Anaphylactic reactions can lead to life-threatening situations. Therefore, a rapid diagnosis and therapy are indicated. Various guidelines recommend immediate treatment with intramuscular adrenaline in severe anaphylaxis. Based on study data from different countries, it has been shown that therapy of anaphylaxis is often not carried out according to existing guidelines.

The aim of the study was an analysis of the emergency treatment and outcome of anaphylaxis in children and adults according to its severity. Focus was placed on the recommended first-line therapy with adrenaline in cases of severe reactions. Further demographic data, triggers, symptoms, and hospitalization rates of anaphylaxis were analyzed.

Data from Emergency Medical Services from Dresden/Germany in cases of anaphylaxis from the start of 2012 through the end of 2016 were retrospectively analyzed. The data of the air rescue were not considered. The severity of the anaphylaxis, the therapy, the further monitoring, and the outcome were analyzed.

A total of 1,131 adults and 223 children with anaphylactic reactions (Grade I-IV) were analyzed. Overall, 591 adults and 102 children showed a severe anaphylaxis. The most common trigger for severe anaphylactic reactions was food in children (61%) and medication in adults (33%). Seven percent of adults and eight percent of children with Grade II or higher anaphylactic reactions received adrenaline. There is a significant correlation between adrenaline therapy and improved condition/outcome in adults and children. Sixty-six percent of adults and 83% of children with severe anaphylaxis were hospitalized. Twenty-one percent of the adults and 13% of the children did not receive further medical observation despite a severe reaction.

The guideline-compliant first-line therapy with adrenaline was not carried out in the majority of the cases analyzed. However, the study shows that treatment with adrenaline for anaphylaxis leads to a significant improvement in the patients’ condition.

Nearly 3% of adults have attention-deficit and hyperactivity disorder (ADHD), although in the UK, most are undiagnosed. Adults with ADHD on average experience poorer educational and employment outcomes, worse physical and mental health and are more likely to die prematurely. No studies have yet used mortality data to examine the life expectancy deficit experienced by adults with diagnosed ADHD in the UK or worldwide.

This study used the life-table method to calculate the life-expectancy deficit for people with diagnosed ADHD using data from UK primary care.

A matched cohort study using prospectively collected primary care data (792 general practices, 9 561 450 people contributing eligible person-time from 2000–2019). We identified 30 039 people aged 18+ with diagnosed ADHD, plus a comparison group of 300 390 participants matched (1:10) by age, sex and primary care practice. We used Poisson regression to estimate age-specific mortality rates, and life tables to estimate life expectancy for people aged 18+ with diagnosed ADHD.

Around 0.32% of adults in the cohort had an ADHD diagnosis, ~1 in 9 of all adults with ADHD. Diagnoses of common physical and mental health conditions were more common in adults with diagnosed ADHD than the comparison group. The apparent reduction in life expectancy for adults with diagnosed ADHD relative to the general population was 6.78 years (95% CI: 4.50 to 9.11) for males, and 8.64 years (95% CI: 6.55 to 10.91) for females.

Adults with diagnosed ADHD are living shorter lives than they should. We believe that this is likely caused by modifiable risk factors and unmet support and treatment needs in terms of both ADHD and co-occurring mental and physical health conditions. This study included data from adults with diagnosed ADHD; the results may not generalise to the entire population of adults with ADHD, the vast majority of whom are undiagnosed.

Involving patients in the health technology assessment (HTA) lifecycle is a core principle at the National Institute for Health and Care Excellence (NICE). To achieve this, NICE has adopted a mixed approach to patient and public involvement and engagement (PPIE) spanning the entire appraisal process. To ensure the PPIE approach enables meaningful involvement, NICE engaged with stakeholders to review its effectiveness and identify areas for improvement.

In 2023, an independent consultant reviewed NICE’s PPIE approach and engaged with NICE staff and external stakeholders from patient organizations, individual patient contributors, and engagement leads at national health and social care organizations. The engagement included interviews with NICE staff (n=19) and external stakeholders (n=13), and an online survey that received 83 responses from patient organizations and patient contributors. Using this feedback, NICE’s patient and public involvement program conducted four focus groups to develop a framework of improved methods and processes for PPIE with NICE staff, patient organizations, and patient contributors.

