9 - New Drug Development Plan

WELCOME TO
Pharmaceutical Technology (Theory)

Lecture No: 09
Dr. Muhammad Asif

Faculty Member
Hajvery University (HU)
New Drug Development Plan
Introduction
•Development of new drug is very difficult, time consuming & very
expensive process.
•During last 50 years, hundreds of new drugs have been introduced,

& many older drugs have been withdrawn.
•< 1% of compounds that go into test eventually become licensed

medicines.
•To bring a new drug to market requires a good understanding of

drug development process & integral role preclinical testing plays.
New Drug
• New drug means new substance, which except during local
clinical trials, has not been used ever before in the country.
• Drug already approved for certain claims but which is now

proposed to be marketed with modified or new claims
(indications, dosage, dosage form, route of administration).
• Fixed-dose combination (FDC) of two or more drugs,

proposed to be combined for first time in a fixed ratio.
Regulatory Authorities
Authority to grant permission for New Drug to test & market

new drug in Pakistan rests with “Drugs Regulatory Authority of
Pakistan (DRAP)”, and is given as per DRAP Act 2012, Drug
Law 1976 and rules framed thereunder.
•US :- FDA (Food and Drug Administration)

•Europe :- EMEA (European Medicines Evaluation Agency)
•UK :- MHRA (Medicines and Healthcare products Regulatory
Agency)
•Japan :- MHLW(Ministry of Health, Labour and Welfare)
Common Ways of Developing New Drugs
• Random screening: It is a sort of blind hitting procedure where
new chemical entities (natural or synthetic) are subjected to
series of pharmacological screening procedures to explore
different types of biological activity.
• From natural products. e.g. Plants and animals

• Studying disease process.
• Using computers to design new drugs.
• Serendipity :- Observation by chance e.g. penicillin

Steps in New Drug Development
A. Idea or Basic Research

B. New drug discovery
C. Screening
D. Preclinical studies
E. Formulation development
F. IND application
G. Clinical studies
H. Official license / Regulations/Marketing
Basic Research
• Start by studying normal & abnormal body functions.
• Investigation of each component of the disease
(pathophysiology).
• Look up information obtained in previous research and
publication.
• Find out at which stage we can stop disease progression
or development.
• Search for targeted drug.
• Isolate the index compound.
• Perform animal testing to obtain safety data.
• Approval to test in humans.
Formulation Development
Pre-formulation:
• Before a drug can formulated, we must known about its physio-

chemical properties and behaviour (e.g. its solubility, its crystal
structure).
• These will dictate, the dosage forms in which medicine will be
marketed and can be used during drug development
investigations.
Drug characterizations
Pre-formulation studies will attempt to characterize the drug by

Spectroscopy, Solubility, melting point, Assay development,
Stability, Microscopy, Powder flow and compression properties,
Excipient compatibility.
Dosage form design
At some stage, a decision needs to be made about the dosage forms

for the delivery of the drug (e.g. tablet, capsule).
Types of dosage form
There are many different dosage forms and they all have their
relative advantages and disadvantages. (e.g. Tablets and capsule,
Injections and Infusion, Pessaries and suppositories, Solution,
Suspension, Elixirs, Ointment, Creams, Paints, Aerosols, Dry
powder inhalations, transdermal patches.)
Clinical Development Of Drug
• A clinical development plan is a comprehensive plan designed to
map out the development of a drug compound from early Phase-1
studies through marketing.
• The clinical development plan contains a summary of the pre-
clinical findings and of market research done for the drug.
Phases of clinical research

• Clinical research is done in four phases(1,2,3&4),each designed
to address different questions.
• Based upon data gathered from pre-clinical testing, the sponsor
has estimation of: the drug’s therapeutic effect and dose levels.
Phase I Clinical Trials
• The main goal of a Phase 1 trial is to discover if the drug is safe

in humans.
• This is a open study conducted in healthy human volunteers (20-

80).
• In special populations (e.g. for anti-cancer drugs).

Phase I Trial Address:
• How rapidly the drug is absorbed?

• Where is the drug distributed in the body?
• Which organ or organ system are involved in metabolism of
drug?
• How quickly is the drug eliminated from the body?
• In phase I studies regulatory bodies (i.e. FDA) can impose a
clinical hold (i.e. prohibit the study from proceeding or stop a
trial that has started) for reasons of safety ,or because of
sponsor’s failure to accurately disclose the risk of study to
investigators.
About 70% of experimental drugs passes Phase I clinical trials.

Phase II Clinical Trials
•Therapeutic exploratory trials.

•First trial in patients with the disease to be treated.
•50 - 300 patients are used for this study.
Objectives
•Effectiveness of the drug

•Common short term side effects and risks with I.N.D.
Phase II Trial address:
•What is the minimum effective dose?

•What is the maximum tolerated dose?
•Is the drug effective in mild, moderate, and severe cases
of the disease or condition?
•Is the drug effective for all expected indications?
•About 35% of experimental drug passes Phase II clinical

trial.
Phase III Clinical Trial
• Phase III studies are extended controlled and uncontrolled

trials.
• Phase III trial involved in patients sub-groups, such as

children, the elderly, and perhaps those with impairments in
liver and kidney.
• Once the phase III completed satisfactory, the drug company

to apply marketing application to the regulatory authorities to
market the drug.
• It carry outs on (300-3000 thousands) patients and lasts 1-

5years.
Clinical Trial Design
• An incredible amount of thought goes into the design of each
clinical trial.
• To provide the highest level of confidence in the validity of

results, many drug trials are :
 Placebo-controlled: Some subjects will receive the new drug

candidate and others will receive a placebo. (In some instances,
the drug candidate may be tested against another treatment
rather than a placebo.)
 Randomized: Each of the study subjects in the trial is assigned

randomly to one of the treatments.
• Double-blinded: Neither the researchers nor the subjects
know which treatment is being delivered until the study is
over.
• This method of testing provides the best evidence of any

direct relationship between the test compound and its effect
on disease because it minimizes human error.
• However, in many instances, alternative trial designs are

chosen based on ethical or other grounds.
• In most cases, two groups are considered: control and test.

