Part 1 in a 2 part series
In medical school and residency, the industry side of medicine is not exactly a topic that gets paid much attention. Countless hours are spent memorizing pharmacology, but the process of how a drug actually makes the transition from bench to bedside is not part of the curriculum. A few years into my own private practice, the opportunity to learn the process of drug development would come to fruition.
The journey started as many innovations do – out of necessity. A breast cancer patient presented with debilitating paronychia consisting of eruptive granulation tissue and onycholysis of her fingernails triggered by her taxane regimen. All conventional treatments had failed, and she was now unable to perform activities of daily living secondary to the pain, and faced discontinuing treatment. As a strong advocate of compounding pharmacies, the decision was made to compound a well-known broad spectrum antiseptic with a penetration enhancer. The results exceeded all expectations. The patient called two days later to say the pain was completely gone, and within several weeks the paronychia was eradicated altogether. All local oncology groups were subsequently called and asked to refer any additional cases, and the results were repetitively duplicated. A few weeks later upon discussion of the findings with a colleague having a background in pharmaceuticals, the pursuance of this new discovery further than just a case series was launched.
When embarking upon a potentially new idea, the first thing that should be done before anything else is a patent search to determine if the idea is in fact novel. You can research this by visiting and becoming familiar with The United States Patent and Trademark Office website (www.uspto.org). On this website, guidelines for writing and filing a provisional patent are explained, which establishes an invention priority date on the patent. The process of filing a provisional patent is inexpensive and gives a one-year period to refine the idea until it must be converted into what is called a utility patent. An experienced patent attorney is essential in this conversion step, and it is what will be the most costly upfront. Choosing correctly pays in dividends as the patent attorney takes the provisional patent and broadens/refines the claims to be written in such a way as to protect the invention in a 360-degree fashion. This is necessary so poachers cannot find cracks in the patent and copycat its ideas without infringing upon them down the road.
In conjunction with working on the patent, the daunting task of FDA approval arises. The first step in developing any new drug or device is to file what is called an Investigational New Drug (IND) Application. An IND is a request for authorization from the FDA to administer an investigational drug or biological product to humans. The main purpose of the IND is for the FDA to review the application for safety to assure that research subjects will not be subjected to unreasonable risk. The website www.fda.gov