Amity Institute of Pharmacy

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Amity Institute of Pharmacy

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 Amity Institute of Pharmacy

Drug Discovery
• Designing and developing new chemical moieties for
disease treatment.

• Involves chemistry, biology, and pharmacology.

• Historically sourced from plants (e.g., Opium, Quinine,

Digitalis).

• Presently, most therapeutic agents are synthetic.

• The process is complex, costly, and time- consuming.

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Stages of Drug Discovery &

Development
1. Discovery
2. Preclinical Testing
3. Clinical Trials
4. Regulatory Approval
5. Post-Marketing Surveillance

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Target Identification
• Identifies a protein or gene responsible for
a disease.
• Drug must interact with this target to be
effective.
• Sources of targets: Scientific literature,
target deconvolution, and discovery.

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Target Validation
• Confirms that the target is responsible for the
disease.
• Helps avoid ineffective drugs.
• Validation steps include:
• Reproducibility (repeat experiments for
accuracy)
• Mutation testing
• Drug interaction analysis

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Lead Compound Identification

• A lead compound is a molecule with desired
pharmacological effects.
• Sources of lead compounds:
• Natural sources (plants, animals,
microorganisms)
• Chemical libraries (large collections of
compounds)
• Computational medicinal chemistry (computer-
aided drug design)
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Lead Optimization
• Enhancing the potency, selectivity, and safety of
lead compounds.
• Involves:
• Structure-activity relationship (SAR) analysis
• Chemical modifications to improve efficacy
• Toxicity reduction

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Preclinical Testing
• Conducted before human trials.
• Involves in-vitro (cell culture) and in-vivo
(animal) studies.
• Assesses drug safety, toxicity, and
pharmacokinetics.
• Helps in selecting promising candidates for
human trials.

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Clinical Trials Overview

• Conducted in human subjects.
• Aim: Evaluate safety, efficacy, and dosage.
• Conducted in four phases:
• Phase I: Small group (safety and dosage)
• Phase II: 100-250 patients (efficacy and side
effects)
• Phase III: Large-scale trials (confirmation of
efficacy)
• Phase IV: Post-marketing surveillance
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Concept of Generic Drug?

• According to FDA:
• A generic drug is a medication created to be the same
as an existing approved brand-name drug in:
• - Dosage form
• - Safety
• - Strength
• - Route of administration
• - Quality
• - Performance characteristics

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What is an Innovator Drug?

• An innovator drug is the first drug created with a
specific active ingredient to receive approval.
• It is the original formulation with established
efficacy, safety, and quality.
• Patents usually last up to 20 years.

• Other companies cannot manufacture or sell the

drug until the patent expires.
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Generic vs. Brand-Name

Differences
• Generic drugs use the same active ingredients
as brand-name drugs.
• Minor differences may exist in inactive
ingredients.
• There is slight, but not medically significant,
variability in batches.
• FDA strictly regulates acceptable variability.

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FDA’s Role in Ensuring Generic Drug

Effectiveness
• FDA ensures that generic medicines:
• Perform the same as brand-name medicines.
• Meet the same high standards of quality and
manufacturing.
• Have the same risks and benefits.
• Undergo rigorous FDA review and inspections.
• Have acceptable variability levels.

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Why do generic medicines often

cost less than brand-name
medicines?
Generic drugs are approved only after a rigorous review
by FDA and after a set period of time that the brand
product has been on the market exclusively. This is
because new drugs, like other new products, are usually
protected by patents that prohibit others from making
and selling copies of the same drug.

Generic drugs tend to cost less than their brand-name

counterparts because generic drug applicants do not
have to repeat animal and clinical (human) studies that
were required of the brand-name medicines to
demonstrate safety and effectiveness. This abbreviated
pathway is why the application is called an “abbreviated
new drug application.” 19
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Are generic drugs as good as brand-

names?

Generic pharmaceutical manufacturers must prove to the FDA

that their version of a drug:

contains the same active ingredient;

 is identical in strength, dosage form, and route of

administration;

has the same indications, dosing, and labeling;

provides the same efficacy and safety profile to patients

(“bioequivalent”) 20
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Generic Drug Development

Current development and approval of generic drug products

were associated with issues concerning. Safety, efficacy and
therapeutic equivalence of such products early compared to the
innovator or brand-name drug product for obtaining marketing
approval.

However, the generic pharmaceutical industry is still challenged

by legislative, regulatory and scientific issues that must be
addressed to allow for the manufacture, approval and marketing
of generic drug products. Generic drug product manufacturers
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must formulate a drug product that will have the same
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Selection of Generic Drugs For Manufacture

The main driving force for the selection of generic drug products
for manufacture is the estimated sales volume for the branded
product.

Formulation considerations for generic

drugs include

The availability of raw materials, chemical purity, and polymorphic

form. Particle size of the active pharmaceutical ingredient

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Steps in Generic Drug Product Development

Process

CONCEPT DEVELOPMENT
The needs of the target market are identified, alternative product
concepts are generated and evaluated, and a single concept is
selected
SYSTEM for further
LEVEL DESIGNdevelopment

It includes the definition of the product into subsystems and

components. The final assembly scheme for the production system
is usually defined during this phase

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DETAIL DESIGN

Includes the complete specifications of the geometry, materials

and tolerances of all the unique parts in the product and the
identification of all the standards parts to be purchased from
suppliers.

A process plan is established and tooling is designed for the each

part to be fabricated within the production system.

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TESTING AND REFINEMENT

Early prototypes are usually built with production-intent

parts(parts with the same geometry and material properties as
intended for the production version of the product).It involves
the construction and evaluation of multiple pre-production
versions of the product.