The engagement identified many positives in NICE’s approach to PPIE, including:

• • lay members sitting on each HTA committee as equal members

• • patient organizations providing written evidence to HTA committees

• • patient experts providing written and verbal testimony to HTA committees

• • support provided by NICE.

The engagement also identified areas where PPIE could have a greater impact, including:

• • improved methods for collecting patient evidence and insight

• • strengthening the role of lay members

• • collating and reusing previously collected patient evidence

• • taking a proportionate approach to involving small organizations

• • allocating staff resources to focus on impactful PPIE practices.

NICE has developed a draft framework for an improved approach to increase the impact of PPIE in HTA decision-making. In 2024, NICE will publicly consult with NICE staff and external stakeholders to review the framework, agree the strategic aims, and develop metrics for measuring success. Following this consultation, the findings and NICE’s updated approach to PPIE will be presented.

The National Institute for Health and Care Excellence (NICE) heard from small organizations how resource intensive and difficult it is for them participate in medicines health technology assessments (HTA) since the COVID-19 pandemic. To provide additional support for these organizations or to provide alternative patient input, NICE explored implementing surveys directly with patients to share with patient stakeholder organizations and NICE’s HTA medicines committees.

Patient organizations and colleagues at NICE were included in the background investigation. Informal interviews were conducted with the HTA bodies in Wales and health technology colleagues in NICE about their experience of this method of patient input.

Two approaches were piloted:

(i) Developing the online questionnaire using the Summary of Information for Patients.

(ii) Developing a jointly branded questionnaire collaboratively with the patient organization and implementing a data-sharing agreement to share the raw data.

The survey was distributed by the patient organization, analyzed by NICE, and shared with the patient organization to inform their submission to NICE.

The results of the background investigation showed that the option to include this additional method of input could provide valuable support for patient organizations and has the potential to increase the amount and quality of patient input to an HTA committee.

Both pilots were successful in:

• • Supporting patient organizations’ input into a medicines HTA

• • Reducing the resources required from patient organizations.

• The second pilot added more value due to:

• • Collaboration, relationship, and building trust

• • Joint development of the survey

• • Data sharing and potential to add to patient evidence about a disease and treatments.

Surveys conducted directly with patients can help patient organizations participate in medicines HTAs, but they are only one element of developing more innovative and sustainable patient involvement in the process. HTA bodies need to innovate and work collaboratively with patient stakeholders to produce a menu of options for involvement so that it can be tailored to stakeholders’ resources.

Pemetrexed and immunotherapies (e.g., pembrolizumab) are approved for first-line maintenance (1LM) treatment of nonsquamous advanced/metastatic non-small-cell lung cancer (NSCLC), but real-world data on their use are limited. The objective of this study was to assess 1LM clinical outcomes, safety, and treatment patterns of immunotherapy versus immunotherapy+pemetrexed among patients with advanced/metastatic NSCLC from the EU4 (France, Germany, Italy, Spain)+UK.

Data from patients in the US, Canada, and EU4+UK with nonsquamous advanced/metastatic NSCLC without targetable mutations were collected via electronic case report form. Physician-identified patients (≥18 y) in the EU4+UK were eligible for this subgroup analysis if they achieved stable disease or complete or partial response with first-line platinum-based chemotherapy+immunotherapy (January 2019 to March 2021) and received 1LM immunotherapy or immunotherapy+pemetrexed. Patients were followed from index (1LM initiation) until last physician contact or death. Outcomes were overall survival (OS), progression-free survival (PFS), treatment patterns and duration, and adverse events.

Among the selected 367 patients (male, 71.9%; mean±StDev age, 63.4±7.2 y; current/former smokers, 85.8%), 203 (55.3%) received immunotherapies, most commonly pembrolizumab (n=173; 85.2%), and 164 (44.7%) received immunotherapy+pemetrexed. Patients receiving immunotherapy had longer median adjusted OS and PFS compared to those receiving immunotherapy+pemetrexed (OS hazard ratio [HR]: 0.63; 95% confidence interval [CI]: 0.36, 0.90; PFS HR: 0.58; 95% CI: 0.38, 0.79). Patients receiving immunotherapy versus patients receiving immunotherapy+pemetrexed had longer median treatment duration (14.0 vs 10.3 mo; p<0.001) and were less likely to experience anemia (19.7% vs 33.5%; p<0.01). Results were similar in the overall study population.

