Introduction: More adjuvant treatment options are becoming available for hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) early breast cancer (EBC) based on results of clinical trials. This study quantified the importance of different attributes of EBC adjuvant therapies to patients and the benefit-risk tradeoffs patients were willing to make. Methods: Women with HR+/HER2- EBC completed an online discrete choice experiment (DCE) survey; the design was informed by clinical data, qualitative interviews (n = 40), and pre-testing interviews (n = 40). Participants (pts) made 10 choices between pairs of hypothetical treatments described by varying levels of 6 attributes. DCE data were analyzed using a correlated mixed logit model. Relative attribute importance scores captured the impact of each attribute across clinically relevant ranges. Benefit-risk tradeoffs were captured as the minimum improvements in 5-year invasive disease-free survival (iDFS) that pts would require to tolerate increases in therapy-associated adverse event (AE) risks. Results: A total of 866 patients from the USA, France, Spain, Canada, the UK, Germany, South Korea, and Australia completed the DCE (mean age: 57.7 years; 76% postmenopausal; 29% stage I disease, 55% stage II, 16% stage III). Improved 5-year iDFS (75.4-82.7% range; associated with combination regimens [CRs] vs. endocrine therapy [ET] alone) contributed the most to treatment preferences (clinically relevant relative attribute importance: 38.4%), followed by reduced risks of venous thromboembolic events (VTEs) (20.4%), neutropenia (20.3%), and diarrhea (15.0%). Treatment type + duration (3.7%) and fatigue (2.3%) were less important. Pts required the largest improvement in 5-year iDFS (3.9%) to tolerate increased risks of VTE (0.7%-2.5%) or neutropenia (5.6%-46%); willingness to accept tradeoffs depended on the AE. Preference heterogeneity was observed across subgroups, but 5-year iDFS improvement was consistently the most impactful on treatment choice in all subgroups. Conclusion: A multicountry sample of patients most valued adjuvant therapies with higher 5-year iDFS and may therefore prefer CRs over ET alone. The value of CRs depends on their specific safety profiles, and shared decision-making should consider this to select treatment options that align with individual preferences.

AimsProlonged waits for hip and knee arthroplasty have raised questions about the equity of current approaches to waiting list prioritization for those awaiting surgery. We therefore set out to understand key stakeholder (patient and surgeon) preferences for the prioritization of patients awaiting such surgery, in order to guide future waiting list redesign.MethodsA combined qualitative/quantitative approach was used. This comprised a Delphi study to first inform which factors patients and surgeons designate as important for prioritization of patients on hip and knee arthroplasty waiting lists, followed by a discrete choice experiment (DCE) to determine how the factors should be weighed against each other. Coefficient values for each included DCE attribute were used to construct a ‘priority score’ (weighted benefit score) that could be used to rank individual patients waiting for surgery based on their respective characteristics.ResultsIn total, 43 people participated in the initial round of the Delphi study (16 patients and 27 surgeons), with a 91% completion rate across all three rounds. Overall, 73 surgeons completed the DCE. Following the final consensus meeting of the Delphi component, the seven final factors designated for inclusion were Pain, Mobility/Function, Activities of Daily Living, Inability to Work/Care, Length of Time Waited, Radiological Severity, and Mental Wellbeing. Output from the adjusted multinomial regression revealed radiological severity to be the most significant factor (coefficient 2.27 (SD 0.31); p < 0.001), followed by pain (coefficient 1.08 (SD 0.13); p < 0.001) and time waited (coefficient for one month additional wait 0.12 (SD 0.02); p < 0.001).ConclusionThese results present a new robust method for determining comparative priority for those on primary hip and knee hip arthroplasty waiting lists. Evaluation of potential implementation in clinical practice is now required.Cite this article: Bone Joint J 2025;107-B(1):89–96.

