Health Technology Assessment System Research Articles

The Polish Society of Gynecological Oncology Guidelines for the Diagnosis and Treatment of Cervical Cancer (v2024.0).

Background: Recent publications underscore the need for updated recommendations addressing less radical surgery for <2 cm tumors, induction chemotherapy, or immunotherapy for locally advanced stages of cervical cancer, as well as for the systemic therapy for recurrent or metastatic cervical cancer. Aim: To summarize the current evidence for the diagnosis, treatment, and follow-up of cervical cancer and provide evidence-based clinical practice recommendations. Methods: Developed according to AGREE II standards, the guidelines classify scientific evidence based on the Agency for Health Technology Assessment and Tariff System criteria. Recommendations are graded by evidence strength and consensus level from the development group. Key Results: (1) Early-Stage Cancer: Stromal invasion and lymphovascular space involvement (LVSI) from pretreatment biopsy identify candidates for surgery, particularly for simple hysterectomy. (2) Surgical Approach: Minimally invasive surgery is not recommended, except for T1A, LVSI-negative tumors, due to a reduction in life expectancy. (3) Locally Advanced Cancer: concurrent chemoradiation (CCRT) followed by brachytherapy (BRT) is the cornerstone treatment. Low-risk patients (fewer than two metastatic nodes or FIGO IB2-II) may consider induction chemotherapy (ICT) followed by CCRT and BRT after 7 days. High-risk patients (two or more metastatic nodes or FIGO IIIA, IIIB, and IVA) benefit from pembrolizumab with CCRT and maintenance therapy. (4) Metastatic, Persistent, and Recurrent Cancer: A PD-L1 status from pretreatment biopsy identifies candidates for Pembrolizumab with available systemic treatment, while triplet therapy (Atezolizumab/Bevacizumab/chemotherapy) becomes a PD-L1-independent option. Conclusions: These evidence-based guidelines aim to improve clinical outcomes through precise treatment strategies based on individual risk factors, predictors, and disease stages.

Priority areas for the development of the health technology assessment system in Ukraine: survey results

The purpose of the study is to analyze the expert opinion on the priority areas of development of the HTA in Ukraine, taking into account the existing condition, problems and possible prospects for effective development. Materials and methods. The expert survey (online questionnaire) included the preparation of a special questionnaire, targeted selection of experts based on their competence in the field of HTA. Three groups of respondents took part in the survey, including 13 HTA developers, 32 users, and 31 informed persons to ensure the representativeness of the survey results. The following methods were used to process and interpret the results: scientific generalization, mathematical and statistical, analytical, and graphical. Results of the study. Based on the results of the expert survey, the role and place of the HTA in the health care system was determined. Experts see the main purpose of the HTA as preparing the necessary information of the relevant level of evidence for decision-making on the selection of optimal MTs (in particular, for budget procurement, inclusion in regulatory lists for price regulation and reimbursement). The respondents' opinions on the participation of different socio-professional groups in the implementation of the HTA and the use of its results were studied. Thus, according to the results of the survey, HTA experts and scientists can carry out the assessment, and in some cases (mini-HTA, hospital HTA for the formation of local formularies and protocols) – specialists of medical institutions. According to the respondents, it is advisable to expand the use of HTA results for decision-making at the national, regional and local levels. According to the survey, the priority areas for the development of HTA are the creation of a professional environment, adequate logistical and informational support, improvement of legislation, creation of an optimal institutional structure, harmonization of HTA methodology and development of international cooperation. Conclusions. The expert survey allowed us to determine the most likely scenario for the further development of HTA in Ukraine. According to the majority of respondents, the main efforts are currently focused on capacity building, formation of a professional environment, active use of evidence, and infrastructure development.

