The Regulatory Maze: What It Means For Our Health

Doc Gumshoe looks at patents, clinical trials and the path to drug approval and sales

By Michael Jorrin, "Doc Gumshoe", December 19, 2017

[ed. note: Michael Jorrin, who I dubbed “Doc Gumshoe” years ago, is a longtime medical writer (not a doctor) who writes for us a couple times a month about health issues and trends. He does not typically focus on specific investment opportunities, but has agreed to our trading restrictions… as with all of our authors, he chooses his own topics and his words and opinions are his alone]

This will not be a primer about how to maneuver your brilliant idea for a drug that treats a disease for which no treatment currently exists into a successful product that will make billions for you.   No, this will be a general backgrounder, which I hope will help Planet Gumshoe understand the factors that essentially dictate the lay of the land with regard to drugs as well as that vast, amorphous, and growing category of stuff that we lump together under the irritatingly imprecise term, “supplements.”   Part of the reason that we call them supplements is that the term “drugs” is reserved for substances that have been officially recognized as treatment for diseases or medical conditions – in other words, substances that have worked their way through the regulatory maze.   However, many of those substances that we call supplements are really, from the perspective of intention, drugs – that is, they exist for no purpose other than to treat, or at least address, diseases or medical conditions.

An example is a supplement that I took for a couple of years (until I became convinced that it was doing little or nothing), that being glucosamine plus chondroitin.   It is clearly not a nutritional supplement; it’s not that we don’t get enough glucosamine/chondroitin in our diets and therefore need to supplement this deficiency by means of pills.   It is purely and simply meant as a treatment, albeit a palliative treatment, for osteoarthritis.    Is it disqualified from being a drug because it does not address the underlying pathology of osteoarthritis?   No.   In fact, lots of drugs are palliative, including the entire category of pain drugs.   Is it disqualified from being a drug because it just doesn’t work?   Again, not really.   It does seem to deliver some benefit, up to a point, and lots of drugs don’t do much more than that – how many drugs do we know about that are labeled as being “for temporary relief” of this or that condition?   Glucosamine/chondroitin does seem to offer some “temporary relief” of the pain and stiffness of osteoarthritis, at least until the condition gets worse.   So why is it a supplement and not drug?

The answer lies in the regulatory maze.   To be considered a drug, the substance in question needs both to be patented and also to be recognized by the Food and Drug Administration – the FDA (or some comparable authority, such as the European Medicines Agency) – as a drug.   The FDA, by the way, does not grant patents – that’s the province, at least in the US, of the United States Patent Office.

First step into the maze: getting a patent

In the development of a drug, obtaining a patent is the absolute sine qua non and the first step.   If an investigator, whether working for a pharmaceutical company or a university or a medical entity or all alone in his/her own laboratory, comes upon a substance or a procedure that appears to have some promise of actually treating or modifying a disease, a patent application is the earliest priority.   Having a patent application at least in the works is a pre-requisite to publishing a report about the potentially promising new thing, or even to dropping hints about it to your trusted colleague.   Why?   Because news of possible treatment options gets around fast, and the investigator cannot risk having somebody else get there first.   And the planet is well-populated with eager researchers who want to get in on the ground floor of the next big thing.

Not being able to get a patent is a huge obstacle in the development of a drug.   No pharmaceutical company (nor anyone else, for that matter) is going to risk the enormous sums necessary to take a potential drug through the clinical trials necessary to gain FDA approval, unless the candidate drug is patented.   (I am using “FDA approval” to stand for regulatory approval in general, by whatever agency.)   And, by the way, those enormous sums are genuinely enormous – estimates are that it may cost as much as $2.5 billion to fund the entire process.

And, of course, getting a patent on a candidate drug is not by any means a sure thing.   The alternative medicine community’s short take is