A Pre-Marketing Activities

OUR LADY OF FATIMA UNIVERSITY
College of Pharmacy
FDA DRUG REVIEW AND

APPROVAL
Unit Outcomes
To demonstrate understanding of FDA drug
approval process, more specifically:
• Discuss the important process of the FDA
review process
• Discuss the strategies for premarketing
activities
DISCOVERY AND DEVELOPMENT
PRECLINICAL RESEARCH
CLINICAL RESEARCH
FDA REVIEW
FDA POST MARKET SAFETY
MONITORING
FDA REVIEW
➢ If a drug developer has evidence from its early tests and
preclinical and clinical research that a drug is safe and
effective for its intended use, the company can file an
application to market the drug.
NEW DRUG APPLICATION (NDA)
➢ After clinical trials have succeeded, a New Drug
Application (NDA) is submitted to the FDA for review
and potential approval.
➢ A drug developer must include everything about a drug—
from preclinical data to Phase 3 trial data in an NDA.
➢ Along with clinical results, developers must include:
❑ Proposed labeling
❑ Safety updates
❑ Drug abuse information
❑ Patent information
❑ Institutional review board compliance information
❑ Directions for use
NDA CONTENTS:
➢ Introduction – brief description of the drug and the
therapeutic class to which it belongs
➢ Chemical and Pharmaceutical information
➢ Animal pharmacology
➢ Animal Toxicology
➢ Human/Clinical Pharmacology Phase I
➢ Therapeutic Exploratory Trials (Phase II)
NDA CONTENTS:
➢ Therapeutic Confirmatory Trials (Phase III)
➢ Special Studies (Geriatrics, Pediatrics, Pregnant or
nursing women)
➢ Regulatory Status in other countries
➢ Prescribing information
➢ Samples and Testing Protocol/s
NDA SECTIONS:
➢ SECTION 1 : INDEX
➢ SECTION 2 : LABELING
➢ SECTION 3 : APPLICATION SUMMARY
➢ SECTION 4 : CHEMISTRY, MANUFACTURING, AND CONTROLS
(CMC)
➢ SECTION 5 : NONCLINICAL PHARMACOLOGY AND
TOXICOLOGY
➢ SECTION 6 : HUMAN PHARMACOKINETICS AND
BIOVAILABILITY
NDA SECTIONS:
➢ SECTION 7 : CLINICAL MICROBIOLOGY
➢ SECTION 8 : CLINICAL DATA
➢ SECTION 9 : SAFETY UPDATE REPORTS
➢ SECTION 10 : STATISTICS
➢ SECTION 11 : CASE REPORT TABULATIONS
➢ SECTION 12 : CASE REPORT FORMS (CRF)
➢ SECTION 13 : PATIENT INFORMATION
➢ SECTION 14 : PATENT CERTIFICATION
➢ SECTION 15 : ESTABLISHMENT DESCRIPTION
NDA SECTIONS:
➢ SECTION 16 : DEBARMENT CERTIFICATE
➢ SECTION 17 : FIELD COPY CERTIFICATION
➢ SECTION 18 : USER FEE COVERSHEET
➢ SECTION 19 : FINANCIAL DISCLOSURE
➢ SECTION 20 : OTHERS
GOALS:
• The drug is safe and effective as a treatment for the condition it
has been developed for.
• The drug’s therapeutic benefits outweigh the risks.
• The drug’s labeling is fit-for-purpose and whether all required
details are included.
• The methods used to manufacture the drug and measures to
ensure the drug's quality are satisfactory.
FDA REVIEW
➢ Once FDA receives an NDA, the review team decides if
it is complete.
➢ If it is not complete, the review team can refuse to file
the NDA.
➢ If it is complete, the review team has 6 to 10 months
to make a decision on whether to approve the drug.
FDA REVIEW
The process includes the following:
❑ Each member of the review team conducts a full review
of his or her section of the application.
✓ For example, the medical officer and the statistician
review clinical data, while a pharmacologist reviews the
data from animal studies. Within each technical discipline
represented on the team, there is also a supervisory
review.
FDA REVIEW
❑ FDA inspectors travel to clinical study sites to
conduct a routine inspection.
❑ The Agency looks for evidence of fabrication,
manipulation, or withholding of data.
FDA REVIEW
❑ The project manager assembles all individual reviews and
other documents, such as the inspection report, into an
“action package.”
❑ This document becomes the record for FDA review. The
review team issues a recommendation, and a senior FDA
official makes a decision.
FDA APPROVAL
▪ In cases where FDA determines that a drug has been shown
to be safe and effective for its intended use, it is then
necessary to work with the applicant to develop and refine
prescribing information.
▪ This is referred to as “labeling”.
▪ Labeling accurately and objectively describes the basis for
approval and how best to use the drug.
FDA APPROVAL
▪ Often, though, remaining issues need to be resolved before
the drug can be approved for marketing.
▪ Sometimes FDA requires the developer to address questions
based on existing data. In other cases, FDA requires additional
studies.
▪ At this point, the developer can decide whether or not to
continue further development. If a developer disagrees with
an FDA decision, there are mechanisms for formal
appeal.
REASONS FOR DRUG APPLICATION FAILURE
New drug applications may fail for a variety of
reasons.
TOXICITY: If the toxicity of a new drug is too high in human
or animal patients, the drug may be rejected due to safety
concerns about its use following manufacture.
EFFICACY: If a new drug’s efficacy is not high enough or

