Weighing Drug Costs and Patient Values in Cancer Care

Living longer with cancer is good news. Paying for expensive drugs for a decade or more is not. And value-based models have not caught up to this new reality in oncology, experts said during 4 dynamic panel discussions April 15, 2025, at Le Meridien in Philadelphia, Pennsylvania, where the Institute for Value-Based Medicine gathered in the shadow of America’s original innovator, Benjamin Franklin, atop City Hall.

Selecting CLL Treatment With an Eye Toward Cost
Having more treatment options in chronic lymphocytic leukemia (CLL) doesn’t mean physicians have fewer questions; in fact, they have plenty. In frontline care, is fixed duration or continuous treatment the way to go? What is the best way to sequence therapies to get the most durable responses? How do prognostic markers factor into decision-making?

Emily Tomasulo, DO, FACP | Image credit: Penn Medicine

Moderator Emily Tomasulo, DO, FACP, assistant professor of clinical medicine at Penn Medicine, guided a discussion that featured panelists Elise Chong, MD, assistant professor of medicine, Penn Medicine; Marcus Messmer, MD, assistant professor, Department of Hematology/Oncology, Fox Chase Cancer Center; and Pierluigi Porcu, MD, professor and director, Division of Medical Oncology and Hematopoietic Stem Cell Transplantation, Jefferson Health.

Frontline decisions are driven by comorbidities, prognostic markers, and CLL factors, Chong said. “It’s really important to know IGHV mutation status,” she explained, referring to whether mutations are present in the immunoglobulin heavy chain variable region; if so, this can mean more favorable responses to chemoimmunotherapy.¹ The presence of high-risk features, such as del(17p) or TP53 mutations, by contrast, might call for venetoclax (Venclexta; AbbVie/Genentech).

Payer coverage can drive how much data are available. “I’m certainly not opposed to knowing additional [next-generation sequencing], although sometimes [there are] varying degrees of coverage with insurance,” Chong said.

Messmer noted that young patients may opt for fixed-duration venetoclax, and Porcu said social determinants of health matter as well. “It really has to be personalized,” he said. Comorbidities may put some treatments off-limits, but, he said, “The good news is that we have options.”

Which drugs to give first can be a complex decision, as Bruton tyrosine kinase (BTK) inhibitors can now be used in a fixed-duration regimen with venetoclax or continuously. But Tomasulo noted that promising results from the phase 3 AMPLIFY trial (NCT03836261) leave unanswered questions because patients with high-risk features were excluded.² These findings showed that acalabrutinib (Calquence; AstraZeneca) with venetoclax beat chemoimmunotherapy in first-line treatment, and adding obinutuzumab (Gazyva; Genentech) to the doublet was better still.³

As more data come in, Porcu said, “We’re going to learn a lot more about the patterns of progression and the patterns of resistance.”

Tomasulo said she looked forward to seeing how patients with high-risk features respond to the AMPLIFY triplet. Both she and Chong said they no longer start patients on the first-generation BTK inhibitor ibrutinib (Imbruvica; Janssen) due to its cardiac effects,⁴ although some patients have taken it for years and are doing fine.

Use of MRD. Panelists had nuanced views on the use of minimal residual disease (MRD) testing to gauge depth of response, following an FDA advisory panel endorsement of its use as a part of a surrogate marker in clinical trials.⁵ Messmer noted that use of MRD testing in AMPLIFY revealed how much the addition of obinutuzumab drove deep responses. He was “not rushing” to change treatment based on MRD results alone, but they have value in evaluating patients 3 months after a fixed-duration regimen ends.
Chong, meanwhile, said the use of MRD could allow patients to take a break from therapy. “I think there’s a lot of promise for using MRD, especially from the standpoint of cost control,” she said. “One of the challenges is, when can we stop?”

Sequencing therapy. Tomasulo shifted the discussion to therapy selection, asking fellow panelists about their approaches to picking the right drug up front, which will allow the longest possible response before patients must shift to a new regimen. “I often think of this as a game of chess and not checkers,” she said. “To get the best mileage out of each therapy involves consideration of cytogenetics and patient factors and optimal timing of each therapy.”

Testing is important here, Porcu said, with decisions driven in part by what adverse events (AEs) are anticipated. Messmer and Chong agreed these decisions are highly specific to each patient, and both said once patients move from a fixed-duration regimen to second-line treatment, chimeric antigen receptor (CAR) T-cell therapy is a viable option. Chong would like to see longer-term data for how patients fare with zanubrutinib (Brukinsa; BeOne Medicines). Even bispecific T-cell degraders, which have promising data, offer risk of infections.

