International Society For Pharmacoeconomics And Outcomes Research Research Articles

The health-economic models: practical aspects and management of uncertainty

Analytic models are a powerful instrument to develop pharmacoeconomic analyses and their importance is growing as they are being increasingly used to make predictions of the consequences of a particular intervention. It is possible to group the most commonly used techniques in three families: decision trees, Markov chains and probabilistic simulation models. Only the last ones take into account a wide range of uncertainties and have the capability to make probabilistic predictions. Discrete-state, discrete-time Markov models are the most used technique, but have some limits due to their structural rigidity that can make appropriate representation of clinical reality difficult. First-order simulation of Markov models produces deterministic results and can be conveniently implemented in a matrix algebra formal framework. In order to take decision based on models prediction deterministic results are not sufficient and it is widely recognized the need to handle uncertainty in its various forms. The task could be accomplished with traditional (deterministic) and/or probabilistic sensitivity analysis. Both analyses provide complementary information on how the parameters and assumptions uncertainty spreads trough the model and are recommended by ISPOR (International Society for Pharmacoeconomics and Outcomes Research) modelling guidelines.

Good Research Practices for Cost-Effectiveness Analysis Alongside Clinical Trials: The ISPOR RCT-CEA Task Force Report

Objectives A growing number of prospective clinical trials include economic end points. Recognizing the variation in methodology and reporting of these studies, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) chartered the Task Force on Good Research Practices: Randomized Clinical Trials—Cost-Effectiveness Analysis. Its goal was to develop a guidance document for designing, conducting, and reporting cost-effectiveness analyses conducted as a part of clinical trials. Methods Task force cochairs were selected by the ISPOR Board of Directors. Cochairs invited panel members to participate. Panel members included representatives from academia, the pharmaceutical industry, and health insurance plans. An outline and a draft report developed by the panel were presented at the 2004 International and European ISPOR meetings, respectively. The manuscript was then submitted to a reference group for review and comment. Results The report addresses issues related to trial design, selecting data elements, database design and management, analysis, and reporting of results. Task force members agreed that trials should be designed to evaluate effectiveness (rather than efficacy), should include clinical outcome measures, and should obtain health resource use and health state utilities directly from study subjects. Collection of economic data should be fully integrated into the study. Analyses should be guided by an analysis plan and hypotheses. An incremental analysis should be conducted with an intention-to-treat approach. Uncertainty should be characterized. Manuscripts should adhere to established standards for reporting results of cost-effectiveness analyses. Conclusions Trial-based cost-effectiveness studies have appeal because of their high internal validity and timeliness. Improving the quality and uniformity of these studies will increase their value to decision makers who consider evidence of economic value along with clinical efficacy when making resource allocation decisions.

Effectiveness of Pharmacoeconomic and Outcomes Research Fellowship Programs Within the United States

Objective. The purposes of this study were to develop outcome measures for pharmacoeconomic/outcomes research (PE/OR) fellowship programs in the United States, and to use these outcome measures as a means to evaluate their effectiveness. Methods. Outcome measures within the cognitive, affective, and psychomotor domains were developed to assess the effectiveness of PE/OR fellowship programs, incorporated in a survey questionnaire, and administered to former fellows via the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Web site. Results. Of the 68 former fellows who completed the survey, 61 met the inclusion criteria. Former fellows reportedly demonstrated the attainment of cognitive and psychomotor skills through tangible products such as abstracts and manuscripts developed during the fellowship program. Concerning affective outcomes, fellows reported that not only were they satisfied with the fellowship experience, but also felt the experience satisfactorily prepared them for t...

Incorporating the Patient's Perspective into Drug Development and Communication: An Ad Hoc Task Force Report of the Patient-Reported Outcomes (PRO) Harmonization Group Meeting at the Food and Drug Administration, February 16, 2001

