BACKGROUND:Epidermolysis bullosa (EB) is a complex group of rare, inherited skin disorders that blister easily, imposing substantial burden on patients and carers. There is a lack of published evidence in the UK quantifying health-related quality of life in EB patients and carers as utility values suitable for use in Health Technology Assessment (HTA). METHODS:A time trade-off (TTO) study was conducted with 120 UK general public members to estimate utility values for EB patient and primary carer vignettes depicting varying EB severity. An exploratory sub-study involving six carer and clinical experts was conducted to estimate utility values of second carers. RESULTS:115 general public participants were included in the final TTO analysis. Mean utility values declined as disease severity increased, from 0.82 in the least severe state, to 0.54 in the most severe. Carer utilities were higher than the equivalent patient health states, but with a similar pattern of decreasing utility with increasing EB severity, ranging from 0.85 to 0.64. The secondary carer sub-study, in six respondents estimated a second care burden factor relative to the primary carer of 77%. CONCLUSIONS:The utilities estimated for both EB patients and their carers have relevance for future economic evaluations and HTA of new EB therapies.
Despite the need for immediate treatment during acute allergic reactions (AARs), many patients do not consistently carry medication. A new mouth-dissolving dexamethasone film offers a portable, easily administered alternative to corticosteroid tablets. This non-randomized, open-label, low-interventional real-world trial assessed satisfaction (accessibility and safety/security) with the mouth film versus betamethasone tablets in adults prescribed tablets for moderate to severe AARs at a Swedish primary care center. Over six months, participants had access to both treatments and reported monthly via an electronic diary; qualitative responses were converted to Likert scales where applicable. Of 50 enrolled, 44 provided diary data (mean age 50 years, 74% female, 58% had an epinephrine autoinjector). In total, 189 responses were collected: 98% stated that the participant was satisfied or very satisfied with mouth film accessibility versus 58% for tablets (mean Likert scores 3.5-3.7 vs. 2.5-2.8; p < 0.001). A higher feeling of safety with the mouth film versus tablets was reported in 78% of responses and improved medication carriage compliance in 77%. At six months, 69% preferred the film. Of 18 AARs in total reported by 13 participants, the mouth film was chosen for treatment of 16 (11 participants); in 94%, the mouth film was found immediately accessible, and in 94%, the efficacy was rated as good or very good. In conclusion, the mouth film improved perceived accessibility, safety/security, and medication-carriage compliance compared with tablets for the treatment of AARs.
BACKGROUND:The objective was to assess how preferences for a self-dissolving dexamethasone mouth film versus conventional tablets for acute allergic reactions may vary according to population characteristics and the level of outcome knowledge for the intervention (i.e., ex ante, interim, and ex post). METHODS:We used preference data from two studies: a questionnaire study with a choice between mouth film and tablets based on a description of the mouth film (ex ante), and a clinical trial with ratings of accessibility and perceived security when the mouth film was available (interim) or used for acute allergic reactions (ex post). Associations between preferences and participant characteristics (demographics and clinical history) were analyzed using logistic regression models. RESULTS:Preference for the mouth film over tablets remained strong as outcome information increased from ex ante to ex post and was consistent across study populations. Higher relative preference was associated with female sex, longer disease duration, older age, and prior severe reactions, whereas satisfaction with current treatment and regular allergy check-ups were linked to weaker relative preference. CONCLUSIONS:Preference for the mouth film over tablets was stable, but the degree of preference may depend on characteristics that influence sensitivity to treatment challenges in acute situations.
The introduction of Joint Clinical Assessments (JCAs) under the European Union Health Technology Assessment Regulation (EU HTAR) represents a major structural reform aimed at reducing fragmentation and duplication in clinical evidence assessment across Member States. While JCAs are intended to support national health technology assessment (HTA) processes through a common EU-level clinical evaluation, their integration into established national HTA and reimbursement systems remains untested at this early stage of implementation. This paper reports findings from two surveys conducted in 2025 among local affiliate representatives with expertise in relevant HTA and market access activities across 25 European countries (comprising 24 EU Member States and Norway), capturing early national perspectives on key aspects of JCA implementation. The surveys explored anticipated impact on national reimbursement timelines, current opportunities for early HTA advice and PICO input, approaches to handling post-JCA data availability, and nationally prioritised policy and implementation issues. Results indicate mixed expectations regarding the potential impact of JCA on reimbursement timelines, with perceived risks of delay largely viewed as possibly transitional and mitigable through national process adaptation. Respondents emphasised the importance of transparency in PICO consolidation and clarity on the use of JCA reports in national appraisals. Overall, the findings suggest that JCA may represent a reconfiguration rather than a centralisation of evidence assessment, with national influence exercised earlier in the assessment lifecycle. These insights provide policy-relevant input to inform ongoing EU HTA implementation and future refinement of the JCA framework.
This paper assesses the impact of uncertainty for investors on the economic valuation of medical innovation projects for orphan drugs or rare diseases. Conventionally, investor evaluation uses the deterministic discounted cash flow (DCF) method with an appropriate sensitivity analysis that captures some level of uncertainty. In healthcare, and particularly for rare diseases, the levels of uncertainty in financial outcomes (return on investment and net present value (NPV)) are broader than the ones normally captured by the DCF formula. Uncertainties include R&D costs, the approval process (level and timing) for obtaining reimbursement, sales, the production cost, and the failure probabilities of the clinical trial phases, to name a few. Additionally, there is not only one type of investor to consider, but different investors exposed to different levels of risk management of their investment. Our analysis tried to capture those two dilemmas (higher levels of uncertainty and different investor types) in two ways. One way was to identify a better method to enhance the different levels of uncertainty. The real option method of evaluation was proposed instead of DCF. For instance, the real option method better captures the uncertainty of the different phases of product development. The other way is to differentiate the investor types through their level of risk assessment perspectives. Small investors and start-up companies may see more benefit in applying the real option methodology to estimate their NPVs at different time points during product development. In summary, our evaluation identified various types of uncertainty when assessing an investment, along with methods to manage their effect on the economic/financial outcomes of medical innovations. Given the high uncertainty associated with early-stage drug development, such as orphan drugs for rare diseases, the real options approach is preferable to traditional DCF models. The analysis also showed that there is not just a single investor perspective to consider but specific perspectives that enhance the prime use of the real option methodology.
