The discipline of clinical pharmacology brings together clinical and scientific practice to support critical and independent appraisal of data pertaining to drugs and therapeutics, and the rational use of medicines. An understanding and knowledge of clinical pharmacology encourages and makes possible the cost-effective use of medicines and vaccines in prevention and treatment of disease at every level of health care and it assists in the making of policies that govern such use. It is important that there should be an educational infrastructure and career path for health professionals in clinical pharmacology.

In its modern form clinical pharmacology was developed in the 1960s, principally in response to public scares about the safety of medicines. The trigger was thalidomide, an incompletely tested drug administered to pregnant women that caused congenital malformations in more than 10,000 newborn infants. In 1961 it was found to be a cause of phocomelia (seal-like rudimentary upper and lower limbs) and other associated abnormalities in infants at birth. The medical world came to realise that the scientific discipline of pharmacology, until then preoccupied with drug action, receptors and laboratory experiments (as important as these are), needed to address more systematically issues of efficacy, safety and rational use of medicines in humans. It was a crucial development that logically followed the earlier contributions of Bradford Hill and others who had systematically developed a logical basis for the controlled clinical trial. The discipline was born of necessity and it held the promise of bringing together drug action, pathology, toxicology, immunology statistics and epidemiology in the interest of safe and effective use of medicines in the clinic and hospital.

Given the public health importance of clinical pharmacology and its potential to contribute to health policy, it is surprising that over the past 40 years it has not thrived, and that it is weakest in the developing world. This chapter reflects the personal experience of the authors, and their efforts to establish clinical pharmacology in a country with a developing economy. It is intended to serve as an affirmation of the need for science and clinical practice to come together in support of rational and cost-effective use of medicines, especially in resource-limited countries and situations. A large proportion of what is expended on medicines in many countries is lost through inefficient systems of procurement and distribution, irrational use, poor adherence, counterfeit and sub-standard medicines, and corruption. Renewed efforts are needed to stimulate clinical pharmacology and to attract inspired leadership. The public needs to have confidence in the medicines available to them, without which people even come to doubt the soundness and reliability of the health system itself. That is a central issue in national and international health policy.