“The Food and Drug Administration (FDA) is responsible for protecting the public health by ensuring the safety, efficacy, and security of drugs, biological products, and medical devices.” It’s right there in their job description.

But with the release of new policies meant to promote innovation for patients with unmet clinical needs, the FDA is asking the American people to “accept some level of uncertainty” when it comes to a handful of gene therapies that have been fast-tracked through the approval process.

By encouraging drug makers to use available incentives tied to rare diseases and orphan designations, the FDA is seeing an explosion of gene therapy applications and fast-track approvals. As if allowing these therapies to come to market sooner by avoiding the time and costs associated with the traditional clinical trial process isn’t enough cause for concern, the FDA doesn’t even consider the issue of value/fair pricing—a standard requirement in other developed nations.

So why would the FDA feel compelled to sacrifice clinical certainty and stay silent on medications with price tags upwards of $2 million? The answer seems simple— the target patient population isn’t large enough to warrant such rigorous reviews.

With the U.S. providing an open door and open checkbook, why aren’t drug companies serving all known cases? Instead, they’re initiating a lottery process for patients in countries where these same medications are not approved yet.

There’s no question—we all want to find a way to end debilitating diseases. Agreeing on the best path to achieve that goal is another story. In the past, it was always pretty clear when patients were taking experimental drugs. Now it seems like new-to-market therapies are in many ways experimental by design.

On one hand, we have the traditional, more deliberate approach to drug approvals with free patient access through controlled, reportable clinical trial experience. And on the other, an accelerated path with sky-high price tags and loosely reported, post-marketing clinical experience.

Ten percent of FDA approved drugs are later pulled from the market under the traditional model. In time, what will we learn about the efficacy and value of medications approved under this new model? I’m concerned the emphasis on speed-to-market in the name of innovation and helping desperate patients may really just be about speed-to-profitability.

~ Kelly Prymicz, PharmD, RPh

Kelly Prymicz, PharmD, RPh, is a consultant with the Chelko Consulting Group where she helps employer-sponsored health plans better understand and manage quality of care and waste issues associated with the prescription drug spend in their medical TPA and PBM administered benefits.