Faster Cures and the Costs of Medical Regulation

I think that it's no great surprise that many people see the US Food and Drug Administration (FDA) and its ilk in other countries as a gargantuan ball and chain dragging down progress. Yet few of these take the fully libertarian position that the FDA should be removed and the demand for safety assurance provided by a marketplace of review and certification organizations. Instead most such advocates argue for a return to the smaller FDA and much less onerous review process that existed in the past. They note that FDA administrators have perverse incentives to block as much progress as possible, and that they have followed these incentives across recent decades to greatly increase the amount of time and money required to obtain approval for new medical technologies.

FDA bureaucrats are blamed for letting through any technology that causes even a tiny amount of harm, while receive no personal benefit for approving something that is safe, and receive no personal penalty from slowing down or blocking perfectly safe technologies from approval. There is no such thing as a perfectly safe medical technology: it is always a cost-benefit analysis for even the most beneficial technologies developed to date, and the mass media tends to inflate every harm done without taking account of the benefits. Everything else proceeds from that, and the consequence is that ever fewer new medicines are approved, there is less funding for development, and many lines of research are abandoned because the cost of regulatory compliance has become too great.

The situation is actually much worse for aging research, as the FDA doesn't recognize aging as a medical condition that can be treated; there is no path through the regulatory process to obtain approval for a treatment for aging, and that fact echoes back down the funding chain to make it much harder to raise money for projects such as SENS rejuvenation research. Potential technologies for the treatment of aging would at best have to be shoved through the approval process as narrow therapies for specific age-related conditions, which may well have a distorting effect on development. Some people are less concerned by this than I am, but changing what the FDA considers to be a disease is a long process of lobbying. This costs money and time that would be better spent on research. Just look at the years of attempts to get FDA bureaucrats to consider sarcopenia a medical condition: that is still going on, with no end in sight.

A number of political advocacy organizations have been founded in past years with the intent of trying to cut down the influence, size, and costs imposed on medical development by the FDA. In most cases they are focused on the time cost rather than the financial cost, possibly because that is an easier rallying cry. These organizations are one of the expressions of frustration with a regulatory process run wild, that serves only itself, and is causing far more harm than good. People don't see the invisible cost of medicines not development and technologies delayed for years. Faster Cures is one of the earlier organizations, now with interests in many approaches to speeding up research, including the venture philanthropy ideals that have been expressed by Peter Thiel for some years. But today I'll point out another lobbying organization created by those frustrated by the FDA and its baleful influence on the pace of medical development:

Tomorrow's Cures Today Foundation

Millions of Americans are in pain and suffer needlessly. Thousands of Americans die unnecessarily as they wait for promising new drugs to make their way through an unnecessarily long approval process. What we see is death and suffering that is attributed to approving drugs with dangerous side effects; but what we don't see is the death and suffering due to regulatory delay. Those victims are invisible.

The Pathway to Faster Cures

Rob Donahue used to ride horses. He was a modern-day cowboy until he was stricken with amyotrophic lateral sclerosis (ALS). Now his muscles are weak. He can't ride horses anymore. And his condition is worsening quickly. ALS will degenerate Donahue's neurons and nervous system, and he will probably die in less than five years. Another ALS sufferer, Nick Grillo, is trying to change all that. He's put together a petition on Change.org to urge the FDA to fast-track approval of a new drug, GM-604, that would help people like Donahue and others like him. "People can't wait five, ten, 15 years for the clinical trial process," said Grillo. "Things need to happen much quicker." But ALS is just one illness, and GM-604 is just one medicine. There are thousands of Americans suffering -- many with terminal illnesses -- while waiting on the FDA approval process.

A paradigm change is essential because FDA culture has led to a situation where it costs an average of $1.5 billion and 12 or more years of clinical testing to bring a new drug to market. Medical innovation cannot thrive when only very large firms can afford to research and develop new drugs. Another problem is that the FDA's first goal is not to maximize innovation, but to minimize the chances that an FDA-approved drug leads to unanticipated adverse side effects and negative publicity. In particular, the FDA's efficacy testing requirements have resulted in an ever-increasing load of money and time on drug developers. We can't count on FDA bureaucrats to fix the broken system they created. Even Congress, whose cottage industry is to regulate, admits that the current FDA system is a roadblock to fast-paced innovation.

The missing seat at the table is for someone who represents freedom -- that is, the right of patients, advised by their doctors, to make informed decisions as to the use of not-yet-FDA-approved drugs. Absent from the congressional hearings over health care, however, has been a freedom agenda, specifically one designed to eliminate the FDA's monopoly on access to new drugs. We hear very little about those who suffer and die because they were not able to access drugs stuck in the FDA's testing pipeline, or about drugs that were never brought to market because FDA procedures made the development costs too high. There is an invisible graveyard filled with people who have died because of drug lag and drug loss. The FDA's deadly over-caution is why venture capitalists shy away from investing in biopharmaceutical startup firms. Venture capitalists are willing to take big risks on ideas that may fail. But failure due to regulatory risk is just too big a hurdle to overcome. Capital providers have other opportunities, even if those opportunities don't involve cures for disease.

