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NCCN Academy Tackles Post-Election Concerns, Real-World Evidence, Cost of Care, and Patient Distress

Katie Kiley Brown, Communications Manager, NCCN

On March 22, 2017, the National Comprehensive Cancer Network® (NCCN®) hosted its Academy for Excellence and Leadership in Oncology™: School of Pharmaceutical & Biotech Business in conjunction with the NCCN 22nd Annual Conference: Improving the Quality, Effectiveness, and Efficiency of Cancer Care™ in Orlando, Florida.

Post-Election Review: Cancer Care under a New Administration

From left to right: Mr. Burkholder, Mr. Garrett, Mr. Samuels, Mr. Simon, and Dr. Goodman.

On the eve of the scheduled vote in Congress to repeal and replace the Patient Protection and Affordable Care Act (ACA)—which was later postponed and eventually cancelled due to lack of support in the House—expert health care policy stakeholders gathered to discuss barriers to appropriate cancer care under the Trump Administration and the then proposed American Health Care Act (AHCA), challenges for the new U.S. Food & Drug Administration (FDA) commissioner, and the focus on value-based medicine.

Moderated by Clifford Goodman, PhD, The Lewin Group, the following panelists participated in the deliberation:

  • Randy Burkholder, Vice President, Policy and Research, PhRMA
  • Peter Garrett, Director, Office of Communications & Public Liaison, National Cancer Institute (NCI)
  • Marc Samuels, Founder and Chief Executive Officer, ADVI
  • Greg Simon, JD, Executive Director of the Biden Cancer Initiative, The Biden Foundation, and Former Executive Director, White House Cancer Moonshot

Upholding coverage for American citizens

Dr. Goodman opened the panel by noting the timeliness of the discussion given the proposed Congress vote on the AHCA and the up-to-the-minute changes being deliberated on the Hill, and Mr. Samuels provided a real-time update on the proposed AHCA legislation to that point in the morning. Panelists then discussed their major concerns with the AHCA.

The panelists cited several concerns, including loss of insurance coverage, disparities in access to quality care, and support for scientific innovation.

Mr. Simon noted that the White House Moonshot Initiative, in 2016, grew into a national movement for greater access to health care. “As part of this movement, we have to have access to insurance and health care. By insurance, I don’t mean tax credits or vouchers, I mean insurance. We must reduce the disparities in access to health care in this country,” he said. Mr. Simon pointed out that the single most important determinant to who survives cancer is who has health insurance (cite ACS study).

“One of the issues we have not addressed is that insured patients are underinsured, and there’s potential for that to continue [under the AHCA],” added Mr. Burkholder. He explained that there are core patient protections built into Medicaid, but it is not clear how lawmakers would uphold the quality of insurance in the individual and small markets under AHCA.

Mr. Simon continued that the working poor are the demographic who will suffer the most under the Republican plan. He explained that, if this group is kicked off of their exchange and is unable to gain access to Medicaid through expansion, they have nowhere to turn. Furthermore, he noted, if the working poor are outside of an urban area and do not have insurance, they will not have access to screening and early detection. Therefore, he noted, access to appropriate cancer care is out of the question.

Mr. Samuels added that, overall, the foundational screening, prevention, and access to early care was missing for this demographic until ACA reform. He also noted that, with the ACA, coverage increased for nearly 40 million men, as women and children are the majority of Medicaid beneficiaries.

Securing a future for scientific innovation

“What’s on my mind is not losing sight of research and the long-haul game,” said Mr. Garrett. “We have a lot of exciting things happening recently, and this is the result of a lot of exciting things happening in research.”

Mr. Garrett explained that since 2004 (with the exception of a special Congressional allowance in 2016) NCI has had a flat budget, which is resulting in a decline in purchasing power. “We can’t fund everything we would like to fund,” he said. With proposed budget cuts to the National Institute of Health (NIH)—of which NCI is a constituent—NCI’s funding capabilities potentially could be even further halted, noted Dr. Goodman.