With CAR T-cell therapy, Chong said, if a patient with CLL can achieve complete remission, “you have a chance at a durable response. And that’s huge.”

In NSCLC, Challenges With Testing and Debate Over Therapy Duration

Martin J. Edelman, MD | Image credit: Fox Chase

Advances in the treatment of non–small cell lung cancer (NSCLC) have changed the outlook for today’s patients—but cost does shape treatment, said experts on the evening’s second panel.

The rise of immuno-oncology (IO) has reset the bar from a clinical perspective, moderator Jessica Bauman, MD, associate professor and chief of the Division of Head and Neck Medical Oncology at Fox Chase Cancer Center, said as she opened the discussion. Her question to fellow panelists: How have cost and access challenges affected its adoption?

Tracey Evans, MD, medical oncologist/hematologist, Jefferson Health, said the cost of PD-1/PD-L1 inhibitors themselves has not been a barrier to adoption where she practices. “There’s a lot more controversy around the molecular testing,” she said, which is important in deciding who should and should not be on certain treatments.

With immunotherapy, Evans said, there is the potential for durable responses “in just about anybody,” regardless of the level of PD-L1 expression. “Even with the heaviest smokers,” she said, “you’ve got the potential that this is going to be a miracle.”

For Lova Sun, MD, MSCE, assistant professor of medicine, Penn Medicine, “IO has revolutionized treatment of metastatic non–small cell lung cancer from a one-size-fits-all chemotherapy approach.” Today, the interest is in moving treatments into earlier-stage settings, as seen in the multitude of studies in the periadjuvant and neoadjuvant stages.^6,7 Like Evans, Sun said she has not encountered insurance barriers for treatments, per se, but she sees roadblocks for molecular testing and referrals to multidisciplinary cancer centers.

“So, that’s a type of access that has been a challenge in the last 5 years or so, as we figure out how to optimize immunotherapy in the earliest setting,” she said.

Martin J. Edelman, MD, FACP, chair, Department of Hematology/Oncology at Fox Chase Cancer Center, agreed with these assessments. “IO therapies have had the greatest impact on the treatment of the broadest population in lung cancer. I never thought in my career I would be putting patients who had metastatic non–small lung cancer onto annual follow-up 5 years after their last treatment,” he said. When Edelman started his career, median survival “was measured in a matter of a few months.”

That said, the big leaps have come in the advanced setting, leaving unanswered questions for other patients: “Who gets immunotherapy by itself? Who gets chemotherapy and immunotherapy? Which of the chemoimmunotherapy regimens do you use?”

Competing therapies for these patients “are probably, for the most part, somewhat similar,” he said. “But there’s likely to be benefits for some based upon comutations. And we don’t really have great data on that.”

Duration of therapy. Bauman opened the discussion to how long patients should stay on immunotherapy. Sun explained that with IO, dosing and timing of treatment are harder to predict because the drugs are activating the immune system. “We’ve all seen patients with just a couple of doses—or even a single dose of immunotherapy—have a great response or terrible toxicity,” she said.

Most seminal trials with IO in lung cancer treated patients with durable responses for up to 2 years; a few allowed indefinite treatment, Sun noted. “It’s kind of notable that those trials had similar outcomes,” she said. “It wasn’t clear that indefinite treatment was beneficial in any way.”

With the cost of pembrolizumab (Keytruda; Merck) exceeding $100,000 per year,⁸ Sun said, it’s worth finding biomarkers that can identify which patients can safely stop therapy or modify treatment.

Evans said when she raises the idea of stopping therapy at the 2-year mark, many patients resist. “They’re not having any toxicity. They come in, they feel reassured that they’re getting their immunotherapy, and they don’t want to stop.”

She added, “I’ve never had an insurance company give me any pushback for continuing a patient’s immunotherapy beyond 2 years…. As hard as it is to get these drugs approved in the first place—get patients on treatment and get the authorizations—nobody blinks when I keep them going for 3, 4, or 5 years.”

Edelman noted that the 2-year mark “is purely arbitrary.” But since that time frame is used in most trials, it’s his outer limit. The prospect of inviting more treatment-related AEs creates added costs, and very few patients have relapsed.

“I do not continue beyond 2 years. It’s not the patient’s decision; it’s a medical decision. That’s where the evidence is.”

$2000 Medicare Cap Helps Patients Manage Myeloma Care

Alfred Garfall, MD | Image credit: Penn Medicine

Quadruplet therapy featuring an anti-CD38 antibody is now standard of care for frontline multiple myeloma treatment, which has increased survival^9,10 but also the complexity of decision-making, as patients may take combinations “for years,” according to the moderator of a panel on cost-effectiveness in treating this blood cancer.

