RESEARCH, DRUG DEVELOPMENT DRIVERS, CHALLENGES, RISKS AND REWARDS

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Ten Days Faculty Development Program
Sponsored by
RUSA, MHRD, Govt. of India
Supported by
Institute of Chemical Technology, Mumbai
Organized by
Rajarshi Shahu College of Pharmacy, Buldana
(15th to 24th November 2019)

2. RESEARCH, DRUG DEVELOPMENT
DRIVERS, CHALLENGES, RISKS AND
REWARDS
11/29/2019
2
--DR.PRAKASH N. KENDRE
RSCP, BULDANA

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What is Research? ? ?
Research is a combination of both experience and reasoning
and can be said to be the most appropriate way of discovering the
truth, precisely in the natural sciences.
Research is the systematic study of trend or event which
involves careful collection, presentation, analysis and
interpretation of quantitative data or facts that relates man’s
thinking with reality.

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 Empirical- research is based on direct experience or observation
by the researcher
 Logical- research is based on valid procedures and principles
 Cyclical - research starts with a problem and ends with a
problem
 Analytical - research utilizes proven analytical procedures in
gathering data, whether historical, descriptive, experimental, and
case study
CHARACTERISTICS OF RESEARCH

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 Critical - research exhibits careful and precise Judgment
 Methodical -research is conducted in a methodical manner
without bias using systematic method and procedures
 Replicability -research design and procedures are repeated to
enable the researcher to arrive at valid and conclusive results

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Research Oriented
Efficient
Scientific
Effective
Active
Resourceful
Creative
Honest
Economical
Religious
QUALITIES OF A GOOD RESEARCHER…

7. CHARACTERISTICS OF RESEARCHER
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Intellectual Curiosity – researcher undertake deep thinking and
inquiry of the things, problems, and situations around him.
Prudence – researcher is careful to conduct his study at the right
time and at the right place wisely, efficiently, and economically.
Healthy Criticism – the researcher is always doubtful as to the
truthfulness of the results.
Intellectual Honesty – researcher is honest to collect or gather the
data or facts in order to arrive at honest results.
Intellectual creativity – a resourceful investigator always creates new
researches. He enjoys inventing unique, novel and original researches, and
considers research as his hobby. (creative researcher is innovative)

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Our Needs and Problems Demand Research…
• how to reduce his burden of work;
• how to relieve suffering;
• how to increase satisfaction in fulfilling his needs,
cravings and aspirations

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VALUES OF RESEARCH TO MAN
Research Improves quality of life – It has led man to search for ways for
improving processes and means which man love and find interesting
Research improves instruction – Modern teacher introduces innovations
and integrates the 3Rs (rational thinking, right conduct, and research) in
all subject areas in his teaching
Research improves students’ achievement
Research improves teachers’ competence
Research satisfies man’s needs – modern facilities that satisfy man’s needs
are all products of research
Research has deep-seated psychological aspects Research responds to
the economic recovery and austerity measures of the country

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TYPES OF RESEARCH
Basic
Research
Applied
Research
Developmental
Research

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CLASSIFICATION OF RESEARCH
Library
Research
Field
Research
Laboratory
Research

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NEED OF RESEARCH
The need to research came due to the following reasons:
To acquire a degree
To get respectability
To face a challenge
To solve a problem
To get Intellectual Joy
To Serve Society by increasing Standard of living for
Science and technology and by showing right path to
society in case of social and behavioral Sciences.

13. COMPONENTS OF RESEARCH
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Problem/Objective
Hypothesis
Theoretical/conceptual framework
Review of literature and Studies
Research Design
Data Collection
Data Processing and Statistical Treatment
Analysis and Interpretation
Summary, Conclusion and Future
Recommendations

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GOOD QUALITY OF RESEARCH
A good research method should lead to…
Originality/ Novelty
Contribution to knowledge
Significance
Technical soundness
Critical assessment of existing work
“If you steal from one author it's plagiarism; if you steal from
many it's research.”
- Wilson Mizner

15. Strategic
Research
Exploratory
Research
Two types
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STAGES OF THE DRUG DISCOVERY AND
DEVELOPMENT PROCESS

