Moving away from a fee-for-service (FFS) reimbursement model to one that rewards value is no small undertaking for a busy practice. As the second day of Patient-Centered Oncology Care

 opened on November 17, 2017, moderator Margaret O’Grady, RN, MSN, OCN, FAAMA, administrative director of the Oncology Service Line at Abington Memorial Hospital, Jefferson Health System, said it’s important to see the best practice methodologies.

In cancer care, that means understanding how practices are adapting to the Oncology Care Model (OCM), which is now in the second year of its 5-year run under the Center for Medicare and Medicaid Innovation.

 Lalan Wilfong, MD, medical director for quality programs at Texas Oncology, shared how adapting to the OCM caused Texas Oncology to dig deeper into what it did well, where it could improve, and where variation and excess staffing had crept in, given that the 420-physician practice had grown through acquisitions.

Wilfong outlined the OCM basics: the model operates in 6-month episodes, which can be repeated, with Medicare paying a services fee for qualifying patients as well as shared savings. Wilfong went through the required OCM elements:

Offering core functions of patient navigation

Documenting a care plan, based on nationally recognized clinical guidelines

Use of quality metrics for shared savings and claims-based metrics

Documentation of care for pain, depression screening, and certain medications 

When Texas Oncology first signed on for OCM, Wilfong said some responded, “What are you doing to me?” But others embraced it.

“What this forced us to do, for the very first time in our practice, it forced us to sit down and think about how we take care of our patients. We always thought we did a good job,” he said. The practice was growing, but oncology was changing so quickly. “What we never really thought through was, ‘Are we doing this well? Are we taking care of our patients well?’”

The number of OCM requirements forces the practice physicians to meet frequently to make sure they are meeting them, a process that has led to many changes that have helped patients, Wilfong said, offering several examples:

Texas Oncology’s physicians always took calls, but what did this requirement really mean? How did it correlate with reducing hospital admission and emergency department (ED) visits? The practice gained access to its CMS data feed to see which patients were ending up in the hospital, and followed that up with 2 things: it set a goal of reducing hospital admissions by 5%, and it said nurses could not refer patients to the ED without a physician’s order. “We hold our physicians accountable for their hospitalization rates. We share that widely in our organization,” Wilfong said.

“Most physicians are fairly anal-retentive people. They were used to being the A+ student in their classes,” he said. “When I give them a score card that shows that they’re red, not green, they don’t tend to like that very much.”

Texas Oncology also redeployed nursing staff to return calls from high-risk patients within 30 minutes, and it set aside appointment slots at each site that can only be used for urgent care—and these are consistently 90% filled.

Gaining access to CMS data showed it made more sense for nurses to contact patients on day 3 of chemotherapy instead of day 2, which is when steroids were still having an effect. “We’re working on a high-risk identification protocol with increasing clinic visits and increasing proactive nursing phone calls,” Wilfong said.

The practice is also using data to optimize its antiemetics protocol for chemotherapy-induced vomiting, which calls on pharmacists to adjust the antiemetics based on the risk of each regimen. In a pilot of this effort, only 2 of 300 patients had a nausea grade of 5 or more on the Likert scale of 1-10.

The OCM process revealed a need for major improvements. “It surprised me, when we looked at our baseline numbers, how poorly our practice was doing at end-of-life (EOL) care,” Wilfong said. The doctors thought they were doing well, but the metrics showed something else: a need for better conversations, fewer hospitalizations near the end of life, and less chemotherapy.

It was a big challenge. “It’s hard to tell a physician who’s been practicing for 20 years that they [are failing] at end-of-life care,” Wilfong said. “They don’t like to hear it, and everybody kind of knows it. All the nurses talk about it behind their backs.”

Identifying those physicians who excel at EOL conversations and arranging for them to mentor those who need improvement is hard, and a work in progress. “I would love to tell you we have this fixed, but we don’t. But we are improving.”

Texas Oncology is using a program, My Choices My Wishes, to help physicians talk to patients, and building a culture of shared decision making. It’s producing results: a high percentage of those who go through the program complete an advanced directive. Among patients with an advanced directive, 76% die in hospice, with a median length of stay of 21 days compared with those who went through the program who did not do advanced directives, a group in which 61% died in hospice and had an average length of stay of 12.5 days.

The compensation changes under OCM took Texas Oncology from 83% compliance with clinical pathways to 90%, a level that Wilfong says “is a perfect number to me.”

When physicians want to treat patients off a pathway, their decision requires medical review, a step that dramatically controls drug spending. “What I tell doctors all the time is that if you’re doing something, you should be able to stand up in a group of your peers and at least half of them agree with you. And if you can’t convince at least [half of] the people that what you’re doing is right, then you probably shouldn’t be doing it in the first place,” he said.

OCM raises the specter of taking on the taboo topic in oncology care. “Drug margins are a revenue source for oncology practices, and we’ve got to start talking about this if we’re ever going to be able to get a handle on our drug spend,” Wilfong said.

When practices have some patients in FFS and others in value-based reimbursement programs, it gets complicated administratively but ethically, too. “This is something...we have to get a handle on in oncology practices in order to be able to succeed in value-based programs in the future,” he said.

1. Oncology Care Model Fact Sheet. CMS website. cms.gov/Newsroom/MediaReleaseDatabase/Fact-sheets/2016-Fact-sheets-items/2016-06-29.html. Published June 29, 2016. Accessed January 7, 2018.

