WASHINGTON—Against a backdrop of the beleaguered roll-out of the Affordable Care Act, the National Cancer Policy Forum (NCPF) of the Institute of Medicine (IOM) held a brainstorming session here to explore the most pressing topics facing the cancer community.
As NCPF Chair John Mendelsohn, MD, Director of the Khalifa Institute for Personalized Cancer Therapy at the University of Texas MD Anderson Cancer Center, explained, the goal of the day-long, jam-packed meeting was to spur dialogue and help the NCPF—which the IOM established in 2005—in its strategic planning. Prior to the meeting, the NCPF invited individuals and groups to submit suggestions for consideration.
In September, the IOM released the sobering “Delivering High-Quality Cancer Care: Charting a New Course for a System in Crisis” report, which describes a fragmented system plagued by soaring costs and which often does not meet patient preferences and that underuses palliative care and hospice services (OT 10/10/13 issue).
Speakers at the summit covered the spectrum of cancer research and care, from basic science to end-of-life services.
Mark McClellan, MD, PhD, Senior Fellow and Director of the Initiative on Value and Innovation in Health Care at the Brookings Institution, warned summit participants not to expect immediate quick fixes for the cancer care system from the ACA's expanded coverage and state insurance exchanges: The ACA launch “has been a bumpy process, but it is moving forward; this is going to take a few years to work out,” said McClellan, former Administrator of the Centers for Medicare & Medicaid Services and former Commissioner of the Food and Drug Administration.
He cited the potential dangers of adverse selection in the new insurance exchanges: the possibility that older, sicker people will sign up in high numbers, but not healthy younger people. “You really need a broad participation,” McClellan said.
NCPF member Otis W. Brawley, MD, Chief Medical and Scientific Officer of the American Cancer Society, the moderator of the session at the summit on cancer control and prevention, warned that as the ACA is implemented, many patients will be under-insured: “This will vary state by state as states that are fighting implementation will have greater disparities in access. We must watch for these worsening of disparities and make people aware of them early so they can be addressed.”
Similarly, the American Society of Clinical Oncology also warned that the ACA is unlikely to fill all access and insurance gaps for cancer patients. In a statement submitted before the summit ASCO urged careful monitoring of section 2709 in the ACA requiring insurance coverage of clinical trials (which the cancer community worked hard to have included in the law): “We should track implementation of this issue closely to ensure comprehensive implementation, particularly in light of the lack of federal regulations or guidance. What are continuing gaps in care and how can we fill that need?”
Basic Research, Enhanced Scientific Training, New Clinical Trial Models
The first high-priority issue discussed at the summit was basic research. William G. Kaelin, Jr., MD, Professor of Medicine at Dana-Farber Cancer Institute and Associate Director of Basic Science for the Dana-Farber/Harvard Cancer Center, said, “The science of basic and translational cancer research—the very foundation of high-quality care—must remain strong and be adequately funded. We must protect and nurture basic discovery in the public sector so as to complement applied research in the private sector.”
He also advocated building a sustainable model for scientific training compatible with a “properly sized and resourced biomedical research enterprise.” And, like many of the speakers at the summit, Kaelin stressed the need for information-sharing and partnerships between the public and private sectors. A priority issue, he said, is to “enhance exchange of information and reagents amongst public sector investigators and between the public sector and the private sector.”
Several speakers discussed the high-priority need for new cancer clinical trial models. As cancer therapies become more fragmented and targeted to ever-smaller populations, there will be a need to reuse the same drugs in different combinations and design multiple-indication therapy models, said Andrea Califano, PhD, the Clyde and Helen Wu Professor of Chemical and Systems Biology at Columbia University, where he is the founding Director and Chair of Columbia's Department of Systems Biology.
Both Califano and Razelle Kurzrock, MD, Senior Deputy Center Director for Clinical Science and Murray Professor of Medicine at the University of California, San Diego Moores Cancer Center, emphasized the need for trials earlier in the course of disease, as well as customized combinations of therapies. Kurzrock noted that while each cancer patient is different—a “malignant snowflake,” as she put it—it is not practical to do “N of one” clinical trials. Therefore, she said, it is becoming important to validate a treatment strategy, not just a drug or a biomarker.
