Randomized controlled trials are ideal for securing marketing authorization (MA) for pharmaceuticals. However, ethical and feasibility constraints have led to the use of noncomparative trials by regulatory bodies to balance evidence uncertainty and patient access, especially for medicines addressing rare diseases or unmet healthcare needs. This research focused on pharmaceuticals approved with noncomparative data and how these translate into reimbursement outcomes.

Indications approved by the European Medicines Agency (EMA) between January 2018 and October 2023 based on noncomparative data were identified. Generics and biosimilars were excluded. Approvals based on non-comparative trial data were identified from European public assessment reports and from “Procedural steps taken and scientific information after authorization” documents. The latter were also used to identify changes in MAs after submission of new data. Missing trial information was extracted from ClinicalTrials.gov. Health technology assessment (HTA) outcomes from the EU4Health programme and the UK were extracted from agency websites.

The EMA approved 46 indications based on noncomparative data: 18 of the 46 (39%) received full MA, 23 (50%) received conditional MA (CMA), and five (11%) had a CMA converted to full MA with additional data. The results for HTAs conducted in various countries were as follows:

• • England: 29 of 46 (63%) indications assessed, (23/29 [79%] recommended);

• • Scotland: 28 of 46 (61%) indications assessed, (20/28 [71%] recommended);

• • France: 33 of 46 (72%) indications assessed, (7/33 [21%] received Amélioration du Service Médical Rendu level I to III);

• • Germany: 37 of 46 (80%) indications assessed, (2/37 [5%] achieved additional benefit);

• • Italy: 37 of 46 (80%) products assessed, (33/37 [89%] reimbursed); and

• • Spain: 34 of 46 (74%) products assessed, (23/34 [68%] reimbursed).

Indications approved by the EMA with noncomparative data achieved mixed HTA outcomes in the EU4Health programme and the UK. More negative outcomes were seen in markets with clinical-benefit payer archetypes (France and Germany). All conversions from CMA to full MA occurred within the last 24 months.

In 2022, a group of health technology assessment (HTA) bodies from Australia, Canada, and the UK announced a collaboration to identify solutions to common challenges. This collaboration was later expanded to include agencies from New Zealand and Quebec, Canada. Since one possible activity of the consortium is joint assessments, we compared the methodologies of the agencies on 11 topics to assess the feasibility of this.

We reviewed the methodological guidelines of the Canadian Agency for Drugs and Technologies in Health (CADTH), L’Institut national d’excellence en santé et services sociaux (INESSS), the National Institute for Health and Care Excellence (NICE), the Pharmaceutical Benefits Advisory Committee (PBAC), the Pharmaceutical Management Agency (Pharmac), and the Scottish Medicines Consortium (SMC). The topics considered were real-world evidence, consideration of health effects, economic reference case, survival analysis, surrogate endpoints, patient involvement, uncertainty, orphan pathways, clinical evidence requirements, carer perspective, and decision modifiers. We analyzed the level of alignment across the collaborating agencies using information from the guidelines, supplemented by published literature where necessary.

Three topics exhibited high alignment: consideration of health effects, clinical evidence requirements and surrogate endpoints. The topics of orphan pathways and carer perspective had low alignment. The remaining topics had moderate alignment. Regarding orphan pathways, NICE and the SMC had separate processes for ultra-orphan drugs, CADTH and INESSS implicitly consider rarity, and PBAC and Pharmac do not appear to consider rarity. Since carer perspective is not commonly accepted in HTA, NICE was the only agency with relevant guidance on this topic. INESSS required the societal perspective as standard, while the PBAC and Pharmac explicitly excluded it. CADTH may consider carer perspective in some circumstances, whereas the SMC guidance was ambiguous.

While there is good alignment on most topics, there are several areas where agencies would need to resolve divergences in preferred methodology if joint assessments are going to be carried out in the future. All relevant stakeholders should be part of this process, including patient groups and industry.

Reimbursement schemes should be regularly updated to maintain a trade-off between costs for the system and access to medicines. The aim of this study was to review reimbursement systems in several European countries in terms of solutions that could have a positive impact on the health technology assessment (HTA) and reimbursement processes in Poland (e.g., increasing patient access to medicines while maintaining payers’ spending).

Secondary publications and the websites of key institutions responsible for drug policy (the Ministry of Health, HTA agencies, and payers) in selected European countries were searched to find unique solutions that could have a positive impact on drug policies in Poland. The keywords used were “drug reimbursement” and “HTA”. A more specific search was conducted as needed.

