Achieving Regulatory Success: Areas of focus for biotechnology companies. Michael J. Schlosser, PhD, DABT April 21, 2013

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1 Achieving Regulatory Success: Areas of focus for biotechnology companies Michael J. Schlosser, PhD, DABT April 21, 2013

2 Regulatory Success Outline Regulatory Initiatives Regulatory Science Pre-Regulatory Science Utilization of science tools for Regulatory Success

3 Regulatory Success Efficacy Compliance Material API Nonclinical Toxicity Probability of Regulatory Success Competitors PK/PD Clinical Safety

4 Regulatory Success Material API FDA Interaction Nonclinical Toxicity Efficacy FDA Interaction Probability of Regulatory Success FDA Interaction Clinical Safety Compliance FDA Interaction Competitors PK/PD

5 What is FDA doing to encourage interactions during drug development? Food and Drug Administration Safety and Innovation Act ( FDASIA ) of 2012 Updated Prescription Drug User Fee Act (PDUFA) Increased transparency FDA performance goals Addresses patent exclusivity Innovation incentives Expedited drug development / review pathways

6 What is FDA doing to encourage interactions? Expedited drug development / review pathways Priority Review status Accelerated Approval Fast Track designation Breakthrough designation FDA Meetings Pre-IND meetings Pre- Pre-IND meetings (OCTGT) End-of-Phase II meetings prenda meetings

7 Does Early FDA Communication Help? Development >3 years shorter with pre-ind meeting

8 Does Early FDA Communication Help? INDs filings are on the rise FDA website

9 Does Early FDA Communication Help? Large number of projects in the pipeline (e.g., oncology) Oncology Projects by Stage Inside BIO Industry Analysis, Jan 2013 Source: PhRMA, 2013

10 Does Early FDA Communication Help? Biotech well represented in 2012 NDAs FDA website FDA website

11 Regulatory Success Efficacy Compliance Material/ API Nonclinical Toxicity Probability of Regulatory Success Competitors PK/PD Clinical Safety

12 What is FDA doing regarding compliance? Guidances GLP, GMP, GCP guidances > 400 related to small and large molecule drugs Draft Guidances (examples) Financial Disclosure by Clinical Investigators Labeling for Prescription Drug and Biological Products Electronic submissions Genotoxic Impurities Combination products Clinical Trial Enrichment

13 What can biotechnology companies do to be compliant? Familiar with relevant guidances Working knowledge of regulatory enforcement in each relevant area (e.g., nonclinical, clinical, CMC) Sponsor SOPs Vendor outsourcing Inspection and monitoring of CROs / CMOs Document control and reviews API shipment to third-parties Issue / crisis management example

14 Regulatory Science Regulatory Science The application of science to drug development and approval Utilizes technology, standards, and approaches for assessing efficacy, safety and quality If applied correctly, enables innovation and speed with dramatic improvements in moving to clinical Proof of Concept and Registration Is starting to alter current drug development and approval Incorporating new science into regulations is slow as new technology (genomics, proteomics, biomarkers, etc.) has outpaced traditional validation approaches

15 What is FDA doing to advance regulatory science? Drug Development Tool Qualification Activities Cardiovascular Safety Research Consortium Serious Adverse Events Consortium Biomarkers Consortium Clinical Trials Transformation Initiative Critical Path Institute Predictive Safety Testing Consortium (PSTC) Patient Reported Outcomes (PRO) Consortium / epro Consortium Coalition Against Major Diseases Consortium Critical Path to TB Drug Regimens Polycystic Kidney Disease (PKD) Consortium Multiple Sclerosis Outcome Assessments Consortium Coalition for Accelerating Standards and Therapies (CFAST) Analgesic Clinical Trials Translation, Innovation, Opportunities and Networks (ACTTION) Initiative FDA Website

16 Regulatory science influences Discovery preind / IND / Phase I Phase II Phase III Post market Lead Optimization and Candidate Selection Candidate Survival Proof-of-Concept and Product Differentiation Labeling Balanced risk-benefit assessment Increasing Confidence in Safety-Efficacy

17 Pre-Regulatory Science Concept Utilize and incorporate best science available into nonclinical and clinical programs ( validated or not) Plan as early as possible and incorporate available tools in both discovery and development activities to improve decision making Biotechnology companies are responsible for providing adequate and appropriate scientific information to justify development approach (nonclinical and clinical) FDA is still learning the safety profiles and biology of new targets -- biotechnology companies can help educate

18 Target characterization Efficacy Target Characterization Safety - Function of target in disease? - Target modulation = effect? - Human tissue for study? - Genetic variants? - Homology across species? - Biomarkers available? - PK/PD relationships Drugable - Related targets? - Homology across species? - Target and normal function? - Side-effects of target modulation? - Biomarkers for side-effects? - PK/side-effect profile? - Target accessible? - Target s family drugable? - Can target modulation be achieved? - Can target be purified? - Requires for specificity and selectivity? - Small versus large molecule?

19 What is the most efficient way to use science tools for project success? Step 1 Define what your drug should look like on the market (e.g.,target Product Profile) Build on global market-based product attributes What science tools / approaches are available for predicting those attributes Examples In vitro models (e.g., herg inhibition, CYP450 inhibition) Modulation of in vivo pharmacological target / disease biomarkers Concentration response in vitro (e.g.,ic50/ic90) versus free drug plasma levels at efficacious dose in pharmacology animal model

20 What is the most efficient way to use science tools for project success? Step 2 Identify gaps in current data set needed to predict drug candidate or product profile Is the gap small or large? Is the gap critical for predicting the profile? Are technology tools available for the prediction? What is the risk of not filling the gap? Gap analysis drives project strategy based on risk

21 What is the most efficient way to use science tools for project success? Often identified gaps in projects IP issues Accessibility and understanding of patient population Preclinical efficacy / toxicity and PK in animal models Cost of Goods at estimated human dose Physio-chemical characteristics compatible with intended route Attributes for differentiating from gold standard Immunogenicity strategy for biologics Appropriate biomarker identified for disease / target Requirements to keep material supply off critical path Product reimbursable Design of clinical proof-of-concept study

22 Proof-of-Concept Is target understood? First-in-Class Second-in-Class No Precedent Second-in-Class Clear Precedent Efficacy / mechanistic PoC Required Identify short-term biomarker or efficacy endpoints Consider other PoC issues (safety, PK, etc.) What other attributes differentiate from first-in-class?

23 Regulatory Success: Risk Management Risk versus Resource Assessment Applied to Gaps Probability that risk will occur Probable Unlikely Cost of Goods (example) Slight Severe Impact if it risk occurs Size of bubble represents cost/difficulty of implementing solution

24 Regulatory Success: Summary Early communication with FDA is critical Establishes relationships and partnerships early which is important for novel targets where FDA education is critical Compliance can be a moving target Understanding regulatory guidances and their level of enforcement is critical for not being under or over compliant Incorporate available predictive tools into projects Allows better decision making throughout every stage of drug discovery and development Identify and analyze project gaps early Strategic risk assessment of each gap allows prioritization of action plans to reach key decisions early and efficiently

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