Mr. Burkholder added that in order to strengthen premier scientific infrastructure in the United States, stakeholders must recognize the importance of private sector infrastructure that need to be upheld, as well. He explained that the private sector is ready to step in and has done so, especially with support for STEM (science, technology, engineering, and math) education.

“NCI recognized that we are part of the solution, but we are not the whole thing,” said Mr. Garrett, citing examples of collaboration between NCI and the pharmaceutical industry, including the drug formulary that cleared the way for investigators to access drugs for trials. Another is the well-known NCI MATCH (Molecular Analysis for Therapy Choice) trial—a large precision medicine trial for patients whose cancer has not responded to treatment. Within this trial, he explained, more than 7,000 patients have been screened, and the trial has a marked uptake in rural communities, thus breaking down disparities to access to personalized medicine. “[The MATCH trial] shows that’s possible when we bring science out into the communities,” said Mr. Garrett.

What’s in store for the new FDA Commissioner?

As of March 22, Congressional hearings for the FDA Commissioner under the Trump Administration had yet taken place, but, according to the panel, sights were set on Scott Gottlieb, MD.

The panelists agreed that, while he may face scrutiny for his previous ties to in the pharmaceutical industry, Dr. Gottlieb stood a good chance of confirmation and would lead FDA through evolution—not revolution.

“We need an agency that evolves as the science an innovation evolves,” said Mr. Burkholder.

We have the fastest cancer drug approval in the world, and the FDA has done a great job dealing with the complexities of medicine…What we need at the FDA is stability—that’s what the agency wants, said Mr. Simon. One issue of importance, he said, is the establishment of a strategy for approving companion diagnostics that would benefit by defining what drugs their cancers will respond to.

Upholding Value in Cancer Care

“You’re under a lot of pressure. What are you thinking about as far as promoting products that cost so much?” Dr. Goodman asked Mr. Burkholder.

“We are under pressure,” Mr. Burkholder responded. “But, that’s what people who go into research sign up for because they know they are signing up for a science career and these companies are derived for the ability to discover something newer and better.” There is also pressure about price and affordability, but these things happen with the patient in mind, he added. Mr. Burkholder explained that, when thinking about the individual patient, one must make sure to think about value, which includes adequacy of insurance coverage and ensure that we support value of the drugs, physician services, and diagnostics—evening that goes into the care of the patient.

 “How do we better communicate value to the patient?” asked Dr. Goodman.

Mr. Burkholder noted that NCCN has begun publishing the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) with NCCN Evidence Blocks™. “The next step is to build off of that for a tool that can better serve shared decision-making, including patient circumstances,” he said.

So how can we make this work?

In closing, Dr. Goodman asked the panel to consider the year 2022—and, against the odds proposed in 2017, the more than 20 million citizens covered under ACA reform did not lose their coverage during the Trump Administration. “What did we do right?” he asked.

Mr. Burkholder suggested a change in the current climate Washington, allowing for bipartisan decision-making where lawmakers reached across the aisle to find ways to compromise.

We looked at the science and followed where things are headed—focusing on how to best care for the growing number of cancer survivors in the country, suggested Mr. Garrett.

Mr. Samuels suggested a two-pronged approach where stakeholders found the right incentives for the “young invincibles” to buy insurance and where breakthroughs in science and better diagnostic coverage allowed for earlier screening and recognition of patient groups who would benefit from particular treatment.

“Republicans lose the house in 2018,” responded Mr. Simon.


Meet the NCCN Guidelines Panel Experts

The day’s second panel featured an in-depth discussion among NCCN Guidelines® Panel members who volunteer their time to

From left to right: Dr. Benson, Dr. Daly, Dr. Pfister, Dr. Plimack, Dr. Mesa, and Dr. Goodman. 

create this library, widely considered the standard of care in oncology in the United States.