A roadmap towards implementing health technology assessment in Oman

PurposeHealth technologies are advancing rapidly and becoming more expensive, posing a challenge for financing healthcare systems. Health technology assessment (HTA) improves the efficiency of resource allocation by facilitating evidence-informed decisions on the value of health technologies. Our study aims to create a customized HTA roadmap for Oman based on a gap analysis between the current and future status of HTA implementation.Design/methodology/approachWe surveyed participants of an advanced HTA training program to assess the current state of HTA implementation in Oman and explore long-term goals. A list of draft recommendations was developed in areas with room for improvement. The list was then validated for its feasibility in a round table discussion with senior health policy experts to conclude on specific actions for HTA implementation.FindingsSurvey results aligned well with expert discussions. The round table discussion concluded with a phasic action plan for HTA implementation. In the short term (1–2 years), efforts will focus on building capacity through training programs. For medium-term actions (3–5 years), plans include expanding the HTA unit and introducing multiple cost-effectiveness thresholds while from 6–10 years, publishing of HTA recommendations, critical appraisal reports, and timelines is recommended.Originality/valueAlthough the HTA system in Oman is still in its early stages, strong initiatives are being taken for its advancement. This structured approach ensures a comprehensive integration of HTA into the healthcare system, enhancing decision-making and promoting a sustainable, evidence-based system addressing the population’s needs.

Topic identification, selection, and prioritization for health technology assessment in selected countries: a mixed study design

BackgroundThere is limited evidence-informed guidance on TISP processes for countries where health technology assessment (HTA) is in a nascent phase. We aimed to explore the range of topic identification, selection and prioritization (TISP) processes and practices for HTA in selected countries and identify aspects relevant to emerging HTA systems.MethodsThis mixed design study included a systematic literature review, an electronic survey, and individual interviews. We conducted a systematic literature review with criteria that were developed a priori to identify countries deemed to have a recently formalized HTA system. Based on the literature review, a twenty-three item online survey was shared with the identified countries, we completed follow-up interviews with ten participants who have experience with HTA. We analyzed documents, survey responses and interview transcripts thematically to identify lessons related to TISP processes and practices.ResultsThe literature review identified 29 nine candidate countries as having a “potential” recently formalized HTA system. Twenty-one survey responses were analyzed and supplemented with ten individual interviews. We found variation in countries’ approaches to TISP — particularly between pharmaceutical and non-pharmaceutical interventions. Results indicate that TISP is heavily driven by policy makers, expert involvement, and to a lesser extent, relevant stakeholders. The use of horizon-scanning and early warning systems is uncommon. Interviewee participants provided further insight to the survey data, reporting that political awareness and an institutional framework were important to support TISP. TISP can be optimized by stronger national regulations and legislative structures, in addition to education and advocacy about HTA among politicians and decision-makers. In some settings regional networks have been useful, particularly in the development of TISP guidelines and methodologies. Additionally, the technical capacity to conduct TISP, and access to relevant local data were factors limiting TISP in national settings. Increased network collaboration and capacity building were reported as future needs.ConclusionsThis study provides current insights into a topic where there is limited published peer reviewed literature. TISP is an important first step of HTA, and topics should be selected and prioritized based on local need and relevance. The limited capacity for TISP in settings where HTA is emerging may be supported by local and international collaboration to increase capacity and knowledge. To succeed, both TISP and HTA need to be embedded within national health care priority setting and decision-making. More in-depth understanding of where countries are situtated in formalizing the TISP process may help others to overcome factors that facilitate or hinder progress.

Impact of medical insurance access negotiation on the utilization of innovative anticancer drugs in China: an interrupted time series analysis

BackgroundThe high costs of innovative anticancer drugs hinder a number of cancer patients’ access to these drugs in China. To address this problem, in 2018, the medical insurance access negotiation (MIAN) policy was implemented, when the prices of 17 innovative anticancer drugs were successfully negotiated and they were therefore included in the reimbursement list. This study aimed to explore the impact of the MIAN policy on the utilization of innovative anticancer drugs.MethodsWith monthly data on drug expenditures and defined daily doses (DDDs) of each innovative anticancer drug from January 2017 to December 2019, interrupted time series analysis was employed to estimate both the instant (change in the level of outcome) and long-term (change in trends of outcomes) impacts of the MIAN policy on drug utilization in terms of drug expenditures and DDDs. Our sample consists of 12 innovative anticancer drugs.ResultsFrom January 2017 to December 2019, the monthly drug expenditures and DDDs of 12 innovative anticancer drugs increased by about 573% (from US$8,931,809.30 to US$51,138,331.09) and 1400% (from 47,785 to 668,754), respectively. Overall, the implementation of the MIAN policy led to instant substantial increases of US$8,734,414 in drug expenditures and 158,192.5 in DDDs. Moreover, a sharper upward trend over time was reported, with increases of US$2,889,078 and 38,715.3 in the monthly growth rates of drug expenditures and DDDs, respectively. Regarding individual innovative anticancer drugs, the most prominent instant change and trend change in drug utilization were found for osimertinib, crizotinib, and ibrutinib. In contrast, the utilization of pegaspargase was barely affected by the MIAN policy.ConclusionsThe MIAN policy has effectively promoted the utilization of innovative anticancer drugs. To ensure the continuity of the effects and eliminate differentiation, supplementary measures should be carried out, such as careful selection of drugs for medical insurance negotiations, a health technology assessment system and a multichannel financing mechanism.

