Pharmacoeconomic trials Phase

To obtain reimbursement and formulary acceptance as health-care dollars become scarcer, the sponsor at launch or soon after should be able to demonstrate cost-effectiveness, superiority, convenience, and patient satisfaction, usually through data from Phase IV pharmacoeconomic trials (see also Chapter 19). 

Therapeutic use (Phase III/IV) Refine understanding of benefit/risk relationship in general or special populations and/or environment Identity less common adverse reactions Refine dosing recommendation Comparative effectiveness studies Studies of mortality/morbidity outcomes Studies of additional endpoints Large simple trials Pharmacoeconomic studies... 

Mauskopf J, Schulman K, BeU L, et al. A strategy for collecting pharmacoeconomic data during Phase 9 II/III clinical trials. PharmacoEconomics 1996 264-77. 

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. 

Mauskopf J, Schuknan K, Bell L, Glick H. A skategy for collecting pharmacoeconomic data during phase lEIII clinical trials. Pharmacoeconomics 1996 9 264-77. 

Not all clinical trials, especially large, multisite, multinational phase III studies and phase IV postmarketing surveillance, pharmacoeconomic, and quality-of-life studies, can be conducted at a CSO facility. These types of studies, and many phase II efficacy studies, are conducted in research- or university-based hospitals or other investigational sites where a sufficient patient population with the disease or disorder to be tested is available. A number of CSOs offer services to support clinical trial studies that are implemented at one or more clinical trial sites. These services can be broken down into relatively broad categories, which are summarized later. 

Fourth, the chapters on Phase I clinical trials and pharmacoeconomic research have been written by experts in these fields. These are very rapidly-developing disciplines. The typical pharmaceutical physician has usually paid little attention to these aspects of clinical development, but it is our belief that these will dictate his/her clinical development plans to an ever-increasing degree in the future. Phase I studies can shorten overall clinical development time, and the pharmacoeconomic leveraging of (especially) Phase III and Phase IV studies (with preparatory Phase II work) are now essential in the modern competitive environment. 

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