The normal steps in Clinical Drug Trials plays a significant role in my novel Double Blind. In the book, eyedrops, nicknamed EZ by the research volunteers, is suddenly fast-tracked to prescription. The fast-tracking of ZeNovo’s EZ eyedrops is a catalyst for Claire Carter, the main protagonist. Claire’s so angry she climbs inside an obscure research building’s air duct to film a board meeting. She’s appalled that while she waits to hear from the Department of Justice on her whistleblower status, she’s yanked into court by ZeNovo for corporate espionage. The poor girl has no idea her lawyer held back data from the DOJ review. She’s doomed to fail and doesn’t know it. But Claire isn’t a quitter, no, she plans to film and upload to YouTube because truth shouldn’t be hijacked.
I don’t go into the clinical trial process much in the book, but I thought readers might be interested in the process. Every drug goes through a pre-clinical (before a human takes it) process. This part covers the drug’s discovery and the researchers are trying to get safety information and efficacy data. (efficacy means a drug produces the intended and desired results). There is a lot of animal research in this stage. I know, I hate this stage too, but the purpose is ultimately to do no harm to humans. Just know it takes ten or more years in this stage before the new drug goes to human trials. (Yes, Zenovo the rotten corporation cheated in this phase in the book). Once safety & efficacy is ensured new drugs progress to human trials.
Phase I Clinical Trials
During this phase the new drug is given to a human for the first time. These studies are safety studies and enroll a small number of healthy volunteers. The research subjects are given increasing doses of the test drug until the side effects reach a point that the subject can no longer tolerate them. Lots of college students sign up for this type of testing. At the end of this trial the PIs (Primary Investigators) know a lot about the drug’s safe dose range and adverse side effects. Assuming there are no deaths or loss of limbs and if the FDA agrees, the drug goes to the next step. All Phase one studies must be concluded before going to Phase II.
Phase II Clinical Trials
These studies are described as rigid and well-controlled studies in a small number of patients who have the symptoms the drug will treat like say for diabetes. The patient subjects all have the target disease but without co-diseases. So the patients enrolled might have well controlled diabetes but no heart disease or cancer. In this phase PIs work with double-blind studies, using a placebo or other comparator drug, or both. The entire purpose of this type of drug trial is to determine if the new drug, now called the investigational drug, works as stated within the safe dose range. Safety is always a big issue and it’s examined closely in all research trials. The FDA must approve the next step or they can request more Phase II studies. Here, EZ was fast-tracked straight to prescription because of its stellar but tainted results.
Phase III Clinical Trials
These studies aren’t done if the outcomes in Phase I or II were poor. To reach this stage the drug has a proven safety profile and evidence of proven efficacy. These studies enroll thousands across the country and internationally. Information in the phase is used to demonstrate the safety (few side effects) and efficacy to assess the risk/benefit relationship for the intended use of the drug. Phase III studies gather the information that goes into a drug product’s package insert. These trials last for years as I can personally attest. After the successful conclusion of this phase, the new investigational drug goes to prescription but may not be approved for special populations, like children.
Phase IIIb Clinical Drug Trials
Some of the drug studies may not have completed because the FDA requested more safety studies, or the FDA ordered extra testing in special populations like the elderly or children. Mostly they are gathering additional data here, but this step doesn’t stop the new, investigational drug from going to prescription market.
Phase IV Clinical Trials
These studies are done after the FDA has approved the new drug for marketing. The company may have gotten the go ahead to market as long as they continued drug studies in this phase. Here the FDA wants long-term safety studies to compare to the short-term safety studies of Phase II. They may be testing the new drug against its older and established competitor drugs.
Double Blind means neither the patient or the PI and his team members know if the patient is taking a placebo or active drug. To find out, the blind must be broken and this is discouraged unless the patient is hospitalized.