Successfully reported this slideshow.
We use your LinkedIn profile and activity data to personalize ads and to show you more relevant ads. You can change your ad preferences anytime.
Expedited Approval Drugs
9 Approaches for Managing Regulatory Approval and Validation
Expedited Drug Approval Classifications
• Fast Track
• Accelerated Approval
• Priority Review
• Breakthrough Therapy
Fast Track Designation
• Drug must be intended to treat a serious condition
• May address an unmet medical need
• Fast Tra...
Accelerated Approval Designation
• Drug must treat a serious condition
• Provide significant improvement in safety or effe...
Priority Review
• Drug must treat a serious condition
• Provide significant improvement in safety or effectiveness when
co...
Breakthrough Therapy
• Drug must treat a serious condition
• Provide significant improvement in safety or effectiveness wh...
Often Dual Designations
• A drug often qualifies for two designations. For example, both
Breakthrough Therapy and Accelera...
Traditional Prescription Drug Development
• On average, 12-year development timeline*
• Including expenditures and opportu...
Expedited Approval Drugs
• Expedited drug development timelines vary
• Chemistry, Manufacturing and Controls (CMC) data of...
9 Approaches for Managing Expedited
Approval
#1
Open and transparent communication with the FDA throughout the
entire appr...
9 Approaches for Managing Expedited
Approval
#2
Additional stability data amendments will likely need to be filed during
t...
9 Approaches for Managing Expedited
Approval
#3
Launch commercial process with limited experience and optimize
post-approv...
9 Approaches for Managing Expedited
Approval
#4
Novel statistical models and approaches will need to be applied in
many ca...
9 Approaches for Managing Expedited
Approval
#5
Manufacturers should freely acknowledge where data is limited,
demonstrate...
9 Approaches for Managing Expedited
Approval
#6
Focus on patient safety and reliable supply of quality product at
launch, ...
9 Approaches for Managing Expedited
Approval
#7
Enhance analytical methods and understanding to offset more limited
proces...
9 Approaches for Managing Expedited
Approval
#8
Some CMC activities may be incomplete at launch. Incomplete
processes coul...
9 Approaches for Managing Expedited
Approval
#9
A post-approval product lifecycle management plan is a must, and it
needs ...
717 Indian Road
Glenview, Illinois 60025
Phone: 847-730-3475
Fax: 847-730-3498
www.biotechlogic.com
Contact: Peter Dellva,...
Upcoming SlideShare
Loading in …5
×

Accelerated approval drugs 9 approaches for managing regulatory approval and validation

841 views

Published on

Approaches to regulatory approval and process validation often must be quite different than traditional approaches. This presentation walks through nine approaches that might be helpful to your project.

Published in: Health & Medicine
  • Be the first to comment

  • Be the first to like this

Accelerated approval drugs 9 approaches for managing regulatory approval and validation