Articles

Evidence-Based Oncology

Coverage of updates in value from Patient-Centered Oncology Care, November 16-17, 2017. 

What Implementing the OCM Looks Like in a Large Practice

In both clinical impact and cost, novel therapies play a significant role for all oncology stakeholders, and experts discussed that impact during Patient-Centered Oncology Care

 in Philadelphia, Pennsylvania. The meeting was presented November 16-17, 2017, by 

The panel featured Bruce Feinberg, DO, chief medical officer at Cardinal Health; Thomas Graf, MD, vice president and chief medical officer at Horizon Blue Cross Blue Shield of New Jersey; Kashyap Patel, MD, a medical oncologist and CEO of Carolina Blood and Cancer Center in Rock Hill, South Carolina; and Kavita Patel, MD, MA, a nonresident senior fellow with The Brookings Institute and co-founder of Tuple Health in Washington, DC.

Dr Kavita Patel explained Medicare’s Novel Therapy Adjustment Factor, which is used to avoid penalizing doctors who are being reimbursed through alternative payment models (APMs) if they prescribe a novel therapy. She then explained the 3 issues involved with Medicare’s Oncology Care Model (OCM):

How do we really know what to make of a utilization rate when we don’t really have comparison groups?

How do the novel therapies apply to the APMs?

Dr Kashyap Patel said that for him, novel therapies are promising, but they come with many complications. He attributed this to the higher cost of care, not only for the cost of therapy itself but also due to the additional time patients must spend with a caregiver. “So, along with trying to reduce hospitalization, plugging numbers into the OCM portal, [and] reporting quality parameters, it’s making things a little harder right now, and we’ll see how it goes over the next 2 to 3 years. But definitely, the novel therapies [have] promise but also [have] complicated the implementation of value- based care in real day-to-day life,” Dr Kashyap Patel said.

Feinberg discussed the Center for Medicare and Medicaid Innovation’s (CMMI) role in novel therapies. He questioned whether CMMI will help align the different stakeholders’ opinions of novel therapies in order to lead to further advancements.

Graf noted the current significance of the term “value” in healthcare and the fact that the meaning differs for every stakeholder. He offered the “episodes approach” for managing costs and quality in oncology. In this approach, multiple bundles would be available for patients and allow them to be stratified so the cost can be assessed.

“But it’s really about the rest of the care that’s critically important and it’s going to determine the outcome for the patients,” Graf explained. “If we can ideally match the patient and their medical needs with the level of resource we deploy, we’re going to minimize the huge increase in costs.”

Furthermore, Feinberg emphasized the need for transparency among stakeholders in order to manage costs and to better understand why other stakeholders make certain care choices. Graf agreed with this concept, while highlighting that trust is required among organizations and patients to produce a successful model of care. Dr Kavita Patel also noted the need for transparency, but called for a “realistic on-ramping for people”—meaning a better understanding of certain healthcare processes among stakeholders.

Said Graf, “If we keep the patients first, and we figure out how to push the money around in the back, we’ll be OK. And so, I think you need a flexible, open, willing payer that’s ready to experiment, that’s willing to understand.” With that, “It’s a great model.”

Dr Kashyap Patel demonstrated the need for transparency by discussing the use of chimeric antigen receptor (CAR) T-cell therapy. He explained that major stakeholders have no way of being prepared for 2-sided risk in the current OCM, especially without any level of transparency. Therefore, 1-sided risk is more comfortable to consider with CAR T-cell therapy because the outcome of the patient would not be compromised.

“So, it’s about really an exploration, and we’re sort of thinking about it the same way we think about transplant, which is, look, we’re going to go in, we know it’s going to be expensive, [but] we’re going to figure it out together,” Graf said in response. “And you need to make a rational margin, we need to pay a reasonable amount, and, if we all agree to that, we can be okay. But, yeah, taking risk when there’s that many unknowns would be crazy.”

The panelists agreed that these types of conversations are important and need to continue around novel therapies in order to produce improvement and changes in healthcare.

Coverage of value updates from Patient-Centered Oncology Care, November 16-17, 2018.

Panel Explores Impact of Novel Therapies on Oncology Stakeholders

Does real-world evidence have a place in increasing the value of care? A panel took on that question during a discussion on the second day of Patient-Centered Oncology Care

The panel included Lalan Wilfong, MD, oncologist at Texas Oncology; Ian Manners, MBA, founder and CEO of Vivor; and Jason Harris, manager of public policy at the National Health Council (NHC), who discussed the role of real-world evidence in the healthcare field.

Harris said the NHC pursues patient engagement both directly and indirectly. The council speaks with patient groups to learn their priorities, but it also has the capacity to draw data from patient registries to learn what learn what patients value. This data can then be used by payers or the FDA to improve overall value for patients.

Wilfong noted that his team is focusing on improving the patient experience, specifically when calling a doctor’s office, through the information gathered from patient satisfaction surveys. He also said that his organization is working to hold other organizations and individual sites within Texas Oncology, which is a multisite practice, accountable for change management and increased engagement at a local level.

Additionally, Wilfong described the challenges involving patient decisions that call for balancing financial considerations without limiting what is needed for good care. “They don’t want to fully engage us in those conversations because they’re afraid that we will limit their care if we realize how financially struggling it is for them,” Wilfong said. “Many times, we don’t realize that, as providers, until it’s too late, until you see not just the patient but the family struggling to make ends meet. And that’s a problem. No matter how you have those conversations, there’s still a disconnect between us and the patient about those needs.”