The panelists included:

  • Al B. Benson III, MD, FACP, FASCO, Professor of Medicine, Associate Director of Cooperative Groups, Robert H. Lurie Comprehensive Cancer Center of Northwestern University; and Chair, NCCN Guidelines Panel for Colon, Rectal, Anal, and Hepatobiliary Cancers
  • Mary B. Daly, MD, PhD, FACP, Professor, Department of Clinical Genetics, Director of the Risk Assessment Program, Timothy J. Talbot Jr. Chair in Cancer Research, Fox Chase Cancer Center; Chair, NCCN Guidelines Panel for Genetic/Familial High-Risk Assessment: Breast and Ovarian; and Member, NCCN Guidelines Panel for Breast Cancer Risk Reduction and Breast Cancer Screening and Diagnosis
  • Ruben Mesa, MD, FACP, Chair, Division of Hematology & Medical Oncology, Deputy Director, Professor of Medicine, Mayo Clinic Cancer Center; Chair, Arizona Cancer Coalition; and Chair, NCCN Guidelines Panel for Myeloproliferative Neoplasms
  • David Pfister, MD, Chief, Head and Neck Surgery, Memorial Sloan Kettering Cancer Center; and Chair, NCCN Guidelines Panel for Head and Neck Cancers
  • Elizabeth Plimack, MD, MS, Chief, Division of Genitourinary Medical Oncology, Director, Genitourinary Clinical Research, Associate Professor, Department of Hematology/Oncology, Fox Chase Cancer Center; and Member, NCCN Guidelines Panels for Bladder/Penile, Kidney, and Prostate Cancers

NCCN Guidelines design

Dr. Goodman opened the session by asking Dr. Mesa to discuss his experience with NCCN. Dr. Mesa noted that the NCCN Guidelines for Myeloproliferative Neoplasms (MPN) are the most recent addition to the Guidelines library, adding that the recommendations are being published in an iterative process.

 “How does NCCN decide it’s time for new Guidelines?” Dr. Goodman asked Dr. Benson.

Often, the suggestion comes from investigators at the NCCN Member Institutions, said Dr. Benson. In diseases, prevention, or supportive care settings, experts will note where the science has evolved relative to treatment or symptom management, he explained. Moreover, he said that resources must be in place for a new panel, as maintaining NCCN Guidelines is an expensive proposition.

Dr. Benson also noted that in addition to new NCCN Guidelines, the panels have begun to split apart.

In the case of genitourinary (GU) cancers, said Dr. Plimack, the division of the kidney and testicular panels was a reflection of scientific progression as specialists garnered increased levels of expertise in each tumor site.

Dr. Daly explained a similar occasion with the creation of the NCCN Guidelines for Genetic/Familial High-Risk Assessment: Colorectal. “The availability of the expertise is really key,” she said. “Someone who spends their life treating the disease may not be up to date with the genetics.”

Seeking the best evidence

“Across the board, what sort of evidence is sought by an NCCN Guidelines panel?” asked Dr. Goodman.

“Of course, we all want the best evidence, which is randomized clinical trials,” said Dr. Daly. Furthermore, Dr. Daly added, when we have a limited trial, we look at similar trials to find consistency.

“One of the bars that we set is something that is peer-reviewed and published for an FDA approval,” said Dr. Plimack. She added that anyone may submit any data for consideration; however, the Panel members appreciate peer-reviewed data illustrating advances to the standard of care—especially for therapeutics.

The panels are looking for double-blind randomized controlled phase 3 trials, said Dr. Benson, but in many cases as the science evolves, these studies do not exist due to external factors such as smaller patient populations. For instance, he noted, with immunotherapy, experts have seen fairly striking results in small trials that are consistent with observations in other diseases. “We are clearly in an era now where we are looking at smaller and smaller subsets of patients, and we are going to have to review data perhaps differently than we have in the past,” he said.

“Certainly, we don’t have randomized clinical trials in all situations,” said Dr. Mesa. He went on to explain the profound importance of panel consensus for NCCN Guidelines recommendations. “One does not only need consensus among the expert panel who represent a core of the key opinion leaders in the field, but each of  these individuals is accountable to their colleagues in the same field at their institutions,” he said.

Dr. Goodman then asked the panel to discuss the role of diagnostics in the Guidelines.

