Value Frameworks in Oncology: Economic Analyses of 4 Clinical Interventions

Chase Doyle

September 2016, Vol 7, No 8 - Value in Oncology


In the past year, the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), and the Institute for Clinical and Economic Review (ICER) have introduced frameworks that seek to determine clinical value in relation to cost for a variety of cancer treatments.

“Given competing priorities on constrained resources, the value frameworks are intended to help providers decide whether a cancer treatment is worth it to a healthcare system. The focus is on prioritization and policy setting, not individual decisions or rationing at the bedside,” said Deborah Schrag, MD, MPH, Chief, Division of Population Sciences, Dana-­Farber Cancer Institute, Boston, at the 2016 ASCO annual meeting.

ASCO’s Value Framework incorporates a scoring algorithm that combines the clinical benefit, toxicity, durability of response, palliation benefit, quality of life, and accomplishing a treatment-free interval. The total points assigned to a specific treatment constitute the net health benefit (from 0 to 130), which is to be considered along with the patient’s out-of-pocket cost and the monthly drug acquisition cost.

ESMO’s Magnitude of Clinical Benefit Scale uses an algorithm for estimating the benefit of a treatment regimen for palliative (grades 1-5) and curative (grades A, B, and C) purpose to help decision makers in the European healthcare systems; grade 5 and grade A represent high clinical benefit.

ICER’s Value Assessment Framework uses incremental cost per outcomes achieved to appraise the value of treatment. For example, high value is defined as <$100,000 per quality-adjusted life-year (QALY) gained; intermediate value ranges from $100,000 to $150,000 per QALY; and low value is >$150,000 per QALY.

Dr Schrag applied these frameworks to estimate how 4 new treatment interventions discussed at the plenary session at ASCO 2016 measured in terms of their cost-effectiveness.

Value of Transplant in Pediatric Neuroblastoma

The value of tandem autologous stem-cell transplantation (ASCT) for pediatric neuroblastoma is likely to be very high, based on an ASCO value score of 76 (of 130), ESMO value score of grade A, and a projected cost of <$50,000 per QALY gained, representing high value for ICER.

The value to the healthcare system is also likely to be very high, and high-income countries should and will likely adopt ASCT for pediatric neuroblastoma. Middle-income countries will likely seek to adopt ASCT as well, although access to care, especially specialty care, may be challenging, Dr Schrag said.

Value of Daratumumab for Relapsed Multiple Myeloma

When adding daratumumab (Darza­lex) to bortezomib (Velcade) and dexamethasone for multiple myeloma, ­ASCO’s value score is 48, which is high; ESMO’s value score is grade 4; and with a projected cost of >$200,000 per QALY gained, would make this a low value for ICER, especially because overall survival benefit has not yet been shown with the addition of this agent. However, the overall value of this novel drug alone will also be very high, because daratumumab has increased progression-free survival, which will likely translate to an overall survival benefit, Dr Schrag reported.

“However, at a cost of more than $10,000 per month, this drug will result in financial strain for individuals who do not have adequate health insurance coverage. Therefore, from a health system value perspective, the rating for this intervention is moderate-to-high,” she said.

High-income countries will likely adopt or seek to negotiate steep discounts, but many middle-income countries will find this promising intervention cost-prohibitive.

Value of Temozolomide for Elderly Patients with Glioblastoma

Adding temozolomide (Temodar) to radiation therapy in elderly patients (aged ≥65 years) with glioblastoma has an ASCO value score of 43 (moderately high); an ESMO value score of grade 3; and based on a projected cost exceeding $100,000 per QALY gained, an intermediate value for ICER.

Because physicians have been able to use this generic drug for some time, the value of temozolomide for glioblastoma in elderly patients is viewed as moderate from a health system perspective, Dr Schrag reported. It has low value in the absence of a specific mutation.

The majority of high-income countries will likely continue to cover this agent, and many will adopt it if they have not already done so, said Dr Schrag, but middle-income countries will vary greatly, with some finding it cost-prohibitive.

Value of Extended Letrozole Therapy in Breast Cancer

An additional 5 years of letrozole (Femara) therapy versus stopping letrozole therapy after the first 5 years in patients with estrogen receptor (ER)-positive breast cancer has an ASCO value score of 8, which has low value, and an ESMO value score of grade B. The projected cost per QALY was not available, because no survival benefit has been shown with extended letrozole therapy.

Dr Schrag emphasized that patients with ER-positive breast cancer who received an additional 5 years of letrozole therapy will be able to make informed decisions that are consistent with their own preferences, toxicities, and side effects.

“Individual value will thus be highly variable, and this will be a preference-sensitive decision based on risk and treatment tolerance,” she said.

Dr Schrag underscored the high risk for osteoporosis associated with extended aromatase inhibitor therapy (ie, letrozole), and the excess costs of treating it. “From a health system value perspective, this intervention is rated as low in each of the value frameworks. It would be a priority to focus on adherence to the first 5 years of aromatase inhibitor therapy,” she said.

A Provocative Beginning

Ultimately, said Dr Schrag, oncology value frameworks are not a solution, but a “provocative beginning” to help providers think more systematically about how to incorporate value in the setting of the ever-increasing costs of cancer therapy.

“These tools are not yet practical for clinical decision-making at the bedside,” said Dr Schrag, noting that improved software will be necessary to make this useful for patient–physician communication.

“That said, we cannot ignore value deliberations. The unaffordability of cancer treatment has adverse consequences on patients, on providers, and on society as a whole….It is critical that we work together to develop better policies to align care value with our core values,” she concluded.