PharmacoEconomics

Accurate prediction of relevant outcomes is important for targeting therapies and to support health economic evaluations of healthcare interventions in patients with diabetes. The United Kingdom Prospective Diabetes Study (UKPDS) risk equations are some of the most frequently used risk equations. This study aims to analyze the calibration and discrimination of the updated UKPDS risk equations as implemented in the UKPDS Outcomes Model 2 (UKPDS-OM2) for predicting cardiovascular (CV) events and death in patients with type 2 diabetes mellitus (T2DM) from population-based German samples. Analyses are based on data of 456 individuals diagnosed with T2DM who participated in two population-based studies in southern Germany (KORA (Cooperative Health Research in the Region of Augsburg)-A: 1997/1998, n = 178; KORA-S4: 1999–2001, n = 278). We compared the participants’ 10-year observed incidence of mortality, CV mortality, myocardial infarction (MI), and stroke with the predicted event rate of the UKPDS-OM2. The model’s calibration was evaluated by Greenwood–Nam–D’Agostino tests and discrimination was evaluated by C-statistics. Of the 456 participants with T2DM (mean age 65 years, mean diabetes duration 8 years, 56% male), over the 10-year follow-up time 129 died (61 due to CV events), 64 experienced an MI, and 46 a stroke. The UKPDS-OM2 significantly over-predicted mortality and CV mortality by 25% and 28%, respectively (Greenwood–Nam–D’Agostino tests: p < 0.01), but there was no significant difference between predicted and observed MI and stroke risk. The model poorly discriminated for death (C-statistic [95% confidence interval] = 0.64 [0.60–0.69]), CV death (0.64 [0.58–0.71]), and MI (0.58 [0.52–0.66]), and failed to discriminate for stroke (0.57 [0.47–0.66]). The study results demonstrate acceptable calibration and poor discrimination of the UKPDS-OM2 for predicting death and CV events in this population-based German sample. Those limitations should be considered when using the UKPDS-OM2 for economic evaluations of healthcare strategies or using the risk equations for clinical decision-making.

High-intermediate grade non-Hodgkin’s lymphoma (NHL) is an aggressive form of the disease, which can respond well to combination chemotherapy, with long-term survival seen in 40–50% of patients. When NHL relapses following standard treatment, high-dose chemotherapy with peripheral blood stem cell or bone marrow support may still cure a significant proportion of patients. Despite a significant rise in the incidence of NHL over recent years, there remains only limited published economic study concerning the overall lifetime cost of treatment, the cost effectiveness of specific treatments or the overall societal cost burden of the disease. The majority of studies identified for the purposes of this review considered the cost of alternative forms of chemotherapy and bone marrow support strategies for patients with advanced disease. Data from these studies suggest that there is a definite trend towards reduced costs for high-dose therapy, possibly reflecting increasing technical experience and improved bone marrow recovery through the use of stem cell transplantation and growth factors. The limited number of cost-effectiveness evaluations suggest that high-dose therapy, following a chemosensitive relapse, is likely to be considered favourable against commonly quoted cost-effectiveness thresholds. Cost effectiveness is becoming an increasingly important factor to consider in the formal assessment of new interventions conducted by groups such as the UK National Institute for Clinical Excellence. In light of the increasing incidence of NHL and the extended use of high-dose treatments in other subgroups of patients, there is a need for increased research into the economics of new interventions for NHL.

Background: Peripheral arterial disease (PAD) is increasingly recognised as an indicator of disseminated atherothrombosis, but its impact on use of healthcare resources is not well understood. Objective: To provide a quantitative description of the resource utilisation and costs incurred following PAD. Methods: Hospitalisations, physician visits and the corresponding direct medical costs were examined in 16 440 patients with a diagnosis of PAD (1985–1995) in Saskatchewan, Canada, and compared with 15 590 reference patients with a diagnosis of myocardial infarction (MI) [1990–1995]. Medical history and patient characteristics were available retrospectively to January 1980 and follow-up to December 2000. Rates and timing of all-cause and cardiovascular hospitalisations and physician visits within discrete periods in the 10 years following PAD diagnosis, and 5 years following MI, were evaluated, as were lengths of stay and predictors of hospitalisation. Results: Average follow-up was 5.9 years among patients with PAD and 3.6 years for MI. Half (55%) of patients with PAD were male versus 64% of reference patients. The mean ages were 67.3 and 66.9 years, respectively. Patients with PAD were hospitalised most frequently soon after diagnosis, with rates subsequently decreasing to 0.14 per month. These rates were similar in the reference group except for the period immediately following MI. The average 5-year cost post-diagnosis (2002 $Can) per patient was $Can41 968 vs $Can48 578 for the reference population. Conclusions: A diagnosis of PAD not only imposes a severe burden on patients and their families, but it also significantly increases the use of healthcare resources and the associated costs. By the end of year 1, this burden is comparable with a diagnosis of MI.

