In 2017, 46 new drug therapies were approved by the FDA, more than twice the number of new meds approved in 2016. The road to drug approval by the Food and Drug Administration isn’t an easy one by any means. From inception to delivery, the journey a new drug has to take is one that is fraught with hurdles and regulations.
The first step in creating a new drug is the idea. Believe it or not, this can be the most difficult step in the entire process. A drug company comes up with an idea that a compound could affect a disease or disorder in a new way. This idea is constantly in a state of change because there are always new compounds being discovered. New properties of existing compounds are also being identified as well as new combinations that could work in novel methods.
Thankfully, drug companies don’t have to worry about keeping their own stock of compounds on hand when they need to test new combinations or different effects. There are huge repositories all over the world that contain samples of nearly every chemical compound that has been created. These compound libraries are extremely helpful in the creation of new drugs, especially when it becomes time to test these new ideas.
However, transporting these chemicals and compounds can be dangerous. You can’t just put them in a box and ship them via UPS. Some are sensitive to heat or light, while others can be volatile. Shipping them requires an innate knowledge of state and Federal regulations, especially in the bulk amounts sometimes required. That’s when drug companies rely on lab movers and material handling specialists.
Once the idea is developed and the new compound works like they want it to, the rest of the road to FDA approval is no less perilous. The next step is to hold preclinical trials. These are usually performed on laboratory animals and allow the manufacturer to determine if there are any unforeseen side effects. One of the primary tests is to ensure that the new compound is not a carcinogen (causes cancer). Other important tests include checking to see if the new drug is mutagenic or teratogenic (affecting DNA or unborn fetal tissue respectively).
After the drug passes the preclinical trials, it will move on to clinical trials on humans. There are several rounds of clinical trials. The first set of trials is usually very small and focuses on patients who have the condition being targeted or volunteers. These trials last about a year and help doctors determine the correct dose and find any side effects that didn’t show up before.
The second trial actually tests how effective the new treatment is against the condition it is treating. This segment of the clinical trials will take years as doctors refine their treatment; this involves adjusting the dosage and testing the drug on larger and larger groups of people.
During this second trial, the drug manufacturer also comes up with its presentation to the FDA. The company has to explain to the FDA what the drug is, what it does, and how it differs from other current treatments, if any.
The last clinical trial is what proves this new drug’s efficacy to the FDA. This stage is the longest and most involved as the study takes place all over the country in research hospitals with larger and larger groups of participants.
These final trials are often double-blind studies, where neither the physicians involved nor the patients know if they’re receiving the drug or a placebo. After successful trials, the company is then free to ask the FDA for approval.
This approval process can take as long as a decade from concept to approval. For example, Viagra (sildenafil citrate) was developed in 1989 to treat high blood pressure and chest pain. During the first set of trials, the side effect of treating erectile dysfunction was revealed. This new discovery caused the drug to go through more trials for that. The drug was finally approved in 1998 by the FDA, eight years later.