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Innovation and Novel Therapies

Evidence-Based OncologyPatient-Centered Oncology Care 2019
Volume 26
Issue 3

During a discussion at The American Journal of Managed Care®’s Patient- Centered Oncology Care® meeting in Philadelphia, panelists outlined the efficacy of the 2 FDA-approved therapies, Medicare reimbursement for CAR T-cell therapies, and the pace of innovation in healthcare.

The Fast Pace of CAR T-Cell Innovation Caused an Array of Treatment Challenges

Evidence shows that chimeric antigen receptor (CAR) T-cell therapies are eff ective, but their price tags are high, raising concerns about how many patients will receive treatment. During a discussion at The American Journal of Managed Care®’s Patient- Centered Oncology Care® meeting in Philadelphia, panelists outlined the efficacy of the 2 FDA-approved therapies, Medicare reimbursement for CAR T-cell therapies, and the pace of innovation in healthcare.

Tisagenlecleucel (Kymriah) has been used successfully to treat children and young adults, up to age 25, with relapsed or refractory acute lymphoblastic leukemia, explained Shannon L. Maude, MD, PhD, assistant professor of pediatrics in the Division of Oncology at the Children’s Hospital of Philadelphia, and medical director of the Center for Cellular Immunotherapies at the University of Pennsylvania Perelman School of Medicine. In the trials that led to FDA approval, patients treated with tisagenlecleucel had a remission rate of 81% after relapsing more than once after the best standard of care.1 In some of the longer-term data now being seen, patients who went into remission have a relapse-free survival rate of 66%.2

The other FDA-approved therapy, axicabtagene ciloleucel (Yescarta), is indicated in adults with diff use large B-cell lymphoma, which typically aff ects people in their 60s and 70s, said John W. Sweetenham, MD, FRCP, FACP, FASCO, professor of medicine and associate director of clinical aff airs at the Harold C. Simmons Comprehensive Cancer Center at UT Southwestern. For 15 to 20 years, treatment for disease has be relatively the same: chemotherapy is front line followed by a bone marrow transplant. But if those 2 treatments are unsuccessful, the patients had essentially no other options.

Now, there have been extraordinary responses with CAR T-cell therapy, he said. There are patients who, in the past, he would have anticipated have a bad outcome after relapsing, who have now survived more than a year after treatment.

“This treatment is like nothing we’ve ever seen before in terms of its ability to turn very sick people around,” Sweetenham said. However, because these therapies are so diff erent from conventional treatments, there are still plenty of unknowns. For example, there have not been any randomized trials to compare the CAR T-cell therapy treatment with more standard treatments.

As a result of how successful CAR T-cell therapies have been and how unique they are, they cost upwards of $373,000 per treatment3—the good news, said Erika Miller, JD, senior vice president and counsel at CRD Associates, is that patients only need the treatment once. The problem is that regulators and legislators are concerned about safeguarding the Medicare trust fund, and these therapies are a big hit, financially.

There is concern that if Medicare pays the full cost, that it “is sending a signal” to drug makers that the price tag is not a problem, and they might even be able to ask for more for the next treatment. “[Regulators and legislators] are concerned about how many patients are going to get this,” Miller said. “They’re afraid of a tsunami. And then, this is all happening at the same time that everyone in Washington [DC] is talking about the price of drugs.”

She echoed Sweetenham and Maude’s comments that everyone is still waiting to see how eff ective the treatments will be in the long term. In addition, a greater concern is that there are other CAR T-cell therapies in the pipeline, which will only add to the costs.

“Medicare doesn’t change on a dime,” she said. “It takes them a long time to change their policy. They have mechanisms for payment that have been in place for a long time that they are reluctant to change.”

The pace of innovation has been, perhaps, too fast. It has outpaced changes in payment, but also, “in some ways, we’re ahead of the evidence,” Sweetenham said.

“We don’t want to end up in a situation where patients are potentially missing out on effective treatment because it’s taking us too long to get the evidence that we really need,” he added. Putting together clinical trials is complicated and expensive, and researchers need a solid partnership with all the stakeholders in terms of getting needed clinical trials moving, Sweetenham said.

Maude added that when trials are set up, they need to be optimized so researchers can identify which patients will benefit the most from CAR T-cell therapies and, thus, improve the current outcomes. There is additional cost in setting up those types of trials, but they will be more cost effective in the long run, she said.

Moving forward, improving patient access to these treatments is critical, Miller said. The cost of CAR T-cell therapies is so high that academic medical centers are losing out on more than $100,000 for each patient treated.4 As a result, there are some centers that are deciding not to off er CAR T-cell therapy.

