Navigating the Roadmap of a Clinical Trial
It’s no secret that clinical trials are quite complex to run. With various steps and phases, most trials take years to get that coveted Food and Drug Administration (FDA) approval.
However, there’s more to it than just the phases of a study. By the time a clinical trial comes to fruition, research teams have likely put in work for years on the front end to get the drug or therapy to a trial. In total, from ideation to FDA approval, a clinical trial may take 10-15 years of researchers time.
This blog outlines the typical roadmap of a clinical trial, from ideation, to preclinical, to trial phases and ultimately, to FDA approval.
The Initial Idea
Clinical trials don’t just simply come to be; there is a lot of ideation and planning upfront. An idea typically comes from something researchers would like to test, and potentially include:
- A new treatment option
- A combination of treatments
- Taking an existing treatment and testing it for a different condition
- New ideas for existing treatment options
No matter how the idea comes to fruition, it is the foundation for a budding clinical trial. From there, research teams will need to develop a clear, testable hypothesis for the basis of the trial. Once the hypothesis is solidified, a clinical trial is ready to enter into the preclinical phase.
Preclinical Phase: Laying the Groundwork
In the preclinical phase, research teams are doing any amount of research they can before the drug moves into phases where they can test on human volunteers. The hypothesis created often leads to a high volume of hypotheses for researchers to sift through and determine what is worth testing. Eventually, through a lot of testing in the laboratory, the goal is to find a singular drug candidate that can move onto the phases of a clinical trial.
Preclinical research is broken down into four parts:
- Basic research
- Drug discovery
- Lead optimization
- Investigational new drug (IND)-enabling studies
With 90% of drug development failing to make it past the preclinical research stage, it’s especially important for research teams to dive in and be thorough in their research as they explore their hypothesis. The ultimate goal during preclinical is to gather enough data to submit an IND application to the FDA, which is a formal application to begin human clinical trials for a drug not yet approved for the general public to use.
Clinical Trial Phases
If a hypothesis makes it out of the preclinical phase, the next step is to conduct a clinical trial. This isn’t just one large study, it’s conducted in four different phases. However, not all trials make it from phase to phase – each phase serves different purposes and has different qualifiers to make it to the next one.
Phase I
These trials are typically very small, about 20-100 volunteers. The purpose of a Phase I trial is to test the medicine in humans for the first time. Researchers are looking for the correct dosage amount for the medicine, as well as learning about the drug’s safety and potential side effects in humans.
Phase II
Phase II trials grow a little more, typically enrolling 100-300 participants. With a slightly larger participant pool, researchers are continuing to study the drug or treatment’s safety, as well as its effectiveness.
Phase III
In Phase III trials, research teams are looking to enroll about 300-3,000 participants. The increased number of participants enables teams to continue to study the drug’s effectiveness and safety. Additionally, with an increased participant pool, research teams are more easily able to monitor side effects and subsequently compare those results with the treatments already on the market.
Phase IV
The last phase in a trial, Phase IV is also known as post-marketing surveillance studies. It occurs after the drug or treatment is publicly available due to FDA approval. Even though the drug is now on the market, researchers will still monitor its safety, effectiveness and long-term effects. These insights will provide a more comprehensive understanding of the treatment’s true risk-benefit profile.
Regulatory Review: FDA Approval Submission
For the drugs that have survived the ideation stage, the preclinical stages, and each research phase, research teams are now ready to file a new drug application (NDA) with the FDA. This application is a formal proposal for the FDA to approve the drug for sale and marketing in the U.S. The NDA needs to be filled out in such a way that the FDA can determine if:
- The drug’s safe and effective, and if the benefits outweigh the risks
- The drug’s proposed labeling consists of all the necessary information
- The manufacturing methods and quality control for the drug are sufficient enough to preserve the drug’s identity, strength, quality and purity
Once a sponsor submits the drug’s NDA, they must pay a fee per the Prescription Drug User Fee Act (PDUFA). In 1992, Congress created this act, which authorized FDA to “collect user fees from persons that submit certain human drug applications for review or that are named in approved applications as the sponsor of certain prescription drug products.” User fees have since been pivotal in expediting drug review and approval processes – before, this process averaged between 21 and 29 months. Now, it’s averaging 10 months for drugs to become approved.
FDA Approval: What Does it Mean?
Once the FDA officially approves the drug or therapy, it can now be marketed and sold for public use. While for research teams, this significant moment marks the end of a long journey, another one is just beginning for patients. An FDA approval opens up many doors for patients, mainly because it provides a new method of treatment that wasn’t previously available. This can lead to improved quality of life for so many patients, which is the biggest benefit of all.
Navigate the Clinical Trial Roadmap With Sabai
At Sabai, our single institutional review board (IRB) and institutional biosafety committee (IBC) board members are ready to help your study as it moves along the clinical trial roadmap. Our ethical review services help deliver protection and oversight for participants involved in clinical research. This enables research teams to focus on what matters most: bringing ideas to fruition, ultimately bringing a drug or therapy to market to help those in need.