Resolving Tension Between Cost-Effectiveness Analysis And Patient Centricity – Forbes

BOSTON, MA - JUNE 6: Steven Pearson, President of the Institute for Clinical and Economic Review ... [+] (ICER), talks with chief medical officer David Rind during a meeting at their offices in Boston on June 6, 2019. (Photo by Craig F. Walker/The Boston Globe via Getty Images)

There appears to be an inherent tension between cost-effectiveness analysis that informs allocative efficiency at the population level, and patient centricity. Here, patient centricity can be defined as incorporation of patient-centric outcomes in value calculations and inclusion of patient input in clinical and economic decisions.

Ideally, precision medicine resolves this tension, as by definition its patient-centric, and when done right, cost-effective. But, theres still a long way to go before precision medicines promise becomes widespread reality.

Through intermediaries, such as payers, cost-effectiveness analysis informs treatment choices made by physicians and patients. For patients and their healthcare providers, issues can arise when reimbursement protocols (e.g., formularies) derived from population-based cost-effectiveness analysis impact (often limit) the choices patients and physicians can make.

Evidence-based value frameworks serve to inform healthcare decision-making in healthcare systems. Sometimes payers employ their own in-house value frameworks. Otherwise, they outsource to entities, such as the Institute for Clinical and Economic Review (ICER), that deploy uniquely crafted value frameworks. The assessment of the value of medical technologies is based on attributes, such as cost, quality of life, life expectancy, toxicity, and side effects. Rising healthcare costs have been a driving force behind the emergence of a host of value frameworks aimed at both defining and quantifying what value means for healthcare.

To work towards a patient-centric and sustainable path for the healthcare system as a whole or one component of it, value frameworks can create patient-outcome adjusted cost-effectiveness models. These account for patient preferences, but also disease severity, burden, among other factors, while addressing distributional issues.

ICER, for example, has included contextual considerations which encompass ethical, legal, or other issues that influence the relative priority of illnesses and healthcare interventions. Presumably, this includes among other things disease severity and burden, rarity of the condition, and lack of treatment alternatives.

ICER has also supplemented the criteria it considers with what it calls other benefits and disadvantages. These are benefits or disadvantages offered by an intervention to the individual patient that may not have been initially considered. An example is consideration of a new formulation or route of administration for an existing drug, which may result in improved patient adherence.

Additionally, ICER is convening three independent evidence review groups to vote on the strength of evidence with respect to the effectiveness of new drugs, devices, and delivery systems.

Changes in ICERs methodology are described in Value Assessment Framework for 2020, which the institute claims will establish a more patient-centered approach to value assessment.

Two things stand out. First, ICER is committed to an increased use of real-world evidence in its reviews. This kind of data will provide a more comprehensive view of how a treatment affects patients in their everyday life. Second, ICERs open comment period is increasingly eliciting and acknowledging more patient input. Specifically, ICER says that "each report is developed with input from multiple sources, including patient advocacy groups, clinical experts, and manufacturers."

What I believe is inconsistent, however, with a genuinely patient-centered approach is that ICER is not looking specifically for members who have clinical expertise in topic areas that will be studied in upcoming reports. Ostensibly, this is done to avoid subjectivity and promote objectivity. Well, sure. But, the reports refer to specific diseases and conditions, and treatments for these diseases and conditions. These must be evaluated by experts in the field as well as patient representatives to gauge their effectiveness profile, safety, burden, as well as healthcare provider and patient preferences.

To exclude disease-specific experts, patients, and caregivers from voting panels doesn't make sense. In the justice system, its essential that judges are impartial. But, this is not a court or judicial review. Moreover, the decisions rendered impact specific subgroups of patients and their healthcare providers, who are optimally suited to do the judging. I believe health technology committees need to know what actual patients, doctors, and caregivers, in a specific disease area care about, and not simply see them as an input into the value assessment process. Of course, healthcare providers, patient (representatives), and caregivers from the impacted disease area wouldnt be the only members with voting rights. Other constituents - payer policymakers, regulators, health economists, others - deserve a seat at the voting panel table. It may turn out that the patients, healthcare providers, and caregivers are voted down. But at least they would feel represented in a material sense.

Ultimately, organizations that have well-established value frameworks, such as ICER, must resolve the difficult dilemma of reconciling the tension between cost-effectiveness analysis and patient centricity. In order to best address this, its imperative to meaningfully engage with patients (representatives) at all stages of health technology assessment, in addition to clinical experts and caregivers in the specific treatment area under consideration.

For this purpose, policymakers must do more to construct patient-centric measures of the benefits conferred by medical technologies, and permit the patient's voice to be heard more incisively, both on the input side regarding value assessment, and decision-making side.

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Resolving Tension Between Cost-Effectiveness Analysis And Patient Centricity - Forbes