Estimated reading time: 7 minutes
- The drug development process involves multiple stages, from preclinical studies to FDA approval and beyond.
- Clinical trials are divided into phases, each with specific goals and ideal participant profiles.
- The entire process typically takes 10-15 years and involves rigorous testing for safety and efficacy.
Clinical Trials Explained
Often, we hear of new treatments becoming available, but have you ever wondered what each new treatment has to go through to get approved by a regulatory body like the FDA? Before a new treatment is approved for commercialization, it needs to go through a meticulous trial process to prove a number of things:
- Is the drug safe?
- What are the potential side effects?
- Does the drug do what it's supposed to do?
All of these questions and more need to be answered before a drug can be considered for approval by the FDA, so that's where clinical trials come in. Here's a breakdown of what's involved in the drug development process:
Preclinical Through Commercialization and Post-Approval Monitoring
Preclinical Studies
Before any human testing can begin, researchers must first demonstrate that the potential new drug is reasonably safe for initial use in humans. This stage involves laboratory and animal studies to gather key information about the drug's toxicity, dosing, and efficacy. These studies help researchers determine if it's reasonably safe to move forward with human trials.
Investigational New Drug Application (IND)
If the preclinical studies are successful, the drug's sponsor (usually a pharmaceutical company) submits an IND to the FDA. This application includes the results from the preclinical studies, the drug's composition, manufacturing details, and a plan for testing the drug in humans.
Phase 1 Clinical Trials
Once the IND is approved, Phase 1 trials can begin. These trials typically involve 20 to 100 participants. The main goal is to evaluate the drug's safety and determine safe dosage ranges. This phase usually lasts several months.
Ideal participants:
Healthy volunteers or, in some cases, patients with the condition under study, particularly for drugs intended to treat serious or life-threatening conditions. Participants should be willing to undergo frequent medical tests and stay at the study site for extended periods.
Phase 2 Clinical Trials
If Phase 1 studies don't reveal unacceptable toxicity, the drug moves to Phase 2. These trials involve several hundred people with the condition the drug is intended to treat. The goal is to obtain preliminary data on whether the drug works in people who have a certain disease or condition. Safety continues to be evaluated, and short-term side effects are studied. This phase typically lasts from several months to two years.
Ideal participants:
Patients with the specific condition the drug is designed to treat. Participants should be willing to follow strict protocols and undergo regular medical evaluations. They often have limited treatment options or have not responded well to existing treatments.
Phase 3 Clinical Trials
Phase 3 studies are larger, involving several hundred to several thousand people. These trials provide more information about safety and effectiveness, study different populations and different dosages, and use the drug in combination with other drugs. This phase can last several years.
Ideal participants:
A diverse group of patients with the condition, representing various ages, ethnicities, and sometimes comorbidities. This phase aims to reflect the general patient population that would use the drug if approved. Participants should be able to commit to long-term follow-up and adhere to the study protocol.
New Drug Application (NDA)
If Phase 3 trials are successful, the drug's sponsor can file an NDA with the FDA. This application includes all animal and human data and analyses of the data, as well as information about how the drug behaves in the body and how it's manufactured.
FDA Review and Approval
The FDA review team thoroughly examines all submitted data on the drug and makes a decision to approve or not to approve it. This process usually takes 6-10 months.
Phase 4 Studies
After a drug is approved and made available to the public, the FDA may require additional studies (Phase 4) to assess long-term safety and effectiveness in various populations.
Ideal participants:
Patients who are prescribed the drug as part of their regular treatment. These studies often involve a more diverse patient population and can include those with comorbidities who might have been excluded from earlier phases.
Understanding the Clinical Trial Process - Not Always Linear
It's important to note that this process is not always linear. Sometimes, phases overlap, or additional studies are required before moving to the next phase. Moreover, many potential drugs fail during this process – only about 1 in 10 drugs that start clinical trials eventually win FDA approval.
The entire process, from preclinical studies to FDA approval, typically takes 10-15 years and can cost hundreds of millions of dollars. This lengthy and expensive process is designed to ensure that new treatments are both safe and effective before they reach patients.
Click to enlarge infographic:
How Can I Participate In Clinical Trials or Learn More?
- Join PatientsLikeMe to sign up for alerts for trials that could be a good fit for you. We are currently recruiting for alzheimer's and lupus clinical trials!
- Check in with local associations and hospitals to see if they are recruiting for any trials
- Talk to your healthcare provider/clinician to see if there are opportunities they are aware of and how you can participate
- If you're a member of PatientsLikeMe, make sure you consistently update your profile so we can let you know about research survey opportunities that are right for you
