The complexity and uniqueness of human metabolism and physiology in the past decade has been highlighted and dissected. Arguments have been made to suggest that our lack of understanding about human individuality has led to the limited efficacy and safety of many marketed therapeutic drugs.
The Drug Discovery and Development (DDD) process continues to slowly evolve to address this perpetual problem. More recently with the advent of personalised and precision medicine, there have been efforts made to address individual complexity. An understanding of personalised versus precision medicine is necessary in order to understand such endeavours. This has led to the development of what have been labelled ‘Targeted’ or ‘Personalised Medicine Drugs’. Based on the differences between the N-of-1 model (personalised medicine) and the 1-in-N model (precision medicine), we propose that a more appropriate name is ‘Precision Medicine Drugs’. Such issues are discussed herein. pleonasm or reality?
There appears to be a considerable disconnect between the expectation of stakeholders and the pragmatic demands of bringing a therapeutic drug to market (1). The Drug Discovery and Development (DDD) process was conceived in the early 1960s and has remained relatively unchanged over the past 50-plus years. It continues to be risk-laden, slow, costly and inefficient, as well as delivering products of questionable value in terms of safety and efficacy (1). For example, cumulative risk is associated with any effort to bring a drug to market. The initial screening of compound libraries (104-106) leads to a single lead compound that has only an ~8% chance of successfully traversing the clinical trials gauntlet (2).
In addition, the failure rate of a drug candidate at each stage of clinical trials is reported to be, 46% (Phase I), 66% (Phase II) and 30% (Phase III) (3). The average time required from drug discovery to product launch remains at an eye-watering 12-15 years (4). Finally, the total capitalised cost of bringing a new drug to market was recently estimated at a staggering $2.87 billion (5).
The metrics associated with the DDD process are clearly problematic. There is also a concern about the value proposition of current, marketed therapeutic drug products produced by the DDD process....
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