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Literature based submission… the options and lessonsRegulatory requirements
Dr Michael Coory
Director, Prescription Medicines Clinical Unit 5
Medicines Authorisation Branch
Market Authorisation Division, TGA
ARCS Scientific Congress 2015
6 May 2015
What is a literature based submission (LBS)?
• Relies on published (non-sponsor) studies to satisfactorily establish
efficacy and safety
• As opposed to conventional submissions, which rely on company-
sponsored studies to satisfactorily establish efficacy and safety
LBS… the options and lessons
Types of submissions
• Conventional
– Full study reports of company-sponsored studies
• Literature based
– Published studies
• Mixed
– Combination of above
LBS… the options and lessons
When is it acceptable to submit a literature based
submission?Case-by-case, depending on the regulatory history in Australia and overseas
*This table is a guide only
Years medicine
marketed
overseas
Type of application
Conventional Mixed Literature
based
< 5 years Yes No No
5-10 years Yes Yes No
10+ years Yes Yes Yes
LBS… the options and lessons
Acceptable overseas countries
• United States
• United Kingdom
• Netherlands
• Sweden
• Canada
• New Zealand
LBS… the options and lessons
Most common types of literature based
submissions
• New product
– but active ingredient (moiety) already registered via a different product
LBS… the options and lessons
• Major variation
– change in posology/dosing instructions
– change in route of administration
– change in strength
• Extension of indications
• New chemical entity (uncommon, but possible)
LBS… the options and lessons
Number of literature-based submissions
Financial year Number
2010–2011 16
2011–2012 16
2012–2013 20
2013–2014 11
2014–Apr 2015
(10 months)
20
LBS… the options and lessons
When is it not acceptable to submit a literature
based submission?
• Module 3
– need a conventional Module 3 for all applications with a quality
component
• Changes to the clinical trials section of the PI
LBS… the options and lessons
Pre-submission
• Contact case manager about intention to submit LBS
• Discussion could be either by email or teleconference or face-to-face
• Need to get confirmation that
– LBS is acceptable
– search strategy is acceptable
LBS… the options and lessons
Why do we need a systematic search of the
literature?
• Cherry pick particular studies
• Narrative versus systematic review
LBS… the options and lessons
Even with a systematic search of literature,
still the potential problem of publication bias
• The research that is published in medical journals is not representative of
all studies that have been done.
• ‘File-drawer’ problem
LBS… the options and lessons
Occasional problems with literature-based
submissions
• Too many studies
– especially for ‘old’ active ingredients
– review of systematic reviews and clinical practice guidelines
– d/w delegate
• No reasoning about efficacy and safety
– dump of studies
• Not enough emphasis on post-marketing experience overseas
LBS… the options and lessons