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Patient-Reported Outcomes: Emerging Developments and Innovative Approaches
Analysis Group led a symposium on patient-reported outcomes (PROs) at the ISPOR 22nd Annual International Meeting.
Many assessment criteria are used by the US Food and Drug Administration (FDA) to help determine the efficacy and safety of a pharmaceutical product. One area that is receiving increased attention from stakeholders across the health care industry relates to PROs, a core set of measures for clinical outcome assessments (COAs) valued by the FDA to support patient-focused care. PROs can assess whether a drug provides benefits by measuring the impact of the treatment on a patient’s symptoms, mental state, or function.
Treatment benefits captured by valid PRO measures have been used to support medical product labeling claims approved by the FDA and/or the European Medicines Agency. As an extension of PROs, the health utilities generated from PRO measures are a critical component for informing decision making in the context of a Health Technology Assessment (HTA), a systematic evaluation of the properties, effects, and impacts of health technologies (i.e., medical interventions).
John E. Ware, Professor, University of Massachusetts Medical School
Given the rising importance of health economic and outcomes evaluations of medical interventions, the need for short, valid, sensitive, and reproducible PRO tools to capture the benefits of treatments has never been greater. During a symposium at the ISPOR 22nd Annual International Meeting, Managing Principal Eric Wu discussed new developments in PROs and HTAs with two leading academics in the field: John E. Ware, professor and chief of outcomes measurement science in the Department of Quantitative Health Sciences at the University of Massachusetts Medical School, and an Analysis Group affiliate; and John E. Brazier, professor of health economics and dean of the School of Health and Related Research at The University of Sheffield, UK. Both are internationally recognized leaders who have established expertise in developing, standardizing, and applying health metrics to assess PROs. The topics discussed during the symposium addressed the growing need for reliable, valid, and responsive health measurement tools that can be readily integrated into the planning and design phase of clinical trials and HTA submissions. Analysis Group Managing Principal Mei Sheng Duh moderated the symposium.
The aging population and rising prevalence of chronic conditions around the world are creating demand for ways to quantify not only patient health, but also other benefits that a patient receives (e.g., dignity, autonomy, sense of belonging). Such measurement tools will lead to better, more accurate, and more sensitive empirical data; will support the claim of a medical product being safe and efficacious; and will provide meaningful benefits to patients for improved health-related quality of life (HRQL).
John E. Brazier, Professor of Health Economics, The University of Sheffield
Professor Ware discussed the foundation of conceptualizing and quantifying HRQL, and emphasized the essential domains of health along with the advances in the standardization of disease-specific PRO measures across health conditions. Professor Brazier then described parallel developments in the utility field, such as condition-specific preference-based measures and how they compare to generic utility measures; extending generic utility measures to incorporate higher levels of functional health and well-being; and calibrating different measures through linking and mapping.
Dr. Wu shared the common challenges encountered in outcomes evaluation in the context of HTAs and discussed the practical approaches for utility generation, including new developments in utility estimation to support HTA submissions.
During his remarks, Dr. Wu noted that HTA agencies generally recommend using utility values directly from clinical trials. However, this is not always available or feasible. Some common challenges include insensitive utility measures, lack of information due to rarity of a disease, underestimation of utility impacts because of the nature of disease progression, and limited duration of a clinical trial. Therefore, careful and strategic considerations should be given for utility assessment during clinical trial planning. Several case studies presented by Dr. Wu addressed the potential approaches to apply for different scenarios in HTA submissions.
Mei Sheng Duh, Managing Principal
As the symposium participants discussed, the recent advances in psychometric and utility methods appear to be increasingly gaining adoption, which will continue to help alleviate patient burden and introduce greater efficiencies. These are critical aspects when it comes to demonstrating product value in health economic and outcomes evaluations. ■
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