Aims Prolonged waits for hip and knee arthroplasty have raised questions about the equity of current approaches to waiting list prioritization for those awaiting surgery. We therefore set out to understand key stakeholder (patient and surgeon) preferences for the prioritization of patients awaiting such surgery, in order to guide future waiting list redesign. Methods A combined qualitative/quantitative approach was used. This comprised a Delphi study to first inform which factors patients and surgeons designate as important for prioritization of patients on hip and knee arthroplasty waiting lists, followed by a discrete choice experiment (DCE) to determine how the factors should be weighed against each other. Coefficient values for each included DCE attribute were used to construct a 'priority score' (weighted benefit score) that could be used to rank individual patients waiting for surgery based on their respective characteristics. Results In total, 43 people participated in the initial round of the Delphi study (16 patients and 27 surgeons), with a 91% completion rate across all three rounds. Overall, 73 surgeons completed the DCE. Following the final consensus meeting of the Delphi component, the seven final factors designated for inclusion were Pain, Mobility/Function, Activities of Daily Living, Inability to Work/Care, Length of Time Waited, Radiological Severity, and Mental Wellbeing. Output from the adjusted multinomial regression revealed radiological severity to be the most significant factor (coefficient 2.27 (SD 0.31); p < 0.001), followed by pain (coefficient 1.08 (SD 0.13); p < 0.001) and time waited (coefficient for one month additional wait 0.12 (SD 0.02); p < 0.001). Conclusion These results present a new robust method for determining comparative priority for those on primary hip and knee hip arthroplasty waiting lists. Evaluation of potential implementation in clinical practice is now required.

INTRODUCTION: Tyrosine kinase inhibitors (TKIs) enable most patients with chronic phase chronic myeloid leukaemia (CP-CML) to achieve near normal life expectancy. However, they are associated with a range of side effects that can impact treatment adherence and eventually, efficacy. Maintaining tolerability is essential for sustaining optimal outcomes. Understanding how people with CP-CML weigh efficacy against toxicity is critical to ensuring treatment decisions reflect what matters most to them. METHODS: This study quantified preferences using a discrete choice experiment (DCE). Adults with CP-CML are recruited by patient advocacy groups from three regions: Europe (United Kingdom, France, Germany, Italy, Spain), North America (United States, Canada) and East Asia (China, Japan). The online DCE survey included 12 choice tasks, each asking participants to choose between two unlabelled, hypothetical TKIs described by six attributes. The six attributes that patients highlighted as important in qualitative interviews, and that were supported by published clinical data on existing TKIs were selected for the DCE and captured aspects of efficacy, toxicity and convenience: 1) severity of fatigue, 2) risk of gastrointestinal (GI) problems, 3) risk of respiratory problems, 4) risk of long-term cardiovascular (CV) problems, 5) chance of achieving deep molecular response (DMR) within two years and 6) mode of administration (MOA; combination of frequency of tablets +/- fasting requirements). The attribute levels were informed by efficacy and safety outcomes from recent TKI trials. The choice data were analysed using conditional logit models, and relative attribute importance (RAI) scores were calculated to capture the impact of each attribute on overall preferences. Benefit-risk trade-offs were estimated by dividing each attribute-level coefficient by the (continuous) DMR coefficient, to assess the trade-offs that patients may be willing to make. This pooled multinational analysis is interim; final results based on the full study sample will be ready and presented at the conference. RESULTS: As of 28 July 2025, a total of 423 respondents completed the survey in Europe and North America (333 and 90, respectively) and were included in the analysis. Overall, 66% of respondents were female and approximately 28% of respondents were aged between 18 and 44 inclusive, with 25% aged 65 and above. 46% of respondents were on first line treatment and 14% were in treatment-free remission. The most to least important attributes, as indicated by the RAI scores (in parentheses) were severity of fatigue (25.9%), chance of achieving DMR within two years (20.9%), risk of GI problems (17.7%), risk of respiratory problems (12.5%), MOA (11.6%), and risk of long-term CV problems (11.3%). There were no statistically significant differences in RAI scores by region (Europe vs. North America). Benefit-risk trade-off estimates showed that, on average, respondents would need a TKI to offer a 30-percentage point (pp) higher chance of achieving DMR within two years to accept a 75% risk of GI problems (compared to a 15% risk). A 21pp increase would be required to accept a 40% risk of respiratory problems (vs. 5%) and a 20pp increase would be required to accept a 25% risk of long-term CV problems (vs. 5%).CONCLUSIONS: In this multinational preference study of people living with CP-CML, attributes reflecting common symptoms and side effects (e.g., fatigue and GI problems) had a substantial impact on (hypothetical) TKI choice. Risk of long-term CV problems was not as important a concern as other side effects, potentially reflecting patients' experiences with these issues and their desire to avoid them. The chance of achieving DMR was a highly important factor in choices, however, the results indicate that patients are willing to accept a lower chance of achieving DMR to avoid side effects. These results highlight the importance of informed and shared decision-making for CP-CML patients in the context of their treatment decisions.

