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Ruxandra Teslo 🧬
@RuxandraTeslo
Writer @WorksInProgMag & @Stripe | Clinical Trial Abundance | long-form
Joined January 2020
3.4K Following    31.4K Followers
In my latest @nytimes article, I wrote how Phase I trials in the US are broken and this being the biggest blocker to cancer cures. The good news: we can borrow from Australia, which keeps patients safe while moving trials faster. W/ @kroetscha for @IFP, we mapped out how. Before getting into the weeds: why do Phase I trials matter so much? 1/ Phase I trials enable iterative loops of learning. Each patient generates information researchers can use to refine the drug, rethink the target, adjust the dose, or redesign the next experiment. Faster Phase I trials mean faster feedback between the clinic and the lab and better drugs. 2/ Phase I is also a crucial financing milestone. Small biotechs often have only a limited runway and getting encouraging human data can unlock the capital needed for further development. This is often the difference between survival and death for a small biotech. 3/ Reforming Phase I trials would pave the way to personalised medicine. Sequencing, biological engineering and A.I. make increasingly personalized therapies possible. But our regulatory system was mostly built for standardized drugs tested in large populations. Crucially, Australia is an important counterexample to the idea that faster trials must mean less safety. We can learn from them! 1/ Depending on modality, Phase I trials can begin 6–12 months sooner there, at substantially lower cost. Australia has run more than 18,000 trials since 2006, with no evidence that this faster system has produced worse safety outcomes. 2/ Australian trial volume has increased 2x in the last decade, driven by American companies taking their studies there. What does Australia do well? 1/ Most important, is Australia’s Clinical Trial Notification Pathway (CTN). Instead of submitting an IND package to their national drug regulator, sponsors simply notify the regulator that they are starting the study. Studies are reviewed by local ethics boards. 2/ Australia embraces a more risk-proportionate approach to study oversight. Requirements for Phase I should not be the same as those for later stage trials commercial drugs, and Australian system implements this distinction well. 3/ In Australia, Phase I studies are formally exempt from full manufacturing requirements, which can 10x costs for some drugs. In the United States, the situation is more complicated. Most sponsors end up implementing near commercial-scale manufacturing. So what can the United States do? 1/ First: create an Australian-style notification pathway for appropriate Phase I trials. Instead of requiring every study to pass through the full traditional FDA review pathway that American companies have to go through, qualified institutions could oversee scientific and ethical review. 2/ Formally exempt Phase I trials from commercial-scale manufacturing requirements and replace them with phase-appropriate manufacturing standards. 3/ Make FDA expectations much more explicit. Sponsors over-engineer studies because they do not know what reviewers will accept. FDA should publish much clearer standards of what is expected. 4/ Operation TrialBlazer, launched by HHS in June, is important for achieving this, by proposing an Australian-like Expedited IND pathway. 5/ But more lasting reform will require Congress. First, the FDA will need explicit authorization to rely on the judgments of local institutions to determine whether a trial may proceed. Second, Congress should formally amend the statutory full manufacturing requirements currently codified in section 501(a)(2)(B) of the FD&C Act (21 U.S.C. § 351(a)(2)(B)) to exempt early-phase trials.
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