A Rare International Dialogue (Saturday May 11, 2019)
Drivers of Drug Development β Regulatory Collaboration
Medical Product Development for Rare Diseases: FDA perspective CDER Larissa Lapteva, US Food and Drug Administration, CBER Medical Product Development For Rare Diseases
Part 2: Regenerative Medicine Therapies
FDA Regulatory Perspective
Similar to STREAM TWO: Larissa Lapteva, Medical Product Development For Rare Diseases Part 2: Regenerative Medicine Therapies FDA Regulatory Perspective
Similar to STREAM TWO: Larissa Lapteva, Medical Product Development For Rare Diseases Part 2: Regenerative Medicine Therapies FDA Regulatory Perspective (20)
2. 2
Efficient Development
Regulatory Programs
β’β― Fast Track
β’β― Breakthrough Therapy
β’β― Regenerative Medicine Advanced Therapy
β’β― Qualified Infectious Disease Product
β’β― Orphan* Product
β’β― Priority review
β’β― priority review voucher program
β’β― Accelerated approval
β’β― Grant programs, user fee waivers, and other
incentives
Product Development
β’β― INTERACT**
β’β― Innovative study designs
β’β― Effective use of βreduce, refine, replaceβ
principle and modeling in preclinical
programs
β’β― Enrichment strategies
β’β― Adaptive and complex innovative
designs****
β’β― Product development tools including
biomarkers and clinical outcomes assessments
β’β― Patient Engagement***
to support
*Designating an Orphan Product: Drugs and Biological Products
https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/default.htm
**INTERACT https://www.fda.gov/BiologicsBloodVaccines/ResourcesforYou/Industry/ucm611501.htm
***Patient-Focused Drug Development https://www.fda.gov/drugs/developmentapprovalprocess/ucm610279.htm
****Complex Innovative Design Pilot Program https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm617212.htm
4. 4
Expedited Programs for Product Development
Fast Track (FT) Breakthrough
Therapy (BT)
Regenerative Medicine
Advanced Therapy (RMAT)
Features
Frequent meetings
Frequent written
communication
Eligibility for *:
ΓΌβ― Accelerated approval
ΓΌβ― Priority review
Rolling Review
*if relevant criteria are met
All of FT Features +
ΓΌβ― Intensive guidance on an
efficient drug development
program, beginning as early
as Phase 1
ΓΌβ― Organizational commitment
All of BT Features +
ΓΌβ― Early interactions to discuss
any potential surrogate or
intermediate endpoints
ΓΌβ― Statute addresses potential
ways to support accelerated
approval and satisfy post-
approval requirements
Guidance for Industry: Evaluation of Devices Used with RMATs
https://www.fda.gov/downloads/biologicsbloodvaccines/guidancecomplianceregulatoryinformation/guidances/cellularandgenetherapy/ucm585417.pdf
6. 6
Draft Guidance Documents To Facilitate
Gene Therapies for Rare Diseases: July 2018
β’β― Human Gene Therapy for Rare Diseases
β’β― Human Gene Therapy for Hemophilia
β’β― Human Gene Therapy for Retinal Disorders
β’β― Chemistry, Manufacturing, and Control (CMC) Information for Human Gene
Therapy Investigational New Drug Applications (INDs)
β’β― Long-Term Follow-up After Administration of Human Gene Therapy Products
β’β― Testing of Retroviral Vector-Based Human Gene Therapy Products for
Replication Competent Retrovirus During Product Manufacture and Patient
Follow-up
https://www.fda.gov/BiologicsBloodVaccines/
GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/
default.htm
9. 9
Patient Engagement and
Patient-Focused Drug Development
β’β― FDA Patient Representative Program
β’β― Patient-Focused Drug Development meetings (FDA and stakeholders)
β’β― Formation of FDA Patient Affairs Staff group in 2017
β’β― FDA Listening Sessions (patient experiences with specific conditions)
β’β― Science of Patient Input: series of guidance documents on methodological
approaches to incorporating patient voice in medical product development and
regulatory decision-making
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm610279.htm
β’β― Inclusion of Patient Experience Data in marketing applications and reviews
The 21st Century Cures Act (2016) and the FDA Reauthorization Act (2017): Plan for Issuance of Patient-Focused Drug Development Guidance
https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM563618.pdf