Publication
Article
Evidence-Based Oncology
Author(s):
Therapeutic innovations in cancer care have the potential to change outcomes significantly for a growing number of patients, but cost and access remain issues that must be addressed to improve care equity and overcome disparities caused by social determinants of health. Sessions at an Institute for Value-Based Medicine event held in Charlotte, North Carolina, featured experts who spoke to recent developments in cancer therapy and ongoing challenges in implementing value-based care in oncology.
Novel Therapeutics and Efficacy End Points in Multiple Myeloma
Donald Moore, PharmD, BCPS, BCOP, DPLA, FCCP | Image credit: Atrium Health
Moderator Donald Moore, PharmD, BCPS, BCOP, DPLA, FCCP, clinical pharmacy manager for hematology-oncology at Atrium Health Levine Cancer Institute, guided a discussion on innovations in multiple myeloma with panelists Christopher Ferreri, MD, a hematologist-oncologist at Atrium Health Levine Cancer Institute focusing on plasma cell disorders, and Barry Paul, MD, a hematologist at Atrium Health Levine Cancer Institute focusing on plasma cell dyscrasias.
In recent years, there has been significant scientific advancement in the multiple myeloma space, Moore noted, including updates in the newly diagnosed multiple myeloma setting, different types of end points, and novel immunotherapeutics coming to market.
One such advance for newly diagnosed patients with multiple myeloma has been the advent of anti-CD38 antibodies, Paul explained, with the gold standard being the addition of an anti-CD38 antibody to a bortezomib, lenalidomide, and dexamethasone (RVd) backbone based on research in transplant-eligible patients. In those who are not eligible for transplant, Paul noted that data from the phase 3 MAIA trial (NCT02252172) have shown unprecedented progression-free survival (PFS) when the anti-CD38 antibody daratumumab is added to lenalidomide and dexamethasone.1 Adding an anti-CD38 antibody to a triplet backbone is also being explored in trials for transplant-ineligible patients,2 and potential benefits may sometimes outweigh toxicity risks on a case-by-case basis.
“This is something that is a conversation to have with your patient, and also it’s important to recognize whether or not a patient can withstand some mild peripheral neuropathy,” Paul said. “A patient who’s already frail, who has baseline peripheral neuropathy, maybe poorly controlled diabetes, would not do well with that.”
Still, Ferreri and Paul agreed that quadruplet regimens may be in the cards for transplant-ineligible patients, particularly those who are not frail. Quadruplet therapy may even provide additional benefit vs triplet therapy among patients who reach a minimal residual disease (MRD) status, Ferreri explained.
“Those patients [who] are fit but transplant ineligible, I think they benefit from the addition of the anti-CD38 treatment, even if you get patients into MRD status,” Ferreri said. “It’s interesting that MRD is basically looking at minimal residual disease at 10–6, and you can get patients into that whether they get a quad or a triplet—but it seems like if they take the quad and they get MRD, they still have a better [PFS] than if they get into MRD with a triplet.”
There has also been much discussion around whether MRD, a relatively new marker for treatment response, can be used to tailor therapy, Moore noted. While Paul said he would likely not base the decision to escalate therapy on MRD status, he may factor it into the decision to hold therapy, he said.
“I think the data need to mature, and I certainly wouldn’t de-escalate therapy in someone who’s high risk, but [with] a standard-risk patient who has had sustained MRD negativity, it’s a conversation, absolutely,” Paul said.
A host of novel therapies are also making their way to the clinic, including autologous chimeric antigen receptor (CAR) T-cell therapies and bispecific antibodies in the relapsed or refractory setting. These therapies have potential to significantly improve survival. “Now we have the benefit of having multiple options in this setting, but it’s our job to figure out how to best sequence them and figure out which line of therapy to best use them in,” Ferreri said.
Both CAR T-cell therapy and bispecifics come with challenges around cost, and CAR T-cell therapy comes with manufacturing challenges because they are manufactured from the patient’s own cells. Despite high costs, Ferreri noted CAR T cells and other novel therapies may be less expensive over time if administered early when patients go into longer-term remissions. Shifts toward outpatient administration of novel therapeutics traditionally administered in the inpatient setting could also help reduce costs, Ferreri said.
Treating Lung Cancer in the Era of Targeted and Immunotherapies
Jai Patel, PharmD | Image credit: Atrium Health
Precision medicine in lung cancer was the focus of a session including lung cancer experts Mohamed Mohamed, MD, hematologist-oncologist at Cone Health in Greensboro, North Carolina; Shelby Quinn, PharmD, oncology clinical pharmacy specialist at Levine Cancer Institute; and Christopher Pallas, MD, a thoracic medical oncologist at Atrium Health Levine Cancer Institute. The session was moderated by Jai Patel, PharmD, chair of the Department of Cancer Pharmacology and Pharmacogenomics and vice president of the translational research program at Atrium Health Levine Cancer Institute.
