Why lifesaving cancer treatments take so long to become commercially available
Kathryn Vinson, MS, CCRC
FDA approval process – In a previous article entitled “Perspectives of a stem cell donor”, I spoke with you about my mom’s journey as a stem cell donor and talked about her little sister’s battle with non-Hodgkin’s Lymphoma (NHL). When Deede got sick in 1992, a lot of the amazing treatments that we have today weren’t yet commercially available. A mere four years following her passing, the FDA approved the use of monoclonal antibodies in the treatment of NHL, ushering in a new wave of innovation in immunotherapy and giving renewed hope to those with this terrible disease. So many times, we have asked, why couldn’t those treatments have been around when Deede was sick? Well, there’s not an easy answer, but I want to explain the FDA approval process here in the U.S. to give you all a better understanding of why this takes so long.
That alphabet soup behind my name means Certified Clinical Research Coordinator – basically I’ve passed tests and have the necessary professional experience to be credentialed. I’ve recruited and coordinated clinical trials, and also managed a late phase research site. Long story short – I can help you understand the process.
In clinical research, we are guided by the Declaration of Helsinki, created by the World Medical Association to lay out the ethical rules for conducting research involving human subjects. These rules are very strict and inspired in part by the atrocities of the Tuskegee experiments and the horrific revelations made during the Nuremburg Trials. Every clinical trial that is conducted must be approved by an Institutional Review Board (IRB). These independent groups are tasked with ensuring the protection of human subjects in research.
Beyond our guiding documents, the flow of research typically goes – laboratory development -> pre-clinical studies -> phase 1 trials -> phase 2 trials -> phase 3 trials – > marketing and production -> phase 4 (aftermarket) trials. Currently, in the U.S. this process takes an average of 12 years! Add to that the fact that only one in 5000 drugs will actually make it all the way through this process, you can begin to see how this is a very costly and time consuming process.
The lab and pre-clinical phases are when the scientist first come up with the new drug. They are working through what they think will work and why, making the experimental compound, and initial testing. The testing in this phase utilizes tissues outside the body. For example, they will take blood cells and make sure that they don’t rupture when in the presence of the compound. After these outside the body tests, they move on to animal testing. I know that this is a controversial issue, but we need to know how a compound will affect an entire organism prior to introducing it to humans. These are overseen by compassionate, loving veterinarians.
Once we have figured out that a drug is safe in the lab, we can move to human trials – only 5 of those 5000 will make it to this stage. After the developer submits an application to the FDA, the compound, now called an Investigational New Drug (IND), can move on to human testing. First up is phase 1, where the IND is tested on healthy individuals. Sounds weird, right? Why on Earth would we put an experimental drug into healthy people? We have to make sure that the IND isn’t going to cause horrendous side effects, and the best way to see that is in healthy people. We start by testing single doses on men. This isn’t sexist, simply the fact that we want to make sure that a woman’s ovaries, and therefore her eggs, are not exposed to a compound that could be harmful down the line. Slowly we add more doses, add women that are not capable of baring children, add women that are on reliable birth control methods, etc. We even study how the IND is absorbed differently when taken with or without food. This is a main time where we discover side effects, how long the IND stays in your system, safety, and toxicology.
When we get into phase 2 and 3 testing, we are looking at a patient population, meaning that they have the condition for which the IND is targeted. Phase 2 are shorter term studies, while phase 3 are more long term. We look at safety, tolerability, and efficacy of these compounds, toxicology, and a myriad of other aspects. What can be difficult for us in clinical research is finding people to volunteer for these studies that have the targeted disease but are otherwise healthy.
After the IND makes it through all of theses hurdles, and the numerous MD’s and PhD’s at the FDA and other regulatory bodies have given their approval, the drug can be marketed. Aftermarket trials look at super long-term safety, as well as comparisons to other marketed drugs.
Clinical research is amazing. These trials have been approved by regulatory agencies for the safety of everyone involved and are run by folks with very specific training and expertise. Participation in these trials provides the scientific community with invaluable data and its participants with early access to potentially lifesaving cancer treatments. Take a look at our page dedicated to clinical trials to see if you might be eligible to participate. We also know that the high cost drug of development is often passed on to the consumer. If you are feeling that burden, please visit our Financial Help section to see our listing of programs that you may qualify for.