I won’t take any position on the debate, but I’d like to focus on the issues that drive those debates.
- Bio-Ethicists- The western construct of medical ethics is highly individualistic, whereas the eastern construct is for “greater common good”. Individuals matter, yes, but large clinical trials help to uncover the broader trends.
- Modelling drugs “in-silico” with state of the art genomics and pharmacokinetics isn’t going to account for uncertainties.
- A lot of debate around scientific processes revolves around hubris- outliers and confirmation biases.
- Smaller clinical trials will involve a lot of statistical gymnastics that would complicate decision making, even for the clinicians.
- Genomics isn’t perfect; there is a considerable discordance in testing and interpretation. As such, you’d see a huge disclaimer with the next-generation genome sequencing.
- What is the “exact numbers-to-treat”?
- At what level should safety testing be really considered “adequate”?
At a recent symposium at the annual meeting of the American Society of Breast Surgeons titled “Why Big Trials Still Matter,” Dr. Norton cast a critical eye on small trials and the current shift toward them. He warned that most discoveries and advances are modest—for example, a drug that reduces the risk of death by 25%—and statistically, they are more likely to be uncovered in the course of large trials and missed in small trials. “I think that we are throwing away a lot of good drugs and a lot of good diagnostics because our trials are too small,” he said.