Summarised results of a survey carried out among pharmaceutical companies and regulatory agencies, by the CMR International Institute for Regulatory Science
Pharmacogenetic (PGt) and pharmacogenomic (PGx) technologies are being applied at some stage during drug development by all 17 pharmaceutical companies that participated in the survey. Such studies are predominantly carried out on selected new active substances in order to investigate target polymorphisms, understand the mechanism of action and to stratify patients for pharmacokinetic (PK) and pharmacodynamic (PD) effects. The use of these technologies is important not only for the selection of patients prior to enrolling them in trials but also to profile patients already in trials, in order to identify genotypes or phenotypes.
At the time of the survey, the majority of companies had already submitted investigational new drug/clinical trial applications and new drug applications that included some PGt and PGx data, to regulatory authorities. These were predominantly submissions to the FDA but also included the EMEA and MHLW. Less than half of those that participated in the study, however, had discussed the use of the techniques with an authority during drug development.
A major driver for industry to utilise PGt and PGx techniques is the need to reduce development times and costs, but there is also a belief that this approach will identify new targets to increase productivity and expand pipelines. For the authorities, the drivers to embrace the new technologies include the promise that these will improve the rationale for medicinal use in terms of indication, dosing and safety management, as well as the demands of industry and patients themselves. Drivers for the healthcare environment are the demand for improved cost-effectiveness and increased drug safety with improved quality, clinical benefit and benefit/risk ratios.
Although the development of regulatory guidance to cover the new sciences is regarded as inevitable, both industry and regulatory authorities expressed concern that premature implementation of guidelines, in the absence of adequate experience and actual case studies, could be detrimental to progress.