www.biopharminternational.com September 2019 BioPharm International eBook 9
Regulatory Sourcebook Regulatory Guidance Update
under medical device application
types or to labeling intended for use
by healthcare providers. It also does
not apply to devices regulated under
a BLA, such as devices associated
with blood collection and processing
procedures.
PHARMACOPEIA UPDATES
FDA released draft guidance on July
10, 2019 that assists applicants in
initiating revisions to an existing
monograph or the development of
a new monograph under the United
States Pharmacopeial Convention–
Pending Monography Process (USP–
PMP) during FDA's evaluation of a
drug substance master file or drug
product application (7). Applicants
of drug approvals must comply with
USP National Formulary (NF) stan-
dards, if applicable. The guidance
assists these applicants in using the
USP–PMP to request a revision of
compendial standards.
In the past, if proposed specifica-
tions in a drug application appeared
to not comply with the current USP
monograph, FDA approval of the
application would be delayed. Until
the USP–PMP was established, there
was not a process for drug develop-
ers to request changes to compen-
dial standards for products that were
not yet approved by FDA. This draft
guidance describes the process for
the "revision of compendial stan-
dards that are harmonized with
the approved quality and labeling
requirements for a drug product
application," including how to initi-
ate the USP–PMP process.
QUALITY REGULATIONS
FDA made M10 Bioanalytical Method
Validation available in June 2019
(8). The draft guidance, developed
by the International Council for
Harmonization (ICH), is intended
to provide recommendations for the
validation of bioanalytical assays for
chemical and biological drug quan-
tification and their application in the
analysis of study samples. The guid-
ance describes the various elements
and expectations to validate specific
tests used to measure the parent and
active metabolites of drugs admin-
istered in nonclinical and clini-
cal studies submitted in regulatory
applications for biological matrices
such as plasma, blood, or serum.
BIOSIMILARS
On May 21, 2019, FDA published
guidance on the design and eval-
uation of comparative analytical
studies used to support the bio-
similarity of a proposed therapeu-
tic protein product to a reference
product licensed under section
351(a) of the Public Health Service
Act (PHS Act) (9). The guidance
also offers recommendations on
the scientific and technical infor-
mation for the chemistry, manu-
fac t ur ing, and controls (CMC)
portion of a marketing applica-
tion for a proposed product sub-
mitted under section 351(k) of the
PHS Act. Among an overview of
the PHS Act and the Biologics Price
Competition and Innovation Act
of 2009 (BPCI Act), the guidance
specifically discusses expression
systems, manufacturing processes,
physicochemical properties, func-
tional activities, target binding,
impurities, reference products and
standards, f inished dr ug prod-
ucts, and stability. Considerations
addressed for a comparative ana-
lytical assessment include refer-
ence and biosimilar products and
data analysis.
T he g u id a nc e i s p a r t o f a
ser ies of doc u ments to fac i l i-
t a t e t h e i m p l e m e n t a t i o n o f
t he BPCI Ac t. Ot her g u ida nce
d o c u me nt s a d d r e s s s c ie nt i f ic
considerations, biosimilar devel-
opment, clinical pharmacolog y
data, labeling of biosimilars, and
demonstrating interchangeability.
REFERENCES
1. FDA, FDA Patient-Focused Drug
Development Guidance Series for
Enhancing the Incorporation of the
Patient's Voice in Medical Product
Development and Regulatory Decision
Making, FDA.gov, www.fda.gov/drugs/
development-approval-process-drugs/
fda-patient-focused-drug-development-
guidance-series-enhancing-incorporation-
patients-voice-medical
2. FDA, Rare Pediatric Disease Priority
Review Vouchers Guidance for Industry,
Draft Guidance (CDER, July 2019), www.
fda.gov/media/90014/download
3. FDA, Providing Regulatory Submissions
in Electronic Format—Certain Human
Pharmaceutical Product Applications and
Related Submissions Using the eCTD
Specifications Guidance for Industry, Draft
Guidance (CDER, CBER, July 2019), www.
fda.gov/regulatory-information/search-
fda-guidance-documents/providing-
regulatory-submissions-electronic-format-
certain-human-pharmaceutical-product-
applications-0
4. FDA, Population Pharmacokinetics,
Guidance for Industry, Draft Guidance
(CDER, CBER, July 2019), www.fda.
gov/regulatory-information/search-
fda-guidance-documents/population-
pharmacokinetics
5. FDA, Risk Evaluation and Mitigation
Strategies: Modifications and Revisions
Guidance for Industry (CDER, CBER,
July 2019), www.fda.gov/regulatory-
information/search-fda-guidance-
documents/risk-evaluation-and-
mitigation-strategies-modifications-and-
revisions-guidance-industry-0
6. FDA, Instructions for Use—Patient
Labeling for Human Prescription Drug
and Biological Products and Drug-
Device and Biologic-Device Combination
Products—Content and Format Guidance
for Industry (CDER, CBER, July 2019),
www.fda.gov/regulatory-information/
search-fda-guidance-documents/
instructions-use-patient-labeling-
human-prescription-drug-and-biological-
products-and-drug-device
7. FDA, Harmonizing Compendial Standards
With Drug Application Approval Using
the USP Pending Monograph Process
Guidance for Industry, Draft Guidance
(CDER, CVM, July 2019), www.fda.gov/
media/128689/download
8. FDA, M10 Bioanalytical Method Validation
(June 2019), www.fda.gov/regulatory-
information/search-fda-guidance-
documents/m10-bioanalytical-method-
validation
9. FDA, Development of Therapeutic Protein
Biosimilars: Comparative Analytical
Assessment and Other Quality-Related
Considerations Guidance for Industry
(CDER, CBER, May 2019), www.fda.
gov/regulatory-information/search-fda-
guidance-documents/development-
therapeutic-protein-biosimilars-
comparative-analytical-assessment-and-
other-quality
BP