In this real-world study, among the selected patients with nonsquamous advanced/metastatic NSCLC who achieved stable disease or complete or partial response with first-line therapy, the addition of pemetrexed to immunotherapy in 1LM did not appear to confer a clinical benefit. Identifying treatments that can improve clinical outcomes for these patients remains an area of unmet need.

The roles and potential value of patient preference (PP) data in health technology assessment (HTA) remain to be fully realized despite an expanding literature and various efforts to establish their utility. This article reports lessons learned through a series of collaborative workshops with HTA representatives, organized by the Health Technology Assessment International’s Patient Preferences Project Subcommittee.

Five online workshops were conducted between June 2022 and June 2023, seeking to facilitate collaborative learning and reflection on ways that PP data can be integrated into HTA. Participants included nine HTA representatives from the United States, Canada, Australia, England, and the Netherlands. Workshops were recorded, transcribed, and thematically analyzed.

Despite appreciating the value of PP data, participants were ambivalent about their use in HTA. Some felt that they were already getting the information they needed from the cost-effectiveness analysis or existing patient involvement processes. Others thought that PP data would be very helpful at the initial and final stage of the decision-making process and, particularly, in the following cases: (a) when technology has important non-health benefits; (b) when the clinical and/or cost-effectiveness evidence is marginal; and (c) when treatment is indicated for a large and heterogeneous population. Issues related to the validity and reliability of PP studies were frequently raised, with preference heterogeneity at the core of these concerns.

Collaborating with HTA representatives in the “co-creation” of PP research can help address their concerns and facilitate mutual learning about how PP data can be used in HTA.

Autistic people have a high likelihood of developing mental health difficulties but a low chance of receiving effective mental healthcare. Therefore, there is a need to identify and examine strategies to improve mental healthcare for autistic people.

To identify strategies that have been implemented to improve access, experiences of care and mental health outcomes for autistic adults, and to examine evidence on their acceptability, feasibility and effectiveness.

A co-produced systematic review was conducted. MEDLINE, PsycINFO, CINHAL, medRxiv and PsyArXiv were searched. We included all study designs reporting acceptability or feasibility outcomes and empirical quantitative study designs reporting effectiveness outcomes. Data were synthesised using a narrative approach.

A total of 30 articles were identified. These included 16 studies of adapted mental health interventions, eight studies of service improvements and six studies of bespoke mental health interventions developed for autistic people. There was no conclusive evidence on effectiveness. However, most bespoke and adapted approaches appeared to be feasible and acceptable. Identified adaptations appeared to be acceptable and feasible, including increasing knowledge and detection of autism, providing environmental adjustments and communication accommodations, accommodating individual differences and modifying the structure and content of interventions.

Many identified strategies are feasible and acceptable, and can be readily implemented in services with the potential to make mental healthcare more suitable for autistic people, but important research gaps remain. Future research should address these and investigate a co-produced package of service improvement measures.

Autistic women are at high risk of developing restrictive eating disorders (REDs), such as anorexia nervosa.

This study provides an overview of the clinical characteristics of autistic women with REDs to (i) enhance understanding of increased risk, and (ii) support the identification of autistic women in eating disorder services.

We compared self-reported autistic and disordered eating characteristics of: autistic participants with REDs (Autism + REDs; n = 57); autistic participants without REDs (Autism; n = 69); and women with REDs who are not autistic (REDs; n = 80). We also included a group of women with high autistic traits (HATs) and REDs, but no formal autism diagnosis (HATs + REDs; n = 38).

Autism + REDs participants scored similarly to Autism participants in terms of autistic characteristics and to REDs participants in terms of experiencing traditional disordered eating symptoms. Autism + REDs participants were distinguished from both groups by having more restricted and repetitive behaviours and autism-specific eating behaviours related to sensory processing, flexibility and social differences. HATs + REDs participants showed a similar pattern of scores to Autism + REDs participants, and both also presented with high levels of co-occurring mental health difficulties, particularly social anxiety.

The presentation of autistic women with REDs is complex, including both traditional disordered eating symptoms and autism-related needs, as well as high levels of co-occurring mental health difficulties. In eating disorder services, the REDs presentation of autistic women and those with HATs should be formulated with reference to autism-specific eating behaviours and co-occurring difficulties. Treatment adaptations should be offered to accommodate autistic characteristics and related needs.

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.

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.

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.

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.