OBJECTIVES:Women with an abdominal aortic aneurysm (AAA) are less likely to receive elective repair than men. This study explored the effect of patient sex and other attributes on vascular surgeons' decision-making for infrarenal AAA repair. DESIGN:Discrete choice experiment. SETTING:Simulated environment using case scenarios with varying patient attributes. PARTICIPANTS:Vascular surgeons. INTERVENTIONS:Surgical decision-making. MAIN OUTCOME MEASURES:AAA repair versus no repair and endovascular versus open repair. RESULTS:182 surgeons completed 2987 scenarios. When all other attributes were equal, a woman was more likely to be offered an AAA repair (marginal rate of substitution (MRS) 3.86 (95% CI 2.93, 4.79)), while very high anaesthetic risk (MRS -4.33 (95% CI -5.63, -3.03)) and hostile anatomy (MRS -3.28 (95% CI -4.55, -2.01)) were deterrents. Increasing age did not adversely affect the likelihood of offering repair to men but decreased the likelihood for women, which negated women's selection advantage from the age of 83 years. Women were also more likely to be offered endovascular repair (MRS 2.57 (95% CI 1.30, 3.84)). CONCLUSIONS:Patient sex alone did not account for real-world disparity observed in selection for surgery. Rather, being a woman was associated with a higher likelihood of being offered AAA repair but also a higher likelihood of being offered less invasive endovascular repair. Increased age decreased the likelihood of surgical selection for women but not men. Preference for less invasive repair, combined with inferior rates of anatomical suitability, and the comparably older age of women at the time of AAA repair selection may account for lower rates of repair for women observed.

The utility of genome-wide sequencing is often quantified in terms of its diagnostic yield. Although obtaining a diagnosis is a fundamental aspect of value, service users also value broader clinical, informational, process and psychological factors in the provision of genomic testing. This study aims to value genome-wide sequencing from the user perspective in Scotland. A survey was developed and administered to 1014 patients and families with experience of genome-wide sequencing to diagnose a rare condition in Scotland. Participants’ willingness to pay for genomic testing was elicited using a contingent valuation payment card. The survey included two genomic-related patient-reported outcome measures: (i) the Personal Utility Scale (PrU) to generate scores for the personal utility of genome-wide sequencing; and (ii) a subscale of the Feelings About Genomic Testing Results (FACTOR) questionnaire to measure negative psychological outcomes. Data were also collected on participants’ prior experiences of genomic testing services. A double-hurdle regression model investigated the predictors of patients' willingness to pay for genomic testing. Of the 1014 invitations sent, 171 contingent valuation questionnaires were returned. Diagnosed participants reported higher personal utility on PrU than undiagnosed participants. However, both groups reported similar negative psychological outcomes on FACTOR. Diagnosed participants were willing to pay £2043 for genome-wide sequencing, compared with £835 for undiagnosed participants. Diagnostic status, waiting time for results and FACTOR scores (negative psychological outcomes) influenced users’ valuations of genome-wide sequencing. Obtaining a diagnosis is a fundamental component of utility in the provision of genomic testing. However, there is still value to those who do not receive a diagnosis. These results have implications for service delivery, such as providing targeted pre-test and post-test genetic counselling, and investing in efficient genome sequencing pipelines to reduce waiting times. Valuing the user experience of genomic testing aligns with patient-centred approaches to the provision of healthcare.

