Perspectives in Pharmacy
Scope and Fields of Pharmaceutical
Services
PHARMACY - art and science of preparing,
dispensing and proper utilization of medications
and provision of drug related information to the
public.
- Preparing from natural and synthetic
sources.
- “The profession responsible for the
appropriate use of medications, devices
and services to achieve optimal
therapeutic outcomes”
-American
Pharmaceutical Association
PHARMACEUTICAL CARE - responsible
provision of drug therapy for the purpose of
achieving definite outcomes that improve a
patient’s quality of life.
PHARMACIST - “educated and licensed to
dispense drugs and provide drug information.”
- Drug experts
- Most accessible member of today’s
healthcare team.
DRUGS - articles intended for use in diagnosis,
cure, mitigation, treatment or prevention of
diseases.
Pharmaceutical Services
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Community Pharmacy - “Retail Pharmacy”
- This is the area of service mostly
strongly identified with the word
pharmacy
Industrial Pharmacy - Research, Production,
Quality Control, Distribution/Marketing
- Research: formulation of the
most suitable dosage forms.
- Production: raw materials to
finished product.
Hospital Pharmacy - Pharmacists dispense
medications, prepare sterile solutions, advice other
professionals and patients on the use of drugs,
monitor drug regimens and evaluate drug use.
Clinical Pharmacy - said to be the saving
grace to the commercialism pervading the
profession.
● Pharmacists in Education - responsible for
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the nature and quality of pharmaceutical
education
- “Shapes the course of profession”
Wholesaling - products of hundred
manufacturers can be obtained from a single
agency.
Pharmaceutical Journalism - offers rewarding
experience for a limited number of pharmacists
with writing and editing skills.
Government Services - Law Enforcers, Board
Examiners, Food and Drug Analyst.
- Organization Management - offers an
opportunity for pharmacists who want to
be officers of national and provincial
associations.
- Philippine Pharmacists
Association (PPhA)
Medical Communication - embraces many
activities including computer handling of
medical data, creation of reference materials,
and research, evaluate, assemble and package
information specifically for the end user.
Nuclear Pharmacy - applies principles and
practices of pharmacy and nuclear chemistry to
produce radioactive drugs used for diagnosis
and therapy.
PPhA and its Affiliate Organizations:
PAPPI - Philippine Association of Pharmacist in
the Pharmaceutical Industry
PACOP - Philippine Association of Colleges of
Pharmacy
PSHP - Philippine Society of Hospital
Pharmacy
CPAP - Community Pharmacist Association of
the Philippines.
PAPVI - Philippine Association of Pharmacists
in Veterinary Industry
DOH-LP - Department of Health-League of
Pharmacists
IPAP - Industrial Pharmacists Association of
Philippines
DSAP - Drugstore Association of the
Philippines
FJCPPhA - Federation of Junior ChapterPhilippine Pharmacist’s Association
YPG - Young Pharmacist Group Philippine.
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Perspectives in Pharmacy
Pharmaceutical Product Development
Definitions of a Drug:
- Article intended for and having as their
main use the diagnosis, cure, mitigation,
treatment or prevention of disease in
man and other animals
- Article recognized by monograph in the
compendia
- Article (other than food) used to affect
the structure or any function of the body
of man and other animals
- Article used as a component of any of
the previously mentioned articles.
Active Ingredient
- any component that is intended to
furnish pharmacological activity or other
direct effect in the diagnosis, cure,
mitigation, treatment or prevention of
disease or to affect the structure or
function of the body of man or other
animals
The Pharmaceutical Product Development
Process
- Research activities directed to the
creation of new drugs for new products
- New drugs may be intentionally or
accidentally produced from natural
sources or by chemical synthesis
Sequence of Events in Pharmaceutical
Product Development
Pharmaceutical Product Development
● Drug Discovery
- Determine target disease.
- Develop hypothesis for a
mechanism of treatment.
- Evaluate hypothesis.
- Determine feasibility of producing
and evaluating the selected
compound.
