2 Purpose of Clinical Trials Assess safety and efficacy ofExperimental treatmentsNew combinations of drugsNew approaches to surgery or radiation therapiesBetter disease prevention approachesBetter 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 effectsTest small group of people (fewer than 100 people)
5 Phase II Trial Learn more about safety and side effects Sharpen estimates of proper dosageDetermine effectivenessTest 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 TrialCollect additional information after the drug enters the market, such as:Drug’s risksDrug’s benefitsOptimal use
8 Types of Clinical Trials Randomized, double-blind trialsOpen trialsFactorial trialsCrossover trialsOrphan 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 drugControl Group:Given standard treatment or a placeboPlacebo: an inactive pill, liquid, or powder that has no treatment value
11 Why Double-Blind?Prevents subjects in different groups from behaving in different waysPrevents 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 TrialsSet-up where researchers and subjects know what treatment is being givenOften used to test surgical procedures and medical devices, that by nature, cannot be done without subject or researcher knowing who is receiving the treatmentMore 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 ASecond group tests therapy BThird group tests therapy A and therapy B combinedFourth group, the control group, tests neither therapy A nor therapy BDifficult to interpret results
15 Crossover Trials Used to test two treatments Set-up where each participant gets both treatments being testedSome participants are assigned at random to receive drug A and when done, receive drug BOther participants receive drug B and when done, receive drug A
16 Orphan Drug TrialsUsed to test drugs designed to treat rare diseases (affecting fewer than 200,000 Americans)Tested on small number of participants who are very sickIf 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 metPotential benefits for participants should outweigh risksParticipants 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.