by pappyvanwinkle | 2025-04-30 01:49:08+00:00
Interesting piece, but I think it is helpful to highlight some points not addressed that provide meaningful context. Though the US system is not perfect, I think we need to acknowledge the US does have a robust, enviable biomedical / regulatory ecosystem, and lots of thought and effort have gone into curating the system that we all enjoy today. To engage with a select few of the points made:

1) Author cites that China introduced priority review mechanisms, conditional approvals, and accelerated timelines to streamline approval pathways. They fail to mention that the US helped pioneer these same mechanisms and that they are currently, actively, in use. We DO have priority review vouchers to incentivize investment in orphan disease, conditional approval pathways, and breakthrough designations for drugs addressing unmet needs to name a few. The US has been, and continues to be an innovator on these topics and credit should be given here - this stuff isn't new to us and there is a rich history of attempts to incentivize innovation ranging from offering prizes for cures to disease, to purchasing experimental therapeutics in advance of approval to ensure sponsors have the cashflow to continue development.

2) Regarding acceptance of foreign data, whose data do you think the rest of the world wants to use by designing their policies the way they have? Many countries are willing to accept foreign data because that includes data generated in the US, where historically and presently, standards are very high and the research infrastructure is well-supported (recent events notwithstanding). One need only look at the story of Dr. Frances Kelsey and thalidomide to understand why the US has gone to great lengths to ensure a high bar that is carefully maintained by its own FDA's standards. This is NOT to say great data isn't generated elsewhere and there are examples where ex-US data has been used to support US approval of promising drugs (particularly in areas of significant unmet need, for example, neurodegenerative disease), but there is rhyme to the reason that goes beyond the argument that the US is antiquated in its approach. That said, one can acknowledge there is a geopolitical component that may wax and wane in relative importance depending on the current political climate.

3) Citing the Lunar trial actually highlights the robustness of the US biomedical enterprise rather than detracts from it - that trial was conducted in patients with stage IV NSCLC after they progressed on platinum therapy. In the US during the enrollment period, ICIs like pembro were being approved as first-line monotherapy displacing platinum-based therapies. So the problem there is when practice changes, you're going to 1) have a harder time finding patients that are progressing on the thing that is no longer first line and 2) remember the intervention arm was ICI + TTF...so you can't intervene when people have already received the drug class that is part of your intervention. Saying they couldn't enroll because of bad infrastructure is like saying the apple store is bad at enrolling customers with dead Iphone 10s to trial your combo therapy when most of the people they see have Iphone 15s. You need to enroll then where most people coming through have iphone 10s and this is precisely why they enrolled a significant proportion of patients ex-US because the standard of care elsewhere had not yet changed.

Props to the author for bringing up the important question of US competitiveness in drug development. But let us not forget that we have an enviable biomedical apparatus for a reason - the US has a strong track record of high standards and innovation because we have a good many rigorous, scientifically-minded folks who work on improving it all the time. It's not perfect, but if we don't call out the good, pioneering work that has been done here, then people may feel justified in questioning if anything has been done at all. One should not get the impression we're just waiting around to get hyperdunked or just lucked into the good things we do have.