The proliferation of new therapies “has created some interesting clinical decision-making and complicated decision-making for our patients,” said Alfred Garfall, MD, associate professor of medicine, Penn Medicine. Questions about value-based care arise, he said, “because all of these therapies are expensive, and they’re often given for a very long time,” as patients live longer.

Until recently, multiyear treatment with daratumumab (Darzalex; Janssen) or isatuximab (Sarclisa; Sanofi), alongside lenalidomide (Revlimid), bortezomib (Velcade), and dexamethasone (VRd), has driven financial toxicity, due to Medicare cost-sharing rules that required higher cost-sharing for lenalidomide, an oral therapy covered by Part D.

However, the January 1, 2025, start of the Part D $2000 out-of-pocket cap, a provision of the Inflation Reduction Act, has been a game changer for patients,¹¹ said panelist Edward Stadtmauer, MD, director of hematologic malignancies clinical/translational research, Division of Hematology/Oncology, Penn Medicine.

“If that really holds together, then that’s going to substantially improve the financial toxicity for patients,” Stadtmauer said. He spoke of the “microeconomics” and the “macroeconomics,” of paying for therapy, and the questions raised. “Certainly for society, these are very expensive things and in some ways, we’re happy victims of our success…. I personally have patients who are 30 years with myeloma, getting [these] therapies.”
But, he said, “In terms of the individual patient, now that should not be a major cost for the patients in their day-to-day lives.”

The panel, which also featured Dan Vogl, MD, MSCE, associate professor of medicine, Penn Medicine; and Peter Abdelmessieh, DO, MSc, of the Department of Bone Marrow Transplant and Cellular Therapies, Fox Chase Cancer Center, discussed how to balance cost concerns and clinical efficacy, given evidence that now shows the quadruplet combination works well in both transplant-eligible and transplant-ineligible patients.^10,12 The arrival of subcutaneous daratumumab has made administration much easier.
While Garfall said frontline use in transplant-ineligible patients is justified, it’s a different story for the others. “I worry the most in the transplant-eligible patients, where we’ve made a wholesale change,” he said. The shift is not very expensive, not to the individual patient, but there is a cost in making “wholesale change…based on a progression-free survival advantage with daratumumab compared [with] the same regimen without daratumumab.”

Garfall continued, “I think we’re going to have the opportunity in the coming years to look at plenty of progression-free survival advantages for even more expensive, more complicated therapies, and we’re going to have to decide: Is that really a good enough end point for additional toxicity, especially, but also for the financial cost?”

Maintenance strategies and MRD. Panelists discussed the merits of adding an anti-CD28 antibody to lenalidomide in the maintenance phase, which may or may not be driven by a patient’s MRD status. Beyond financial considerations, Abdelmessieh said the key question is, how bad was the patient’s disease up front?

Many factors contribute to this clinical decision, and he would consider adding daratumumab in high-risk patients post transplant, especially if they have residual disease. Stadtmauer noted results from the phase 3 PERSEUS study (NCT03710603) showed adding daratumumab to lenalidomide in the maintenance phase increased PFS over 4 years.¹⁰

Vogl concurred with Abdelmessieh. “I totally agree that the main situation where I’ve added daratumumab during the maintenance therapy is for people with residual disease after stem cell transplant, because I’m kind of worried that just lenalidomide by itself isn’t going to be enough to hold them, and the toxicity of [daratumumab] is so low,” he said. “It is not yet part of my practice to do a posttransplant bone marrow biopsy and send it for MRD, so I’m mainly looking at their serum markers as a measure of whether they have residual disease.

Use of MRD may be valuable in evaluating groups of patients, Vogl said, but that doesn’t mean it’s as valuable for decision-making with an individual patient. “I think there certainly could be an interesting discussion of, do we really know what to do with MRD enough to be doing it as part of routine clinical practice, and is there truly value in measuring MRD in myeloma at this point, outside of a clinical trial?”

Moving CAR T up in the queue. The panel concluded with a discussion of where CAR T-cell therapy and bispecifics fit into treatment sequencing. Garfall noted that most of the studies involve high-risk patients, but the panelists weighed whether more typical patients could benefit from fewer toxicities and a “fixed-duration” solution, especially if CAR T could be offered on an outpatient basis.

Abdelmessieh said being at a smaller facility offers advantages with more complex treatments such as CAR T. “Everyone knows who you are, and your patients aren’t going to get lost,” he said.

