Platform Trials as a Service: A CRO Model for Rare Diseases?

In the mid-1990s, Ford spent four years building the new Taurus. Before a single car hit the market, they made 99 prototypes. Each one cost $400,000 and took weeks to assemble. Only when the assembly line was ready could they build a car for $20,000 in ten hours.

That’s the difference between prototypes and production. Prototypes are expensive, slow, fragile. Production is efficient, repeatable, scalable.

Clinical trials today are still prototypes. Every program is hand-built from scratch: new sites, new protocols, new control groups, new data systems. It’s artisanal craftsmanship at industrial scale. And like Ford before the assembly line, it’s slow, costly, and fragile.

Most companies still run trials this way. They set up their own sandbox, enroll their own patients, recruit their own investigators, and carry the entire cost. It’s as if every carmaker had to pave its own road before testing a model.

That’s the status quo. We barely notice how wasteful it is because it’s all we’ve known.

But what if we built machinery that enabled the conduct of clinical trials at a 20-fold efficiency, just like Ford did with cars? That’s what platform trials promise.

Platform Trials

Platform trials flip the model. Instead of building a new road each time, you build one highway and let many cars run on it. A shared backbone. A single trial where multiple therapies can be tested at once, often against the same control group. That means faster recruitment, fewer patients stuck on placebo, lower costs, and more shots on goal.

This approach is relevant as pharmaceutical and BioTech pipelines are increasingly chasing the same targets. Here Cremieux, commenting on the L.E.K. analysis:

Considering the data, the Platform Trial approach is needed! And the concept is neither new nor theoretical. Versions of this model are already alive in the wild. Take I-SPY 2, the breast cancer platform launched more than a decade ago. Born at UCSF in collaboration with the FDA, it blurred the line between academia and industry. Companies like Genentech, Pfizer, and Amgen didn’t have to spend years setting up bespoke trials. Instead, they plugged their molecules into the I-SPY 2 infrastructure and got answers faster, at lower cost. What started as an academic experiment became a hybrid machine where pharma footed the bill but benefited from a smarter, shared testing ground.

Glioblastoma, one of the most brutal cancers known to medicine, has its own version: GBM AGILE. Organized by a nonprofit consortium, it was explicitly designed to welcome commercial entrants. Bayer and Kazia Therapeutics are among those who’ve slotted their drugs into it. For smaller biotechs, it opened a door that would otherwise have been locked. They could never afford to build their own global glioblastoma trial; AGILE made it possible.

In the emergency of COVID-19, the adaptive platform trials RECOVERY in the UK and ACTIV in the US became household names, government-led, fast-moving, decisive. But even here, companies ran their own versions. Gilead, for instance, structured its remdesivir program with an adaptive backbone that allowed them to pivot quickly. It wasn’t a fully open platform, but the DNA was the same: reuse infrastructure, move faster, avoid dead ends.

Most recently, Roche and Day One Biopharmaceuticals launched FIREFLY-2, a global platform for pediatric high-grade gliomas. Built to be continuous, it’s designed to test multiple investigational agents over time. This is a glimpse of the future: a commercially driven platform operating not as a one-off, but as a standing backbone for ongoing discovery.

And yet, these are the exceptions. When you step back, the balance is clear. The vast majority of platform trials have been initiated and anchored by academics, governments, or nonprofit consortia. Industry’s participation, while growing, is still hesitant. Pharma tends to treat platforms less as infrastructure to co-own and more as convenient bolt-ons, temporary roads they can drive on until the next bespoke trial is built.

Why? The reasons are depressingly familiar. The industry is fragmented. Small biotechs don’t have the money to build platforms. Large pharma is siloed, with each therapeutic area guarding its turf and reluctant to cede control. Investors prefer quick milestones over long-term infrastructure plays, so executives shy away from explaining why they’re funding a platform instead of another trial. And even if the will existed, governance questions loom: who owns the master protocol, who controls access, how results are shared, how intellectual property is protected. Without a neutral builder, the coordination rarely materializes.

And it is here, in rare diseases, where the absence of platforms is felt most acutely.

Rare Diseases

A rare disease sounds… rare. But together they’re common: one in ten Americans lives with one. Yet fewer than 10 percent have an approved treatment, and only 22% have even been studied in trials.

On paper, the rising FDA approvals rate, from 29% of all FDA drug nods in 2010 to 54% in 2022, looks encouraging. Yet for every win, dozens of programs collapse. Giants like Pfizer and Takeda have scaled back. Small biotechs face brutal economics. Investors treat rare diseases as danger, not opportunity.

The reason is structural. Clinical trials for rare diseases are a logistical nightmare. Patient populations are tiny and scattered, often too sick to stomach placebo arms. Recruitment is brutal. Thirty percent of rare disease trials fail before completion, with poor enrollment a leading cause.

Per-patient costs are crushing, especially for lean biotech startups. Add to that the economic headwinds: post-pandemic valuations for rare disease-focused firms have plummeted more sharply than broader pharma sectors.

Legislation tries to help but often misfires. The 1983 Orphan Drug Act pulled tax breaks and exclusivity to spur development, and it worked, sort of. But the Inflation Reduction Act, meant to tame drug prices, exempts orphan drugs only if they stick to one rare indication. That’s like telling a startup to build one feature and never iterate. It blocks the efficient repurposing of drugs across multiple uses, a common path to broader impact. It makes rare diseases less market-sexy… again.

So how does one act when both patients and resources are rare?

One answer is regulation. If it’s unethical to spread tiny patient populations across dozens of trials placebo arms, then regulators could mandate a master trial structure for certain rare diseases, a standing platform companies must join. Enrollment would be pooled, control arms shared, and every six months (as example) new investigational therapies could enter the system.

But governments have proven unable to use regulations for efficiency, quite the opposite. Regulations rarely streamline; they usually complicate and add burden. So, I’m not a fan of that.

Another answer lies in the market itself. Contract research organizations could offer platform trials as a service, becoming trusted third parties that manage logistics, protect intellectual property, and balance competing interests. For big pharma, this might feel unnecessary. But for early-stage biotechs with limited resources, the ability to “subscribe” to a ready-made trial infrastructure could be transformative.

Whether regulation forces the change or CROs seize the business opportunity, the outcome is the same: a backbone that makes rare disease trials possible at scale.

The gains are obvious: shorter timelines, lower costs, greater patient willingness, standardized execution, lower entry costs for biotechs, derisked incentives for pharma. Most importantly, for patients, it means shaving years off the wait for treatment.

Platform trials won’t make rare disease drug development easy. They’ll make it possible. And that’s the leap, from hand-built prototypes to scalable infrastructure, that clinical research needs. We’ve done it before: Ford with cars, everything we order and get shipped home. Multiple products relying on one infrastructure. So why not for clinical trials, especially in rare diseases?