Application of natural language processing to predict final recommendation of Brazilian health technology assessment reports.

Health technology assessment (HTA) plays a vital role in healthcare decision-making globally, necessitating the identification of key factors impacting evaluation outcomes due to the significant workload faced by HTA agencies. The aim of this study was to predict the approval status of evaluations conducted by the Brazilian Committee for Health Technology Incorporation (CONITEC) using natural language processing (NLP). Data encompassing CONITEC's official report summaries from 2012 to 2022. Textual data was tokenized for NLP analysis. Least Absolute Shrinkage and Selection Operator, logistic regression, support vector machine, random forest, neural network, and extreme gradient boosting (XGBoost), were evaluated for accuracy, area under the receiver operating characteristic curve (ROC AUC) score, precision, and recall. Cluster analysis using the k-modes algorithm categorized entries into two clusters (approved, rejected). The neural network model exhibited the highest accuracy metrics (precision at 0.815, accuracy at 0.769, ROC AUC at 0.871, and recall at 0.746), followed by XGBoost model. The lexical analysis uncovered linguistic markers, like references to international HTA agencies' experiences and government as demandant, potentially influencing CONITEC's decisions. Cluster and XGBoost analyses emphasized that approved evaluations mainly concerned drug assessments, often government-initiated, while non-approved ones frequently evaluated drugs, with the industry as the requester. NLP model can predict health technology incorporation outcomes, opening avenues for future research using HTA reports from other agencies. This model has the potential to enhance HTA system efficiency by offering initial insights and decision-making criteria, thereby benefiting healthcare experts.

OP58 Challenges In Maintaining Up To Date Health Technology Assessments in Rare Diseases: Lessons From Fabry Disease In Australia

IntroductionFabry disease is a rare, inherited X-linked lysosomal storage disease characterized by a wide spectrum of heterogeneously progressive clinical phenotypes, and which results in progressive kidney disease, cardiomyopathy, cerebrovascular disease, and reduced life expectancy. Disease-specific therapy aims to improve symptoms, stabilize current disease and delay progression. In Australia treatment access requires that patients meet pre-specified criteria, which have been in place for more than 15 years. Patient questions prompted the patient organization, Fabry Australia, to investigate why these criteria had remained unchanged despite significant progress in the understanding and management of Fabry disease.MethodsA panel comprising two members of Fabry Australia and its Medical Advisory Committee conducted a review of the literature. The aim of this was to inform the clinical quality of the Australian treatment access criteria with reference to international guidelines and contemporary data. The findings from the literature were applied to develop consensus recommendations for classification and Fabry-specific treatment initiation criteria in diagnosed patients.ResultsEvidence supports earlier treatment with reduced barriers to access in some circumstances. Australian access criteria are misaligned with this. They do not distinguish between classical and non-classical Fabry phenotypes, neglect the impact of quality of life and gastrointestinal symptoms, and impose symptom-severity related criteria, which may lead to unnecessary treatment initiation delay. An updated framework is presented. It differentiates phenotypes, facilitates more timely access to Fabry-specific treatment for classical males, and supports relevant organ involvement criteria in classical females and patients with non-classical disease.ConclusionsA well-performing health technology assessment system facilitates patient access to cost-effective treatments that improve health outcomes. Timely treatment initiation is important to avoid irreversible organ damage in Fabry patients. Patients’ questions about out-dated access criteria has prompted research and uncovered barriers that are no longer clinically valid. The perspectives of the patient as a stakeholder in their disease management should not be overlooked when assessing the value of health technologies in the rare disease setting.

