Research regarding new treatments (drugs, biological products, and high-risk devices) often begins with a broad assessment of disease epidemiology, disease burden, and shortcomings of existing treatments. That research may draw from diverse data sources, including real-world data generated by health system operations.
The clinical research phase of treatment development typically follows a well-established pathway from initial evaluation of safety to preliminary evaluation of therapeutic efficacy to pivotal trials intended to support regulatory approval for marketing. Those pivotal trials focus on key questions of efficacy (typically in comparison to placebo or some analogous control condition) and safety (especially serious or previously unrecognized adverse effects). This focus is consistent with the responsibility of the U.S. Food and Drug Administration (FDA) for assuring the safety and efficacy of drugs, biological products, and medical devices at the time of approval.
Regulatory approval is sometimes followed by systematic postmarketing evaluation to address a wider range of practical or real-world questions. This more pragmatic research again draws from more varied sources of data drawn from diverse clinical settings.