Health Policy$ense

Value Frameworks for Cancer Drugs, US Style

Bringing transparency to clinical and coverage decisions

Developing a value framework for cancer drugs can sound like an arcane exercise without much relevance to clinical care. Restate it as a question of how, and how much, to pay for cancer drugs, and you’ve got everyone’s attention. Current and potential value frameworks are the subject of a special issue of Value in Health and ongoing examination by the Gant Precision Cancer Medicine Consortium at the University of Pennsylvania.

Last month, the group reviewed international frameworks, particularly those of Germany, the UK, and Canada, and the regulatory levers used to assess and pay for value. This month they reviewed relevant US frameworks developed by the National Comprehensive Cancer Network (NCCN), the American Society of Clinical Oncology (ASCO), and the Institute for Clinical and Economic Review (ICER).

These frameworks differ from the international ones, and from each other, in their goals and audience. NCCN and ASCO are designed to help providers and patients make informed choices; ICER’s framework (more along the lines of international frameworks) is designed to inform population decisions, such as pricing, payment, and coverage, rather than individual clinical decisions. This difference lies at the heart of how each organization assesses and presents its framework.

The NCCN uses “Evidence Blocks™” to visually represent value along five dimensions, each scored on a scale of 1-5 (higher being better):

NCCN Guidelines panel members score each dimension using their knowledge of published data as well as their clinical experiences. Affordability is based on an estimate of the total costs of the therapy, rather than on direct costs to the patient. The goal is to provide an easy way for patients and providers to compare the value of possible therapies.

Similarly, ASCO’s value framework is designed to make it easy to compare therapeutic alternatives, for treatments that have been studied head-to-head in prospective randomized clinical trials. Unlike the visual representation of evidence in the NCCN, ASCO creates a “net health benefit (NHB)” score, a weighted measure of a treatment’s benefits and side effects. The NHB is composed of the incremental health benefits, minus incremental toxicities, with bonus points awarded for palliation of symptoms, quality of life, treatment-free survival, and potential for long-term survival.  The NHB is presented in the context of the drug’s acquisition cost (curiously, not its incremental cost, despite the fact that the NHB is incremental rather than absolute) and patient co-payment (although how that is to be determined is not known).

In contrast, ICER reports take a population-based perspective, and do not purport to be a shared decisionmaking tool. ICER assesses value in terms of comparative clinical effectiveness, incremental dollars per quality-adjusted life year (QALY), and a value-based price benchmark (usually at willingness-to-pay thresholds of $50,000 to $150,000 per QALY). It also assesses the affordability of a new treatment by evaluating its 5-year impact on a large health system budget.

You might wonder whether these frameworks actually provide reliable and valid measures of value. In a recent pilot study, eight panelists applied these frameworks to five advanced lung cancer drugs to assess the rankings across frameworks and the degree to which they provide stable and consistent results (excluding measures of affordability). Despite their different measures and scales, the frameworks ranked the five drugs similarly, with stronger agreement between ASCO and NCCN than ICER and NCCN. Inter-rater reliability for each drug was highest for ASCO, and lowest for ICER, which has a much broader range of drugs it evaluates. The study suggests that the frameworks are measuring a similar concept, despite their different inputs and scales.

So how are these frameworks used, and can they help us achieve and sustain access to high value cancer care for all patients? One consortium member pointed out that although the frameworks are relatively new, the decisions about what drugs to use and what drugs to pay for were being made in exam rooms and boardrooms anyway. The impetus for these frameworks, he noted, was to bring transparency and structure to these decisions that can be otherwise idiosyncratic and opaque.

As of now, the NCCN and ASCO frameworks are not commonly used in the clinical encounter, although ASCO plans to develop a software tool for that purpose. Thus far, the primary users of the ICER framework have been payers, who have worked it into their coverage decision processes. A number of consortium members voiced concerns that payers were using the framework to deny coverage, and that too much emphasis on the short-term budget impact would lead to the wrong discussions that focus only on achieving a lower price. Patients worry that cost-effectiveness will override all other imperatives, and that patient-reported outcomes such as returning to work may not carry enough weight in the process.

Other members of the group pointed out that the goal was not to deny coverage but to support innovative ways to pay for high-value drugs, acknowledging the tension that often exists between long-term value and short-term financial considerations. For example, the frameworks can support outcomes-based risk agreements, where drug manufacturers are at financial risk for achieving certain outcomes.

One member noted that value frameworks used by third-party payers in coverage decisions and those used by clinicians and patients in clinical decisionmaking can, and should, work in concert with one another. In a broader sense, the coverage decision should lay out the sustainable treatment options available to the patient and clinician. Without the coverage decisions, in this view, “you run out of options because you run out of money.”

One key question, which the Gant Precision Cancer Medicine Consortium will continue to consider, is whether and how the cumulative decisions of third-party payers in our system can produce the desired end, which is access to high-value care for all patients in an era of increasingly expensive precision cancer drugs.

The Penn Precision Cancer Medicine Consortium is a multidisciplinary group of more than 20 experts and stakeholders that has come together at Penn to develop a new framework for the economic sustainability of precision cancer medicine. Through multiple discussions culminating in a conference in May 2017, the group will tackle the hard questions that precision medicine raises for patients, providers, and payers.

The Consortium is made possible through a philanthropic gift to the University of Pennsylvania by Donald R. Gant, Wharton '52 and the Gant Family. It is led by LDI Senior Fellows Justin Bekelman and Steven Joffe. Other members of the Consortium and their backgrounds are here.