Clinical trials are crucial to ensure that treatments are safe and effective.
These research studies test the safety of new medical approaches, including the development of new medications and devices (and even combination products such as stents that release medication!).
Clinical trials follow a protocol which is an action plan for the study. The protocol describes what will be done in a study, how it will be conducted, and explains why each portion of the study is necessary. Depending on what is being studied, each clinical trial will have different requirements for participants. Some require volunteers with particular diseases while others require healthy people. Age, sex, medication history, and more can be determining factors (or inclusion/exclusion criteria) for a clinical trial.
How are clinical trials monitored and approved?
A clinical trial must be reviewed by an Institutional Review Board (IRB) if it is studying a drug, biological product, or medical device regulated by the Food and Drug Administration (FDA), or if the study is funded or conducted by the federal government. Institutional Review Boards have been established to ensure:
- studies are ethical
- participants are protected – both their rights and their welfare
- the risks of the study are reasonable as compared to the potential benefits.
IRBs can private companies (often called independent of commercial IRBs) or affiliated with universities that conduct clinical trials. They are typically staffed by physicians, statisticians, and ethicists. There must be at least five members on an IRB with varying backgrounds.
Read the government requirements for IRBs at 21 CFR 56.107
The drug development process
It is expensive and time consuming to bring a new drug or medical device to market. Estimates on cost and timeline vary. However, the Association of Clinical Research Organizations reports that it takes 15 years and as much as $1.2 billion. In a similar vein, PhRMA reports that it can take a drug six to seven years simply to complete clinical trials. And, each successful is estimated to cost $2.6 billion. Clearly, this is a long, expensive process.
Pre-Clinical Research
Duration: 1 – 3 years
Objective: Testing the drug on animals. If the test finds that the product is safe, it is cleared for human clinical trials
Phase 1 – Initial Human Clinical Trials
Duration: 6 months – 1 year
Objective: Test the drug on 20 – 80 healthy individuals. Ensure the drug is safe and generate some pharmacological data
Phase 2 – Expanded Human Clinical Trials
Duration: 1 – 2 years
Objective: Test the drug on 100 – 200 patients who have the disease or condition for which the drug was created
Phase 3A
Duration: 2 – 3 years
Objective: The drug is tested on several hundred to several thousand participants at numerous sites to test the safety and efficacy on a large scale. There is also a focus on regulatory issues.
When complete, the sponsor of the clinical trial submits all relevant data (clinical information, plans for production, packaging, and labeling information) to local regulatory agencies for review. This review can take up to 30 months.
Phase 3B
Duration: 6 months – 1 year
Objective: Test the drug on large groups of patients. Evaluate for cost-effectiveness and efficacy as compared to existing approved drugs.
Phase 4
Duration: Ongoing following regulatory approval
Objective: After a drug is approved, it is monitored to ensure that it is safe and effective if used beyond the original application. Additionally, it allows for testing of dosage strengths and formulations and can confirm extra-clinical benefits, like cost-savings or improved quality of life.