Early prototypes are tested to determine whether the product

will work as designed and whether it satisfies the customer’s
needs. The goal of prototypes is usually used to answer
questions about performance and reliability in order to identify
changes for the final product 25
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PRODUCTION RAMP UP

The purpose is to train the work force and to work out any
remaining problems in the production process.

The artifacts produced during production ramp up are sometimes

supplied to the preferred customer and are carefully evaluated to
identify any remaining flaws

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Generic Drug Approval Process:

The FDA’s Office of Generic Drugs is responsible for reviewing the

ANDA and approving the drugs products marketing.

The FDA’s Office of Generic drugs has a website

http//www.fda.gov.org that provides additional information for
manufacturers of generic drug products including a flow chart
presentation of the ANDA review process.

It also describes how FDA determines the quality, safety, and

efficacy of generic drug products prior to the approval for 27
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Generic drug application reviewers focus on bioequivalence data,

chemistry and manufacture quality, microbiology data where
relevant, requests for plant inspection, and drug labelling
information

The ANDA for generic drug product approval is based on

bioequivalence to the brand name product, appropriate chemistry
and manufacturing information, and appropriate labelling

Generic drug sponsors do not have to perform the nonclinical

animal toxicity studies or expensive clinical efficacy and safety
studies that are included in the new drug application .NDA which
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is submitted to the FDA for market approval of the brand name
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The ANDA contains data which is then submitted to FDA’s Center

for drug evaluation and research for the generic drugs.

FDA-approved generic drugs must meet the same rigid

standards as the innovator drug.

To obtain FDA approval, a generic drug product must- contain

the same active ingredient as an approved drug product the
inactive ingredients may vary.

Be identical in strength, dosage form, route of administration,

same indications, bioequivalent, and meet the batch
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QUIZ
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Section 1: Stages of Drug Discovery

1. What is the first step in the drug discovery process?
a) Pre-clinical testing
b) Target identification and validation
c) Clinical trials
d) Drug manufacturing

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2. Which of the following methods is commonly used for drug

discovery?
a) High-throughput screening
b) Random trial-and-error testing
c) Traditional herbal medicine only
d) Marketing analysis

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3. What is the role of computational drug design in drug discovery?

a) Predicting drug-target interactions
b) Conducting human trials
c) Manufacturing large-scale drugs
d) Replacing laboratory experiments completely

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4. Which organization regulates the approval of new drugs in the United

States?
a) WHO
b) FDA
c) EMA
d) ICH

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Section 2: Drug Development Process

5. Which of the following is NOT a stage in the drug development
process?
a) Pre-clinical testing
b) Clinical trials
c) Marketing surveillance
d) Drug advertising

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6. The purpose of drug formulation development is to:

a) Determine drug safety and efficacy
b) Optimize drug delivery and stability
c) Conduct human trials
d) Assess legal aspects of drug marketing

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7. What is the term for modifying an existing drug to improve its safety or
effectiveness?
a) Drug repurposing
b) Drug optimization
c) Drug recycling
d) Drug withdrawal

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Section 3: Pre-Clinical and Non-Clinical Activities

8. What is the main goal of pre-clinical studies?
a) To test the drug in human volunteers
b) To assess safety and efficacy in animal models
c) To conduct large-scale manufacturing
d) To determine the drug price

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9. Which of the following is NOT a type of pre-clinical study?

a) In vitro testing
b) In vivo animal studies
c) Human clinical trials
d) Toxicology studies

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10. Which regulatory document is submitted before beginning human

clinical trials?
a) Investigational New Drug (IND) application
b) New Drug Application (NDA)
c) Drug Master File (DMF)
d) Abbreviated New Drug Application (ANDA)

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Section 4: Clinical Studies

11. Which phase of clinical trials primarily tests for drug safety?
a) Phase I
b) Phase II
c) Phase III
d) Phase IV

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12. Phase III clinical trials focus on:

a) Testing in a small number of healthy volunteers
b) Determining large-scale efficacy and safety in patients
c) Post-marketing surveillance
d) Identifying drug toxicity in animals

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13. Which phase of clinical trials continues after the drug is

marketed to monitor long-term effects?
a) Phase I
b) Phase II
c) Phase III
d) Phase IV

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Section 5: Innovator and Generic Drugs

14. What is the key difference between an innovator drug and a generic
drug?
a) Innovator drugs are patented, while generics are not
b) Generics have different active ingredients than innovator drugs
c) Innovator drugs are more effective than generics
d) Generics require longer approval time than innovator drugs

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15. Which regulatory submission is required for the approval of a generic

drug in the U.S.?
a) IND
b) NDA
c) ANDA
d) BLA

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16. Bioequivalence studies for generic drugs compare which of the

following with the innovator drug?
a) Chemical composition
b) Manufacturing process
c) Pharmacokinetic parameters
d) Brand name

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Section 6: Generic Drug Product Development

17. A generic drug must be equivalent to the innovator drug in:
a) Pharmacokinetics and pharmacodynamics
b) Marketing and packaging
c) Price and availability
d) Route of administration only

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18. What does "therapeutic equivalence" mean in generic drug development?

a) The generic drug is identical in all aspects to the innovator drug
b) The generic drug has the same clinical effect and safety profile as the
innovator drug
c) The generic drug is manufactured by the same company as the innovator drug
d) The generic drug does not require FDA approval

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19. Which of the following is a challenge in generic drug development?

a) Establishing bioequivalence
b) Conducting pre-clinical animal studies
c) Obtaining a patent for the generic drug
d) Conducting Phase III clinical trials

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20. Generic drug manufacturers must prove their product has the same:
a) Active ingredient, dosage form, strength, and route of administration as
the innovator drug
b) Brand name and packaging as the innovator drug
c) Clinical trial results as the innovator drug
d) Higher effectiveness compared to the innovator drug

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