Defining Value and Ensuring Affordable Access in Modern Oncology

Andrew Chapman, DO, FACP | Image credit: Jefferson Health

The evening’s final panel delved into thorny questions of finding value in cancer care, with panelists highlighting examples of good intentions running into roadblocks. Moderator Andrew Chapman, DO, FACP, director, Sidney Kimmel Cancer Center, Jefferson Health, promised a “spicy conversation” with panelists of varied backgrounds. They were as follows:

Valerie Csik, MPH, CPPS, director, Quality and Care Transformation, Jefferson Health; Henry Fung, MD, FACP, FRCPE, chair and professor, Department of Bone Marrow Transplant and Cellular Therapies, Fox Chase Cancer Center; and Ramy Sedhom, MD, clinical director for medical oncology at Penn Medicine Princeton Health and associate director, Penn Center for Cancer Care Innovation, with leadership roles in geriatric oncology and palliative medicine for Penn Medicine.

Chapman, who is also codirector of the Senior Adult Oncology Center at Jefferson, started by “ripping the Band-Aid off” with a question about what metrics matter in measuring value.

“How do we even define value?” Sedhom asked. “Value might have one definition for a payer, which might be what’s the most cost-effective therapy, or what’s the therapy that keeps the patient out of the hospital and decreases utilization. But sometimes we really have trouble defining what benefits are most important to our patients.”

Older patients, for example, might ask whether drugs will affect cognition or put new burdens on their caregiver. It’s not that cost does not matter, as Sedhom explained, but “the value-based models that have been proposed are probably not the most patient-centered.”

Csik agreed that many value-based models “are not prioritizing the value of patients.” So far, she said, “there’s a lot more emphasis on the cost component than on the quality component…. The answer to the question, in my mind, is giving more emphasis to the quality.”

Too often, Fung said, value is not discussed. It’s not taught at medical or business schools. “So, we don’t talk about it, and then we don’t really have a system to measure it,” he said.

Patient outcomes are not the only measures that count, Fung said. He shared an account from a man who did not want any treatment—it would not improve quality of life for his wife or his daughter; the daughter had just lost her job, and bringing her father for treatment would cost her time with her children. Including caregivers in decisions matters, Fung said, as does redefining cost.

“Do we measure [whether a] patient is still disabled after treatment? We never measure about this. How many patients go on disability? How many patients go on Medicare and Medicaid?”

Chapman noted that many measures penalize hospitalizations, although some are necessary. Sedhom decried the historic lack of emphasis on palliative care, although it was a major theme of the 2024 Plenary Session at the American Society of Clinical Oncology (ASCO).¹³ A study at the ASCO Quality Care Symposium that repurposed a drug from the 1980s for nausea represents “a really low bar,” he said.¹⁴

Unintended consequences. Medicare’s oncology models, first the Oncology Care Model and now the Enhancing Oncology Model, have not gone far enough in addressing health equity, Sedhom said. He cited an example that is a huge point of frustration among community oncologists—the design of Principal Illness Navigation (PIN) codes to help compensate practices for guiding patients through treatment.

“We wanted to incentivize community navigators and community health workers,” after clinical trials have shown these services improve outcomes, he said. “But thanks to Medicare, there’s a co-pay for it, so that’s not really helping your patient who probably needs it most.”

Later, Sedhom noted that if practices accept PIN funds to pay for navigation, it can undermine their ability to find new savings in the total cost of care.
Chapman asked whether there any lessons from these examples.

“First, we don’t have the metrics that we all agree on,” Fung said. Academic medical centers and community practices offer different levels of care, and perhaps the same models should not apply. Too few patients are enrolling in clinical trials, due to funding and other constraints.

“Things are not working, he said. “We need to build something completely different.”

Sedhom said with the bulk of the cost of treatment being tied to drug costs, it’s unfair to criticize practices for being unable to shave off more savings from what’s left. He sees a need to reform the Orphan Drug Act,¹⁵ because the abundance of new genomic markers and new indications means the cost of drugs never comes down.

Said Csik, “We need to flip the script on the model—focus on the quality, and the cost will follow.”

Fung favored solutions that home in on the cost of cancer drugs, because this is where patients and their caregivers feel the most strain. Now that patients are living longer, the accumulating costs of therapy over multiple years needs more attention. Medical students need early exposure to these realities, he said.

Payers, Sedhom said, “in the spirit of doing the right thing,” sometimes adopt their own pathways that differ from the ones in use, or the ones that make the most sense for a specific patient. Sedhom said his 90-year-old patient with metastatic HER2-positive breast cancer should be approved for an off-pathway treatment if she doesn’t want HER2-directed drugs, for example.
When Chapman asked what policies needed reform, Csik said prior authorization topped the list. “I think that would be a tremendously valuable metric to be included as part of some of these value-based contracts.”
Added Fung, “I think we need a revolution.”

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