Journal Wrap | Patient-reported outcomes (PROs) represent powerful tools for patients, clinicians, and policy-makers, helping to shape therapeutic choices, disease management practices, reimbursement decisions, and health policy. However, PROs are not routinely used as key outcome measures in cardiovascular clinical trials. In a recent review paper published in the European Heart Journal, the European Society of Cardiology (ESC) issued a call to integrate PROs into cardiovascular clinical trials evaluating therapeutic interventions—for the good of both patients and clinicians.

To understand the benefit that is lost when PROs are excluded from major research, it is essential to understand the concept of PROs: they measure a specific concept or constructs from the patient's perspective, including health-related quality of life, symptoms, functioning, utility, and knowledge of or adherence to therapy, and measure different components of perceived patient well-being.

Of course, without properly validated instruments to measure PROs, they hold little value. The ESC paper provided the following prescription: the selected instrument must demonstrate good reliability and reproducibility, construct validity, and responsiveness to change. There are two types of instruments, both of which aid in different aspects of clinical care:

• Generic instruments allow comparisons to be made across clinical groups, and may be more useful for policy making and quantifying disease burden
• Disease-specific measures allow comparisons to be made across patients with given conditions, and may be more useful to inform clinical decision making and patient-centered care

Translating these findings into clinical practice exemplifies another hurdle that clinicians and health care decision makers must overcome. Patient-reported data can, for instance, guide a discussion between patients and health care providers regarding the net clinical benefit of a new therapy, or when balancing a potential therapy's risk-benefit profile, say antithrombotic agents that reduce the risk of recurrent cardiovascular events such as myocardial infarction or revascularization procedures but increase the risk of bleeding. "Patients are key stakeholders in health care decisions," Anker et al. stressed. "Patients may view the balance between such benefits and risks differently from clinicians, and PROs provide applicable data in this regard."

However, the authors noted that "determining an evidence-based level of meaningful change in a PRO measure" presents a real challenge in both cardiovascular clinical trials and clinical practice, and that "consensus has not been reached on what constitutes a clinically meaningful change."

While further research efforts are needed to resolve this major issue, the ESC ultimately encourages the incorporation of appropriate PRO measures in all pivotal cardiovascular trials and registries, providing a list of recommendations to achieve these goals:

• PROs reflect a key dimension of overall disease burden, and they should be a primary aim of disease management to improve patient well-being.
• Publication of high-quality research papers that describe the development and validation of PRO instruments or that report results of studies where PRO measures were primary or secondary endpoints should be encouraged.
• Patient-reported outcomes measures should be reported in all trials alongside mortality/morbidity outcomes (i.e., as major secondary endpoints) in accordance with the CONSORT (Consolidated Standards of Reporting Trials) PRO Extension.
• PROs should be available and considered for future practice guidelines.
• Physicians should be trained in the application and interpretation of PROs.
• PROs should inform clinical decisions and evidence-based guidelines.

"Professional societies can take action to influence the uptake of PRO data in the research and clinical communities," the authors concluded. "This process of integrating PRO data into comprehensive efficacy evaluations will ultimately improve the quality of care for patients across the spectrum of cardiovascular disease."

Anker SD, Agewall S, Borggrefe M, et al. Eur Heart J. 2014 June 5. [Epub ahead of print]

Keywords: CardioSource WorldNews Interventions

< Back to Listings