Manners’ software company Vivor acts as a financial platform that helps providers find resources that are underused for patients, like assistance from pharmaceutical companies or nonprofit foundations. The company finds solutions for providers to help their patients afford treatments, Manners explained. He emphasized that it is part of a provider’s responsibility to consider the patient’s financial experience.

Manners also discussed a trial that Vivor is conducting to see if providing patients with financial resource information at the point-of-care would reduce out-of-pocket (OOP) costs. “[At] least they can start there, explain what the patient’s out-of-pocket costs are going to be as they go through a treatment plan, and then bring up the other issues, [such as, ‘How is this going to affect your work? Is it going to change the insurance that you can afford?’” Manners noted.

The panelists agreed that when it comes to cancer treatment, most individuals are willing to spend whatever it takes for the best treatment and doctors, but according to Wilfong, their ability to make a choice is eliminated. Furthermore, Harris emphasized the importance of accountability when considering the limited choices for cancer treatment, especially for those that are uninsured and underinsured.

“When it comes to looking for positive outcomes, health outcomes [come] first, of course, but when we say this pathway, this treatment plan, was the best one based on real-world evidence, we need to account for the patient’s financial experience as well,” explained Manners. “And we need to start doing that by first

In reference to the Oncology Care Model (OCM), Manners considers it to be vague and open to interpretation. He called for a more specific financial plan through the OCM that would help patients understand insurance benefits and OOP costs.

Overall, the panelists emphasized the importance of providing patients with the necessary resources and to work on minimizing the disconnect between physicians and patients, especially when it involves treatment costs.

“So, where we’re at right now is sometimes there’s a big disconnect between the different stakeholders and what that value means to that patient sitting in front of you,” Wilfong said. “Many payers are having their own pathways that may be discordant from our pathways for various reasons. And so, we’re having those discussions more.”

Coverage of value updates from Patient-Centered Oncology Care, November 16-17, 2018. 

Adopting Real-World Evidence and Value Into a Payment Model

Science is yielding more options than ever to treat cancer. That’s the good news.

The bad news is that as cancer care becomes more complex, so do choices for patients and their families. How much does a wonder drug help patients if they can’t afford it or if the adverse effects (AEs) make quality of life poor or if it forces them to spend down retirement savings or puts the family home in foreclosure?

These are the real challenges facing patients that are now being studied and measured by the Cancer Support Community, according to Joanne Buzaglo, PhD, senior vice president of the group’s Research and Training Institute.

Buzaglo noted that the idea of “value” in cancer care has gained attention in recent years. The American Society of Clinical Oncology, the National Comprehensive Cancer Network, and Peter Bach, MD, MAPP, of Memorial Sloan Kettering Cancer Center have all developed value frameworks.

But simply looking at a drug’s cost compared with how much it extends a person’s life misses the mark if the patient’s perspective is left out.

The Research and Training Institute works to collect that patient perspective through its Cancer Experience Registry that develops data that can lead to system change. Buzaglo described how the registry collects information on patient demographics, cancer-related distress, symptom management, AEs, and the clinical trial experience and how cancer affects a person’s ability to work. Of great importance, it collects information on financial toxicity and shared decision making and planning.

She shared data on the top 10 concerns among cancer patients in the registry, which included eating and nutrition (62%), remaining physically active (55%), worrying about the future (53%), feeling too tired (50%), and having sleep problems (46%). Nearly half of cancer survivors (47%) are at risk for clinically significant levels of depression.

What causes this? Communication gaps with the healthcare team don’t help, Buzaglo said:

1 in 3 patients said their healthcare team did not explain short-term AEs

52% received guidance on long-term side effects of treatment, which means nearly half did not

 The term is in the eye of the stakeholder, Buzaglo said, and the patient’s view is often missing. “By understanding how patients define value, we can identify strategies to better engage the patient in value-based cancer care,” she said.

The institute asked patients, “When considering your cancer experience, how would you define value?” and the results led to 2 reports: findings on 769 patients with metastatic breast cancer (MBC) and comparative findings in MBC and breast cancer.

It turns out that value not only means different things to differ- ent people, but sometimes that meaning has nothing to do with a patient’s health: When making treatment decisions, 93% of patients consider quality of life, 79% consider length of life, and 74% consider the impact a decision has on their family. And although patients are increasingly involved in decisions (66% said they were very involved), less than half (46%) felt they had sufficient knowledge to make good choices and only 38% felt fully prepared.

 Buzaglo and her team’s research also showed that while three-quarters of patients said they knew what clinical trials are, 80% said they were uncomfortable being randomly assigned to a treatment andnd 77% say their insurance would not cover the cost of treatment.

The financial burden of cancer care weighs on patients: 58% of registry patients said it had significantly affected them and 37% were experiencing anxiety. Yet the topic of financial burdens presents the greatest communication gap between the care team and patients: 73% said they had not discussed their financial concerns with their healthcare providers, but they were making significant trade-offs or cutting corners on care:

13% had applied for or used public assistance

17% put off a complementary appointment, such as one to a physical therapist

5% had taken an extra job while undergoing cancer treatment to pay medical costs

Buzaglo said this Web-based program was created by the Cancer Support Community, incorporating its research findings into a screening tool to evaluate distress and make referrals to improve quality of life and health outcomes and help patients deal with the cost of care.

