The Weird Idea That Could Speed Up Drug Approvals and Save Lives

The FDA has a fast-track system that gets animal drugs approved in 2 years instead of 10—but won't use it for humans, despite similar biology. Meanwhile, life-saving human vaccines stay stuck.

Photo by CDC on Unsplash

Two senators recently launched a bipartisan call for moonshot ideas to accelerate American science, asking questions like "how do we cut drug approvals from ten years to one?"

I’m glad we’re asking questions like these. Cutting drug development timelines would go a long way in getting more treatments, allowing non-mega-corporations to submit drugs, and upping our biosecurity resilience.

Right now, after proving a drug is safe, the FDA requires companies to prove efficacy through expensive, multi-year trials before approval. These studies often cost tens of millions of dollars and can take a decade to complete. Only the largest corporations can afford to try. Few patients can access experimental treatments during this period, even when they lack other drug options. The high cost of these clinical trials mean corporations must ensure a big financial return, which is why the adult tuberculosis vaccines is still in limbo years after early efficacy proof (the 1.25 million people dying from tuberculosis each year are in low income countries).

There are better ways to prove efficacy without the huge tradeoffs we’re paying now.

An Alternative Approach

Allow conditional approval based on a broader range of evidence—including real-world data from informed patients and promising biomarker improvements—rather than requiring companies to prove perfect outcomes first. Companies would demonstrate the same high safety standards to gain market access, then complete comprehensive trials within five years or face withdrawal.

This expanded conditional approval pathway would:

• Expand access for patients with limited options

• Reduce approval timelines through earlier market entry

• Lower development costs

• Encourage treatments for rare and neglected diseases that lack large commercial markets

• Generate a lot of data about disease and biomarkers

The FDA has some conditional pathways, but they’re very limited.

This expanded conditional pathway already exists. But it’s only for the FDA’s veterinary medicine approvals.

Borrow from the Veterinary FDA “Expanded Conditional Approval Pathway”

The FDA's veterinary division allows conditional approval for animal drugs that address unmet health needs. Companies demonstrate safety and reasonable effectiveness, get market access, then have five years to complete full studies or lose approval. Doctors and patients decide whether the risk profile is worthwhile.

A longevity drug for dogs recently got approved this way. A traditional clinical trial would have been impossibly long and expensive, requiring years of tracking and huge numbers of test subjects to notice gradual aging effects. After submitting 20,000 pages of evidence—including third-party research and effectiveness data, the FDA waived the full trial requirement. Instead, the FDA allowed the company to instead submit results about aging biomarkers in different types of dogs and efficacy data showing the drug reduced relevant aging biomarkers. This allowed the drug to be feasible and available for those who want to try it. The drug goes on the market after only two years—after safety, stability, and early efficacy data at a cost of ~$1 million.

Because of alternatives to the full clinical trial, a small company brought a treatment to market. Even if the drug ends up not as effective as early evidence showed, the study still generated real-world data on aging.

Targeting aging might sound outlandish, but any breakthroughs could have large impacts on treating age-related diseases like dementia, Parkinsons, etc.

Allowing for targeting biomarkers like this would also unlock treatment for more ailments. This veterinary conditional approval system has approved treatments for seizures, pancreatitis, and congestive heart failure in dogs—conditions that remain poorly addressed in human medicine despite similar underlying biology.

When Only Giants Can Play

With our current expensive system, only large corporations can afford multi-million dollar, multi-year trials, so only they decide which drugs get developed. And people are dying because of it.

Consider tuberculosis: A co-inventor of a vaccine that prevents over half of infections handed it to the pharma company GlaxoSmithKline (GSK) for development. Five years later, GSK still hasn't brought it to trial, choosing instead to focus on a shingles vaccine for wealthy, insured patients. Shingles causes far fewer deaths than tuberculosis (less than 100 US deaths yearly) but generates better returns.

Despite backing from multiple governments and the Gates Foundation, the vaccine remains stuck.

We could treat tuberculosis deaths with the urgency they deserve by considering giving people at high risk of tuberculosis the option to try it after safety testing.

We Might Be Structuring Trials Wrong

A growing consensus of the scientific community suspect our understanding of chronic conditions might be fundamentally wrong. Conditions like Alzheimer's, Parkinson's, and chronic kidney disease increasingly appear to be collections of distinct subtypes rather than single, uniform diseases.

If so, effective drugs will be doomed to fail trials. If treatments work for specific subtypes, the treatments will fail in broad trials that lump all patients together.

Physicians sometimes notice that certain drugs help specific patient groups, but lack resources to investigate systematically. We could accelerate discovering how they actually function. Real-world evidence would reveal the true patterns of human illness—if we let it.

I realize this is a crazy idea. But we live in shocking times where Strep A and Hepatitis C vaccines are achievable but won’t exist anytime soon. 800,000 people die from those diseases each year.

Drug quality and public trust in medications didn't collapse when we created this system for veterinary medicine. But this approach would still need to be paired with greater awareness and understanding of the nuanced role the FDA plays. But if we demonize the FDA or any government agency whenever rare adverse events occur for drugs they conditionally approve, we're biasing toward preventing deaths caused by the FDA instead of preventing deaths overall.

We should care about all these preventable deaths, not just the ones that make headlines.