International Journal of Technology Assessment in Health Care最新文献

Biosimilar products that would enhance the patient’s access to treatments have emerged as a product group that is becoming more widespread globally. Since Turkiye is an emerging market in the pharmaceutical area in the Middle East and North Africa region, and being a reference country in pricing processes for some countries, the number of products in the market and reimbursement decisions are important. The aim of this study is to evaluate duration of inclusion of biosimilars into reimbursement lists after registration in Turkiye.

This study used website-based information (Turkish Medicine and Medical Devices Agency Registered Products List and Detailed Pharmaceutical Price List, Social Security Institution Reimbursed Product List) to analyze the reimbursement approval duration for registered biosimilars in Turkiye after receiving registration approval. A study has been conducted on the launch period of biosimilar products to patients access with reimbursement and the evaluation period of reimbursement applications after registration approval. Even though they might not be active on the reimbursement list right now, products that have previously been approved for payment have been included.

Between 1995-2022, biosimilars of 13 active substances in total were registered by the Ministry of Health in Turkiye. Thirty-three different brands and 105 biosimilars with all pharmaceutical forms are registered. As of November 2022, 72 biosimilars were in the reimbursement list. Twenty-two of reimbursed biosimilars were deactivated or excluded from reimbursement. It is calculated that the average evaluation and approval timeline for reimbursement of biosimilars between 2009 and 2022 is 9 months. When biosimilars in the European Medicines Agency (EMA) and Turkiye are compared, 13 biosimilar active substances are licensed in Turkiye compared to 19 for the EMA. When the total number of brands is compared, it has been observed that while 33 brands are registered in Turkiye, this number is 73 for the EMA.

This study, in which reimbursement approval dates for registered biosimilars in Turkiye have been compared based on years and access timelines of biotechnological products, shows that biosimilars have been launched to patients access with reimbursement much faster than biotechnological products.

According to the Quebec law on health and social services, health technology assessment (HTA) is part of university hospital centers’ mission, together with training, research and care. However, unlike these other functions, HTA is not covered in current affiliation agreements that bind a university with academic health institutions. Université Laval and its affiliated health institutions set up a consultation committee whose mandate is to propose the terms of an agreement to specify collaboration regarding HTA between the university and its affiliated institutions. This study investigates perceptions and needs of stakeholders from Université Laval and its affiliated health institutions with respect to the HTA mission.

Semi-structured interviews were conducted with four types of participants, namely university faculty members, health professionals and managers from institutions with and without HTA units, as well as external partners. Interview guides were developed based on the integrated analysis framework of Greenhalgh et al., and adapted to each group of respondents. Most interviews were conducted in groups and were facilitated by a research associate and a senior investigator. Interviews were recorded and uploaded to NVivo 1.6.2 software for codification and analysis.

A total of 57 people were interviewed (nine group interviews and 35 individual interviews). Three main themes emerged, namely knowledge of HTA, factors related to the relevance of the HTA function, and organizational factors. Results showed that half of the respondents have a vague knowledge of HTA or have never heard of it. Most of the respondents agreed that the HTA function fits well with the mission of a health institution. They would accept getting involved with HTA activities at different levels if all conditions are met. Nevertheless, almost half of respondents believed that others strategies than including HTA into contracts of affiliation should be explored to regulate this function. Finally, organizational obstacles such as the lack of funding, shortage of staff and the lack of well-structured collaborations were highlighted by respondents.

Despite recognition of the importance of the HTA function in university health centers, interventions will be required at different levels to support the development of local HTA capacity. In particular, popularization of the HTA function and collaboration networks through specific projects should be developed.

The REAL World Data (RWD) In Asia for Health Technology Assessment (HTA) guidance was developed by a regional working group to facilitate the increasing acceptance of real-world evidence (RWE) in Asia. We compared the consistency of REALISE against guidance from Japan and China.

Country-specific guidance for RWE/RWD use in pharmaceutical development were identified in May 2022 through governmental websites, with validation searches via Google. Sections from local guidance were mapped onto REALISE and categorized as “agree”, “mixed”, “disagree” or “missing” based on coverage and consistency.

Five Japanese and three Chinese documents were mapped. Most sections in Chinese guidance (77%) and 36 percent of sections in Japanese guidance were tagged “agree” or “mixed”, with general alignment on definitions and good practice considerations (study design, accountability); however, 63 percent of Japanese sections were tagged “missing” from REALISE. As local documents took the regulatory perspective, they lacked REALISE’s discussion of translating RWD to RWE for HTA/economic evaluations specifically. Local guidance focused on practicalities of RWD collection in local contexts, including descriptions of specific actions (e.g., evaluating RWD sources, ensuring data security) rather than overarching principles described in REALISE; specifically, Japanese guidance described how to access and analyze databases/registries, reflecting Japan’s landscape of robust sources of national healthcare data, but lacked discussion of other RWE study types, data sources and specialized analytical methods. While Chinese guidance had a broader view of RWD types (more similar to REALISE), they also contained discussions on pharmacovigilance and omics data, communication with regulatory bodies, and incorporation of RWE into the approval pathway for traditional Chinese medicines.

