Below are the first 10 and last 10 pages of uncorrected machine-read text (when available) of this chapter, followed by the top 30 algorithmically extracted key phrases from the chapter as a whole.
Intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text on the opening pages of each chapter. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.
Do not use for reproduction, copying, pasting, or reading; exclusively for search engines.
OCR for page 1
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care INTRODUCTION Many ongoing changes are likely to have an impact on cancer research and care. For example, technological advances are rapidly changing the way cancer research is conducted, and the recently passed healthcare reform legislation has many implications for cancer care. There is a growing emphasis on molecularly targeted therapies, information technology (IT), and patient-centered care, and clinical cancer research has become a global endeavor. At the same time, there are concerns about shrinking research budgets and escalating costs of cancer care. Considering such changes, the National Cancer Policy Forum (NCPF) of the Institute of Medicine held a National Cancer Policy Summit on October 25, 2010. The Summit convened key leaders in the cancer community to identify and discuss the most pressing policy issues in cancer research and cancer care. With panel presentations and discussions led by experts in topics such as healthcare policy, oncology research, public health, palliative care, and behavioral economics, the Summit explored policy issues related to cancer research, implementation of healthcare reform, delivery of cancer care, and cancer control and public health needs. These discussions among Summit participants and Forum members will guide the Forum’s strategic planning and ensure that NCPF activities are responsive to priorities in the cancer community. This document is a summary of the Summit, authored by NCPF staff. The agenda is in the Appendix.
OCR for page 2
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care Several recurring themes surfaced during the meeting that participants identified as important policies issues in cancer. These themes included providing IT to support a learning healthcare system; determining new and financially sustainable models for cancer care; facilitating patient-centered cancer research and care, including research that documents patient quality of life and care that considers pain management and the support needed by caregivers; restructuring the research, clinical, and regulatory arenas to address current challenges; fostering precompetitive collaboration; taking a global approach to both health research and regulation; adopting standards of care and essential health benefits in the implementation of the Affordable Care Act (ACA);1 and advancing successful cancer risk reduction and prevention efforts. Many of the panelists also put forth suggestions for policy actions to address the needs and challenges they had identified. For example, it was pointed out that although a goal of healthcare reform legislation was to reduce healthcare disparities, some provisions are not consistent in this regard. The ACA prohibits health insurers from denying an individual access to an “approved” clinical trial (including those funded by the National Institutes of Health [NIH]) and requires coverage for routine patient care costs in those trials; but the legislative provisions pertaining to Medicaid do not include such language, so low-income patients could still be denied access to novel therapies in clinical trials. Several panelists suggested that this could easily be remedied. However, Medicaid is administered at the state level, and many of the healthcare reform provisions will be implemented at the state level, so there likely will be 50 different approaches to healthcare reform and Medicaid coverage policies. Participants identified several major challenges again and again, and offered similar potential solutions. For example, combination therapies that target multiple key pathways in cancer cells are increasingly seen as the primary hope for new breakthroughs in cancer treatment. Yet the 1 In this workshop summary, the Affordable Care Act refers to the final version of the healthcare reform law, including the Patient Protection and Affordable Care Act (P.L. 111-148) signed into law by President Barack Obama on March 23, 2010, and amended by the Health Care and Education Reconciliation Act of 2010 (P.L. 111-152) on March 30, 2010.
OCR for page 3
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care co-development of novel agents entails unique challenges that are not encountered when developing single agents. Panelists and Forum members repeatedly asserted that new incentives, such as increased market exclusivity, are needed to encourage drug developers to overcome these obstacles. Many participants also stressed that interoperable informatics systems will be critical for making advances in cancer research and care. RAPID LEARNING HEALTHCARE SYSTEMS Many of the comments voiced at the Summit centered around the goal of harnessing information technology to create rapid learning healthcare systems, in which data collected on patients can be used in real time to better tailor treatments and determine care that is the most effective with the fewest risks and costs. “The entire backbone of everything we do, for all stakeholders, will be in the area of information—gathering information in a quality way so health information exchange can take place in a real-time system,” said Dr. William Dalton, president, chief executive officer (CEO), and director of the Moffitt Cancer Center. He suggested building this information system so it is useful to researchers, patients, clinicians, policy makers, and administrators, all of whom are aiming to create an evidence-based approach that will make health care more affordable, more accessible, and of higher quality. “To do this we have to build an information system that has the ability to extract data and follow patients throughout their lifetimes…. The patients themselves are the centerpiece—we always need to come back and say what is best for the patient,” said Dr. Dalton. He added that “we can’t assume we know what patients want.” Many patients want to know what research is being done with their data and biological samples, so it will be important to create patient portals for information exchange, he said. The role of patient consent and information dissemination in research using patient databases was addressed in a recent IOM consensus report (IOM, 2009). Citing the NCPF’s report on rapid learning healthcare systems (IOM, 2010b), Dr. Dalton said that it described the direction in which cancer research and care should be heading, adding, “It’s a grand idea, but it doesn’t exist yet and must be built in an iterative process.” He noted that the healthcare reform legislation will help foster a rapid learning healthcare system, with its support of accountable care organizations. These organizations provide a medical home for patients that consolidates healthcare providers and tracks the effectiveness of their care, making it
OCR for page 4