Maude and Sweetenham also highlighted the access challenges. Because so few centers offer CAR T-cell therapy, patients often have geographic barriers and must drive long distances in order to get treatment. Patients with commercial insurance tend to have less trouble getting the treatment approved than patients who

receive Medicare.

Although the current administration has been reluctant to pay the full price tag for these therapies, it has shown it is very focused on promoting innovation, Miller said. “There’s recognition that there’s innovation here that can’t be choked off,” she added. The administration is listening, and there has been some progress

with the increased new technology add-on payment,5 but with an election next year, there could be a new administration in the White House with a diff erent perspective.

When asked to peer into the future, Miller predicted there would be a payment model for CAR T-cell or cellular therapies being tested. Next year, there might be a CAR T-cell therapy for multiple myeloma, which has a large patient base, so there will be more pressure on CMS to create a payment model.

Maude and Sweetenham are hoping to see more longer-term follow-up data and better predictions about which patients will benefit the most. Sweetenham is also anticipating that these treatments will move more into the outpatient setting and hopes to see patients getting better access to the treatments.

“Pessimistically, I haven’t seen the field really move that far in 5 years,” Sweetenham said.“


1. Maude SL, Laetsch TW, Buechner J, et al. Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. N Engl J Med. 2018;378(5):439-448. doi:10.1056/NEJMoa1709866.

2. Grupp SA, Maude SL, Rives S, et al. Tisagenlecleucel for the treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic

leukemia: updated analysis of the ELIANA clinical trial. Biol Blood Marrow Transplant. 2019;25(3):S126-S127. doi: doi.org/10.1016/j. bbmt.2018.12.410.

3. Andrews M. Staggering prices slow insurers’ coverage of CAR T cancer therapy. Kaiser Health News. http://www.khn.org/news/staggering-prices-

slow-insurers-coverage-of-car-t-cancer-therapy/. Published July 17, 2018. Accessed January 2, 2020.

4. Manz CR, Porter DL, Bekelman JE, et al. Innovation and access at the mercy of payment policy: the future of chimeric antigen receptor therapies [published online ahead of print November 1, 2019]. J Clin Oncol. doi: 10.1200/JCO.19.01691.

5. Rosenberg J. CMS proposes increased reimbursement for CAR T-cell therapy; price hikes for rural hospitals. The American Journal of Managed Care.® http://www.ajmc.com/newsroom/ms-proposes-increased-reimbursement-for-car-tcell-therapyprice-hikes-for-rural-hospitals. Published April 24, 2019. Accessed

Innovation in Care Also Means a Better Patient Experience

January 2, 2020.When one hears the term innovation in oncology care, the first thought can be keeping up with unprecedented advances in therapy. But changes to the delivery system—the use of navigators, the rise of data-driven quality measurement, the advance of decision-support tools—are equally important in bringing therapies to patients. How do health systems keep up and turn the corner? It’s not easy, according to a panel, Innovation in Oncology Care and Treatment, at Patient-Centered Oncology Care® in Philadelphia, Pennsylvania, held November 8, 2019.

“Innovations are coming at a pace that I’ve never seen before. And I’ve been doing this for 23-plus years,” said event Cochair Joseph Alvarnas, MD, who moderated the panel featuring:


director of Strategic Practice Initiatives for the Community Oncology Alliance;


, vice president, Network Solutions for the Blue Cross Blue Shield Association;


vice president, Innovation and Solution Development, Premier Inc, of Charlotte, North Carolina; and


, chief operating officer at Fox Chase Cancer Center, Philadelphia.

“It’s an exciting time because there’s so much happening so very fast,” Gamble said. The infrastructure of oncology practice—especially reimbursement systems—has not caught up to advances in therapies and technology. On one hand, Gamble said, CMS tells oncologists that the old fee-for-service billing model is “archaic,”

and many doctors agree. But when it comes to finding new ways to measure the value of new technology, agreement has proven elusive, he said.

“We can’t even agree on which is better: overall survival or progression-free survival,” Gamble said.

Hospitals operating on slim margins must fi gure out how to advance the delivery system while staying at the forefront of care, said Bachman. “We want to invest in all these new technologists,” but a new diagnostic tool must be balanced against deferred maintenance and “all the bread-and-butter stuff ,” such as replacing software systems.

“You’re constantly rolling the dice and trying to measure the risk for the organization,” she said. “And very few businesses run on the kinds of margins that we run on. I mean, even a good hospital today, they’re lucky if they’re making 6% to 8% margin. I don’t think any of the companies that we have to interface with would tolerate that.”

