Pharmacoeconomics study design

Ungar W (1997a) The choice of pharmacoeconomic study design during drug development -Part 1. Pharmacoepidemiology and Drug Safety 6 391-397. 

We have already addressed some of the general issues in the design and interpretation of pharmacoeconomic studies. Yet, prospective pharmacoeconomic studies, especially within phase III clinical trials, are often our only opportunity to collect and analyze information on new therapeutic products before decisions are made concerning reimbursement and formulary inclusion for these agents. We now address issues that arise in the design of these studies. 

Wider range of patients than in NDA/PLA database Larger numbers of patients Active comparator study designs Supplemental efficacy studies Additional approvals in non-ICH countries Pharmacoepidemiology and pharmacoeconomics in particular healthcare environments Post-marketing commitments All of the above... 

Both the quantity and the types of data able to be collected will be affected by the nature of the clinical study patients may be inpatients or outpatients, and this in turn will govern the nature of pharmacoeconomic data that can be recorded. It is also important whether a clinical trial is intended as a pivotal trial for registration or not if a study is pivotal, then a clinical efficacy measure will have to be the primary end point. Pharmacoeconomic parameters can still be incorporated into such a study as secondary end points, and still provide valuable information. If, on the other hand, the clinical research addresses a health system delivery issue, then the pharmacoeconomic end points may well be primary, and the study design need not be constrained by FDA-mandated requirements for the double-blind, placebo-controlled aspects of proof of efficacy. 

As the product moves from phase II into phase III, the number of seemingly redundant instruments should decline as the obvious choice, or best guess should rise to the top. If the goal of phase III studies is to file an NDA or gain regulatory approval, the studies may not be appropriately designed to capture the additional information deemed necessary for the product s success. In some cases, separate pharmacoeconomic studies may be needed prior to marketing. 

When evaluating published pharmacoeconomic studies, the following factors should be considered study objective, study perspective, pharmacoeconomic method, study design, choice of interventions, costs and consequences, discounting, study results, sensitivity analysis, study conclusions, and sponsorship. 

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