Our latest research, all in one place. Browse our collection of journal articles, reports and conference proceedings to see how we’re contributing to HEOR research. Remember to:
AIM: Late-onset Pompe disease (LOPD) is a rare lysosomal disease primarily impacting muscle strength andrespiratory function. LOPD has a substantial burden despite the availability of alglucosidase alfa (alg). Patients often require mobility and respiratory support over time. Cipaglucosidase alfa in combinationwith miglustat (cipa + mig) is one of two more recently approved treatments for adults with LOPD. Given limited data on the lifetime trajectory to mobility and respiratory support in LOPD, a patient-level simulation model was developed to compare the long-term impact of cipa + mig with alg on these outcomes.
MATERIALS & METHODS: The patient-level simulation predicts lifetime mobility and respiratorydisease progression outcomes based on the 6-min walk distance and % predicted forced vital capacity for alg and cipa + mig for the overall LOPD population using available data and assumptions from experienced clinicians. PROPEL/PROPEL open-label extension (NCT03729362) and ATB200-02 (NCT02675465) studies informed outcomes for four years with cipa + mig and one year with alg. French Pompe disease registry data were used thereafter.
RESULTS: Based on the available data and clinical assumptions, the model predicts cipa + mig slows the overall progression of LOPD, allowing patients an additional 2.72 years without mobility or respiratory support compared with alg. People receiving alg may be wheelchair dependent and require invasive respiratory support for an additional 2.57 and 1.55 years, respectively.
CONCLUSION: Cipa + mig may delay disease progression compared with alg over the lifetime of a patient with LOPD, which would increase the amount of time spent without mobility and respiratory support dependency.
BACKGROUND: Inflammatory bowel diseases (IBD), including ulcerative colitis (UC) and Crohn’s disease (CD), are chronic conditions affecting around 500,000 people in the UK and carries rising prevalence and substantial economic burden. Despite expanding therapeutic choice, contemporary UK cost analyses are scarce. A flexible cost of illness model was developed to estimate the excess costs associated with CDUC in the UK at a population level, including direct NHS costs and societal costs. The model estimates how the direct healthcare and indirect societal costs of IBD change over time, helping to analyse and manage key cost drivers. This supports planning and policy decisions by outlining how interventions and external influences affect long-term spending patterns.
METHODS: The study used public data, published research, and the 2023 IBD UK survey to create a cost model covering prevalence, diagnosis, management, complications, and mortality of CD and UC. Clinical management inputs included resource use associated with the ideal pathways in line with IBD Standards. This model estimated and compared healthcare use and complications in people with CD and UC to the general population over 15 years, including costs from flare-ups, remission, and societal impacts.
FINDINGS: The estimated total annual direct healthcare cost based on an optimal patient journey for CD and UC is £3 billion. Ongoing management makes up around 93% of this cost (£2.8 billion), of which 84% of this makes up remission-related treatment in people with active disease. The ongoing management costs of active disease is almost 3 times higher costs in remission, at £185,903,000 and £67,557,000 respectively. The annual lost productivity cost of CD and UC is estimated at around £0.8 billion, mostly due to inability to work. The total healthcare costs are £3.8 billion. In the absence of robust population based real-world data, and given the complexity of IBD care, this model offers a practical solution for estimating population costs, which are likely underestimated, and can be adapted as more accurate datasets become available.
INTERPRETATION: Although pharmacological treatment accounts for a substantial proportion of IBD expenditure, effective control of inflammation and maintenance of remission are likely critical for reducing downstream complications, hospitalisation, surgery, disability, and productivity losses.
The Health Technology Assessment International (HTAi) 2025 annual meeting featured three main
plenaries to explore next generation (NextGen) evidence in health technology assessment (HTA). In this
commentary we capture the discussions of Plenary Two: NextGen Methods: Hype or Here to Stay? Each
plenary panelist was tasked to convincingly debate the need, rigor, and implementability of one of three
emerging methods domains in HTA: 1) Environmental Sustainability, 2) Adaptive HTA, and 3) Artificial
Intelligence (AI)-enabled Real-World Evidence (RWE). The three panelists convincingly debated that their
method would endure beyond initial hype; all three methods were perceived to have a moderate to
high level of need, rigor, and implementability by the audience. Key questions from the audience
included a request for examples of where HTA reviews have considered environmental sustainability, a
challenge for adaptive HTA to embrace other value elements outside of cost-effectiveness, and asked
how the human-in-the-loop principle fits into AI-driven RWE and what this means for HTA agencies that
are already stretched for resources. In this commentary we summarize the presentations, discussions
and audience engagement to provide readers with accessible insight into the debate about which
method(s) are anticipated to endure beyond their initial hype.
Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra rarehaematological disorder. This study aimed to estimate the clinical burden, healthcareresource use (HCRU) and associated costs of cTTP in England using primary andsecondary care data. A retrospective cohort study was undertaken using the ClinicalPractice Research Datalink (Aurum) linked to Hospital Episode Statistics data. Thestudy period was from 01 January 2000 to 31 December 2019. Patients with cTTPwere identified using an algorithm and matched 1:2 to patients without thromboticthrombocytopenic purpura (TTP) (non-TTP cohort) to contextualise the findings. Atotal of 36 cTTP patients were identified. The mean follow-up for cTTP patients was4.4 and 5.7 years in primary and secondary care, respectively, and 4.9 and 5.8 yearsfor the non-TTP cohort. Of the 36 patients, 38.9% experienced an acute episodeand 19.4% experienced an organ damage event during follow-up. HCRU (includinginpatient admission, outpatient appointments and primary care appointments) washigher in the cTTP cohort relative to the non-TTP cohort. The average yearly cost ofhealthcare for a cTTP patient was £6155, relative to £858 in the non-TTP cohort. Thisstudy demonstrates the high clinical and economic burden of cTTP and providesevidence to inform healthcare planning.