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care easier to gather patients’ longitudinal data and to collect and store their biospecimens. Analytical tools still have to be created, however, to enable investigators to conduct research on the data collected, according to Dr. Dalton. He envisions such tools as especially useful to patients and their physicians in helping them make informed decisions about the optimal care most likely to work for each individual patient. Ideally such decision assist tools would be rapidly construed from the data collected and analyzed in the system, he said. In addition, Dr. Dalton envisions being able to survey a database to find the most appropriate patients for enrollment in a clinical trial and contacting them directly about their participation. Policy makers will also find a rapid learning healthcare system useful for conducting comparative effectiveness research and cost analyses, he added. “My plea as we move forward is that we consider gaining expertise and input on those that build these sort of information exchanges from all facets—academia, industry, and users. We, as a body of researchers, policy makers, and advocates, can influence how the healthcare reform is implemented and deployed, but we must be active and have a major priority be the architecture of the health information exchange that would be required,” Dr. Dalton said. Dr. Russell Glasgow, deputy director of dissemination and implementation science at the Division of Cancer Control and Population Sciences at the National Cancer Institute (NCI), and Dr. Douglas Lowy, deputy director of the NCI agreed. Dr. Lowy stressed, “The issue of information technology is one of paramount importance. It cuts across all the different aspects of research to the point of implementation. As we try to develop evidence-based research, being able to have platforms that can speak to each other is going to be of critical importance.” Interoperability Dr. Harold Varmus, director of NCI, noted that a report from the President’s Council of Advisors on Science and Technology on the design of health information systems will provide guidelines for how information technology in the healthcare arena might be implemented, including the need for standardization and interoperability of hospital computer systems (PCAST, 2010). He and others at NCI have been consulting with the White House Office of Science and Technology Policy on how to create “in the world of oncology a kind of knowledge repository that could be of immense
OCR for page 5
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care benefit to patients, healthcare providers, and the research community. We welcome any advice you want to give for this active process that is currently under development,” Dr. Varmus said. Dr. Ellen Sigal, chairperson and founder of Friends of Cancer Research, also stressed the need for interoperability of health information systems and the data collected by government agencies. “How are we going to synthesize this information and make it useful to make informed decisions?” she asked. “To coordinate all the data collected is a very important opportunity and very daunting responsibility. There is no way that the Patient-Centered Outcomes Research Institute (PCORI)2 or any of these other initiatives will be meaningful unless we work with one another and figure out the interoperability,” she said. Electronic Medical Records Drs. Dalton and Glasgow also stressed the importance of having electronic medical records (EMRs) that are complete and implemented within a healthcare information system. Dr. Dalton suggested that researchers and policy makers voice their concerns about the need for EMRs to include information that will be useful for cancer researchers. As he noted, fewer than 10 percent of ambulatory medical practices currently use EMRs with key functionalities identified by the Institute of Medicine (IOM, 2003b; Jha et al., 2006). Given the current push for the implementation of EMRs and the $19 billion in funding that the Health Information Technology for Economic and Clinical Health (HITECH) Act provided to advance health information technology, Dr. Dalton said now is an opportune time to ensure that the architecture of EMRs will be sufficient for the type of research that could be done using the data these records provide. “This community is the one that needs to influence that architecture so that research can be performed,” Dr. Dalton said. Dr. Glasgow added that EMRs should 2 PCORI was established by the Patient Protection and Affordable Care Act of 2010 (P.L. 111-148) for the purpose of “assist[ing] patients, clinicians, purchasers, and policymakers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis that considers variations in patient subpopulations, and the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriateness of the medical treatments, services, and items.” See also Box 2.
OCR for page 6
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care include patient-reported quality-of-life measures. “A number of our colleagues could provide you with a list of very practical, well-validated, very reliable quality-of-life measures that could be routinely included as part of quality metrics,” Dr. Glasgow said. Validity of Data and Observational Studies There was also recognition of the adage “garbage in, garbage out,” as Dr. Glasgow put it. That is, the usefulness of the data collected will depend on their quality, lack of bias, and thoroughness. He pointed out that there are methods for ensuring that the type of data collected in healthcare systems is sufficiently complete, accurate, and standardized to be useful for research. The U.S. Department of Veteran’s Affairs is an international leader in such data curation and use in research, according to Dr. Glasgow, and the Cancer Research Network, consisting of 14 health maintenance organizations (HMOs) around the country, also has a lot of expertise and publications on this topic. The usefulness of the data collected in a learning healthcare system also depends on their completeness. Dr. Marcus Plescia, director of the Division of Cancer Prevention and Control at the Centers for Disease Control and Prevention (CDC), pointed out that the biggest challenge the CDC has in ensuring the completeness of cancer registries is not the data coming from hospital systems but the lack of data, or lack of complete data, on people who are diagnosed with cancer in community-based practices. He expects these data to improve when more practices use electronic reporting formats. The CDC also has tried to work out ways to get better electronic reporting from sources, such as pathology laboratories, that do not currently report to it. “If we could get pathology labs to report, that would be an enormous way to pick up on a lot of these cases that are slipping through the cracks,” he said. He noted that CDC has also been involved in determining what criteria physicians should have to report on in the electronic records they submit to cancer registries. Dr. Rachel Ballard-Barbash, associate director of the Applied Research Program of the Division of Cancer Control and Population Sciences at NCI, added that NCI has made an effort to link data systems related to quality of life and patient-reported outcomes to cancer registry data to further their clinical usefulness. “The CDC and the NCI are trying to figure out how to harness the potential for informatics to do this more rapidly. But I think doing it within the context of healthcare delivery systems, where
OCR for page 7
