The problem with the FDA: Joseph Gulfo offers expert testimony before US Senate committee

Interview by Dan Landau


Dr. Joseph Gulfo, executive director of the Rothman Institute of Innovation and Entrepreneurship in FDU’s Silberman College of Business, was recently invited to testify as an expert witness before the U.S. Senate Committee on Homeland Security & Governmental Affairs in Washington, D.C. The focus of the hearing was on “Connecting Patients to New and Potential Life Saving Treatments.”

At the hearing, Gulfo argued that dysfunction on the part of the Food and Drug Administration (FDA) is preventing patients from accessing experimental treatments that could save their lives. He shared the gist of his testimony in an interview here:

Fairleigh Dickinson University: How did you come to be called as a witness?
Joseph Gulfo:
Sen. Ron Johnson, R-Wis., chairs the committee and his constituents raised the issue of the FDA obstructing access to experimental treatments and “right-to-try” laws (which allow terminally ill patients to try experimental therapies), and he decided to hold a hearing to investigate. At the same time as this was happening, I wrote a paper called “The Proper Role of FDA in the 21st Century” and Johnson was impressed with it and invited me to testify before his committee.

FDU: As you see it, what is the problem?
The mission of the FDA, as stated in the Food, Drug and Cosmetic Act, is to “promote health by promptly and efficiently reviewing clinical research. This includes ensuring that drugs are safe and effective.”

That’s a very simple mission; however, we have seen a steady erosion of the safety and effectiveness cornerstone upon which the FDA law has been built. Instead of just looking at safety and effectiveness, the FDA is requiring huge amounts of data on long-term outcomes and benefit-risk aimed at ensuring the drug improves the average patient’s lifespan. But the FDA is supposed to assure safety and effectiveness of drugs, not life outcomes for patients. This focus on the “average patient” masks positive outcomes for specific cases and deprives individual patients and doctors from making choices on what is best for that individual—that is precision medicine.

Over time, the FDA has increased the hurdle that new drugs must clear to garner approval. FDA routinely takes the position that new drugs must offer substantial improvement over existing therapies. This has the consequence of discouraging pharmaceutical companies from making drugs that treat illnesses which millions of Americans suffer from, like diabetes and cardiovascular disease. Instead, drug companies have a regulatory incentive to develop specialty for rare diseases with tiny patient populations (thousands, instead of millions) for which no other therapies exist.

Drug companies are like electrons and they follow the path of least resistance. In 2014, a record 40% of all new drugs approved by the FDA were orphan drugs. That number increased to 48% in 2015 and will likely continue to go up, unless the FDA makes regulatory incentives for companies to develop drugs for common ailments.


Above: The panel of experts testifying before the U.S. Senate Committee on Homeland Security & Governmental Affairs on “Connecting Patients to New and Potential Life Saving Treatments.” L-R: Darcy Olsen, president and CEO of the Goldwater Institute; Laura McLinn; Diego Morris; Joseph Gulfo, executive director of FDU's Rothman Institute of Innovation and Entrepreneurship; and Nancy Goodman, executive director of Kids v Cancer.


FDU: The FDA says it does this to make drugs safer. Why is this a problem?
If the FDA only reviewed drugs on the basis of safety and effectiveness, we would have more drugs available. With the FDA vetting drugs for clinical benefits and long-term outcomes, the development cycle is so much longer. Drugs can now take a decade to be approved by the FDA.

Also, by focusing long-term outcomes, the FDA is effectively keeping terminally ill patients from accessing experimental therapies. The terminally ill patient doesn’t care that long term use of the drug may result in heart problems—the terminally ill have only months to live anyway and they want to live a few months more.


FDU: What is the solution to the FDA’s mission creep?
We need to love the FDA. The FDA does great work and they constantly get beat up in the press, even when they do things right.

We need to encourage the FDA and show them the support they need to feel comfortable to promote health instead of protecting health. Promoting health is optimistic, while protecting health is a defensive posture. In the fear-based protect health model, safety and effectiveness no longer mean substantial evidence of a decrease in disease activity.

We need to redefine effectiveness for the FDA and we need to stop thinking that 100% safety can be guaranteed, because it can’t. The expectation that the FDA should be 100% successful has got to stop.

The FDA was invited to testify at this hearing, but they didn’t. Why? Because usually when they do come to hearings, grandstanding politicians attack them. We need to police the FDA and ensure it is doing its job, but we can’t keep calling them on the carpet and viciously abusing with congressional hearings that are nothing more than opportunities for politicians to score political points.

Things will go wrong and when they do, the FDA needs to be encouraged to continue to promote health.

Also, right to try laws would go a long way towards helping terminally ill patients access experimental therapies. Right now, 24 states have right to try laws on the books, with laws pending in 16 more states (including New Jersey).


FDU: Anything else?
Let me leave you with this story, which I think sums up my points:

I was in the elevator in my hotel in D.C. and I was holding my notes for the hearing. A man got on and saw me reviewing them and asked what my speech was about. I told him it was about the right to try initiative, which helps facilitate dying patients to get access to experimental drugs that have promise. A woman on the elevator with us jumped into the conversation, saying “the heck with waiting until I’m terminal, I want the drugs now!”

If we can get the drugs approved more quickly, then everyone can benefit from them.

Editor’s Note: A video of the hearing is available here. Gulfo’s testimony begins just before the 48-minute mark. More about Gulfo and his crusade to reform the FDA is available here.