High costs and slow innovation are the hallmark of a monopoly. And, as medical science continues its rapid pace of innovation, the cost of lost opportunities for better health will increase even faster. The solution is to introduce consumer choice and competition. FDA proponents would bolster the fear that "unsafe" drugs could flood the marketplace. But the FDA cannot define what is "safe." Only patients with their unique health conditions, treatment profiles, and preferences for taking risk can define what is safe for them.

Comments

One of my bigger fears as far as aging research and therapies goes are the regulatory bodies like the FDA. If nothing changes, and these therapies come to fruition, then what happens? Medical tourism for everyone? Will big pharma companies open up shop in more lax countries to provide treatment? I know groups like Bioviva want to do their tests offshore, then go to the FDA with their positive (hopefully) results.

Posted by: Ham at June 11th, 2015 8:26 PM

Ham, what's the problem with medical tourism for everyone? Travel is cheap in comparison to disease and treatment.

Given the possibility of development and treatment anywhere in the world, the world is closer to the fully libertarian position than one might think given the existence of the FDA and similar, and one might conclude that "[lobbying] costs money and time that would be better spent on research", including lobbying with zero chance of obtaining its stated goal such as eliminating the FDA.

Idle (because I haven't done more than a naive search and am not well read on medical tourism) question: is anyone meeting "the demand for safety assurance provided by a marketplace of review and certification organizations"? Naive searches turn up rating sites, but I have no idea which if any are useful. My bias is that demonstrating and improving how marketplaces satisfy demand for safety and efficacy in a competitive marketplace (global medical tourism) would in the long term be more useful than merely advocating for deregulation (of whatever degree). Though perhaps still not as useful as research. :)

Posted by: Mike Linksvayer at June 11th, 2015 10:57 PM

"High costs and slow innovation are the hallmark of a monopoly."

Like the monopoly of a patent?

"FDA proponents would bolster the fear that "unsafe" drugs could flood the marketplace. But the FDA cannot define what is "safe." Only patients with their unique health conditions, treatment profiles, and preferences for taking risk can define what is safe for them."

Yeah, that's because so few people use homeopa... oh, wait!

Posted by: Antonio at June 11th, 2015 11:16 PM

It's sad that companies are having to jump through hoops to get these things tested but whats the alternative? Like Liz Parrish from Bioviva says ten years and a million plus dollars to develop a single new drug is just unworkable. Take into account the failure rate on translating drugs from mice to men which is very high you might be looking at a handful of new drugs in that time.

Right to try needs to be seriously ramped up, people have the right to try these therapies and many want to do so but red tape stops them doing so! Their life their right!

So the alternative is offshore testing and proof of Efficacy in the hope the FDA sees the results and agrees to fast track the therapy to a reasonable time scale. Companies like Bioviva are going offshore with an IRB and FDA overseen tests so things can happen faster. This also has the advantage in helping those like Dr Fossel who are taking things like Telomerase therapy to the FDA under an IND.

I wonder how SENS factor in the time to deliver therapies and if Aubrey's prediction of 25-30 is due to the drag factor of the FDA? How much faster would SENS deliver with a far less restrictive process? What if the FDA only enforced safety not efficacy?

Simply put we cannot continue with the current system and the paradigm needs to change. The pace of progress is advancing rapidly and is in direct conflict with the infrastructure in place.

Posted by: Steve H at June 12th, 2015 3:43 AM

Great article, Reason. Thank you.

The problem we see in many areas of research is the high hetereogeneity of patients and the lack of regulatory authority to account for this. Population based studies always try to find wonder drugs, which work in all humans. This not only gets harder and harder but it also prevents individual patients from getting the drug they need because. Trastuzumab for breast cancer would have never been approved if patients weren't stratified based on HER2 status. The problem we have with many diseases is that stratifying is difficult or not possible and it can dramatically slash the number of possible customers. Therefore it gets unattractive to go for FDA approval because the costs are so high. In the end only patients and doctors should decide what drugs they use. I find it atrocious that the FDA can decide over life and death of patients. The FDA has no right to prevent a patient from taking a risk instead of dying from a disease.

Posted by: Waverunner at June 12th, 2015 6:46 AM

I don't think Aubrey's prediction of 25-30 years had much to do with FDA slowdown. It's possible, but at the same time, they still don't even consider aging a disease yet. Which is why I've been wondering what's going to happen when SENS, Calico, or one of these Pharmaceutical companies actually releases some sort of treatment. Like I asked earlier... are they going to open up shop off shore? Or will they release things as a "nutraceuticals"? Could it all be for nothing if the FDA won't approve any of it?