A new tool called VOICE (Value Outcomes in the Cancer Experience) is being tested with patients identified in the cancer registry. VOICE would be focused on the future and what patients want to achieve with their treatment.

Buzaglo posed these questions: What if patients were screened for the outcomes that were important to them? What if healthcare professionals were paid for properly informing patients?

Finally, she asked, what if reimbursement was tied not to giving patients drugs, but to giving them the outcomes they sought?Reference 

1. Peer Exchange: 2016 Oncology Stakeholders Summit. 

 2016. ajmc.com/ journals/evidence-based-oncology/2016/peer-exchange-spring-2016-oncology-stakeholders-summit. Published May 10, 2016. Accessed January 9, 2018.

Coverage of patient-centered care updates from Patient-Centered Oncology Care, November 16-17, 2017. 

Using Data on Patients' Cancer Experiences to Change Payment Paradigms

For all of the innovations in cancer therapy and diagnostic tools, one of the most profound advances in care may be the helping hand. The role of the nurse navigator continues to evolve, and evidence shows giving patients this source of support works.

Different aspects of the navigator and support roles were the topic of a panel on the second day of Patient-Centered Oncology Care

, the multistakeholder gathering in cancer care held in Philadelphia, Pennsylvania.

The insurer, Aetna, is incorporating nurse navigators throughout the cancer care continuum due to the “vital impact” these professionals have on patients, said Roger Brito, DO, senior medical director of Oncology Solutions at the payer. Navigators wear multiple hats and reach out immediately after a biopsy—they don’t wait for the results to come back, he said. The biopsy itself is “an anxiety provoking procedure,” Brito said.

“They follow our members through their cancer journey by getting them into the right treatment plan” he said. “Our philosophy is, ‘You don’t join us, we join you.’”

Bo Gamble, director of strategic practice initiatives for the Community Oncology Alliance, has seen the evolution of the navigator’s role, especially over the last 5 years. “I’ve really seen it take hold,” Gamble said, adding that the presence of navigators affects not only the quality of care for patients but also affects physician recruitment.

Navigators have communication skills, particularly as mediators between providers and a patient’s employer, that physicians lack. “They have a magic touch,” Gamble said, and are so needed that the challenge now is freeing up time for professional development.

As an oncology financial navigator for St. Agnes Cancer Institute in Ellicott City, Maryland, Nicole Taglione connects with nurse navigators at the beginning of a patient’s treatment to figure out what insurance will pay and whether there is need for assistance from private foundations or drug manufacturers. “We help the patient to understand what to expect before they get the bills,” she said.

Marie Kelly Pressler, RN, MSN, OCN, and Karon Martyn, MSN, ANP-BC, AOCNP, both of Abington Hospital-Jefferson Health, a hospital north of Philadelphia, discussed the sequence of their roles in a patient’s care. Pressler serves at the initial point of contact and empha- sized how important that is for setting the tone for the rest of the patient’s experience.

“That first touchpoint is so very important for the patient,” Pressler said. It’s essential to communicate a sense to the patient that “I’m safe. I’m comfortable with the decision I make after this phone call.”

When Pressler transitions a patient to Martyn’s care, the process is very careful and deliberate. Martyn knows the patient is headed her way and assists in a shared decision-making process that she says is a far cry from the 1950s model of care where physicians did little to inquire what patients wanted.

“We make sure that treatment decisions are best for them,” Martyn said, and she makes sure patients understand that things can change.

Moderator Margaret O’Grady RN, MSN, OCN, FAAMA, the administrative director for Abington Hospital-Jefferson Health Oncology Service Line, asked each member of the panel to discuss how they address acute episodes as well as the topic of palliative care—a challenging but necessary step.

Brito said the more that Aetna does to increase the “stickiness” of the relationship between the navigator and the patient, the more likely they are to prevent issues of toxicity of medication from turning into a trip to the emergency department. The growing emphasis on survivorship care, and the rising number of survivors, extends the length of these relationships.

Pressler was more direct. “The first thing is, you have to be nosy.” Nurses in the infusion room, for example, must be trained to ask the patients about adverse effects. When patients say nothing is wrong, nurses must be willing to dig a little deeper. Developing that rapport will allow patients to open up and share things before they turn into big problems, as well as make the appointment with the physician more efficient.

Gamble agreed. His organization has done extensive surveys with patients about their experiences and learned, for example, that sometimes the stress level within families is so high that a patient’s workplace is the only refuge. Again, he said, the navigators seem better equipped at finding out these things. “They’ve become the heroes in this generation.”

Coverage of patient-centered care from Patient-Centered Oncology Care, November 16-17, 2017. 

Navigators Offer Touch Points on Patients' Cancer Journey

Patient care is a team effort, and this is truly evident in oncology care. A panel on the importance of clinical and non-clinical stakeholders in a patient’s care trajectory brought together a diverse group convened by 

, held November 16-17, 2017, in Philadelphia, Pennsylvania.

Panelists included Rose Gerber, director of patient advocacy and education, Community Oncology Alliance; Stacey McCullough, PharmD, senior vice president for pharmacy, Tennessee Oncology; Michael Diaz, MD, director of patient advocacy, Florida Cancer Specialists & Research Institute; and Rebekah Gilbert, RN, BSN, OCN, nurse practitioner, Hematology Oncology Associates of Central New York.