The following solutions considered worthy of further consideration:

• • central organization of tenders for hospital drugs and conducting price negotiations with regional financial responsibility;

• • determining the maximum annual copayment per patient for reimbursed drugs;

• • complementary private health insurance that reduces patient copayments;

• • creating a separate path for hybrid drugs in the HTA and pricing processes;

• • increasing the number of consultations with the market authorization holder, clinical experts, and the public during drug evaluation; and

• • not accounting for the costs of lost productivity during HTA due to the discrimination of seniors and children.

Minor changes in the HTA process, such as increasing the role of consultations, as well as major systemic changes (e.g., introducing complementary private insurance, creating a separate path for hybrid drugs, and introducing a maximum annual copayment for reimbursed drugs) could improve patients’ access to drugs. Implementing these solutions requires significant adaptation of the local legal framework.

Access to innovative and expensive medicines is a significant challenge for Poland’s healthcare system. These therapies often do not meet the reimbursement criterion that is currently set at three times the gross domestic product per capita. Nevertheless, there are ongoing efforts to identify funds that can cover the cost of innovative and expensive medicines.

A new legal act established the Medical Fund, which is an addition to the regular National Health Fund. The Medical Fund finances medical technologies recognized by the Polish healthcare system as highly innovative or of high clinical value. Lists of such therapies are prepared by the health technology assessment agency, in consultation with clinical experts, and then approved by the Ministry of Health. Simplified marketing authorization applications based on a budget impact analysis, a more straightforward assessment process, and a separate budget may allow patients to access these therapies faster.

Since January 2022, ten highly innovative therapies have been funded for patients with conditions such as spinal muscular atrophy, acute hepatic porphyria, and primary hyperoxaluria. The reimbursement decisions were issued for a two-year period, during which data on treatment efficacy were collected. If the data collected after two years is insufficient to assess the treatment’s efficacy, the decision can be extended without an additional procedure for another two years. After two or four years, the marketing authorization holder must submit a reimbursement application based on a full health technology assessment report. Risk-sharing schemes based on clinical outcomes are mandatory.

The Medical Fund has granted early access to modern therapies. The decision to continue funding for a particular drug depends on whether registry results confirm data from experimental studies.

The decision-making process in public administration can be analyzed from different perspectives. Evidence-based policies are not the only support for public policy design. High variability was observed regarding the health technologies used during pandemics. The processes and results of decision-making on health technologies in 25 health ministries in Argentina during the COVID-19 pandemic were analyzed.

This retrospective study utilized triangulation of quantitative and qualitative methods. Information was retrieved for the years 2020 to 2021 through document review of official web pages, surveys, and interviews with decision-makers and advisors of the national ministry of health and 24 Argentinian provinces. Recommendations and reimbursement policies for seven health technologies were considered as tracers. Official health technology assessment (HTA) reports, laws, judicial rulings, journalistic news, and civil society actions on social networks and the internet regarding these technologies were analyzed. The ruling political party in each province was mapped, with the aim of exploring the political influences and intervening actors.

Contrary to World Health Organization (WHO) recommendations, ivermectin, inhaled ibuprofen, convalescent plasma, and equine serum were widely recommended and reimbursed outside a clinical trial context by most of the Argentinian ministries of health, leading to risks for patients and a huge opportunity cost. Health ministries with institutionalized HTA units had significantly higher adherence to WHO recommendations than other health ministries. Legislative and judicial powers influenced the use of health technologies through laws and judicial rulings. Researchers and civil society also influenced decision-makers. Partisan political issues did not fully explain the heterogeneity of the decisions made by the health ministries during the pandemic.

The impact of HTA organizations and their technical reports was limited. Health Ministries with institutionalized HTA units were more likely to adhere to WHO recommendations. The influence of different technical and political criteria, power relations within and outside the administrations, the pharmaceutical industry and academics, the media, social pressure, judicial and legislative powers, and the political context strongly influenced decision-making.

The World Health Organization (WHO) recommends population-based cancer screening programs for colorectal cancer (CRC). However, CRC screening is not available in Chile´s public health system. The Arturo Lopez Perez Foundation (FALP) is interested in implementing a CRC screening program for their insurance beneficiaries, despite some uncertainties regarding cost effectiveness and budget impact. Exploring co-financing scenarios could reveal feasible choices for program implementation.