However, these designs can be adapted to facilitate more
complex subgrouping.
New Drug Application (NDA) and Approval
•Once all three phases of the clinical trials are complete, the
sponsoring company analyzes all of the data.
•If the findings demonstrate that the experimental medicine is both

safe and effective, the company files a New Drug Application
(NDA) — which can run 100,000 pages or more — with the FDA
requesting approval to market the drug.
•The NDA includes all of the information from the previous years of
work, as well as the proposals for manufacturing and labeling of the
new medicine.
•FDA experts review all the information included in the NDA

to determine if demonstrates that the medicine is safe and
effective enough to be approved. By law, FDA is allowed six months
to review an NDA.
Following rigorous review, the FDA can either:
1) Approve the medicine,

2) Send the company an “approvable” letter requesting more
information or studies before approval can be given, or
3) Deny approval.
•Review of an NDA may include an evaluation by an advisory
committee, an independent panel of FDA-appointed experts who
consider data presented by company representatives and FDA
reviewers.
•Committees then vote on whether the FDA should approve an
application, and under what conditions.
•The FDA is not required to follow the recommendations of the
advisory committees, but often does.
Phase IV Clinical Trial(post Marketing Surveillance)
•Research on a new medicine continues even after approval.
•As a much larger number of patients begin to use the drug,

companies must continue to monitor it carefully and submit periodic
reports, including cases of adverse events, to the FDA.
•In addition, the FDA sometimes requires a company to conduct

additional studies on an approved drug in “Phase 4” studies.
•These trials can be set up to evaluate long-term safety or how the

new medicine affects a specific subgroup of patients.
Abbreviated New Drug Application (ANDA)
•ANDA is submitted to regulatory bodies to obtain the approval to

market a generic drug product.
•It contains data which when, provided for the review and once
approved, an applicant may manufacture and market the generic
drug product as a low cost alternative.
•Generic drug applications are termed “abbreviated” because they

are generally not required to include pre-clinical and clinical data to
established safety and effectiveness.
•Generic drug application reviewers focus on Bioequivalence data.

Summarization
Conclusion
• The drug discovery and development process is a long and
complicated process.
• Before any newly discovered drug is placed on the market, it must
undergo extensive testing.
• Each success is built on many, many prior failures.
• Advances in understanding human biology and disease are opening
up exciting new possibilities for breakthrough medicines.
• At the same time, researchers face great challenges in understanding
and applying these advances to the treatment of disease. These
possibilities will grow as our scientific knowledge expands and
becomes increasingly complex.
• Research-based pharmaceutical companies are committed to
advancing science and bringing new medicines to patients.

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WELCOME TO

Pharmaceutical Technology (Theory)

Dr. Muhammad Asif

•During last 50 years, hundreds of new drugs have been introduced,

•< 1% of compounds that go into test eventually become licensed

•To bring a new drug to market requires a good understanding of

• Drug already approved for certain claims but which is now

• Fixed-dose combination (FDC) of two or more drugs,

Authority to grant permission for New Drug to test & market

•US :- FDA (Food and Drug Administration)

• From natural products. e.g. Plants and animals

• Using computers to design new drugs.

• Serendipity :- Observation by chance e.g. penicillin

A. Idea or Basic Research

• Before a drug can formulated, we must known about its physio-

Pre-formulation studies will attempt to characterize the drug by

At some stage, a decision needs to be made about the dosage forms

Types of dosage form

Phases of clinical research

• The main goal of a Phase 1 trial is to discover if the drug is safe

• This is a open study conducted in healthy human volunteers (20-

• In special populations (e.g. for anti-cancer drugs).

• How rapidly the drug is absorbed?

About 70% of experimental drugs passes Phase I clinical trials.

•Therapeutic exploratory trials.

•Effectiveness of the drug

•What is the minimum effective dose?

•About 35% of experimental drug passes Phase II clinical

• Phase III studies are extended controlled and uncontrolled

• Phase III trial involved in patients sub-groups, such as

• Once the phase III completed satisfactory, the drug company

• It carry outs on (300-3000 thousands) patients and lasts 1-

• To provide the highest level of confidence in the validity of

 Placebo-controlled: Some subjects will receive the new drug

 Randomized: Each of the study subjects in the trial is assigned

• This method of testing provides the best evidence of any

• However, in many instances, alternative trial designs are

• In most cases, two groups are considered: control and test.

•If the findings demonstrate that the experimental medicine is both

•FDA experts review all the information included in the NDA

1) Approve the medicine,

•Research on a new medicine continues even after approval.

•As a much larger number of patients begin to use the drug,

•In addition, the FDA sometimes requires a company to conduct

•These trials can be set up to evaluate long-term safety or how the

•ANDA is submitted to regulatory bodies to obtain the approval to

•Generic drug applications are termed “abbreviated” because they

•Generic drug application reviewers focus on Bioequivalence data.

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