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The advent of effective agents for the treatment of osteoporosis has led to the view that placebo-controlled trials to test new agents for efficacy are no longer appropriate. Rather, studies of superiority, equivalence, or non-inferiority have been recommended. Such studies require very large sample sizes, and the burden of osteoporotic fracture in a trial setting is substantially increased. Studies of equivalence cannot be unambiguously interpreted because the variance in effect of active comparator agents is too large in osteoporosis. If fracture studies are required by regulatory agencies, there is still a requirement for placebo-controlled studies, although perhaps of shorter duration than demanded at present.

Advancing the diagnosis and treatment of osteoporosis will require progress on several fronts: we must overcome the current widespread failure to fully employ the means already at our disposal for the evaluation, prevention and treatment of osteoporosis. Diagnostic testing must become more effective at characterizing the bone and mineral metabolism of the individual patient. Testing must do far more than identify candidates for treatment; it must permit monitoring of the response to therapy or progression of disease. New therapies should have well-characterized, very specific physiological effects. It is critical that they do not produce adverse effects on the bone remodeling mechanism. Gaps in our understanding of the relationships between the mechanical functions of the skeleton and the biology of bone tissue must be filled in order to provide a basis for evaluation and treatment of the skeleton as an integrated organ system.