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National Comprehensive Cancer Network

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NCCN Academy Brings Together Experts to Deliberate Challenges and Opportunities for Patient Access, Collaboration, and Guidelines Development

By Katie Kiley Brown, Manager, Corporate Communications, NCCN

On July 11, 2017, the National Comprehensive Cancer Network® (NCCN®) hosted its Academy for Excellence and Leadership in Oncology™: School of Pharmaceutical & Biotech Business in Philadelphia, Pa.

The day’s first session, Ensuring Development and Access to Innovative Therapies, featured the following panelists:

  • Niesha Griffith, MS, RPh, FASHP, Vice President, Cancer Services, West Virginia University Health System
  • Ohad Oren, MD, Department of Internal Medicine, Hospital of the University of Pennsylvania
  • Alex Thole, Head of Oncology, Sales & Marketing, Sandoz Biopharmaceuticals
  • Patricia Wolfangel, Vice President, Xcenda, AmerisourceBergen

Dr. Goodman began the module on the topic of formularies and the challenges that facilitators face when making decisions surrounding immunotherapy and combination therapies.

Ms. Griffith said that a lot has changed with formularies. While cost, efficacy, and safety were always considerations, she explained, now centers need to consider whether they will be paid for the drug or, if a patient cannot afford it, is there support available to them. Moreover, she noted the difficulty in obtaining payer coverage for off-label drug use.

Bringing immunotherapies into the mix is going to be a true challenge for payers, said Dr. Oren. “For conventional therapies, we have decade’s worth of experience with the side effect profiles. That’s a challenge for the payers as they bring on new immunotherapies—we don’t have the data for immunotherapy-related auto-immune toxicities,” he said.

As stakeholders, Dr. Oren explained, we have to do things somewhat blindly at first, but the “holy grail” will be long-term surveillance.

“Our pharma colleagues in the room are really looking to define value. What type of evidence do they need to bring you?” Dr. Goodman asked Ms. Wolfangel.

“We have gateways with the 21st Century Cures Act,” said Ms. Wolfangel, adding the importance of getting value studies into the hands of the payers in a timely manner.

“Defining value in these circumstances is very challenging…are you seeing this as another hurdle?” Dr. Goodman asked Mr. Thole.

“I think it’s an opportunity for us,” he answered. The task for the formulary committees is measuring value and understanding that there are multiple stakeholders. With biosimilars, he said, there is a lot of education, but there is a wealth of data that we have to make available to the committees. “We give formularies the same information we give FDA,” he said.

Dr. Goodman then moved the discussion toward the use of value frameworks in oncology. “At this point, how relevant are they?” he asked the panelists.

“They’re in their infancy,” said Ms. Griffith. “Value frameworks are something we refer to, but it’s not something that we use for decision-making.”

“What I see [with value frameworks] is a tremendous effort put forth to develop these responses,” said Ms. Wolfangel. “We are on a learning curve, and it’s pretty steep, but the level of effort being put into it is tremendous.”

Mr. Thole added that the industry has a lot of experience with efficacy and safety, but the challenge is where to include the economics. He added that developers of the value frameworks must be thoughtful about the customer and how they are going to use it.

“I look forward to the future when we can have the value framework in health information technology at the point of care,” said Dr. Oren. “NCCN Evidence Blocks™ do a terrific job of truly integrating the important parameters that impact a patient’s future,” he said.

Moving from value to quality, Dr. Goodman asked the panel to consider quality measurement in oncology.

“In the United States, it’s a long time coming. We need to take quality into consideration, but I do not believe it’s being used for decision-making today,” said Ms. Wolfangel.

Mr. Thole said, “Developing a value-based strategy—while it will take a lot of work—is the best strategy.

Creating a shared-risk model pharma companies can join is going to require much more active participation from manufacturers, explained Dr. Oren. He added that treatments with truly unique mechanisms of action would translate into superior outcomes for patients.

Dr. Goodman asked the panel to differentiate between the various measurements when contracting in oncology.

“The best metric, of course, is overall survival,” said Dr. Oren. Oftentimes, progression-free survival is a value measurement, because overall survival is not available for many new interventions, he said.

