Value Frameworks in Cancer Care: A Beginning, Not the Solution

Roxanne Nelson, BSN, RN

June 28, 2016

Defining the value of a drug in relation to its cost has become a rapidly emerging theme in the cancer field, and several frameworks have emerged in the last year or so to help oncologists grapple with this issue. However, these value frameworks are not a solution but rather "a provocative beginning, to help us think more systematically about how we incorporate value in ever-increasing costs of cancer therapy," said Deborah Schrag, MD, MPH, a professor of medicine at Harvard Medical School in Boston, Massachusetts.

It is not yet practical to use frameworks for making clinical decisions at the bedside, she warned, speaking at the plenary session of the recent American Society of Clinical Oncology (ASCO) 2016 Annual Meeting.

"One reason is that treatment costs vary enormously and it is difficult to obtain that information," she said. "Software tools may help and ASCO is developing those."

But that said, she emphasized that "deliberations about value cannot be ignored…even though they are noxious."

Deliberations about value cannot be ignored…even though they are noxious. Dr Deborah Schrag

"The unaffordability of cancer treatment has adverse consequences on our patients, on us, and society as a whole," she added.

The cost of cancer treatment is rapidly rising, with no end in sight, and this is most evident by the ever-growing price tag on new drugs coming out of the pipeline.

The average monthly cost for an FDA-approved drug in 2015 "broke the $12,000 watershed," Dr Schrag noted.

"Promising drugs and combinations discussed at this meeting have pushed beyond that ceiling and are at the $30,000 per month mark," she said.

Thus, even though oncology drugs are highly valued, they are becoming increasingly unaffordable for many patients, and this is true in affluent countries such as the United States as well as elsewhere around the world.

In an ideal world, and if resources were not an issue, any new innovation could be adopted. "But that is not the case," Dr Schrag pointed out. "We need to make choices, and how do we decide which innovations to adopt?"

That is where value frameworks come in.

Five Frameworks Launched Recently

Value is often defined as the net health outcome achieved over the cost required to achieve it. But both the numerator and denominator in this "deceptively simple formula" are exceedingly challenging to measure, she cautioned.

With treatment becoming increasingly expensive, the question that arises is how to decide whether a treatment is "worth it" to a healthcare system given competing priorities on constrained resources, Dr Schrag said.

"Value frameworks are intended to help with difficult decisions we wish we could avoid, and ASCO and other groups have decided to tackle this head on," she added.

Value frameworks are intended to help with difficult decisions we wish we could avoid. Dr Deborah Schrag

Five different value frameworks launched in oncology in 2015–2016, and while they have overlapping similarities, each one differs with respect to purpose, focus, and means of assessment.

Peter Bach, MD, director of the Center for Health Policy and Outcomes at Memorial Sloan Kettering Cancer Center, New York City, designed the DrugAbacus, a tool that may help determine a more appropriate price for a specific agent, based on what experts feel are possible components of a drug's value.

The Institute for Clinical and Economic Review (ICER), an independent, nonprofit, research-based organization, released a Value Assessment Framework that is composed of both a clinical care value assessment and a health system value assessment that includes cost modeling and expert input.

The National Comprehensive Cancer Network (NCCN) issued its NCCN Evidence Blocks, which uses "blocks" to visually represent five key value measures — efficacy, safety, quality of evidence, consistency of evidence, and affordability — to provide important information about specific recommendations found in the NCCN guidelines.

ASCO has introduced its Value Framework, which defines value as a measure of clinical benefit, toxicity, and cost.

The European Society for Medical Oncology (ESMO) has issued its Magnitude of Clinical Benefit Scale, which seeks to "stratify" a drug's clinically meaningful benefit and includes measures of efficacy (ie, survival) as well as the prognosis of the condition and the toxicity of the drug.

How Do They Rate?

Dr. Schrag showed how these frameworks could work in practice by applying 3 of them — the frameworks devised by ASCO, ESMO, and ICER — to new treatment approaches that were discussed at 4 presentations made at the plenary session of the annual meeting.

The first of these presentations, which received the "highest value scores" from the frameworks, was a trial that showed improved 3-year overall survival in children with high-risk neuroblastoma who received tandem myeloablative autologous stem cell transplant using peripheral blood stem cell as consolidation

Dr Schrag said that, from the perspective of the healthcare system and the individual, the value for tandem transplant was very high because it increased cure rates for young children at a cost of approximately $50,000.

ASCO scored it at 76, which is very high (range, 0 to 130), while ESMO gave it a grade A. ICER assessed the treatment intervention as falling in the range of greater than 50,000 per quality-adjusted life-year (QALY) gained.

The second study found that the combination of daratumumab (Darzalex, Janssen) with bortezomib (Velcade, Millennium Pharmaceuticals), given with dexamethasone, achieved about a 60% reduction in death or progression in patients with relapsed or refractory multiple myeloma compared with bortezomib with dexamethasone.

This abstract also received high value scores, but they were more tempered because of the high cost of treatment.

ASCO gave it a high score of 46 (range, 0 to 130); ESMO, a grade 4; and ICER, $200,000/QALY gained (overall survival is not yet reported).

There was a significant progression-free survival benefit (hazard ratio, 0.39), which could potentially translate to improved overall survival, but the cost per month is greater than $10,000 per patient.

The individual value is high but will result in financial strain, and value to the healthcare system was evaluated as moderate to high, Dr Schrag pointed out.

The third abstract showed improved survival in elderly patients with glioblastoma multiforme who received chemotherapy with temozolomide in addition to radiotherapy. Chemoradiation is already the standard of care in this disease, and these new findings showed that it can be successfully used in elderly patients (all older than age 65 years).

"Temozolomide is generic, it is already available, and physicians have been able to use this for quite some time," said Dr. Schrag, but despite this the framework value scores hovered in the moderate-high range.

ASCO scored it at 43 (range, 0 to 130), which is moderate-high, ESMO gave it a grade 3, and ICER projected greater than $100,000/QALY gained.

There is a significant overall survival benefit in this uncommon cancer with a poor prognosis, and treatment runs about $400 to $4000 per month, Dr Schrag pointed out. "From a health system perspective it was viewed as moderate and the drug is of low value in the absence of MGMT mutation."

The last abstract revealed the lowest value scores. The results from the MA.17R study showed small but significant improvement in disease-free survival by adding an additional 5 years of therapy with letrozole in postmenopausal women with early-stage breast cancer, but there is also an increase of adverse effects.

ASCO scored it 8 (range, 0 to 130), which is low value; ESMO gave it a grade B; and ICER could not evaluate it in terms of QALY gained because there is no survival benefit.

There is better disease-free survival (hazard ratio, 0.66), fewer second breast events, but identical survival so far at 6 or more years, Dr Schrag noted. "There is also an increased risk of osteoporosis and fractures and excess cost required to treat those complications."

The costs of the drug are relatively low at less than $500/month, but from a health system perspective this intervention is rated as low in each of the value frameworks, she said. "Instead, it is a priority to focus on adherence to the first 5 years of therapy."

Individual value is highly variable because it is a preference-sensitive decision based on risk and treatment tolerance, she added.

Dr Schrag reported a consulting or advisory role with New Century Health, Ohio State University, and a relationship with the Journal of the American Medical Association.

American Society of Clinical Oncology (ASCO) 2016 Annual Meeting. No abstract. Presented June 5, 2016.

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