HHS (FDA, NIH, ARPA-H and related agencies) is moving forward to accelerate clinical trials in what they call Operation TrialBlazer (kudos on the pun). The promoter, of course, is China:
China has made biotechnology a national priority, systematically expanding its clinical research infrastructure through government funding, simplified regulatory mechanisms, and continued investment. In 2021, China’s global share of Phase 1 tests surpassed that of the United States for the first time, a milestone that would have seemed impossible a decade earlier. And in 2024, China surpassed the United States in the total number of registered clinical trials, with more than 7,100 registered, representing 39% of global trials…. For some advanced procedures, including cell and gene therapy, radioligand therapy, and stem cell therapy, China uses investigator-initiated trials to provide more flexibility, albeit with some trade-offs in terms of oversight and quality control. This means that drugs can go into human trials if the researcher is interested and funded. In the US, similar trials can wait years to begin.
I am also pleased to see that they mention Australia, another advanced democracy, as a leader in clinical trial regulation:
Australia’s Clinical Trial Notification System allows trials to begin in less than 70 days after final protocol submission, with regulatory approval granted in 21 to 28 days and sites activated within 6 to 12 weeks.
Remember those comparisons. Operation TrialBlazer suggests positive changes similar to the CMC specification. CMC is Chemistry, Manufacturing, and Controls–and deals with the basics of drug manufacturing. The FDA, however, is very dangerous and companies know that so they often skip the CMC: for example, to prove the stability of the formula in 6+ months when the test will only take a few weeks or to write their full commercial production process before they know if the drug works and they know very well that the process will be changed many times before the drug is actually sold. In short, many costs for very little benefit. The FDA is now clarifying that this kind of thing is not necessary. Well, that’s low-hanging fruit. There are other good ideas too.
But notice what they don’t suggest. Apart from using China and Australia as examples they are not going down any path. Where China is most advanced is in cell therapy, gene therapy, radioligand, and stem cell work and in these areas, China allows trials to proceed on an investigator-initiated basis: as the TrialBlazer document puts it, the drug can enter humans “if the researcher has interest and funding.” China then combines this open (or lax) front (for these products) with government-wide industrial policy to accelerate the winners.
The US refuses to go down that path. Ok, not my phone, but I get it. But they also refuse to follow Australia. In Australia there is also There is no prospective government review of many early phase clinical trials. Under the Clinical Trial Notification (CTN) system, the sponsor submits their protocol package to the Human Research Ethics Committees (HRECs)–Australia’s IRBs–and once the ethics committee approves, the sponsor it informs the regulator, the Therapeutic Goods Administration (TGA), and pays the fee. The TGA does not read and clear the package before the trial begins. Estimates of approximately 21 to 28 days for the first and less than 70 days for the document are faster precisely because the control step is not a test. The government regulator has consistently come out on top in many clinical trials, although unlike China it is involved in serious biological risks. China decided, high risk, high reward.
Australia certifies the certificates, HRECs. Europe uses a similar system for medical device approval. It’s a plan proposed by former FDA chief medical officer Henry Miller and one that I’ve long supported in the US. China is more laissez-faire.
The US formulation in contrast relies on the FDA’s “golden review” and “the FDA will retain full regulatory and decision-making authority.” In short, all the changes to TrialBlazer are about making the gatekeeper faster, cheaper to prepare, and less guaranteed. Nothing about eliminating the gatekeeper.
Addendum: Full disclosure, I have done consulting with ARPA-H for related work. See also my previous post on the repeal process, Montana’s SB535 and America’s Potential Biotech Renaissance.
