Most companies have some form of standard operating procedure by which they generate clinical study reports. Pharmacoeconomic data should be handled and reported in a similar manner. In some cases it may be appropriate to issue the pharma-coeconomic component of a study as an appendix to a larger clinical report. This will depend on the
Table 19.3 Points to consider: incorporating pharmacoeconomic measures in clinical trials
• Document the pharmacoeconomic objectives, methodology and analysis plan within the study protocol
• Measure outcomes in the most appropriate and most disaggregate units. Categories can always be collapsed at a future time, but is impossible to split out variables beyond their original units. The sources of process and outcomes data may vary
• Clinical data may be captured from providers, patients, and medical records
• Resource use data may be obtained from patient, administrative databases, providers or charts
• Quality of life data should come from the patient. In some cases (very young, very old, mentally unstable) patient proxies are used, but the patient should be considered the optimal choice
• The study design can affect the types of outcomes that can be reliably collected, and the manner in which the outcomes can be collected
• Study design affects several parts of the evaluation process:
Cost of evaluation
Time required to conduct the evaluation
Accuracy of the information gained
Ease of defending subsequent decisions made, based upon the evaluation level of pharmacoeconomic involvement in the study and how closely related the end-points may be to the pathological measures. If there were just a few pharmacoeconomic measures that were being tested, an appendix to a clinical report might be appropriate. In contrast, for example, where recovery from anesthesia is measured by 'street fitness' (the humanistic outcome) and neurological measures of balance and coordination (the physiological end-point), then it could be cogent to report these two types of data together, and to examine how well they correlate; this would not be suited for an Appendix for the humanistic data.
External reports are most likely going to be manuscripts submitted to peer-reviewed journals. Placement of pharmacoeconomic articles in non-specialty journals is important but difficult; some editors do not understand the intrinsic properties of pharmacoeconomic data, and some reviewers will blindly apply statistical constraints that are inappropriate or not valid to humanistic outcomes (e.g. power calculations to measures of the adverse effects of drugs on quality of life measures).
The basic principles of scientific writing and reporting apply to pharmacoeconomic research, and little need be said here. The structure of the paper is the same (Introduction, Methods, Results, Discussion, etc.). It is important to be consistent and appropriate in the use of terminology (e.g. 'costs' is not synonymous with 'charges', and cost-effectiveness is not a cost-benefit analysis; Sanchez and Lee 1994). New mediums such as the Internet offer new possibilities for publication, dissemination, and debate (Medical Outcomes Trust (2001) www.outcomes-trust.org; American College of Clinical Pharmacy 1996).
It must be said that how such information gets disseminated is controversial in the USA. A good recent example is an investigation of atovaquone vs. i.v. pentamidine in the treatment of mild to moderate Pneumocystis carinii pneumonia. This report included a decision tree to estimate the costs and cost-effectiveness of atovaquone vs. Pentami-dine for cotrimoxazole-intolerant patients (Zarkin et al 1996). Clinical outcomes were based on data from a previous randomized controlled trial (RCT) (Phase III) comparing the two medications, while economic outcomes were based on treatment algorithms derived from discharge data, published reports and clinical judgments by the co-authors. The clinical data was from a randomized, double-blind study, a key issue with the agency. A sensitivity analysis was conducted. The major conclusion of the study was that there were significant cost savings to be had from treating Pneumocystis carinii pneumonia on an outpatient basis. An FDA representative, during a platform presentation of this paper, even indicated that this data could be used in promotion.
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