Existing studies on adaptation to health shocks primarily focus on whether health and well-being return to baseline levels over time. However, little is known about the factors associated with the extent of adaptation. This study examines how patterns of time use across daily activities relate to the extent of adaptation. Focusing on women diagnosed with breast cancer, with women without a cancer diagnosis as a comparison, we apply an event study design to examine whether time spent on paid work and physical leisure are associated with varying degrees of adaptation, measured by self-reported health status and life satisfaction. Our findings suggest that full-time paid work and more than 10 h of physical leisure per week at baseline are associated with slower and less complete adaptation in the domain of self-reported health. Conversely, the degree of adaptation in the domain of life satisfaction does not significantly vary based on baseline levels of paid work and physical leisure. Furthermore, a reduction in time allocated to paid work or physical leisure following a cancer diagnosis is associated with slower adaptation in the domain of self-reported health compared to maintaining pre-diagnosis levels. These findings suggest an interplay between pre- and post-diagnosis time allocation that warrants further investigation.

BackgroundNext generation sequencing (NGS) can decrease the diagnostic odyssey for patients with rare diseases. However, valuing the combination of health and non-health outcomes associated with NGS is challenging. While stated preference methods can be used for monetary valuation of outcomes, frameworks that jointly account for both costs and benefits to determine cost-acceptability are limited. Insights into cost-acceptability can help inform pricing and access decisions where competition among NGS alternatives is imperfect or diagnostics are provided as a public good.MethodsWe used stated preference data to estimate the cost-acceptability of exome sequencing (ES) (i.e., cost at which ES provides value and users are willing-to-pay) in a user-based valuation. We estimated the benefit of ES as an alternative to all other diagnostic tests using a compensating variation model. Based on estimated net-benefit, we determined the proportion of users with positive expected net-benefit for varying cost (CAD$0-$15,000) and chance of diagnosis (10%-90%). We created a cost-acceptability frontier of costs and chance of diagnosis for a range of scenarios. Expected net-benefit and cost-acceptability were estimated for low-cost (CAD$1,600) and high-cost (CAD$11,660) ES scenarios.ResultsWe find that at least half of users consider costs of up to CAD$10,000 acceptable if the chance of obtaining a diagnosis of ES is at least 50%. However, at least some users are willing to accept a chance of diagnosis below 50%, even if the associated cost are high.ConclusionOur proposed valuation framework suggests that many potential users of ES are willing to accept various combinations of cost and chance of diagnosis. Cost-acceptability is especially high if the chance of diagnosis is larger the 50%.

Abstract Background A rare disease affects fewer than 5 in 10,000 people. More than 6,000 known rare diseases affect up to 6% of the European population. A national rare disease strategy sets out a country’s plan to reduce disease burden and improve quality of care for people with rare diseases. This review, requested by the Department of Health in Ireland to inform a new national strategy, described national rare disease strategies in 13 selected countries. Methods Strategies published from 2013 to 2023 were identified via online searches for Austria, Australia, Denmark, England, Finland, France, Germany, Ireland, the Netherlands, Northern Ireland, Portugal, Scotland and Wales. National representatives were contacted to confirm the identified resources. Data were extracted for the domains of: aims, priorities, implementation, governance and funding models. Descriptive analysis and narrative synthesis was undertaken. Results Improving treatment and coordination of care was a common overall aim. Priorities noted in all strategies were: screening and diagnosis; access to healthcare and coordination of services; rare disease research; and patient representation and empowerment. Implementation details varied to reflect the national context and time of strategy development. More recently-developed strategies included more precise actions and greater emphasis on innovations in genomics than older strategies. Strategies were developed, implemented, monitored and evaluated via combinations of newly-established bodies and or existing health authorities. Six countries did not specify a funding model or dedicated budget for strategy implementation. Conclusions National rare disease strategy contents and implementation approaches varied between countries and reflected innovations over time. This review will inform the development of Ireland’s new national rare disease strategy. Key messages • This review provided insights into national rare disease strategies over a 10-year period. • The findings will inform strategy development in Ireland, and are relevant to other countries.

