The American Society of Clinical Oncology (ASCO) has published an initial version of a conceptual framework for assessing the value of new cancer treatment options based on clinical benefit, side effects and cost. The framework will serve as the basis for user-friendly, standardized tools that doctors can use with their patients to discuss the relative value of new cancer therapies compared with established treatments.
“Value and cost are among the biggest issues in healthcare today, but there are few tools to help doctors and patients objectively assess benefits, side effects and costs,” said ASCO President Julie M. Vose. “Our goal is to help oncologists and their patients weigh potential treatment options based on high-quality scientific evidence and a thoughtful assessment of each patient’s needs and goals. In publishing this initial version of the framework, just the beginning of the process, we hope to drive discussion and debate about a critically important issue.”
The ASCO Value Framework, published in the Journal of Clinical Oncology, was developed by ASCO’s Value in Cancer Care Task Force, with input from oncologists, patient advocates, representatives of the pharmaceutical and insurance industries, and others. ASCO is soliciting comments on the framework.
Cost - an Increasing Burden for Cancer Patients
ASCO is developing the framework at a time when patients are increasingly affected by the costs of cancer care. Costs have risen sharply in recent years, and cancer drugs are a significant driver of these increases. Newly approved cancer drugs now cost an average of $10,000 per month, with some exceeding $30,000 per month. Many patients are feeling the impact because they pay a significant share of drug costs through health insurance deductibles, co-payments, and other out-of-pocket expenses.
“Cancer patients are increasingly burdened by the rising costs of care,” said Lowell E. Schnipper, Chair of ASCO’s Value in Cancer Care Task Force. “Even well-insured patients are often unprepared for the high out-of-pocket cost of some cancer therapies. Too often, that leads to severe financial strain and even bankruptcy.”
Studies have also shown that some cancer patients take less medication than prescribed, or avoid filling prescriptions altogether, because of concerns about cost.
Value Framework Draws on High-Quality Evidence
The ASCO Value Framework proposes a methodology to compare the relative clinical benefits, side effects, and costs of treatment regimens that have been tested head-to-head in randomized clinical trials.
Data on the clinical benefits and side effects of each regimen are used to calculate a combined “Net Health Benefit” score, or NHB. The NHB represents the added benefit that patients can expect to receive from the new therapy, versus the current standard of care. The NHB is calculated based on improvement in overall or progression-free survival, and on the number and severity of toxicities. For patients with advanced cancer, a higher NHB is awarded for regimens that also offer relief from cancer-related symptoms or allow patients a treatment-free period.
The framework relies on high-quality scientific evidence available from randomized clinical trials, including commonly-reported data such as clinical outcomes and toxicity. Other important measures, such as quality of life and patient-reported outcomes, are not used in the framework, because they are not reported consistently enough to be reliable.
The NHB is presented alongside the patient’s expected out-of-pocket costs for the regimens being compared, as well as the overall drug acquisition cost.
“It’s critical to distinguish between value and cost,” said Dr Schnipper. “Sometimes the more valuable treatment will be the more expensive one and sometimes it won’t be. Ultimately, the definition of ‘value’ will be highly personalized for each patient, taking into account an individual’s own preferences and circumstances. For example, in the setting of advanced cancer, is length of life the most important goal or is quality of life? Is the proposed treatment affordable? That’s why we’re proposing to provide information on net health benefit and cost side-by-side.”
The framework is intended for doctors to use on an individual basis with their patients, and would not provide generalizable scores or rankings. Once the framework is finalized and adapted into a practical tool for clinical use, doctors would be able to adjust parameters based on an individual patient’s health needs, preferences, and financial situation — resulting in a personalized value assessment.
To help demonstrate the potential utility of the framework, the ASCO Task Force applied its methodology to four clinical scenarios: metastatic lung cancer; advanced multiple myeloma; metastatic prostate cancer; and adjuvant therapy for HER2-positive breast cancer. The results were striking: in some clinical scenarios, a newer, more expensive regimen had a much larger NHB than the previous standard. In other scenarios, the newer, more expensive regimen showed little or no net health benefit. These are sample scenarios only, and NHBs for the same regimen may vary in different types of cancer or treatment settings.
ASCO emphasizes that the proposed framework is intended to empower patients with clear information, and should not be used to limit patient choice. “This framework is about weighing the options, not limiting them,” said Dr Vose. “It should not be used to replace physician judgment or patient preference.”
Seeking Comment from all Stakeholders
Comments will inform the evolution of the value framework, which will be modified in response to feedback and updated as new data are developed about the utility and impact of new treatments in different clinical scenarios. Ultimately, ASCO plans to use the framework as the basis of physician-guided tools for day-to-day use in clinical settings. ASCO will move quickly to address input received, although the specific timeline for future steps is not yet determined.
