Aspects of health important to patients are difficult to validate and difficult to quantitate: one person’s “I feel terrible” could be another person’s “I am OK;” one person’s “I can’t do anything” could be another’s “I have a few things I can’t do any more.” Nevertheless, the essential focus for physicians should be that which is important to patients, specifically how they feel and how they function. This goal—putting patients’ hopes and concerns first—demands eliciting from individual patients the nature of their hopes and fears. This is difficult to do. Understanding patient’s preferences demands knowledge, practice, social skills, a desire to listen, and empathy. And yet, relevant quantitation is essential to comprehending how patients feel, what outcomes are important to them, and how to address these issues in ways that are easily understood.
In recent years, a variety of methods have been designed with the goal of learning more about (1) what is important to patients and (2) how to learn what is important to patients. There are now methods to assess quantitatively how patients feel and function. These include the National Eye Institute 25-Item Visual Function Questionnaire, which assesses feelings and health-related quality of life; other questionnaires, such as the Glaucoma Symptoms Scale, evaluate visual and ocular symptoms and how bothersome they are to patients. Performance-based measurements of function include—among others—reading speed, driving simulation, and comprehensive tests such as the Assessment of Disability Related to Vision. Unlike visual acuity and visual fields, these are direct evaluations of actual ability to perform the activities of daily living.
Physicians tend to use information they believe will be most likely to help them care for patients. They want “hard” information that can be easily measured to help them achieve this goal. Ophthalmologists, for example, often use information such as intraocular pressure (IOP), age, central corneal thickness, visual acuity, and visual fields to make treatment decisions, believing them most likely to lead to decisions beneficial to patients; they rely on these measures as surrogates for feeling and function. However, relationships between quality of life/ability to perform the activities of daily living and standard markers such as IOP and central corneal thickness can often be poor. For other surrogates, such as cup-to-disc ratio, relationships exist, but they are difficult or impossible to establish. For still others, such as the visual field, the large intertest variabilities require long intervals of time to obtain enough useful information to arrive at a valid conclusion. When discussing treatment options such as surgery with patients, doctors generally make a sincere effort to understand what they believe to be of most importance to each individual patient. However, how well the surgeon’s understanding accurately reflects what is actually of highest priority to the patient is not clearly known and has not been intensively studied. At present, surgical studies, for example, focus by and large on surrogates such as IOP, visual fields, or rates of complications, and rarely consider direct measures of cost, convenience, quality of life, days of disability, or ability to function.
The United States Food and Drug Administration (FDA) Center for Devices and Radiological Health has noted that devices are often not evaluated from the patient’s point of view or in ways that take individual patient preferences into account. Through a number of initiatives, the FDA has committed to measuring and incorporating the patient’s perspective in the evaluation of medical products. To assess existing measures and to develop better measures for evaluating patient-focused outcomes, the FDA has co-developed projects with the American Glaucoma Society (AGS) and two Centers of Excellence in Regulatory Science and Innovation (CERSI http://www.fda.gov/ScienceResearch/SpecialTopics/RegulatoryScience/ucm301667.htm ) based at the Johns Hopkins University and the University of California San Francisco (UCSF)/Stanford University. The researchers selected Minimally Invasive Glaucoma Surgical (MIGS) devices as their focus. This topic seems an excellent choice, as these surgeries are in the midst of rapid development and are of great interest to ophthalmologists, patients, and industry.
Earlier this year, researchers from the UCSF/Stanford CERSI collaborated with the RAND Corporation in Santa Monica, the FDA, and the AGS to convene a physician focus group, and have planned 3 patient focus groups at different sites within the United States to accumulate the background information necessary to develop a web-based questionnaire. This questionnaire will serve as a publicly available patient-reported outcome tool that can be used in future submissions to regulatory agencies designed to assess glaucoma surgical devices. Although this is currently a U.S. initiative, European glaucoma colleagues have expressed great interest in the development of such a tool and trans-Atlantic collaboration for similar future initiatives has been explored. In parallel, the Johns Hopkins CERSI is focusing on developing tools to assess patient preferences for patients with mild to moderate glaucomatous diseases who are candidates for MIGS procedures. The 2 CERSI centers, RAND Corporation, FDA, and AGS communicate at least quarterly via teleconference to provide updates to all stakeholders in this FDA-funded initiative. The tools developed from this project will undoubtedly have relevance beyond MIGS devices.
The AGS is pleased to be part of this initiative, which will provide an opportunity for many members of the society to be involved in dialogue that will establish tools for assessing the patient’s perspective, encourage implementation of these tools in clinical trials, and facilitate the incorporation of this information in discussions with glaucoma patients. This is an important and exciting project that may have valuable conclusions, not only for patients with glaucoma and those who care for them, but for persons with all matter of maladies.
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