Background The extent to which patient-based outcomes can be used to evaluate and communicate the effect of new drugs and devices is a subject of much debate. Criteria for evaluating the scientific quality of data to support health-related quality of life (HRQL) and other patient-based labeling and promotional claims in the United States and Europe have been proposed by various scientists and organizations. Since March 2000, a working group composed of members of the International Society for Quality of Life Research (ISOQOL), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the Pharmaceutical Manufacturer's Association Health Outcomes Committee (PhRMA-HOC), and the European Regulatory Issues on Quality of Life Assessment (ERIQA) met to discuss and coordinate the various recommendations by their respective groups and address the need to harmonize outcomes review criteria within and across United States and European regulatory agencies. Over time, the discussion expanded from HRQL outcomes to include any outcome based on data provided by the patient or patient proxy, that is, patient-reported outcomes (PROs). The working group therefore became known as the PRO Harmonization Group. Methods Working with a member of the US Food and Drug Administration (FDA), four key issues requiring clarification were identified: how PROs are defined and put into operation for research purposes; the added value of PROs in the drug review and evaluation process; selected questions related to the PRO measurement and research methodology; and the interest and demand for PRO information by decision makers. On February 15, 2001, all members of the PRO Harmonization Group attended a meeting in Rockville, Maryland, to discuss these four issues further, and on February 16, 2001, a formal presentation was made to representatives from various departments and reviewing divisions of the FDA. These presentations are summarized in this report. Results All participants agreed that PROs are important for understanding the impact of treatment on patient functioning and well-being. They also stressed the need to communicate PRO information to key decision makers, including regulatory agencies, clinicians, patients and their families, and payers. Finally, the meeting resulted in plans for continuing the dialogue on PRO measurement and interpretation. Summary The February 16, 2001, meeting represented an important step in harmonizing efforts across various organizations and in opening a dialogue with the FDA around major issues related to methodologic standards for measuring and interpreting PROs in the drug evaluation process.

Organizational Structure and Educational Components of Pharmacoeconomic Fellowship Programs: A Fellows' Perspective

Objective. The purpose of this study was to describe the organizational structures and educational components of the pharmacoeconomics and outcomes research (PE/OR) fellowship programs in the United States. Methods. A survey questionnaire was administered to current and past fellows of PE/OR fellowship programs via the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) web site. Results. Of the 102 fellows who completed the survey, 71 met the inclusion criteria. A common profile emerged regarding the organizational and educational features of PE/OR fellowships. Fellows reported that their PE/OR fellowship programs were 2 years in length, typically sponsored by the pharmaceutical industry, and conducted predominantly in academic and pharmaceutical industry sites. Fellows indicated that their fellowships provided them with a variety of coursework and a diversity of research skills to enhance their knowledge about PE/OR. Conclusions. The characteristics of PE/OR fellowships revealed fr...

Pharmacoeconomics in Europe

There is a trend towards greater use of pharmacoeconomics and outcomes research in Europe indicating that health economic researchers in the European Union (EU) are not wasting their time, assured Professor Michael Drummond at the inaugural European conference of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) [Cologne, Germany; December 1998]. However, other than in centralised drug pricing and reim bursement discussions, he believes that the use of pharmacoeconomic evaluation in Europe is still limited. Furthermore, the great diversity in the healthcare systems of Europe makes it difficult to foresee harmonisation of drug pricing and reimbursement decisions at least in the short term.

WOSCOPS economic model results

Strong evidence of the favourable cost effectiveness of pravastatin prophylaxis to prevent cardiovascular disease in Canadian men has been shown by adapting the economic model of the West of Scotland Coronary Prevention Study (WOSCOPS) to the Canadian setting.*So said Jaime Caro, one of the developers of the WOSCOPS economic model, at the Third Annual Meeting of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) [Philadelphia, US; May 1998]. He believes that ‘the richness of WOSCOPS data permits reasonable adjustment’,so that the economic model from Scotland can be applied to other countries. Although the model was successfully adapted to the Canadian setting, such application is ‘not straight-forward’,and involves more than cost-adjustment, he said.

New oral CMV prophylactic more cost effective than current standard?

Testing and ‘pre-emptive’ therapy with IV ganciclovir for 2 months in the presence of viral shedding is currently the most favourable of 3 ganciclovir-based IV strategies for the management of cytomegalovirus disease in renal transplant recipients. This is the finding of an economic study that used a model to ascertain the likely value of a new oral drug for such patients before clinical data are available.*At the Third Annual Meeting of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) [Philadelphia, US; May 1998], Dr Josephine Mauskopf from the Research Triangle Institute, North Carolina, US, used this model to show that if a new oral drug with a similar price and efficacy to IV ganciclovir was available, prophylaxis could be more cost effective than either the pre-emptive or the ‘wait-and-treat’ options.