Title: Facilitating informed decision-making in haemato-oncology
Funder: Department of Health and Social Care

Abstract: Aims and Objectives The underlying hypothesis is that delivery of personalized evidence-based information at key decision points along the care-pathway will facilitate engagement of patients and clinicians in shared decision-making that will, in turn, support and improve clinical practice. Such models will also provide information on the cost-effectiveness of alternative interventions to managers and commissioners. Background and Rationale Characterized by their remitting/relapsing nature, the pathways of patients with chronic haematological malignancies are diverse; some patients having long periods without treatment, others receiving multiple courses of chemotherapy and a few, particularly those with myeloma, having high dose chemotherapy and bone marrow transplants. While such cancers are presently incurable and their management is often based on subjective assessment of symptom burden, their prevalence is increasing as the population ages, new diagnostic techniques are developed, and the numbers and combinations of life-prolonging treatments continue to expand. Furthermore, the availability of increasing numbers of patient and tumour associated prognostic markers, and the incorporation of these data into existing prognostic scores, means that personalized/stratified approaches to the treatment of these cancers are becoming progressively more possible. All of this means that patients, clinicians and commissioners face increasingly complex choices about therapeutic options that differ in efficacy, toxicity, and cost (personal and financial). However, whilst information on the likely need for and effectiveness of treatment, its consequences for the patient s lifestyle, and costs to the healthcare system lie at the heart of effective shared decision-making, there is an acknowledged dearth of appropriate information for haematological cancers &ndash; and it is this challenge that provides the underpinning rationale for this programme. Research Plan Representative of the spectrum of chronic haematological malignancies, this programme focuses on the information needs of patients with three exemplar chronic haematological malignancies: chronic lymphocytic leukaemia, follicular lymphoma and myeloma &ndash; which combined account for around 45% of newly diagnosed lymphoid malignancies. The programme of work is predicated on the established expertise and infrastructure of the Haematological Malignancy Research Network (www.hmrn.org), which was successfully initiated in 2004 with the aim of providing robust generalizable data to inform research and clinical practice &ndash; both locally and nationally. HMRN s catchment population (>3.8 million) has a similar socio-demographic profile to the country as a whole, and patient care is provided by a unified clinical network organized into five multi-disciplinary teams (MDTs) that work to common guidelines. Importantly, all diagnoses (>2,200 new patients annually), including disease progressions and transformations, are coded to the latest WHO classifications by clinical staff at a single integrated haematopathology laboratory that contains all of the technology and expertise required for diagnosis and on-going monitoring. Within HMRN all patients have full treatment, response and outcome data collected to clinical trial standards; and the study operates with Section 251 support under the NHS Act 2006, enabling linkage to nationwide information on deaths, cancer registrations, and Hospital Episode Statistics. The programme will be carried out in five distinct, but overlapping, work packages: Exploration and analysis of information needs, decision-making processes and preferences Population-based pathway analyses and prognostic model development Cost effectiveness/economic analysis, and economic evaluation Development of information resources to support decision-making Feasibility testing of patient experience survey methods and use of information resources in clinical practice (MDT and clinic settings) Using an iterative approach to the analysis of information from around ten focus groups and around 60 interviews, WP1 will provide unique insights into patient and practitioner experiences of decision-making, and information needs across different disease trajectories; which will inform the development of resources in WP4. In addition to delivering stand-alone outputs developed using existing data from approximately 6,500 patients (diagnosed 2004-14) and new data from a further 2.5 years collected during the programme, the closely linked WP2 and WP3 will also feed into WP4. Finally, WP5 will test the feasibility of introducing the information resources, iteratively developed in WP4, into routine clinical practice in a single MDT. The feasibility of introducing patient experience survey methods into routine practice across the 14 hospitals (5 MDTs) will also be established in WP5. A protocol for a future cluster randomized trial will be developed based on these findings. Projected Outputs and Dissemination Plan All WPs will produce individual outputs that will inform the evidence-base, both applied and theoretical. The integrated information resources and associated NHS delivery mechanisms are, however, the primary goal. Research findings will be disseminated throughout via peer review, conference presentations, public meetings, professional bodies, and national patient organisations/partnerships/charities. Progress will be monitored by the Programme Steering Committee. Benefit to Patients and the NHS Provision of high-quality prognostic material will benefit all stakeholders; informing MDT recommendations, increasing efficacy and satisfaction of clinician-patient consultations, and providing data for commissioners. Such evidence is currently unavailable and this programme will facilitate the development of stratified/personalized medicine, as well as informed shared decision-making, both central NHS goals.

Lay abstract: Chronic blood disorders (including chronic lymphocytic leukaemia, follicular lymphoma and myeloma) are complicated diseases that cannot be cured. Many different approaches are available to manage these cancers, including active monitoring ( watch and wait ), multiple courses of drugs (chemotherapy), and bone marrow transplants. Choosing the best option can be difficult, as research evidence often comes from studies with patients who are younger and fitter than average, and may not keep-up with the rapid pace of developments. Recent scientific advances mean it is now possible to know the risks/benefits of some treatments, including whether the treatment will work, how it will affect quality of life/lifestyle and length of time a patient may live, as well as how much it will cost. Our research develops this individualised information, so it can be made available in an accessible and usable format to patients, doctors and nurses to share and use, in order to make the best treatment decisions. Information will also be available to commissioners, so they can make sure treatments are available. Working with patients with chronic blood disorders, as well as their doctors and nurses, we will find out about their information needs and preferences. We will develop and provide information that is specific for individual patients, taking into account their type of disease, how far it has spread, treatment already received; and other things, such as their age and general health. During the programme, we will test the feasibility of introducing this information in hospitals in the study area. This will allow us to check what patients and staff think about this information, whether it helps patients to become more involved in decisions about their care, and to assess the economic costs. The proposed research will build on a study in the North of England where all chronic blood disorders are diagnosed at a single hospital laboratory, and information about treatments and use of services is collected and linked with laboratory results. The study team are experienced in doing work of this type; and some of them (researchers, doctors and nurses) have worked together since 2004 when they set up the project supporting the new research proposed here. Others, with experience in decision-making and health costs, have recently joined the team. The two patient applicants are members of our established Patient Partnership, and will help to make sure the information we develop is suitable. We hope this information will eventually be used in hospitals across the whole country.

Research activity codes: 7.1 INDIVIDUAL CARE NEEDS;7.3 MANAGEMENT AND DECISION MAKING
Health categories: CANCER AND NEOPLASMS