Introduction and aimDiabetes is a global health emergency with increasing prevalence and diabetes-associated morbidity and mortality. One of the challenges in optimising diabetes care is translating research advances in this heterogeneous disease into clinical care. A potential solution is the introduction of precision medicine approaches into diabetes care.We aim to develop a digital platform called ‘intelligent Diabetes’ (iDiabetes) to support a precision diabetes care model in Scotland and assess its impact on the primary composite outcome of all-cause mortality, hospitalisation rate, renal function decline and glycaemic control.Methods and analysisThe impact of iDiabetes will be evaluated through a cluster-randomised controlled study, recruiting up to 22 500 patients with diabetes. Primary care general practices (GPs) in the National Health Service (NHS) Scotland Tayside Health Board are the units (clusters) of randomisation. Each primary care GP will form one cluster (approximately 400 patients per cluster), with up to 60 clusters recruited. Randomisation will be toiDiabetes (guideline support),iDiabetesPlusorusual diabetes care(control arm). Patients of participating primary care GPs are automatically enrolled on the study when they attend for their annual diabetes screening or are newly diagnosed with diabetes. A composite hierarchical primary outcome, evaluated using Win-Ratio statistical methodology, will consist of (1) all-cause mortality, (2) all-cause hospitalisation rate, (3) proportion with >40% estimated glomerular filtration rate [eGFR] reduction from baseline or new development of end-stage renal disease, (4) proportion with absolute HbA1C reduction >0.5%. Outcomes will be evaluated after a 2-year median follow-up period. Comprehensive qualitative and health economic analyses will be conducted, assessing the cost-effectiveness, budget impact and user acceptability of the iDiabetes platform.Ethics and disseminationThis study was reviewed by the NHS Health Research Authority and approved by the East of Scotland Research Ethics Committee (reference: 23/ES/0008). Study findings will be disseminated via publications, presented at scientific conferences and shared with patients and the public on the study website and social media.Trial registration numberISRCTN18000901.

Abstract Background The COVID-19 pandemic highlighted the need to improve preparedness for emerging health threats. This includes reserving assets for use in health emergencies, such as, shelters, energy supply items, and a stockpile of medical countermeasures (MCMs). Learnings from the recent pandemic are key to improving future strategies for preparedness in Ireland and across Europe. Methods Semi-structured interviews were conducted with key representatives in France, Latvia, Lithuania, the Netherlands, and Norway. These countries were chosen as they have a similar threat profile to Ireland. The interviews aimed to understand the national approach taken in each country to stockpiling of MCMs for public health emergencies. Interview summaries were developed and reviewed by key representatives for clarity. Thematic analysis was conducted. Results All of the key representatives interviewed were working within their associated Department of Health, or a government affiliated public health agency. Themes and key findings identified included scope and current stockpiling approaches; cost considerations and efficiency, and an understanding of how stockpiling approaches have evolved overtime. While some countries stockpiled items such as facemasks, gloves and ventilators during the COVID-19 pandemic, this is being reviewed, due to the high costs involved. Additionally, all countries reported that waste due to unused stock was a challenge, however, the use of stock rotation can reduce stock waste. Choice of supplier is also key for security of supply. Using indigenous suppliers can facilitate a scale up of supply in times of crises. Conclusions National MCMs stockpiles are a key resource that may be deployed as part of a response to health emergencies and disasters. Reducing waste was a key priority and challenge for all countries. Measures identified to reduce waste in future included active management of stockpiles, stock rotation and the use of indigenous suppliers. Key messages • As a result of the COVID-19 pandemic, many countries are reviewing their current approach to stockpiling. • Key issues for countries included preventing waste and ensuring security of MCM supply.

