Health technology assessment (HTA) entities adopt different value frameworks as a means of assessing the benefits and costs of (new) medical technologies. Benefit attributes include reduction or amelioration of symptoms, quality of life, life expectancy, and improvement of functioning and productivity, discounted for possible toxicity and side effects related to the use of medical technologies. In this context, HTA value frameworks are increasingly incorporating the patient’s voice as input in the value assessment of medical technologies, but not necessarily giving patients a say in decision-making regarding prescribing and coverage recommendations.
In the U.S., patients spend an increasing amount out-of-pocket on prescription drugs, and specialty pharmaceuticals in particular. This is due to rising deductibles and co-insurance, which is calculated as a percentage of ever-climbing list prices. Despite this, patients play a very limited role in the design of specialty drug coverage policies.
Both the British National Institute for Health and Care Excellence (NICE) and the American Institute for Clinical and Economic Review (ICER) employ a specific form cost-effectiveness analysis; the cost-per-Quality-Adjusted Life Year (QALY). In the case of NICE, cost-per-QALY findings inform prescribing and reimbursement recommendations. The QALY’s distinct appeal is its ability to combine morbidity (represented by the quality of life adjustment factor) and mortality into one figure, which allows for comparisons across different diseases.
But, there is an inherent tension between cost-effectiveness analysis that informs prescribing as well as pricing and reimbursement decisions related to allocative efficiency at the population level, and actual treatment decisions made by physicians and patients. Friction occurs when prescribing recommendations or reimbursement protocols – in the form of formulary restrictions, for example – based on cost-effectiveness analysis, impacts (limits) individual decisions taken by patients and physicians.
Furthermore, a common criticism of the cost-per-QALY method is that it maximizes benefits subject to a budget constraint, across the entire population without regard to the distribution of the benefits. The mantra “a QALY is a QALY is a QALY” implies that it doesn’t matter to whom QALYs accrue. On one hand, this is considered egalitarian. On the other, it doesn’t necessarily account for issues such as initial health status, disease severity, or lack of treatment alternatives.
Important changes have been underway in two of the globe’s most influential HTAs. Britain’s NICE is considering equity, specifically disease severity, in its appraisal process. Accounting for disease severity introduces a fairness dimension into the allocation calculus. NICE also points to the importance of shared decision-making between patients and medical professionals for “preference-sensitive decisions.” But, the shared decision-making NICE envisions doesn’t involve handing decision making to the patient. In fact, NICE circumscribes patient decisions by narrowing choices to the “selected range of options recommended by NICE guidance.”
The U.S.-based ICER has responded to criticism that it is not sufficiently patient-centered by adding to the value criteria to be considered, including what it calls “other benefits and disadvantages.” Other benefits or disadvantages are items conferred by a medical technology to the individual patient, caregivers, or the delivery system that would not have been initially considered as part of the evidence package on comparative clinical effectiveness. To illustrate, these may include new methods of formulation or routes of administration that improve patient adherence. The ease of treatment (for example, whether a patient must spend several hours on an intravenous infusion, or can instead take an oral tablet), constitutes an important aspect of value, from both a patient and health system perspective.
ICER is also supplementing contextual considerations which include ethical, legal, or other issues that influence the relative priority of diseases and medical technologies. For instance, similar to NICE, ICER says it is considering the severity associated with a disease or condition, or the lack of existing treatment alternatives.
Also, HTA agencies worldwide – from Australia to Canada – have begun utilizing patient-reported outcome (PRO) instruments and inclusion of the patient voice when evaluating medical technologies. Broadly, these agencies are adopting practices that were already being implemented by regulatory entities, such as the Food and Drug Administration (FDA). The FDA will soon be expanding the role of what it calls “patient focused drug development” to include patient involvement in clinical study design and execution in new lines of products, such as cell-mediated gene therapy.
However, among HTAs and payers, inclusion of the patient voice is not universal. The world’s best known HTA, the British NICE suggests that PRO data aren’t used as part of their technology appraisals; rather, NICE may utilize PRO data as a surrogate endpoint for other parts of its evaluations (for example, as part of a cost-effectiveness calculation).
Changes in ICER’s methodology are described in the Value Assessment Framework for 2020, which the organization claims establish a more patient-centered approach to value assessment.
ICER also says it is committed to an increased use of real-world evidence in its reviews of medical technologies. This kind of data will provide a more comprehensive view of how a treatment affects patients in their everyday lives. Further, ICER’s 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.” Third, 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.
In selecting participants in the evidence review groups, ICER states it 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.
It would seem, however, that the disease areas and medical technologies included in the reports must at least in part be evaluated by experts in the field as well as patient representatives to properly gauge effectiveness, safety, adverse effects, and other disease-specific factors that help shape the medical technology’s comprehensive profile.
Also, to exclude disease-specific experts, patients, and caregivers from voting panels may not make sense. Policymakers need to find out what actual patients, doctors, and caregivers, in a specific disease area care about, that is, what matters to them.
It’s encouraging that NICE, ICER, and other HTA entities are aiming to engage with stakeholders to pursue a shared goal of patient-centric care. But, there’s still more work to be done.
As HTAs evolve, they will seek additional patient-centric measures of the benefits conferred by medical technologies. Further, it’s expected that their value frameworks will permit the patient’s voice to be heard more incisively, both as an input for value assessment, and as part of the decision-making process regarding prescribing and coverage recommendations.