International Journal of Technology Assessment in Health Care最新文献

Objectives: To assess the health technology assessment (HTA) process in Greece from its establishment in 2018 until 2023 in terms of timeliness and productivity.

Methods: Data were collected from the HTA Committee's database and other publicly available sources. The overall study timeframe was divided into three periods: (i) July 2018-January 2020, (ii) January 2020-July 2021, and (iii) July 2021-February 2023.

Results: During the study period, a total of 1,157 applications for medicinal products (MPs) (including 219 new active substances (NAS) and orphans) were submitted to the HTA Committee. The number of HTA recommendations increased from 60 (first period) to 641 (third period), while the backlog of MPs pending HTA and price negotiations decreased from 89 and 106 (January 2020) to 8 and 44 (February 2023), respectively. The median time intervals for all application types decreased significantly over time. In February 2023, the median time for clinical data assessment of NAS (excluding orphans) almost halved from 207 days in the first period to 114 days; median times for NAS and orphans from regulatory approval to HTA application were 420 and 457 days, and from HTA application to reimbursement 228 and 417 days, respectively.

Conclusions: The performance of the HTA process in Greece improved significantly over time, with increased MP appraisals, backlog reduction, and decreased timelines. Delays in reimbursement of NAS were mainly caused by the long gap between regulatory approval and HTA application. Overall, HTA review times in Greece are now on par with that of well-established European HTA systems.

Objective: The management of rare diseases is rarely addressed among policymakers and public communities. It is hindered by the lack of information on its epidemiology and burden, especially from the perspective of patients and families with rare diseases. This study aims to understand the perceptions of rare disease patients and their families in the management of rare diseases in Malaysia.

Methodology: A qualitative interview was used to explore the perceptions of patients and families regarding the management of rare diseases in Malaysia. In-depth interviews were conducted with the rare disease patients or their parents/guardians provided by three major rare disease advocacy groups, between 1 July and 15 September 2016. The interviews focused on two key areas: the challenges associated with rare disease and the issues related to accessing medication.

Findings: Out of the nineteen recruited participants, seventeen (89.5 percent) completed the interview sessions. The significance of awareness, knowledge, and support from others emerged as crucial for families and patients living with rare diseases. Despite facing delayed diagnosis and treatment, a majority of patients and parents expressed satisfaction with the advancements in rare disease management. Nevertheless, a prominent challenge revolves around access to enzyme replacement therapy for eligible patients.

Conclusion: This study emphasizes the importance of healthcare professionals understanding patient with rare diseases perceptions to tailor communication strategies, provide accurate information, and address concerns effectively. The message underscores the significance of collaboration between healthcare providers and patient support groups to deliver adequate health information, potentially enhancing patients' understanding and their illness perceptions.

Innovative health technologies offer much to patients, clinicians, and health systems. Policy makers can, however, be slow to embrace innovation for many reasons, including a less robust body of evidence, perceived high costs, and a fear that once technologies enter the health system, they will be difficult to remove. Health technology funding decisions are usually made after a rigorous health technology assessment (HTA) process, including a cost analysis. However, by focusing on therapeutic value and cost-savings, the traditional HTA framework often fails to capture innovation in the assessment process. How HTA defines, evaluates, and values innovation is currently inconsistent, and it is generally agreed that by explicitly defining innovation would recognize and reward and, in turn, stimulate, encourage, and incentivize future innovation in the system. To foster innovation in health technology, policy needs to be innovative and utilize other HTA tools to inform decision making including horizon scanning, multicriteria decision analysis, and funding mechanisms such as managed agreements and coverage with evidence development. When properly supported and incentivized, and by shifting the focus from cost to investment, innovation in health technology such as genomics, point-of-care testing, and digital health may deliver better patient outcomes. Industry and agency members of the Health Technology Assessment International Asia Policy Forum (APF) met in Taiwan in November 2023 to discuss the potential of HTA to foster innovation, especially in the Asia region. Discussions and presentations during the 2023 APF were informed by a background paper, which forms the basis of this paper.

Objectives: We draw from the Health Technology Assessment (HTA) literature to propose how hospitals and local health networks can prepare the key components of early economic evaluations to support the development and management of health service interventions.

Methods: Using the case example of a proposed intervention for older people in the Emergency Department (ED), a conceptual logic model of a new health service intervention is articulated to inform the structuring and population of a decision-analytic model using observed data on the existing care comparator and structured elicitation exercise of initial stakeholder expectations of intervention effects.