Despite differing purposes (with no RWE guidance from local HTA bodies), local and regional guidance align on general principles/good practice in generating/using RWE, providing common ground for increasing usage of RWE in HTA in Asia.

In order to increase the value of its services and activities, the Agency for Health Quality and Assessment of Catalonia (AQuAS) has incorporated in its strategic plan the commitment to improve the processes, quality and people, while ensuring transparency, independence, rigor and efficiency following the guidelines of the European Foundation for Quality Management. We aim to present the standardization processes to improve the efficiency in elaborating health technology assessment (HTA) reports at AQuAS.

Process standardization has been developed in seven stages: (i) definition of scope, objectives and creation of working groups; (ii) mapping and analysis of all ongoing processes to determine whether they needed improvement or were already optimal; (iii) creation of new processes by evaluating the inclusion of automatic tools and their possible digitization; (iv) creation of the process map; (v) communication to the team for its implementation after training; (vi) dissemination on corporate website; (vii) monitoring and evaluation of their impact.

The creation of AQuAS’ HTA report development process map has involved 14 people over the past two years. After an initial two-day workshop, the team was organized in working groups of two to three people, with regular monitoring and the creation of a specific knowledge management unit led by the AQuAS’ evaluation manager. The process map was configured based on three axes. The strategic axis contains legal frameworks, ethical principles, good practices and methodological frameworks (29 specific for HTA reports). The key axis presents in an integrated way the process and methodology followed from the request of the HTA report to the final product’s dissemination. The support axis includes, among other, the management of 31 requests, information specialist processes (37 searches), training (51 courses) and 6 internal procedures with their corresponding 26 templates and 27 tools.

With a process reengineering approach, HTA reporting has become more efficient. We believe this approach can help other agencies improve their internal processes and subsequently improve team and customer satisfaction.

Health technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to describe the European Network for HTA (EUnetHTA) and HTA agencies expectations regarding PED to support reimbursement in France, Germany, Italy, Spain and the UK.

Published HTA guidance documents were reviewed to identify recommendations related to clinical outcomes assessment (COA) (including disease-specificity, validation, analyses, endpoints and interpretation) and other forms of PED (e.g., patient preference information) in HTA decision-making. Insights from guidance documents were supplemented with a review of literature and published HTA cases and interviews with key opinion leaders (KOLs) focused on current and future states.

The German and French guidance documents include PED recommendations focused on relevant COA and health-related quality of life data, without detailing preferred COA measures. However, key differences were noted between these two countries in the methodological approaches regarding responder definitions, acceptable missing data threshold and multiplicity analyses. These differences were reinforced by the case studies and the KOLs. UK’s sources also focused on COA, in general proposing specific use of the EQ-5D to derive utility values for modelling, but included limited details on other PED-related elements. The Italian and Spanish guidance documents do not detail COA or other PED expectations, but the Italian KOL described that COA is considered if submitted. The currently developed EUnetHTA21 guidelines include PED-related information that bear the signature of certain individual HTA bodies. Globally, there is limited interest in PED beyond COA across the agencies.

The level of expectations with regards to PED varies across EUnetHTA and several European HTA agencies. Interest in PED derived from non-COA sources is limited across the countries. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development. Global harmonization would help advancing PED measurement standards.

An estimated 17.9 million people died from cardiovascular diseases (CVDs) in 2019, which is 32 percent of all global deaths and 85 percent were due to heart attack and stroke. Chest pain is one of the most common reasons for presenting to the emergency department (ED). It is increasingly recognized that artificial intelligence (AI) will have a significant impact on the practice of medicine in the near future and may help with diagnosis and risk stratification. We aim to estimate a diagnostic prediction of acute myocardial infarction by the development and validation of an AI model.

Data on 134 variables of 3,986 consecutive patients who presented to the ED with non-traumatic chest pain were included in the analysis. Using AI tools, a neural network model was developed to establish the risk of acute myocardial infarction (AMI) to achieve n=150 patients over 18 years of age attending the ED.

The mean age was 65.5 (±13.7) years and 63.6 percent were male. Most (60.1%) patients were admitted to hospital, with only 20.3 percent diagnosed at hospital discharge with ischemic heart disease (IHD). All patients were followed up for two months, and 6.3 percent were readmitted to the ED, but none presented with an episode of IHD. In the data analysis of the entire sample we obtained a probability of diagnosing IHD by the SmartHeart model (S=93.1%, E=47.3%, PPV=31.0%, and NPV=96.4%). When we analyzed the sample of patients with no history of IHD (n=104), the diagnosis accuracy was as follows (S=100%, E=77.5%, PPV=42.8%, and NPV=100%).

Our AI model provides information to predict patients who are suffering from acute IHD. AI has been reported to outperform emergency physicians and current risk stratification tools to diagnose IHD, but has rarely been integrated into practice. This study highlights the diagnostic applicability and accuracy of this type of tool and that is why studies should be implemented to see its effectiveness in routine practice in EDs.