Gerber, a breast cancer survivor, narrated her personal experience as a patient and a survivor, which serve as her inspiration for her current role as a patient advocate and educator. Having received her diagnosis while she was raising a young family, Gerber said that she and her husband were devastated. Overwhelmed by the news, and vulnerable, Gerber and her husband were ready for her to participate in any clinical trial that was offered. “However, when I looked at the pages and pages of consent forms and dis- closure forms, I was scared,” she said, adding that she values the importance of clinical trials as an option for patients and firmly believes that patients should be engaged to participate in trials.

Gerber also noted that care transitions are extremely important for patients. “I never thought, 14 years later, that I’d still be actively seeing my oncologist, as I continue dealing with some of my health issues,” she said, explaining that her chemotherapy (trastuzumab) and other cancer treatments increased her susceptibility to secondary health issues. So, in addition to an oncologist, a radiologist, and a surgeon, Gerber’s care plan includes a cardiologist because of heart conditions developed as an adverse effect of the trastuzumab, an endocrinologist, and a neurologist. Additionally, she struggles with weight issues, which could be related to some of the treatments that were administered. “But at the center of it all has been my oncologist,” Gerber said.

Gilbert said that each care team in her practice includes a patient navigator, a nurse, an advanced practitioner, and a doctor. “We huddle each morning to discuss our patients who will be visiting our clinic that day, as well as the patients who have been identified as being high risk.” This keeps everyone on the team abreast of what’s going on with their patients. Additionally, the clinic has a telephone triage system, with 3 dedicated nurses on call all day, who either bring in a patient or give them home-care instructions over the phone. “Very rarely do we send a patient to the ER [emergency room],” Gilbert said.

McCullough explained that, following the advent of oral oncolytics, Tennessee Oncology established an in-house retail system that operates as a specialty pharmacy. “Our healthcare system has, so far, underutilized pharmacists, but a pharmacist can definitely step in and be a part or a better partner along with nurses and physicians,” she said. Pharmacists have access to the electronic health record as well as the doctor’s notes. Also, when they access the system to refill prescriptions, the pharmacist can keep track of when a patient was due for a clinic visit but may have missed the appointment and can be the point of contact for the patient. “So, a pharmacist can play a more proactive role in patient care,” McCullough said.

Diaz noted a very specific challenge at Florida Cancer Specialists: Consolidation has increased its size to over 100 sites across the state, with more than 200 oncologists on the team in addition to advanced practitioners. “Each site had their own model on how they operated, and we needed a process so everyone would work in a similar fashion to be able to provide all the advanced care that patients need,” Diaz explained. Their 40 care coordinators have been divided into 3 teams:

Care coordination for patients on active treatment

The team that works with active patients has protocols in place so it knows how often they need to contact the patients. They also have specific questions when they reach out to these patients, in addition to routine clinic visits.

The team dedicated to survivorship coordinates care with the doctors and calls the patients to ensure they are aware of upcoming appointments.

The team that manages transitional care coordination has access to software that allows a coordinator to follow all of the information and ensure patients are taken care of during their transition out of a hospital, in case they need extra care post discharge, because they may or may not always know whether they need to get in touch with a doctor with regard to a specific concern. “So, the bulk of the care we are trying to provide to our patients is handled by our care coordinators,” he said.

As a nurse navigator in her practice, Gilbert noted that she works closely with the pharmacist on patient education, especially with oral oncolytics, which is at the top of their plan. A nurse also makes follow-up phone calls with the patient at pre-determined intervals, and if there are any issues, the nurse will let the team know to follow up with the patient.

“While the patient should be at the center, I think the oncologist, the nurse, the pharmacist, and even family caregivers should be part of the patient’s care team,” Gerber said, aptly summarizing the discussion.

The Importance of Teamwork in Oncology Care Transitions

The headlines of the 21st century Cures Act said it provided $6.4 billion for scientific research for cancer, Alzheimer disease, and opioid addiction, as well as steps to speed drug development, including new approval pathways for certain devices and biologics.

But beyond that, said the FDA’s Frank Weichold, MD, PhD, of the Office of the Commissioner and the Office of the Chief Scientist, the Cures Act brings a new way of thinking about the agency’s role: For decades, most regulation was a reaction to some tragedy or weakness in existing rules. Cures ushers in an era of the FDA as a partner in drug development, Weichold said, as he kicked off Patient-Centered Oncology Care®, held November 16 and 17, 2017, in Philadelphia, Pennsylvania.

, Weichold explained how the FDA has created a culture of regulatory science and that the Cures Act brings the next step. Regulatory science is not “an attempt to regulate science,” he said, but an effort to guarantee that rulemaking for drugs, foods, and consumer products under the agency’s purview follows a process that ensures safety and quality under standards that are consistent and not based on unexpected whims. The less evidence there is, the more individual judgment creeps into the process, Weichold explained.

With the Cures Act, drug development will be simultaneously more evidence driven and patient centered. “It makes the FDA an active participant in drug development, and it requires us to focus more on patient-focused drug development using novel innovative trial designs, applying real-world evidence, and create drug development tools to speed this up,” Weichold said. Clinical trials as we know them are “essentially disconnected from the real word,” he noted, and that sometimes results in real-world outcomes that don’t match what happened in studies—to say nothing of the high research costs.

FDA Commissioner Scott Gottlieb, MD, supports the use of computer modeling in the drug development process, Weichold said, as well as adaptive and novel trial designs that are already in use. But this requires good data—and Weichold said there’s much to be done to get data out of silos and to encourage sharing to create algorithms that can speed medical decisions, with fewer errors.

