Direct to Consumer: Patient-Reported Outcomes

New clinical endpoints mean exacting messages for patients.
Jun 01, 2005

"I feel better." "I'm in less pain." "I'm thinking clearly." "Breathing is easy for me." "I'm able to complete the things I want to do."

Michael Parisi
These comments may represent clinically meaningful results for your brand—results that may not be appreciated by you or your customers. After all, the industry suffers from a general lack of understanding around quality of life (QOL) parameters, including symptom improvement, well-being, and overall satisfaction with treatment.

And with good reason. Although many debilitating chronic illnesses gradually erode patients' QOL through associated symptoms of fatigue and pain, as well as behavioral changes, these symptoms cannot be measured objectively in the same way, for example, as blood pressure or blood glucose levels. Therefore, the challenges in interpreting—and then communicating—QOL improvements are major obstacles in brand communications.

That's where patient-reported outcomes (PROs) come in. PROs assess the impact of a disease and its treatment from the patient's perspective. Researchers collect data using patient diaries and scientifically validated assessment scales.

When appropriately studied, PROs can help:

  • facilitate patient-provider communications
  • aid in clinical decision-making
  • build the understanding of frequently overlooked problems of a disease or its treatment (like fatigue or pain)
  • improve side-effect management and patient compliance
  • evaluate the impact of treatment or interventions
  • increase patient satisfaction.

PROs: Basis for FDA Approval
Looking Back The study of quality of life dates back many years, to the work of J. Ware and the development of the SF-36 Health Survey—a 36-item short-form survey developed as part of the Medical Outcomes Study, a two-year study of patients with chronic conditions. Conducted by RAND, the study looked at general and specific populations, the burden of disease, and the effect of treatment. The SF-36 was intended for use in clinical practice and research, health policy evaluations, and general population surveys to aid in the development of better screening, diagnosis, and treatment for many diseases.

But the term QOL often has been misunderstood by clinicians and researchers, who sometimes refer to the measure as "soft," making it difficult to quantify. Because of that, FDA researchers looked into finding a term that more accurately reflected what QOL sought to do. The agency's ad division found that PROs did a better job of communicating the goal of measuring the impact of treatment from the patient's perspective. Therefore, the term PROs began to replace QOL.

Despite its favorable position, FDA said it would delay the development of formal guidance surrounding PROs until it collected more information. To that end, the agency created the Patient-Reported Outcomes Harmonization Group, a coalition of four organizations—the Pharmaceutical Research and Manufacturers of America (PhRMA), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the International Society for Quality of Life Research (ISOQOL), and European Regulatory Issues on Quality of Life Assessment (ERIQA) Group. These four organizations were charged with working closely with FDA to identify the information gaps and clinical needs in the area of PROs, prior to the issuance of any guidelines. After initial meetings, the Patient-Reported Outcomes Harmonization Group agreed that the PROs evaluation is a valid concept that should be measured in clinical trial settings.

Following ongoing discussions with FDA, and a March 2002 meeting that focused on identifying the proper measurement instruments, statistical measurements, and appropriate handling of missing data, the group is planning to publish an official document that could be used in the development of guidelines for the use of PROs measurements in clinical research and brand communications.

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