Title: Evaluation of the clinical and cost-effectiveness of Short-term Integrated Palliative Care Services (SIPC) to OPTimise CARE for people with advanced longterm Neurological conditions (OPTCARE Neuro)
Funder: Department of Health and Social Care

Abstract: DESIGN: Follows MRC guidance for evaluation of complex interventions: (i) set up, feasibility, mapping; (ii) randomised pragmatic trial of Short-term Integrated Palliative Care (SIPC) offered from a Multiprofessional Palliative Care Team compared to best usual care; (iii) a qualitative component, explores change process, how SIPC might be improved, interprets quantitative results, plus survey of health professionals; (iv) economic modelling. Methods build on those tested in our phase II randomised trial for MS patients, and longitudinal, economic and qualitative studies with patients and caregivers severely affected by these and other longterm conditions. SETTING: Five areas from across the UK; South London, Cardiff, Brighton and Sussex, Liverpool and Nottingham, all with palliative care, neurology/rehabilitation services and sufficient catchment population - representative of people with longterm neurological conditions in mixed urban, suburban and rural areas. TARGET POPULATION: Patients severely affected by advanced or progressive stages of the longterm neurological conditions (LTNCs) of either: • Multiple Sclerosis (MS) - primary or secondary progressive or aggressive relapsing remitting disease • Parkinson’s Plus (PP) i.e. Idiopathic Parkinson’s Disease(IPD) and related movement disorders of Progressive Supranuclear Palsy(PSP) or Multiple System Atrophy(MSA) - stages 3-5 Hoehn and Yahr(H&Y) or related measures for PSP/MSA • Motor Neurone Disease (MND) All are assessed (by clinicians) to have to have a least one unresolved symptom (e.g. pain, breathlessness, nausea/vomiting) which is continuing despite usual care and at least one of the following: 2nd unresolved symptom, cognitive problems; complex psychological (depression, anxiety, loss, family concerns) and/or complex social needs. INTERVENTION: SIPC runs from existing palliative care teams, linked with local neurology and rehabilitation services. Following referral a SIPC keyworker visits and makes a comprehensive palliative care assessment and proposes treatments, within 5 working days. There are 2-4 follow-up visits and referral to other services as appropriate. SIPC is offered in addition to usual services. CONTROL: Best usual services, including nurse specialists, neurology and rehabilitation. DATA COLLECTION: Face to face interviews at baseline (pre-randomisation), and follow up at 6, 12, 18 and 24 weeks. MEASUREMENT OF COSTS AND OUTCOMES: Recorded in face to face interviews with trained interviewers. Primary outcome is combined score of POS-S5 a validated measure of 5 core symptoms: pain, nausea, vomiting, sleeping difficulty and mouth problems at 12 weeks. Costs are estimated at baseline, and at 12-and 24-week follow-up from service use during the previous 12 weeks, recorded using an adapted version of the Client Service Receipt Inventory(CSRI). Secondary outcomes are: caregiver burden, caregiver experience, patient’s psychological distress, quality of life, palliative needs and symptoms recorded by patient, caregiver, presence of future care plans and a short observer (researcher) assessment. We survey health professionals on their views of SIPC. QUALITATIVE COMPONENTS: Interviews and focus groups with patients, families and staff in the set up to explore ways to: enhance recruitment; improve documentation; hone the components of SIPC and how introduced. During the trial: study the recruitment progression; consider any improvements. In order to understand the effects of SIPC we will conduct qualitative interviews with patients, caregivers and professionals to explore their experience of SIPC; explore change process; interpret the quantitative data, ascertain whether other aspects of their care need addressing. SAMPLE SIZE: We recruit 356 patients for the main trial. In view of the advanced illness in this patient group we have allowed 20% attrition (phase II trial attrition from death or illness: 3/52) to the primary 12-week outcome, giving 296 patients, or 148 in each arm with both baseline and 12-week outcome data. Each centre will recruit 70-90 patients over 27 months. Qualitative interviews are with 7 patients / caregivers and 6 staff per centre. The survey of health professionals is of 40 per centre, 200 total. ANALYSIS: Quantitative trial data follows CONSORT guidance on an intention-to-treat basis using generalised linear mixed model with centre as a fixed effect, adjusting for baseline score of POS-S5 and for variables from the baseline assessment for which treatment-group imbalance is found. Confidence intervals and test statistics are calculated using cluster robust estimates to account for therapist effects. The comparative effectiveness of the SIPC and standard care is explored and analysed within and across centres. The missing data are imputed using appropriate imputation techniques, depending on missing mechanisms. Robustness of findings is assessed, further uncertainties are addressed by sensitivity analysis. Secondary outcomes(hospital admissions, POS, ZBI, HAD, EQ-5D) are analysed using similar framework with that of primary outcome. The economic evaluation is conducted from(i) the health/personal social services perspective and (ii) a societal perspective. Qualitative data are analysed using the framework approach, in relation to aspects of study design and care. PROJECT TIMETABLE: Months 1-5 -Map current practice, set up trial, qualitative exploration, training, database preparation. Month 6-Trial commences. Monitor recruitment with feedback to centres. Months 6-33 – Recruitment of patients into study at rate of 8-15 patients per month (ie 1-3 patients per centre). Qualitative data collection. Months 11, 18, 24, 30-Formal reviews of recruitment rates. Data checking and cleaning. Months 12, 18, 24, 30-Interim dissemination of study, including mapping, qualitative exploration and trial recruitment and baseline needs, at conferences and with a six monthly e-newsletter. Months 33–Recruitment of new patients to trial ends, follow up continues for 6 months. Month 33-36-Analysis of all baseline data–Continued collection follow-up data Month 36-39 – Main trial analysis. Month 39–42 Analysis of final follow up data in both arms. Dissemination. PATIENT/PUBLIC INVOLVEMENT: 3 or more representatives (one is co-applicant), plus recruitment of individuals locally and from national groups on the Project Steering Committee, and integrated in all phases.

