Using fast-track trial methodology to evaluate a breathlessness intervention service for patients with advanced disease Morag Farquhar Macmillan Post-Doctoral Research Fellow GPPCRU University of Cambridge IPH Showcase 14 th February 2011

Breathlessness Intervention Service (BIS) aims to help people live with breathlessness in advanced disease any diagnosis (cancer or non-cancer) works jointly with patients & carers advises referrers multi-disciplinary hospital-based service functioning in the community ‘toolkit’ of pharmacological and non-pharmacological interventions complex intervention (MRC framework) – Phase III RCT

3 RCTs in palliative care Randomised Controlled Trials (RCTs): –‘gold standard’ for evaluating services –intervention & control group > compare outcomes RCTs in palliative care problematic: –recruitment - patients (& referrers) reluctant to randomisation to non-intervention arm –attrition – deterioration / death non-RCT: –before-after study (observational study) –but are any identified changes due to the intervention?

Fast-track methodology fast-track RCT: –‘fast-track’ group (intervention immediately) –‘control’ group (intervention after waiting list) also called: –‘delayed intervention trial’ –‘deferred intervention trial’ –‘waiting list trial’ or ‘wait list trial’ fast-track RCT: –strength of an RCT, but appears acceptable to palliative care patients & referrers [Farquhar M, Higginson IJ, Booth S. Fast-Track Trials in Palliative Care: An Alternative Randomized Controlled Trial Design. Jnl Pall Med 2009;12(3):213]

5 BIS Phase III fast-track RCT fast-track single-blind pragmatic RCT of BIS versus standard care mixed methods RCT (qualitative & quantitative data) multiple perspectives: patients, carers, referrers, providers economic evaluation design informed by Phase II pilot RCT [Farquhar M, Higginson IJ, Fagan P, Booth S. The feasibility of a single-blinded fast-track pragmatic randomised controlled trial of a complex intervention for breathlessness in advanced disease. BMC Palliative Care 2009; 8:9] different disease trajectories: BIS service model varies by disease group (interventions & timing)…

Phase III: BIS service model patients with non-malignant conditions (e.g. COPD, heart failure): 3 visits 3 phone calls over 4 week period patients with malignancies (any cancer): 1 visit (ideally with primary care) 2 phone calls over 2 week period two sub-protocols (two RCTs in one…)

WEEK Baseline interview t1 (n=87) Withdrawn (significant deterioration between agreeing to recruitment visit and visit) / died (n=11) Eligible patients (n=158) Starts to receive BIS (n=44) Mid-intervention interview t2 (n=41) End of intervention interview t3 (n=41) Mid-waiting interview t2 (n=38) End of waiting interview t3 (n=38) Waiting list group starts to receive BIS Mid-intervention interview t4 (n=37) End of intervention interview t5 (n=37) Taken off study n=0 (died) = intervention Fast track group Agreed to recruitment visit n=98 Waiting list group Referrer interview Follow up interview t5 (n=36) Receives standard care (n=43) Referrer interview BIS Phase III: non-malignant conditions sub- protocol n=87 patients 4 week intervention n= 24/33 referrers (73% RR)

WEEK Baseline interview t1 (n=?) Withdrawn (significant deterioration between agreeing to recruitment visit and visit) (n=?) Eligible patients (n=?) Starts to receive BIS (n=?) End of intervention interview t3 (n=?) End of waiting interview t3( n=?) Waiting list group starts to receive BIS (n=?) End of intervention interview t5 (n=?) Taken off study n=? (died) = intervention Fast track group Agreed to recruitment visit n=? Waiting list group Referrer interview Receives standard care (n=?) Follow up interview t5 (n=?) Referrer interview BIS Phase III: malignant conditions sub- protocol n=26 / 60 patients 2 week intervention

Phase III fast-track RCT: other data as well as patients’ & carers’ and outcomes within RCT… views of (qualitative interviews – process & outcome): –referrers –providers intervention data (fidelity): –BIS records –audio-recordings

BIS evaluation key collaborators: Dr Morag Farquhar (University of Cambridge) Prof Irene Higginson (KCL) Prof Chris Todd (University of Manchester) Dr Toby Prevost (University of Cambridge & KCL) Prof Paul McCrone (KCL) Dr Sara Booth (CUHNFT) RA: Barbara Brafman-Kennedy (CUHNFT) Funders: NIHR Research for Patient Benefit (BBK) Macmillan Cancer Support (MF) This presentation presents independent research commissioned by the National Institute for Health Research (NIHR) under its Research for Patient Benefit (RfPB) programme (Grant Reference Number PB-PG ). The views expressed are those of the author and not necessarily those of the NHS, the NIHR or the Department.