The document discusses the purpose and requirements of an Investigational New Drug (IND) application submitted to the FDA. An IND allows a company to ship an experimental drug and conduct clinical trials in humans. It provides safety data and details of planned trials for FDA review to ensure risks are minimized for subjects. A complete IND includes chemistry, manufacturing, pharmacology, protocols and other application materials for FDA evaluation before trials may begin.

1. IND enabling studies
Presented By : Dolly Chauhan
M.Pharm (Pharmacology)
2nd Semester

2.  An IND is a submission to the food and drug administration
(FDA) requesting permission to initiate a clinical study of a
new drug product.
 The Federal Food ,Drug and Cosmetic act requires that
drugs have an approved marketing application before they
can be shipped in interstate commerce

3. The IND application allows a company to initiate and
conduct clinical studies for their new drug products.
The IND application provides the FDA with the data
necessary to decide whether the new drug and the
proposed clinical trial pose a reasonable
risk to the human subjects participating in
the study.

4.  FDA's role in the development of a new drug begins when the drug's
sponsor has screened the new molecule for pharmacological activity
and acute toxicity potential in animals, wants to test its diagnostic or
therapeutic potential in humans
 The molecule changes in legal status under the Federal Food,
Drug, and Cosmetic Act and
 becomes a new drug subject to specific requirements of the drug
regulatory system
 Drug is to be the subjected to an approved marketing application
before it is transported or distributed across state lines
 IND- notice of claimed investigational exemption for a new drug
must be filed with regulatory body.

5.  Submission application to FDA requesting
permission to initiate the study of Newdrug
product.
 Pharmaceutical company obtains permission to
clinical trial before a marketing application for
the drug has been approved

6.  FDA reviews the IND application for safety
to assure that research subjects will not be
subjected to unreasonable risk
 Product is reasonably safe for initial use in
humans
 If the compound exhibits pharmacological
activity that justifies commercial development

7. Pharmaceuticals may move across state lines
during two stages of human use
• Research prior to “approval”
– Requires research permit: e.g., Investigational
New Drug Exemption (IND)
• Marketing after “approval”
– Requires marketing permit: e.g., New Drug
Application (NDA)

8. Applicant (Drug Sponsor):
 who assumes responsibility for the marketing
of a new drug
 IND application provides FDA with data
necessary to decide :
New drug
proposed Clinical trial pose a reasonable risk
to human subjects participating in the study

9. The IND application allows the company to
initiate & conduct the clinical studies of their
new drug Product.
• The safety of the Clinical trial Subjects is
always the primary concern of FDA.

10. Primary goal of the Sponsor should be to
demonstrate to the FDA that the
- new drug
- proposed trial
- entire clinical development plan described in
the IND is designed to minimize the risk to
the trial subjects.

11. New chemical entity not approved for
the indication
Being administered at the new dosage
level
combination with anther drug
All clinical studies where the new drug
is administered to human subjects

12. Non clinical studies
Approved drug and the study is within its
approved indication for use

13. COMMERCIAL IND : goal is to obtain
marketing approval for a new product.
NON-COMMERCIAL IND : It includes
 INVESTIGATOR IND :
In this case ,the physician is both the sponsor and
investigator

14. Investigator IND
o Submitted by a physician who both initiates and conducts an investigation, and
under
 whose immediate direction the investigational drug is administered or dispensed.
o Physician might submit a research IND to propose studying an unapproved drug,
or an approved product for a new indication or in a new patient population
Emergency Use IND
o Allows FDA to authorize use of an experimental drug in an emergency situation
o Does not allow time for submission of an IND in accordance with 21CFR ,
PART 312 (INDA)
Treatment IND
o Submitted for experimental drugs showing promise in clinical testing for serious
or immediately life-threatening conditions while the final clinical work is
conducted and the FDA review takes place

15.  EMERGENCY IND : FDA authorize immediate
dispensing of a non-approved drug in a life
threatening situation when no standard
acceptable therapy is available.
TREATMENT IND : FDA will permit
investigational drug to be used to treat a
serious or life threatening disease or if there
is no comparable alternative drug available.