“We are on an inflection point when it comes to the reductionist position and thinking in a holistic world, and what’s between is a lot of data,” he said.

Knowledge and data sharing isn’t where it should be, Weichold said, and “we need better communication, and we need better transparency opportunities” that are not a burden for investigators. Yet he said every person in the room could likely think of an example of a hospital administrator or rule maker who prevented data sharing.

The 21st Century Cures Act calls on regulators to “get engaged,” Weichold said, and facilitate sharing by creating frameworks to make it happen. Because when it happens, patients will benefit, he said.

“Essentially, to enter the right data once and use it many times is something that we have to accomplish,” Weichold said. Regulators can help health systems address privacy and safety concerns, but “we have to change the culture,” he added.

He pointed out examples from the FDA’s Oncology Center of Excellence, which collaborated with the Harvard Business School to develop master trial protocols; he also discussed the I-SPY 2 breast cancer trial, which follows an adaptive design that takes the response times from patients as they move through the trial to make treatment decisions for future patients.

Data sharing is key in developing better biomarkers, Weichold said, which the I-SPY trials and other analyses have shown can be used as surrogate end points, to help bring drugs to market fast and identify positive responders. He cited work by Laura Esserman, MD, MBA, at the University of California at San Francisco in the I-SPY trials, which has shown how trials can work with real-time feedback.

Data and technology alone are not enough, however. “We have most of the things that it takes to have a continuous collab- orative and transparent system, including data exchange, data liberation, [and] data markets, if you want to call it that way,” he said. “But what we need is to build trust and build relationships, and as such, it all hinges on the people—people who are also depending on the FDA.”

Who are the final collaborators? Patients. “We need to include patients in a different way—make them co-researchers, codevelopers&mdash;and leverage the tools that we have in our advanced age of technology.”

“And with that,” he concluded, “we will have better outcomes, better performance, and, perhaps, happier people.”References:

1. 21st Century Cures Act. FDA website. fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendmentstotheFDCAct/21stCenturyCuresAct/default.htm. Updated September 14, 2017.

2. DeBonis M. Congress passes 21st Century Cures Act, boosting research and easing drug approvals. Washington Post. December 7, 2016. washingtonpost.com/news/powerpost/ wp/2016/12/07/congress-passes-21st-century-cures-act-boosting-research-and-easing- drug-approvals/?utm_term=.aade676a9585. Accessed January 4, 2018.

3. The I-SPY trials. ispytrials.org/home. Accessed January 4, 2018.

Coverage of regulatory changes from Patient-Centered Oncology Care, November 16-17, 2017. 

FDA Moves to Era of Active Participant in Drug Development

The FDA's role in establishing the standards and regulations for public health would benefit from increased transparency, according to stakeholders who included a representative from the agency. The group was part of a panel that appeared on the first day of Patient-Centered Oncology Care®, presented November 16 and 17, 2017, in Philadelphia, Pennsylvania, by 

Panelists included Roger Brito, DO, senior medical director of Oncology Solutions at Aetna; Mark Fleury, PhD, principal of policy development at the American Cancer Society Cancer Action Network; and Frank F. Weichold, MD, PhD, director for Critical Path and Regulatory Science, Office of Regulatory Science and Innovation, Office of Chief Scientist, in the Office of Commissioner of FDA.

Brito discussed expanding the oncology footprint at Aetna through potentially collaborating with the FDA to increase data-sharing transparency; this would benefit clinical trials and pilot studies. Brito explained that such transparency could be achieved through top-down engagement not only with providers but also with Aetna members. “I think it’s an exciting time to be in healthcare. I think we need to collaborate, include the patients, include pharmaceutical industries, [the] FDA, [the] healthcare company,” he said.

Referring to the cost of care, Brito emphasized that transparency will allow all stakeholders to know about different therapies and treatments earlier in the pipeline, which will improve the cost-containing measures among therapeutics.

Weichold also explained that transparency is necessary to identify areas of improvement between the drug regulators and the patients. He expressed the need for building a new culture in which all stakeholders are contributing resources. “We need to be able to engage the stakeholders and include experts, not just opinion leaders but actual experts that can, through scientific means, define opportunities for solutions,” he said.

Weichold noted that increased transparency could be achieved through embracing the opportunities that technology provides. For example, he mentioned the app HUGO, which gives physicians easy access to patient records. Weichold also noted the signifi- cance for high-quality data and access to such data—a component that the United States could adopt from European models.

Fleury discussed the role of advocacy organizations in working to bring people together and sparking policy conversations among groups of stakeholders. He offered a recent example of a pediatric cancer drug that had biological, regulatory, and financial factors that were different from those of a drug developed for adults; stakeholders came together to reach a common understanding of the problem.

The FDA faces an ongoing challenge of deciding whether the benefits outweigh the risks for the average patient for a condition—although all patients are different, Fleury said. Therefore, he explained, the system is moving toward gathering the patient’s experience and perspective so the FDA does not have to make decisions based on the “average” patient.

Additionally, Fleury noted the importance of providing education to individual patients while ensuring that they trust their physician, as part of building the new culture. “I do think there’s an important role to make sure that we inform the system and create a system that makes sure that they are getting the best possible care and not rely on that patient on day 1 knowing everything that they should ask and everything that they could do,” Fleury said.

