H Kim

Overview of methods for comparing the efficacies of drugs in the absence of head-to-head clinical trial data

In most therapeutic areas, multiple drug options are increasingly becoming available, but there is often a lack of evidence from head‐to‐head clinical trials that allows for direct comparison of the efficacy and/or safety of one drug vs. another. This review provides an introduction to, and overview of, common methods used for comparing drugs in the absence of head‐to‐head clinical trial evidence. Naïve direct comparisons are in most instances inappropriate and should only be used for exploratory purposes and when no other options are possible. Adjusted indirect comparisons are currently the most commonly accepted method and use links through one or more common comparators. Mixed treatment comparisons (MTCs) use Bayesian statistical models to incorporate all available data for a drug, even data that are not relevant to the comparator drug. MTCs reduce uncertainty but have not yet been widely accepted by researchers, nor drug regulatory and reimbursement authorities. All indirect analyses are based on the same underlying assumption as meta‐analyses, namely that the study populations in the trials being compared are similar.

Overview of pharmacoeconomic modelling methods

In the current climate of burgeoning health care costs, pharmacoeconomics is becoming increasingly important, but knowledge about pharmacoeconomic methods is limited among most clinicians. This review provides an introduction to, and overview of, common methods used in pharmacoeconomic modelling: decision analysis, Markov modelling, discounting and uncertainty analyses via Monte Carlo simulation. It will conclude with a suggested approach to reading and appraising published pharmacoeconomic analyses.

From Regulatory Approval to Subsidized Patient Access in the Asia-Pacific Region: A Comparison of Systems Across Australia, China, Japan, Korea, New Zealand, Taiwan, and Thailand

Objectives: To compare processes and timings of regulatory and subsidized access systems for medicines across seven jurisdictions within the Asia-Pacifi cr egion.Methods: A questionnaire was developed focusing on regulatory and health technology assessment– based subsidized access processes and timings in each of the seven surveyant’s jurisdictions. Results: Australia and Thailand are the only two jurisdictions that formally allow the subsidized access evaluation process to be conducted in parallel with the regulatory evaluation process. Australian, Japanese, Korean, New Zealand, and Taiwanese systems afford broad coverage, whereas Chinese and Thai systems provide limited coverage for medicines under patent. Subsidized access systems for all jurisdictions except Thailand have an associated patient co-payment for each medicine/prescription. The biggest disparity across the study group relates to time from regulatory submission to subsidized access of patented medicines— ranging from just over 1 year (Japan) to a minimum of 5 years (China). Conclusions: There is consistency across the seven jurisdictions studied in relation to regulatory and subsidized patient access processes—that is, regulatory approval is required before subsidized access review; subsidized access coverage is broad; and the cost of medicine subsidization is offset, in part, by patient co-payments. Although local differences will always exist in relation to budget and pricing negotiation, there may be efficiencies that can be applied across systems to improve time to subsidized access. Closer understanding of regulatory and subsidized access systems can lead to best-practice sharing and, ultimately, timely access and better health outcomes for patients.

PHP32 PROBABILISTIC SENSITIVITY ANALYSIS—A NECESSARY EXTRA?

PHP31 WILLINGNESS TO PAY PER QUALITY ADJUSTED LIFE-YEAR: IS ONE THRESHOLD APPLICABLE FOR ALL DECISION-MAKING? Zhao FL, Yue M, Yang H, Wang T, Wu JH, Li SC University of Newcastle, Callaghan, NSW, Australia; 306 Hospital of PLA, Beijing, China; The First People’s Hospital of Yunnan Province, Kunming, Yunnan, China OBJECTIVES: To estimate the Willingness to pay (WTP) per quality-adjusted life-year (QALY) ratio with the stated preference data and compare the results obtained between chronic prostatits (CP) patients and general population. METHODS: CP patients were recruited from two tertiary referral hospitals and the general populations were randomly approached in China at the beginning of 2009. WTP per QALY was calculated with a formula combining the subjects’ own health-related utility and the WTP value. Two widely used preference-based health-related quality of life instruments, EQ-5D and SF-6D, were used to elicit utility for participants’ own health. The monthly WTP values for moving from participants’ current health to a perfect health described by “11111” status of EQ-5D were elicited using closed-ended iterative bidding contingent valuation method. RESULTS: A total of 268 CP patients and 364 participants from general population completed the questionnaire. We obtained four WTP/QALY ratios ranging from

A Cost-Effectiveness Analysis of Nivolumab Compared with Ipilimumab for the Treatment of BRAF Wild-Type Advanced Melanoma in Australia

4700 to

A Comparison of Three Survival Models to Estimate The Cost-Effectiveness of Cancer Immunotherapy In The Treatment of Advanced Melanoma

7400, which were lower than the proposed thresholds and published researches eliciting the preference for avoiding the risk of death. In addition, the WTP/QALY ratios from the general population were signifi cantly lower than those from the CP patients and different determinants were associated with the within group variation identifi ed by multiple linear regression. CONCLUSIONS: Preference elicitation methods are acceptable and feasible in the socio-cultural context of an Asian environment and the calculation of WTP/QALY produced meaningful answers. The lower WTP/QALY elicited than published values and higher value from CP patients compared with the general population highlight the necessity of considering disease specifi c QALY in estimating WTP/QALY. Our results inferred that one threshold might not be enough to serve all decision-making under different situations. Further studies using the same methods to confi rm whether the WTP/QALY value would be dissimilar among diseases with different impact on QoL would be needed.