Launching Clinical Trials:

1
Launching Clinical Trials Small Company vs Large
Company Environment
Laurie A. Ridener, RAC Senior Director, Clinical
and Regulatory Affairs Pervasis Therapeutics,
Inc. Cambridge MA
2
Background

• Pervasis Therapeutics, Inc.
• Combination Biologic / Device Product
• 15 Employees
• EMD Serono, Inc.
• Recombinant Therapeutic Proteins, Novel Device
  Delivery Systems
• 3,000 Employees WW
• 700 in US (125 in Clinical Affairs, 20 in
  Regulatory Affairs)
• Biolink, Inc.
• Hemodialysis Access Device
• 30 Employees
• Continuum Biomedical, Inc.
• Medical / Dental Laser Systems
• 75 Employees
• Medical Device CRO
• 5 years

3
Clinical Development Program

• Small Company
• University-based Research Laboratory
• Engineer
• Physician
• Entrepreneur
• Resources limited
• Timelines fixed
• Delays terminal
• Expert management

• Large Company
• Internal R D
• Marketing
• Clinical Affairs
• Senior Management
• Business Development
• Resources available
• Timelines extendable
• Delays accommodated
• Management diffuse

4
Basic Documentation All Companies

• Approved IDE / IND
• Protocol
• Investigators Brochure/Instruction Manual
• Informed Consent
• Investigational Device / Product Supply
• Investigators
• Evaluated, Qualified, Initiated
• Study Subjects
• Data to Support Marketing Application

5
Launching a Clinical Trial Small Company

• Full Service CRO
• SOP Development
• Protocol Development
• Site Monitoring
• Project Management
• Data Management
• Statistical Analysis
• Medical Writing
• Training

• Niche Service Providers / Independent Contractors
• Monitoring
• Statistical Analysis
• Project Management
• Medical Writing
• Vertical Integration
• Hire key personnel
• SOPs in place

6
Launching a Clinical Trial Small Company

• Full Service CRO
• The bigger the better
• Proven Track Record in managing clinical trials
• All the resources within one organization
• One check to write each month
• High level of expertise of consultants
• Too big to manage
• Large team membership, often at many locations
• Involved with too many clinical trials, no
  individualized attention
• Procedural flexibility limited
• Review / processing time extended
• Contract negotiations / changes arduous
• High turnover rate

7
Launching a Clinical Trial Small Company

• Niche Service Providers / Independent Contractors
• Customer Oriented
• Individualized service and contracts
• Low overhead
• Pay only for service actually needed
• Lots of Moving Parts
• Expertise not guaranteed
• Project management becomes full time job
• In-house leadership critical

8
Launching a Clinical Trial Small Company

• Vertical Integration
• Robust Team Composition
• Direct control / influence over study progress
• Reaction time swift and nimble
• SOPs need to be in place
• Liability
• Huge milestone, big commitment
• Harder to exchange a bad hire
• Office space a consideration

9
Launching a Clinical Trial Small Company

• Planning for a Smooth Launch
• Investigator involvement in protocol development
  AND case report form content
• Test drive CRFs with study coordinators
• Know IRB meeting dates, assist investigators in
  preparing IRB application
• Schedule Site Qualification visits parallel with
  IDE/IND preparation
• Determine packaging and shipping requirements
  confirm ship to addresses
• Pre-assemble Study Notebooks

10
Launching a Clinical Trial Small Company

• Investigator Meeting
• Generates enthusiasm and momentum
• Chance to clarify end-points, data collection
  requirements
• Present timeline and anticipated enrollment
  activity
• Allows investigator concerns to be address with
  collegial support
• Include Study Coordinator
• cultivate a relationship of collaboration and
  teamwork
• Include other direct participants
• Radiologists, independent assessors, lab
  personnel
• Schedule in conjunction with society meeting

11
Launching a Clinical Trial Small Company

• Intense pressure to enroll that first patient
• Start contract / agreement / indemnification
  process early
• Submit to IRBS concurrent with IDE/IND submission
• Risk FDA requires revisions, need to resubmit
  to IRB, additional cost
• Benefit study sites ready to enroll upon FDA
  green light
• Be ready to turn around IRB requested changes to
  the consent document quickly
• Case Report Forms printed or EDC built, tested,
  on-line
• Collect Investigator regulatory documents early
• CV, Financial Disclosure, copy of medical
  license, agreement

12
Launching a Clinical Trial Small Company

• Key Considerations for Success
• Director/Senior level FTE
• Strong leadership skills with extensive clinical
  trial expertise and project management oversight
• Small company experience mandatory
• Hands-on experience is equally important as
  project oversight
• Consider a small to medium sized CRO to get the
  best of all possibilities
• Full service
• Personal attention
• Flexibility and quick turnaround time on
  deliverables

13
Clinical Development Program

• Small Company
• University-based Research Laboratory
• Engineer
• Physician
• Entrepreneur
• Resources limited
• Timelines fixed
• Delays terminal
• Expert management

• Large Company
• Internal R D
• Marketing
• Clinical Affairs
• Senior Management
• Business Development
• Resources available
• Timelines extendable
• Delays accommodated
• Management diffuse

14
Basic Documentation All Companies

• Approved IDE / IND
• Protocol
• Investigators Brochure/Instruction Manual
• Informed Consent
• Investigational Device / Product Supply
• Investigators
• Evaluated, Qualified, Initiated
• Study Subjects
• Data to Support Marketing Application

15
Launching a Clinical Trial Large Company

• In-house capabilities
• SOP Development
• Protocol Development
• Site Monitoring
• Project Management
• Data Management
• Statistical Analysis
• Medical Writing
• Training

• Niche Service Providers / Independent Contractors
• Monitoring
• Statistical Analysis
• Project Management
• Medical Writing
• Vertical Integration
• Key personnel in place

16
Launching a Clinical Trial Large Company

• Firm commitment from Senior Management
• Set priorities
• Allocate resources
• Strong leadership
• Project Management
• Diverse team members, multinational
• Other priorities, not customer focused
• Large number of clinical studies

17
Launching a Clinical Trial Large Company

• Weekly Cross Functional Team Meetings
• Clinical Trial Leaders
• Clinical Operations, Monitors
• Manufacturing
• Data Management
• Biostats
• Regulatory Affairs
• Pharmacovigilance
• Follow-up with Team Members between meetings
• Assign and track action items, hold members
  accountable

18
Launching a Clinical Trial Large Company

• Planning for a Smooth Launch
• Minimal Investigator involvement
• Kick-off meeting still valuable
• IRB applications prepared by site personnel
• Site Qualification visits are scheduled as
  monitors are available
• Shipping clinical trial materials (Supply Chain,
  outsource)
• Study Notebooks (outsource)

19
Launching a Clinical Trial Large Company

• Key Considerations for Sucess
• Senior level Clinical Trial Leader
• Strong leadership skills with extensive clinical
  trial expertise and project management oversight
• Budgeting expertise, knowing where to spend,
  where to save
• Consider outsourcing when appropriate
• Excellent interpersonal communication skills
• Bi-directional
• Different management styles for different
  personalities

20

• Thank you!
• Further questions
• Laurie Ridener
• lridener_at_pervasistx.com
• 617.871.1211