Asked for the payer’s perspective, Atkins said, the Blues are focused on improving coordinating of care and improving patient experience. “Everything that we do as a payer is focused on [the] member and that member experience,” she said, and fixing “what we continue to see as a fragmented system is our ultimate calling.”

“I do think that we have made a lot of progress,” Atkins said.

Advances in treatment such as chimeric antigen receptor (CAR) T-cell therapy initially caught payers off guard, but the Blues responded by designating facilities as CAR T-cell specialists. “I felt like the manufacturers had gotten a little bit ahead of us, but we were able to launch our Blue Distinction Centers on January 1,

2019, for CAR T therapy,1 and we envision that as a chassis to build off of for additional therapies,” she said.

Oskouei, whose fi m provides actionable data to improve delivery and pharmacy operations for health systems, said just keeping up with the tidal wave of information is a challenge. “There’s a statistic that there’s new evidence development every 26 seconds,” she said. Converting all that information to “optimize patient care is a big challenge.”

Giving providers good point-of-care clinical decision-support tools lets them off er patients the best outcomes, she said. With all these challenges, Alvarnas asked, “How do you make sure that innovation stays patient centric?” And, how can health systems make the pace of change sustainable from a physician point of view?

Atkins said keeping innovation patient centric starts with asking, “What are your goals in treatment? Do you want to go to your daughter’s wedding? Is this about getting to a graduation?” This shifts the defi nition of patient-reported outcomes to one “that I think we can certainly drive in a meaningful way.” So, Alvarnas asked, what are the barriers to more of this happening now?

“You know, I’m going to be really transparent with you,” Atkins said. Incorporating patient-recorded outcomes is not easy, because the data are less traditional and not as rigorous as registry data. As a payer, “I’ve had to challenge my own thinking on it.” Bachman said listening to the patient is “a huge part” of what happens at Fox Chase—via patient surveys and service gap analyses. Through this process, the staff learned that what matters most to patients is reliability.

“Imagine being in the hospital and having a schedule and actually knowing when something is going to happen to you,” she said. Fox Chase built systems with navigators and better scheduling to make commitments to its patients and deliver on them “and measure our ability to do that.”

Gamble said in other areas, technology makes it possible for consumers to expect greater value for what they pay, but oncology and healthcare generally haven’t followed this approach. Alvarnas pointed out that the system is increasingly unsustainable—more and more responsibility is being pushed onto the physician as

science rapidly evolves. Oskouei said that’s where technology can play a role in clinical decision support—by updating staging criteria, new treatment guidelines, and rapidly changing product indications. “Having that information at the fi ngertips is critical,” she said. But, Alvarnas said, patient education must be part of the equation, too. “At a deep level, you’re talking about investment, when someone is at home using wearables or other technology to monitor them.”

“We need to have specifi c education strategies for all the stakeholders, patient fi rst and foremost, but also for the payers themselves, [and for] the employers are having to pay for the insurance,” Gamble said.” Federal rules can create barriers to innovation that make change unaff ordable.

“It’s going to take a lot of teamwork and bringing up bright minds to say, ‘Lead the charge.’ Let’s not be afraid of it. Let’s tackle it,” he said.

But how? Bachman said information technology investments will be essential. Physicians must be surrounded by teams. Navigators work. Alvarnas said much of this change must be captured in coding, so that payers will reimburse. Atkins highlighted a Blue Cross Blue Shield of Minnesota program that is trying to do this.

And then, Alvarnas asked, how will we know change is working?

“So, we focus on measurement, measurement, measurement measurement, and we are not measured developers, you know, we rely on our colleagues across the industry,” Atkins said, referring to groups such as the National Comprehensive Cancer Network. Transparency is paramount, which includes using data sources

everyone can see. Gamble raised the issue of measuring survival, and there was a discussion of using “workarounds” to get past the limits of claims data.

As the discussion concluded, Lalan Wilfong, MD, of Texas Oncology said, “One of the issues that I struggle with a lot is, just because something’s new doesn’t make it novel or innovative. There’s a lot of things coming out that really don’t move the bar in patient care,” he said. Immunotherapy works well in certain disease types, but makes no diff erence in others.

“As leaders in this space, how do you distinguish between innovation that’s real and impactful for patient care versus something that’s just new?” he asked.

Gamble offered his thoughts. “Insert guidelines for any new pharmaceutical drug, so that you’ve got some sort of requirement says, this is how I’m meeting a universal mission for quality and value,” he said. “Then, I have standard metrics for all innovations. So that we could look at in a way that says yes, this is really improving the life of the patient.”

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