NIPTE Seminar at US FDA, 16 March 2016. QBR as an Organizing Principle for the Proposed NIPTE Center of Excellence for Pharmaceutical Formulations (CEPF)

1. The Next Decade: Visioning a Collaboration
between NIPTE & FDA CDER OPQ
Ajaz S. Hussain, Ph.D., President
The National Institute of Pharmaceutical Technology & Education, Inc.

2. NIPTE
501(c)(3) Non-profit organization
Founded in 2005
Incorporated in 2007
Headquarters: Minneapolis, MN
12 Schools of Pharmacy, 3
Schools of Engineering, 1
Medical School
Improving Quality and Lowering
Costs with Confidence
March 16, 2016 2FDA CDER White Oak, Building 22, Room 2205

3. Pharmaceutical Science & Quality: Looking Back
Regulatory Science: FDA Review & Inspection
Thawing the “PSF”: OPS, SUAPC, BCS,…, PQRI,…
PAT to Pharmaceutical Quality in 21st Century to Critical Path Initiative & NIPTE
OPQ: One Voice for Quality, Integrated, Metrics, Culture …… & NIPTE
Pre-OPS OPS- Early
Days
OPS in 21st
Century
OPQ
March 16, 2016 3FDA CDER White Oak, Building 22, Room 2205

4. NIPTE 2005 – 2015 and beyond
2005 – 2015 Highlights
• FDA – NIPTE MOU, June 2005
• Developing QbD guidance elements on
process design space (2008-2010)
• Critical Path Manufacturing Research Sector
Initiative Grant (U01) Awarded by FDA in 2011
for 5 years with funding at up to $7,000,000
annually
• Reviewer Education in State of the Art
Pharmaceutical Manufacturing Technology
Lessons & Aspirations
• What worked: Integrated multi-disciplinary
research based solutions to complex
development/regulatory challenges
• For example - Gabapentin stability design space
• What can and should work better
• Knowledge transfer and management
• Understanding FDA’s challenges & FDA feed-
back
• Proactive planning for ‘grand’ vs ‘brushfire’
challenges
• NIPTE’s Centers for Excellence – aligned with
OPQ: One Voice of Quality
March 16, 2016 4FDA CDER White Oak, Building 22, Room 2205

5. Optimal efficiency via stochastic basis for clinical relevance
March 16, 2016 FDA CDER White Oak, Building 22, Room 2205 5
To optimal efficiency with confidence Via Stochastic, Clinical Relevance
Research Related to Formulation and Pharmaceutical Product Stability. Advisory Committee for Pharmaceutical Science. April 14, 2010

6. Integrated solutions need multi-disciplinary expertise
March 16, 2016 FDA CDER White Oak, Building 22, Room 2205 6
Integrated Solution Needs Multi-disciplinary Expertise
Research Related to Formulation and Pharmaceutical Product Stability. Advisory Committee for Pharmaceutical Science. April 14, 2010

7. FDA CDER OPQ’s mission is to assure that quality drugs
are available to the American public.
Assure that all human drugs meet the same quality
standards to safeguard clinical performance;
Enhance science- and risk-based regulatory approaches;
Transform product quality oversight from a qualitative to
a quantitative and expertise-based assessment;
Provide seamless integration of review, inspection,
surveillance, policy, and research across the product life
cycle; and
Encourage development and adoption of emerging
pharmaceutical technology
• NIPTE’s Mission
• The mission of NIPTE is to improve human
health through multi-university collaborative
research to advance the quality, safety,
affordability and speed to market of medicines
through interdisciplinary research and
education in pharmaceutical technology
March 16, 2016 FDA CDER White Oak, Building 22, Room 2205 7

8. Grand Challenge in Pharmacutical Quality
• The FDA’s PAT Guidance opened the door to continuous manufacturing; ‘tipping point’
reached in 2015
• ICH Q8 outlined a methodology for ‘Quality by Design’; methodology without 21st century
ontology curtails progress
• Weak epistemology reduces confidence generally and in rapidly globalized supply chain it
raises serious concern such as “are these data too good to be true?”
• Through its research and educational programs NIPTE will contribute towards realizing CDER
OPQ vision, in this context the Nation needs a Center of Excellence for Pharmaceutical
Formulations (CEPF)!
March 16, 2016 8FDA CDER White Oak, Building 22, Room 2205

9. Today we share our initial thoughts on CEPF
• NIPTEs’ Center of Excellence for Pharmaceutical Formulations (CEPF): Making the case
• Stephen R. Byrn, Ph.D., Purdue University
• QBR as one of the Organizing Principles for NIPTE CEPF
• Kenneth R. Morris, Ph.D., Long Island University– Brooklyn Campus
• Critical Roles of Raw Materials and Manufacturing Processes in Product Quality
• Stephen W. Hoag, Ph.D., University of Maryland Baltimore
• Robert (Bill) O. Williams, Ph.D., University of Texas at Austin
• Feng Zhang, Ph.D., University of Texas at Austin
• Advanced Characterization of Drug Substance and Drug products
• Raj Suryanarayanan, Ph.D., University of Minnesota
• Eric J. Munson, Ph.D., University of Kentucky
• Q&A and FDA Feedback
March 16, 2016 FDA CDER White Oak, Building 22, Room 2205 9