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The Need For A New Patient-Centered Decision Tool For Value-Based Treatment Choices In Oncology

Over the past decade, breakthroughs in oncology have improved overall and progression-free survival for cancer patients. The rapid pace of drug development and approval has made available multiple therapeutic options for a wide range of cancers. In some cases, these options differ on attributes other than efficacy, such as route of administration (for example, oral versus infusion) or type of side effects. At the same time, the high costs of these novel anticancer medications have placed considerable financial pressure on both the US health care system and—with the continuing insurance trends toward high deductibles and coinsurance levels—on patients.

Recognizing that costs and value should be important considerations in patients’ and providers’ cancer treatment discussions, US clinical organizations such as the American Society of Clinical Oncology (ASCO) and the National Comprehensive Cancer Network (NCCN) have developed value frameworks. Unlike other value framework initiatives, these tools are not geared toward supporting payer coverage decisions. Instead, they are intended to help guide shared patient/physician decision making for cancer treatment choices, presumably among the clinical alternatives approved for coverage by insurers. While such efforts to integrate cost and value into treatment planning are laudable, these patient-level frameworks have attracted a variety of criticisms. Some have identified technical issues with the metrics used to define clinical benefit and toxicity. Others have raised concerns over the (lack of) reliability and consistency in the decisions that result from the use of these frameworks. From our perspective, however, the most significant problem with these two cancer value frameworks is that they are insufficiently patient-centered.

Need For A More Patient-Centered Cancer Value Framework

Patients, clinicians, advocates, and policy makers are increasingly recognizing and calling for patient-centered value assessments. It is critical that frameworks used to influence treatment decisions accommodate heterogeneity in patients’ clinical characteristics and personal preferences. Given the highly individualized nature of decisions involving life-threatening conditions such as cancer, it is imperative that cancer value frameworks accommodate individual patient preferences. They must be comprehensive in capturing specific treatment attributes and must also accommodate the relative priorities (or weights) assigned to those attributes.

Both the ASCO and NCCN initiatives have been faulted for failing to reflect the full range of perspectives and priorities of patients and their families. The ASCO framework generates a net health benefit score based on efficacy (overall survival, progression-free survival, disease-free survival, or response rate) and safety (toxicity), including bonus points for tail-of-the-curve survival benefits, palliation of cancer symptoms, quality of life, and treatment-free interval. The overall net health benefit score and drug cost (drug acquisition costs and patient out-of-pocket expenses only) are then used to inform the value of the cancer treatment choices. The NCCN framework rates five components—treatment efficacy, safety, quality and consistency of evidence (two), and affordability of the overall treatment (including other therapy costs such as hospitalization, supportive care, and administration).

While these are all important considerations when choosing treatments, they do not necessarily encompass all of the factors that are relevant to patient decision making. For instance, travel required for an infusion (versus an oral) anticancer treatment may be a critical factor in choosing a treatment for some patients. Patients may have transportation challenges or may cringe at the idea of their family member having to take time off from work to drive them for the weekly infusions. Furthermore, criteria in the framework such as toxicity may lack the specificity needed for patient decision making. Patients may be more concerned by a specific type of toxicity than aggregate toxicity grades associated with the treatments. For example, a musician may deem loss of the ability to play an instrument due to neuropathy worse than a loss of survival probability but may be less concerned about other side effects. Similarly, a single earner in the family may be especially concerned about fatigue and other side effects affecting his or her ability to work and maintain his or her job and health insurance. A value framework that is truly patient-centric will acknowledge these tradeoffs.

The ad hoc approaches to scoring in both frameworks also limit their relevance to real-world patient decision making. In the ASCO framework, a therapy can achieve a maximum of 130 points based on its clinical benefit (80 points), toxicity (20 points), palliation (10 points), and increased time off treatment (20 points). However, does such an arbitrary scoring system necessarily represent how an individual patient may weight these different attributes given their preferences and circumstances? For instance, one elderly patient may place a high priority on extending survival at the expense of toxicities and quality of life to attend his granddaughter’s pending wedding, whereas another elderly patient may be less concerned about survival and more concerned about the quality of her remaining months of life.