Although many changes to the care system are needed, the experts all agreed on the progress that has been made, and they believe progress will continue to be made by the FDA in 2018.

“The agency, I can tell you, from its leadership but also in particular from its staff, [is] very much interested and feels a high level of responsibility and also a sense of public service to make significant contributions to that,” Weichold said.

Building a Culture of Transparency Is Key to FDA in 2018, Experts Say 

Just a month before attendees gathered in Philadelphia, Pennsylvania, for the annual meeting of Patient-Centered Oncology Care

, legislators in the state’s capital of Harrisburg made it the 38th to pass a right-to-try law. That was 6 states in 2017 alone.

 Championed by conservative groups like the Goldwater Institute as well as Vice President Mike Pence,

 the laws sound good on their face: they can connect terminally ill patients with experimental treatments; even opponents say the idea of giving dying patients “one last chance” is hard to oppose.

But as panelists discussed during the session presented by 

, these state laws are not necessarily good for patients or long-term drug development, and there is a better alternative: the FDA’s newly streamlined Expanded Access program, which allows patients to gain access to investigational therapies in a more regulated way, with greater accountability. Thus, there is great concern about a proposed federal right-to-try legislation, including a version that has passed the United States Senate.

The panel featured W. Kevin Kelly, DO, director of the Division of Solid Tumor Oncology at the Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia; Marjorie A. Speers, PhD, executive director for the WCG Foundation; and Diana Zuckerman, PhD, president of the National Center for Health Research, board member of the Reagan-Udall Foundation.

As Speers explained, the Expanded Access program began decades ago during the AIDS crisis, when patients demanded protocols be established for those not enrolled in a clinical trial who wanted to try a potentially life-saving medication when nothing else was available. Once FDA approves a request, the patient’s doctor supervises administration of the drug as an extension of an ongoing clinical trial, with significant reporting requirements. The modern proliferation of right-to-try laws has occurred even though FDA has streamlined its process and increased its approval of individual Expanded Access applications, from 1200 to 1500 in recent years, she said. “There has been a lot of attention and pressure on the FDA to make more of these drugs available,” Speers said. “And much of that has been through social media.”

Zuckerman said that so far, state-level right-to-try laws have not had a significant effect, but the proposed version of a federal law could do harm by letting patients obtain drugs independent of the FDA. The Senate version was amended so that drug makers are not required to sell investigational drugs to patients outside of clinical trials, but if they do, they must report adverse events to the FDA.

 Typically, patients receiving investigational drugs while they are going through FDA approval can only be charged for the drug’s manufacturing cost, and patients usually receive them for free. Zimmerman said an early version of the bill did not cap sale prices, but the bill that passed the Senate only allows pharmaceutical companies to charge manufacturing costs.

Still, if patients obtain drugs this way, there are far fewer requirements than there are currently under the FDA’s Expanded Access program.

And this appears to be central to the appeal: Right-to-try laws purport to cut the FDA out of the picture, connecting patients directly with pharmaceutical companies to reduce burdens on patients and physicians, if drugs have cleared phase 1 safety hurdles. But some see this as a threat to the drug development process; FDA’s Expanded Access requires at least some evidence of efficacy in addition to phase 1 results.

Speers discussed the 3 categories of use under the FDA’s Expanded Access program. Applications can be submitted for (1) individual patients, (2) intermediate-size patient populations, (3) wider treatment use ahead of distribution after approval.

Because this process includes patients with life-threatening diseases, Speers noted that the FDA has ensured transparency in the process of

accessing the necessary experimental drugs. Therefore, a patient must complete an application, and a specific judgment about the individual patient is made by the FDA. The agency also can also review the requests with a clinical trial’s Institutional Review Board.

Speers also discussed the accessibility of experimental medications for patients through the Expanded Access program. Some of the issues raised through the expansion of right-to-try laws concern the low number of patients who take part in clinical trials—it’s only about 10%.

“There are things that we can do to move trials along more quickly, and it’s across a whole range of things: recruitment, data, and design, and what’s required as a standard to make the decision. That’s I think where we want our emphasis to be, and for this to be a smaller program but remove those barriers that can be removed from it,” she said.

Kelly, a clinician, emphasized the importance of safety when evaluating patients for investigational drugs, considering that many clinicians would not have experience in administering these drugs.

“Can we get more expansion protocols to expand to more patients, [and] decrease the eligibility criteria, so we can treat these patients on a study [by] physicians who actually know how to use the drugs?” Kelly asked.

Still, he has seen both sides of the issue. “I spent several years at the FDA on the advisory committee, so I know the safety issue that they grapple with,” he said. “But being on the front line, it’s a lot different. I have patients coming to me to all the time asking me about investigational drugs. Some are appropriate, some are not ... And it’s a lot of education that the patients actually need to understand what is their disease, what are the treatments, and what is a reality.”

The rise of checkpoint inhibitors offers a great example, he said, in that there are significant toxicities involved—if a physician has never seen them he or she might not know how to handle them. To expand access and knowledge among clinicians, collaborative databases should be developed so data can be shared among a large range of individuals.

Zuckerman also noted the importance of physicians and patients report- ing the serious adverse events from individual patient uses of investigational drugs. In the Expanded Access program, physicians administer the drugs on behalf of the FDA and there are significant reporting requirements, which demands accountability, according to Zuckerman.

“I think we really should focus on education for physicians who want to use an investigational drug through Expanded Access,” she said.

