1. Clinical Trials

2. Purpose of Clinical Trials
Assess safety and efficacy of
Experimental treatments
New combinations of drugs
New approaches to surgery or radiation therapies
Better disease prevention approaches
Better diagnostic approaches

3. Clinical Trial Phases Phase I trials Phase II trials Phase III trials
Phase IV trials

4. Phase I Trial Evaluate safety of drug Determine safe dosage range
Identify side effects
Test small group of people (fewer than 100 people)

5. Phase II Trial Learn more about safety and side effects
Sharpen estimates of proper dosage
Determine effectiveness
Test larger group of people (up to 200 participants)

6. Phase III Trial Determine effectiveness Determine side effects
Test large groups of people (1,000-3,000 participants)

7. Phase IV Trial
Collect additional information after the drug enters the market, such as:
Drug’s risks
Drug’s benefits
Optimal use

8. Types of Clinical Trials
Randomized, double-blind trials
Open trials
Factorial trials
Crossover trials
Orphan drug trials

9. Controlled, Randomized, Double-Blind Trials
Controlled: One group receives the treatment and another group does not.
Randomized: control group and treatment group are chosen at random.
Double-blind: Neither subjects nor scientists know who is assigned to which group until after the data are collected.

10. Treatment Group vs. Control Group
Given experimental drug
Control Group:
Given standard treatment or a placebo
Placebo: an inactive pill, liquid, or powder that has no treatment value

11. Why Double-Blind?
Prevents subjects in different groups from behaving in different ways
Prevents scientists from introducing any unconscious bias into the data collection process

12. Single-Blind Subjects do not know which group they are assigned to.
Researchers know who is assigned to which group.
Prone to researcher bias.

13. Open Trials
Set-up where researchers and subjects know what treatment is being given
Often used to test surgical procedures and medical devices, that by nature, cannot be done without subject or researcher knowing who is receiving the treatment
More prone to error and bias than double-blind studies

14. Factorial Trials Used to test medicines in combination
Set-up in the following manner:
First group tests therapy A
Second group tests therapy B
Third group tests therapy A and therapy B combined
Fourth group, the control group, tests neither therapy A nor therapy B
Difficult to interpret results

15. Crossover Trials Used to test two treatments
Set-up where each participant gets both treatments being tested
Some participants are assigned at random to receive drug A and when done, receive drug B
Other participants receive drug B and when done, receive drug A

16. Orphan Drug Trials
Used to test drugs designed to treat rare diseases (affecting fewer than 200,000 Americans)
Tested on small number of participants who are very sick
If drug works, improved health is usually readily apparent

17. Safety and Ethical Regulations
Clinical trials are designed to safeguard the health of the participants.
Clinical trials done in the United States must be approved and monitored by an Institutional Review Board (abbreviated IRB)

18. Institutional Review Boards
IRBs are independent committees comprised of physicians, statisticians, community advocates, and others. The role of the IRB is to do the following:
Ascertain that clinical trials are scientifically worthy and that ethical guidelines are met
Potential benefits for participants should outweigh risks
Participants must be informed of all potential risks and agree to willingly participate and can drop-out at any time (called informed consent)
Monitor the trial during its run and may even halt the trial if serious problems are reported

19. What Happens After Clinical Trial
When the trial ends, the drug company submits all data to the Food and Drug Administration (FDA) for approval to market the drug.
If FDA agrees that the drug is safe and effective, it grants approval and the drug is moved into everyday medical practice.
Once on the market, the FDA continues to survey the side effects experienced by patients within the general population.