A roadmap toward implementing health technology assessment in Egypt

BackgroundThe Egyptian healthcare system is currently in the early phase of health technology assessment (HTA) implementation. The aim of this study is to propose an implementation roadmap based on the national healthcare system status.MethodsA survey was conducted among Egyptian healthcare sector decision-makers to assess the current and future (preferred) HTA implementation status in Egypt based on a widely used international scorecard methodology. Subsequently, interviews were conducted with experts representing middle- and top-tier management in the Egyptian healthcare system to interpret the survey results and recommend specific actions.ResultsExperts recommended more capacity-building programs for HTA and health economics. Additionally, they proposed establishing HTA units in separate healthcare authorities and merging them into a single central HTA unit in the long term. Regarding the scope of implementation, experts recommended commencing with the assessment of innovative pharmaceuticals, and thereafter, expanding the scope to cover all health technologies in the long term. Additionally, they recommended using innovative tools such as “multi-criteria decision analysis (MCDA)” for tendering, and “managed entry agreements” for reimbursement decisions. Local burden of diseases and costing studies were also recommended to facilitate the implementation of HTA.ConclusionExperts agreed that several actions are required for successful HTA implementation in Egypt, including coordination between HTA bodies, application of an explicit MCDA framework, and strengthening of local evidence generation. To implement these actions, investment in technical capacity-building is indispensable. Most experts favored using multiple and soft cost-effectiveness thresholds. Efforts should be made to publish HTA submission guidelines and timelines of the processes

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

Objectives Building 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. Methods We 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. Results It 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. Conclusions Overall, 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 Summary Health 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. Therefore we 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. We found 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

Health technology assessment in Türkiye: Current status and perspectives on future implementation

Objective:: Türkiye's health care system reforms have led not only to increased access to health care but also to rising pharmaceutical expenditures. Therefore, health technology assessment (HTA) has become an important tool for evaluating priorities in reimbursement and budget allocation. Our study aimed to describe the current HTA environment in Türkiye and explore long-term perspectives from a broad spectrum of Turkish stakeholders on the development of HTA in the next ten years. Methods:: In 2019, we used a convenience sampling method to conduct an online survey with stakeholders from different areas in the health system. Additional face-to-face discussions were conducted to clarify answers when needed. We assessed the current evaluation process for pharmaceuticals and examined the need for HTA in Türkiye. Online survey data were extracted into Microsoft Excel for analysis. Quantitative data were summarised descriptively. Results:: A total of 27 Turkish stakeholders completed the survey; 21 were employed in the public sector, and 6, in the private sector. The majority of participants (18/27) suggested introducing HTA for all new health technologies considered for public reimbursement and instituting an additional review process for currently reimbursed technologies. Most respondents (25/27) agreed that a threshold for cost-effectiveness should be applied in the next ten years. Conclusion:: The stakeholders concurred that Türkiye must implement an HTA process soon. However, further discussion and interaction between stakeholders are essential to ensure a broad commitment to the implementation of a structured HTA process in Türkiye

Clinical utility of NGS diagnosis and disease stratification in a multiethnic primary ciliary dyskinesia cohort

Background Primary ciliary dyskinesia (PCD), a genetically heterogeneous condition enriched in some consanguineous populations, results from recessive mutations affecting cilia biogenesis and motility. Currently, diagnosis requires multiple expert tests.Methods The diagnostic utility of multigene panel next-generation sequencing (NGS) was evaluated in 161 unrelated families from multiple population ancestries.Results Most (82%) families had affected individuals with biallelic or hemizygous (75%) or single (7%) pathogenic causal alleles in known PCD genes. Loss-of-function alleles dominate (73% frameshift, stop-gain, splice site), most (58%) being homozygous, even in non-consanguineous families. Although 57% (88) of the total 155 diagnostic disease variants were novel, recurrent mutations and mutated genes were detected. These differed markedly between white European (52% of families carry DNAH5 or DNAH11 mutations), Arab (42% of families carry CCDC39 or CCDC40 mutations) and South Asian (single LRRC6 or CCDC103 mutations carried in 36% of families) patients, revealing a striking genetic stratification according to population of origin in PCD. Genetics facilitated successful diagnosis of 81% of families with normal or inconclusive ultrastructure and 67% missing prior ultrastructure results.Conclusions This study shows the added value of high-throughput targeted NGS in expediting PCD diagnosis. Therefore, there is potential significant patient benefit in wider and/or earlier implementation of genetic screening