“We look at diagnostics to divide patients into who would and would not respond to a particular drugs,” said Dr. Daly. She explained that the clinical trial population for these drugs often is heterogeneous.

“In prostate and bladder cancers, we are trying to get to where lung is today,” said Dr. Plimack. She explained that it has been known for years that EGFR will show if a patient’s cancer will respond to either immunotherapy or chemotherapy. Now, she noted, researchers look to such testing to guide enrollment into clinical trials.

“When I approach this particular area,” said Dr. Pfister, “I ask to what extent does the result change what we do?” He explained that, related to multi-gene panels, the benefit of knowing which treatment will best benefit the patient may outweigh the cost of the test.

Another important component in the NCCN Guidelines is imaging, said Dr. Benson. The NCCN Imaging Appropriate Use Criteria (NCCN Imaging AUC™) forces panels to look at imaging very carefully and be very specific, he said. “We are trying to give much more direction where imaging is helpful—and where it’s not—to discourage inappropriate use,” he said.

The role of real-world evidence in Guidelines recommendations

Dr. Goodman asked, “To what extent are you including real-world data in decision-making?”

“Generally, real-world retrospective data is not something we use because it’s prone to bias, but real-world prospective data is something we would consider. We place it all in context and form as decision,” said Dr. Plimack.

“We have fairly rigorous standards for reviewing data of any type,” said Dr. Benson.  Claims data have a lot of gaps, he noted. “I think most panels would view claims data with a great deal of skepticism. Nevertheless, if data are presented, we have to be rigorous,” he said.

Dr. Pfister said that there is good work being done looking at SEER-Medicare data that has revealed insights into mortality outcomes, and that there is a role for robust observations into alternative sources of information to help panelists make the best decisions regarding patient management. “We have to evaluate based on their scientific rigor,” he said.

Dr. Mesa explained that claims data may be helpful in rarer diseases. “There can be a tremendous amount of bias in retrospective data from large centers or NCI-designated centers. There are clearly limitations in claims data…but it can show what is being done in the bulk of U.S. practices,” he said.

Weighing affordability in recommendations

“NCCN instituted the Evidence Blocks recently, how is that working out?” asked Dr. Goodman.

The NCCN Guidelines with NCCN Evidence Blocks™ for MPN are still being finalized, noted Dr. Mesa. “It’s a very complementary set of information. There’s a spectrum of the weight of evidence and they provide more granularity in an interpretable format,” he said.

“Does the cost of a particular therapy impact the decision for inclusion in the Guidelines?” asked Dr. Goodman.

“No,” said Dr. Pfister. “We make recommendations independent of cost.”

If NCCN Evidence Blocks™ are a tool for initiating shared decision-making with patients, how well-equipped are oncologists to have that discussion? asked Dr. Goodman.

Dr. Benson explained that there are programs at medical schools now on how to incorporate economics into medicine.

Dr. Pfister added that these conversations tend to focus on drugs, but stressed that affordability goes far beyond drugs into imaging, surgery, antiemetics, and other areas of treatment.

“Affordability and cost to the patient is much different than affordability and cost to society,” said Dr. Plimack. She explained that, for someone without insurance coverage, the drug can often be attained free of charge. However, with an insurance plan, there may be a high-cost co-pay for a rather inexpensive drug, she said.

Dr. Mesa said that oncologists are heading in the right direction with training for cost discussions. “There will never be enough time so that the provider is the expert,” he said. The treatment team—physicians, nurses, financial counsellors, social workers—need to huddle together about the best options for each patient, he explained.

In closing, Dr. Goodman asked the team what the pharmaceutical and biotech industry should be looking for as the NCCN Guidelines program evolves over the next two to five to 10 years.

“The science will drive the evolution of the guidelines and the science is moving into patient selection criteria based on biological principles,” said Dr. Benson.

“Where the science is moving towards personalized medicine, we need cooperation and enthusiasm from the companies who develop therapies for those used in smaller subsets of patients,” said Dr. Daly.

