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The Trump Administration has signaled it intends to revamp the Food and Drug Administration to speed the approval of drugs. Some FDA commissioner candidates have proposed radical reform, including an end to the requirement that drugs must be effective to be approved. Experts discuss what reform might look like and what the FDA needs to better succeed.

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Peter Pitts, President, Center for Medicine in the Public Interest and former Food and Drug Administration Associate Commissioner

Dr. David Gortler is a professor of pharmacology and a former Food and Drug Administration senior medical officer, now a Pharmacology expert and FDA expert with FormerFDA.com  He was also the former FDA/health care policy and Obamacare/ACA repeal and replacement advisor for the 2016 Ted Cruz presidential campaign

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Remaking the FDA

Reed Pence: Healthcare is one of the functions of the federal government that President Trump promised to shake up most. Dismantling the Affordable Care Act has gotten most of the headlines, but the new administration is also considering major changes to an agency that has its hand in virtually ever Americans life – The Food and Drug Administration.

Peter Pitts: The FDA is a very tough job; it regulates about 30% of the whole US economy beyond just drugs, also food and medical devices and cosmetics and dietary supplements, but when it comes to drugs what a lot of people don’t understand is that one of the FDA’s key jobs is make sure that the drug isn’t just safe – but safe and effective. And in these days of very large molecule drives drugs, drugs for serious life threatening disease, it takes time – the science is extremely difficult.
Pence: Peter Pitts is President of the Center for Medicine in the Public Interest and a former Associate Commissioner for the FDA.

Pitts: The last time most people touch science was in high school where there’s a right answer and a wrong answer. When it comes to the type of regulatory science the FDA has to do, it’s extremely difficult – there are nuances, there’s really a direct right or wrong answer, so these things take time and I think what people want is for the FDA to get it right, not necessarily not faster – certainly faster if they can do it “as well” but safety comes first. I think no ones going to argue about that.

Pence: But just “what” constitutes safety can produce plenty of arguments. Dr. David Gortler is a former FDA medical officer and former advisor to the FDA and healthcare team of presidential candidate Ted Cruz. He says the agency has approved plenty of drugs whose safety is debatable. One example, he says, is a drug called Addyi.

Dr. David Gortler: I was pretty surprised that this drug was approved cuz, this is a drug which according to the Journal of the American Medical Association, only worked 8-12% of the time in women, with female sexual dysfunction – with anorgasmia. And, maybe that’s not so bad but the problem is this drug has some very, very serious safety concerns. And not to exaggerate or overstate the facts at all but if a woman, while taking Addyi, were to take some of the most common antibiotics out there, the drug interaction could cause them to die. If they were to take this drug with recreational amounts of alcohol use, again, the drug interaction or the alcohol interaction could cause them to get very sick and die. And so, the bottom line is with that drug talking about something which doesn’t really work very well 8-12% of the time, that means almost never works and there’s some very significant safety risks. I don’t understand why the FDA would approve a drug like that.

Pence: But Pitt’s says that’s the kind of decision that makes the FDA’s job difficult.

Pitts: All drugs have risks as well as benefits, some more than others, and it’s the FDA’s job to weigh the risk and benefit based on the severity of the disease. So for example, not going approve a drug for headaches that have a 20% chance of death, but when it comes to ovarian cancer for example or lung cancer, the FDA is regularly approving drugs with a high mortality because the death rate for the disease is so high. So again, it comes down to a balance. I think the FDA does a pretty good job, could they do a better job? I think they definitely could, because they could play better intramural and by that I mean, they could do more work in collaboration with academia, with industry, with patient groups to understand the new types of tools that can be brought to bear drug regulation.

Pence: Among the new tools, biomarkers, which would allow drug approval based on statistical analysis.

Pitts: These tools have to be validated, but I believe that the FDA can work with outside bodies to validate these individual biomarker tools faster so that more of them can be used to do better research. And I think ultimately, one of the first jobs of the FDA is to help people that are developing new drugs to fail faster – if you fail faster you can invest billions of dollars that you’re saving in new types of drugs. And I think that’s crucial, the FDA has to be both a regulator ‘of’ and a partner ‘with’ industry and it’s a tight rope, I understand that, but the FDA needs to embrace that concept.

Pence: Gortler says it would also be nice if FDA approvals relied a little more on the reviewers who spent months pouring over the evidence. He says too often their recommendations are overturned by top brass, he says the FDA could also remove testing hurdles that exist partially because they’ve simply always been there.

Gortler: The FDA could always be working a little faster, they could always be doing things a little bit better, even when it comes to some of the preclinical testing on animals – I mean nobody really likes that preclinical animal testing and I can tell you right now a lot of those animal tests really just aren’t very useful. They don’t elucidate that much information when it comes to clinical testing, but these are legacy tests which are required by the FDA and many animals are harmed or killed testing drugs meant for people.

Pence: The FDA has been under pressure for some time to approve drugs faster, it’s one reason President Trump said he wants to reform the FDA and speed or a lack of it in its approval process is perhaps the biggest criticism of the agency. However, Pitt’s says simply regulation won’t speed up the words and he says it certainly won’t take cost cutting.

Pitts: If you want the FDA to do a better job that means doing it faster, and faster means being able to hire more staff to do it appropriately and that’s not being discussed. You know, nothings for nothing and people need to understand how this thing works. It’s easy to say, “the FDA is standing in the way of progress” when you say we want to have less regulation, we want to have less spending, we want fewer employees – I understand that from a philosophical position but actions have consequences and if you don’t have the bodies you need to get the job done with excellence, you’re not going to get the results that you want.

