The US moves to reclaim the therapeutic pipeline

A welcome move from the FDA and HHS

Today, the Department of Health and Human Services (HHS) announced new plans aimed at restoring American leadership in clinical research by removing unnecessary barriers to clinical trials — in other words, properly doing Clinical Trial Abundance.

In a call with journalists, Acting FDA Commissioner Kyle Diamantas and NIH Director Jay Bhattacharya outlined a series of reforms designed to hasten Phase I trials and encourage companies to conduct studies in the US. The FDA’s efforts are intended to reduce Phase I timelines by six to twelve months. They include an expedited investigational new drug pilot program, updated guidance on the use of advanced computer models for dose selection, and updated frameworks for clinical trial master protocols.

I am particularly excited about the focus on clarifying what is required for a first-in-human Phase I study with respect to chemistry and manufacturing controls (CMC). Excessive CMC requirements are some of the most onerous and unnecessary barriers to clinical trial starts. In conversations with leading oncologists working on cell therapy, they have repeatedly told me they could test as many as 10x more cell therapies with minimal increase in risk (and in the process treat some patients), were it not for the requirement to do a “full-GMP” process.

It is extremely encouraging to see these announcements from the administration and to see this topic being taken seriously, after decades during which clinical trial reform remained at the bottom of policy priorities. Biotech policy in the United States is moving in a direction I have long supported and argued for, particularly in the case of accelerating Phase I trials. The proximate cause for these announcements appears to be the growing recognition of competition from China: writing in Fox News today, HHS Secretary Robert F. Kennedy Jr. calls on the US to win the race for medical innovation, citing China’s surpassing of America in early-stage clinical trials. Kennedy has previously told Congress that China is “eating our lunch” on new drug approvals and clinical trial starts.

This news today coincides with another announcement, an announcement that may perhaps serve to illustrate why the administration is so concerned about China. For the first time in history, a CAR-T cell therapy has been approved to treat a solid tumour. Cell therapies have transformed blood cancers for almost a decade now, but solid cancers, which comprise around 90% of all tumours, have remained out of reach. That changed today: CARSgen Therapeutics announced that China’s National Medical Products Administration has approved satricabtagene autoleucel for the treatment of patients with a specific molecular subtype of advanced gastric and gastroesophageal adenocarcinoma who have failed at least two prior lines of therapy

What is notable about this development, aside from the scientific achievement, is that this therapy was developed by a Chinese company and approved by a Chinese regulator, with no appearance at all from the United States. I have written before about the rise of China in biotech, using the example of another CAR-T therapy, Carvytki. Carvytki was first tested in humans almost a decade ago by a biotech company from China called Legend Biotech. After impressive Phase I results, an American large pharma company licensed the asset from Legend and carried Carvytki to approval, beating the American-originated therapy Abecma in Phase 3 trial results.

The Carvykti case should have been an early warning. Already by 2016, the same year Legend began the clinical trial that would first reveal Carvykti’s potential, China had overtaken the United States in the number of cell-therapy clinical trials. The gap has been growing since. Indeed, a decade later, we see another first: this time, it is not only the early stages, but the whole pipeline, up to approval, that has moved to China. This progression shows something that many of us have been warning for a while: being able to do fast iterations in humans through efficient Phase I trials is key to biotech advancement.

As I wrote at the beginning of this month:

The most valuable thing early-stage trials enable is iteration. They allow tight feedback between the clinic and the lab. There are countless ways to engineer a better CAR-T cell, and many cannot be evaluated in the laboratory alone. No cell culture or animal model fully replicates the complexity of a human tumor, and AI is unlikely to close that gap anytime soon. We simply lack the training data to capture what tumors are actually like in vivo: their geometry, vascularization and biomechanical properties.

China’s ability to run these trials quickly and at scale gave it a structural advantage in that learning process, whereas the US is currently undermining itself through burdensome manufacturing requirements and regulatory bureaucracy that make early experimentation slower and more costly than it needs to be. The policy world has, belatedly, taken notice of this. A proposal in the President’s 2027 FDA budget would streamline early-stage trials. This a welcome recognition that regulatory friction on early experimentation is a competitive liability. But a budget proposal is not a policy and will remain aspirational unless Congress acts.

For now, American and European pharmaceutical companies largely retain the upper hand in the later stages of clinical development, as shown by the fact that Legend ultimately licensed Carvytki to a large American biopharmaceutical company to get it approved. But the pipeline that feeds those late-stage trials is increasingly Chinese. Such early-stage dominance turned into vertical integration of the entire chain in solar panels, batteries and electric vehicles, and LCD panels. The question is how long Western companies can sustain their advantage at the later stages, when the discoveries that make those stages possible are increasingly being made elsewhere.

The announcement of the first solid cancer therapy approved in China and China only prove the veracity of these words. And although I am optimistic due to today’s moves from the FDA and HHS, those of us excited about speeding up Phase I trials and therapeutic abundance should take these announcements as the first steps, not a done deal. Much has to happen, including legislative action on the part of Congress, in order to make more efficient early-stage trials a reality.