“It’s a bright future, but a long horizon,” said Ms. Wolfangel. “We need to make thoughtful trade-off decisions about where to put our resources.”

“The minute you start thinking about value and bringing a therapy to market you need to think about value-based contracting and frameworks,” said Mr. Thole.

“We need to make sure we are looking at what’s important to the patient, too,” said Ms. Griffith.

The panel deliberated about the best approach to defining value for single agents within combination therapies.

“We don’t know the outcome versus toxicity of the single agents of a combination therapy,” said Dr. Oren. “In the absence of robust literatures, there is no answer…we are not there yet.”

Will the introduction of biosimilars to the market create a meaningful impact on cost and patient access, asked Dr. Goodman. 

Mr. Thole explained that the introduction of biosimilars has injected a 15% savings into the market. According to Mr. Thole, the FDA spends a lot of time with industry to define the evidence levels necessary for biosimilars. “Most of the work you see started before the agency formalized their guidance. At the same time, we recognize that the agency is a critically important stakeholder, but so are the providers and patients.”

“There has to be some patient education around biosimilars,” said Ms. Griffith. People do not have a good understanding about the clinical and scientific information that it takes to get a drug approved, she said. “From a patient perspective, when I think about biosimilars, anything that is going to decrease cost is a good thing,” she added.  

Ms. Griffith also mentioned concern about payers’ reactions to biosimilars on the market, noting that her pharmacy will only carry one biosimilar agent based on what the payers will reimburse.

Mr. Thole discussed the fact that the biosimilar production process has been happening for decades across different manufacturing sites, explaining that each production site is, essentially, manufacturing a biosimilar to the original. “[We’ve seen] 375 million days of patient experience across 75 countries with the same outcomes as the originator,” he said.

“We are living long on these fabulous therapies,” added Ms. Wolfangel. “We also have to think about the longer view.” Longer progression-free survival opens patients up to financial toxicity and treatment teams need to be prepared to counsel patients, she explained.

In closing, Dr. Goodman asked the panelists what advice they have for industry to ensure patient access to innovative therapy. 

“We have to start preparing for the future,” said Ms. Griffith. She shared that the value-based payment model is “here to stay”. “My other really important message is that one has to consider the patient.”

Looking at long-term innovation, Dr. Oren stated that industry must not only achieve fantastic achievements in remission, but also improve quality of life for the patients.

Industry must put the same effort into communicating value as they do communicating evidence-based safety and efficacy, added Mr. Thole.

“If you’re in the value space, you’re in a good place,” said Ms. Wolfangel.


The second module, titled, Meet the NCCN Guidelines Panel Experts, featured the following panel:

  • Deborah K. Armstrong, MD, Professor of Oncology, Professor of Gynecology and Obstetrics, The Sidney Kimmel Comprehensive Cancer Center of Johns Hopkins, and Interim Chair of the NCCN Guidelines Panel for Ovarian Cancer
  • Patrick Brown, MD, Associate Professor of Oncology, Assistant Professor of Pediatrics, Director, Pediatric Leukemia Program, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, and Co-Chair of the NCCN Guidelines Panel for Acute Lymphoblastic Leukemia
  • Joan McClure, MS, Senior Vice President, Clinical Information & Publications, NCCN
  • Mary Lou Smith, JD, MBA, Co-Founder, Research Advocacy Network, and Member of the NCCN Guidelines Panels for Breast Cancer and Breast Cancer Screening

Dr. Goodman opened the day’s second panel with a discussion about evidence submissions to the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) and the panel members’ expectations.

Dr. Armstrong explained that most of the panel members are practicing at academic institutions, so most of the submissions are not surprises. However, she noted that the Ovarian Cancer panel prefer that submissions include peer-reviewed publication.

“The gold standard is peer-reviewed clinical trial data for therapeutics,” explained Dr. Brown. For a therapeutic agent to be included within the Guidelines, he said, it requires FDA approval in some indication, but not necessarily the indication for which it is recommended in the Guidelines.

“I look very favorably when a stakeholder submits a well-written summary of the information, but it has to be supported by data…the level of evidence is something we take very seriously,” said Dr. Brown.

