FDA Regulatory Strategies

1
FDA Regulatory Strategies

• Larry Kleinman, Paul Jackson, Kari Nadeau,
  Michelle Rohrer
• Wednesday, March 5, 2002

2
Mission

• BioE2E brings together an active community of
  life-science entrepreneurs by creating a lively
  and useful forum for the exchange of practical
  real-world entrepreneurial experiences.
• Our programs foster education, collaboration,
  idea exchange, networking, and professional
  development among community members.

3
BioE2E Panel Events

• First Wednesday of each month
• 7pm wine food, 730pm workshop845pm
  networking
• Wilson Sonsini Goodrich Rosati (here)
• Cost, 20 cash, at the door (dont forget)
• Check e-mail, get on the list _at_ bioe2e.org
• BioE2E is affiliated with the MIT Club of
  Northern California Entrepreneurship Program
  (http//www.mitcnc.org/www/Entrepreneurship.htm)

4
Events

• Alternative Funding Fair (February 28, full
  day)_at_ SRI International in Menlo Park
• FDA Regulatory Strategies(March 5 today)
• Business Models What They Are, and What's
  Getting Funded Now (April 2, Wednesday)

5
Todays speakers

• Larry Kleinman, MS
• Founder and VP of Development, Pacific
  BioDevelopment firm offering strategic drug
  development consulting
• Senior Director of Regulatory Affairs, Genentech
• US Public Health Service (retired)
• FDA
• National Cancer Institute

• Paul Jackson, MD, PhD
• Chief Medical Officer, Endovasix
• Product design
• Design and monitoring of clinical trials
• Residency in neurosurgery at Brigham and Womens
  Hospital
• PhD in neurobiology and MD from Harvard Medical
  School

6
Todays speakers (2)

• Kari Nadeau, MD, PhD
• VP, Chief Scientific and Medical Officer,
  Clearview Projects
• Medical director of global clinical trials at
  Biogen, Coulter Pharmaceuticals and Roche.
• 22 INDs, 2 BLAs and 1 FDA meeting
• Residency in oncology and hematology at Dana
  Farber Cancer Institute
• PhD in Biochemistry and MD from Harvard Medical
  School

• Michelle Rohrer, PhD
• 10 years of experience in the biotechnology
  industry
• Associate Director of Regulatory Affairs,
  Genentech
• Clinical Scientist and International Clinical
  Team Leader in biologics for AIDS wasting,
  diabetes, neuropathy
• Managed and monitored clinical trials across all
  phases and across many indications including
  psoriasis, age-related macular degeneration, RA,
  diabetic wounds and acute coronary syndrome

7
Defining the drug/device label

• Target labels drive -
• Clinical Development Plan
• Product claims (delivery, safety, efficacy,
  dosing)
• Criteria for termination of project
• Commercial/marketing strategy

Target Label
Define Hypothesis

• Target indication(s)
• Patient population (e.g. adults, children,
  neonates)
• Treatment/prophylaxis
• Superiority over standards of care
• Method of delivery
• Definition of appropriate endpoints

8
Creative Strategies in Devices 510(K) vs PMA?

• 510(K)
• Concept of equivalence
• May 28, 1976 Medical Devices Amendments to the
  FDA
• Pros
• Speed
• Lower risk
• Cons
• Low barriers to entry
• 510(K) with clinical trials
• PMA Pre Market Approval
• Clinical trials for safety and efficacy of device
• Pros barriers to entry
• Cons time, expense and risk

9
Key Considerations in Crafting a Clinical
Development Plan?

• Key parameters in developing a clinical
  development strategy
• How do I mitigate/minimize
• Risk of Failure
• Cost of development
• Time to market
• How do I maximize
• Commercial opportunity
• Product exclusivity
• Development flexibility

10
Alternative Regulatory Tracks

• Compare and contrast -
• Humane Device Exemption (HDE)
• Orphan Drug
• Fast Track
• Accelerated Approval
• Other

11
How do I best manage my relationship with the
FDA? What (if any) are the points of leverage?

• What is the FDA agenda?
• What to expect from the FDA at each stage of
  development?
• FDA treatment of devices

12
How do I factor reimbursement into my product
development strategy?

• Reimbursement for therapeutics
• Reimbursement for devices

13
Considerations for overseas markets

• Europe
• Japan
• ROW

14
Where do I get more information?

• Devices
• http//www.rsna.org/REG/research/regulatory/wfprfc
  .html
• http//www.601help.com/index.html
• Drugs
• http//www.fda.gov

15
Closing remarks
16
Thank you Good luck!Next Event April 2,
2003Biotech Business Models
17
Appendix
18
What is life cycle planning?
Discovery

• Marketing
• Sales
• Clinical trials

• Development
• Manufacturing
• Pre-clinical
• Clinical

Approval (registration)
19
FDA Definitions of Device Classes

• Class I devices are defined as non-life
  sustaining. These products are the least
  complicated and their failure poses little risk
• Class II devices are more complicated and present
  more risk than Class I, though are also non-life
  sustaining. They are also subject to any specific
  performance standards
• Class III devices sustain or support life, so
  that their failure is life threatening

Source www.601help.com, www.fda.gov