Transcript Slide 1

FDA Regulatory Considerations
in Launching Products
WITI
(Women In Technology International)
San Diego Conference
February 14, 2005
Michael A. Swit, Esq.
Vice President, Life Sciences
What Data – Quantity & Quality –
Will FDA Require?
• Will vary -- FDA has extensive discretion
here
• Key task -- try to get clarity as soon as
possible in the process -- Ways to do so:
– Pre-IND meeting -- encouraged by FDA prior to start of
human clinicals
– End of Phase 2 Meeting - CRITICAL -- here's where
you want to "lock" them in
What Data – Quantity & Quality
– Will FDA Require?
• FDAMA § 119(a) --
– FDA must meet with you on design of studies; and
– Any agreement on study design must be written and
can't be changed later w/o your consent unless a
new safety or effectiveness issue arises later
– “Special Protocol Assessments” – FDA process for
implementing
• FDAMA § 115(a) -- data from one adequate and wellcontrolled study and confirmatory evidence can be used
to show substantial evidence of effectiveness
What Data – Quantity &
Quality – Will FDA Require? …
• "Pure" proof of clinical effectiveness may
not be needed -- e.g., under “Fast Track,”
may be able to use:
– Surrogate endpoints
– Clinical endpoints
– Phase IV study will be needed usually
The FDA Review -- Priority
and Speed
• "Fast Track" -- FDAMA § 112
– treats a "serious or life threatening condition"
– shows "potential to address unmet medical needs for
such condition"
– If so, FDA must "facilitate the development and
expedite and review" of the drug
– Request at time of or after IND filing
– Allows for “rolling” submissions
See 1998 Guidance on Fast Track
http://www.fda.gov/cder/guidance/2112fnl.pdf
The FDA Review -- Priority and
Speed
• Accelerated Approval
– Similar to Fast Track (predates it), but does always get priority review
– For promising products for life threatening diseases, using preliminary
results -- thus, surrogate markers possible
– May lead to provisional approval and a requirement to do post-approval
studies – aka “Phase IV”
– Under criticism due to Vioxx
• Priority Review
– Results from FDA’s common approach to all NDAs
• P - Priority -- significant advances over existing treatments.
• S - Standard -- drugs similar to currently available drugs
– Priority – gets 6-month review clock
– Standard – gets 10-month review clock
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Pre-Approval – The Home Stretch
• Pre-Approval Inspection
– Be prepared
– For established firms, may not be needed if meet certain
criteria
– For new firms – key to final approval
• Advisory Committee Meetings
– Final resolution, often, of key issues
– Highly public process – if you get backing, chances of
approval are high (but not a lock)
– Need to know how to prepare for
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Pre-Approval – The Home Stretch
• Press Releases and Other Publicity/Advertising
– FDA will insist you submit for “prior” review
– Must be vetted for compliance with approved labeling –
Regulatory plays key role
– Need interdisciplinary team with all parts of company – not
just Sales & Marketing
• Regulatory
• Investor Relations
• see next slide for other reasons why
• Promotional Offers – tap into your health law
attorneys – landscape is very complex
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Tips – Some Hard Lessons
• Make sure R&D and Sales & Marketing are talking
early on –
– ensure the indication being studied is one you want to sell
– Product name –
•
•
must jibe with approval – e.g., Regaine (no good) vs. Rogaine
Not be confusing with another approved drug
• Know who owns your data – especially when costs are
shared.
• Don’t assume you get patent extensions or HatchWaxman Exclusivity – validate in advance
Tips – Some Hard Lessons
• Understand, that an approval is not enough – you
need to get Medicare &/or private payer
reimbursement
– Start the reimbursement qualification process early
– Design clinical protocols to address payer
expectations
• Private & Government
• Example -- study your drug in Medicare-age patients
• Don’t wait until Phase III to tackle tough issues
– e.g., -- client completed Phase III, but has never fully
characterized molecule
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Tips – Some Hard Lessons
• If outsourcing, audit aggressively your "vendors:”
– CROs, clinical investigators, contract manufacturers, API makers
– IRBs – they have been shut down in past
– Joint venture partners – e.g., Cialis® – Lilly manufacturing plant
problems – delayed about one year
– Remember – even when you outsource, you are still ultimately
responsible for what happens and you still need to have systems
and people in place to ensure your vendors are working correctly
• Don't bury your head to problems -- investigate and
disclose promptly
Questions?
Call, e-mail, fax or write:
Michael A. Swit, Esq.
Vice President, Life Sciences
THE WEINBERG GROUP INC.
336 North Coast Hwy. 101
Suite C
Encinitas, CA 92024
Phone 760.633.3343
Fax 760.633.3501
Cell 760.815.4762
D.C. Office 202.730.4123
[email protected]
www.weinberggroup.com
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THE WEINBERG GROUP when their products are at risk.
Our technical, scientific and regulatory experts deliver the crucial
results that get products to market and keep them there.
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