Wrong questions, wrong answers? Are we getting the drugs we need?

Abstract

While medicines usually promote health, they can also be expensive and cause harm. It is, therefore, important that needed medicines are developed, and that they are safe, effective and affordable. Unfortunately, progress towards this goal is inconsistent. We argue that forces other than commercialisation need to be considered, and that there is a need to advocate for a drug development process that fills important gaps, reduces clinical uncertainty and promotes the rational use of medicines. The trouble with drug development To maximize clinical benefit and minimize harm to patients, clinicians are increasingly aspiring to an ‘evidence-based’ or ‘rational’ approach to prescribing (1). The relevance and quality of the evidence available about pharmacotherapies is, therefore, essential to good clinical care. Ideally, drug discovery and clinical research (‘drug development’) should serve to advance medical knowledge and improve the prevention and management of disease. In reality, however, it is increasingly apparent that drug development is failing in fundamental ways and that there are major gaps in the evidence available to clinicians