● Screening
- Combination Chemistry
- Make many possible compounds
at one time.
- Focus on quantity of possible
compounds, not purity of each.
- High Throughput Screening
- Test hundreds at a time for
activity.
- Process requires serious technology.
- 1 in 10,000 makes it to the
market.
Two Main Stages of Drug Development
● Preclinical Research
- involves synthesis and purification
testing in the lab and animal testing
- thousands of compounds are tested
in preclinical research before a
handful are chosen to enter the
second stage, which requires filing
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of an IND
Pre-Clinical Testing
- Evaluate acute and short term
toxicity in animals (one rodent,
one non-rodent).
- Dose at increasingly high
levels to induce toxicity.
- Determine lethal dose.
- Dose at normal levels for
short and long term.
- Assess how drug is absorbed,
distributed, metabolized, and
excreted in animals.
Primary goals of early preclinical
development
- to determine if the product is
reasonably safe for initial use in
humans
- to determine if the compound
exhibits pharmacological activity
that justifies commercial
development.
- to determine recommended dose
for initial use in humans
- to establish the physicochemical
properties of the drug: its
chemical makeup, stability,
solubility
Objective 1: Safety
- Toxicity Studies - done on
experimental animals: murine
(rats or mice), canines (dogs),
primates (monkeys), porcine
(pigs)
- Dose at increasingly high levels
to induce toxicity
- Determine lethal dose
- Dose at normal levels for short
and long term
Objective 2: Pharmacologic activity
- Pharmacodynamics – what the
drug does to the body
- Pharmacokinetics – what the
body does to the drug ADME –
absorption, distribution,
metabolism excretion
Objective 3: Recommended Dose
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Safe starting dose for clinical
trials
- Objective 4: Physicochemical
properties
- Structural characterization
- Impurity identification
- Solubility assessment
- Stability
Output – Raw Material
Specifications
Pharmaceutical Manufacturing Research
- The aim of pharmaceutical
manufacturing development is to
design a quality product and its
manufacturing process to
consistently deliver the intended
performance of the product.
- The information and knowledge
gained from pharmaceutical
manufacturing development studies
and manufacturing experience
provide scientific understanding to
support the establishment of the
design space*, specifications, and
manufacturing controls.
Investigational New Drug (IND)
- Application filed by a sponsor to get
FDA approval to conduct clinical
studies of an IND on human subjects
- Investigational new drug means a
new drug or biological drug that is
used in a clinical investigation. The
terms ‘‘investigational drug’’ and
‘‘investigational new drug’’ are
deemed to be synonymous for this
purpose.
- Clinical investigation (or trial) means
any experiment in which a drug is
administered or dispensed to, or
used involving, one or more human
subjects.
- Sponsor means a person who takes
responsibility for and initiates a
clinical investigation.
- Contract research organization
means a person that assumes, as
an independent contractor with the
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sponsor, one or more of the
obligations of a sponsor, e.g., design
of a protocol, selection or monitoring
of investigations, evaluation of
reports, and preparation of materials
to be submitted to the Food and
Drug Administration.
- Investigator means an individual
who actually conducts a clinical
investigation (i.e., under whose
immediate direction the drug is
administered or dispensed to a
subject).
- Sponsor-Investigator means an
individual who both initiates and
conducts an investigation, and under
whose immediate direction the
investigational drug is administered
or dispensed.
- Subject means a human who
participates in an investigation,
either as a recipient of the
investigational new drug or as a
control.
Institutional Review Board (IRB)
- Reviews, approves, and monitors
research involving human subjects
in order to evaluate the ethical
acceptability and the scientific
validity of any such studies.
- An IRB is formally designated by an
institution in which research takes
place, such as a hospital or
university.
- Research cannot begin until the
IRB approves.
Clinical Studies
- Clinical trials are research studies
done to evaluate whether a drug or
treatment is both safe and effective
for people.
- Prior to being used in clinical trials,
drugs or procedures are first
evaluated extensively in the lab
and/or in animal studies.