A Markov model was developed to assess the cost effectiveness of a biennial fecal immunochemical test (FIT) strategy for individuals aged 50 to 69 years from a private insurer perspective. Survival probabilities and average costs for each cancer stage at diagnosis were derived from our CRC patients. Benefits were measured as life-years gained. The budget impact was calculated using the expected population of beneficiaries for 20 years. Cost-effectiveness results were reported using the incremental cost-effectiveness ratio (ICER), with a referential threshold of one to three times the gross domestic product per capita (USD16,000 to USD48,000). Shared funding scenarios with national public insurers and out-of-pocket payments were simulated.

CRC screening was cost effective in the base case and shared funding scenarios. Indeed, it showed some benefits and savings in the latter scenario. On the other hand, budget impact values were unaffordable for the base case scenario, revealing that implementing the program would be difficult even with the promising ICER. However, in shared funding scenarios, the budget impact decreased and even showed savings after five years due to the decrease in cancer treatment costs.

CRC screening with a biennial FIT is a cost-effective strategy for FALP beneficiaries, but the budget impact is substantial and poses challenges for its implementation. The use of shared funding strategies could help alleviate the budget impact, making the implementation of a screening program more feasible.

Breast cancer is the most frequently diagnosed cancer among women in Poland. Molecular diagnosis of cancer, including identification of mutations in the BReast CAncer 1 and 2 (BRCA1/2) genes, is publicly funded in Poland. However, the diagnostic process is hampered by various billing models that use different testing materials and methods, so the diagnostic scheme is not considered optimal by physicians or patients.

A cost-utility analysis was conducted to evaluate the cost effectiveness of modifying the molecular diagnosis protocol for breast cancer in Poland. The analysis compared the current diagnostic method with a new schema, which involves next-generation sequencing (NGS) for mutations in the BRCA1/2 genes for all patients with breast cancer. The model was developed using a comprehensive review of relevant literature, reimbursement data, and consultations with clinical experts.

If all patients with breast cancer undergo molecular diagnosis, the number of BRCA1/2 gene mutation carriers detected will increase from 213 to 971 per year. Despite the increase in costs to the public payer, compared with the existing scenario, the incremental health effect of the new scenario generated an incremental cost-effectiveness ratio of EUR37,274 per quality-adjusted life-year. This finding effectively disproves the claim that it is not cost effective to perform tests using the NGS technique in all patients with breast cancer.

As a part of the cost-utility analysis it was shown that patients newly diagnosed with breast cancer should undergo molecular testing for mutations in the BRCA1/2 genes using NGS. This schema ensures patients’ access to appropriate targeted therapy. In addition, extended diagnosis of family members can prevent cancer in relatives who carry the mutation.

Crohn’s disease and ulcerative colitis are chronic conditions that primarily affect young individuals of working age and can lead to severe and irreversible complications, which may result in the temporary or permanent inability to work. The objective of this report was to determine whether including broader reimbursement of innovative drugs would result in tangible benefits from a public expenditure perspective.

Social security and social insurance fund benefits paid to incapacitated patients between 2012 and 2021 were used to measure the effectiveness of the innovative drug reimbursement policy. The analysis included expenditures per patient and the full population. Disability and sickness benefits were classified as public transfers and were not included in the costs for the pharmacoeconomic analyses. However, they are crucial in evaluating the efficacy of treatment for a specific disease because they broaden the perspective of public spending. An increase in social benefit expenses per patient suggests a decline in the patient’s health status, whereas a decrease indicates an improvement.

The cost of disability benefits for patients diagnosed with Crohn’s disease and ulcerative colitis was approximately EUR5,814 annually. However, the cost of reimbursing drugs used in drug programs per patient was notably lower. The analysis results indicated positive changes in the amount and structure of social benefits for this patient population (e.g., fewer patients receiving disability benefits). This conclusion was further strengthened by an analysis of the data for each province separately.

The expansion of reimbursement for innovative drugs in the treatment of inflammatory bowel disease provides benefits for public spending.

Successful development of technologies to support older people in care homes remains challenging. The strategic establishment of a group of residential settings providing 24-7 living labs plus an innovation hub is described. Acoustic monitoring (AM) for continuous nighttime checking of residents was selected for assessment. A pilot study in 2016 identified potential cost savings, reduced nighttime falls, and improved daytime well-being.

