An NDA is the
Only "Form" of a Drug That Most Regulators
Will Ever See.
A Firm Should Look
at its NDA as
an Example of its
Finest Work.
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their respective owners
Keywords: Integrated summary of
effectiveness; Integrated summary of safety; Summary
of clinical efficacy; Summary of clinical safety;
Common Technical Document
Understanding the Differences and
Effectively Transitioning Between the US
Integrated Summaries of Effectiveness and
Safety (ISE/ISS) and the CTD Summaries of
Clinical Efficacy and Safety (SCE/SCS) by David N. Schwartz, BS
Michael J. Umen, PhD
Kathy Nomides
Mary Vanderhoof, MS
As printed in Drug Information Journal,
Vol. Vol. 44, pp. 641-648, 2010.
The adoption in 2000 of the Common
Technical Document (CTD) format for marketing
applications notwithstanding, the US regulations
requiring an integrated summary of effectiveness
(ISE) and an integrated summary of safety (ISS)
remain in effect. Many applicants, however, have
attempted to use the CTD module 2 clinical
summaries, specifically the summary of clinical
efficacy (SCE) and the summary of clinical safety
(SCS), alone to fulfill the regulatory requirements
for an ISE and ISS, arguing that it is redundant to
submit a separate ISE and ISS in addition to the SCE
and SCS. Consequently, the US FDA has issued
numerous guidances and made podium presentations
communicating the message that, except in rare
circumstances, New Drug Applications should contain
the ISE and ISS documents as well as the CTD
summaries of clinical efficacy and safety (SCE and
SCS). The core difference between the ISE/ISS and
their corresponding clinical summaries is in the
depth of the analyses and the amount of information
needed to support the analyses. While documenting
the larger integrated analyses of efficacy and
safety in the ISE and ISS, applicants should develop
a strategy and process for deriving the SCE and SCS.
Ultimately, submitting detailed and fully
comprehensive ISE and ISS documents not only enables
applicants to comply with regulations, but may also
facilitate quick and efficient preparation of the
SCE and the SCS.
Keywords: Clinical review
template; Clinical safety reviewer; FDA guidance;
Common technical document; Medical review officer;
Clinical overview; Clinical Summary
Preparing Regulatory
Submissions with the FDA Reviewer in Mind:
Use of the Clinical Review Template
by Mary Vanderhoof, MS
Michael J. Umen, PhD
David N. Schwartz, BS
As printed in Drug Information Journal,
Vol. 42, pp. 349-357, 2008.
Both the guidance documents issued
by FDA to industry and the guidance documents
intended to be used internally by FDA's own staff of
medical review officers (MROs) can be effective
tools to help sponsors prepare their regulatory
submissions. Two recent reviewer guidance documents,
the clinical review template and the corresponding
guidance, "Conducting a Clinical Safety Review of a
New Product Application and Preparing a Report on
the Review," provide useful insight into the FDA
review process. The review of the clinical data has
always been on the critical path to market approval
for new molecular entities, and the recent
reauthorization of the Prescription Drug User Fee
Act (PDUFA IV) has underscored both the importance
of an efficient review and postmarketing safety
monitoring. The more FDA and the sponsors harmonize
their efforts, with sponsors providing the summaries
and analyses (including comprehensive tables,
figures, and listings) that MROs need to adequately
assess the safety data and document their reviews,
the greater the opportunity for FDA to meet the
performance goals set out in PDUFA and for FDA and
industry together to deliver safe and effective new
therapies to US patients in a timely fashion.
Key
Words: New Drug Applications; Principles; Labeling
Six Enduring Principals
for Preparing NDAs by Michael J. Umen, Ph.D.
As printed in Drug Information Journal,
Vol. 34, pp. 703-707,2000.
Six enduring principles for
preparing New Drug Applications (NDAs) are advanced:
Principle Number 1: An NDA is the document that
supports the labeling of a drug, Principle Number 2:
An NDA is the only "form" of a drug that most
regulators will ever see. A firm should look at its
NDA as an example of its finest work, Principle
Number 3: NDAs are organized to facilitate review by
the authorities, Principle Number 4: Group studies
in a logical order, Principle Number 5:
Standardization of format and consistency of content
make for high quality applications, and Principle
Number 6: Getting it submitted on time is important;
but getting it right is more important if your goal
is an approved application.
Key
Words: New Drug Applications; pharmaceutical
executives;
principles.
COPYRIGHT NOTICE:
Reprinted with permission from Pharmaceutical
Executive, February 1984. Pharmaceutical Executive
is a copyrighted publication of Advanstar
Communications Inc. All rights reserved.
Winning Plays in the R&D Game by Robert W. Keidel Ph.D. and Michael J. Umen,
Ph.D. As printed in the Pharmaceutical Executive,
February 1984.
Are my medicinal chemists playing
baseball but being managed like a football team?
Are my new-drug application (NDA) teams playing
baseball instead of basketball? These may
appear to be strange questions for pharmaceutical
executives to ask themselves? However, such
questions can be relevant and may provide powerful
insights to help managers score points in the
industry. Omit the sports terms, and they
become questions pharmaceutical executives
frequently ask themselves....