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care we are looking at integrating information at the point of care and providing that information back, is probably a model that will be more relevant for immediate clinical interaction,” she said. The benefits and challenges of linking patient data from different sources (especially if the data have been de-identified) was described in a recent IOM consensus report (IOM, 2009). Dr. Plescia added that “it’s challenging to make these big state government bureaucratic systems [responsible for the cancer registries] able to respond at the kind of speed that researchers need to be able to do their work.” Dr. Patricia Ganz, professor at the University of California, Los Angeles, Schools of Medicine and Public Health, suggested that the cancer registry information collected by states for the CDC be expanded to include information collected by patient insurers. In response, Dr. Plescia noted that information from health insurance systems would be extremely helpful, but it is often difficult to acquire. There was some debate on how observational studies conducted using the data collected from healthcare information systems should be utilized in addition to the gold standard of randomized, controlled studies. “We probably won’t be able to just take an electronic medical record and use it for research because it wasn’t created for that purpose, but for the purpose of treating the patient. It’s not going to be our savior,” said Dr. Kay Dickersin, professor of epidemiology and director of the Center for Clinical Trials and U.S. Cochrane Center at Johns Hopkins Bloomberg School of Public Health. Dr. Dalton agreed that using standardized electronic health records as they now exist will not be sufficient. He stressed that there is an urgent need to incorporate research into the concept of meaningful use as efforts continue to expand the use of electronic health records in clinical practice. There is now a window of opportunity to build the necessary architecture into electronic systems to facilitate research, he said. Dr. Dickersin added that observational studies are very important for identifying harms associated with treatments and such studies are already commonly included in systematic reviews that focus on harms, but much work remains to develop the methodology for using observational data in systematic reviews more broadly. She noted that a current active area is research on methods related to systematic reviews using observational data to determine, for example, how bias might affect the validity of findings. “I don’t think anybody is excluding the idea of observational studies, it’s just that we have an awful lot to learn, and it depends on the question you are asking,” Dr. Dickersin said.
OCR for page 8
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care Although recognizing that observational studies using data collected in healthcare information systems have their limitations, some participants pointed out that there are also limitations of randomized controlled trials. These trials evaluate treatments in relatively small, carefully selected populations, often without the confounding morbidities that would make the results applicable to the general population in real-world settings. “One of the biggest challenges facing cancer right now is the issue of whether the findings we are generating are valid externally for patients that cannot be entered into randomized, controlled trials, because we are not going to test all of our questions in randomized, controlled trials,” Dr. Ballard-Barbash said. “We need to think about how to use statistics in our large data systems to really look at this across populations,” she added. Dr. Dalton also noted, “The more patients you study that are participating in a real-time learning information system, the more you learn.” Examples of Rapid Learning Healthcare Systems Dr. Ballard-Barbash cited the HMO Cancer Research Network as an example of a rapid learning healthcare system. This collaboration of HMOs around the country covers 11 million patients and, in one year, diagnoses more than 100,000 cancer patients. The Cancer Research Network has funding from several sources in addition to NCI, such as the Agency for Healthcare Research and Quality (AHRQ) and the CDC, who are interested in its potential to be a “population laboratory,” Dr. Ballard-Barbash said, and they have agreed to a centralized approach to institutional review board (IRB) oversight for projects, with the lead institution on a particular research project managing the IRB issues. Dr. Ballard-Barbash gave this example to illustrate that large healthcare systems already are making progress in learning from the patient data they collect. “While it is true that they are not standardized, there is a lot happening related to natural language processing and other ways to capture data that are currently in many records and are going to evolve over time,” she said. Dr. Kirsten Anderson, chief of staff to the chief medical officer and senior medical director at Aetna, added that one-third of Aetna focuses on information technology that generates and stores a tremendous amount of data that is used in meaningful research. “Information technology is going to be key if we are going to make any kind of inroads to efficiency and productivity,” Dr. Anderson said. Dr. Lloyd Everson, vice chair and member of the board of directors of
OCR for page 9
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care US Oncology, Inc., agreed that “IT is key to the future,” but added, “The question is, Where is the capital going to come from [to pay for IT]? Who can afford this?” EVIDENCE-BASED MEDICINE In addition to primary research conducted in randomized clinical trials and observational studies, evidence-based medicine relies upon systematic reviews and comparative effectiveness studies to ascertain which treatments work best for which patients. Dr. Dickersin focused her remarks on how systematic reviews can inform the practice of evidence-based medicine. Dr. Dickersin explained that a systematic review is a review of existing knowledge that uses explicit, scientific methods in a structured and transparent process. Systematic reviews entail a comprehensive search for relevant articles and, unlike other literature reviews, apply explicit scientific methods of appraisal and synthesis of the findings. The IOM has since released a report on standards for systematic reviews (IOM, 2011). Practice guidelines are increasingly relying on systematic reviews, according to Dr. Dickersin, who argued these reviews are essential for a well-performing healthcare system. Dr. Dickersin noted that there are a fair number of systematic reviews being conducted at the federal level (including by the AHRQ, CDC, Substance Abuse and Mental Health Services Administration, Centers for Medicare and Medicaid Services, U.S. Department of Veterans Affairs, and U.S. Preventive Services Task Force), but the majority of these reviews are using internal programs or agency or contractual investigators. She said there is little funding for systematic reviews conducted outside of internally supported federal programs. NIH provides the major source of funding for external systematic reviews and funds systematic reviews conducted by the Cochrane Collaboration (Box 1). PCORI will also likely make funding available for systematic reviews. Dr. Dickersin made several recommendations regarding systematic reviews. One was for NIH to provide more funding for such reviews, so that there is coverage of specific disease-related topics in the reviews that have clinical relevance. AHRQ tends to focus its systematic reviews on broad-based topics that cut across health fields and has done only a few on cancer, for example. Dr. Dickersin also echoed several IOM recommendations made in its 2008 report Knowing What Works in Health Care (IOM, 2008), namely, that there be more investment in advancing the scientific methods used to