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Participating in a clinical trial can
provide researchers with valuable
information on new treatments; in
some cases, it may also offer the
chance for a cure or improvement in
a patient's quality of life that's not
provided by standard therapy.
Participation in clinical trials is
voluntary, and individuals are
allowed to discontinue the treatment
at any time .
The purpose of clinical trials is to
decide if a new medication or
treatment is safe and effective.
Clinical trials are research studies
that are conducted on human
subjects to find more effective ways
to treat, prevent, or diagnose
disease.
They may also be done to find
treatments with fewer side effects, or
treatments that are easier for
patients to tolerate.
Phase 0
- A recent designation for exploratory,
first-inhuman trials conducted in
accordance with the US FDA’s 2006
Guidance on Exploratory IND Studies.
- They are exploratory studies that often
use only a few small doses of a new
drug in a few patients
- Also known as human microdosing
studies
- Designed to speed up the development
of promising drugs by establishing very
early on whether the drug behaves in
human subjects as was expected from
preclinical studies.
- Involves the administration of single
subtherapeutic doses of the study drug
to a small number of subjects (10 to 15)
to gather preliminary data on the agent's
pharmacokinetics (what the body does
to the drug).
- Help researchers find out whether the
drugs do what they are expected to do.
- May help avoid the delay and expense
Perspectives in Pharmacy
of finding out years later in phase II or
even phase III clinical trials that the drug
doesn’t act as expected to based on lab
studies
- If there are problems with the way the
drug is absorbed or acts in the body, this
should become clear very quickly in a
phase 0 clinical trial.
- Not yet being used widely, and there are
some drugs for which they wouldn’t be
helpful.
- Studies are very small, often with fewer
than 15 people, and the drug is given
only for a short time.
- They’re not a required part of testing a
new drug.
Phase I
- This phase consists of short-term
clinical tests of the drug on 20 to 80
healthy volunteers to determine basic
pharmacological and toxicological
information in humans especially as
regards safety. The FDA can stop
clinical testing if they deem the drug
unsafe.
- Studies that are usually conducted with
healthy volunteers and that emphasize
safety.
- The goal is to find out what the drug's
most frequent and serious adverse
events are and, often, how the drug is
metabolized and excreted.
- The aims of this initial trial include:
- Determining that the drug is safe for
humans
- Gaining information about proper
dosage amounts
- Assessing side effects at different
dosage amounts
- Determining how the drug is
metabolized by the human body
- Assessing the effect of the drug on
target symptoms
- Phase I typically takes about one year,
which is much shorter than other phases
of the trial period.
- The short time period is because a
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Phase 1 trial is not used to see if the
drug is effective, only that it is mainly
safe for humans to use.
Some of the questions researchers may
be looking to answer about a drug with a
Phase 1 trial include:
• Does it cause any serious side
effects?
• Are patients able to tolerate the drug?
• What's the safest route of
administration (such as pill, injection,
infusion)?
• How is it metabolized by the body?
• What's the highest dose that is
tolerable to patients?
Phase II
Conducted on a larger group of people
to further evaluate the safety of the
treatment and to determine how well it
works.
Typically the drug is tested in 100 to 300
patients.
Dosage levels are experimented with to
find optimal dosage levels, and further
information on safety is collected
The main purpose of a Phase II clinical
trial is to begin to assess whether a drug
is effective while exposing as few people
as possible to any unwanted side
effects.
All study participants will have been
diagnosed with the condition that the
new medication has been designed to
treat.
Phase III
Designed to provide proof of the drug's
effectiveness in treating the target health
problem and to assess the risk vs.
benefits of using the drug
Drug is given to large groups of people
often numbering in the thousands (1,000
to 3,000)
Data is gathered regarding side effects
and optimal dosage amounts in order to
provide labeling and physician
prescription information
Trials are tightly controlled and can take
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several months to several years to
complete
Done to determine if the new treatment
is more effective, or has less side
effects, than the standard (currently
approved) method.
This phase answers the question "does
it work better than the standard
treatment?"
