). Within its first year of existence, the OCE approved 17 drug and biologic applications, 32 supplemental drug and biologic applications, and two biosimilar applications in oncology (252).
Policy mandates established by the 21st Century Cures Act and other legislation accelerated efforts to characterize regulatory-grade real-world evidence and develop methodologies to support its regulatory use. Real-world evidence is clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data (see sidebar on Real-world Evidence: Regulatory Practice and Promise). Use of real-world evidence has broad potential applications to improve clinical care, accelerate research, and support regulatory decision-making.
To support these critical regulatory efforts and more, Congress provided the OCE with its first directed appropriation of $15 million into the Agency's base budget in FY 2018. There is broad support for providing the Center with $20 million in FY 2019.
In addition to the 21st Century Cures Act, another effort that gained significant momentum and resulted in the passage of new legislation in May 2018 by Congress is the concept of expanded access to experimental therapies, or “right-to-try” (see sidebar Expanded Access and Right-to-Try).
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Policies to Advance Cancer Prevention and Control for Individuals and Communities
Public policies must support both scientific research and the application (or implementation) of that research through programs that benefit people, families, and communities (see sidebar on CDC Cancer Prevention and Control Programs). Multiple factors, in addition to genes and biology, affect cancer mortality and quality of life. These factors include the environment in which people live and the impact of health behaviors, community support networks, and local, state, and federal policies and programs. Cancer prevention, detection, treatment, and survivorship programs are most effective when all these factors are considered. Scientific research can inform efforts to address these factors, so that we can reduce cancer rates, reduce cancer mortality, and improve the quality of life for cancer patients and survivors.
Among the most important factors in preventing and treating cancer is access to comprehensive health care coverage. The Affordable Care Act (ACA) (see sidebar on Major Benefits Related to Cancer in the ACA) continues to provide major benefits for all Americans, but especially for cancer patients and survivors. Particularly valuable in terms of cancer prevention and control are provisions mandating coverage for preventive cancer screening and prohibiting denial of insurance coverage based on pre-existing conditions. Any modifications to existing health care coverage policy should maintain and strengthen these provisions to ensure all Americans share in the benefits of our national commitment to cancer prevention and control.
Elimination of HPV-related cancers is one area where advances in basic and clinical research, paired with results of population research, can be combined to spur policy that substantially reduces cancer mortality. In June 2018, the AACR joined all 70 NCI-designated cancer centers and five national cancer organizations in urging increased HPV vaccination and screening access to prevent the human devastation caused by HPV-related cancers. The organizations collectively recognized insufficient vaccination as a public health threat and called upon the nation’s policy makers, physicians, parents, and young adults to take advantage of this unprecedented opportunity through vaccination to eliminate several different HPV-related cancers in men and women. Achieving the HPV-cancer elimination goal will require effective collaboration among a broad range of stakeholders, as well as policies and programs to strengthen HPV vaccination rates at the national, state, and local levels.
Growing differences in risk factor exposure and cancer outcomes between racial and ethnic groups, rural and urban populations, and insured/uninsured Americans highlight the need for health policy solutions that address disparities. Policy solutions that support coordination among federal agencies, state and local governments, and the communities they serve are important to reduce inequities in health care access, ensure high-quality care for all, and address social determinants of health. Improved engagement of minority communities as partners in research is one notable way in which NIH is working toward better understanding of disparities to help drive development of solutions.
The NIH All of Us program (a prospective study of one million Americans funded through the NIH Innovation Fund) is engaging communities across the country to ensure participants in this extensive research cohort reflect American diversity. Such diverse inclusion is intended to lay the groundwork for enabling men and women of all ages, races, and ethnicities to benefit from coming advances in precision medicine. In another example of NIH leadership on disparities, NCI is spearheading a rural cancer control initiative that funds population research and promotes national-level interagency collaboration to better understand rural disparities and test strategies for mitigating challenges rural communities face in cancer prevention and control.
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Targeting Tobacco Products through FDA Regulation
Restricting the use of tobacco through regulatory policies has long had an important role in cancer prevention and improving outcomes for cancer patients (see sidebar on Highlighting the New Corrective Advertisements). The AACR strongly believes that further progress can be made against cancer through the use of evidence-based strategies to prevent and control tobacco use (230) (253).
In July 2017, FDA Commissioner Scott Gottlieb, MD, announced that the agency’s Center for Tobacco Products will focus their tobacco regulatory efforts on nicotine addiction. Although nicotine is not directly responsible for cancer-related deaths, it is a highly addictive compound, which makes it extremely difficult for users to quit using tobacco products. Through an Advanced Notice of Proposed Rulemaking (ANPRM), the FDA is beginning to look at lowering the nicotine content of combustible tobacco products and sought public input on the potential public health benefits and any possible adverse effects of lowering the nicotine content of combustible cigarettes. The FDA has issued additional ANPRMs on the role of menthol and kid-appealing flavors in tobacco products in attracting youth and on the definition and use patterns of premium cigars. The AACR, through its Science Policy and Government Affairs Committee, Tobacco and Cancer Subcommittee, publicly submitted ideas and suggestions on all of these particular requests for comments from the FDA.
In recent years, electronic nicotine delivery systems (ENDS) have become popular with adolescent and young adults. Some e-cigarette devices contain high levels of nicotine and have strong youth appeal as they come in a variety of flavors and are easily concealed through their resemblance to USB flash drives and a vapor cloud that dissipates quickly.
To address these concerns, the FDA has:
conducted a nationwide crackdown on the sale of e-cigarettes to minors at both brick-and-mortar and online retailers.
contacted eBay to raise concerns over several listings for a specific, novel END product called JUUL, on its website. As a result, eBay swiftly removed the listings and voluntarily implemented new measures to prevent new listings from being posted to the web retailer’s site.
contacted e-cigarette manufacturers directly to request marketing information to understand and address why kids are finding these products appealing.
issued in conjunction with the Federal Trade Commission warning letters to companies that misleadingly labeled or advertised nicotine-containing e-liquids as kid-friendly food products like juice boxes, candies, and cookies. E-liquids can be harmful, even deadly, to children.
Policies to Overcome Challenges in Pediatric Cancer
Cancer remains the second leading cause of death in children ages 1 to 14 (10). Over the past year, important strides have been made to further progress against pediatric cancers through the passage and implementation of two key pieces of legislation. In August 2017, Congress passed key provisions of the Research to Accelerate Cures and Equity (RACE) for Children Act of 2017. The RACE Act requires companies developing targeted cancer drugs for adults to develop those drugs for children with cancer as well. The law is a critical update to the Pediatric Research Equity Act (PREA) of 2003, which has led to important new safety and labeling information for other children’s diseases but has had a minimal impact on childhood cancer due to several exemptions. Importantly, the RACE Act ends an exemption to PREA obligations for cancer drugs with orphan designations if the molecular target of the drug is relevant to a pediatric cancer. Since the passage of the RACE Act, the FDA has worked with NCI and other key opinion leaders to develop a list of pediatric-relevant molecular targets that will address regulatory uncertainty for industry and guide decision-making.
In May 2018, Congress passed the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act of 2018 (see sidebar on The STAR Act Provisions). This is the most comprehensive childhood cancer legislation passed by Congress to date, and it aims to address some of the most challenging issues in childhood cancer research and care.
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