Title: CONCORD: CO-ordiNated Care Of Rare Diseases
Funder: Department of Health and Social Care

Abstract: IMPORTANCE: Depending on the disease and where they live, patients with rare diseases may receive co-ordinated care at specialist clinical centres, which bring together various professionals so patients can see them all in a single visit. Unfortunately for many people it is usual to have to visit different hospitals several times for treatment, which can be inconvenient, costly and reduce the likelihood of attendance. Also, most people with rare diseases receive some of their care at their local hospital or GP practice. This can cause problems because co-ordination between different organisations is often not very good. Some people have access to named care co-ordinators to help with this, but many do not. In 2013 the UK governments published the UK Strategy for Rare Diseases, which said it is essential to co-ordinate care for people with rare diseases. Unfortunately, there is not good evidence as to how this should be done, and the Strategy called for further research in this area. RESEARCH QUESTIONS: RQ1: What does “co-ordinated care” mean, what are the components of co-ordinated care, and in what ways and why may co-ordinated care for people with rare diseases be similar or different to co-ordinated care for people with other conditions? RQ2: Is care for people with rare diseases in the UK co-ordinated, and if so, how? RQ3: What are the preferences of patients and families and professionals in relation to how care for rare diseases is co-ordinated? RQ4: What are the different ways in which care for people with rare diseases might be co-ordinated RQ5: How much do these options cost? METHODS: For RQ1 we will undertake a scoping review (not only in rare diseases) focusing on care co-ordination across organisational boundaries and interventions employed to support and improve this. For RQ2 and RQ3 we will create a questionnaire-based survey of current experiences and costs, incorporating a discrete choice experiment of preferences. The questionnaire will be co-produced with patients and families. RQ4 will involve interviews, focus groups and workshops with stakeholders (patients and families affected by rare diseases, local and national commissioners, national patient groups and charities, providers of co-ordinated care from health care social care and voluntary sectors) to develop a taxonomy of co-ordinated care for rare diseases covering health, social and third sector care. RQ5 will calculate the costs incurred in setting up and running these services. We will hold a dissemination event to discuss lessons learned and how these might be applied more widely. MAIN BENEFITS: The proposed research will contribute to better co-ordination of care for people with rare diseases. It will improve understanding about what co-ordinated care should entail for people with rare diseases, provide evidence about how care is currently co-ordinated, and ascertain preferences for co-ordinated care (i.e., what type of co-ordinated patients and families affected by rare diseases, and others, would like and need). This will inform how care co-ordinated might be centred around the needs and preferences of patients and families affected by rare diseases.

Lay abstract: A rare disease is one that affects fewer than 5 people in 10,000 in the general population. So by definition, a single rare disease affects a small number of people. However, there are over 8,000 rare diseases and if all the people suffering from these were added together, the total in the UK would come to more than 3.5 million people. So, altogether, rare diseases affect a large number of people. Rare diseases can be severe, disabling and affect more than one part of the body. As a result, patients often require treatment from different experts. Depending on the disease and where they live, patients may receive treatment at specialist clinical centres, which bring together various professionals so patients can see them all in a single visit. Unfortunately for many people it is usual to have to visit different hospitals several times for treatment, which can be inconvenient and costly, and also reduce the chance that people will turn up for their appointment. Also, for most people with rare diseases some of their care is provided nearer to home by their local hospital or GP. This can cause problems because co-ordination between different organisations is sometimes not very good. Some people have access to named care co-ordinators to help with this, but many do not. In 2013 the UK governments published a document called the UK Strategy for Rare Diseases, which said it was essential to co-ordinate care for people with rare diseases. Unfortunately, there is not good evidence as to how this should be done, and the Strategy also called for further research in this area to address this issue. To meet the study objectives we will: 1. Undertake a review to find out what “co-ordinated care” means. 2. Run a survey of people affected by rare diseases (patients and carers) to ask how care is co-ordinated. 3. Include in this survey questions about how people would prefer their care to be co-ordinated. 4. Develop a way of classifying how care of rare diseases can be co-ordinated. 5. Calculate how much these different ways of co-ordinating care might cost to run and to set-up and implement To be successful the study requires input from patients and families. This will help to ensure it meets the needs of people affected by rare diseases. To achieve this, our study team includes representatives from national patient organisations with direct experience of living with rare diseases. They will contribute to research design, patient recruitment, data collection, interpretation of study findings, and dissemination. They will also run the project’s PPI Advisory Group (PPIAG), which will involve working with a group of patients/carers to advise and support the study. The research team also includes experts in rare diseases, and academics who are experienced in undertaking this type of research. We want our research to be useful to a wide range of people, so we will hold workshops and events for professionals, people who commission services, patients and families who are interested in the care of people with rare diseases, so they can tell us how to make our findings helpful to them.

Research activity codes: 7.1 INDIVIDUAL CARE NEEDS;8.1 ORGANISATION AND DELIVERY OF SERVICES
Health categories: GENERIC HEALTH RELEVANCE