Us2.ai receives FDA clearance for Us2.v2,transforming…

By Arthur Pappas, Pappas Ventures
February 1, 2012

A few tweaks to the approval process would help serve innovators and investors in a sensible way.

Despite recent sniping, the Food and Drug Administration doesn’t need an adversarial relationship with drug and device makers. We’re partners in a shared mission: to bring life-saving innovations safely and efficiently to market.

That mission can succeed under stringent standards. No one is calling for laxity. But it also takes transparency, a process that values innovation, and a smart balance between risk and reward.

With just a few changes, everyone—chiefly the public—would benefit.

Toward consistency and transparency. The clinical trial process is unnecessarily daunting when the goal posts change from one product to another, or from one phase of development to another. As science accelerates, a mid-course change is sometimes necessary, but the FDA has to judge whether such a twist outweighs the need to be consistent.

Here, the FDA needs to recalibrate. It should give more weight to the value of regulatory expectations that are clear and predictable. The agency can also promote fairness by respecting statutory timelines. Currently, it can “stop the clock,” restart it months later, and count a project as on-time. Our nation’s pipeline of new cures and therapies should run on real time.

Communication is not collusion. Today’s FDA is too wary of informal dialogue. The result means a delay, at best, and at worst a chilling effect that stymies useful conversation. The process would benefit from an “open file” approach that lets applicants learn what FDA scientists and consultants are thinking during the review process, not after approval or rejection.

Reform the appeal process. There are no official penalties for appealing an FDA determination. But FDA regulators are human, and we all know how relationships suffer when one person goes over another’s head.

A small, underfunded applicant may stake its entire future on its reviewers’ good graces. Both sides would be better served by a formal, non-confrontational appeals process. This could take the form of a rapid-response arbitration panel, or possibly even a market for appeal rights that combines the spirit of cap-and-trade with the peremptory challenges of jury selection.

Be aggressively progressive. The FDA works hard to reduce one kind of risk: bringing something unsafe or ineffective to market. It needs to work harder to avoid the opposite risk: delaying or rejecting innovations that people need.

The agency was founded to counter both threats, but its culture has swung too far toward holding things back. One solution is an aggressive program of “progressive approval,” that allows regulators to make case-by-case, risk-reward judgment calls and approve products for use when the evidence tells them the benefits likely outweigh the risks.

I’ve been on the front lines for both triumph and failure in this process. My firm played a part in one recent success story, a product that uses genetic targeting to combat melanoma. Its creators were able to keep up a robust dialogue with their FDA counterparts, and together, the innovators and the regulators brought physicians a new treatment much faster than usual.

Unfortunately, my experience tells me that’s an exception. I’ve seen the chilling effect of regulatory uncertainty at work. I’ve been in the room when we had to shut down funding for promising avenues of research because the path through the FDA was just too unclear.

By its own description, the FDA is “responsible for advancing the public health by helping to speed innovations.” It is meant to be both a policeman and a partner.

No one—neither venture investors nor the people in lab coats—wants to be hasty in the way we approve drugs or medical devices. We’re not asking for an easy process. We want a transparent, predictable one that lets innovators and investors plan and carry out their work in a sensible way.

Arthur Pappas is managing partner of Pappas Ventures, a Durham, N.C.-based venture firm that specializes in life sciences.