Patient-centred healthcare outcome measures: towards a unified architecture

Introduction

Measuring appropriate outcomes is at the core of patient-centred medicine. With an ever-increasing number of clinical trials being published, the heterogeneity of outcome measures used in different trials makes direct comparisons difficult. Despite this, clinical effectiveness research is increasingly influential in making decisions about which treatments are best for patients. A recent judgment has strengthened the importance of commissioners in the UK National Health Service (NHS) of recommendations made by the National Institute of Health and Clinical Excellence (NICE), which are based on outcomes research. ¹ In the USA, The Patient Protection and Affordable Care Act created the Patient-Centered Outcomes Research Institute, to further assess options for prevention, diagnosis and treatment of disease.

Clinical researchers use outcomes for treatment guidelines

In an attempt to address this concern, clinical researchers recommend the development and use of core outcome sets. A core outcome set is a minimum set of outcome measures that is assessed and reported in all trials for a particular condition. Validated core outcome sets in conditions such as rheumatoid arthritis, stroke and traumatic brain injury are currently in use, ² and their use has now been advocated by funding bodies such as the National Institute for Health Research (NIHR). These outcome sets are developed by consensus, taking into account the viewpoints of a range of stakeholders including patients, clinicians, academics, statisticians and clinical trialists. The incorporation of patients in this process has been proven to enhance the inclusion of important patient-related outcomes and explore research agendas previously not considered. ³

The development of core outcome sets has been assisted by organisations and projects such as the Core Outcome Measures in Effectiveness Trials (COMET) initiative ⁴ and the National Institute for Neurological Disorders and Stroke (NINDS) Common Data Elements (CDE) project. ⁵ These initiatives have provided guidance and support for investigators to develop core outcome sets in their respective fields.

Policymakers use outcomes to analyse health systems

Interest in core outcome sets extends beyond clinical trials. Health policy experts and health economists see core outcome measures as a key tool in assessing the ‘value’ of interventions. ⁶ In healthcare, ‘value’ is defined as a function of outcomes produced in a patient pathway expressed in relation to the cost required to service the pathway. ⁶ Without a validated set of core outcome measures, assessments of ‘value’ would not be comparable. The development of core outcomes sets in this sphere has been pioneered by organisations such as the International Consortium for Health Outcome Measures (ICHOM). ⁶

Opportunity for collaboration

Clinical trial academics and health economists have independently noted the importance of developing a validated set of patient-centred core outcome measures. Since two different interest groups are collecting data, there is the potential for heterogeneity and replication of work. This wastes resources and is therefore best avoided.

Consequently, we feel that collaboration between the two groups, sharing outcome set development methodology and developing a unified set of core outcomes could be beneficial to all stakeholders. Clinical trialists and health economists stand to benefit from improved methodologies and the increased availability of comparable core outcomes for their respective purposes, and clinicians benefit from a clearer understanding of the most effective treatments. Most importantly, patients benefit from better healthcare.

Patient involvement is key

Involving patients in designing outcome measures helps ensure that they are able to influence clinical priorities for the care they receive. For example, in Germany, most centres have similar five-year mortality rates for prostate cancer, but there is a wide variability in outcomes that are also important to patients, such as rates of incontinence and erectile dysfunction. ⁷ Outcomes collection of patient-relevant measures across the country can help to highlight this variability to patients and to their doctors, enhancing the work organisations such as NHS Right Care, which currently produces atlases of variability in healthcare services. In turn, doctors may be able to recognise opportunities to improve their practice and outcomes, and even be offered financial incentives to drive-up quality. ⁸

Furthermore, ensuring that patient-centred outcomes are collected is important because doctors may underestimate or overestimate the importance of certain complications or side-effects to patients and therefore may underemphasise or overemphasise at the point of treatment decision making. Lower take-up for prostate surgery was demonstrated after showing patients videos explaining the outcomes of surgery, developed after gathering patient concerns through open-ended questions in patient interviews. ⁹

Recommendations

A recent systematic review of core outcome sets developed for comparative effectiveness research shows variability in extent to which patients were involved in the choice of outcomes and the method by which patients were involved. ² Groups examining outcomes from a policy perspective, such as the Dartmouth Atlas and ICHOM, have developed standardised methodologies for this involvement. We therefore advocate the development of best practice guidelines on patient involvement methodology for outcomes research. We believe that NHS England should encourage partner clinical and research organisations to make use of these guidelines, both in terms funding outcomes-based research and utilising this research in commissioning services. Organisations such as INVOLVE (funded by NIHR to increase patient involvement in research) and COMET would be useful in achieving this aim.

Second, there is a mismatch between diseases for which outcome sets have been developed ² and diseases which represent the highest proportion of morbidity to the domestic population, such as hypertension, obesity, ischaemic heart disease, COPD and depression. ¹⁰ We therefore call on NICE to consider the development of a patient-centred outcomes framework using approaches supported by COMET and core outcome sets. This would require interaction between patient groups, clinicians, health economists, policy makers and academics in order to produce core sets for each condition as they next come under review.

Only by finding out what our patients most value from healthcare can we truly prioritise their needs. Emerging technologies may allow us to take advantage of collecting patient-reported outcomes in routine clinical practice, using remote and electronic means, so that in future, we may be able to more rapidly incorporate such data to feed back into our own clinical practice.