THREAD: In response to common but incorrect orthodoxy that FDA is approving new products “too fast,” echoed in today’s @nytimes, some reflections on what’s changed in science and medicine and why certain aspects of drug/device development have changed alongside the advances.
First on product safety: We have many more tools to evaluate safety pre and post approval. Our expectation for better drug and device safety has increased over time, but that’s wholly appropriate because so too has our ability to assure the safety of new products.
On drugs, more new drugs are targeted to narrow populations and significant unmet needs. Financial incentives drove development into these areas but science made it possible. We’re able to target the genomic and proteomics basis of disease in a way not possible in the early 2000s
The result? We often see a robust, outsized treatment response in small patient series in settings we’re disease is serious and options few. This opportunity fueled bi-partisan congressional consensus to make development more efficient through paths like breakthrough designation
When a drug is advanced the biological basis for how it works to achieve its effect is well understood. Gone are days of drugs screened at random. Rational drug design and firmer knowledge of biology means we know more about biological plausibility of new drugs than ever before.
I’m studying this empirically, but it’s likely that when we look at recent cohorts of new drugs against older cohorts we’ll find that the absolute treatment effect offered by new drugs has, on the whole, improved over time. In other words, new drugs offer more health improvement.
#FDA itself has undergone a transformation over the last few decades in terms of its professional efficiency and scientific prowess. The Office of New Drugs is a crown jewel of the federal service and the entire organization is marked by deep expertise and mission driven focus->
This fidelity translates into a better overall drug and device development and review process. The improvements in people and process have made the agency’s review programs more efficient. #FDA’s singular strength remains its people and it’s culture of science and public health.
A multitude of recent policies have also improved trial design, signal detection, and product safety. The net result is a renaissance of scientific opportunity that’ll define our age as one marked by our ability to crack the biology of disease and increasingly transcend illness.
