Advocating for New Patient-Centered Tools for Value-Based Treatment Choices in Oncology

Abstract: The cost of medical drugs continues to increase, and costs of novel therapies for oncology are particularly expensive—for patients as well as health systems in general. Many health care stakeholders are concerned about financial toxicity for patients and how to ensure access to innovative therapies. There is a large and growing consensus that costs and value should be important considerations in patients’ and providers’ cancer treatment discussions. Many leading organizations have created value frameworks to help assess treatment options and their relative value. The American Society for Clinical Oncology and the National Comprehensive Cancer Network have each created their own value frameworks, but many think that patient preferences are not adequately integrated into these frameworks and that the metrics they employ do not produce reliable information. We provide a brief assessment of these frameworks and how they further need to evolve to meet the needs of patients in a health system shifting to value-based care.

The past decade has witnessed remarkable breakthroughs in oncology that have improved survival outcomes for patients with cancer. The unprecedented speed of drug development and approval has made available multiple therapeutic options for a wide range of cancers. However, the high costs of many of these novel anticancer medications have placed considerable financial pressure on both the US health care system and—with growing cost shifting from payers through high deductibles and coinsurance levels—on patients.^1,2 

Faced with a growing consensus that costs and value should be important considerations in patients’ and providers’ cancer treatment discussions, several clinical organizations such as the American Society of Clinical Oncology (ASCO) and the National Comprehensive Cancer Network (NCCN) have developed value frameworks.^3,4 These frameworks differ from existing efforts (such as the Institute for Clinical and Economic Review [ICER]’s value assessment framework,⁵ Memorial Sloan Kettering’s Drug Abacus⁶ and ESMO [European Society for Medical Oncology]’s magnitude of clinical benefit scale [MCBS])⁷ as they are resources intended to help guide shared patient/physician decision-making for cancer treatment choices, presumably among the clinical alternatives approved for coverage by insurers.⁸ While these value frameworks represent an important first step in integrating costs and value into treatment planning, these patient-level frameworks have been criticized because of the metrics used to define clinical benefit and toxicity as well as concerns over the lack of reliability and consistency in the resulting decisions. However, the most significant problem with these cancer value frameworks is that they fail to consider what is most important to the individual patient.

Building upon our previous work,⁹ in this article we review current ASCO and NCCN value frameworks and discuss how they still need to evolve to meet the needs of patients and the shift to value-based care delivery and reimbursement.

Reviewing the Current Cancer Value Frameworks as Shared Decision-Making Tools

Patients, clinicians, advocates, and policymakers are increasingly calling for patient-centered value assessments. Frameworks utilized to influence treatment decisions must accommodate heterogeneity in patients’ clinical characteristics as well as their personal preferences. Given the highly personal 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 toxicities and must also accommodate the relative importance assigned to those attributes. Both the ASCO and NCCN frameworks have been faulted for failing to reflect the perspectives and priorities of patients and their families.¹⁰ In both cases, the stated intention is for the frameworks to serve as a starting point for a physician-patient discussion. The absence of prompts that address patient-specific preferences and priorities, however, suggests these topics are not likely to be discussed. 

ASCO Value Framework

The ASCO framework is intended to be used as a shared decision tool that allows physicians and patients to compare treatment options. The analysis 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 related to the regimen) are then used to inform the value of the cancer treatment choices. The efficacy score is based only on the results of randomized controlled trials (RCTs), while safety is determined by the RCTs’ findings as to frequency and grade of side effects. 

NCCN Value Framework

The NCCN framework rates 5 components: treatment efficacy, safety, quality and quantity of evidence, and affordability of the overall treatment (including other therapy costs such as hospitalization, supportive care, and administration). The evaluations are driven by consensus on the basis of published data, panel members’ clinical experience, and case reports. Safety is rated on the likelihood of side effects from an intervention, with fewer side effects being rated more highly.

Where the Frameworks Fall Short

Components

These frameworks do not incorporate real-world evidence, patient-reported outcomes, or consider sub-population analyses. More importantly, from the patient perspective, there is no consideration for factors that reflect patients’ priorities and preferences. For instance, travel required for an infusion (vs an oral) anticancer treatment may be a critical factor in choosing between treatments for some patients. Patients may have transportation challenges or may not have care partners who can 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. 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. Similarly, a dentist may want to avoid any risk of peripheral neuropathy. A value framework that is truly patient-centric needs to acknowledge these tradeoffs and consider quality-of-life priorities in the context of each patient’s personal circumstances.

