Title: Cost-effectiveness of osteoporotic fracture risk assessment in people with intellectual disabilities
Funder: Department of Health and Social Care

Abstract: Research question: What is the most cost-effective method for osteoporotic fracture risk assessment in people with intellectual disabilities? Background Our originating study &ldquo;Fractures in people with intellectual disabilities: comparison with the general population and development of a fracture risk calculator specific to these patients&rdquo; (PB-PG-1216-20017), included all patients with intellectual disabilities [ID] (n= 43,176) and five age and gender matched controls without ID per patient in the Clinical Practice Research Datalink (CPRD) linked to the Hospital Episode Statistics (HES) database (1998-2017). It included the largest cohort of people with Down s syndrome ever studied (n= 4,119). Unpublished results showed a higher incidence rate of major osteoporotic (MOP) fractures (vertebra, shoulder, wrist, hip) and of hip fracture in adults with ID. The difference is outstanding for hip fracture, for which incidence rate ratios (95%CI), between subjects with and without ID varied between 2.3 (1.9-2.7) and 7.8 (4.1-14.6) according to age and gender. We developed a fracture risk score (IDFracture) estimating the 10-year risk of hip, MOP or any fracture for adults with ID 30-79 years old. Aim To determine the most cost-effective risk assessment method for osteoporotic fractures. Short-term objective To reach a policy change in assessing osteoporotic fracture risk. Long-term objective To reduce the risk of osteoporotic fractures. Methods We will validate the IDFracture risk scores on the Aurum database (from 23 million people) with full linkage to the HES dataset. For the cost-effectiveness analyses we will use the subset of people aged 40-79 years from the Aurum cohort. We will use a Markov model with an annual transition cycle projecting life-long incidence of fractures and death. To estimate cost-effectiveness, the model will be run assuming several strategies: Current strategy, i.e. NICE and National Osteoporosis Guidelines Group, using QFracture for risk score calculation Risk assessment by IDFracture in all patients at age 40 years at their yearly review, with bone mineral density scan (DXA) in those between predefined age-related risk thresholds. To perform DXA in all patients at age 40 (follow up according to result). For each of these strategies, total lifetime costs and outcomes plus incremental cost-effectiveness ratio (ICER) will be calculated against the next most effective strategy. The main analyses will be done from the NHS perspective. The impact of fracture on health-related quality of life will be taken from the literature. Timeline Months 1-5: regulatory approval, data management, model validation, calculation of risk score algorithms Months 3-11 Cost effectiveness analyses Months 11-12 Final report/ manuscripts writing Anticipated Impact and Dissemination We will use our links to national bodies: the National Institute for Health and Care Excellence (NICE), the Royal Colleges of Physicians, of Psychiatrists, of General Practitioners, the British Medical Association, the Society for Academic Primary Care, the Royal Mencap Society, the Down s Syndrome Association, the Royal Osteoporosis Society, the Society for Endocrinology, the Bone Research Society. The latter three societies are part of the NICE accredited National Osteoporosis Guideline Group. All this will guarantee widespread dissemination of results, and lead to rapid clinical impact.

Lay abstract: People with intellectual disability (ID) are at higher risk of fractures, particularly of the hip. These are devastating events causing persistent disability for the individual, extra work for carers, and a high cost to health and social care services. Such fractures are often preventable, and are due largely to osteoporosis (thinning of the bones), a very treatable condition. Osteoporosis occurs earlier in the life of a person with ID, at ages when bones would be stronger in the general population, and is particularly frequent in people with Down's syndrome. People with ID do not generally have their fracture risk assessed, and when they do, standard risk scores are poorly tailored to this population. Their risk therefore goes unrecognised, and few are offered the opportunity to prevent fractures. Through this study we aim to identify the most effective strategy for assessing risk of fracture and offering preventive treatments in people with ID. Treatments include bone protecting medication, vitamin D supplements, changes to drug therapy, and lifestyle measures. We have developed a new score for assessing fracture risk, IDFracture, specifically tailored to the ID population. People with ID have an annual health check at their general practice, which does not currently include an estimate of bone health and fracture risk. This health check would be a good opportunity to discuss the issue with the person and their carers, calculate their risk using the IDFracture score, and where needed arrange to have the bone density measured through a DEXA scan. At present, we do not know which of several possible strategies would be the most cost-effective and acceptable for individuals. Using information already published in the medical literature, a health economist will estimate the benefits and costs of a number of strategies. These will include regular risk assessments, followed by DEXA scan in those above a range of risk levels and treatment of osteoporosis according to current guidelines. We will include the option of arranging DEXA in everyone over 40 years (irrespective of the risk score). We will take account of the benefits, costs, inconvenience, possible adverse effects, and impact on quality of life of each strategy. This will result in policy recommendations that could be implemented immediately. Our research is supported by the Royal Osteoporosis Society, the Down's Syndrome Association, and Mencap. A group of patient and public representatives advises us and one of them is a co-applicant. We will disseminate our findings and the policy recommendations through scientific manuscripts and to all relevant national bodies including the National Institute for Health and Care Excellence (NICE), the Department of Health and Social Care, through the Royal Colleges of General Practitioners, Physicians and Psychiatrists, and through the organisations supporting our research.

Research activity codes: 2.5 RESEARCH DESIGN AND METHODOLOGIES (AETIOLOGY);8.2 HEALTH AND WELFARE ECONOMICS
Health categories: MUSCULOSKELETAL;MENTAL HEALTH