CLINICAL TRIALS

MRS. STEWARTMEDICAL INTERVENTIONS

CENTRAL MAGNET SCHOOL

Essential Question:

◦How are clinical trials set up to ensure all data collected is valid and that all human subjects are treated ethically?

THINK – PAIR – SHARE

◦What is a clinical trial?

◦Clinical Trials 101: How do we develop and test a new vaccine

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

Clinical Trial Phases

◦Phase I trials

◦Phase II trials

◦Phase III trials

◦Phase IV trials

Phase I Trial

◦Test small group of people (fewer than 100 people)

◦Evaluate safety of drug

◦Determine safe dosage range

◦Identify side effects

Phase II Trial

◦Test larger group of people (up to 200 participants)

◦Learn more about safety and side effects

◦Sharpen estimates of proper dosage

◦Determine effectiveness

Phase III Trial

◦Test large groups of people (1,000-3,000 participants)

◦Determine effectiveness

◦Determine side effects

Phase IV Trial

◦Collect additional information after the drug enters the market, such as:◦Drug’s risks◦Drug’s benefits◦Optimal use

Direct to Consumer Drug Advertisements

◦The United States and New Zealand are the only two countries where direct-to-consumer (DTC) advertising of prescription drugs is legal.  

◦Every $1.00 spent advertising prescription drugs is estimated to increase their retail sales by $4.20. 

◦Example: Lyrica

◦Example: Cymbalta

Reference: American Medical Association, "AMA History Timeline,” www.ama-assn.org (accessed Mar. 21, 2014)

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.

TYPES OF CLINICAL TRIALS

Randomized, double-blind trials

Open trials

Factorial trials

Crossover trials

Orphan drug trials

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.

Treatment Group vs. Control Group

◦Treatment Group:◦Given experimental drug

◦Control Group: ◦Given standard treatment or a placebo◦Placebo: an inactive pill, liquid, or powder that has no treatment value

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

Single-Blind

◦Subjects do not know which group they are assigned to.

◦Researchers know who is assigned to which group.

◦Prone to researcher bias.

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

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

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

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

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)

Institutional Review Boards

◦Established by gov’t May 30, 1974◦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

Grey’s Anatomy

◦Study: New alzheimers drug – phase I

◦Adele◦Friend of the doctor’s conducting the study◦Wife of the chief of surgery◦Has alzheimers

◦Alzheimer’s Drug Clinical Trial - Adele’s results

◦Alzheimer’s Drug Clinical Trial – Meredith

You Are the IRB: Was this trial ethical?

conducting high-altitude experiments on prisoners, using a low-pressure chamber, to determine the maximum altitude from which crews of damaged aircraft could parachute to safety

freezing experiments using prisoners to find an effective treatment for hypothermia

scientists tested immunization compounds and sera for the prevention and treatment of contagious diseases, including malaria, typhus, tuberculosis, typhoid fever, yellow fever, and infectious.

determine how different "races" withstood various contagious diseases by infecting them against their will.

(Prisoners in Nazi concentration camps during WWII were experimented on.)

Examples of unethical experiments in the U.S.

1932400 African-American men

from the Tuskegee Institute left untreated from syphillis despite cure available so that scientists could study the “natural course of the disease”

(http://oreilly.com/medical/trials/news/ethics_of.html)

THINK – PAIR – SHARE

◦How does the US IRB prevent future experiments from violating ethics?

Essential Question: Exit Ticket◦How are clinical trials set up to ensure all data collected is valid and that all human subjects are treated ethically?

Assignment: lab groups ◦Develop a new drug ◦Determine what disease/disorder this drug will treat ◦Describe how it will work specifically

◦Design the clinical trial to test it for safety and efficacy

◦(must be approved by an IRB (Mrs. Stewart)

◦Due Monday at end of class