Results: The elicited patient pathway probabilities and lengths of stay quantities profile which of the existing types of patients are expected to avoid the ED and how this impacts the lengths of stay across the system. The exercise also quantifies the stakeholders' uncertainty and disagreement, with qualitative insights into why. The elicitation exercise participants draw upon the rationale for how the intervention is expected to affect a change within the local context, as captured within the logic model, together with the descriptive analyses of the characteristics and utilization of their target population. Feedback indicates the methods are acceptably robust yet pragmatic enough for healthcare delivery settings.

Conclusions: As proposed in this paper, HTA methods can be used to capture how key stakeholders initially expect a service intervention to affect a change within their local context. The example results can be used in a decision-analytic model to guide the development and management of an intervention.

Objectives: Total hip arthroplasty (THA) is an orthopedic intervention that generates substantial costs to national healthcare systems due to the number of interventions and the cost per intervention. We performed a cost comparison analysis in Austria and Switzerland.

Methods: Data from the national joint arthroplasty register in Switzerland and internal information from the national healthcare services in Austria and Switzerland were compared for patient demographics, interventional characteristics, and costs adjusted for inflation and purchasing power from 2015 to 2021.

Results: The average age for primary THA in Austria was from 67.4 to 67.8 years with 55.9-57.2 percent female patients and from 68.5 to 69.3 years with 52.4-53.8 percent female patients in Switzerland. The annual incidence rate for primary THA rose from 210.28/100k to 216.6/100k in Austria and from 212/100k to 250/100k in Switzerland. After correction for inflation, costs were -1.91 percent lower in Austria in 2021 than in 2015 and -2.57 percent lower in Switzerland. After correction for purchasing power, costs were higher in Austria. The average hospital stay after THA in Austria was reduced by 20 percent (11.7 days/2015 vs. 9.4 days/2021) and 25 percent in Switzerland (8.4 days/2015 vs. 6.4 days/2021). Revision rate was 2.5-3.2 percent in Austria and 2.8-3.2 percent in Switzerland.

Conclusions: The patient population was comparable while patients undergoing primary THA in Austria stay longer in hospital and have relatively higher costs when adjusted for currency, purchasing power, and inflation. The use of standardized registers would be helpful to compare outcomes and costs.

Objectives: Rare diseases (RD)-related policies have received significant attention due to the pressing medical requirements associated with these medical conditions and the substantial impact and treatments they may have on healthcare budgets. Nevertheless, policymakers frequently encounter difficulties in managing issues concerning resource allocation and prioritization within this population. Realizing the need to address such problems, this study was conducted to develop a framework based on the multicriteria decision analysis to improve RD reimbursement prioritization in Malaysia.

Methods: Primarily, a scoping review was performed to identify the methods and criteria used for the reimbursement of RD treatment, followed by strategic stakeholder engagement and a deliberative process on determining the best approach for the framework, including criteria identification, elicitation of weights, and a pilot assessment using the framework.

Results: The findings reflected the priorities and perspectives of the stakeholders, which identified eight key criteria and their associated weights, namely effectiveness (19.6 percent), disease severity (15.6 percent), safety (14.2 percent), access to treatment (12.6 percent), economic consideration (12.2 percent), type of therapeutic treatment (11.5 percent), availability of alternatives (8.3 percent), and population group (6 percent).

Conclusions: In summary, the developed framework was well-accepted by the Rare Disease Committee, which will be applied as part of the committee deliberation for transparent and equitable decision making on fund allocation and reimbursement of orphan and RD treatment in Malaysia.

Background: This study investigated how patient representatives have experienced their involvement in medicines appraisal and reimbursement processes with the Council for Choices in Health Care in Finland (COHERE) and the Pharmaceuticals Pricing Board (PPB) and how authorities perceive the role of patient organizations' input.

Methods: Semi-structured thematic individual and pair interviews were conducted in 2021 with representatives (n = 14) of patient organizations and government officials (n = 7) of the Ministry of Social Affairs and Health. The interview data were analyzed using qualitative content analysis.