The AM system was systematically assessed in four WCS Care Group Ltd care homes following demonstration and testing in an innovation hub. The first author undertook a mixed-methods study (2019 to 2023) to assess the technology in terms of falls prevention, practical implementation, and future benefits of enhanced sound classification using artificial intelligence (AI). In 2020, a network of care providers, the National Care Forum (NCF), was funded by National Health Service Digital to develop three similar innovation hubs across England. The NCF undertook a national survey to identify priorities in terms of implementing new technologies in care homes.

Structured interviews with care workers and observational assessment in four care homes confirmed that the AM system has the potential to identify active residents and reduce the risk of falls, although it cannot offer fall prediction or prevention functionality. Two new functions were proposed. Eight machine learning models for sound classification and demonstration tests in three simulated settings supported feasibility and adoption. A post-demonstration survey (n=39) identified a high probability of adoption for these additional functions. The NCF survey found that the top four new technologies of interest to care homes were medication management systems, electronic care planning, wearable GPS trackers, and AM systems.

Care homes report interest in procuring AM systems, which could reduce the risk of falls. In addition, new AI-supported functions are acceptable to care providers. The UK government has proposed that 20 percent of care homes introduce AM by March 2024. Guidelines for future assessment of sustainable care technologies in living lab settings now need to be developed.

The diagnosis intervention packet (DIP) is a health insurance payment method with Chinese characteristics. Because the DIP is easy to understand and operable, it has become the main payment method promoted by China’s National Health Insurance Bureau. The development and reform of the DIP policy, which was introduced in 2020, is worth evaluating.

Policy texts were selected using conceptual sampling and expert interviews. The basic framework of policy evaluation was determined using text mining and statistical analysis methods. Word frequency analysis of the DIP payment policy content was undertaken using NVivo and Gephi software to compare the scope of concern before and after policy implementation. Quantitative evaluation of representative DIP health insurance payment policy content at the national level in China before and after policy implementation (2020 to 2022) was conducted by constructing a text mining and policy modeling consistency (TM-PMC) index model containing nine primary variables and 38 secondary variables.

Policy content analysis using text mining tools revealed that DIP-related policy themes were relatively concentrated, primarily focusing on disease types, medical institutions, and directories. These themes continue to be consistently updated. The quantitative results of the TM-PMC index model showed that the overall design of the policies was reasonable, but there was a noticeable variation in differentiation between the policies. Out of the eight policies analyzed, five were rated as excellent, two as good, and one as acceptable.

The DIP policy is subject to continuous supplementation and optimization. The main factors that influence the value of the TM-PMC index for the policy include the following: policy objectives, policy objects (the groups for which the policy is implemented), policy tools, and policy perspectives. Therefore, various reforms related to collection and payment should be carried out in a locally adapted and standardized manner.

As there is no standard definition of unmet patient or societal needs, the Needs Examination, Evaluation, Dissemination (NEED) project defined criteria for assessing these needs. This study describes the creation of an exploitable evidence database on unmet health-related patient and societal needs for various health conditions, and how this can be used in decision-making.

The NEED framework defines explicit dimensions (patient, societal, and future needs), domains (health, healthcare, and social needs), and criteria, along with specific measurable indicators, to identify unmet health-related patient and societal needs for various health conditions. For each indicator, information sources were sought, including both quantitative and qualitative evidence. For some indicators, guidance on evidence generation was developed. Possible uses of the NEED database were discussed with national and international panels of experts, stakeholders, and decision-makers.

Data sources were identified for some but not all patient and societal needs criteria. They included existing databases (e.g., the Global Burden of Disease database and administrative databases), literature, and primary data collection. A standardized methodology was defined for primary data collection (quantitative and qualitative). The NEED evidence database can be used by researchers, research funders, regulators, health technology assessment agencies, policymakers, patients, healthcare providers, etcetera for prioritizing areas of research as well as—thanks to the level of detail provided in the database—to assess the extent to which proposed “solutions” meet the most pressing unmet needs of patients or society.

Once operational, an unmet patient and societal needs evidence database, anchored to an explicit framework, can serve different stakeholders and decision-makers for various purposes. It collects evidence on unmet health-related patient and societal needs for various health conditions, given the current standard of care, independent of any new product or service, and therefore supports a needs-driven healthcare innovation and policy system.

