5 Tips from Former FDA Commissioner Janet Woodcock on How to Win Over Regulators

The biggest mistake biotech entrepreneurs make when approaching the FDA? Trying to hide potential problems.

“I think that’s one of the biggest misconceptions—that you have to have the whole story complete before approaching us,” Woodcock said during a recent Cure webinar. “It’s much better to put it all out there and show the data.”

Woodcock spent decades at the agency, shaping nearly every major regulatory advancement in modern medicine. She directed the Center for Drug Evaluation and Research for most of her career, overseeing the world’s gold standard for drug approval and safety. She also led the therapeutics effort for Operation Warp Speed, helping bring COVID-19 treatments to patients.

In conversation with Cure CEO Seema Kumar, she shared guidance for founders navigating the FDA therapeutic application process.

Click here to view the full 60-minute conversation titled Navigating FDA Regulation, only accessible to Cure Members.

1. Don’t Wait Until Everything Is Perfect

Regulators understand the risks and unknowns when it comes to drug development, Woodcock said. Their main concern is ensuring patient safety while evaluating therapies that may offer new approaches.

“There’s always uncertainty and ambiguity in drug development, that’s why it’s risky,” she said. “Trying to put on the best face possible isn’t the best strategy with the FDA.”

RELATED: How Decentralized Clinical Trials are Reshaping Biotech Strategy

2. Present a Balanced Case

Woodcock said FDA reviewers are evidence-driven; they start by looking at the unmet medical need, then examine what alternatives patients have. After that, they dig into the applicant’s data.

"Companies shouldn't overstate their case; a balanced presentation builds credibility," Woodcock said. "If reviewers raise a potential risk, don't dismiss it—evaluate it seriously."

The green flag the FDA looks for? A company that presents both good and bad news honestly. The best approach, she said, is to talk about potential harms and how you have or will try to address them. In other words, show that you’ve done your homework.

"Give a balanced, scientific presentation—that instills confidence," Woodcock said.

On the flip side, if you can't answer basic questions about your preclinical work, you will lose credibility.

3. Understand the Risk–Benefit Calculation

The FDA's tolerance for risk depends heavily on what you're treating. For cancer, rare disease or fatal conditions with no or few treatment options, patients have made clear they're willing to accept more uncertainty for a chance at relief. But for common conditions with existing treatments, the bar is higher.

“The balance is different for cancer than for headaches,” she said. “Founders should think about the benefits versus the potential harms—that's how FDA looks at it."

While patients may be willing to accept more risk, regulators may balk. The balance is different depending on the disease area and the reviewers' experience, she said, suggesting sometimes you need to educate reviewers about the burden of the condition you're targeting.

RELATED: FDA's Latest Move Could Pave the Way for Cheaper Complex Medicines

4. Know When to Challenge Guidance

FDA guidance documents include a disclaimer: This is our best thinking, but you can do it another way if you have a better idea. This matters especially for new modalities like gene therapy or CRISPR where precedent is limited.

"If a company is convinced that the advice they're getting is too conservative or not the right method, they can and should propose alternative approaches—and explain why they'd serve equally well," Woodcock said. "Scientifically, you shouldn't use the wrong method just because you're used to it," she said.

But don't go rogue, she said. Make a proposal and explain your reasoning. Work with the agency, don't work around it.

5. Think Beyond Traditional Trials

For rare diseases or precision-medicine applications, traditional randomized controlled trials often are not feasible because sample sizes are too small. The FDA recognizes this, Woodcock said, and is increasingly receptive to alternative approaches, especially those driven by patient groups.

“When groups propose trial designs or endpoints appropriate for their disease, that’s powerful,” she said.

She said to remember reviewers share your goal of improving health, but they won't be as excited as you are about your molecule. Their primary allegiance is making sure first-in-human studies are as safe as possible.

Understanding that perspective, and working with it rather than against it, is the key to a productive regulatory partnership.