Commentary: Incorporating Patient-Reported Outcomes in Periodontal Clinical Trials

• Autores: Michael K. McGuire, Dennis Tarnow, E. Todd Scheyer
• Localización: Journal of periodontology, ISSN 0022-3492, Nº. 10, 2014
• Idioma: inglés
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• Resumen

  • The authors review patient-reported outcome (PRO) metrics for dentistry, and in particular, periodontics. The PRO commentary for periodontics includes a review of split-mouth, randomized, controlled clinical trial results that specifically tracked pain at different sites over time after intervention and provided guidelines for peak pain time points and evidence for referred pain assessment when studying soft tissue augmentation procedures. Both the questions that are asked of patients and the timing of those questions are important study design considerations. The authors suggest PRO methodology for periodontal clinical trials that can be used to identify information important to patients and clinicians.

    As a measure of dental practices, the ultimate patient outcome is whether a patient preferentially returns to the dental offices, or better yet, refers a friend. In this regard, the patient�s opinions about treatments are fundamental to what dental professionals do. But in clinical research, patient-reported outcomes (PROs) � what the patient reports about his or her treatment experience � are fast becoming the metrics that will also help determine the services provided.

    Intuitively, one knows that PROs such as quality of life should help find the best therapies, but the practice of effectively incorporating PROs in periodontal clinical studies is only now being refined. By definition, a PRO is �any report of the status of a patient�s health condition that comes directly from the patient, without interpretation of the patient�s response by a clinician or anyone else.�1 This can include reports about specific symptoms, such as pain at a particular site in the mouth, or more general concepts, such as difficulty eating or, more generally, treatment satisfaction � always reported directly by the patient. However, creating valid PRO instruments and metrics requires more than simply removing clinicians and staff from the interpretation of patient opinions. The US Food and Drug Administration and the European Medicines Agency have provided position papers and guidelines for the development, implementation, and interpretation of PROs in clinical trials.2 Indeed, PRO instruments have served as the basis for regulatory approval and appeared in labeling for products across several therapeutic areas.3 In essence, PRO instruments should: 1) be free from error (be reliable); 2) measure what they are intended to measure (be valid); 3) be sensitive to changes in the patient�s condition (be able to detect treatment differences); and 4) be interpretable (be clinically meaningful).4 Although these goals might also sound easy to reach, effective PRO methodology is surprisingly difficult to develop.

    PROs, by definition, are subjective, i.e., they involve the measurement of a wide spectrum of patient opinions and experiential responses to difficult-to-quantify endpoints, such as anxiety, pain, and satisfaction. Because many important patient experiences occur sporadically (for example, sudden pain when chewing), they need to be recorded in near real time, as they are occurring in the patient�s natural environment. Also, patients may be reluctant to provide honest, unguarded answers, particularly when speaking with, and trying to please, their caregivers, so PROs must be administered and recorded in a way that minimizes these �demand characteristics.�5 For example, computerized assessments or questions administered by staff members who are not involved in the patient�s treatment may yield more accurate responses. Exacting item development procedures, usually including an iterative process of interviews with patients,6 and innovative trial designs must be implemented to best develop and administer clear and quantifiable PROs. This is critical in periodontal clinical trials, where patient experiences, such as pain, are central to evaluating new treatments.