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PURPOSE: Qualitative accounts and studies employing disease-specific outcome measures have demonstrated that people with dystrophic epidermolysis bullosa (DEB) display impaired health-related quality of life (HRQoL) due to wound-related challenges. There is a paucity of utility data necessary to inform the health economic evaluation of emerging treatments for cutaneous open wounds.
METHODS: The objective was to implement a vignette study with a representative sample of the United Kingdom (UK) general population (N = 1139) and people with DEB (N = 44) to estimate utility values. Vignettes were defined by various attributes, including the number and area of wounds, pain, itch and hours spent managing wounds. Participants valued the vignettes from the perspective of a patient or a caregiver proxy using the EQ-5D-3L or EQ-5D-Y-3L, respectively.
RESULTS: The mean utilities elicited from the patient perspective ranged from 0.491 to − 0.448 and 0.457 to − 0.462 for the general population (N = 627) and DEB (N = 31) sample, respectively. The utilities elicited from the proxy perspective ranged from 0.443 to − 0.543 and 0.079 to − 0.550 in the general population (N = 512) and DEB (N = 13) sample, respectively.
CONCLUSION: The findings join a limited evidence base indicating that the symptoms associated with the wounds experienced by people with DEB have a considerable detrimental effect on their HRQoL.
AIMS: Asymptomatic severe aortic stenosis (aSAS) is associated with morbidity, with mortality increasing once symptoms develop. Benefits of early aortic valve replacement (AVR) over clinical surveillance (CS) with delayed AVR upon symptom onset have been demonstrated, and it is now recommended from the latest European Guidelines if procedural risk is low (Class IIa, Level A). The aim of this study is to estimate the economic impact of transcatheter aortic valve implantation (TAVI) for the treatment of aSAS.
METHODS AND RESULTS: A cost-utility analysis compared TAVI using the SAPIEN 3/SAPIEN 3 Ultra valve vs. CS in patients with aSAS across nine European countries. A lifetime Markov model captured peri-procedural and long-term outcomes across three health states: alive and well, stroke, and death. Inputs were derived from the EARLY TAVR trial (NCT03042104) and literature sources. Clinical event rates were estimated via parametric survival analysis. Transcatheter aortic valve implantation treatment for patients with aSAS was modelled to be the economically dominant strategy for treating aSAS across all nine European healthcare systems, with cost-effectiveness probabilities ranging from 97.3% (UK) to 99.9% (Belgium). Incremental results per person included cost savings from −£1788 (UK) to −CHF15 802 (Switzerland), quality-adjusted life years gains of 0.18 (Germany, UK) to 0.23 (Switzerland), and life-year gains of 0.12 (UK) to 0.17 (Belgium). Annual discounting of upfront TAVI costs vs. delayed AVR costs drove results.
CONCLUSION: For patients with aSAS, early intervention with TAVI is estimated to deliver greater health benefits and reduce costs compared with CS. These findings support policies promoting early detection and timely intervention before symptom onset.
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.
