Patient-reported outcomes and health-related quality of life in effectiveness studies: Pros and cons

The importance of patient perspectives on disease impact and response to therapy is increasingly recognized. Patient-reported outcomes (PROs), including health-related quality of life (HRQL), provide information on the effectiveness of medical interventions that cannot be obtained using objective clinical measures. For drug development, PROs such as symptom questionnaires are accepted by the European Medicines Agency (EMEA) and US Food and Drug Administration (FDA) in the approval process. HRQL assessments can help to establish the niche for a new product to support marketing efforts. Utilities can also be derived from health outcomes measures for cost-effectiveness analyses required by health plans for granting drug reimbursement. Because they more closely reflect routine clinical care, effectiveness studies are often a superior setting than traditional randomized controlled trials (RCTs) for conducting comprehensive PRO assessments to determine the real-world impact of therapy on symptoms and other aspects of everyday life. Screening, quality of care, self-reported adherence to treatment, and patient monitoring are important potential applications of PROs in effectiveness studies. However, well-known limitations of effectiveness studies also apply to PROs; these sometimes include a lack of randomization, controls, and blinding. It may be difficult to obtain patient compliance and clinician assistance with PRO assessments in a real-world setting. PRO data may be biased by nonrandom patient dropout, errors in timing of PRO assessments, and the environment in which questionnaires are administered. Additionally, the drug development schedule often requires outcomes results before effectiveness studies can be conducted. Addressing these difficulties during study design and the use of short and targeted questionnaires will improve the usefulness and feasibility of PRO measures in effectiveness studies.