Axicabtagene ciloleucel (axi-cel) đã nhận được sự cấp phép tiếp thị tại Canada cho việc điều trị u lympho B cell lớn tái phát hoặc kháng trị sau hai hoặc nhiều hơn hai đợt điều trị hệ thống. Giá trị lâm sàng và kinh tế của axi-cel đối với bệnh nhân và hệ thống y tế nên được xem xét. Mục tiêu của phân tích này là xác định, từ quan điểm của xã hội và người chi trả y tế công cộng, tính hiệu quả chi phí của axi-cel so với chăm sóc hỗ trợ tốt nhất cho bệnh nhân mắc u lympho B cell lớn tái phát hoặc kháng trị tại Canada. Một mô hình kinh tế dược phẩm đã được phát triển và tìm kiếm dữ liệu lâm sàng từ các nghiên cứu ZUMA-1 và SCHOLAR-1 bằng cách sử dụng so sánh ghép cặp theo điểm xu hướng. Một cách tiếp cận mô hình hỗn hợp phân đoạn sống sót đã được áp dụng để mô tả hiệu ứng tiềm năng chữa trị của phương pháp điều trị axi-cel trong u lympho B cell lớn. Việc sử dụng tài nguyên y tế và dữ liệu sự kiện bất lợi dựa trên kết quả từ ZUMA-1, và giá trị tiện ích lấy từ dữ liệu ZUMA-1 được bổ sung bởi tài liệu đã công bố. Chi phí (tính bằng đô la Canada năm 2021) được lấy từ các cơ sở dữ liệu chi phí công khai của Canada và tài liệu đã công bố. Lợi ích và chi phí được chiết khấu ở mức 1,5% mỗi năm, và các phân tích độ nhạy đã được thực hiện để đánh giá độ chắc chắn của các kết quả. Trong trường hợp cơ bản, axi-cel đã tạo ra thêm 6,2 năm sống so với chăm sóc hỗ trợ tốt nhất, tương đương với 4,6 năm sống điều chỉnh theo chất lượng bổ sung, và có liên quan đến chi phí bổ sung là 606,010 đô la. Tỉ lệ chi phí-lợi ích gia tăng là 132,747 đô la cho mỗi năm sống điều chỉnh theo chất lượng so với chăm sóc hỗ trợ tốt nhất từ góc độ xã hội (106,392 đô la cho mỗi năm sống điều chỉnh theo chất lượng từ góc độ người chi trả y tế công cộng). Những yếu tố chính của phân tích bao gồm giá trị sống không tiến triển và sống toàn bộ cho axi-cel. Các kết quả của phân tích này gợi ý rằng axi-cel có thể được coi là phân bổ tài nguyên hiệu quả về chi phí so với chăm sóc hỗ trợ tốt nhất trong việc điều trị bệnh nhân lớn tuổi bị u lympho B cell lớn tái phát hoặc kháng trị tại Canada.

The role of the Pharmaceutical Management Agency (PHARMAC) is to manage pharmaceutical subsidy expenditure in New Zealand. PHARMAC has adopted a proactive approach. It selects the drugs that are to be subsidised and declines to subsidise others. It has established reference pricing across many drug groups, has entered into a range of innovative commercial contracts with pharmaceutical companies, and has encouraged greater price competition among pharmaceutical companies in order to lower prices and control expenditure risk. These initiatives have all been part of an overarching strategy to improve the value of the government’s expenditure on pharmaceuticals. PHARMAC has also developed techniques of cost-utility analysis to assess the value of expenditure. PHARMAC has slowed pharmaceutical expenditure growth, culminating in a fall in expenditure in the 1998/1999 year. At the same time, patient access has continued to expand, with more prescriptions being written and new drugs being subsidised. Therefore, PHARMAC has made dramatic strides to improve the value of the government’s expenditure on pharmaceutical subsidies and its actions have meant that more funds have been available for investment in other health services, than would have occurred if previous policies had remained unchanged.

Cost-effectiveness (CE) thresholds are being discussed more frequently and there have been many new developments in this area; however, there is a lack of understanding about what thresholds mean and their implications. This paper provides an overview of the CE threshold literature. First, the meaning of a CE threshold and the key assumptions involved (perfect divisibility, marginal increments in budget, etc.) are highlighted using a hypothetical example, and the use of historic/heuristic estimates of the threshold is noted along with their limitations. Recent endeavours to estimate the empirical value of the thresholds, both from the supply side and the demand side, are then presented. The impact on CE thresholds of future directions for the field, such as thresholds across sectors and the incorporation of multiple criteria beyond quality-adjusted life-years as a measure of ‘value’, are highlighted. Finally, a number of common issues and misconceptions associated with CE thresholds are addressed.