An integrated approach to outcome evaluation : incorporating patient reported outcomes in heart failure

Abstract

Globally individuals and health care systems are facing the burden of chronic illness. The impact of the increasing burden of non-communicable diseases is experienced by individuals and health care systems. Across the globe health care systems are struggling to meet the increasing demands for services within the confines of rising costs and needs for accountability. Beyond costs and treatment allocations, there is an increasing mandate to provide care that is patient centred and appropriate to the needs of the individual. The Innovative Care in Chronic Condition (ICCC) framework has been successful in driving health care reforms to meet the needs of individuals with chronic illness internationally. Deriving metrics that allow monitoring of conditions at the level of the patient, provider and health care system are of increasing importance. Comprising this thesis is a series of studies to investigate outcomes that includes the patient’s perspective in the evaluation of clinical interventions. To achieve this, chronic heart failure, was used as an exemplar of a chronic condition.Chronic heart failure (CHF) is the final common pathway for many cardiac conditions. As a consequence has emerged as a major public health problem and represents as an excellent exemplar of living with a chronic illness. CHF patients commonly experience high levels of ill-health, disability and mortality placing a heavy burden on health care systems. Hospitalisations are frequent and costly to both CHF patients and to society. People with CHF live with a limited quality of life and physical ability and the prognosis for CHF is poor. Given the nature of debilitating symptoms, and their potential impact on physical, social and psychological aspects of life, patient’s perspective in outcome assessment is essential in providing effective care.Specifically this study sought to: •Examine patient reported outcomes in clinical management and in clinical research •Investigate patient important outcomes, their utility, relevance and acceptability amongst patients, clinicians, researchers and administrators •Test composite outcomes model that integrate patient important outcomes in clinical trials researchPatient reported outcomes (PROs) is a strategy to capture the patient perspective and experience on their health status. The use of PROs can be incorporated in clinical assessments, monitoring of clinical progress as well as clinical research. Despite their frequent use in research, evidence suggests that to date they have had a limited influence on clinical practice and policy. As part of this thesis an integrative review was conducted to explore the potential utility of PROs at the policy level. By using the ICCC framework, PROs were indeed essential to improve the management of CHF at the micro, meso and macro levels of decision making.One of the key challenges in using PROs and outcomes important to individuals in CHF is limited methodological and reporting quality. This is cited as a reason why many clinicians are sceptical of the utility of PROs. To explore issues in reporting a review was conducted on RCTs of pharmacological therapy in CHF that reported health related quality of life (HRQoL) as a primary or secondary outcome. Using the Minimum Standard Checklist for evaluating the quality of reporting of HRQoL outcomes resulted in 26 (19.1%) studies being considered ‘very limited’ in terms of methodological and reporting rigour, and 91 (66.9%) were evaluated as ‘limited’ and only 19 (14.0%) studies were considered to be of a ‘probably robust’ quality. In fact, the quality of HRQoL reporting has not improved over time. Some of the issues identified are limited discussions, methodological shortcomings, and poor HRQoL reporting. This review has underscored the importance of standardising of the reporting of HRQoL measures.Although capturing the patient’s perspective via PROs is important, they may not be the only outcome measures important to patients. Currently, no single CHF outcome measure captures all dimensions of the quality of care from the patient’s perspective. To identify outcome measures in CHF deemed important to patients, a structured literature review was undertaken. The conceptual and methodological challenges and opportunities in each outcome measure were identified as important to patients with CHF. That is mortality, hospitalisation and PROs were identified as important to patients but also meaningful and relevant to the provider and health care system as well. These outcome measures were proposed as a core outcome set that represent the minimum set of outcomes that should be measured and reported in CHF.A number of composite outcome measures have been developed to capture the perspective of the patient, clinician as well as including objective measures of health. Three validated composite outcomes, the Packer’s Score, Cleland’s Patient Journey and the composite endpoint used in the African American Heart Failure Trial (A-HeFT) were examined in a secondary analysis of a prospective, multi-center randomized controlled trial of 280 hospitalized CHF patients in the Which Heart failure Intervention is most Cost-effective & Consumer Friendly in Reducing Hospital Care (WHICH?) Trial in order to assess the comparability and interpretability of the measures in a pragmatic clinical trial. Correlation coefficients demonstrated substantial associations amongst all three composite endpoints. Although there was a considerable agreement across the three measures when estimating deteriorating condition, these was less when estimating improvements.This thesis has described both the importance and complexity of including outcome measures that are meaningful to patients in both the assessment of individuals’ needs, testing interventions, monitoring outcomes and assessing process and outcome measures at a health systems level. This thesis has also extended the discussion and debate around PROs to discuss Patient Important Outcomes, which is outcomes that patients notice and for which they would be willing to undergo a treatment with associated risk, cost, or inconvenience for it to be the only thing that changed. Using CHF as an exemplar has provided useful insights into the dimensions and complexities of measuring outcomes in chronic and complex conditions. As the burden of chronic disease continues to increase refining the metrics of outcome measurements will be equally as important as refining novel therapies. This will be critical to develop and implement interventions to meet the growing numbers of people living with chronic illness.