I'd really like to see some results from bioviva. In that conference video, Liz Parrish said they were going to have age reversal proof within a year, and that they were going to take that to the FDA. We'll see. I'm still going with the assumption I'll have to partake in medical tourism to get something done years down the line, because the FDA isn't likely to change and I'm ok with that. I kind of have to be, I guess.

Posted by: Ham at June 12th, 2015 11:29 AM

Let’s not do away with the FDA. Instead, let’s remove its authority to dictate what treatments may or may not be used and make the FDA an advisory agency instead.

As an advisory agency the FDA’s mission would be to ensure that patients received complete informed consent. Drug companies would be obliged to label their products with statements that accurately and clearly describe what is known about the drug’s safety, effectiveness and side effects. The statements themselves would have to be approved by the FDA. The well-informed patient would then have the option (in consultation with his/her doctor) to either use the drug or not.

This system would stop the FDA from acting like our parent – making decisions about what is good for us or not. Drug companies could still work toward earning the coveted “FDA approved” label which would certainly improve the drug’s marketability. But until then, the drug could still be marketed to informed patients who deem the drug’s effectiveness worth the risk.

No false or misleading advertising allowed. Any liability would fall on the patient who made the informed decision to use the product.

Posted by: Allen Alger at June 12th, 2015 4:11 PM

As soon as we have a treatment that by all accounts does work, that does reverse aging by, say, 30 or so years (enough to enter the longevity escape velocity), all hell is going to break loose. It's going to be the news of the century and it's going to change everything. At this point many are going to find it unacceptable that FDA stands in the way, or for that matter that only the well-to-do might be able to afford it.

Posted by: Northus at June 12th, 2015 10:10 PM

Governments are unable in the current climate to fund the kinds of radical science we are hoping for. Reason and others here are completely right the current situation is a joke.

What will happen if and when SENS or Calico or someone else in the US develops a working therapy? Can we expect a ten year delay from the FDA? Can we expect it to be blocked by political interest? Will it trigger calls for a moratorium like CRISPR has which will further delay things as they are endlessly debated? Gene therapy is struggling due to the same hesitance to move forward.

People who try to fight the system and rail against it will ultimately get nowhere, we need to change that system from the outside. Bypass it, and ethically demonstrate the technology.and the rest will follow. I believe that advocacy, grass roots funding and private enterprise will deliver the first pass rejuvenation technologies, it is up to us to do what we can to help that. We can change the system from the outside.

A single demonstration of robust rejuvenation in a human is all we need, show it to the world and watch the system change as the public demand those changes. That is the Catalyst in my opinion and nothing short of that will trigger rapid change.

To quote Sun Tzu I killed you....without lifting a finger"

Posted by: Steve H at June 13th, 2015 4:08 AM

The current climate with the FDA and them not considering aging as a disease is perplexing to me, with all these groups looking into anti aging now... A moratorium would be crushing if there was something developed. I'd like to think people would bypass it if there were something developed though. But as it stands now, more people say they are against this research than those who say they are for it, so who knows if people will be up in arms for access to treatments.

Posted by: Ham at June 13th, 2015 9:32 AM

The regulatory challenges facing rejuvenation biotechnologies were addressed in two "Question of the Month" columns in the SENS Research Foundation Newsletter. As discussed there (see two links below), I am happy to say that while there remain things to be worked out to ensure the smoothest and most rapid track toward clinical development and licensure of rejuvenation biotechnologies, the challenge is far, far less than many in the community (including most of you) believe.

First and foremost, the old bogeyman that "aging" is not recognized as a "disease" for which the FDA and other regulatory bodies license therapies should not actually pose a significant hurdle to getting rejuvenation biotechnologies licensed and into widespread use (although it would be one of several things that would likely hold up CR mimetics and similar one-shot systemic "anti-aging" therapies).

Moreover, the remaining regulatory hurdles for the development of rejuvenation biotechnologies are already being tackled thanks to years of effort and goodwill amongst patient advocates (with heroic end-of-career effort by Dan Perry, retiring head of the Alliance for Aging Research and the Healthspan Campaign (of which SRF is a member), working with the Alzheimer's disease patient community) who have very productively engaged pharma and enlightened regulators at FDA.

There remains a lot to be done. If there are any lawyers or law students out there seeking work that could really help humanity and advance the vision of a future free of degenerative aging, there is a clear mission for you if you can find a way to creatively engage the system.

Posted by: Michael at June 13th, 2015 4:45 PM

Thanks Michael and I know we can do this!

Posted by: Steve H at June 14th, 2015 2:54 AM

I just noticed that I accidentally linked the same issue of the Newsletter for both halves of the question. Here is the correct link on the worry that "aging" is not recognized as a "disease" for which the FDA and other regulatory bodies license therapies.

Posted by: Michael at July 14th, 2015 4:55 PM

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