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https://www.fda.gov/ForPatients/
FDA’S DEFINITION OF A NEW DRUG
Pre-discovery
Understand the disease
Target identification
Choose a molecule to target with a drug
Target validation
Test the target and confirm its role in the disease
Drug discovery
Find a promising molecule (a “lead compound”)
that could become a drug
..... DRUG DEVELOPMENT
High-throughput Screening
A most common way that leads are usually found.
Advances in robotics and computational power
allow researchers to test hundreds of thousands
of compounds against the target to identify any
that might be promising.
Biotechnology
Scientists can also genetically engineer living
systems to produce disease-fighting biological
molecules.
THE ASSIGNMENT OF FUNCTION TO THE
PRODUCTS OF SEQUENCED GENES CAN BE
PURSUED VIA VARIOUS APPROACHES:
Sequence homology studies – degree of
similarity between sequences
Phylogenetic profiling – predict functional
relationships
Rosetta stone method
Gene neighborhood method – the genes that
encode the metabolic pathways
Knockout animal studies
.....SEQUENCED GENES APPROACHES
Preclinical Research
Drugs undergo laboratory and animal testing to
answer basic questions about safety.
Before testing a drug in people, researchers must
find out whether it has the potential to cause serious
harm, also called toxicity.
Can take up to three years to complete.
During this stage, researchers must also work out
how to make large enough quantities of the drug for
clinical trials.
This is the first scale up.
.... PRECLINICAL RESEARCH
In Vivo
FDA requires researchers extremely thorough testing
and use good laboratory practices (GLP), defined in
medical product development regulations, for
preclinical laboratory studies.
Good Laboratory Practice
study reports
and a system of quality assurance oversight
for each study to help assure the safety of
FDA-regulated product
Usually, preclinical studies are not very large.
However, these studies must provide detailed
information on dosing and toxicity levels After
preclinical testing, researchers review their
findings and decide whether the drug should be
tested in people.
THE RANGE OF MAJOR TESTS
UNDERTAKEN
Pharmacokinetic profile
Pharmacodynamic profile
Bioequivalence and bioavailability
Acute toxicity
Chronic toxicity
Reproductive toxicity and teratogenicity
Mutagenicity
Carcinogenicity
Immunotoxicity
STEP 3: CLINICAL RESEARCH
Clinical Research
Drugs are tested on people to make sure they
are safe and effective.
1. Fast Track
Fast track is a process designed to facilitate
the development, and expedite the review of
drugs to treat serious conditions and fill an
unmet medical need.
2. Breakthrough Therapy
A process designed to expedite the
development and review of drugs which may
demonstrate substantial improvement over
available therapy.
....FDA REVIEW
3. Accelerated Approval
These regulations allowed drugs for serious
conditions that filled an unmet medical need
to be approved based on a surrogate endpoint .
4. Priority Review
A Priority Review designation means FDA’s
goal is to take action on an application within
6 months.
NEW DRUG APPLICATION
Supplemental Applications
INDs for Marketed Drugs
Manufacturer Inspections
Drug Advertising
Generic Drugs
Reporting Problems
Active Surveillance
SUPPLEMENTAL APPLICATIONS
RA 6675
An act to promote, require, and ensure the
production of an adequate supply, distribution, use,
and acceptance of drugs and medicines identified by
their generic names.
Approved: September 13, 1988
RA 9502 – Universally, Accessible Cheaper and
Quality Medicines Act of 2008; amending RA 8293
(Intellectual Property Code), RA 6675 and RA 5921
(Pharmacy Law)
Approved: June 06, 2008
.....GENERIC DRUGS