Brazil’s public health system serves most of the population, but 25 percent of citizens rely on private health insurance. The National Regulatory Agency for Private Health Insurance and Plans (ANS) regulates private medicine reimbursements, which diverge from the public sector threshold. In 2022, the National Committee for Health Technology Incorporation (CONITEC) set a willingness-to-pay benchmark of BRL40,000 (USD8,215) per quality-adjusted life-year. The ANS has no such benchmark, highlighting a pivotal gap in economic evaluations for private health care.

This quantitative study investigated the Incremental cost-effectiveness ratios (ICER) for reimbursed medicines in Brazil’s private health sector, comparing them with CONITEC’s benchmarks and international thresholds. Data were extracted from industry reimbursement submissions to the ANS and analyzed for statistical disparity and policy implications.

Preliminary findings found an ICER peak of BRL619,900 (USD127,220) per quality-adjusted life-year for talazoparib, which is used to treat certain advanced breast cancers. This contrasted sharply with CONITEC’s established threshold, indicating a critical need to evaluate ANS policies.

Early results indicate that the ICERs for some medicines surpass CONITEC’s willingness-to-pay limit, suggesting that the ANS should consider establishing a defined cost-effectiveness threshold. This is imperative to harmonize with global standards and maintain sustainable health financing.

In Brazil, equitable access to medications is critical. There are significant pricing disparities between the National Health System and private health care, which are influenced by the National Committee for Health Technology Incorporation (CONITEC) and Law 14.307. This study investigated these disparities, with aim of proposing strategies for equitable access and sustainability in health care.

This analysis compared prices between the public and private sectors for trastuzumab and adalimumab. Public sector prices were obtained from the Health Prices Database (HPD) and private sector prices were obtained from the Unimed National Table of Materials and Medications (TNUMM), as of May 2023. The study evaluated the extent of pricing discrepancies, considering Drug Market Regulation Chamber ceiling prices and industry discounts.

The cost of the trastuzumab biosimilar, KANJINTI® (Amgen Inc.), was BRL15.79 (USD3.24) per mg in the private sector, compared with BRL4.50 (USD0.92) per mg in the public sector (a 250% difference). The original version of adalimumab, HUMIRA® (AbbVie), was priced at BRL5,450.38 (USD1,120.53) in the TNUMM versus BRL2,445.46 (USD502.33) in the HPD (a 123% difference). The adalimumab biosimilar, HYRIMOZ® (Sandoz Inc.), was priced at BRL7,723.99 (USD1,586.87) in the TNUMM compared with BRL2,449.19 (USD503.05) in the HPD (a 215% price discrepancy).

The study highlights significant disparities in drug pricing between Brazil’s public and private healthcare sectors. These disparities affect the financial sustainability of private health entities and elevate costs for consumers, potentially increasing reliance on the National Health System. Policy revisions, price parity strategies, and further studies are vital for a sustainable healthcare system.

Informed healthcare policies in Brazil rely on robust health technology assessment (HTA), especially for conditions like non-small cell lung cancer (NSCLC). We present an efficiency frontier analysis to evaluate NSCLC treatments that correlates annual treatment costs with clinical outcomes, offering a systematic approach to enhance decision-making in the Brazilian healthcare context.

This quantitative study analyzed NSCLC drug costs within the Brazilian healthcare system and the clinical efficacy data of pivotal studies. The data were analyzed using Python and R software. The dataset comprised drug costs and hazard ratios for overall survival. After data preparation, which involved normalization and outlier management, we constructed an efficiency frontier by ranking drugs based on cost and effectiveness. A linear regression model was then developed to extrapolate this frontier, deriving a formula that predicts treatment costs for specified improvements in overall survival.

The analysis delineated an efficiency frontier and revealed cost-effective NSCLC treatments in Brazil. The following linear regression equation was derived: overall survival = (1.033551 − 0.000003) × treatment cost (USD). This allows for the estimation of appropriate treatment costs for new therapies based on their expected clinical outcomes. This initial model provides a foundation for estimating the economic impact of new treatments.

This preliminary efficiency frontier analysis offers a novel perspective for evaluating NSCLC treatment strategies in Brazil to support sustainable healthcare policy decisions. The model is subject to limitations due to the absence of a systematic literature review. However, it represents an initial step towards a more comprehensive HTA framework. Further research should refine the model by including systematic data collection and analysis.

