Drug Discovery and Clinical Trials
Clinical Trials: Research studies involving people
Why are clinical trials important:
A. Translate results of basic scientific research into better ways to prevent, diagnose,
or treat disease
B. The more people take part, the faster we can → Answer Questions → Find better
h
treatments and prevention methods
es
Did it happen before to give drugs without trial? Yes
Tragedies: Thalidomide Tragedy (Contergan scandal)→ Used to relieve nausea and
w
vomiting → Birth of Many Deformic Babies
ai
Few people participate in clinical Trials Causes:
hr
Finding volunteers is difficult
Inconvenient
Risk
Ik
Cost of Volunteers (They get paid for TIME SPENT not for Risk)
h
ee
Do we have trials in Jordan → Yes, Bioavailability Trials
Types of Clinical Trials:
m
Treatment (DM Drugs)
Prevention (Vaccines)
Sa
Early detection/screening
Diagnostic
Quality of life/supportive care
ik
al
The first steps in the development of a new
drug are:
M
Discovery or synthesis of a potential new
drug molecule
r.
Animal studies (Pre-clinical)
Human testing (Clinical studies)
D
1
, How are drugs discovered:
1. Identification or elucidation of a new drug target
2. Rational drug design of a new drug by: understanding of biologic mechanisms,
drug receptor structure, and drug structure
3. Chemical modification of a known molecule→ Start with one drug and look for
derivatives
4. Screening for biologic activity of large numbers of natural products, banks of
previously discovered chemical entities (Other uses of the same drug), and large
h
libraries of peptides, nucleic acids, and other organic molecules banks of previously
es
discovered chemical entities
5. Biotechnology and cloning using genes to produce peptides and proteins (Most
Expensive)
w
6. Combinations of known drugs to obtain additive or synergistic effects → Panadol Plus
or Chemotherapy
ai
hr
Confounding Factors in Clinical Trials:
1. Variable natural history of most diseases
Ik
2. Presence of other diseases & risk factors
3. Subject & observer bias → Avoided by Single Blind and Double Blind Studies
h
We avoid Confounding Factors → to avoid errors, a crossover design is used (Bioequivalence)
ee
Same ccc → 20 volunteers (First week 10 take panadol other 10 revanin)--> Wash out → اعكس
Subject & observer bias → Patient knwos he took a drug so might get better psychologically →
m
Decreased by Blinding (Useally Single Blinding)
Sa
We can also decrease Confounding Factors → Randomization: very important! (Distribution
without Bias)
ik
The Food & Drug Administration (FDA)
al
Administrative body that oversees the drug evaluation process in the USA and grants
approval for marketing of new drug products.
M
If a drug has not been shown through adequately controlled testing to be "safe and
effective" for a specific use → it cannot be marketed for this use
r.
responsibility of those seeking to market a drug to test it and submit evidence on its
relative safety and effectiveness.
D
In Jordan → JFDA → Before JFDA → Drug Directorate ()مديرية الدواء
From 0 to FDA Approval Duration: 15-20 Years
2
Clinical Trials: Research studies involving people
Why are clinical trials important:
A. Translate results of basic scientific research into better ways to prevent, diagnose,
or treat disease
B. The more people take part, the faster we can → Answer Questions → Find better
h
treatments and prevention methods
es
Did it happen before to give drugs without trial? Yes
Tragedies: Thalidomide Tragedy (Contergan scandal)→ Used to relieve nausea and
w
vomiting → Birth of Many Deformic Babies
ai
Few people participate in clinical Trials Causes:
hr
Finding volunteers is difficult
Inconvenient
Risk
Ik
Cost of Volunteers (They get paid for TIME SPENT not for Risk)
h
ee
Do we have trials in Jordan → Yes, Bioavailability Trials
Types of Clinical Trials:
m
Treatment (DM Drugs)
Prevention (Vaccines)
Sa
Early detection/screening
Diagnostic
Quality of life/supportive care
ik
al
The first steps in the development of a new
drug are:
M
Discovery or synthesis of a potential new
drug molecule
r.
Animal studies (Pre-clinical)
Human testing (Clinical studies)
D
1
, How are drugs discovered:
1. Identification or elucidation of a new drug target
2. Rational drug design of a new drug by: understanding of biologic mechanisms,
drug receptor structure, and drug structure
3. Chemical modification of a known molecule→ Start with one drug and look for
derivatives
4. Screening for biologic activity of large numbers of natural products, banks of
previously discovered chemical entities (Other uses of the same drug), and large
h
libraries of peptides, nucleic acids, and other organic molecules banks of previously
es
discovered chemical entities
5. Biotechnology and cloning using genes to produce peptides and proteins (Most
Expensive)
w
6. Combinations of known drugs to obtain additive or synergistic effects → Panadol Plus
or Chemotherapy
ai
hr
Confounding Factors in Clinical Trials:
1. Variable natural history of most diseases
Ik
2. Presence of other diseases & risk factors
3. Subject & observer bias → Avoided by Single Blind and Double Blind Studies
h
We avoid Confounding Factors → to avoid errors, a crossover design is used (Bioequivalence)
ee
Same ccc → 20 volunteers (First week 10 take panadol other 10 revanin)--> Wash out → اعكس
Subject & observer bias → Patient knwos he took a drug so might get better psychologically →
m
Decreased by Blinding (Useally Single Blinding)
Sa
We can also decrease Confounding Factors → Randomization: very important! (Distribution
without Bias)
ik
The Food & Drug Administration (FDA)
al
Administrative body that oversees the drug evaluation process in the USA and grants
approval for marketing of new drug products.
M
If a drug has not been shown through adequately controlled testing to be "safe and
effective" for a specific use → it cannot be marketed for this use
r.
responsibility of those seeking to market a drug to test it and submit evidence on its
relative safety and effectiveness.
D
In Jordan → JFDA → Before JFDA → Drug Directorate ()مديرية الدواء
From 0 to FDA Approval Duration: 15-20 Years
2