Ms. McClure explained the levels of evidence used within the NCCN Guidelines®, noting that Level 2A data is the default. 

On the subject of evidence, Ms. Smith noted that data is a challenge within the patient community, as well. Recently, Research Advocacy Network hosted a focus group and a major concern for patients was understanding how the clinical trial participants compared to the general patient population. According to Ms. Smith, patients have reached a level of sophistication where they are calling for trials to better reflect their general population.

Dr. Armstrong added that patients are not necessarily interested in “statistically significant” findings typically reported in trial outcomes data, but instead, they are interested in overall survival benefits of a therapy.

“How does this thinking get included in the Guidelines development process?” Dr. Goodman asked Ms. Smith, who is the longest serving patient advocate on an NCCN Guidelines panel.

“First of all, you have to have the patient at the table,” she answered. Ms. Smith discussed the importance not only of the Guidelines development, but insisted that it is imperative to get the word out to the public about specific NCCN Guidelines updates.

“Patients will not make decisions until they see what the Guidelines say,” said Ms. Smith. “Now we have the patient guidelines, which can include information that are not needed in the [clinical] algorithms.”

Dr. Brown and Dr. Armstrong discussed the difficulty in making recommendations for patients with recurrent diseases, as well as across different demographics, including age, comorbidities, and prior treatments.

“When the Guidelines first came out, we had a statement that, for patients with two or more treatments, best supportive care was the care options. But then, in our clinics, we realized that this was not the real-life situation,” said Dr. Armstrong. She explained that the typical patient with metastatic disease would receive more than three or four therapies with a good performance status throughout the course of the disease. The Guidelines recommendations were changed accordingly, but she explained that the best supportive care should always be an option.

Ms. McClure explained that big data might help in predicting patient performance across different disease and demographic subtypes in the clinical setting. However, she noted that there are inherent biases in how oncologists treat patients with metastatic disease.

Dr. Brown added that specific patient characteristics and variables play a huge role in determining treatment. Such variables include demographics, molecular information about the tumor, molecular information about the patient, patient age, and patient fitness, as well as the patient’s age—especially in diseases like ALL where diagnoses span the full spectrum of age.

“Where does personal judgment and experience weigh in when making recommendations?” asked Dr. Goodman.

“Ultimately the provider’s judgment and the patient’s wishes are what drive treatment decisions,” replied Dr. Brown. “The goal of the Guidelines is not to replace judgment and preference…I would say they are decision-support.”

“The NCCN Guidelines have a statement in them that they should NOT replace clinical judgment,” said Ms. McClure. “When looking at large patient populations, we see that 80 to 85% are treated on Guideline. That’s a reasonable benchmark to look for.” Ms. McClure explained that the Guidelines cannot pertain to every patient and that there are small subsets of patients for whom the Guidelines are not appropriate.

“Where is the role of biomarkers in developing the Guidelines?” asked Dr. Goodman.

Dr. Brown explained that there is a necessity for clinical information and trials data for a biomarker to be included in the NCCN Guidelines recommendations, as well as a link to an improved outcome. “There is a page in the Guidelines within the Diagnosis and Work Up section that includes biomarker tests recommended to have the complete understanding of a person’s disease, he said, noting the added importance of biomarkers in determining prognosis and risk stratification.

“Biomarkers are lab-developed tests so they’re in a different category, unlike therapeutics where there is a huge infrastructure of regulation—that is still in development,” he added.

Dr. Goodman asked Ms. Smith to discuss her experience as a patient advocate on an NCCN Guidelines panel. “Can you assure me that if I were to come on as a patient advocate that I would be taken seriously?” he asked.

“Yes,” she responded. Ms. Smith explained that patient advocates need to be prepared and know what the treatment recommendations are, as well as connect to the research.

“I see my role as bringing the patient experience to the panel. To do that, I’m to help the panel members understand what the questions are that the patients have. I have been very well-accepted by the other panel members,” she said.

Dr. Goodman switched the topic from the panels to the Guidelines recommendations. “Was there ever a situation where the Guidelines were more restrictive than FDA?” he asked Ms. McClure.

