The process of testing drugs in humans—clinical development—is a massive, under-studied bottleneck, accounting for 70% of drug development costs. Despite its importance, there is surprisingly little public knowledge, academic research, or even basic documentation on how to improve this crucial stage.
Critical knowledge on how to run clinical trials is not formalized in textbooks or courses but is passed down through a slow apprenticeship model. This limits the spread of best practices and forces even highly educated scientists to "fly blind" when entering the industry, perpetuating inefficiencies.
While the UK's world-class universities provide a rich pipeline of scientific talent for biotechs, the country's clinical trial infrastructure is a significant hurdle. Immense pressure on the NHS creates delays in site opening and patient recruitment, creating a fundamental friction point in the biotech value chain.
A COVID-19 trial struggled for patients because its sign-up form had 400 questions; the only person who could edit the PHP file was a grad student. This illustrates how tiny, absurd operational inefficiencies, trapped in silos, can accumulate and severely hinder massive, capital-intensive research projects.
While the FDA is often blamed for high trial costs, a major culprit is the consolidated Clinical Research Organization (CRO) market. These entrenched players lack incentives to adopt modern, cost-saving technologies, creating a structural bottleneck that prevents regulatory modernization from translating into cheaper and faster trials.
While AI can accelerate the ideation phase of drug discovery, the primary bottleneck remains the slow, expensive, and human-dependent clinical trial process. We are already "drowning in good ideas," so generating more with AI doesn't solve the fundamental constraint of testing them.
While most focus on AI for drug discovery, Recursion is building an AI stack for clinical development, where 70% of costs lie. By using real-world data to pinpoint patient locations and causal AI to predict responders, they are improving trial enrollment rates by 1.5x. This demonstrates a holistic, end-to-end AI strategy that addresses bottlenecks across the entire value chain, not just the initial stages.
A-muto's CEO argues that shaving months off discovery isn't the real prize. The massive cost in drug development comes from late-stage clinical failures. By selecting highly disease-specific targets upfront, their platform aims to reduce the high attrition rate in clinical trials, which is the true driver of cost and delay.
With clinical development cycles lasting 7-10 years, junior team members rarely see a project to completion. Their career incentive becomes pushing a drug to the next stage to demonstrate progress, rather than ensuring its ultimate success. This pathology leads to deferred problems and siloed knowledge.
The company intentionally makes its early research "harder in the short term" by using complex, long-term animal models. This counterintuitive strategy is designed to generate highly predictive data early, thereby reducing the massive financial risk and high failure rate of the later-stage clinical trials.
The median $40,000 cost per trial enrollee is high because pharma companies essentially run a parallel, premium healthcare system for participants. They pay for all care and level it up globally to standardize the experiment.