18. 312.20 REQUIREMENTS OF AN IND
• A sponsor shall submit an IND to FDA who
intends to conduct a clinical investigation.
• Investigation is not supposed to begin
without prior written authorization of FDA

19. • Phase 1 - ADME (20- 80) healthy subjects
• Phase 2 – effectiveness in particular
indication (several hundred patients)
• Phase 3 – safety and effectiveness ( 100-
1000) subjects.

20. To assure safety and rights of the subject.
• To assure the scientific quality of
investigation will yield data capable of
meeting statutory standards for marketing
approval
• Focus should be on general investigational
plan & protocol which should be supported
by additional information including animal
toxicological studies

21. 1. Cover sheet (FORM FDA 1571)
2. Table of contents
3. Introductory statement and a general investigational
plan
4. Investigators brochure
5. Protocols
6. Chemistry , manufacturing and control information
7. Pharmacology and Toxicology information
8.Previous human experience with the investigational
drug
9.Other relevant information like no of IND
submissions
10. Protocol amendments, any changes in the protocol

22.  Name of the sponsor
 Date of submission
 Address
 Telephone number
 Name(s) of Drug
 IND Number
 Indication
 Phase of clinical investigation to be conducted
list no.of all investigational new drug application
 Serial no.
 Contents of application

23. Description of clinical studies planned for the
experimental drug
Purpose of the study
Indication to be studied
Types of trials to be initiated
Number of study subjects
Risks involved

24. Structural formula of drug
Summary of pharmacological , toxicological ,
pharmacokinetic effects in animals
Safety and efficacy
Purpose of study
Dose / dosefrequency
Monitoring procedures

25. - It describes – the objective of the
study
- the trial design
- how subjects would be selected
- how the trail is to be conducted.

26. Determines the adequacy of methods used to
manufacture and assay investigational
compound
Safety concerns
Describe drug substances
Method of preparation
 Reagent and solvents
 Acceptable limits and analytical methods to
ensure quality and purity of drug.

27. non-clinical safety data that sponsor
generated to prove that the IPis safe for
clinical study .
the amount & type of data depends on class
of new drug duration of proposed clinical
trial, patient population that will be exposed
during the trial

28. Pk studies
Pd studies
observed adverse event profile

29. purpose of this section is to facilitate the
availability of promising new drugs to
desperately ill patients
In the case of an immediately life-threatening
disease, a drug may be made available for
treatment use under this section earlier than
Phase 3, but ordinarily not earlier than Phase
2.

30. The “treatment use” of a drug includes the
use of a drug for diagnostic purposes.
If a protocol for an investigational drug meets
the criteria of this section, the protocol is to
be submitted as a treatment protocol

31. A treatment protocol is required to contain the
following:
• The intended use of the drug.
• An explanation of the rationale for use of the
drug,
• A brief description of the criteria for patient
selection, The method of administration of
the drug and the dosages.
• A description of clinical procedures,
laboratory tests, or other measures.

32. Once the IND is stamped as received , it is
sent to CDER for review.
• It is further categorically divided into
different sections –
 Medical
 Chemistry
 Pharmacology / Toxicology
 Statistics

33.  Safety Review
 Clinical Hold Decision
 Notify Sponsor
 Sponsor Notified of Deficiencies
 Study Ongoing
• Once CDER's 30-day initial review period
expires, clinical studies can be initiated,
unless a clinical hold has been placed.

34. 1.Helps in the result of successful preclinical
development program.
2.IND is also the vehicle through which a
sponsor advice the next stage of drug
development i.e. clinical trial.
3.The preclinical study, helps the sponsor’s
primary goal to determine that the product is
reasonably safe for initial use in human.
4.It is important in the
development of compounds
commercial
if it exhibit
pharmacological activity.
5.It is important in assuring the marketing of a
new drug and responsibility for compliance of
sponsor.

35. 6.It is important for the company to initiate
and conduct the clinical studies of their new
product.
7.It secure the safety and effectiveness of the
clinical trial subjects.
8.IND can be alternative in a life threatening
situation when no standard acceptable
therapy is available.
9.It gives the indication of new drugs.
10.Pharmacology and toxicology information
of new drugs.