“We’ve had an unprecedented number of approvals and innovations within the lung cancer space over the last several years,” Patel began, asking panelists for their input on the recent changes in the treatment space.
Mohamed spoke to the significant advances made since the days when chemotherapy combinations were the only way to approach cases of advanced lung cancer. Then, with the revelation that certain patients have mutations that can be targeted via therapy, the potential survival benefit of oral agents such as erlotinib became clearer. In the time since, targeted therapies have changed the game for patients with actionable mutations.
“I think what speaks a lot to lung cancer is the fact that we’re finding targeted agents for patients [who] have mutations that are in 2% of non–small cell lung cancer,” Quinn added. “ROS1, MET, HER2—so that these patients with such rare mutations still have treatment options. And I think that just says a lot about non–small cell lung cancer and the advancements we’re making for not just all our patients but those [who] have these more rare mutations as well.”
In addition to targeted therapies, immunotherapies have improved the outlook for patients who do not have targetable mutations, Pallas said. Mohamed echoed the sentiment, adding that he had his reservations when immunotherapies were first introduced in lung cancer.
“At the beginning, I was very skeptical that immune therapy would help in lung cancer,” Mohamed said. “Now, I see a lot of patients [who] completed 2 years of treatment with immune therapy and are still coming to me after 5 or maybe more years that they are not on treatment, and they still have no evidence of disease progression.” Targeted therapies are different, he noted, in that patients must remain on them because disease can flare if they stop.
Patel posed the question of how clinicians should sequence the growing number of therapies in the lung cancer treatment armamentarium. While treatment is becoming more nuanced and complex with these developments, Pallas said the most important element is shared decision-making with each individual patient.
Generally, Pallas explained, the goal of treatment in stages I through III has curative intent, while stage IV lung cancer is typically treated with palliative care. In patients with resectable disease, he noted a potential shift from strictly adjuvant therapy to neoadjuvant immunotherapy, resection, then adjuvant immunotherapy for potentially improved outcomes.
Regarding sequencing in those with stage IV disease, broad molecular profiling is a crucial step to identify any targeted therapies and PD-L1 status. Some patients, specifically those with EGFR or ALK mutations, may be immune resistant, Pallas noted. Mohamed also highlighted the importance of molecular profiling, which should be done earlier in cases where neoadjuvant immunotherapy is being considered.
“As we try to streamline clinical trial options, a big part of our molecular tumor board is highlighting those patients [who] do have a clinical trial based on their results, so we can make sure we get the patients—especially if a trial is available, we usually feel very strongly about that—the care that we think is going to be better and the new treatment in the future for them,” Pallas said.
Aligning Definitions of Value in Cancer Care
Defining value from the perspectives of various stakeholders is key in oncology care, and multidisciplinary collaboration can help ensure optimal treatment, experts in a session covering pharmacy decision-making in oncology agreed. The panel, which was moderated by Donald Moore, included Alexandra Wolff, PharmD, clinical pharmacist coordinator at Atrium Health Levine Cancer Center; Andre Harvin, PharmD, chief pharmacy officer and vice president of procurement at Cone Health; and Heather Moore, PharmD, clinical pharmacist in breast oncology at Duke Cancer Center.
At Levine, an outpatient stem cell transplant unit opened in November 2024, which has allowed for the administration of autologous transplants and CAR T-cell therapies entirely in the outpatient setting, Heather Moore explained.
Outpatient vs inpatient transplants and CAR T could lower the cost of care but also improve the patient experience and align multiple definitions of value in cancer care.
“Value is one of those words that we use a lot in health care, and I think it has really different meanings depending on what chair you’re sitting in,” Wolff said. “One of the things I’ve gotten to see a lot in the outpatient unit is that value to patients means things that are a lot different in terms of time, in terms of quality, in terms of autonomy.” For many patients, she said, being able to continue with a relatively normal life outside of their health care is a valuable aspect of outpatient procedures.
The panel also agreed on the importance of multidisciplinary care, which Wolff credits with the success of the outpatient transplant unit at Levine Cancer Center. It is especially important in this setting to learn what other team members do and be willing to assist in a variety of ways rather than being siloed, she explained.
Harvin agreed and spoke to how having a clinical pharmacist practitioner (CPP) focusing on breast cancer at Cone Health has helped get more patients seen each day and helped oncologists manage their workloads. For patients with stage IV disease, they could see their oncologist every 5 to 6 weeks instead of every 3 to 4 weeks and see a CPP in between.
“They established a relationship with those patients and then even started doing things more on the front end with greater education for the patients as they were diagnosed and when they started to come into the cancer center,” Harvin said. The CPP became a crucial role at the center, he explained.
Panelists also discussed integrating data into coverage and treatment decisions. While Harvin noted the importance of real-world evidence for coverage decisions, Wolff said that although real-world evidence can play into treatment discussions and decisions, earlier data are typically factored in at Levine to get new drugs to patients as soon as possible.