Scoring

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, toxicity, palliation, and increased time off treatment. However, does this scoring system necessarily represent how an individual patient may weigh these different attributes, given their individual preferences and circumstances?

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 5 domains: efficacy, safety, quality of evidence, consistency of evidence, and affordability. The framework uses 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 struggles, however, to deliver a meaningful assessment of value from the patient perspective, especially because oncologists—rather than individual patients—rate the affordability of cancer treatment. The NCCN considers the overall cost of an intervention, including the drug, infusions, supportive care, toxicity monitoring and management, and the probability of care being delivered in the hospital. However, the extent to which the oncologists are truly informed regarding these costs is debatable.¹¹

Creating a More Patient-Centered Decision Support Tool

Recent findings 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 including 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.^12,13

Enhancing Provider-Patient Communications

As a first step, resources are needed to help patients articulate their concerns and to help providers, both in training and in practice, 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, family celebrations, or planned travel), 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 over time. Hence, revisiting patient priorities and preferences over the course of treatment and disease progression is also important, particularly at points of transition in the treatment regimen.

Patient Preferences in Real Time

Real-time measurement of priorities and preferences remains elusive. Patients and clinicians may have ad hoc conversations that reflect patients’ and physicians’ perspectives and values, however, there is rarely any formal process for collecting and documenting this information early in their relationship. None of the leading oncology pathways, guidelines, or electronic health records (EHRs) incorporate references to patients’ priorities or capturing data regarding what is important to them (though sometimes clinician notes are recorded in open fields). One of CancerCare’s initiatives is to develop and test simple tools that can be used in oncology clinics to capture and document such information on patients’ priorities and preferences. Quantitative research among oncology nurses, physicians, and patients has probed the extent to which each of these respondent groups perceives this information to be discussed and documented before treatment decision-making is finalized. The research also requested clinician feedback on alternative ways to ensure that these conversations occur and are saved in the patients’ EHR. Importantly, implementation of these tools must be turn-key and extremely efficient, because constraints on patients’, providers,’ and clinics’ time are a major barrier to altering clinician work flow.

Patient-Perspective Value Framework

An important proposed example of a patient-centered decision framework for value-based treatment choices is the Patient-Perspective Value Framework (PPVF), developed by Avalere/FasterCures.¹⁴ It relies on a value assessment methodology that incorporates patient preferences and can be applied to a variety of settings and used by multiple audiences. The PPVF includes 5 patient-centered domains with criteria and measures, and uses a broad (non-condition-specific) methodology for assessing value driven by patient preferences. This work attempts to capture a more comprehensive set of factors that are relevant and important to patients.¹⁵

In May 2018, Avalere announced a quantitative scoring methodology that adds further granularity to the PPVF methodology.¹⁶ The scoring approach aligns with the PPVF’s domains and weighs patient preferences separately within each of these domains to generate varying preference-weighted scores. Pilot testing with patients is planned for early 2019, and ongoing efforts are aimed at how to best apply the PPVF to real-world solutions.

Multiple Criteria Decision Analysis

The next challenge to be addressed by a customized patient-centered decision tool is that patients need not equally prioritize 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 or more formal rating exercises. The latter could be accomplished through the use of multiple criteria decision analysis (MCDA), which 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 in likely clinical or quality-of-life outcomes.¹⁷ While MCDA methods need further development and refinement to be routinely used, they hold promise in helping cancer patients make personalized treatment choices based on what is most important to them.¹⁸

Conclusion

Continued innovations in oncology hold the promise of survival improvements and, in many cases, eventual cures. However, these new treatments will introduce significant challenges for patients, such as higher out-of-pocket expenses and new toxicities. Hence, we echo our previous call for a methodologically rigorous approach to incorporating patient-centered value elements to inform shared patient/physician decision-making regarding various cancer treatment alternatives. Further, oncologists in practice and in training should be provided with educational opportunities to learn about truly patient-centered value-based treatment planning. For patients to reap the benefits of innovative cancer therapies, shared decision-making tools must be created that manage to balance clinical evidence and patient priorities without being too burdensome to effectively integrate into daily oncology practice.

References

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