OCR for page 10
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care BOX 1 The Cochrane Collaboration The Cochrane Collaboration is an international collaboration with more than 25,000 contributors that prepares, maintains, and promotes the accessibility of systematic reviews of the effects of healthcare interventions. It has done more than 4,000 systematic reviews, 314 of which involve cancer. All of the Cochrane Collaboration’s reviews are published in The Cochrane Library, which is updated monthly. The Cochrane Collaboration is currently working with other groups, such as the AHRQ and its evidence-based practice centers, to make the data from their systematic reviews publicly available so that other researchers can use these data in their own systematic reviews. SOURCE: Dickersin presentation (October 25, 2010). do systematic reviews and that the capacity to conduct systematic reviews be built in the workforce by expanding training opportunities in systematic review and comparative effectiveness research methods. Additional suggestions from Dr. Dickersin included that efforts be made to learn from the methods research that has been done in other countries and that the federal government provide training grants specifically for systematic reviews, as well as infrastructure funds for investigator-initiated systematic reviews. “Let’s ensure that these mechanisms are sustained and we don’t break down the groups as soon as the funding is over. That’s one of the advantages of funding large groups who build infrastructure and hubs of knowledge and activity,” Dr. Dickersin said. Dr. Dickersin’s final suggestion was that systematic reviews be required before clinical trials are funded and that the purpose of the trials should be put within the context of a systematic review. “The bottom line is that systematic reviews are very important. I would like to see NIH, AHRQ, and all the federal agencies engaged in a cooperative effort here,” she said. Dr. Lowy agreed that it is an appropriate role for NIH to fund systematic reviews and that it should leverage its resources with other agencies in order to create maximum benefit in this regard.
OCR for page 11
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care FINANCIALLY SUSTAINABLE MODELS OF CARE A major focus of comments and discussion at the Summit was on how to provide financially sustainable models of cancer care, given the current economic downturn, healthcare costs that have risen exponentially in recent years, and the current interest in healthcare reform. “The economic crisis in health care is very real and it threatens the delivery of cancer care in our country,” stressed Dr. Everson. “Costs are increasing more rapidly than revenue.” This is especially true for cancer care, which is particularly complex and thus more costly, he noted. Dr. Allen Lichter, chief executive officer of the American Society of Clinical Oncology, concurred, noting that each oncologist supports, on average, eight staff in their office, which requires a large, reliable revenue stream. Yet he said there has been increasing reluctance of health insurers to reimburse expensive cancer care. This is driving cancer care to be offered only within hospital settings, which further increases the cost of the care, Dr. Lichter noted. “We must address cost. This is the single most poisonous thing that is going on in the field of oncology. If it continues unchecked at the pace it has been going, it will destroy the oncology care delivery system in the United Sates,” he said. Participants made several suggestions for what the elements of a financially sustainable model of cancer care would be, including the following: Incentives that are aligned with costs and quality Value-added or appropriate care that is indicated by evidence-based medicine Quality standards Coordinated care that has a team or network approach Innovative payment schemes Appropriate Incentives Dr. Everson pointed out that incentives drive the use of procedures, and the financial incentives currently in the healthcare system contribute to the fragmentation of care in many settings, with little coordination between the outpatient and inpatient settings or from primary care to specialty care. There also is no real incentive for providing quality care that leads to efficient and effective outcomes, he added. For example, Drs. Ganz and Everson both pointed out that chronic conditions account for more than 75 percent of all healthcare costs, and many chronic conditions can be
OCR for page 34
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care their clinical significance in ways that can bring new drugs forward in a more expeditious manner,” Dr. Rothenberg said. Dr. Lowy responded that “what we actually need are validated surrogate end points. Clearly biomarkers end up being of critical importance.” He noted that in the HPV (human papilloma virus) vaccine trials, there was sufficient understanding of the pathogenesis of cervical cancer that researchers could use moderate and high-grade dysplasia as an end point. “Because this was a prevention trial, it would have been unethical to wait until cancer developed in these patients,” Dr. Lowy added. PRECOMPETITIVE COLLABORATION As researchers continue to uncover the molecular complexity of the network of pathways that foster various cancers, there is increasing interest in combining multiple drugs, each of which targets a key player in the network. Such combination therapy will likely require precompetitive (i.e., premarket) collaboration if these therapeutic agents are not already on the market and are sponsored by different drug companies. Dr. Rothenberg noted that there are four major obstacles to developing combination therapy: determining targets, validating those targets, determining how those targets interact, and clinically validating the drugs aimed at those targets when used in combination. Each of these tasks can be more effectively addressed by collaboration, Dr. Rothenberg said. Dr. Dalton added that “there are definite synergisms between what different technologies, different companies, can bring. Companies are also realizing that by partnering, they actually increase their market considerably. So some strategic collaborations and partnerships are developing.” Dr. Pazdur suggested such collaborations could be fostered with appropriate incentives, such as increased market exclusivity. FDA successfully used incentives to encourage drug companies to test their adult drugs in pediatric populations, he said. The incentive FDA gave companies was increased exclusivity for six months for the entire drug moiety when it was used for a pediatric indication. “From a regulatory point of view, it is a suboptimal situation if we have to make somebody do something…. When you have a program that has incentives—the only thing we have, from an FDA point of view, is exclusivity—then that means something.” The NCPF recently held a workshop on precompetitive collaboration (IOM, 2010a).
