While preclinical research answers basic questions about a drug’s safety, it is not a substitute for studies of ways the drug will interact with the human body. “Clinical research” refers to studies, or trials, that are done in people. As the developers design the clinical study, they will consider what they want to accomplish for each of the different Clinical Research Phases and begin the Investigational New Drug Process (IND), a process they must go through before clinical research begins.
On this page you will find information on:
- Designing Clinical Trials
- Clinical Research Phase Studies
- The Investigational New Drug Process
- Asking for FDA Assistance
- FDA IND Review Team
- Approval
Designing Clinical Trials
Researchers design clinical trials to answer specific research questions related to a medical product. These trials follow a specific study plan, called a protocol, that is developed by the researcher or manufacturer. Before a clinical trial begins, researchers review prior information about the drug to develop research questions and objectives. Then, they decide:
- Who qualifies to participate (selection criteria)
- How many people will be part of the study
- How long the study will last
- Whether there will be a control group and other ways to limit research bias
- How the drug will be given to patients and at what dosage
- What assessments will be conducted, when, and what data will be collected
- How the data will be reviewed and analyzed
Clinical trials follow a typical series from early, small-scale, Phase 1 studies to late-stage, large scale, Phase 3 studies.
Phase 1
Study Participants: 20 to 100 healthy volunteers or people with the disease/condition.
Length of Study: Several months
Purpose: Safety and dosage
During Phase 1 studies, researchers test a new drug in normal volunteers (healthy people). In most cases, 20 to 80 healthy volunteers or people with the disease/condition participate in Phase 1. However, if a new drug is intended for use in cancer patients, researchers conduct Phase 1 studies in patients with that type of cancer.
Phase 1 studies are closely monitored and gather information about how a drug interacts with the human body. Researchers adjust dosing schemes based on animal data to find out how much of a drug the body can tolerate and what its acute side effects are.
As a Phase 1 trial continues, researchers answer research questions related to how it works in the body, the side effects associated with increased dosage, and early information about how effective it is to determine how best to administer the drug to limit risks and maximize possible benefits. This is important to the design of Phase 2 studies.
Approximately 70% of drugs move to the next phase
Preclinical
Phase 0
Phase I
Phase II
Phase III
Phase IV
Preclinical
Primary goal: Testing of drug in non-human subjects, to gather efficacy, toxicity and pharmacokinetic information.
Number of participants: NA
Phase 0
Primary goal: Pharmacokinetics; particularly, oral bioavailability and half-life of the drug.
Number of participants: 10 people
Phase I
Primary goa: Testing of drug on healthy volunteers for safety; involves testing multiple doses.
Number of participants: 20 to 100 normal healthy volunteers
Phase II
Primary goal: Testing of drug on patients to assess efficacy and side effects.
Number of participant:100 to 300 patients with specific diseases.
Phase III
Primary goal: Testing of drug on patients to assess efficacy, effectiveness and safet.
Number of participants: 300 to 3,000 patietns with specific disease.
Phase IV
Primary goal: Post marketing surveillance – watching drug use in public
Number of participant: Anyone seeking treatment from their physician