evidence is inconclusive, the FDA may reject it.
Pharmacokinetic Properties or Bioavailability : PK
properties or poor bioavailability due to low aqueous
solubility, or high first-pass metabolism, may also cause a
drug to fail FDA review. PK causes of drug failure include
inadequate action duration and unanticipated human drug
interactions.
Inadequate Drug Performance: If the new drug performs
the desired function, but only at a shallow level, the FDA
may reject the application in favor of a formulation that
performs better.
PRODUCT LAUNCH
Once the drug receives approval from the relevant regulatory
authority, numerous activities will need to be initiated to prepare
for the launch of the product.
These include:
➢ Manufacturing scale-up and serialization
➢ Printing of final product label information, packaging and
artwork
➢ Product storage, shipping and distribution arrangements
➢ Production staff and quality team availability
Biologics License Applications (BLA)
Process (CBER)
➢ The Biologics License Application (BLA) is a request
for permission to introduce, or deliver for
introduction, a biologic product into interstate
commerce (21 CFR 601.2).
Biologics License Application (BLA) Process (CBER)
➢ A BLA is submitted by any legal person or entity who is engaged in
manufacture or an applicant for a license who takes responsibility
for compliance with product and establishment standards.
➢ Form 356h specifies the requirements for a BLA.
This includes:
▪ Applicant information
▪ Product/Manufacturing information
▪ Pre-clinical studies
▪ Clinical studies
▪ Labeling
ORPHAN DRUGS AND BIOLOGICAL PRODUCTS
➢ Orphan drugs/biological products are registered following
a facilitated registration process and may be accessed by patients
using a Compassionate Special Permit (CSP).
➢ A CSP is a special permit signed by the FDA Director granting a
Specialized Institution (SI) and Specialty Society (SS) the privilege
to avail of an unregistered drug and device product through a
certain licensed establishment for certain kind/type of patients,
specific volume and period.
➢ Patients having the following conditions are allowed to use

CSP:
1) Acquired Immune Deficiency Syndrome (AIDS)
2) Cancer
3) Life-threatening conditions
➢ The requirements for securing a CSP are the following:
1) Letter of Application
2) Name and contact details of Specialist of the patient
3) Prescription
4) Medical Abstract of Patient
✓ A maximum of 15 calendar days is given by the FDA in the
processing of CSP.
References:
• Allen, L. V., Popovich, N. G., Ansel, H. C., & Ansel, H. C. (2011). Ansel's
pharmaceutical dosage forms and drug delivery systems. Philadelphia:
Lippincott Williams & Wilkins.
• Brody. T. (2020). Clinical Trials : Study Design, Endpoints and Biomarkers,
Drug Safety, and FDA and ICH Guidelines. Elsevier Inc. (Available in
ebscohost.com)

A Pre-Marketing Activities

Uploaded by

Copyright:

Available Formats

A Pre-Marketing Activities

Uploaded by

Document Information

Copyright

Available Formats

Share this document

Share or Embed Document

Sharing Options

Did you find this document useful?

Is this content inappropriate?

Copyright:

Available Formats

A Pre-Marketing Activities

Uploaded by

Copyright:

Available Formats

OUR LADY OF FATIMA UNIVERSITY

FDA DRUG REVIEW AND

EFFICACY: If a new drug’s efficacy is not high enough or

➢ Patients having the following conditions are allowed to use

You might also like