BEAT CF pulmonary exacerbations core protocol for evaluating the management of pulmonary exacerbations in people with cystic fibrosis.

Background: Cystic fibrosis (CF) is a rare, inherited, life-limiting condition predominantly affecting the lungs, for which there is no cure. The disease is characterized by recurrent pulmonary exacerbations (PEx), which are thought to drive progressive lung damage. Management of these episodes is complex and generally involves multiple interventions targeting different aspects of disease.

Protocol for establishing a core outcome set for evaluation in studies of pulmonary exacerbations in people with cystic fibrosis.

Introduction: Pulmonary exacerbations are associated with increased morbidity and mortality in people with cystic fibrosis (CF). There is no consensus about which outcomes should be evaluated in studies of pulmonary exacerbations or how these outcomes should be measured. Outcomes of importance to people with lived experience of the disease are frequently omitted or inconsistently reported in studies, which limits the value of such studies for informing practice and policy.

Preferred health outcome states following treatment for pulmonary exacerbations of cystic fibrosis.

Background: Treatment for pulmonary exacerbations of cystic fibrosis (CF) can produce a range of positive and negative outcomes. Understanding which of these outcomes are achievable and desirable to people affected by disease is critical to agreeing to goals of therapy and determining endpoints for trials. The relative importance of outcomes resulting from treatment of these episodes are not reported.

Novel method to select meaningful outcomes for evaluation in clinical trials.

Background: A standardised framework for selecting outcomes for evaluation in trials has been proposed by the Core Outcome Measures in Effectiveness Trials working group. However, this method does not specify how to ensure that the outcomes that are selected are causally related to the disease and the health intervention being studied. Causal network diagrams may help researchers identify outcomes that are both clinically meaningful and likely to be causally dependent on the intervention, and endpoints that are, in turn, causally dependent on those outcomes.

Long-term use and cost-effectiveness of secondary prevention drugs for heart disease in Western Australian seniors (WAMACH): a study protocol.

Introduction: Secondary prevention drugs for cardiac disease have been demonstrated by clinical trials to be effective in reducing future cardiovascular and mortality events (WAMACH is the Western Australian Medication Adherence and Costs in Heart disease study). Hence, most countries have adopted health policies and guidelines for the use of these drugs, and included them in government subsidised drug lists to encourage their use. However, suboptimal prescribing and non-adherence to these drugs remains a universal problem.