A Nuanced Opposition to the FDA and Similar Regulatory Agencies

As regular readers will know, I am no great admirer of medical regulation as it presently exists in the wealthier parts of the world. It is a burdensome system, in which whatever power good intentions have to make the world a better place has long been eroded away by the short-term human incentives present in any large bureaucratic organization. What is left is a system in which it costs multiples of what it should cost to bring medicine into the clinic at an appropriate level of risk, a system that acts primarily to suppress rather than encourage development of new medical technology, and a system that tramples upon the rights of patients to make their own informed choices. In the past my prescription for a better future has been one in which medical tourism flourishes: the use of regulatory arbitrage to bring medicine to the clinic in a responsible way in places outside the US. The eminently dysfunction US medical system, in which no party is incentivized to control costs, the most influential parties collude to prevent cost discovery, and quoted costs have little relation to actual costs, leads to a situation in which it is cheaper to fly halfway around the world to another country to receive even modest procedures. So let medical tourism grow as a form of pressure upon the existing regulatory system, because competition from outside is the only thing that has a hope of resulting in significant reform. Any number of people work within the system on initiatives to make it better. There are non-profits and high net worth individuals and patient advocates all calling for reform of the FDA in one way or another. All are distinguished by having had very little positive effect over the years of their efforts. Indeed, over the last decade the cost of FDA approval has doubled, the time lengthened, and the rate at which new medical technologies are approved is stagnating. This has happened over the course of a period of enormous, staggering progress in biotechnology, in which costs of the underlying technologies have fallen dramatically. How can this be the case? Cynically, I would say this was also a period of great progress in connectivity and media exposure. Thus the tendency of bureaucrats to prioritize the minimization of bad press and accountability leads to ever greater demands, costs, and delays placed on regulatory applications, in search of a mythical, non-existent minimum level of risk that can only really be obtained by preventing new technologies from arriving at all. Medical tourism, however, has challenges, primarily that it remains a highly disorganized market. Few of the people who might benefit significantly in fact do so. It requires a fair amount of research and determination; there is no easy on-ramp. I think that it will require universally desired enhancement therapies, such as follistatin or myostatin gene therapies for muscle growth, to obtain a large enough number of potential customers to generate sufficient organization in this marketplace to build that on-ramp. The number of people with severe illnesses is simply too small for the self-organization of entrepreneurship and venture funding to snowball in medical tourism - if it was going to happen, it would have happened already. Here is another question: how do new therapies become available via medical tourism? Just as medical tourism is disorganized, so too there are large challenges facing company founders who want to responsibly develop and offer a therapy outside the established mainstream regulatory systems. A clear path forward to this goal has not been built; there is no roadmap. It is the jungle of uncertainty, and investors fear uncertainty. The founders of a company heading in this direction, such as BioViva Sciences and a few others, are faced with not just the challenging of building a therapy that is safe and reliable, but also building the entire infrastructure - the relationships, the legal understandings, the third party validation of safety and effectiveness - to bring that therapy into a clinic. Then they must also survive the bombardment of negative press from organizations invested in the status quo, hostile to the idea that proving safety and responsibility really doesn't require more than a fraction of the costs that the FDA imposes. Further, the founders must successfully woo investors who are very used to the present beaten path and nervous regarding anything new. Taken together, this is a tall order. It is a task suited to a non-profit, or more accurately a dedicated organization, or a distributed bootstrapping process of cultural change driven by many such groups. The prize is well worth it, in terms of an acceleration of progress towards many new classes of therapy, a removal of the dead weight cost that slows and suppresses advances in medicine. As is so often the case, it is far easier to articulate the change we'd like to see than to make it happen, however. So if I started a medical biotech company, I'd have to say that I would take it through the current regulatory system, while (a) being an honest player by the rules of that system, because that is the best way through the gauntlet, and (b) continuing to hold the view that the whole thing should be replaced with a far less self-serving, onerous, expensive, and terrible option. Unfortunately, the standard regulatory path is the only fairly reliable way to put a therapy in the hands of large numbers of patients. The other, ethically far better paths, such as that pursued by BioViva, have the unfortunate outcome of great uncertainty in whether patients will ever be able to use the treatment in large numbers. I'm sure I'm not alone in having come to this position. The change the world needs here is clear: a far more organized industry of medical tourism, and an infrastructure for responsible medical development and validation of therapies, outside the established regulatory systems, that is generally accepted by investors. These challenges are easy to state, but resistant to any sort of easy solution that can do better than reaching a fraction of the patients who might benefit. At present any one company might succeed against the odds in obtaining enough funding and support to set up an offshore clinic - but how many patients can they reach? Small heroic battles, one company at a time, will not be enough. Until greater change is achieved, near everyone who starts the process of medical development will likely look at the landscape and make the rational short-term choice to pass through the existing regulatory gauntlet.

https://www.fightaging.org/archives/2018/04/a-nuanced-opposition-to-the-fda-and-similar-regulatory-agencies/

About Robert Zinn

Robert Zinn, M.D., Ph.D. is a medical doctor, physician, and web entrepreneur, who, for over 15 years was employed by academic and research institutions and focused his clinical practices on very specialized patient populations, such as those with rare genetic diseases or rare cancers. He shares his knowledge through his website, NutritionTheory.org

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