Results: Patient representatives expressed their appreciation for the PPB and the COHERE in creating consultation processes and systematic models that support involvement. However, there were many challenges: patient representatives were uncertain about how their submissions were utilized in official processes and whether their opinions had any significance in decision-making. Patients or patient organizations lack representation in appraisal and decision-making bodies, and patient representatives felt that decision-making lacked transparency. The importance of patient involvement was highlighted by the authorities, but they also emphasized that the patient organizations' contributions were complementary to the other materials. Submissions regarding the medications used to treat rare diseases and those with limited research evidence were considered particularly valuable. However, the submissions may not necessarily have a direct impact on decisions.

Conclusions: The interviews provided relevant input for the development of involvement processes at the PPB and COHERE. The interviews confirmed the need for increased transparency in the medicines assessment, appraisal, and decision-making procedures in Finland.

Objectives: In France, decisions for pricing and reimbursement for medicinal products are based on appraisals performed by the National authority for health (Haute Autorité de Santé (HAS)). During the appraisal process, additional real-world evidence can be requested as "Post-Registration Studies" (PRS) when there are uncertainties in evidence that could be resolved by additional data collection. To facilitate PRS planning, a retrospective exploratory analysis was conducted to identify the characteristics of medicinal products associated with a PRS request.

Methods: This analysis encompassed all appraisals finalized between January 1, 2016 and December 31, 2021 and compared products for which the appraisal led to a PRS request with those that did not.

Results: Six hundred positive opinions for reimbursement were identified, with a PRS request present in 17 percent (n = 103) of cases. The independent characteristics associated with a PRS request were a mild or moderate clinical benefit score, a major to moderate or minor clinical added value score, previous availability under an early access program, and certain therapeutic areas (neurology, pulmonology, and endocrinology). These findings suggest two different profiles of PRS requests: (i) products for which there is uncertainty in the size of the clinical benefit and (ii) innovative products for which a substantial benefit is expected but uncertainties persist.

Conclusions: These results will assist health technology developers to better anticipate data generation to promptly address uncertainties identified by HAS. It may also help HAS and other assessment agencies to work together to improve postlaunch evidence generation according to the characteristics of the medicinal products.

Objectives: Patients with hematological malignancies are likely to develop hypogammaglobulinemia. Immunoglobulin (Ig) is commonly given to prevent infections, but its overall costs and cost-effectiveness are unknown.

Methods: A systematic review was conducted following the PRISMA guidelines to assess the evidence on the costs and cost-effectiveness of Ig, administered intravenously (IVIg) or subcutaneously (SCIg), in adults with hematological malignancies.

Results: Six studies met the inclusion criteria, and only two economic evaluations were identified; one cost-utility analysis (CUA) of IVIg versus no Ig, and another comparing IVIg with SCIg. The quality of the evidence was low. Compared to no treatment, Ig reduced hospitalization rates. One study reported no significant change in hospitalizations following a program to reduce IVIg use, and an observational study comparing IVIg with SCIg suggested that there were more hospitalizations with SCIg but lower overall costs per patient. The CUA comparing IVIg versus no Ig suggested that IVIg treatment was not cost-effective, and the other CUA comparing IVIg to SCIg found that home-based SCIg was more cost-effective than IVIg, but both studies had serious limitations.

Conclusions: Our review highlighted key gaps in the literature: the cost-effectiveness of Ig in patients with hematological malignancies is very uncertain. Despite increasing Ig use worldwide, there are limited data regarding the total direct and indirect costs of treatment, and the optimal use of Ig and downstream implications for healthcare resource use and costs remain unclear. Given the paucity of evidence on the costs and cost-effectiveness of Ig treatment in this population, further health economic research is warranted.

Operationalization guidance is needed to support health technology assessment (HTA) bodies considering implementing lifecycle HTA (LC-HTA) approaches. The 2022 Health Technology Assessment International (HTAi) Global Policy Forum (GPF) established a Task Force to develop a position paper on LC-HTA. In its first paper, the Task Force established a definition and framework for LC-HTA in order to tailor it to specific decision problems. This second paper focused on the provision of practical operational guidance to implement LC-HTA. Detailed descriptions of the three LC-HTA operational steps are provided (defining the decision problem, sequencing of HTA activities, and developing optimization criteria) and accompanied by worked examples and an operationalization checklist with 20 different questions for HTA bodies to consider when developing an LC-HTA approach. The questions were designed to be applicable across different types of HTA and scenarios, and require adaptation to local jurisdictions, remits, and context.