Abstract

Observational Therapeutics: A Watershed of Hits for New Drugs

Rama Vaidya, Dean- Medical Research Centre, Kasturba Health Society

Observational studies are gaining ground in the field of drug discovery besides its well accepted importance in epidemiological and social studies. Experimental studies like randomized control trials (RCT), systematic and meta-analysis of RCTs are considered the Gold Standard for “the best available evidence”. However, glaring examples of the fault lines within the presumed gold standard are “the saga of Viox”, Women’s Health Initiative (WHI) study for postmenopausal health issues and the number of drug-withdrawals within a few months to years after the market launch of those drugs.

India, with officially recognized multiple systems of medicine and wide spread use of the system-specific therapies, provides unique opportunity to discover and develop new drugs through well designed observational field studies (cohort or case-control). Evaluation of objective markers (clinical, imaging, biochemical, endocrine, metabolic, molecular etc.) of the condition in question at baseline, during and post intervention can provide outcome information about the effectiveness and safety of the given therapeutic modality. Observed clinical changes with objective markers of response (resolution) in temporal relation to the given intervention would suggest a causal relationship. Few studies have elegantly shown that well-designed observational studies contrary to the existing belief had not overestimated the magnitude of the exposure-outcome associations. “Bedside to bench and back to bedside”, an emerging thought, has similar scientific elements and processes. Observational studies in Ayurvedic therapeutics would thrive on the basis of well studied, carefully harnessed and meticulously documented Ayurvedic field experiences.