An Effective Health Care System through Health Technology Assessment and Evidence Based Payment System in India

The purpose of this research is to discuss the significance of health care insurance compensation for obtaining comprehensive medical treatment in India. Through a systematic review survey focusing on the keywords like out-of-pocket, Health Technology Assessment and Health care system was conducted by using the searches from Google Scholar, Science Direct, PubMed and ProQuest Database, it is observed that India ranks among the greatest out-of-pocket (OOP) healthcare expenditures in the globe. This seems to be notwithstanding the deployment of several public health plans and the provision of a significant variety of both private and public health coverage schemes. Decrease in the treatment quality across many healthcare institutions of government is a key cause causing the typical Indian individual to constantly rely on medical services provided by private firms, heading up OOP expenses. Among the most significant problems in implementing universal health care (UHC) in India is a poor acceptance of insurance products and awareness between many Indians. The burden of large OOP expenditures on people might be lessened by increasing medical insurance acceptance, which may be accomplished by enhancing India's medical claims system.

Scientific generalization of approaches to the development of the Health Technology Assessment system in international practice

Scientific generalization of approaches to the development of the Health Technology Assessment system in international practice

The Polish Society of Gynecological Oncology Guidelines for the Diagnosis and Treatment of Endometrial Carcinoma (2023)

Due to the increasing amount of published data suggesting that endometrial carcinoma is a heterogenic entity with possible different treatment sequences and post-treatment follow-up, the Polish Society of Gynecological Oncology (PSGO) has developed new guidelines. to summarize the current evidence for diagnosis, treatment, and follow-up of endometrial carcinoma and to provide evidence-based recommendations for clinical practice. The guidelines have been developed according to standards set by the guideline evaluation tool AGREE II (Appraisal of Guidelines for Research and Evaluation). The strength of scientific evidence has been defined in agreement with The Agency for Health Technology Assessment and Tariff System (AOTMiT) guidelines for scientific evidence classification. The grades of recommendation have been based on the strength of evidence and the level of consensus of the PSGO development group. Based on current evidence, both the implementation of the molecular classification of endometrial cancer patients at the beginning of the treatment sequence and the extension of the final postoperative pathological report of additional biomarkers are needed to optimize and improve treatment results as well as to pave the route for future clinical trials on targeted therapies.

Current Medical Technology Reimbursement System in Japan

ObjectivesThe process for reimbursement of medical technologies in Japan is complex, and to date, it has not been well described overall. This article aims to provide an overview of the reimbursement system for medical technologies in Japan, including the reimbursement application process and the payment decision making. MethodsConduct review for relevant health policy and regulation and gather opinion from the key stakeholders. ResultsThe Japanese functional category listing system for the reimbursement of medical technologies is a unique fee-for-service payment system, and the timing for the listing is dependent on the application category. A key positive aspect of the current system is the level of transparency and the predictable pathway for reimbursement of new medical technologies. Conversely, the current reimbursement process may not capture the true extent of the innovation of new technologies, especially when creating a new functional category and/or a new medical procedure coding. ConclusionsThere are potential areas where changes could improve access, efficiencies, and value, such as the price revision system based on the market survey, the foreign average price assessment, and the health technology assessment system. These additions and modifications in policy and regulation of reimbursement will help facilitate the effective and efficient access to new innovative medical technologies within the context of a sustainable and affordable National Health Insurance system in Japan.

Deliberative processes in health technology assessment of medicines: the case of Spain.

Spain incorporated in 2020 changes in its health technology assessment (HTA), pricing, and reimbursement system for medicines including publishing reports, development of networks of experts, or consultation with stakeholders. Despite these changes, it is unclear how deliberative frameworks are applied and the process has been criticized for not being sufficiently transparent. This study analyses the level of implementation of deliberative processes in HTA for medicines in Spain. We review the grey literature and summarize the Spanish HTA, pricing, and reimbursement process of medicines. We apply the deliberative processes for HTA checklist, developed to assess the overall context of the deliberative process, and identify the stakeholders involved and type of involvement following the framework for evidence-informed deliberative processes, a framework for benefit package design that aims to optimize the legitimacy of decision making. In the Spanish HTA, pricing, and reimbursement process deliberation takes place in order to exchange viewpoints and reach common ground, mainly during the prioritization, assessment, and appraisal steps. It is closed to the public, not clearly summarized in published documents and limited to the Ministry of Health, the regulatory agency, other Ministries, and experts with mostly clinical and/or pharmaceutical background. The views of stakeholders are only represented through consultation. Communication is the most commonly used form of stakeholder engagement. Despite improvements in transparency of the Spanish HTA process for evaluating medicines, aspects related to stakeholder involvement and implementation of deliberative frameworks need further attention in order to achieve further legitimacy of the process.