Ms. McClure explained that there indeed have been cases of NCCN restriction greater than FDA. For example, she said, FDA may approve a therapy for all patients with metastases for a specific cancer, but the NCCN  panel may restrict the recommendation only to patients with good performance status. “If toxicity outweighs the benefit, they may restrict,” said Ms. McClure.

In closing, Dr. Goodman asked the panelists to discuss what is most important and unique about the NCCN Guidelines.

The Guidelines are credible and open to change, said Ms. Smith.

“Data, data, data,” said Ms. McClure. “The more and better the data, the better the outcome is likely to be.”

Dr. Brown noted that stakeholders must be transparent as the oncology landscape changes, and encourages outside parties to use the NCCN Guidelines submission process to provide data-driven recommendations to the panels.

Dr. Armstrong echoed her fellow panelists, noting that the NCCN Guidelines are a fluid process, and recommendations can be reconsidered if data improve.


The third panel of the day, Patient Advocacy Update: Policy, Programs, and Effective Collaboration, featured the following leaders in oncology patient advocacy:

  • Karen R. DeMairo, BA, MHSA, Executive Director, Education & Integration, The Leukemia & Lymphoma Society
  • Elaine Grobman, Chief Executive Officer, Susan G. Komen Philadelphia
  • Marialanna Lee, Senior Director, State Government Affairs, The Leukemia & Lymphoma Society
  • Katherine Sharpe, MTS, Senior Vice President, Patient and Caregiver Support, American Cancer Society
  • Kirsten Sloan, Vice President, Public Policy, American Cancer Society Cancer Action Network, Inc.
  • Meryl Weinreb, BA, MA, Chair, Education & Public Policy, Susan G. Komen Philadelphia

Dr. Goodman opened the discussion by asking the panelists to consider the burden of cost on patients with cancer.

“Patients are no longer a captive audience,” said Ms. Sharpe. She explained that with outpatient treatment, the cost of treatment quickly grows when co-pays are charged each time a patient walks in the door.

Cost burden has a direct impact on access to care and patient outcomes, said Ms. DeMairo. If a patient cannot afford a co-pay, they may not see the doctor; if the patient cannot afford the medicine, they may not fill their prescriptions, she said. For some patients, she explained, “the bottom line comes down to whether to buy the meds or pay the co-pay or pay the electric bill and buy groceries.”

“All of our organizations have been collaborating on policies that are in the patients’ best interests,” said Ms. Lee. “The ACA made huge strides in taking cost from an insurmountable barrier to something that patients still have to think about, but there are protections that keep it from being a complete barrier.”

Ms. Weinreb explained that the way a drug is administered is becoming another barrier to patient access. Because a large number of therapies in the pipeline are oral, the current reimbursement structure would place them under the pharmacy benefit rather than a patient’s medical benefit, she said. With co-pay as high at 30%, pharmacy benefit exposes patients to a greater cost burden, she added.

To combat this issue, there are a number of co-pay programs offered by patient advocacy organizations, Ms. DeMairo said.

Stakeholders have to look beyond cost of care, explained Ms. Grobman. “We’ve spent years trying to educate the population to get into the doctors’ offices earlier so that they don’t present at stage III or IV. There are women—thousands of them—who are penalized by where they live,” she said. “Those women can’t afford to listen. They don’t have the transportation. They don’t have the babysitters. They don’t have the food…We have the best care possible here in the community. We need to get them into the clinic to get that care and get it earlier and keep costs down.”

Ms. Sloan explained that there are Medicare/Medicaid patients that cannot use co-pay assistance programs, so their options are lower than others’. When ACA was enacted, she said, it set into motion a system to keep costs down, while eliminating life-time maximums. Now, she said, the system is moving away from co-pays to coinsurance, which is percentage-based. “Now, coinsurance for a $100,000 therapy is 20% instead of a $20 co-pay.”

Ms. Lee added that this move is happening in employer-sponsored programs, as well.

“I think that we’re all in this together,” said Ms. Sharpe, who called on the patient advocacy groups and industry to work together to help with what the patients are experiencing. “There is nothing more devastating then working with a patient and knowing that there are absolutely no financial options available.”