Pence: But to truly be a partner with a pharmaceutical industry, Pitts says the agency needs more than just speed.

Pitts: I believe that what the pharmaceutical industry wants more than anything else isn’t speed, although speed is important, what they really want is a greater degree of predictability. Because predictability allows them to invest their research and development funds more smartly to understand what’s gonna happen all throughout the process. Now the bad news is that regulators on the other hand, love ambiguity because ambiguity gives them endless power to basically do whatever they want. Predictability begins to draw pretty clear white lines around the regulatory process; so that things can be done in a solid predictable manner and over and over again, industry has said what they really want is a more predictable FDA. That way they know what to do to get programs to move forward. Politicians talk about speed because its something people understand – kind of a general talking point that “Faster is always better” – what industry wants in term of being able to invest more smartly to develop better clinical programs is a more predictable FDA.

Pence: However, pharmaceutical companies seem reluctant to support some of the most radical proposals for FDA reform. Among those that have been under consideration for FDA commissioner are those that would reduce drug approval to a ‘Yes’ or a ‘No’ on safety only. It wouldn’t matter if the drug works or not. The FDA would have no say over that and it would be up to consumers to watch out for themselves. Both Gortler and Pitts say that would be dangerous.

Gortler: I don’t know if I’m really prepared to start letting, you know the average, consumer make complicated pharmacological decisions about pharmacogenomics, and long term safety, and dosing, things like that.

Pitts: That’s naïve and foolish and dangerous, because you can’t really ever separate safety from risk, it’s a team – Safety/benefit, risk/benefit, it comes together. Allowing drugs to go on the market that are viewed only on safety, I don’t even know what that means, because safety trials are done very very early in the development process so I don’t see how you can even brings a drug to market and give it a label that can be understood by doctors and patients because we wouldn’t know what it does. It sounds sexy to say “we’re gonna get this drugs approved quickly, because they’ll all be safe,” that really means nothing; it’s a meaningless statement.
Pence: Supporters of such an idea say the market would steer consumers to the drugs that work. Some of have suggested that the equivalent of a yelp for drugs would be more helpful than the FDA is. But pharmaceutical companies like having the FDA there to keep snake oil off the market. And Gortlers says if safety were the lone standard for drug approval at least it would force the FDA to get its act together on that.

Gortler: One of the things that could be potential good outcome of that, is that the FDA would have to beef up their safety testing and right now I can tell you, the FDA is doing a very bad job of measuring its safety. What we have now is the FAERS or the AERS database, which stands for the Adverse Event Reporting System, and they’re just according to the official of the FDA, they say they only about a 1-10% of adverse events that occur in the general public are ever collected. So one of the good aspects that can be on letting a drug go after safety testing is that the FDA would have to do a much much better job of collecting safety data.

Pence: Speeding up the process or approving more drugs by ignoring effectiveness, may be too much for people to swallow. But are those the right yardsticks to use to judge the FDA?

Gortler: I’ve read in a bunch of places online, that the FDA in 2016 have had it’s lowest number of approvals in the past 6 years and that’s really not a great metric. You don’t just look at the overall number of approvals like the overall number of widgets that are produced, it really matters what those drugs are for. What I hope will be done – this is something we depend on the drug companies for – is to have more of a blockbuster drugs, which we really need cures for. Cuz there’s still some very serious conditions out there and we haven’t really had a really good blockbuster drug in a long time. Some of the major killers in the United States include heart disease, and Alzheimer’s disease, and stroke, and diabetes, and certain kinds of cancer. I think its important not just to look at the quantity, but the quality.

Pence: Another possible change? Perhaps the FDA will let drug companies promote off label uses of drugs approved for other purposes. Now they can’t, the doctors use drugs for unapproved purposes all the time.

Pitts: Companies want to do more because they’re saying, “listen, we have legitimate information about how our drugs work in the real world that’s peer reviewed, its truthful, accurate, it’s non misleading and there’s significant value to the public to be able to share those with doctors and patients.” And what the FDA has said is, “we want to work with industry to get this done responsibly,” and I believe that they will. They’re really holding back, this goes back to the issue of predictability vs. ambiguity – the FDA, I think unfortunately, is allowing these rules to remain ambiguous; they can deal with these cases one by one as they see fit and the federal courts have seen otherwise. The federal courts have said, the FDA has no authority to ban the sharing of truthful, accurate and non-misleading information. So there’s still a ways to go with having industry and having the FDA work together to create rules and regulations to do this responsibly because I don’t believe the way to get this done is through the courts, you want the players who are involved to do it between them rather than having a judge flip a coin and decide which way to go.

Pence: The FDA can still be a protector of the public health and safety without coming off as the food and drug police, but that will take credibility. It’s one reason that Pitts believe the commissioner’s job should be filled by a doctor.

Pitts: I think it sends a message to the staff and to the public that they understand, first and foremost, that the job of the FDA commissioner is to be a steward and a guardian of public health. Secondly, I think we need somebody who understands the staff inside the FDA, the career staff, and the process. Because if you don’t, you have somebody walking into that chair who has a tremendously steep learning curve and even the best commissioner is gonna serve 8 years and that rarely ever happens, you really want somebody who can step into the job, knows the players, knows the process, has an agenda and can make it happen. One of the things I understood when I worked at the FDA is that you can’t walk into the FDA and be confrontational, that way you fail. But if you walk in and you’re confrontational, the senior staff says, “listen, I was here before you got here, I’ll be here after you leave, See you later” – you need to be a partner and a leader to get people to bind to your agenda.

Pence: So even when the public health is at stake, politics are not far away. You can find out more about all our guests on our website, RadioHealthJournal.net. I’m Reed Pence.

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