  1. 1. Expedited Approval Drugs 9 Approaches for Managing Regulatory Approval and Validation
  2. 2. Expedited Drug Approval Classifications • Fast Track • Accelerated Approval • Priority Review • Breakthrough Therapy
  3. 3. Fast Track Designation • Drug must be intended to treat a serious condition • May address an unmet medical need • Fast Track designation request can be submitted at any time during drug development process • Supporting data can be clinical or nonclinical • Earlier and more frequent communication with the FDA during development • Rolling review of application • Fast Track designation may be withdrawn if drug no longer meets qualifying criteria
  4. 4. Accelerated Approval Designation • Drug must treat a serious condition • Provide significant improvement in safety or effectiveness when compared to drugs currently on the market • May use surrogate endpoints to demonstrate clinical benefit • Approval is granted on conditional basis – post-approval trials are necessary • Earlier and more frequent communication with the FDA during development • Application is submitted in one package • Drug is subjected to expedited withdraw
  5. 5. Priority Review • Drug must treat a serious condition • Provide significant improvement in safety or effectiveness when compared to drugs currently on the market • Drug review process is shortened to 6 months
  6. 6. Breakthrough Therapy • Drug must treat a serious condition • Provide significant improvement in safety or effectiveness when compared to drugs currently on the market • Supporting data must be clinical • Intensive FDA guidance and involvement throughout development process involving senior FDA officials • Rolling review of application • Although clinical data is required, an all hands on deck approach is taken to get the drug to market
  7. 7. Often Dual Designations • A drug often qualifies for two designations. For example, both Breakthrough Therapy and Accelerated Approval designations are granted to qualifying drugs
  8. 8. Traditional Prescription Drug Development • On average, 12-year development timeline* • Including expenditures and opportunity costs, an average of ~$2.6 billion to bring a drug to market* *Tufts Center for the Study of Drug Development (CSDD) report
  9. 9. Expedited Approval Drugs • Expedited drug development timelines vary • Chemistry, Manufacturing and Controls (CMC) data often needs to be developed in about half the time of the traditional development process • Patient safety and product supply cannot be compromised • Analytical methods creation and product and process characterization must be started earlier in the development process • Some CMC activities may be incomplete at launch
  10. 10. 9 Approaches for Managing Expedited Approval #1 Open and transparent communication with the FDA throughout the entire approval and post-market process. The pharmaceutical company mindset of not wanting to learn certain information for fear of needing to revalidate based on those discoveries has no place in this new reality. New information will be learned pre- and post-launch, and amendments will need to be filed.
  11. 11. 9 Approaches for Managing Expedited Approval #2 Additional stability data amendments will likely need to be filed during the review process and in some cases post-market. This approach is required given that less data will be available at submission due to compressed timeframes.
  12. 12. 9 Approaches for Managing Expedited Approval #3 Launch commercial process with limited experience and optimize post-approval–the classic three runs is not the guiding force within this construct. The level of flexibility regulators will extend is determined for each specific product. Factors taken into consideration include: riskiness of product characteristics, seriousness of the condition and medical need, complexity of manufacturing processes, state of the innovator’s quality system and merits of the innovator’s risk-based quality assessment including Critical Quality Attributes (CQA).
  13. 13. 9 Approaches for Managing Expedited Approval #4 Novel statistical models and approaches will need to be applied in many cases. Representative samples and assays for these models will likely need to be acquired from sources, like prior knowledge and use of comparability protocols. Also, determination of the appropriate use of stability data from representative pilot scale lots will be required.
  14. 14. 9 Approaches for Managing Expedited Approval #5 Manufacturers should freely acknowledge where data is limited, demonstrate that the missing data pose no risk to patient safety or product supply and outline post-market strategy for acquiring the missing data. Conversations with the FDA are clearly required for successful outcomes here.
  15. 15. 9 Approaches for Managing Expedited Approval #6 Focus on patient safety and reliable supply of quality product at launch, not process optimization. In addition, begin critical product attributes and process characterization work much earlier than a typical pharmaceutical development process. In many cases, consider broader product quality ranges for non-Critical Quality Attributes until further manufacturing experience is acquired post-approval.
  16. 16. 9 Approaches for Managing Expedited Approval #7 Enhance analytical methods and understanding to offset more limited process understanding and to support future comparability work. Extremely important, involve commercial Quality Control representatives in the development assay design.
  17. 17. 9 Approaches for Managing Expedited Approval #8 Some CMC activities may be incomplete at launch. Incomplete processes could include: Process Validation, stability studies on commercial product, manufacturing scale/tech transfer data and complete control system data.
  18. 18. 9 Approaches for Managing Expedited Approval #9 A post-approval product lifecycle management plan is a must, and it needs to be included in the filing to support deferred CMC activities.
  19. 19. 717 Indian Road Glenview, Illinois 60025 Phone: 847-730-3475 Fax: 847-730-3498 www.biotechlogic.com Contact: Peter Dellva, Head of Business and Finance PD@biotechlogic.com

×