Lay abstract: Longterm neurological conditions (LTNCs) are a group of progressive disorders, affecting approximately 10 million people in the UK. People with LTNCs commonly have physical disabilities affecting arms, legs, bladder, bowel, eyesight, or speech, for example, plus other intractable symptoms including pain spasms and fatigue. In addition, there is often a double burden of psychological distress or cognitive dysfunction. Over one third of people with LTNCs are estimated to need help with daily living, and nearly one million informal carers support someone with a neurological disease. Earlier phases of neurological illness can be managed well with medical treatments, rehabilitation, care and support. However, some diseases are or become invariably progressive. In the later stages those affected experience increased problems with greater need for personal, social, psychological and spiritual support. Care needs for those severely affected by LTNCs rise sharply once the disease reaches more advanced stages. Research has shown that these are not managed well by existing standard care; patients live with continuing symptoms, emotional and social problems. Their families also carry increasing burdens. While costs to the NHS increase, not all the care given is experienced as appropriate according to reports from some patients and families, with failures in coordination between acute and community care. Palliative care is a multi-professional system that addresses the needs of the whole person – physical, emotional, social and spiritual. Specialists in palliative care have trained in these specific aspects, and how to communicate well during difficult times. They work in multi-professional teams, in-patient hospices, hospitals, care-homes and in home care. Palliative care has been suggested in NHS policy documents, such as the National Service Framework for LTNCs (quality standard 9) as a way to better help people severely affected by longterm neurological conditions, but it has not been rigorously tested. There is also uncertainty about when to introduce palliative care. Palliative care services are unevenly distributed, many managing on voluntary funding. Primary Care Trust spend on specialist palliative care ranges from £186 to £6,213 per death, with most of their funding supporting cancer patients. Without robust evidence about the best models of providing palliative care for people with longterm neurological conditions, it is very unlikely that palliative care services will be offered appropriately. We have conducted preliminary research to understand the needs of people affected by the late stages of several LTNCs. We found that in addition to pain and spasms many patients experienced symptoms such as nausea and sleeping difficulties which were overlooked. People had on average 11 physical symptoms, plus emotional concerns and these became steadily worse over time. We then conducted a successful small scale randomised trial in one condition, multiple sclerosis, and found that referral to short term integrated (with neurology and rehabilitation) palliative care improved symptom control, reduced caregiver burden and costs to the NHS (largely by reducing inappropriate hospital admissions) over a 12 week period, with effects lasting beyond that. In this study we aim to find out whether offering Short-term Integrated Palliative Care Services (SIPC) to people affected by a wider group of neurological conditions and in several centres can improve their care experience, particularly regarding management of pain and symptoms but also quality of life, anxiety and depression, communication, and caregiver burden. It will also consider whether SIPC may be more cost-effective than standard care, or affects hospital admissions. We have chosen to concentrate on 3 main groups of LTNCs, which are common, distressing and may be suitable for palliative care. These are Multiple Sclerosis, Parkinson’s Plus disorders (Idiopathic Parkinson’s Disease(IPD) and related movement disorders of Progressive Supranuclear Palsy(PSP) and Multiple System Atrophy) and Motor Neurone Disease(MND). During the development and set-up phase we will determine what constitutes SIPC and will map standard NHS practice. Through this process, a manual will be prepared to guide clinicians and practitioners in order to establish a high level of consistency. Open interviews will be conducted with patients and caregivers to assist with the development of good quality, accurate patient information materials to ensure the operation of informed consent and to support high rates of recruitment and retention. Then we will commence the randomised trial in our centres. We will check we achieve our recruitment rates and that procedures are feasible. All necessary ethical and NHS approvals will be gained before starting the study, and all who take part may withdraw at any time without their standard care being affected. People who meet our inclusion criteria (suffering from the neurological conditions, and with symptoms and problems) in each UK centre, will be invited to take part in the study. It is entirely up to individuals whether they decide to participate. Those who agree will be randomly allocated, by a computer (randomised controlled trial), to receive either: i) Standard Care plus SIPC or ii) Standard Care. They will know which group they are in, but are asked not to tell the research nurse. After 12 weeks the Standard Care group will also be offered SIPC, as a delayed intervention. Participants and their caregivers will be asked to complete a series of questionnaires (about symptoms, quality of life, mood and services received) at the start of the study, then at 6, 12, 18 and 24 weeks. We will collect information about hospital admissions and services used from clinical records. The study will focus mainly on the difference between scores at the outset and at 12 weeks for the two groups (Standard Care plus SIPC versus Standard Care). Assessment to 24 weeks will determine how long SIPC effects are maintained and whether there are benefits of earlier referral. Open interviews with patients, caregivers and staff will help us understand more about what they value most about SIPC and how it may be working. The study takes place over 42 months, with the set-up and development phase in months 0-5, the main trial in months 6-39 (recruitment ending month 36), followed by analysis and dissemination

Research activity codes: 8.1 ORGANISATION AND DELIVERY OF SERVICES;8.2 HEALTH AND WELFARE ECONOMICS
Health categories: NEUROLOGICAL