OCR for page 35
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care GLOBALIZED HEALTH RESEARCH AND REGULATION Increasing globalization of biomedical research, especially drug development, testing, and regulation was also raised as an important issue among participants. Dr. Pazdur noted the international scope of oncology and the globalization of clinical trials, and stressed “our need to embrace it rather than flee from it.” The vast majority of clinical trials accrue patients internationally, not only from Western Europe but from Eastern Europe and Asia. “Is this a good thing? Yes. It provides answers more quickly. It provides information on subpopulations in the United States, as we are a nation of immigrants with an ever-increasing presence of Hispanic and Asian populations. So to have representation of these people in clinical trials is extremely important,” Dr. Pazdur said. Dr. Lowy agreed with this statement, adding that the HPV vaccine he was involved with was tested internationally, which he thought was important given that the vaccine is destined to go to different populations, and “it is critical to be sure that your intervention is going to work across populations, and not just be useful for a particular population,” he said. Dr. Pazdur added that “it doesn’t work in the economics sphere to be a protectionist, nor does it work in the scientific sphere.” He suggested that the Cooperative Groups, which are responsible for running many of the major clinical trials of oncology drugs in this country, coordinate their efforts with international studies. He noted that there have been international clinical trials in which Cooperative Group trials have participated, with pharmaceutical companies running the European components of the trials. Dr. Pazdur encouraged more of these collaborations, rather than viewing the Cooperative Group trials as being in competition with international clinical trials. He said the quality of data emanating from these international trials is generally good, and the trials represent the standard of care in the United Sates. “For us to try to cast doubt on these trials by implying that they are inferior really kind of smacks of a protectionism that probably doesn’t benefit anybody in the long run,” Dr. Pazdur said. Dr. Rothenberg pointed out, however, that international trials can have their own set of complications. For example, in a crossover trial, patients whose disease progresses on an experimental therapy are allowed to cross over to another therapy. Yet the type of subsequent therapy given to patients may vary from country to country, so “how will the selection of regions in
OCR for page 36
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care which you choose to do the trial impact the subsequent therapies of those patients, and therefore impact the outcomes of the trials?” he asked. Dr. Pazdur also pointed out the need for consistent international regulation of drugs. He noted that the innovative regulation FDA is currently devising in relation to combinations of unapproved drugs and drug-diagnostic combinations has to harmonize with international regulations, especially with the European Medicines Agency. The FDA has regular discussions with this agency about these topics, he said. Recognizing the rising importance of global health, the NCI is creating a new center for global health that will bring together several existing international programs of NCI and will pave the way to better partnership in research with developing countries, Dr. Varmus reported. “I believe quite strongly that, while it’s difficult to bring sophisticated radiotherapy, complicated surgery, and highly expensive drugs to poor countries, there are many things we can do to lower the burden of cancer in the low- and middle-income populations of poor countries by better prevention practices, especially vaccinations against HPV and HBV (hepatitis B virus), smoking cessation programs, pain management, early detection, and using therapies that are inexpensive because they are off-patent,” Dr. Varmus said. Dr. Seffrin pointed out that the United Nations (UN) is having its first-ever, high-level meeting on noncommunicable diseases in September 2011. There have only been 28 high-level UN meetings since World War II, he noted, and only one so far has dealt with a health problem (HIV/AIDS). “This is not only our first time to get up to bat, but it’s probably our only time to get up to bat, about what we might be able to do to change the fact that noncommunicable diseases are not on the global health agenda,” he said. He noted that the Millennium Development Goals of the UN are silent about cancer, even though it is the number one cause of death in the world and the single greatest economic burden on the world economy—three times more costly than HIV/AIDS, tuberculosis, and malaria combined (John and Ross, 2010). He added that “if we just were able to provide what we know works today to everyone when they need it and as they need it, by 2030, we could be talking about averting … over 15,000 deaths per day globally.” HEALTHCARE REFORM—AFFORDABLE CARE ACT Dr. Horton gave a presentation on the major provisions of the recently passed ACA, which are summarized in Box 2. Dr. Horton noted that much
OCR for page 37
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care of the implementation of the ACA will be occurring within the next four years. Mr. Weil pointed out that states will be instrumental in implementing many of the provisions in the legislation, including those to expand coverage and preventive health measures. Dr. Horton, Mr. Weil, and others then pointed out ways in which oncology stakeholders can monitor and influence the implementation of the ACA, especially in regard to determining the essential benefits that will have to be offered by qualified health insurance plans, the funding that is allocated to carry out some of its provisions, and gaps in coverage that need to be closed. Essential Health Benefits Dr. Horton noted the importance of defining the essential health benefits package of qualified plans. “What do you get as part of this minimal package or standardized package? How do you influence that at the federal level? That’s very important,” Dr. Horton said. Both she and Dr. Seffrin noted that such a package could specify palliative care, for example. Dr. Horton added that there is opportunity to inform and influence the National Prevention and Health Promotion Strategy that will be developed and its advisory council. Mr. Joseph Glick, founder of Expertool Software, suggested developing standards for risk-benefit analysis and cost calculation. These standards could perhaps be required on both the provider and the payor side and considered when determining essential benefits. “Neither the payors nor the providers are looking at cost-benefit in a standardized way,” he said. Funding and Implementation “There is a lot of authorization language in [the ACA], but not many appropriations. So if Congress makes an effort to defund, most of these provisions are out of luck,” Dr. Horton pointed out. She added that the legislation could potentially be repealed or deemed unconstitutional at some point in the future. Dr. Seffrin doubted that the legislation would be repealed, although he acknowledged that it is likely to undergo changes and that “the important point is that we will be dealing with a new platform for delivery and we have to make sure that we build a better platform than what we have—that we get it right.” Dr. Seffrin noted that the American Cancer Society is focusing more than three-quarters of its advocacy activities for the next four years on