16. Strategic research of industry is based on….
Inherent research competence &
expertise
Therapeutic areas of unmet medical
needs
Market potential/commercial
viability
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17. Exploratory research of industry is based on….
Investigation of the biological mechanism and identification
of a “chemical lead” that interferes with it.
• Combinatorial
Chemistry
Diverse
synthetic
molecules
•HTSBio-synthetic
molecules
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18. In addition to the use of HTS for pharmacological activity,
HTS tests have been developed for assessing metabolism and
pharmacokinetic and toxicity factors to speed up the drug
discovery process.
Othersupporting factors to speed-up the drug discovery process
Rational drug design
QSARs
Genomics
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19. Drug
Discovery Drug
Product
Pre-formulation
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22. The objective of pharmaceutical R&D can be defined as
“converting ideas into candidate drugs for development”
and the objective of product development defined as
“converting candidate drugs into products for registration
and sale”.
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23. In reality, these goals are extremely challenging and difficult to
achieve because of the many significant hurdles a pharmaceutical
company has to overcome during the course of drug development.
Today, a pharmaceutical scientist is well versed with the fact
that the overall action of a drug molecule is not merely
dependent on its inherent therapeutic activity, rather on the
efficiency of its delivery at the site of action.
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24. Research : Novel compound (patentable?)
Novel biological mechanism (patentable?)
Unmet medical needs
Potent and selective
Safety : High margin of safety
Non-toxic (not carcinogenic, tetratogenic,
mutagenic, etc.)
Clinical : Tolerable side-effects profile
Efficacious
Drug process : Bulk drug can be synthesized/scaled up
Pharmaceutical : Acceptable formulation/pack (meets
customer needs)
Drug delivery/product performance
acceptable
Stable/acceptable shelf-life
Clinical trial process robust and can be
scaled up
Activity Requirements
Major Hurdles to Successful Product Registration and Sale
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25. Marketing/commercial Competitive Meets customer needs
Value for money Commercial return
Regulatory Quality of data/documentation
Manufacturing Manufacturable
Able to pass pre-approval inspection
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26. Only 1 in 5,000 to 1 in 10,000 compounds
screened in research will reach the market
The failure rate will vary from 1 in 5 to 1 in 10 compounds that
will achieve registration and reach the market-place.
There is a significant commercial risk from those that are
marketed; only 3 out of 10 are likely to achieve a fair return on
investment (ROI)
THE HIGH RISK OF FAILURE IN DRUG DISCOVERY AND
DEVELOPMENT
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27. The products which give poor return on investment are often the
result of poor candidate drug selection, the compound does not
have the desired properties of …..
•Safety, purity and or
•selectivity
•efficacy
•potency or duration
Poor product
development
The latter scenario should, and can be, avoided by careful
assessment at the “product design” stage of development
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28. STRATEGIC
RESEARCH
MARKETING &
COMMERCIAL
EXPLORATORY
RESEARCH
EXPLORATORY
DEVELOPMENT
CANDIDATE
SELECTION
FULL
DEVELOPMENT
Based on company
strategy
Further
market/medical needs
new indications
Launch
Regulatory
submissions
£/$
Sales &
profits
Safety and efficacy
demonstrated
Proof of concept
demonstrated
Candidate drug
selected
Further market/
medical needs
product line
extensions
Product life cycle
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A simple definition of “product design”
is: “the initial stage of product
development, where ‘global’
agreement is required about the nature
of the product to be developed”

31. Quality function deployment (QFD), referred to as
“customer driven engineering” and “matrix product
planning”, is a useful quality and planning tool which
uses a structured approach in defining all the customer
needs or requirements and translates these into design
requirements for product development.
Quality Function Deployment (QFD)
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House of
Quality

34. 1
• To provide clear direction and objectives for the project team
2
• To gain buy-in and input from all the key functions at the start of
development (such as Pharmaceutical Development, Safety,
Clinical, Manufacturing Operations, Quality Assurance,
Regulatory and Marketing)
3
• To assess the feasibility of the project in commercial and technical
terms
4 • To identify any risks early and hence manage them
5
• To avoid wasting valuable resources on developing a product
which is not needed or wanted
6 • To provide a good reference source for the development plan
Important Benefits of Effective Product Design
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35. Product Design Report
1
• Target product profile/minimum product profile
2
• Design specification and critical quality parameters
3
• Commercial and marketing considerations
4
• Technical issues and risk assessment
5
• Safety assessment considerations
6
• Environmental, health and safety considerations
7 • Intellectual property considerations
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Commercial and Marketing Considerations
Development costs
Timing to market
Market size (disease prevalence, diagnosis and
treatment rates, market value)
Competition (current, developing and impact
on future market)
Cost of goods (target)
Unmet medical need (effectiveness of current
treatment, improvements required)
 Pricing and reimbursement (current and future)

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THANK YOU!