To speed up the construction of grassroots medical and health teams in China, free training of rural order-oriented medical students was launched in June 2010. Based on the theory of policy tools, a quantitative analysis of policy texts at the national level was conducted to explore the use of policy tools and to put forward corresponding suggestions for adjustments.

From January to February 2023, the research team searched the Peking University Treasure Database and the official websites of the State Council, the National Health Commission, the Ministry of Education, and other ministries for national policy documents related to free training of order-oriented medical students published from June 2010 to May 2023. A policy tool and policy target analysis framework were used to quantitatively analyze the policy documents.

A total of 16 policy documents were included and 213 policy provisions were extracted. From the perspective of policy tools, the proportion of policy provisions using imperative policy tools was the highest (63.4%), followed by advisory policy tools (18.8%). and reward-based policy tools (13.6%). Functional expansion tools (2.8%) and authoritative restructuring tools (1.4%) accounted for a relatively low proportion. The institutional education stage is the main policy target, with provisions accounting for 75 percent (162 articles), followed by the continuing education stage (17.6%; 38 articles), and the postgraduate education stage (7.4%; 16 articles).

The distribution of policy tools for the free training policy of rural order-oriented medical students in China needs to be balanced, and the internal combination of the same policy tools needs to be optimized. The policy targets were mainly concentrated in the education stage of universities.

The National Centre for Pharmacoeconomics (NCPE) in Ireland conducts health technology assessments (HTAs) of drugs under consideration by the decision-maker for reimbursement. We analyzed how clinical expert opinion obtained by applicant pharmaceutical companies is used to inform HTA submissions made to the NCPE. We also describe how clinical opinion obtained by the NCPE is used to inform NCPE assessments.

We conducted a retrospective review of HTA submissions made to the NCPE from July 2019 to June 2020 inclusive. Data were extracted using a bespoke data collection instrument created in Microsoft Excel. To describe how clinical opinion informed the NCPE assessments, we extracted data from NCPE HTA Technical Summary Reports available on the NCPE website.

A total of 18 HTA submissions were reviewed. Clinical expert opinion was used by applicants to support all submissions. The median number of clinical experts who informed each individual HTA submission was seven (range 1 to 33); the majority were hospital physicians. Clinical opinion was used to inform HTA domains, including patient and population estimates (n=14; 78%), use of drugs in clinical practice (n=13; 78%), treatment effectiveness (n=6; 33%), healthcare resource use (n=14; 78%), and health-related quality of life (n=5; 28%). We present examples where clinical opinion, obtained by the NCPE, was used to inform NCPE assessments.

Clinical expert opinion informed all 18 applicant HTA submissions made to the NCPE during the study period. The NCPE also seeks clinical expert opinion to inform their assessments. Healthcare professionals make an important contribution to HTA and, thus, inform the decision-making processes on drug reimbursement in Ireland.

In 2017, the Brazilian Health Regulatory Agency (Anvisa) and the Oswaldo Cruz Foundation (Fiocruz) in Brazil entered into a collaboration that enabled the production of evidence and dissemination of technical scientific information in the regulatory field, with aim of supporting the decision-making process on regulatory issues.

This qualitative and descriptive study was designed to report on the technical cooperation between Anvisa and Fiocruz in Brazil during from 2017 to 2020.

The studies developed by the Decentralized Execution Term assisted in the formulation of health policies at the macro, meso, and micro political levels, supporting the strengthening of institutional regulatory policy. The medicines agenda was the most recurrent in cooperation outputs, followed by the food and smoking agendas. The postmarket surveillance agenda played a leading role in actions carried out with the updating of the Technovigilance Manual and the creation of pharmacovigilance bulletins, both of which were available to health professionals and citizens. Finally, 27 courses were offered, and 1,276 certificates were issued (606 to workers linked to health regulation).

The Brazilian experience enabled to capacity building and critical analysis of evidence in regulatory scope. It has facilitated the preparation of productions that align with the regulatory agenda. Health technology assessment and health regulation need to converge in monitoring processes to reduce uncertainties and increase user safety.

Emergency department (ED) visits for epilepsy are common, costly, and often clinically unnecessary. Configuration of care pathways (CPs) that could divert people away from ED offer an alternative. The aim was to measure patient and carer preferences for alternative CPs and to explore the feasibility of implementing the preferred CPs in the National Health Service (NHS) England with a wider group of stakeholders.