Institutionalizing health technology assessment in Ethiopia: seizing the window of opportunity.

Ethiopia's commitment to achieving universal health coverage (UHC) requires an efficient and equitable health priority-setting practice. The Ministry of Health aims to institutionalize health technology assessment (HTA) to support evidence-based decision making. This commentary highlights key considerations for successful formulation, adoption, and implementation of HTA policies and practices in Ethiopia, based on a review of international evidence and published normative principles and guidelines. Stakeholder engagement, transparent policymaking, sustainable financing, workforce education, and political economy analysis and power dynamics are critical factors that need to be considered when developing a national HTA roadmap and implementation strategy. To ensure ownership and sustainability of HTA, effective stakeholder engagement and transparency are crucial. Regulatory embedding and sustainable financing ensure legitimacy and continuity of HTA production, and workforce education and training are essential for conducting and interpreting HTA. Political economy analysis helps identify opportunities and constraints for effective HTA implementation. By addressing these considerations, Ethiopia can establish a well-designed HTA system to inform evidence-based and equitable resource allocation toward achieving UHC and improving health outcomes.

PP82 First Educational Trainings According To New Health Technology Assessment Guideline For Medicines In Ukraine

IntroductionThe success of the health technology assessment (HTA) implementation depends on the level of communication efficiency between all stakeholders. Taking this into consideration, the leading HTA experts of HTA Department of State Expert Center of the Ministry of Health of Ukraine launched the new educational initiative which includes training programs for pharmaceutical companies.MethodsСomprehensive review of the first developed HTA training programs based on the HTA Guideline (Number 593) “The state health technology assessment for medicines” approved on 29 March 2021 was conducted.ResultsLeading HTA experts of the HTA Department developed a training program, which was conducted for industry representatives according to the provisions of the HTA Guideline. In 2021 over twelve training sessions and two webinars were conducted. These learning events reflect harmonized international recommendations and approaches to HTA training programs, The Professional Society for Health Economics and Outcomes Research, ISPOR short courses in particular, European Network for Health Technology Assessment (EUnetHTA), The National Institute for Health and Care Excellence (NICE), Institute for Clinical and Economic Review (ICER), Agency for Health Technology Assessment and Tariff System (AOTMiT) guidelines. Two-hour webinars were aimed at giving theoretical and practical bases for building a Markov model with the help of Excel and TreeAge Software. Training sessions titled “HTA as a tool for assessing the value of health technology” differ by duration (7-hour, 4-hour, 3-hour) and cover core topics adapted to the needs of the audience. The main program components include HTA Roadmap in Ukraine, clinical section, economic section and practical case studies of building a Markov model. All training options included questionnaires at the beginning and i end to assess the quality of each program. Questionnaires are the tool that gives presenters the possibility to trace progress and transform the training material accordingly.ConclusionsDevelopment of extended capacity building programs in HTA for users and doers is highly prospective for further steps in HTA institutionalization in Ukraine.

OP27 Health Technology Assessment Processes, Characteristics, And Key Differences In High, Middle, And Low-Income Countries Of Asia Pacific Region

IntroductionThe healthcare sector in the Asia Pacific (APAC) region is in a period of rapid growth and exciting innovation. This has led to an increase in the number of APAC countries adopting and implementing health technology assessment (HTA) to assess the clinical, ethical, economic, and societal aspect of healthcare technology. The aim of this study is to provide an updated snapshot of the status of HTA and key differences in selected countries of APAC region.MethodsHTA robustness and gap requirements, including its structure, process, use in decision-making and resource allocation processes were assessed through a review of published and gray literature for each of the selected country. A qualitative analysis was carried out by using a set of 15 principles of an International Working Group for HTA Advancement to identify the robustness and key differences in HTA based decision-making process in scoping countries.ResultsThe finding of this study reveals that maturity of HTA determined by country-specific factors, such as presence of independent HTA agency, healthcare funding and expenditure, etcetera, and varies across the high-, middle-, and low-income countries of APAC region. Based on the study’s results, HTA ecosystem of selected countries categorized into rising HTA followed by advancing and mature HTA categories. In addition to the differences in HTA structure, the influence of stakeholder engagement differs among HTA bodies. The variation in the time frame of HTA decisions was significant among countries, with a general lack of awareness and transparency among health policy decision-makers and resulted in longer time for assessment for rising HTA category compared to the advance and mature HTA categories.ConclusionsThe vision of a comprehensive and robust HTA system can be achieved by implementing a transparent, independent, decision-making, and strongly integrated HTA process in the region. We recommend that efforts should be directed to promote a transparent and sustainable HTA, throughout the low- and middle-income countries of APAC region which eventually, lead to more effective HTA ecosystem.

