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Regulatory News | 24 January 2017 | By Zachary Brennan
A potential President Donald Trump choice for US Food and Drug Administration (FDA) commissioner spoke with Focus on Tuesday.
Here’s a lightly edited version of the interview with Dr. Joseph Gulfo, the executive director of the Lewis Center for Healthcare Innovation and Technology at Fairleigh Dickinson University, the author of “Innovation Breakdown: How the FDA and Wall Street Cripple Medical Advances” and the former President and CEO of the medical device firm MELA Sciences:
Focus: First off, congratulations on being considered for FDA commissioner – can you talk about what kind of discussions you’ve had with the Trump transition team?
Gulfo: I don’t want to speak out of turn, but I had some people approach me and had some discussions.
Focus: You’ve written in the Wall Street Journal and elsewhere about the need for FDA to be overhauled and for red tape to be removed – can you elaborate on those plans? Are there specific types of biopharma and medical device companies that you think would benefit from such changes? What specifically isn’t working at FDA and for whom is it not working?
Gulfo: “I was commenting on Trump transition goals, if you will, about cutting red tape. I think the agency has the best intentions but there’s a fear – and even when they get it right they get beat up.
Out of that fear, the intent and letter of the original law [establishing FDA] is to promote health by ensuring safety and effectiveness [of medical products], but that has migrated over the years slowly and steadily…like a chronic, insidious disease. It’s been slow but I’ve watched it over my career. When I came into the industry – drugs were labeled for safe use and now there’s this absolute notion of safety, we’ve moved from safety and effectiveness…
And now safety has become a discussion of benefit and risk and in big diseases, this has become long-term outcomes and survival. That’s all happened out of fear and is not in the regulations. What I’ve proposed is getting FDA back to safety and effectiveness…and we can do this one of four ways: with biomarkers, clinical science and symptoms (ie. blood pressure, tumor size), with disease modification (bouts of migraines per month) and lastly with long-term outcomes.
I don’t think I’m calling for a major overhaul…what I love the most about being called the [former Supreme Court justice Antonin] Scalia of life sciences – I want to get back to original intent of the law. I want to get back to ways to not make FDA be in fear. When I was at the Senate Oversight Committee hearing last year, Sens. [Ron] Johnson [R-WI] and [Daniel] Carper [D-DE] both said the same thing to me and they flattered me saying they read every word of my testimony, and then they told me, ‘We invited FDA here and they wouldn’t come, and you basically said ….all we do is beat up FDA…we do call those gotcha hearings, and that has to stop…stop the fear.’”
Focus: Can you expand on this idea of using an “Internet of Things,” as you said in the WSJ op-ed to ensure patients and doctors have the best information? Do they not have the best information now?
Gulfo: “Maybe I didn’t artfully explain that. When FDA laws were written, if you wanted to get something written into a scientific meeting, you had to do it 10 months in advance. But now, if you have something profound, can be at the ACC [American College of Cardiology] meeting, for example, next Thursday…you can get really earth-shattering news out really quickly. I’m not saying we should ‘rely on it’ but suggesting we live in a different environment of information dissemination and that has to be considered in the way drugs get approved. Post-approval studies were only observational when I first started. Show us the data. Now, post-approvals are re-adjudications of initial studies…we need to get back to observational studies and disseminate that info quickly, via Twitter or medical conferences or wherever, but where we don’t need to re-adjudicate the information.”
Focus: You seem to be in favor of Right-to-Try laws at the state level, which skirt around FDA’s authority – can you elaborate on your position?
Gulfo: “At the Senate hearing I was at, I surprised everyone when I basically said something against Right-to-Try and everyone looked at me and I said I want to make it where it’s not needed, either via conditional approval, like in the EU…Right-to-Try is just a compromise, it’s not the first answer you’d want. But I sympathize with patients, and it’s a compromise.”
Focus: Can you talk about reforms you’d like to make to the US drug approval process? What’s the benefit of speeding up the approval process even more? What’s the benefit of not looking for long-term outcomes?
Gulfo: "FDA’s pathways have led to drugs that aren’t even being developed and innovation really happens in small companies. Look at last year’s approvals: There were none for cardiovascular disease and none for Alzheimer’s – as you’ve written, FDA can’t approve what they don’t have before them. But I was at a biotech showcase recently and saw 95 companies in cancer, ¾ of them developing a drug for AML and pancreatic cancer because there are significant, unmet needs there and these companies can gain incentives if they approvals....But the pathway is so onerous that you’re not even seeing the development…I believe, and I make my point with some hyperbole: The low-hanging fruit of single drug with single target, I’m not going to say it’s over, but maybe we’ll get one every five years. It’s not as easy as it used to be.
The only way you’re going to get a meaningful impact is with combination therapies, so some nuance here: if we get things on the market that show activity with a biomarker, always start with safety, and some activity, at least some patients helped, then get them on the market and then they can be used in combination with other drugs coming onto the market. Change the path and get more products that may only be a single, or a single stretched into a double, but get more out there and then we might hit some grand slams."
Focus: Do you have any concerns that speeding up approvals and increasing approvals would put patients at risk?
Gulfo: “FDA has an awesome, daunting responsibility and no, we don’t want to compromise that. We want safe products. No buts about that. I always go to the mission of the FDA in the law: To promote health. A client once said: there’s got to be a balance. I believe safety and effectiveness with yes, continuing safety, observational studies to better inform the label, pull it or black box it or what not is the way to go. I don’t want toxic stuff out there. I believe you can do it without putting people in jeopardy. FDA gets hit from all sides, groups say they’re not doing enough or moving too slow."
Focus: President Trump has made comments recently about cracking down on drug prices. Do you think this is an area FDA can get into?
Gulfo: “I think the FDA has a significant role in drug prices in three ways. If approvals on a calendar basis were shortened, even by just two years, there are models that the contribution of development time to approval would mean less of a need for price increases and more exclusivity…those two years would reduce the development expenses and then with longer exclusivity, companies can recoup their R&D funds easier.
Also, more approvals is more competitors which equals lower prices.
The third effect is the following: Companies raise prices to meet their quarterly earnings. I know because I’ve run companies and I would much rather meet my earnings with a steady stream of new approvals than raising prices.”
Tags: FDA commissioner, Gulfo, Trump and FDA