Clinical Trials
A Real World Application of the
Scientific Method
Copyright 2010. PEER.tamu.edu
How do you know if the
medicines you take are safe?
Did you know that all
medical drugs,
treatments, preventions
and even medical devices
have to go through a
thorough process before
they are available to you?
This process is call a
Clinical Trial.
What are Clinical Trials?
Clinical Trials are medical or health-related
research studies done in human beings (or
in animals if the study is a veterinary study).
Why are Clinical Trials
Important?
In Clinical Trials, researchers take the results from
basic scientific research and translate them into
ways to prevent, treat, or diagnose disease.
Without them, we would could not ensure safe,
effective treatments for diseases.
The Scientific Method
Clinical Trials are “real
world” applications of the
Scientific Method.
Each time a drug, medical
device or procedure, is
tested, a question is
asked, a hypothesis is
made, an experiment is
conducted, results are
analyzed, and a
conclusion is reached.
Think Break:
What are Clinical Trials?
Why are Clinical Trials important?
How are Clinical Trials like the Scientific
Method?
Types of Clinical Trials:
(as defined by the National Institutes of Health)
Treatment Trials - test new treatments, new
combination of drugs or new approaches to
surgery or radiation.
Prevention Trials - look for better ways to prevent
diseases.
Types of Clinical Trials:
Diagnostic Trials - determine
better tests or procedures for
diagnosing a particular disease
or condition.
Screening Trials - test the best
way to detect or treat diseases.
Quality of Life Trials - explore
and measure ways to improve
the comfort and quality of life of
people with a chronic illness.
Sponsors
Clinical trials are usually
sponsored or funded by
companies that make
pharmaceuticals or medical
devices.
Trials can occur at sites as
varied as hospitals,
universities, doctors’ offices,
community clinics, or in the
offices of clinical-trial
contractors.
Clinical Trials are Done in
Phases:
First, a Pre-Clinical Trial must
be done before the Clinical
Trial starts.
Preclinical trial – research
on a new drug or a new
medical device or procedure,
usually done on animals, to
learn about mechanisms of
action, determine how well the
treatment works, and see if it
is safe to test on humans.
Think about it:
Who sponsors Clinical Trials?
Why is it important to test drugs, treatments
and devices on animal cells or subjects
before testing them on humans?
Clinical Trials are Done in
Phases:
Phase I
Researchers test an experimental drug or
treatment in a small group of people
(approximately 20-80) for the first time. The
purpose is to evaluate its safety and identify
side effects. If this is a veterinary study, it is
conducted in animals.
Where do the people come
from?
Clinical Trials require
people to volunteer to be
tested! They are often paid.
Would you volunteer to be a
subject in a Clinical Trial?
What if it meant you
received a treatment for a
disease that wasn’t
available to anyone else?
Phases of Clinical Trials:
Phase II
The experimental drug or treatment is
administered to a larger group of
people/animals (approximately100-300) to
determine its effectiveness and to further
evaluate its safety.
Phases of Clinical Trials:
Phase III
The experimental drug or treatment is administered to
a large group of people/animals (300-3,000 or more)
to confirm its effectiveness, monitor side effects, and
compare it with standard or equivalent treatments.
Research Concepts
In many studies, the new drug is compared to a placebo. A
placebo is a product that looks like the new drug, but it does
not have the active ingredient in it. People do not know that
they are getting the placebo.
Sometimes the test compares the new treatment against an
existing treatment to see if better results can be obtained.
Research Concepts
Blind and Double Blind Trials are frequently
done.
A Blind Trial is a trial in which the patients do not
know if they are receiving the treatment or a
placebo.
A Double Blind Trial is a trial in which the
patients and the researchers do not know who is
receiving the treatment.
Why would the above be good ideas?
Research Concepts
Randomization is the process by which patients
are assigned a group for the Clinical Trial.
Groups are assigned randomly, not purposefully.
Some people will receive the new treatment, some
may receive an already approved treatment, and
some may receive a placebo.
If one treatment is found superior, the trial is
stopped so that the fewest patients possible
receive the less beneficial treatment.
Approval must be gained:
Once a drug has proven satisfactory after Phase III trials,
the trial results are usually combined into a large document
containing a comprehensive description of the methods
and results of human and animal studies, manufacturing
procedures, formulation details, and shelf life.
This collection of information makes up the "regulatory
submission" that is provided for review to the appropriate
regulatory authorities like the U.S. Food And Drug
Administration (FDA) so they can then grant the sponsor
approval to market the drug, device or treatment.
The Results!
For approximately every 5,000 to 10,000 compounds that enter preclinical testing,
only one is approved for marketing.
Cost of the failures has to be borne by the price of the one success.
Phases of Clinical Trials:
Phase IV
After a drug is licensed (approved by the FDA) or
treatment is launched, researchers track its safety,
seeking more information about a drug or
treatment’s risks, benefits, and optimal use. These
long-term studies involve large groups of
participants and are designed to reveal if any
unexpected side effects occur in a small
percentage of individuals. 2010 2011
Timeline Estimate
Below are some estimates on the amount of
time it takes for this process in cancer
treatment research.
Pre-clinical Trials - 4.5 years
Phases I-III - 8.5 years
FDA Approval - 1.5 years
Phase IV - Ongoing for the duration of the
use of the drug
How long is this whole process?
Costs
On average, pharmaceutical
companies are spending anywhere
between $100 and $800 million per
each drug tested!
Spending on clinical trials in the
U.S. is forecasted to rise to $32
billion by 2011.
Why would anyone spend that
much money on drug development?
Think about it:
Why does it cost so much to conduct a
clinical trial?
Why does it take so much time to conduct a
clinical trial?
Mind Map
To pull together what you have learned, you are going
to construct a “Mind Map” or concept map relating to
clinical trials.
A Mind Map is a special form of a web diagram for
exploring knowledge.
You start with the main idea and branch off from there.
Use different shapes, sizes or colors to show different
ideas and use lines to connect the related ideas.
On each of the lines, you must write the relationship
between the ideas or topics.
Here are Two Simple
Mind Maps.
• Can you tell the main topic of each map?
• What do the lines mean?
• Notice that the words on the lines show relationships
between the topics.
Here is one made using a computer
program:
In this example, the relationships are not written on the lines yet.
This is used mainly to show the use of colors and shapes.
Here is a Population Mind Map
This Mind Map is on how to make Mind Maps!
Mind Map
Your job is to make a Clinical Trials
Mind Map.
Be creative, your map will be different
from your neighbor’s map.
There is no “one right way” to do this.
Your map needs to make sense to you!