OCR for page 38
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care BOX 2 AFFORDABLE CARE ACT (ACA) The main goals of the ACA are to provide broad health insurance coverage and improve the fairness, quality, and affordability of that coverage, improve healthcare value and efficiency, strengthen primary care access, and invest in public health. The ACA establishes a health plan exchange marketplace for insurance products that meet certain federal and state standards, which are called qualified health benefit plans. These plans are required to cover “essential benefits,” which have yet to be fully defined. The stipulations of the ACA that are especially relevant to cancer patients and providers include the following: Children up to age 26 can enroll on a parent’s plan, and exclusion based on preexisting conditions is prohibited for children, effective July 1, 2010. Adults with preexisting conditions, beginning in 2014, cannot be denied coverage or be forced to pay more based on their health status or gender. Insurers cannot drop coverage because of an individual’s participation in a clinical trial. The legislation prohibits the denial of coverage of routine care costs of participants in certain clinical trials, including FDA-approved drug trials and federally funded clinical trials that treat cancer or other life-threatening diseases. However as currently devised, it does not mandate coverage of routine care costs of Medicaid participants in clinical trials, although that is an option for states, if they are willing to fund it. Medicaid will be expanded to cover all nonelderly, nondisabled citizens and legal U.S. residents with family incomes below 133 percent of the poverty level and will provide preventive care for those individuals. However, coverage of preventive screening efforts to ensure that the many regulations related to ACA implementation meet the needs of cancer patients. Dr. Seffrin said there is a need for scientifically based recommendations in the areas of cancer prevention, palliation, quality of life, cost containment, and appropriate care. Dr. Glasgow suggested conducting mathematical modeling of healthcare reform so as to see the effects of different policy options. He suggested that such modeling
OCR for page 39
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care benefits for adults in “traditional” Medicaid eligibility categories is not required. Medicare benefits will be expanded to include preventive measures deemed effective by the United States Preventive Services Task Force, and screening for women and children recommended by the Health Resources and Services Administration. There will be no cost sharing for these preventive measures. Another new Medicare benefit will be an annual wellness visit and the development of a personal prevention plan. A multipayor national quality improvement strategy will be offered, as well as continued movement toward provider reimbursement tied to quality outcomes. Demonstration projects will be done on medical homes, gain sharing, medical liability, bundling, geographic payment variation, and accountable care organizations. The Patient-Centered Outcomes Research Institute will be established. The Center for Medicare and Medicaid Innovation will align payment incentives in areas of treatment planning and follow-up care planning with nationally recognized evidence-based guidelines for cancer care. There will be increased scrutiny and control over health insurer cost increases. The act calls for sustained funding for prevention and public health and the establishment of a National Prevention and Health Promotion Strategy and Council, and provides a new grant program for community prevention. Lifetime and certain annual coverage limits will be banned. Such caps have been a prominent reason for underinsurance and delays in seeking tests and treatment. SOURCE: Horton presentation (October 25, 2010). could detect unanticipated outcomes “before we have to go through the pain of a large clinical trial to find that out.” Mr. Weil noted that states could hamper effective implementation of ACA by not providing sufficient funding or by further limiting coverage. “All roads to implementation run through the states. If we are going to get this right, it’s not just a matter of rules at the federal level, but also imple-
OCR for page 40
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care mentation choices at the state level,” Mr. Weil said. For example, half the expansions in coverage that the legislation stipulates are in the Medicaid program, which states administer. “We know that, for cost containment reasons, states often set up administrative barriers that go beyond the eligibility criteria, and outreach is done at the state and local levels,” Mr. Weil said. This means that federal eligibility expansions essentially will not be implemented unless states make their eligibility systems work effectively, Mr. Weil said. States can also influence the continuity of care as low- to middle-income patients move from being covered by the Children’s Health Insurance Program or Medicaid to participating in a qualified insurance plan. States may or may not put continuity rules in those programs that specify benefit design and actual providers. “Continuity is going to be a huge issue, largely determined at the state level,” Mr. Weil said. He also pointed out that although the federal law changes how insurance is regulated, for example, by eliminating preexisting condition exclusions and by rating changes, it is up to the states to enforce those rules. “If states take those changes seriously and have the resources to do the oversight with health plans, we will see improvements. If they don’t have the resources, those changes will be on paper, but they won’t be in reality,” Mr. Weil said. State budgets and rules will also determine implementation of many of the public health measures the ACA stipulates, as well as quality standards and pay-for-performance rates. States are also responsible for the risk adjustment systems within the insurance exchange, which will ensure that health plans have appropriate incentives to cover people with higher costs. “We have tremendous potential for improvement in cancer care associated with the implementation of the Affordable Care Act, but effective implementation is going to rely heavily on the states. So as you are thinking about how to make a difference, how to achieve the potential, do not just look at the federal government, but also support the efforts going on within your states,” Mr. Weil said. One participant pointed out the lack of capacity and sophistication of some state health departments and legislatures and the need for strategies to influence implementation of the ACA at the state level. Yet as Dr. Glasgow and Mr. Weil both pointed out, an advantage of the state implementation process is that states often take the lead in public healthcare innovations with their experimental programs, whose results pave the way for universal implementation nationwide.
