FDA Regulatory Considerations in Launching Products Michael A. Swit, Esq. Vice President, Life Sciences WITI (Women In Technology International) San Diego Conference February 14, 2005

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 well- controlled 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

6 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

7 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

8 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

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 Hatch- Waxman Exclusivity – validate in advance

10 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

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

12 Call, , fax or write: Michael A. Swit, Esq. Vice President, Life Sciences THE WEINBERG GROUP INC. 336 North Coast Hwy. 101 Suite C Encinitas, CA Phone Fax Cell D.C. Office Questions?

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