Complex treatments—A challenge for clinical research

In complementary medicine, the so-called “whole medical systems” use a complex approach for the diagnosis and treatment, such as Chinese medicine or homeopathy. According to NCCAM whole medical systems are systems that are built upon complete systems of theory and practice and have evolved prior and separate from the conventional medical approach. Clinical research can focus on a variety of different questions, including efficacy and effectiveness. There is an ongoing discussion about the advantages and limitations of both efficacy and effectiveness research. ‘Efficacy’ refers to the extent to which a specific intervention is beneficial under ideal conditions, while ‘effectiveness’ is a measure of whether a specific intervention when applied in a usual care setting has the desired effect. For efficacy studies whole medical systems have to be broken down to single interventions to allow for comparison with a placebo. Furthermore, in these efficacy studies the interventions are mainly applied in a standardized manner, whereas in practice they are usually tailored to the individual patient. The advantage of effectiveness studies is that they allow for an evaluation of whole medical systems by comparing these, for example, with complex conventional standard treatments. When developing a study protocol for an effectiveness study, the theoretical basis of the medical system and the available health service data on the usage in routine care should be considered, to assure that this trial reflects usual care. The relevance for decision making from both efficacy and effectiveness studies is still open to discussion. This is not only a methodological discussion, but also an ethical and political discussion. The core question to be answered is whether the evidence for a specific effect or the evidence that a specific intervention is beneficial in usual care should have more impact, when deciding on the availability of a treatment.

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