OCR for page 41
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care Mr. Weil noted that although many of the provisions of the legislation will be implemented by 2014, some will not be in effect until as late as 2021, so there is still the opportunity to engage in workshops or studies that would influence the implementation of the bill. “Think about the time line that health reform is under. Some things are already out of the chute, and other things have yet to happen,” Mr. Weil said. Dr. Ferrell added that “health reform is more than just shifting of the funding…. In each of these provisions there is also an opportunity for us to really rethink the quality of care that’s delivered to real patients and families.” Coverage Gaps There will be several potential gaps in coverage with the advent of the ACA that Dr. Horton pointed out. She noted that the National Breast and Cervical Cancer Early Detection program and similar existing programs will expire within several years. It is not clear if the ACA will be sufficient to extend the preventive care those programs provide in an environment of tight discretionary dollars. “How do you begin to make the case to continue other programs like this as needed?” Dr. Horton asked. While the ACA provides coverage of routine care costs related to clinical trial participation for state-regulated insurance and employer-sponsored plans, this provision is not applicable to Medicaid, although states may cover the routine costs associated with clinical trial participation through their own funding initiatives (Rosenbaum et al., in press). Dr. John Hohneker, senior vice president and head, Global Development and Integrated Hospital Care at Novartis Pharma AG, asked how to achieve uniform implementation of the legislation in this regard at the state level so that “cancer patients in California are treated the same as cancer patients in Iowa,” he said. Mr. Weil agreed that attention to this is warranted. Dr. Horton also pointed out that Medicaid will be expanded to cover all nonelderly, nondisabled citizens and legal U.S. residents with family incomes below 133 percent of the poverty level and will provide preventive care for those individuals. However, the ACA doesn’t require coverage of preventive screening benefits for adults in “traditional” Medicaid eligibility categories, which tends to comprise the poorest women who are caretakers of minor children and disabled nonelderly adults, creating a gap in coverage for this vulnerable population.
OCR for page 42
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care CLOSING REMARKS At the conclusion of the Summit, Mr. Kean provided a wrap-up of the day’s discussions and offered some thoughts for consideration. He noted that the concept of keeping the patient at the center of focus came up again and again. Many participants spoke about patient-focused or patient-centered services, about gathering patient perspectives on their experiences, and about patient empowerment. With regard to cancer prevention, there were some provocative ideas around diet, weight control, physical activity, and reducing tobacco use, he said. Many discussions focused on research opportunities, but there was also frequent mention of a lack of metrics, methodologic and analytic tools, and technologies needed to accomplish the goals identified. For example, health IT is often looked upon as the solution to many ongoing challenges in cancer research and care, but there are also many challenges associated with implementing effective, integrated IT systems. Many participants emphasized the need to both increase the quality and lower the cost of cancer care. In some ways, these goals may seem at odds with each other, but aligning incentives of patients, physicians, and payors could help to achieve both goals, Mr. Kean observed. At the same time, impending workforce shortages in oncology care and continuing disparities in access to health care among various populations and geographical regions will have to be considered as well, he noted. The ACA aims to address such disparities, but implementation of many provisions remains uncertain and will likely vary among the states. Mr. Kean noted that several participants suggested that the cancer field is in a position to lead the charge in trying to solve some of these difficult challenges, because of the infrastructure and mechanisms that are in place. At the same time, he noted that the cancer community is under incredible pressure to do more with fewer resources. Acknowledging these challenges, he called on Summit participants to work on concrete ideas to improve the quality of cancer research and care. REFERENCES ACS (American Cancer Society). 2010. Cancer facts and figures 2010. Atlanta, GA. Brookings Institution. 2011. 2010 Conference on clinical cancer research. http://www.brookings.edu/events/2010/1020_clinical_cancer.aspx (accessed January 10, 2011). Bruckbauer, S., J. Contreras, and P. Tuovinen. 2010. The 111th Congress winds down. NCI Cancer Bulletin 7(22):8.