OP88 Drawing Lines In The Sand: How Do We Define The Scope Of Analysis In HTA And Economic Evaluation?

IntroductionWe explore how the scope of analysis is defined in health technology assessment (HTA) and economic evaluation and consider the potential implications of these decisions.MethodsThe scope of analysis, including decisions about which methods and domains of HTA to include in the assessment, which costs, and health outcomes are most meaningful, and which comparators are the most relevant are typically informed by the needs of the decision-maker. We undertook two systematic scoping reviews to assess: (i) to what extent systems thinking is considered in literature-based technology assessments; and (ii) how the scope of the analysis is defined in economic evaluation using Clostridioides difficile infection as an exemplar. We synthesized the findings from these reviews and offer three key observations for future research and exploration in the field of HTA.ResultsOur scoping reviews found that the scope of analysis in economic evaluations typically focus on single interventions, often ignoring upstream and downstream interventions. Similarly, published technology assessments have narrowly defined and inconsistent scopes of analysis, with limited consideration of indirect health and non-health impacts. Three key observations for the field of HTA include: (i) economic evaluations focus on the value of single heath interventions. A focus on a single health intervention may simplify the analysis; however, will this siloed decision-making lead to optimal health resource allocation? (ii) published assessments have inconsistently defined scopes of analysis. A decision problem that focuses on the needs of the decision-maker is practical; however, will inconsistencies in perspectives across assessments create unfair conceptualizations of value? (iii) HTA is technology-focused, not patient-focused. A technology-focused HTA system aligns with the technology diffusion process; however, does this move us away from the patient-centered mandate of HTA?ConclusionsThe dynamic nature of HTA leads to many conceptualizations of value. Considering the potential implications of narrowly defined, inconsistent, and technology-focused scopes of analyses may have consequences on achieving a patient-centered high-quality health system.

PP115 Analysis Of Previous Joint Clinical Assessment And Potential Transferability To Four European Countries: Case Study And Conceptual Approach

IntroductionThe European Network for Health Technology Assessment (EUnetHTA) has led together, with member states, several joint actions, including Joint Clinical Assessments (JCA), under the form of project-based voluntary cooperation, which outputs and transferability of those projects in other European countries remains somehow limited. In June 2021, the European Council has reached an agreement on the European Health Technology Assessment (HTA) regulation, which is entering into force gradually. Initially limited to oncology products, then extended to orphan/advanced therapy, and after a five to eight-year additional period, it will apply to all centrally approved products. The JCA will consist of a focused scientific analysis on relative effectiveness assessment, including the health condition, technology description, clinical effectiveness, and safety. These analyses will also include information relating to the degree of certainty. We consider that the evidence appraisal might have limitations, and transferability would not be generalizable. We aim to determine the potential drivers and barriers for HTA transferability in EU4, employing the analysis of a case example where JCA was conducted.MethodsEmploying an oncology JCA, we will compare an HTA analysis conducted in EU4 countries (Germany, France, Italy, and Spain). Overview and background information on countries involved in the JCA, and EU4 HTA system will be provided, followed by HTA outcome and main evidence requirements, reimbursement outcome, and pricing agreements.ResultsStudy results supporting HTA outcomes may focus on the population assessed, the comparator considered, and uncertainty management. A conceptual adaptation about the scope of the EU JCA regulation will be discussed, to understand its potential advantages to individual HTAs in Europe and remaining gaps to effectively inform HTA or decision-making process.ConclusionsThe analysis of pricing and reimbursement outcomes can further help understand potential barriers and drivers for JCA transferability and potential areas of evidence generation requirements.