Despite the risks and challenges involved in administering these inves- tigational drugs, Zuckerman said there are ways to streamline the application and monitoring process, so that safety can still be the highest priority while making it easier for patients and physicians to participate.

Each panelist expressed the issues and potentially negative effects of the federal right-to-try laws. Each concluded by offering an ideal system that would address the different concerns. Zuckerman proposed a simpler system that makes it easier for patients, Speers proposed limiting the use of expanded access until after a drug has been approved by the FDA but before it is on the market, and Kelly proposed a patient-centric system that provides patients with a direct source for information.References:

1. Pennsylvania becomes the 38th state to pass right to try. Goldwater Institute website. goldwaterinstitute.org/article/pennsylvania-becomes-38th-state-to-pass-right-to-try/. Published October 13, 2017. Accessed January 16, 2018.

2. Karlin-Smith S, Kim SM. Senate approves ‘right-to-try’ drug bill. Politico. politico.com/story/2017/08/03/senate-right-to-try-drug-bill-241293. Published August 3, 2017. Accessed January 16, 2018.

3. FDA Expanded Access (Compassionate Use). FDA website. fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm. Updated January 4, 2018. Accessed January 16, 2018.

Weighing the Merits of Right-to-Try Laws and FDA's Expanded Access Program

Clinical care pathways have come a long way, according to Kathleen Lokay, the president and CEO of Via Oncology, who discussed the real-world impact of digital decision-support solutions at Patient-Centered Oncology Care

 in Philadelphia, Pennsylvania. The meeting was held November 16- 17, 2017, presented by 

Lokay emphasized the importance of content in treatment pathways. She explained that treatment pathways typically start out small, with tracks for only a few diseases, and then evolve to incorporate different phases of care, like symptom management, workup, or surveillance.

At Via Oncology, Lokay said the evolving pathway model builds accountability among physicians by forcing them to consider the patient perspective in pathway development. This is accomplished through a committee system, with 36 panels that meet throughout the year to adapt and update pathways.

The 3 pathway determinants Lokay discussed in her presentation include:

 If one treatment represents the clear choice, it becomes the pathway.

 If efficacy between different treatments is comparable, the pathway calls for treatments with less toxicity to improve quality of life and reduce hospitalization and emergency department visits.

 Only if efficacy and toxicity are comparable will the pathway choose the lower cost treatment for the payer.

Lokay also discussed the need for multiple options that accommodate different patient preferences in the pathway. “Even though we haven’t changed the construct about how we develop the pathways, we’ve had to get smarter about giving more information if [patients] need it and creating situations where maybe the pathway even actually has a recommendation for a financial toxicity situation that would take you away from the single best and take you to something that has sort of that economic trade-off for the patient,” Lokay stated.

Despite the efforts and evolution of the pathway, real-world evidence has not yet been incorporated—but it could be added at a later point. “I always have trouble saying that phrase, ‘real- world evidence.’ And partially that’s because what we really need to be able to do is make sure that that real-world evidence has the same kind of voracity that the published trial data do,” Lokay explained. “But I think, until it rises to that level to actually make pathway decisions on treatment recommendations based on that, we’re just not there yet.”

She noted that pathways need to have a way of remaining up-to-date. She mentioned iKnowMed, which customizes and regularly updates the pathway so it can be easily shared. The output can then be used by the physicians to make treatment decisions.

The electronic health record (EHR) is also critical to decision support, according to Lokay. Integrating the EHR with the pathway decision-support software, like iKnowMed, would produce a high level of connectivity and common set of content for physicians to use. Additionally, making sure information from the most recent clinical trial results finds its way into pathways is critical, as this ensures the content of the pathway provides value to decisions.

Lokay separated the players involved into 3 categories: precision medicine support companies and big data companies, those that create services for payers, and those that are trying to create their own pathways.

At the conclusion of the presentation, when asked about the provider versus the payer pathway, Kathy discussed the content and software parts of the pathways. She overall predicts that pathways will expand into more areas of oncology and evolve as multidisciplinary pathways.

“So, the content is really not the issue that we find. What we find the rub in is that the physicians will use our pathway and then their team will still have to use the software to submit data for the other pathway...The problem is how to keep up with that because our pathways are changing, their pathways are changing, [and] they’re changing at different points in time,” Lokay said. “It is a real challenge.”

Coverage of digital technology updates at Patient-Centered Oncology Care, November 16-17, 2017. 

Via Oncology's Lokay on Real-World Impact of Digital Decision-Support Solutions

The use of digital data has become a prominent aspect in medicine as technology continues to advance. A panel at the Patient-Centered Oncology Care

meeting held in Philadelphia, Pennsylvania, November 16 and 17, 2017, explored the impact of digital data on different aspects of healthcare. 

The panel featured Torrie K. Fields, MPH, senior program manager for Advanced Illness and Palliative Care at Blue Shield of California; Viraj Narayanan, director of Life Sciences at Cota; and Kathleen Lokay, president and CEO of Via Oncology.

Fields said Blue Shield uses digital data to allow for greater flexibility in treatment pathways for those with serious illnesses. She emphasized the need to ensure that oncologists and patients are communicating and fully understand their treatment options. The 2 things to consider when assessing quality and utilization are understanding variance and considering the patient perspective.

Coverage of digital technology updates from Patient-Centered Oncology Care, November 16-17, 2017. 

Patient Centered Oncology Care 2017