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Drug Development Process - Part 1

The document provides an overview of the drug development process from target identification through pre-clinical trials. It discusses key stages including: 1. Target identification which involves discovering and validating disease targets through screening and identifying hit and lead compounds. 2. Pre-clinical research, which includes in vitro and in vivo studies to evaluate efficacy, dosing, and toxicity before human trials. Pre-clinical studies must follow good laboratory practices. 3. In vivo pharmacokinetic studies including RACE (rapid and efficient compressed) screening in small animal cohorts to determine properties and comprehensive PK analysis on further developed compounds. The drug development process aims to discover and optimize compounds through several stages before progressing to clinical trials in

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296 views19 pages

Drug Development Process - Part 1

The document provides an overview of the drug development process from target identification through pre-clinical trials. It discusses key stages including: 1. Target identification which involves discovering and validating disease targets through screening and identifying hit and lead compounds. 2. Pre-clinical research, which includes in vitro and in vivo studies to evaluate efficacy, dosing, and toxicity before human trials. Pre-clinical studies must follow good laboratory practices. 3. In vivo pharmacokinetic studies including RACE (rapid and efficient compressed) screening in small animal cohorts to determine properties and comprehensive PK analysis on further developed compounds. The drug development process aims to discover and optimize compounds through several stages before progressing to clinical trials in

Uploaded by

thang nguyen
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© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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Download as PDF, TXT or read online on Scribd

DRUG DEVELOPMENT PROCESS –

Part 1

29 Jun 2020
Objectives
1. Drug invention and development
timeline
2. Pharmaceutical and biotechnology
landscape
3. Compound selection and preclinical
trials
Overview of drug development
process
Overview of drug development
process - timeline
Overview of drug development
process - Cost
Overview of drug development
process - Cost
Target identification
Target identification
Target identification
• Target discovery, which involves the identification and early
validation of disease-modifying targets, is an essential first
step in the drug discovery pipeline.
• A hit compound is a molecule that shows the desired type
of activity in a screening assay
• Hit to lead (H2L) stage of drug discovery which aims to
discover compounds of interest (leads) from a selection of
compounds (hits) that demonstrate promising therapeutic
effects.
• The hit to lead process, which is also referred to as lead
generation, typically occurs following the completion of
target validation, assay development, and high-throughput
screening studies
• A lead compound has pharmacological or biological activity
likely to be therapeutically useful
Target identification
Target identification
• Phenotypic screening is a type of screening used in
biological research and drug discovery to identify substances
such as small molecules, peptides, or RNAi that alter
the phenotype of a cell or an organism in a desired manner.
• Target validation is the first step in discovering a new drug
and can typically take 2-6 months. The process involves the
application of a range of techniques that aim to
demonstrate that drug effects on the target can provide a
therapeutic benefit with an acceptable safety window
• Lead optimization aims at enhancing the most promising
compounds to improve effectiveness, diminish toxicity, or
increase absorption. Many of the technologies for lead
discovery overlap with lead optimization as researchers
attempt to incorporate the best drug characteristics early in
the process
Target identification
Pre-clinical research
• The two types of preclinical research are: in vitro and in
vivo
• 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.
– evaluate the efficacy of new drug products quickly and
accurately
– provide critical information for clinical trials
– help ensure patient safety
– meet FDA and international requirements for new drug
submissions
Pre-clinical research
• FDA requires researchers to use good laboratory practices
(GLP), defined in medical product development regulations,
for preclinical laboratory studies.
• Good laboratory practices (GLP), set the minimum basic
requirements for:
– study conduct
– personnel
– facilities
– equipment
– written protocols
– operating procedures
– study reports
– and a system of quality assurance oversight for each study to
help assure the safety of FDA-regulated product
Animal use in preclinical research
3R principles

Russell WMS, 1959, The principles of humane experiment technique, London Methuen, pp. 17-23 15
In Vitro PK and In Vivo PK
In Vitro PK and In Vivo PK

• RACE: This experiment is a rapid and efficient


compressed in vivo PK screening method to determine
the pharmacokinetic attributes of novel chemical
probes. A small cohort of animals (4 mice or 2
rats/experiment). R.A.C.E. is also to evaluate varying
formulation excipients for improving solubility and
absorption
• Comprehensive PK: compounds that show promising
PK profiles or that are further developed, can be
subjected to a comprehensive pharmacokinetic
analysis and metabolite identification study
In Vivo PK - RACE
In Vivo PK – Comprehensive PK

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