Formative work (provider survey, service-user interviews, knowledge exchange, and think-aloud piloting) informed a discrete choice experiment (DCE) with six attributes: access to care plan, conveyance, time, epilepsy specialist today, general practitioner (GP) notification, and epilepsy specialist follow-up. This was hosted online with random assignment to two of three scenarios (home, public, or atypical). Logistic regression generated preference weights that were used to calculate the utility of CPs. The highest ranked CPs plus a status quo were discussed at three online knowledge exchange workshops. The nominal group technique was used to ascertain stakeholder views on preference evidence and to seek group consensus on optimal feasible alternatives.

A sample of 427 people with epilepsy and 167 friends or family completed the survey. People with epilepsy preferred paramedics to have access to care plan, non-conveyance, one to three hours, epilepsy specialists today, GP notification, and specialist follow-up within two to three weeks. Family and friends differed when considering atypical seizures, favoring conveyance to urgent treatment centers and shorter time. Optimal configuration of services from service users’ perspectives outranked current practice. Knowledge exchange (n=27 participants) identified the optimal CP as feasible but identified two scenarios for resource reallocation: care plan substitutes specialist advice today and times of strain on NHS resources.

Preferences differed to current practice but had minimal variation by seizure type or stakeholder. This study clearly identified optimal and feasible alternative CPs. The mixed-methods approach allowed for robust measurement of preferences, whilst knowledge exchange examined feasibility to enhance implementation of optimal alternative CPs in the future.

An early dialogue (ED) is non-binding scientific advice given to industry in the initial stages of technology development to help create evidence that the health technology assessment (HTA) agency will request. ED could also be used in the academic ecosystem. We report our experience with the clinical validation of an algorithm to predict persistent remission in patients with rheumatoid arthritis treated with biological therapy.

A systematic review (SR) was undertaken to compare optimization algorithms with current clinical management. The review focused on the effectiveness and safety of these tools and included clinical practice guidelines, SRs, and primary studies. Several meetings took place between the research team and HTA researchers to integrate HTA requirements (e.g., choice of comparators, relevant outcomes, quality of life, and patient groups) into the study design to ensure the quality and accuracy assurance of data collected as well as the proper monitoring of good clinical practice.

Local clinical practice guidelines pointed to the importance of optimization strategies to select the most suitable patients in remission. However, there is currently no validated algorithm to select these patients. The literature search retrieved 1,809 references. There were no primary studies identified and only two ongoing randomized controlled trials met the inclusion criteria: REMRABIT-Plus (OPTIBIO) and PATIO. There were some important differences between the studies with respect to the patient populations and stages of the disease. Based on these results, the review will continue in “living evidence” mode, with the aim of collecting new evidence as it becomes available.

There is currently an unfulfilled need between research projects in the academic context and HTA that can be resolved with ED. Collaboration in the early stages of technology development is fundamental to improving the appropriateness of data produced for future HTAs. This will facilitate quality decision-making on the incorporation of procedures into the National Health Service.

Developing a health technology assessment (HTA) report requires the involvement of relevant stakeholders, specifically healthcare professionals, patients, and industry representatives. Evaluating the satisfaction of participants in HTA reports is crucial to improving internal procedures and increasing the participation of stakeholders, who play a crucial role in adding knowledge and experience to the reports.

We developed a nine-question survey to assess the satisfaction of healthcare professionals and patients involved in HTA reports. The questions evaluated global satisfaction with participating in the HTA report (scored 0 to 10), as well as with the information received to develop tasks within the report and the time given to perform them. Healthcare professionals (n=86), patients (n=3), and contributors to outcome prioritization (n=4) involved in 12 HTA reports developed in Catalonia, Spain were invited to participate.

Thirty-two contributors (34.4%) responded. Twenty-nine were healthcare professionals (33.7% response rate) and three were patients (100% response rate); 59.4 percent of respondents had only participated in one HTA report. Contributors rated their participation with 8.53 points, with external reviewers and patients being the most satisfied collaborators (9.33 and 9.00 points, respectively). Twenty-eight participants (87.5%) felt they had received enough information to develop their tasks in the report, while the other four (12.5%) would have preferred more specific information about the aims of the evaluation. All but one contributor was satisfied with the amount of time given to perform the required tasks.

Collaborators in HTA reports were satisfied with their participation. Both the information provided about the tasks and the time provided to perform them were adequate. Nevertheless, the assessment team must always ensure that the aims of the evaluation are clear to everyone. We plan to evaluate the satisfaction of industry collaborators in the near future.