Abstract Issue In 2023, in Ireland, a public health policy research team was established in the Health Information and Quality Authority to conduct evidence syntheses and provide evidence-based advice to inform national decision-making, at the request of the Department of Health. One year on, we reflect on the impact of this team and outline lessons learned. These findings may support other countries in creating and developing public health policy research teams. Description of the problem We aim to answer the following questions: What impact can a public health policy research team have and what are the lessons learned, one year on? Projects completed by the team in 2023 were reviewed for stakeholder engagement (such as expert and public and patient involvement) and impact (feedback from policymakers, resulting public health actions or policies and online report downloads). Results Seven public health projects were completed in 2023, in areas including: Long COVID, public health reform, rapid multiplex testing, and medical countermeasure stockpiling. National and international stakeholders were engaged with via expert advisory group membership, document review and project presentations. Patient representatives were included from Long COVID Advocacy Ireland and Rare Diseases Ireland. Project reports were downloaded a total of 281 times. Positive feedback was received from all stakeholders. Project findings were presented to policymakers to inform national public health and rare disease strategy development, and the Long COVID model of care in Ireland. Lessons Early and ongoing stakeholder engagement is key when translating research to practice, via policy. Innovative methods of engagement, including public and patient involvement, can ensure meaningful collaboration. Adapting methodologies to meet stakeholder needs, such as rapid evidence synthesis, enhances impact. Lastly, research impact on policy takes time, and an understanding of national policy development is required. Key messages • A public health policy research team provides impactful evidence synthesis to advise national policymakers. • Stakeholder engagement is key to developing impactful policy advice.

Introduction Climate change poses a major threat to our health, livelihoods and the planet. In 2020, the UK National Health Service (NHS) committed to reducing its Scope 1, 2 and 3 emissions to reach net zero by 2045. Although a net zero NHS would help to limit the consequences of climate change, little is known about the UK general public’s values and preferences for the proposed service changes needed to reach net zero.Methods This study will elicit the public’s preferences for actions to help achieve net zero NHS in England and Scotland using a discrete choice experiment (DCE). The DCE attributes and levels describe actions that can be taken by the NHS across key areas: buildings and estates, outdoor space, travel and transport, provision of care, goods and services and food and catering. The survey was designed using online think-aloud interviews with 17 members of the public. Two versions of the survey will be administered to a sample of up to 2200 respondents. One will include a payment vehicle as income tax increases. We will estimate the relative importance of each attribute and, for the former survey, the monetary trade-offs which individuals are willing to make between attributes. Where possible, we will match both samples to gauge preference robustness with the inclusion of the monetary payment. We will test whether respondents’ preferences differ based on their socioeconomic circumstances and attitudes toward the NHS and climate change.Ethics and dissemination The University of Aberdeen’s School of Medicine, Medical Sciences and Nutrition Ethics Research Board has approved the study (reference: SERB/690090). All participants will provide informed consent. Results will be submitted to peer-reviewed publications and presented at relevant conferences and seminars. A lay summary of the research will be published on the Health Economics Research Unit website.

OBJECTIVES:While patient input to health technology assessment (HTA) has traditionally been of a qualitative nature, there is increasing interest to integrate quantitative evidence from patient preference studies into HTA decision making. Preference data can be used to generate disease-specific health utility data. We generated a health utility score for patients with chronic obstructive pulmonary disease (COPD) and consider its use within HTAs. METHODS:Based on qualitative research, six symptoms were identified as important to COPD patients: shortness of breath, exacerbations, chronic cough, mucus secretion, sleep disturbance, and urinary incontinence. We employed a discrete choice experiment (DCE) and the random parameter logistic regression technique to estimate utility scores for all COPD health states. The relationship between patients' COPD health utility scores, self-perceived COPD severity, and EQ-5D-3L utility scores was analyzed, with data stratified according to disease severity and comorbidity subgroups. RESULTS:The COPD health utility score had face validity, with utility scores negatively correlated with patients' self-perceived COPD severity. The correlation between the COPD health utility scores and EQ-5D-3L values was only moderate. While patient EQ-5D-3L scores were impacted by comorbidities, the COPD health utility score was less impacted by comorbid conditions. CONCLUSIONS:Our COPD utility measure, derived from a DCE, provides a patient-centered health utility score and is more sensitive to the COPD health of the individual and less sensitive to other comorbidities. This disease-specific instrument should be considered alongside generic health-related quality of life instruments when valuing new COPD therapies in submissions to licensing and reimbursement agencies.

