A Peek at CMO East 2013

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A Peek at CMO East 2013

  1. 1. Development Process: Plan with the End in Mind Commercialization NDA (BLA)/MAA Submission(s) Registration Strategy Integrated Summaries Final Reports Study Conduct Protocols Protocol Outlines Clinical Development Plan Non-Clinical Plan 1  
  2. 2. Drug Development Regulatory Provisions §  Fast Track Development Status - To expedite product development §  Orphan Drug Status - To encourage development of drugs for rare diseases §  Accelerated Approval - To speed approval in a life-threatening disease based on surrogate marker data with a commitment to conduct confirmatory studies §  Key Consideration - Benefit risk assessment 2  
  3. 3. Drug Development Imperative: Prepara&on  of  Target  Product  Profile   §  Considerations –  Indications –  Target patient populations –  Efficacy profile –  Safety profile –  Formulations –  Dosage regimens 3  
  4. 4. Input Into Drug Development Program §  Involve Constituencies   -­‐  Regulatory Agencies -­‐  Key Investigators & Opinion Leaders (KOLs) -­‐  Patient Advocacy Groups 4  
  5. 5. Clinical Trial Statistical Considerations Two Separate Assessments Pre-­‐Study  Sample  Size  Determina3on                Post-­‐Study  Sta3s3cal  Analysis   (Output:    number  of  pa3ents)                          (Output:    p  value  result) Assump&ons   Power (80%)                          -­‐                  Probability of sufficient number of patients studied to detect a difference (avoid false negative) §  Assessments   §  Primary  endpoint  (eg,  pa3ent  survival)   §  Sta3s3cal  test  to  determine  p  value  (eg,   modified  log-­‐rank  test  to  compare  Kaplan-­‐ Meier  pa3ent  survival  curves)   [Post  Hoc  Sensi&vity  Analyses]   §           α  value  (≤0.05)                          -­‐                  Probability of result occurring by chance (avoid false positive) §         Difference  to  be  detected   §  Enrollment  period   §  Follow-­‐up  dura3on   [Prespecified  Sta&s&cal  Analysis  Plan]     5      
  6. 6. Regulatory Meeting Preparation   § Back  to  Basics   • WHO   • WHAT   • WHERE   • WHY   • HOW     6  
  7. 7. Regulatory Preparation: Back to Basics   § WHO    (is going to be involved)   • Iden6fy  individuals  from  the  Regulatory  Agencies  who  will  be     involved  in  interac6ons  so  the  right  company  and  external   experts  are  available  for  mee6ngs  and/or  teleconferences   • Contact  external  experts  as  soon  as  possible  to  ensure  their   availability         7  
  8. 8. Regulatory Preparation: Back to Basics   § WHAT  (needs to be done)   • Compile  a  complete  list  of  ac6vi6es  that  need  to  be        accomplished  for  the  mee6ng   • Do  not  overlook  details  which  can  cause  delays  or  inconvenience   such  as  booking  a  sufficient  number  of  hotel  rooms     8  
  9. 9. Regulatory Preparation: Back to Basics   § WHERE   (will prep meetings and actual meeting be held)   • Determine  loca6on  of  mee6ngs  early  on  to  facilitate      travel  and  hotel  bookings   • Prepare  for  con6ngencies  such  as  the  possibility  of  extra      aRendees         9  
  10. 10. Regulatory Preparation: Back to Basics   § WHY  (the  need  for  a  mee6ng  should  be  asked                      to  provide  clarity  of  mee6ng  goals  and  details  to                      facilitate  prepara6on)                         • Establish  rapport  as  possible  with  Regulatory  Agency  staff  to   assist  with  planning     • Hold  prep  teleconferences  and/or  mee6ngs  to  minimize   disconnects       10  
  11. 11. Regulatory Preparation: Back to Basics   § HOW    (will logistics be coordinated to avoid duplication of   activities)                                 • Prepare  a  6me  and  event  schedule  to  delineate      expecta6ons         11  
  12. 12. Regulatory Preparation   §   Needs       • Iden6fy  the  goals  for  the  mee6ng     • Understand  how  work  is  distributed     • Foster  collabora6on       12  
  13. 13. Regulatory Preparation   §   Details       • Know  the  audience   -­‐    Obtain  informa6on  on  their  exper6se     • Try  to  preempt  an6cipated  ques6ons  in  the  briefing   document   -­‐    Understand  Agency  stance  on  topic(s)  to  be  discussed     13  
  14. 14. Drug Development §  Critical Success Factor −  Upfront  planning  to  prevent  problems   14  
  15. 15. Drug Development Key Factors in Drug Development - Balance Quality Drug Development Time Cost Align Goals and Incentives for Success 15  

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