Controlled clinical trial is a type of
treatment research study in which a
treatment is compared to a control
group. This control group may be either
another active treatment (e.g., another
medication), a non-active treatment
(e.g., a placebo), or some other type of
control group.
In some forms of research, a control
group is used to provide a basis of
comparison to the group of individuals
who are being studied in support of the
research theory
A placebo is a substance given to a
study participant that has no known
treatment value.
Placebos are made as similar to the
actual treatment as possible. If the new
medication being studied is in the form
of an injection, the placebo will be an
injection. The idea is that the study
participants and the researchers do not
know who is getting the real treatment
and who is getting a placebo.
Trials are typically conducted in a
"double-blind" manner, where neither
the researchers nor the participants
know who received the placebo and
who received the medication.
In this study, half of the participants
usually receive a placebo and the other
half get the actual medication of interest.
Double-blind studies are conducted to
prevent bias on the part of the
researchers and the participants.
Any effects seen as a result of taking the
placebo are usually attributed to the
expectations of the patient is known as
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the "placebo effect."
New Drug Application (NDA)
The NDA application is the vehicle
through which drug sponsors formally
propose that the FDA approve a new
pharmaceutical product for sale and
marketing.
The data gathered during the animal
studies (preclinical) and human clinical
trials of an Investigational New Drug
become part of the NDA.
The goals of the NDA are to provide
enough information to permit the FDA
reviewer to reach the following key
decisions:
• Whether the drug is safe and effective
in its proposed use(s), and whether the
benefits of the drug outweigh the risks.
• Whether the drug's proposed labeling
(package insert) is appropriate, and
what it should contain.
• Whether the methods used in
manufacturing the drug and the controls
used to maintain the drug's quality are
adequate to preserve the drug's identity,
strength, quality, and purity.
The documentation required in an NDA
is supposed to tell the drug's whole
story, including
• what happened during the clinical
tests,
• what the ingredients of the drug are,
• the results of the animal studies,
• how the drug behaves in the body,
• how it is manufactured, processed and
packaged
Abbreviated New Drug Application
(ANDA)
- Contains data which when submitted
to the provides for the review and
ultimate approval of a generic drug
product.
- Once approved, an applicant may
manufacture and market the generic
drug product to provide a safe,
effective, low cost alternative to the
public.
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Generic Drug Product
• Product that is comparable to
an innovator drug product in
dosage form, strength, route of
administration, quality,
performance characteristics and
intended use.
• Generic drug applications are
termed "abbreviated" because
they are generally not required to
include preclinical (animal) and
clinical (human) data to establish
safety and effectiveness.
• Instead, generic applicants are
required to scientifically
demonstrate that their product is
bioequivalent (i.e., performs in
the same manner as the
innovator drug).
• Generic applicants are required
to scientifically demonstrate that
their product is bioequivalent
(i.e., performs in the same
manner as the innovator drug).
• One way scientists
demonstrate bioequivalence is to
measure the time it takes the
generic drug to reach the
bloodstream in 24 to 36 healthy
volunteers.
• This gives them the rate of
absorption, or bioavailability, of
the generic drug, which they can
then compare to that of the
innovator drug.
• The generic version must
deliver the same amount of
active ingredients into a patient's
bloodstream in the same amount
of time as the innovator drug. bioequivalence
Marketing Authorization (MA)
Certificate of Product Registration
(CPR)
Routine Commercial Production New
Product Launching Marketing
Phase IV
- Done in the post-marketing stages after the Food and Drug
Administration (FDA) has approved
the product for general use
- Studies are conducted on hundreds
to thousands of people
- Study answers the question "does it
work over time and for purposes
other than what it was initially meant
for?"
- done to continue monitoring the risks
and benefits of the drug
- may look at the drug's effect on
different subgroups of individuals
(such as different genders or ages)
or to gather information about the
long-term effects of drug usage
- may be conducted in order to get
FDA approval for the drug to be
used for a different purpose than the
target symptoms it was originally
approved for