Dr. Pfister noted that there will be increased scrutiny and analysis along the line of what has been done with drugs, applying it to other parts of the continuum of care.

Dr. Plimack said that, with expanding basket trials in therapeutics, it will be hard to consider non-randomized trials without a comparator arm.

Dr. Mesa added that it would be important to see “evolving quality metrics based on Guidelines so there is greater accountability in diseases that have not yet been represented in the first pass in applying quality metrics in oncology.”


Perspectives on the State of Cancer Care from Patients to Providers to Employers

The day’s third module focused on patient access, benefit design, and incorporation of value-based care in oncology. The panel, moderated by Dr. Goodman, included the following experts:

  • Marcus Neubauer, MD, Vice President and Medical Director, Value-Based Reimbursement, McKesson Specialty Health
  • Lee Newcomer, MD, MHA, Senior Vice President, Oncology and Genetics, UnitedHealthcare
  • Laurel Todd, Vice President, Healthcare Policy & Research, Biotechnology Innovation Organization (BIO)
  • Lee Tomlinson, Patient and Healer Advocate, and Founder, The C.A.R.E. Effect Movement
  • Clorinda Walley, Executive Director, Good Days

Patient protections in health care reform

Dr. Goodman opened the day’s final module by asking the panelists to discuss their concerns about the new administration and the proposed AHCA’s effect on patient access to care.

Ms. Walley discussed the fact that, at the time of its implementation, ACA gave the illusion of coverage when, in actuality, the number of uninsured increased. “My concern is that we give adequate coverage,” she said. The groups that will suffer the most under the new proposed rule are  chronically ill patients, she added.

“The benefit of being an innovator is that you can work within any environment, but our biggest concern is patient access,” said Ms. Todd. From the biomedical technology standpoint, she explained, predictability is most important as the manufacturers she represents require stability over time to bring innovation to market and get it into the hands of patients.

Dr. Newcomer agreed: “If we could have stability and predictability, we would all benefit…It would be great if we could get a decision point that says, ‘These are going to be the rules and they will continue to be the rules so you can plan.’” There are going to be changes, Dr. Newcomer predicted.

Mr. Tomlinson said he is terrified of the proposed AHCA. Mr. Tomlinson is a survivor of throat cancer, who, after treatment and being unable to work for a prolonged period, was forced to claim bankruptcy despite having a successful career. Mr. Tomlinson expounded on the profound effect that a cancer diagnosis has on  family, career, and budgets and explained there are costs beyond treatment that a family must contend with.

“There are insured and uninsured, but there is also underinsured,” said Dr. Neubauer. “Drugs are expensive, but so are imaging, radiation, surgery, and procedures, and what we’ve seen with exchange programs are very high deductibles,” he added. Dr. Neubauer explained that even if deductibles on the exchange and through private payers are not that high, out-of-pocket costs are still making treatment unaffordable. “When I practice, I do the best I can to explain the cost up front,” said Dr. Neubauer, who still cares for patients one day a week.

“How are you seeing financial toxicity?” Dr. Goodman asked Ms. Walley.

“Generally speaking, about 30% of insured individuals can’t afford their out-of-pocket costs,” she said. Good Days, which provides financial assistance for patients’ treatment and travel needs, as well as assists patients in finding appropriate health insurance, works with about 750,000 patients per year with an average household income of $39,000, she explained.

At what cost treatment?

To help combat the high-cost of new, innovative treatments, manufacturer-sponsored patient assistance programs have been put in place, explained Ms. Todd. She noted, however, that with the coupon system, patients on Medicare and Medicaid are unable to participate in the programs; moreover, certain states are also blocking use. Manufacturer coupon programs provide patients with drugs at a discounted rate, making regimens more affordable for patients who qualify for the benefit.

Benefit design and how much of the cost is being shifted towards patients has a lot to do with the ability to pay for treatment. In order to deal with the cost of care, industry has to look across the totality of care, said Ms. Todd. “What we have is a health care cost problem—not a drug cost problem or even a cancer cost problem.”

