A Targeted Review of Worldwide Indirect Treatment Comparison Guidelines and Best Practices.

OBJECTIVES: Controls and governance over the methodology and reporting of indirect treatment comparisons (ITCs) have been introduced to minimize bias and ensure scientific credibility and transparency in healthcare decision making. The objective of this study was to highlight ITC techniques that are key to conducting objective and analytically sound analyses and to ascertain circumstantial suitability of ITCs as a source of comparative evidence for healthcare interventions. METHODS: Ovid MEDLINE was searched from January 2010 through August 2023 to identify publicly available ITC-related documents (ie, guidelines and best practices) in the English language. This was supplemented with hand searches of websites of various international organizations, regulatory agencies, and reimbursement agencies of Europe, North America, and Asia-Pacific. The jurisdiction-specific ITC methodology and reporting recommendations were reviewed. RESULTS: Sixty-eight guidelines from 10 authorities worldwide were included for synthesis. Many of the included guidelines were updated within the last 5 years and commonly cited the absence of direct comparative studies as primary justification for using ITCs. Most jurisdictions favored population-adjusted or anchored ITC techniques opposed to naive comparisons. Recommendations on the reporting and presentation of these ITCs varied across authorities; however, there was some overlap among the key elements. CONCLUSIONS: Given the challenges of conducting head-to-head randomized controlled trials, comparative data from ITCs offer valuable insights into clinical-effectiveness. As such, multiple ITC guidelines have emerged worldwide. According to the most recent versions of the guidelines, the suitability and subsequent acceptability of the ITC technique used depends on the data sources, available evidence, and magnitude of benefit/uncertainty.

Olaparib versus Placebo in Maintenance Treatment of Germline BRCA-Mutated Metastatic Pancreatic Cancer: A Cost-Utility Analysis from the Canadian Public Payer's Perspective.

Pancreatic cancer has an annual incidence of 2/10,000 in Canada, with a one-year mortality rate greater than 80%. In the absence of a cost-effectiveness analysis in Canada, this study's objective was to assess the cost-effectiveness of olaparib versus a placebo in adult patients with deleterious or suspected deleterious BRCA metastatic pancreatic adenocarcinoma, who did not show any progression for at least 16 weeks with first-line platinum-based chemotherapy. A partitioned survival model with a 5-year time horizon was adopted to estimate the costs and effectiveness. All of the costs were extracted from the public payer's available resources, effectiveness data were obtained from the POLO trial, and Canadian studies were used for utility inputs. Probabilistic sensitivity analyses and scenario analyses were performed. The total costs of olaparib and the placebo over five years were CAD 179,477 and CAD 68,569, with overall quality-adjusted life-years (QALYs) of 1.70 and 1.36, respectively. The incremental cost-effectiveness ratio (ICER) of the olaparib group compared with the placebo was CAD 329,517 per QALY. With a commonly cited willingness to pay (WTP) threshold of CAD 50,000 per QALY, the drug does not achieve acceptable cost-effectiveness mainly due to the high price of the medication and insufficient impact on the overall survival of patients with metastatic pancreatic cancer.

Teriparatide in the Treatment of Severe Postmenopausal Osteoporosis: A Cost-Utility Analysis.

Teriparatide is a new agent serves as a treatment of choice for severe post-menopausal osteoporotic patients who are at high risk of fracture or have failed or been intolerant of previous osteoporosis therapy. The objective of this study is to estimate the cost-utility of teriparatide compared with no treatment from health system perspective in Iran. A micro-simulation model was developed for a cohort of hypothetical Iranian patient population (women aged 70 years, T-score -2.5 with previous fracture or T-score -3.0 without prior fracture) over a lifetime horizon. The model consisted of the seven health states. During each cycle, patients could have a fracture, remain healthy, remain in a post-fracture state or die. Background fracture risks, mortality rates, persistence rates, utilities, medical and drug costs were derived using published sources. Total accumulated life-time costs and quality-adjusted life years (QALYs) were estimated. Teriparatide was associated with 4.786 QALYs and total direct costs of IRR 143,168,259 over a lifetime horizon. Compared to no treatment, teriparatide provided an additional 0.145 QALY at an incremental cost of IRR 33,511,013. The resulting incremental cost-effectiveness ratio was IRR 230,333,030/QALYs gained. The probabilistic analysis showed that accepting a willingness-to-pay 2 and 3 GDP/capita in Iran, the probability of teriparatide being cost-effective were 51% and 83%, respectively. Compared to no treatment, teriparatide was indicated to be more costly and associated with fewer fractures, more life-years, and more QALYs. The result showed that teriparatide may be considered a cost-effective intervention when targeted to the appropriate patients.