A trial design that might make sense with the current rapid rate of progress in development of effective new cancer drugs is to study multiple similar new therapies using the approved treatment as the control arm, said NCPF member Richard Pazdur, MD, Director of the FDA's Office of Oncology Drug Products. For example, in the case of new drugs for ALK-positive lung cancer, new therapies could be studied against the ALK-inhibitor crizotinib in the control arm.
Pazdur also put in a plea for a renewed emphasis on safety. “We have a love affair, so to speak, with the efficacy issue,” he said. But safety is equally important, and just because a patient has a malignant disease it does not give a researcher carte blanche to test an investigational drug with no safety data. “We have to have some humility here when we approach these diseases,” he said.
‘Immunotherapy at a Tipping Point’
In a keynote address, Carl H. June, MD, Director of Translational Research at the Abramson Cancer Center at the University of Pennsylvania, said that immunotherapy is now at a tipping point and is poised to become an important new tool for oncologists. Citing FDA approval of the immunotherapy agents sipuleucel-T for prostate cancer and ipilimumab for melanoma, he predicted that the investigational immunomodulating drug nivolumab has the potential to greatly improve treatment of non-small cell lung cancer (NSCLC).
“I think it's fair to say that in a few years immunotherapy will be front-line therapy for NSCLC,” he said. June noted that in some cancers chemotherapy can be combined with immunotherapy to overcome drug tolerance. But he cautioned that multi-center immunotherapy trials are difficult, with complex manufacturing and liability issues and there is need for physician education on management of toxicities.
Curb Spiraling Costs
Several speakers at the summit cited the urgent need to contain the spiraling costs of cancer care. For example, said Lowell E. Schnipper, MD, the Theodore W. and Evelyn G. Berenson Professor in the Department of Medicine at Harvard Medical School and Clinical Director of Beth Israel Deaconess Medical Center, “Physicians have not exerted sufficient leadership in bending the cost curve. Physicians need to own more of this problem than we have traditionally done.”
Schnipper, Chair of ASCO's Task Force on the Cost of Cancer Care, praised programs such as the Choosing Wisely campaign, which helps physicians use medical resources prudently. He, along with several other summit speakers, urged curtailing active cancer treatment when it is likely to be futile.
He said that to help control rising costs, he also advocates allowing CMS to negotiate prices for anti-cancer drugs and other therapies. In prepared remarks submitted before the summit, ASCO urged a much stronger focus on cancer therapy cost issues, stating, “As $100,000 price tags become almost routine, cancer therapies will move beyond the reach of many patients, not just the un- or under-insured.”
Other Key Areas
Summit speakers also discussed the following key issues, among others.
* The desperate need for a much stronger commitment to cancer prevention, including tobacco cessation and reduction of high caloric intake and lack of physical exercise leading to obesity. Prevention also encompasses the need to treat premalignant conditions to prevent their progression to cancer, said William N. Hait, MD, PhD, Global Head of Research and Development at Janssen Research & Development, LLC. Prevention also includes full uptake of cancer prevention vaccines such as the HPV vaccine to prevent cervical and anal cancers, said Denise Galloway, PhD, Associate Director of the Human Biology Division at Fred Hutchinson Cancer Research Center.
* Better public education on cancer screening programs. The public needs more accurate information and education on the potential harms as well as the benefits of cancer screening, said Barnett S. Kramer, MD, MPH, Director of the National Cancer Institute's Division of Cancer Prevention). He pointed out the dangers of false reassurance, false positives, potential harms ensuing from the screening tests themselves (such as sepsis following a biopsy), anxiety, detection of a lethal cancer without changing the outcome, and detection of non-lethal cancers. When evidence-based screening tests are offered, it helps to have employer rewards and incentives to get employees screened, and consumer incentives through rewards programs offered by insurance companies, said Lillie B. Schockney, RN, MAS, Administrative Director of the Johns Hopkins Breast Center and a breast cancer survivor.
* Stronger emphasis on helping cancer survivors stay healthy. With 14 million U.S. cancer survivors, who are at known risk of late effects of treatment, cancer recurrence, and secondary cancers, more efforts need to be focused on survivor wellness, said Catherine M. Alfano, PhD, MS, Deputy Director of NCI's Office of Cancer Survivorship. “What we need is a new model of survivorship” that encompasses tobacco cessation, healthy eating, weight control, and exercise, she said.
* Full and wise use of e-health information technology, including novel applications of electronic health records, rapid learning systems such as ASCO's CancerLinQ, and distance-medicine pro that can monitor symptoms and improve treatment compliance via electronic reminders.