“I think prospective and real-world data both have their place, and both are important,” Heather Moore said. “When I think of the perfect example of this, I think of antibody-drug conjugates.” She explained that in breast cancer, there are approved antibody-drug conjugates that showed better PFS and overall survival in specific patient populations in clinical trials but tend to have different outcomes when utilized in clinical practice or sequenced in certain ways, and those data are key.
Karen Winkfield, MD, PhD | Image credit: Vanderbilt University Medical Center
Karen Winkfield, MD, PhD, professor of radiation oncology at Vanderbilt-Ingram Cancer Center and executive director of the Meharry-Vanderbilt Alliance, a strategic partnership between Meharry Medical College and Vanderbilt University Medical Center, moderated a final session on value-based oncology care. The panel included Greg Knight, MD, a hematologist-oncologist focusing on bone marrow transplant at Levine Cancer Center who also researches financial toxicity in cancer; Justin Favaro, MD, PhD, of Oncology Specialists of Charlotte; and Stacey Hammer, PharmD, director of pharmacy for population health at Cone Health.
Favaro spoke to the need for a value-based system in oncology care, noting that in recent years, fewer health systems function within the Charlotte area, and a lack of true competition between health systems allows prices to stay high.
“The markup for chemotherapy is ridiculous when patients get treated at the hospital, so we don’t have a value-based system,” Favaro said. “We have a system where really there’s lack of value. You have the same exact chemotherapy drug [that] can be given at an outpatient setting for much lower cost, or a hospital-based network for a much higher cost. And we’re seeing that just doesn’t make sense.”
Winkfield asked how value-based care models might be used to reduce disparities in care access, emphasizing value from a societal perspective. The shift away from fee-for-service payment could help increase access, as would a greater focus on social determinants of health, Knight explained.
“You’re extracting the maximum amount you can, and there [are] huge disparities between service lines and different disease types in terms of how much you’re getting reimbursed for what you’re doing, and it leads to massive disparities in care,” Knight said of the fee-for-service system. With value-based care, the discussion is around how patients are affected by their care and whether being reimbursed for care based on performance or outcomes would create a more accessible, equitable system.
Favaro’s interest is in why the health care system in the US is as expensive as it is, noting that while many advocacy organizations point to aspects of insurance, such as prior authorization, another major piece of the puzzle is prevention. Up to 40% of cancers are preventable,3 he explained, but this concept is not given enough attention.
“All of us up here should be really going out to the community and saying, ‘These are the things you need to do so you never become our patient,’” Favaro said. “Diet, exercise, get your vaccines, etc. But it’s also the way our health insurance system is set up.” The structure and overregulation of insurance has also played a part in inflating health care costs, he said. And consolidation—both between payers and health systems as well as the formation of massive health care systems—is another factor that comes into play, Favaro explained.
In terms of how value-based models might alleviate the factors driving high costs, Hammer said that in addition to things like bundled payments or accountable care organizations taking on full risk in payment models, lowering out-of-pocket costs for patients can include steps like utilizing different team members or locations with lower facility fees. At Cone Health, they also leverage community health workers and meeting the patients where they are with mobile units and telehealth.
Medication costs and insurance costs are both ongoing issues when it comes to care access, the panelists agreed. Addressing disparities overall will take policy-level changes.
“A lot of reform needs to be done to actually make it affordable but also encourage guideline-directed therapy and make sure that the access is there,” Hammer said. “Looking back to our first question—the disparities and the [social determinants of health] and all of those barriers that the insurances don’t necessarily help us with, we’ll have a lot of patients [who] might be able to get transportation to their oncologist but can’t get transportation then to the pharmacy, so then they don’t pick it up. The insurance will pay for the visit to the doctor, but they won’t pay for transportation to the pharmacy. I think there [are] broken pieces there where they’re not taking care of the patient as a whole, and it’s still very episodic.”
References
1. Study comparing daratumumab, lenalidomide, and dexamethasone with lenalidomide and dexamethasone in participants with previously untreated multiple myeloma. ClinicalTrials.gov. Updated April 1, 2025. Accessed April 22, 2025. https://clinicaltrials.gov/study/NCT02252172
2. Usmani SZ, Facon T, Hungria V, et al. Daratumumab plus bortezomib, lenalidomide and dexamethasone for transplant-ineligible or transplant-deferred newly diagnosed multiple myeloma: the randomized phase 3 CEPHEUS trial. Nat Med. 2025;31(4):1195-1202. doi:10.1038/s41591-024-03485-7
3. Steinzor P. New study reveals 40% of US cancer cases, nearly half of deaths linked to modifiable risk factors. AJMC. July 11, 2024. Accessed April 22, 2025. https://www.ajmc.com/view/new-study-reveals-40-of-us-cancer-cases-nearly-half-of-deaths-linked-to-modifiable-risk-factors