OCR for page 43
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care Burns, D. M., C. M. Anderson, and N. Gray. 2010. Has the lung cancer risk from smoking increased over the last 50 years? Cancer Causes and Control, December 25. ———. 2011. Do changes in cigarette design influence the rise in adenocarcinoma of the lung? Cancer Causes and Control 22(1):13-22. CDC (Centers for Disease Control and Prevention). 2008. Smoking-attributable mortality, years of potential life lost, and productivity losses—United States, 2000-2004. Morbidity and Mortality Weekly Report 57(45):1226-1228. ———. 2009. Chronic diseases: The power to prevent, the call to control: At a glance 2009. http://www.cdc.gov/chronicdisease/resources/publications/AAG/chronic.htm (accessed January 6, 2011). Demark-Wahnefried, W., N. M. Aziz, J. H. Rowland, and B. M. Pinto. 2005. Riding the crest of the teachable moment: Promoting long-term health after the diagnosis of cancer. Journal of Clinical Oncology 23(24):5814-5830. FDA (Food and Drug Administration). 2010. Draft guidance for industry codevelopment of two or more unmarketed investigational drugs for use in combination. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM236669.pdf (accessed January 10, 2011). Goldberg, P. 2010. FDA to reorganize oncology office into four divisions by disease type. Cancer Letter 36(30):1-4. Hoffman, H. J., N. L. LaVerda, P. H. Levine, H. A. Young, L. M. Alexander, and S. R. Patierno. 2011. Delays in breast cancer diagnosis: Having health insurance does not eliminate race/ethnicity-associated delays in breast cancer diagnosis in the District of Columbia. Cancer. Published online February 24, 2011. IOM (Institute of Medicine). 2003a. Fulfilling the potential for cancer prevention and early detection. Washington, DC: The National Academies Press. ———. 2003b. Key capabilities of an electronic health record system. Washington, DC: The National Academies Press. ———. 2007. The learning healthcare system: Workshop summary. Washington, DC: The National Academies Press. ———. 2008. Knowing what works in health care: A roadmap for the nation. Washington, DC: The National Academies Press. ———. 2009. Beyond the HIPAA Privacy Rule: Enhancing privacy, improving health through research. Washington, DC: The National Academies Press. ———. 2010a. Extending the spectrum of precompetitive collaboration in oncology research: Workshop summary. Washington, DC: The National Academies Press. ———. 2010b. A foundation for evidence-driven practice: A rapid learning system for cancer care: Workshop summary. Washington, DC: The National Academies Press. ———. 2011. Finding what works in health care: Standards for systematic reviews. Washington, DC: The National Academies Press. Jha, A. K., T. G. Ferris, K. Donelan, C. DesRoches, A. Shields, S. Rosenbaum, and D. Blumenthal. 2006. How common are electronic health records in the United States? A summary of the evidence. Health Affairs 25(6):w496-w507. John, R. M., and H. Ross. 2010. The global economic cost of cancer. Atlanta, GA: American Cancer Society and Austin, TX: Livestrong. Kelley, A. S., and D. E. Meier. 2010. Palliative care—A shifting paradigm. New England Journal of Medicine 363(8):781-782.
OCR for page 44
The National Cancer Policy Summit: Opportunities and Challenges in Cancer Research and Care Lynge, D. C., E. H. Larson, M. J. Thompson, R. A. Rosenblatt, and L. G. Hart. 2008. A longitudinal analysis of the general surgery workforce in the United States, 1981-2005. Archives of Surgery 143(4):345-350. Morrison, R. S., J. D. Penrod, J. B. Cassel, M. Caust-Ellenbogen, A. Litke, L. Spragens, and D. E. Meier. 2008. Cost savings associated with US hospital palliative care consultation programs. Archives of Internal Medicine 168(16):1783-1790. NCAB (National Cancer Advisory Board). 2010. To create a strategic scientific vision for the National Cancer Program and review progress of the National Cancer Institute: Report of the National Cancer Advisory Board Ad Hoc Working Group. Bethesda, MD: National Cancer Institute. Neubauer, M. A., J. R. Hoverman, M. Kolodziej, L. Reisman, S. K. Gruschkus, S. Hoang, A. A. Alva, M. McArthur, M. Forsyth, T. Rothermel, and R. A. Beveridge. 2010. Cost-effectiveness of evidence-based treatment guidelines for the treatment of non-small-cell lung cancer in the community setting. Journal of Oncology Practice 6(1-7). PCAST (President’s Council of Advisors on Science and Technology). 2010. Realizing the full potential of health information technology to improve healthcare for Americans: The path forward. Washington, DC: Office of Science and Technology Policy. Rosenbaum, S., J. Lee, M. P. Chapman, and S. R. Patierno. 2011. Cancer survivorship and national health reform. In Health services for cancer survivors, edited by M. Feuerstein and P. A. Ganz. New York: Springer. Sher, D.J. Cost-effectiveness studies in radiation therapy. 2010. Expert Review of Pharmacoeconomics & Outcomes Research,10(5):567-582. Sima, C. S., K. S. Panageas, and D. Schrag. 2010. Cancer screening among patients with advanced cancer. Journal of the American Medical Association 304(14):1584-1591. Spettell, C. M., W. S. Rawlins, R. Krakauer, J. Fernandes, M. E. S. Breton, W. Gowdy, S. Brodeur, M. MacCoy, and T. A. Brennan. 2009. A comprehensive case management program to improve palliative care. Journal of Palliative Medicine 12(9):827-832. Temel, J. S., J. A. Greer, A. Muzikansky, E. R. Gallagher, S. Admane, V. A. Jackson, C. M. Dahlin, C. D. Blinderman, J. Jacobsen, W. Pirl, J. A. Billings, and T. J. Lynch. 2010. Early palliative care for patients with metastaic non-small-cell lung cancer. New England Journal of Medicine 363(8):733-742. Thompson, M. J., D. C. Lynge, E. H. Larson, P. Tachawachira, and L. G. Hart. 2005. Characterizing the general surgery workforce in rural America. Archives of Surgery 140(1):74-79. UnitedHealth Group. 2010. New UnitedHealthcare cancer care payment model to focus on best treatment practices and better health outcomes. http://www.unitedhealthgroup.com/newsroom/news.aspx?id=efeefe23-c20a-47b1-ad72-73bba875d46c (accessed January 5, 2011). Williams, T. E., Jr., and E. C. Ellison. 2008. Population analysis predicts a future critical shortage of general surgeons. Surgery 144(4):548-556. Wolin, K. Y., K. Carson, and G. A. Colditz. 2010. Obesity and cancer. Oncologist 15(6):556-565.