Impact of health technology assessment implementation with a special focus on middle-income countries

ObjectivesBuilding an efficient health technology assessment (HTA) system requires significant effort and political commitment, in addition to human and financial resources. Expectations of what HTA can offer to middle-income countries (MICs) are continuously rising, which drives health policymakers to raise the question of whether HTA could help secure the financial sustainability needed to implement universal health coverage. In this study, we explored the impact HTA adoption may have on the countries and its impact on health system objectives, as well as transferability of benefits and drawbacks observed in higher-income to middle-income countries. MethodsWe utilized secondary data from a systematic literature review and primary data by disseminating a survey among local stakeholders in three MICs across three continents to capture their perspective on the impact of HTA implementation from a local context. ResultsIt was evident from the results of both the literature review and survey that the positive impacts of HTA implementation outweigh the negative impacts. Most of the reviewed literature discussed the impact of HTA on the intermediate objectives of the health finance policy in relation to the broad health system goals. According to the survey respondents, the most evident benefit of HTA implementation is improving the transparency and accountability of healthcare decisions. ConclusionsOverall, HTA implementation can introduce a myriad of benefits to healthcare systems in MICs as well. Our findings show that while HTA implementation may have the potential to generate cost savings in specific areas, there is no guarantee that HTA can generate savings at the macro level. Public Interest SummaryHealth technologies (medicines, devices, and interventions) are rapidly increasing in complexity and cost. Health Technology Assessment (HTA) guides healthcare decision-makers in choosing the most suitable, effective, affordable, and acceptable health technology to invest limited healthcare resources. However, healthcare decision-makers in middle-income countries (MICS) are still uncertain about whether adopting HTA would help them achieve the financial sustainability needed to achieve universal health coverage. Thereforewe sought to gather evidence on how HTA has affected the health systems of countries that have already adopted it by reviewing published research reports. In addition, healthcare decision-makers from three MICs were questioned about their perception of how HTA implementation will affect their country's health system. Wefound that the positive effects of HTA implementation outweigh the negative ones; specifically, the transparency and accountability of decisions are improved. However, although HTA implementation may generate cost savings in specific areas, it may not significantly contribute to overall financial sustainability.

Does health technology assessment compromise access to pharmaceuticals?

In response to rapidly rising pharmaceutical costs, many countries have introduced health technology assessment (HTA) as a ‘fourth hurdle’. We evaluated the causal effect of HTA based regulation on access to pharmaceuticals by using the introduction of Germany’s HTA system (AMNOG) in 2011. We obtained launch data on pharmaceuticals for 30 European countries from the IQVIA (formerly IMS) database. Using difference-in-difference models, we estimated the effect of AMNOG on launch delay, the ranking order of launch delays, and the availability of pharmaceuticals. We then compared the results for Germany to Austria, Czechia, Italy, Portugal, and the UK. Across all six countries, launch delays decreased from the pre-AMNOG period (25.01 months) to the post-AMNOG period (14.34 months). However, the introduction of AMNOG consistently reduced the magnitude of the decrease in launch delay in Germany compared to the comparator countries (staggered DiD: + 4.31 months, p = 0.05). Our logit results indicate that the availability of pharmaceuticals in Germany increased as a result of AMNOG (staggered logit: + 5.78%, p = 0.009). We provide evidence on the trade-off between regulation and access. This can help policymakers make better-informed decisions to strike the right balance between cost savings achieved through HTA based regulation and access to pharmaceuticals.

Consensus on methods of development of clinical practice guidelines in oncology under the auspices of Maria Sklodowska-Curie National Research Institute of Oncology and the Agency for Health Technology Assessment and Tariff System

Introduction. As the changes leading to improvement of cancer care in Poland have shown the need to introduce clinical practice guidelines into the health care system, it has become clear that no methodological standard of the process for guidelines preparation has been established so far. The following process aims to present a unified and comprehensive clinical practice guidelines (CPGs) development methodology. Materials and methods. A review of globally recognised methods used by guideline development groups was prepared, informing the discussion during three plenary meetings and extensive consultations in writing. The resulting document was unanimously approved by a group of 24 methodologists and clinical experts, and has been formally recognized as a standard for CPGs development by the management of the National Institute of Oncology and the Agency for Health Technology Assessment and Tariff System. Results. Within the process, 43 recommendations were formulated to create unified and comprehensive rules for guideline development within the Polish healthcare system. Conclusions. The presented methods are consistent with the globally recognized tools and methods of guideline development, such as GRADE and ADAPTE, and follow quality criteria described by AGREE II. The process supports the development of high-quality guidelines within a resource-constrained setting by allowing to choose between adoption, adaptation, or de novo development of either the whole document of guidelines or particular recommendations.