Regular readers will know that I'm not in favor of the present state of medical regulation. In this I am not alone. Many people think that some fraction of the cost of obtaining regulatory approval of new therapies is entirely unnecessary, some fraction of the degree of rigor imposed on manufacture and clinical trials is entirely unnecessary. Clinical trials conducted in Australia cost half of those conducted in the US or Europe, because the Australian community has declared that full Good Manufacturing Practice (GMP) procedures mandated by the FDA in the US and EMA in the EU are in fact unnecessary. Something like 10% of all early stage clinical trials worldwide take place in Australia. In that country the government has delegated ethics and assessment of risk to their equivalent of competing institutional review boards, each associated with a specific clinical trial center. It is a good example of the way in which centralization and diminished competition penalizes progress.
Do you think that the right number for the degree of waste currently imposed by regulators is half? More than half? Less than half? It is a huge cost in a world in which $30M to $40M is needed to move from preclinical proof to completing a phase 1 safety trial in a small number of volunteers in the US or EU. That cost means that a sizable fraction of potential medicines are never developed. Halve that cost and more medicines will be developed. Yet those within the system are very quick to defend the excess: regulatory capture rules, and the established pharma industry uses the regulatory system in order to reduce competition from upstate therapeutic developers. None of this is to the benefit of humanity as a whole.
Since the turn of the century, the cost of developing new therapies has more than doubled. The regulators ask for ever more proof, ever more tests, ever more rigor, never strongly penalized for the invisible graveyard of therapies and patients that results. This is how complex systems trapped in the later stages of regulatory capture move forward. The dominant players retain their dominance by becoming a part of the system that suppresses the potential for progress. This is widely recognized, and numerous patient advocacy groups have come, tried to change the system from within, largely failed, and vanished. The Clinical Trials Abundance project is one such, and by no means the most radical. I think their proposals change too little to make a difference even if implemented. I believe that the only path likely to lead to radical change is the development of a robust clinical development ecosystem outside the FDA, EMA, and related regulatory systems, built atop the present medical tourism infrastructure. Something to compete at a much lower price point.
The Case for Clinical Trial Abundance
The need to make drug development more efficient has become increasingly pressing. US healthcare spending growth is predicted to reach nearly 20% of GDP by 2032 and exceed GDP growth itself for structural reasons, like an aging society. Meanwhile, given high medication prices and little political appetite to cut Medicare spending, there is mounting pressure to reduce drug development costs. In the face of these cross-pressures, the best policy approach is a supply-side innovation agenda, aimed at lowering the costs of trials.
We have several reasons to be optimistic about our ability to cut clinical trial costs and timelines. One proof-of-concept is the RECOVERY trial, which cost about 1/80th of a traditional randomized controlled trial (RCT) and likely saved hundreds of thousands of lives by demonstrating the efficacy of steroids for COVID-19. RECOVERY showed the enormous cost and time savings possible if trials are kept tightly focused on important questions and trial enrollment/organization is made as easy as possible. We can also look at historic examples of large trials (e.g., the polio vaccine field trials) that ran on time and answered important questions, by avoiding cumbersome and unnecessary administrative delays.
Many stakeholders agree on the urgency of the problem, often framed as clinical trial modernization. Reducing the cost and difficulty of generating high-quality medical evidence is a rare area where most experts agree on the goals. Beyond these specifics, many of our memos follow the guiding question: "What would a permanent, US-scale RECOVERY trial look like and accomplish?" With dramatically cheaper trials, we would more quickly sift through poorly evidenced clinical practice. New therapies would cost less to test in humans, and we would have answers and innovation sooner. Beyond speeding up the approval of new drugs, cheaper and faster trials would also allow more kinds of questions to be asked. When a large trial costs $100 million to carry out, some questions simply don't get asked.
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