Postapproval Changes for Drugs: A Practical Guide
3
Introduction
Globally, the term “marketing application” (MA)
is applied to the collection of data, analyses, and
summary reports (“the dossier”) pharmaceutical
applicants submit to regulators to support a formal
request for commercial distribution of medicinal
products. MAs contain evidence intended to
demonstrate a medicinal product’s safety, effec-
tiveness, and quality and are subject to many
technical, discipline-specific, and administrative
regulatory requirements as a condition of approval.
Even after an MA is approved, there are reg-
ulatory requirements that continue to apply, e.g.,
when an MA is transferred from one applicant to
another annual updates and other administrative
changes in the elements that comprise the MA
or that impact the MA overall. While it may be
tempting to deprioritize or overlook compliance
with the regulatory requirements that govern
these changes, they serve an important function:
facilitating the continuous medicinal product sur-
veillance through which regulators support and
maintain public health. Further, in some cases,
noncompliance can be associated with significant
adverse consequences, including proposed with-
drawal of the MA or financial penalties.
This chapter reviews applicable US regula-
tory requirements for MA transfers and other
Chapter 1: United States
By Melodi McNeil, RPh, MS
administrative changes. The requirements that
govern administrative changes to drug master
files (DMFs) are also reviewed.
Key Terms
The US Food and Drug Administration (FDA)
recognizes three broad categories of MAs for
human drug/biologic products: the new drug
application (NDA), the biologics license appli-
cation (BLA), and the abbreviated new drug
application (ANDA).
The term NDA describes the dossier appli-
cants use to propose marketing of a new drug,
i.e., one not generally recognized, among experts
qualified by scientific training and experience
to evaluate the safety and effectiveness of drugs,
as safe and effective for use under the condition
prescribed, recommended, or suggested in the
labeling.1 The regulations having to do with
NDAs are found in 21 CFR Part 314. The appli-
cable statute is Section 505 of the Federal Food,
Drug, and Cosmetic Act (FD&C Act).
Similarly, a BLA is a request for permis-
sion to introduce, or deliver for introduction, a
biologic product into interstate commerce. The
BLA is regulated under 21 CFR Parts 600–680.2
Biologics are regulated under Section 262 of the
Public Health Service Act.
3
Introduction
Globally, the term “marketing application” (MA)
is applied to the collection of data, analyses, and
summary reports (“the dossier”) pharmaceutical
applicants submit to regulators to support a formal
request for commercial distribution of medicinal
products. MAs contain evidence intended to
demonstrate a medicinal product’s safety, effec-
tiveness, and quality and are subject to many
technical, discipline-specific, and administrative
regulatory requirements as a condition of approval.
Even after an MA is approved, there are reg-
ulatory requirements that continue to apply, e.g.,
when an MA is transferred from one applicant to
another annual updates and other administrative
changes in the elements that comprise the MA
or that impact the MA overall. While it may be
tempting to deprioritize or overlook compliance
with the regulatory requirements that govern
these changes, they serve an important function:
facilitating the continuous medicinal product sur-
veillance through which regulators support and
maintain public health. Further, in some cases,
noncompliance can be associated with significant
adverse consequences, including proposed with-
drawal of the MA or financial penalties.
This chapter reviews applicable US regula-
tory requirements for MA transfers and other
Chapter 1: United States
By Melodi McNeil, RPh, MS
administrative changes. The requirements that
govern administrative changes to drug master
files (DMFs) are also reviewed.
Key Terms
The US Food and Drug Administration (FDA)
recognizes three broad categories of MAs for
human drug/biologic products: the new drug
application (NDA), the biologics license appli-
cation (BLA), and the abbreviated new drug
application (ANDA).
The term NDA describes the dossier appli-
cants use to propose marketing of a new drug,
i.e., one not generally recognized, among experts
qualified by scientific training and experience
to evaluate the safety and effectiveness of drugs,
as safe and effective for use under the condition
prescribed, recommended, or suggested in the
labeling.1 The regulations having to do with
NDAs are found in 21 CFR Part 314. The appli-
cable statute is Section 505 of the Federal Food,
Drug, and Cosmetic Act (FD&C Act).
Similarly, a BLA is a request for permis-
sion to introduce, or deliver for introduction, a
biologic product into interstate commerce. The
BLA is regulated under 21 CFR Parts 600–680.2
Biologics are regulated under Section 262 of the
Public Health Service Act.