“We are having the conversation on repeal and replace [of ACA]. Can you start us off by commenting on the impact that would have on the cancer community?” Dr. Goodman asked Ms. Weinreb.

“We really don’t know what they’re doing at this point,” she responded. “A lot of this is a mystery. Clearly everyone is terrified. We know what it was like before ACA, and it was not a good situation.” She explained that repealing the essential health benefit would be devastating, especially for patients with cancer and survivors.

Ms. Sloan called on both the patient advocacy groups and industry to work together to help show policymakers how interrelated development, access, and patient outcomes are. “All of us as stakeholders need to help policy makers understand that the protections are critical to ensure that patients get the care and services they need. There is no question that the ACA needed to be improved. We have to work together to make these treatments affordable,” she said.

“There is no one entity or one stakeholder group that’s going to be the one to carry the burden of reducing cost of care,” said Ms. Lee. “We are representing who is at the very heart of the cancer care system…we shouldn’t do this without the patients.”

Dr. Goodman asked the panel to discuss consideration of the ‘Right to Try’ legislation.

“I can tell you that all of the groups on the stage have signed a set of principles—the idea is that it’s a very difficult issue because what’s at the heart of these issues is that we help patients to get access,” said Ms. Sloan.

Panelists agreed that there is a challenge in weighing patient access to innovative therapy when they have exhausted other options with the risk of allowing patient access to drug that has not gone through extensive clinical trials—both for the patient and for the innovators.

Dr. Goodman asked the panelists to describe some of the impactful collaborations their organizations have had with industry.

Ms. Sharpe answered that the American Cancer Society has worked with several pharmaceutical companies on initiatives to better engage and acclimate patients around their care which have resulted in pilot programs around breast and lung cancers.

Ms. DeMairo described a master trial that LLS has begun for patients with Acute Myeloid Leukemia. For the trial, she said, patients who present in the ER newly diagnosed with AML undergo genomic testing and are then put into one of a number of arms that include drugs from a number of different manufacturers.

Another project, she explained, to reach out to communities and develop relationships with national black churches to raise awareness of Multiple Myeloma in black men in the United States.

“I am heartened by the pharmaceutical companies who are targeting these particular patient populations,” said Ms. Sharpe. “We all want to do this work, but without the resources, it’s a challenge. Support from industry enables us to work within the medical home and get them access to what they need.”

The panel discussed the fact that patients have trouble finding information about available clinical trials.

“One of the services that LLS offers is a clinical trial search center,” said Ms. DeMairo. Presently, she explained, there are two nurse navigators who work with newly referred patients, review their medical records, examine financials, and find possible trials for them.

“We have also asked to review consents that the company is putting together for a particular trials,” said Ms. DeMairo. She said that it is important to have an advocacy expert review consents, as they are another barrier to access for patients.

“How do you manage relationships with pharma so as not to seem biased?” asked Dr. Goodman.

“Our relationships with pharma are based on advancing our missions,” said Ms. Sharpe.

“I have found pharma to be extremely honorable partners,” said Ms. Grobman.

In closing, Dr. Goodman asked the panelists, “If you could enact a policy change where you would partner with industry or seek industry to further your organization’s mission, what would that one policy change be?”

Ms. DeMairo answered that providing access is key.

Ms. Grobman called for industry to stand with the advocacy communities in the community to reach patients who do not have access or cannot afford access.  

“We need to put our best minds together to figure out how to take it to the next level of value,” said Ms. Lee.

Ms. Sloan called on industry to keep working on the new innovations. “There is a lot that we have to deliver to make sure that the patient is at the center of the care.”

Ms. Weinrab discussed the importance of the oncology medical home model, as well as educating primary care physicians about survivorship issues to ensure that survivors receive quality care long after cancer treatment.

“The solution centers around access,” said Ms. Sharpe. “Having a diagnosis is hard enough. Not being able to access the care you need shouldn’t 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 will be held in conjunction with the NCCN 23rd Annual Conference: Improving the Quality, Effectiveness, & Efficiency of Cancer Care™ on March 21, 2018, at the Rosen Shingle Creek in Orlando, Fla.

For more information about NCCN Academy programs, visit NCCN.org/academy.