Dr. Newcomer challenged the benefit of industry-sponsored patient assistance programs for newer, pricier drugs that provide little benefit over less expensive alternatives. He said that, in such cases, the system could pay up to $90,000 more than necessary. Alternatively, if the program provides assistance for a regimen that provides unique benefit, the program is very helpful, he said.

“If you’re in the business of trying to cure or at least lessen the impact of cancer, recognize that the cost may keep someone from taking advantage of all of your hard work,” said Mr. Tomlinson.

Managing the psychosocial burden on patients

Dr. Goodman asked the panelists to touch on the psychosocial encumbrance to proper care, noting that financial toxicity is not the only barrier to access and successful treatment.

Ms. Walley noted that the state of mind of a patient can modify or impact whether they heal or not. “We’re finding more and more that patients with psychosocial support needs can’t find it, and they’re suffering beyond what the disease is doing to them,” she said.

The World Health Organizations (WHO) says that provision of psychosocial care in patients with cancer is essential to adequate treatment, Mr. Tomlinson added.

“I can’t tell you how close I was to suicide,” he said. “I was so despondent and alone.” Mr. Tomlinson added that he finally found the help he needed through the Cancer Support Community—in group therapy he was able to connect with other patients who had a “shared language”, and he also sought individual counseling.

Today, with such programs as the Oncology Care Model (OCM), practices are driven to provide psychosocial services and practices are expected to hire patient navigators and counselors, said Dr. Neubauer. At large academic centers, these services often are included, but work needs to be done for reimbursement of such services in the smaller community settings, he added.

Dr. Newcomer added that the episode-based payment model, which has been piloted for a number of years by UnitedHealthcare, reimburses groups for increasing quality of care while lowering cost. This model, an alternative to fee-for-service design, includes aspects such as patient navigators and other patient-centered care that improves quality and outcomes.

Value in real world data?

Traditionally, said Dr. Goodman, when decisions are made in clinical care, coverage, and payment, the data came from large clinical trials. Today, how are you using new data sources and how is that changing your decision-making? he asked.

Drs. Neubauer and Newcomer discussed tools that UnitedHealthcare has implemented, including the aforementioned episode-based care model and a newer prior authorization tool, that allow providers to look at real-world data based on particular patient profiles treated with particular regimens. This data complements shared decision-making with patients, allowing them to see how other patients in similar settings faired with treatment, they explained.

Ms. Walley said such data would definitely be beneficial for patients, as long as it is presented in a clear, understandable way.

“My ability to analyze such data may have been compromised, but I at least would like to be asked,” said Mr. Tomlinson.

Next steps in closing the gap

In closing, Dr. Goodman asked the panelists, “What do you think you can do next that will help close the chasm between oncology breaking away from the people who can’t afford it anymore?”

With regard to Good Days, Ms. Walley said, “Education and awareness—our role will be helping navigate [patients to an appropriate pathway.”

“I’m a believer in value-based care,” said Dr. Neubauer. “I think the transition needs to complete from fee-for-service models to value-based care.”

Dr. Newcomer noted the importance of making information and data available to patients to help them make informed decisions about their care.

“Embrace the data and meaningfully demonstrate the value that any given therapy provides,” said Ms. Todd. “For me, as a trade association, breaking down the barriers—the hurdles—that allow for that to happen.”

About NCCN Academy

Pharmaceutical and biotechnology professionals are given the rare opportunity to view the oncology space, its future and its current operational issues from the provider and payer perspective. Participants will improve their working knowledge of key business, policy, coverage, reimbursement, informational, and operational issues in oncology and will gather valuable insights material to developing effective strategies for navigating the various constituencies in cancer care. Through this interactive program, pharmaceutical and biotech professionals will learn from key stakeholders what they view to be the most pressing issues in oncology today and how to apply this knowledge to create mutually-successful working relationships.

The next NCCN Academy is scheduled for Tuesday, July 11, 2017, at The Rittenhouse in Philadelphia, PA. For more information, visit NCCN.org/academy.