NCCN: Costly Innovative Cancer Therapies Pose New Challenges-Financial and Otherwise

Innovative treatment strategies such as chimeric antigen receptor (CAR) T-cell therapy offer dramatic benefits for some cancer patients, but integrating them into clinical practice can be challenging and may require new models of reimbursement, according to panelists at the 2019 National Comprehensive Cancer Network (NCCN) Annual Conference, held March 21–23 in Orlando, Florida.

“Innovation has very broad implications, like pebbles dropped in a pond,” said roundtable moderator Clifford Goodman, PhD, of The Lewin Group in Falls Church, Virginia. “There are differences between outcomes and patient experiences in clinical trials and in the real world.”

Integrating CAR T-cell therapies into clinical practice has required hard work and organization, said panelist Frederick L. Locke, MD, of the Moffitt Cancer Center in Tampa, Florida. The expertise required means that CAR T is administered at relatively few regional centers, and the astronomical cost of the engineered T cells themselves-which can run anywhere from $500,000 to $1.5 million per patient-are just part of the overall cost of these treatment strategies, panelists noted.

Less frequently discussed are the “hidden costs” of coordinating care and managing adverse events, said John W. Sweetenham, MD, of the Huntsman Cancer Institute at the University of Utah in Salt Lake City. “It’s important to take a holistic approach to the true cost of treatment, rather than just the cost of the agent,” he said.

In the United States, physician practices or hospitals, rather than biopharmaceutical companies, are reimbursed by insurance systems, noted panelist Jennifer Malin, MD, PhD, of the UnitedHealth Group-typically at a margin above the actual cost of the product.

“That can be as low as 6% or as high as three or four times the cost of the product at elite institutions,” she said. “The ‘margin’ approach from the early days of Medicare made sense for, say, doxorubicin, but a number of new innovative therapies are very expensive.”

Immune checkpoint inhibition can cost just under $20,000 per month, for example-just for the agent. “So, a three or four-times multiplier is quite a bit,” Malin said.

Therapeutic radiopharmaceuticals are in the range of $50,000 to $100,000 per dose, she noted.

“One big challenge-the elephant in the room-is, what is the reasonable profit margin for administering a drug for a practice or hospital?” Malin said. “Is $50,000 or $100,000 reasonable for administrative costs for a patient’s treatment? A million dollars seems excessive.”

The high cost of innovative cancer treatments makes it difficult to cover the costs of treatment under current Medicare fee schedules, Malin added. And demand for such treatments will climb.

“Hospitals performing CAR T therapy are adhering to the FDA label, but we anticipate studies showing it’s effective outside the FDA label,” Malin noted. “So how will we ensure there isn’t regional variation in who gets it? We don’t want to see inequities based on where patients live.”

It is not yet clear how best to reimburse for such therapies. However, because CAR T-cell therapies require weeks to prepare for each patient, it’s important to begin patient education conversations early, panelists agreed.

“The price tag is expensive, but that should not stop you from referring your patients,” Locke said. “You need to advocate for your patient and get them to centers. Get patients to centers early.”

One urgent goal that could benefit patients while reducing costs is identification of better biomarkers and risk-stratification systems that can spare patients who are unlikely to benefit from a given therapy due to its potential side effects.

“As we develop these therapies, the better you can identify the patient population that [will] benefit, the better able you are to cover that population,” noted Lalan Wilfong, MD, of Texas Oncology in Dallas.

It is also important to consider how innovative treatments fit into “the total continuum of care” a patient will undergo during her cancer treatment, Wilfong added. New treatments represent “tremendous innovation for some patients who are living longer and better lives, but for others, innovation’s benefits have been small,” he noted.

Patient-reported outcomes (PROs) are playing an increasingly important role in clinical trials, roundtable panelists agreed. Doing so ensures that patients are not only living longer, but are also enjoying a good quality of life, Malin said.

“How are we going to know which additional supportive care products or approaches will produce the best outcomes for patients, as opposed to just survival time?” Malin asked.   

“When we are asked only a few specific, limited questions, that limits what data are available,” said panelist Stefanie Joho, a cancer survivor and patient advocate. “I know from my experience, I was very frustrated because I experienced side effects [not included in the study] that I felt were important to get on record for an entirely new drug or modality. I think until we really treat patients as research partners, we’re selling ourselves short.”

Successfully implementing innovative treatments like CAR-T cell therapies requires a coordinated team approach, Joho said. “That includes everything from managing side effects [and working with] ER doctors [and] nurses-it really takes a team to make these treatments effective.”

The roundtable was sponsored by Bristol-Myers Squibb and supported by Abbvie; Astellas; AstraZeneca; Celgene Corporation; Eisai Inc.; Foundation Medicine; Gilead Sciences Inc; Halozyme; Janssen; Karyopharm Therapeutics Inc.; Loxo Oncology; Novartis; Pharmacyclics, an AbbVie Company; and Sanofi-Genzyme. Sponsored by Bristol-Myers Squibb.The Lewin Group is owned by Optum, a UnitedHealth Group company.