While the NCCN framework does not synthesize values across different domains to arrive at a single score, it uses an expert panel of oncologists to rate the five domains (efficacy, affordability, and so forth) using a standardized scale from 1 to 5 (where 1 is the least favorable and 5 is the most favorable). It then creates a visual plot of the scores using a 5-by-5 matrix. The NCCN framework also struggles to deliver a meaningful assessment of value from the patient perspective, especially because oncologists, instead of patients, rate the affordability of cancer treatment.

Designing A Patient-Centered Decision Tool For Value-Based Treatment Choices

So how do we develop and implement a patient-centered decision tool for value-based treatment choices that truly meets the goals and priorities of cancer patients in daily clinical practice? The Patient-Perspective Value Framework, recently developed by Avalere/FasterCures, is an important first attempt to capture a more comprehensive set of factors that are relevant and important to patients. However, it is a generic framework in the early stages of development and is not specifically tailored for shared decision making in cancer.

Recent findings from initiatives led by CancerCare (where one author, Ellen Miller Sonet, serves as chief strategy and alliance officer) provide several additional insights into the needs and challenges to be addressed in developing an effective tool for shared decision making in cancer treatment planning. Focus groups conducted as part of CancerCare’s 2017 Patient Values Initiative—a project that seeks to reshape the value debate by exploring what is important to patients and families in treatment decision making—revealed that newly diagnosed patients are overwhelmed by treatment planning discussions and can rarely raise their questions, concerns, or priorities in the first few meetings they have with their providers. As a first step, resources are needed to help patients articulate their concerns and to help providers solicit and understand patient priorities and preferences during treatment planning. One approach may be to modify intake forms to include questions about work life, household responsibilities, favorite activities, pending events (such as weddings), and personal goals. However, the focus groups also found that patients’ priorities and perceptions of their treatment change over time and that the “personal calculus” used to arrive at treatment decisions is constantly evolving: Family circumstances change, side effects wax and wane, and costs of care become more burdensome. Hence, revisiting patient priorities and preferences over the course of treatment and disease progression is also important.

Real-time measurement of priorities and preferences remains problematic. A CancerCare initiative is developing and testing simple turnkey tools that can be used in clinics to capture and communicate such information on patients’ priorities and preferences. Another initiative led by Carevive Systems uses technology to facilitate the incorporation of cancer patients’ values, preferences, and goals into treatment decision making. This remains an important area of development and testing to support evidence-based approaches to capturing this information.

The next challenge to be addressed by a customized patient-centered decision tool is that patients need not equally prioritize (weight) all of the factors that are important to them in arriving at a treatment decision. Approaches for incorporating this lack of equality into a patient-centered decision-making tool may include assignment of patient rankings to these factors (importance buckets) or more formal rating exercises. The latter could be accomplished through the use of multiple criteria decision analysis (MCDA). MCDA provides a way to make complex decisions, such as those faced by cancer patients who are often presented with several treatment options that involve tradeoffs. While MCDA methods need further refinement, they hold promise in helping cancer patients make personalized treatment choices.

In summary, continued innovations in oncology ensure survival improvements and even cures. However, these new treatments bring an array of challenges for patients, such as higher out-of-pocket expenses and new toxicities. Hence, there is an urgent need for a methodologically rigorous approach to incorporating patient-centered value elements to inform shared patient/physician decision making regarding various cancer treatment alternatives. The ultimate goal should be to create cancer treatment decision tools that generate a tailored output based on clinical evidence as well as patient priorities.

Authors’ Note

The writing of this blog post was supported by funding from the Pharmaceutical Research and Manufacturers of America. The funding source had no role in developing the contents of the article or approving the final blog post for publication. Jalpa A. Doshi, PhD, has received research funding and served as a consultant to various biomedical companies and payers.

This post originally appeared in the Health Affairs blog.