Women’s preferences for time allocation reveal how they would like to prioritise market work, family life, and other competing activities. Whilst preferences may not always directly translate to behaviour, they are an important determinant of intention to act. We present the first study to apply a discrete choice experiment (DCE) to investigate time allocation preferences among women diagnosed with breast cancer and women without a cancer diagnosis. Time attributes were paid work, household work, caregiving, passive leisure and physical leisure. An income attribute was included to estimate the monetary value of time. The study took place in the UK and the DCE was completed by 191 women diagnosed with breast cancer and 347 women without a cancer diagnosis. Responses were analysed using a mixed logit model. Women diagnosed with breast cancer have stronger positive preferences for daily activities compared to women without a cancer diagnosis. They require less compensation (not significant) for an additional hour of paid work (£5.58), household work (£7.92), and caregiving (£8.53). They are willing to pay more for an additional hour of passive leisure (£1.70, not significant) and physical leisure (£13.66, significant). The heterogeneous preferences for time allocation among women have policy implications and are significant for welfare analysis.

Abstract Background Respiratory syncytial virus (RSV) can cause severe acute respiratory tract infection in infants and older adults. However, until recently, no immunisation interventions were available to reduce the burden of RSV disease in the general population. New health technologies have now been authorised in Europe for infants (a long-acting monoclonal antibody (nirsevimab) and a maternal vaccine (RSVpreF)), and two vaccines for adults aged ≥60 years (RSVPreF3 and RSVpreF). A rapid health technology assessment (HTA) was requested by the Irish Department of Health to support an interim decision on RSV immunisation. Methods The rapid HTA commenced in January 2024, and comprised description of technology, a summary of international practice, national and international epidemiology and burden of disease, and a costing analysis. The summary of international practice (including underpinning evidence) was undertaken rather than a systematic review of effectiveness and safety due to the rapidly changing evidence base and short timeline within which information was required. Results While new recommendations regarding immunisation against RSV are emerging internationally in response to recently authorised technologies, implementation and funding strategies are not currently widespread. Compared with a full HTA, a rapid HTA enabled an efficient and flexible approach to producing a high quality evidence synthesis to inform a temporary policy decision, taking approximately 6 months to complete rather than 12 months or longer. A full HTA is planned to inform long-term decision-making on this topic, but would not have been feasible to inform an interim decision. Conclusions This rapid HTA will inform a temporary decision on immunisation policy for a single RSV season in Ireland. Conducting a rapid HTA prior to a full HTA has enabled timely decision-making, with implications for Ireland’s national healthcare system and people at risk of severe RSV-related disease. Key messages • Innovations in immunisation against RSV are leading to changes in policy across Europe. • Rapid HTA can support timely, evidence-based public health decision-making in a fast-evolving field.

Aims This study aims to evaluate the cost effectiveness of genetic and genomic testing strategies for the diagnosis of rare developmental disorders in NHS Scotland. Methods Six genetic and genomic testing strategies were evaluated using a decision tree model. First-line, second-line and last-resort trio genome sequencing (GS), and second-line and last-resort trio exome sequencing (ES) were compared with standard genetic testing. The cost effectiveness of each strategy was expressed in terms of incremental cost per additional diagnosis. The impact of uncertainty on cost-effectiveness results was explored using deterministic and probabilistic sensitivity analysis. Results 2nd-line ES was a cost-saving option, increasing diagnostic yield by 13.9% and decreasing cost by £1027 per trio compared to standard genetic testing. Compared to ES, strategies involving GS increased costs significantly, with only a moderate or zero improvement in diagnostic yield. Sensitivity analysis indicated that significant reductions in cost or improvements in diagnostic yield are required before 1st-line GS becomes cost effective. Conclusion 2nd-line ES (after chromosomal microarray; replacing gene panel testing) for the diagnosis of developmental disorders is a cost-saving option for the Scottish NHS. Ongoing economic evaluation is required to monitor the evolving cost and diagnostic yield of GS and ES over time.