Authors: Franz Porzsolt, Manfred Weiss, Christel Weiss.
Sir Archibald Cochrane requested about 100 years ago answers to three questions before implementation of innovations in health care. These answers request the confirmation of efficacy, i.e. the “Proof of Principle (PoP)”, of the objective “Real-World Effectiveness (RWE)” and of the subjectively perceived value (VAL) of health care services. At the same time, Sir Ronald A. Fisher developed the concept of the Randomized Controlled Trial (RCT) for research in agriculture. This concept has become the accepted gold standard for demonstration of effects in health care research in the past 50 years.
RCTs are a useful study design to answer the first of the three Cochrane questions “Can it work?”. A wide range of different tools is available to answer the third (subjective) Cochrane question “Is it worth it?”, but none of these tools can answer the second question “Does it work?” in an unselected sample of patients of day-to-day care who present a variety of different comorbidities. We need to consider that the patient individual baseline risk profile may have a stronger effect on the assessed outcomes than our interventions. Without knowledge of the individual patient risk profile, we may probably disregard the strongest effect on the assessed outcomes.
Our comment substantiates and justifies the need of a new method than the RCT for demonstration of RWE. For that, we propose the Pragmatic Controlled Trial (PCT) and describe 1) the methods we used for analysis of data and the emerging strategy recommended for the three-dimensional assessment of outcomes, 2) the identification of the terminology conflict, 3) example to illustrate the evaluation using a PCT, 4) limitations of a PCT, 5) potential impactof the PCT, 6) literature on similar studies.
In summary, The PCT enables the answer to the second Cochrane question. This answer is based on the important effects of the “Endpoint-Specific Risk Profiles (ESRPs) of individual patients on the explored endpoints. In addition, the PCT facilitates the standardized analysis of revenue and expenses. The time and costs of documentation for approval of drugs and medical devices can be effectively reduced due to considerable reduction of necessary experimental RCTs for demonstration of PoP. The results of PCTs also enable new justifications of ethical, medical, economic, and political decisions.
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