BACKGROUND: The long-term use of strong opioids for chronic non-cancer pain puts people at risk of serious harm.
OBJECTIVES: To test the effectiveness and cost-effectiveness of a multicomponent intervention targeting opioid use for the treatment of chronic pain.
INTERVENTIONS: Participants were randomised 1 : 1 (using a minimisation programme stratified by geographical locality, baseline pain intensity score and baseline morphine equivalent dose) to either usual care (an educational booklet and relaxation compact disc) or usual care plus the I-WOTCH intervention; 3 day-long group sessions delivered by a nurse and lay facilitator, plus a one-to-one session and ongoing telephone contact from the nurse to support opioid tapering.
RESULTS: We randomised 608 people. At 12 months, there was no between-group difference in Patient-Reported Outcomes Measurement Information System Pain Interference Short Form (8A) scores; mean difference, −0.52 (95% confidence interval −1.94 to 0.89). At 12 months, 65/225 (29%) of people in the intervention group and 15/208 (7%) of people in usual-care group reported using no opioids [odds ratio 5.55 (95% confidence interval 2.80 to 10.99)], absolute difference, 21.7% (95% confidence interval 14.8 to 28.6). Over a lifetime horizon, I-WOTCH is on average associated with an incremental cost of £9277 per person, and provides an additional 0.314 quality-adjusted life-years. The deterministic incremental cost per quality-adjusted life-year gained was £29,543. The I-WOTCH intervention may be cost-effective compared to best usual care. The process evaluation suggested group support and shared experience were important to those trying to taper.
LIMITATIONS: The opioid use analysis is based solely on participant self-report. The findings only apply to people willing to consider opioid reduction and may not apply to a more complex secondary care population. The results may not be applicable to people using very high opioid doses.
CONCLUSIONS: The I-WOTCH intervention helps substantially more people stop opioids than best usual care without adversely affecting pain interference.
BACKGROUND: Modifiable risks—tobacco use, poor diet, alcohol consumption, physical inactivity and mental ill-health—drive substantial disease in UK. NICE evidence on preventive interventions targeting these risks and estimates of eligible populations and disease burden demonstrate the potential for prevention and reducing health inequalities.
METHODS: Deaths and DALYs from behavioural risks and mental health conditions were estimated using GBOD 2021 data. Prevalence by deprivation quintile came from national surveys. NICE guidelines were reviewed to identify cost effective preventive interventions.
RESULTS: In 2021, tobacco and dietary risks caused the highest mortality and disease burden: tobacco nearly 58 000 deaths and 1·4 million DALYs; diet 48 000 deaths and 1.05 million DALYs. Mental health had low mortality (5·7 deaths) but high DALYs (1·45 million), especially in younger people. Alcohol and inactivity had lower death counts and moderate DALY impacts. Most risks were more prevalent in deprived areas. NICE-recommended interventions such as smoking cessation, alcohol brief advice, obesity treatments, and psychological therapies, are relevant to millions, especially high-need groups.
CONCLUSION: (Re-)investing in prevention and equitable strategies could substantially reduce disease burden and address inequalities, with the greatest gains likely to arise from sustained and targeted investment in populations and areas that have been left behind.
Poor mental wellbeing is one of the leading causes of long-term sickness absence from work and may lead to absenteeism, presenteeism and staff turnover, costing UK employers an estimated £51 billion annually. This study uses economic modelling to provide data on costs and benefits to employers who are considering implementing a workplace intervention to improve wellbeing. Additionally, the analysis is used to assess any changes in employee outcomes (e.g., productivity and staff turnover). A cost-consequence model with a one-year time horizon was developed to assess the impact of workplace mental wellbeing interventions. Because all workplaces are different, it is not useful to present one single base case to generalise across all settings. Instead, the model generates a series of hypothetical case studies, with varying levels of absenteeism, presenteeism and staff turnover, as well as different levels of productivity and staff replacement costs. Several mental wellbeing interventions are compared with ‘no intervention’ (current practice) to calculate the total incremental costs and incremental cost per employee. In a hypothetical case study with 50 employees and an intervention cost of £100, the intervention has a net cost saving of £4,207 per employee. Savings are due to reductions in absenteeism and presenteeism. Sensitivity analysis and scenario analysis assess the impact of varying each input, to reflect that inputs will vary substantially for each individual organisation and setting. The intervention is more likely to be cost saving when the baseline levels of absenteeism, presenteeism and staff turnover are high, and the intervention cost is low. Mental wellbeing interventions may influence a range of outcomes, but outcomes demonstrating a mental wellbeing benefit to employees may be challenging to translate into monetary value. The model can be used by decision makers and employers to understand the potential economic and wellbeing implications of implementing workplace mental wellbeing interventions.
