DIFFERENTIATING CRO-SPONSOR PARTNERSHIPS THE WORLDWIDE WAY

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1 DIFFERENTIATING CRO-SPONSOR PARTNERSHIPS THE WORLDWIDE WAY FROM TRANSACTIONAL TO TRANSLATIONAL Outsourcing in Clinical Trials Israel Angelico Carta, MD 20 March 2018

2 EXPERIENCED MANAGEMENT Neal R. Cutler, M.D. Chief Executive Officer Angelico Carta, M.D. President Michael Murphy, M.D., Ph.D. Chief Medical and Scientific Officer Henry J. Riordan, Ph.D. Executive Vice President, Medical and Scientific Affairs Founder and Chairman of Worldwide Board-certified psychiatrist and is also boardqualified in both neurology and clinical pharmacology NIH for over nine years in a variety of positions Current President of the American Foundation for Clinical Pharmacology Developed and obtained a FDA-approval for schizophrenia drug, FazaClo Board-certified neurologist Cofounder of Worldwide Former Managing Director of a private pharmaceutical development consultancy specializing in CNS drug development services Board-certified in psychiatry Doctorate in Pharmacology Faculty member within the Center for Experimental Pharmacology and Therapeutics at Harvard-MIT Founder, R&D Editor, American Health and Drug Benefits Clinical Research & Excellence (CARE) Lifetime Achievement Award for 2017 Licensed psychologist, with specialty training as a neuropsychologist Responsible for the scientific conduct and service delivery of all CNS clinical research initiatives Dr. Riordan has been involved in the assessment, treatment and investigation of various CNS disorders in both industry and academia for the past 20 years Authored over 275 publications, including nine books on the topics of neurology and diabetes. PharmaVoice 2017 s 100 Most Inspiring People in the life-sciences industry 2

3 BUILT ON A FOUNDATION OF MEDICINE AND SCIENCE Therapeutically-Aligned IMID* Investment and Continuous Improvement in World-Class Delivery via People, Tools, Process Neuroscience Cardiovascular and Metabolic Rare Disease Deep Therapeutic and Clinical Expertise Technology and Systems (Tools to Support Our Strategy) Reliable and Repeatable Clinical Execution Processes (Efficiency and Quality) *Immune Mediated Inflammatory Diseases 3

4 SIZE & GEOGRAPHIC PRESENCE Western Europe Nottingham Berlin Paris Rome Russia & Eastern Europe St. Petersburg Belgrade Bucharest Budapest Yekaterinburg Kemerovo Kharkov Kiev Moscow Skopje Sofia Tbilisi Warsaw Zagreb Corporate Headquarters Morrisville, NC, USA North & South America Beverly Hills, CA King of Prussia, PA Austin, TX Morrisville, NC San Antonio, TX São Paulo, Brazil Asia Pacific Singapore Seoul Taipei Australia Middle East & Africa Istanbul Award-Winning, Full-Service CRO Services Professionals Operating in 60 Countries 4

5 DIFFERENTIATING CRO-SPONSOR PARTNERSHIPS THE WORLDWIDE WAY Working with the right partner has significant influence on the success of a clinical program Exploring how CRO-sponsor partnerships can add value and address common challenges Assessing the benefits of therapeutic focus, expertise and experience Highlighting the importance of rigorous trial methodology, access and selection of the right patient population Encouraging accountability and open communication from every level of the organisation 5

6 DOES YOUR WORLDWIDE FOOTPRINT MAKE SENSE FOR MY STUDY? 6

7 HOW FOCUSED ARE YOU ON MY THERAPEUTIC AREA? 7

8 CRO PARTNERSHIP WORLDWIDE/SPONSOR ENGAGEMENTS What Activities Are Outsourced or Retained? What Data Facilitate Milestone Decisions? Strategic Initiatives or Transactional Requests? How Should the Pipeline Be Prioritized? Is This "Discovery in Clinical Development?" How to Create Value at Key Inflection Points? 8

9 FOCUS ON ANSWERING KEY QUESTIONS Therapeutic Experts 1 Can the study be enrolled in the desired time based on RFP specs? Any specs? 2 How many sites and countries will be required? Are they available? Regulatory Experts Operational Experts 3 What is the optimal distribution of countries and sites to maximize success? Publications Trial Databases 4 Who are the key investigators and are they committed to the proposed study design? Site Representatives 9

10 IT S CHESS, NOT CHECKERS Checkers Chess Predictable Intuitive Highly Functional Project Teams Slow Strategic Visionary Approach Capacity Driven Business Model Cognizant of Multiple Downstream Events 10

OPERATIONAL CONSIDERATIONS OF STRATEGIC PARTNERSHIP Governance Performance reviews at set times and across all levels Creation of Governance Charter and escalation pathway Executive committee review

11 OPERATIONAL CONSIDERATIONS OF STRATEGIC PARTNERSHIP Governance Performance reviews at set times and across all levels Creation of Governance Charter and escalation pathway Executive committee review joint committee Operational team review joint committee Involvement of SMEs and KPIs Review internally via Diurnal MPR Two way Early involvement Communication and Transparency Development Planning and Medical/Scientific Input Early involvement of Worldwide SME Enhance speed and reduce cost of development 11

SPONSOR & WORLDWIDE MOST FREQUENT DISCUSSIONS

Hybrid monitoring strategies? Historical data?

12 SPONSOR & WORLDWIDE MOST FREQUENT DISCUSSIONS Fast-track & breakthrough? Adaptive designs? Patient mapping and retention? Placebo? A One and done strategy? Hybrid monitoring strategies? Historical data? Registry? Reimbursement? Multi-organ assessments? PROs? Single patient IND/ named patient? At home or in clinic? 12

13 SCIENTIFIC/CLINICAL INTERFACE WITH FDA/EMA What Has Worldwide Done since 2008? Pre-IND meeting; EOP2; pre-anda; MHRA DNP (Neurology) DPP (Psychiatry)* DAAA(Analgesia) DPARP(Rheumatology)* Division of Bone, Reproductive and Urologic Products (DBRUP) Office of Cellular, Tissue, and Gene Therapies (OCTGT) Office of Generic Drugs (OGD) Ant-Infective Products (DAIP)* Hematology Products (DHP)* How Do We Proceed? Sponsor/Worldwide IND team What Do We Discuss? Regulatory Strategy Bioanalytic Review Primary & Secondary pharmacology Safety pharmacology Regulatory ADME-TOX What Do We Offer? FIH in NVs (small molecules, biologics) FIH in patients (small molecules, biologics, ATMP) Proof of Mechanism (Target engagement) Proof of Concept within ADME-TOX package Potentially pivotal trials Design, assessments, analyses *Includes orphan indications 13

WORLDWIDE ORPHAN INDICATIONS EXPERIENCE Acromegaly Amyotrophic Lateral Sclerosis Behavioral variant, Frontotemporal Dementia, Granulin mutation Cystic fibrosis Duchenne muscular dystrophy Down

14 WORLDWIDE ORPHAN INDICATIONS EXPERIENCE Acromegaly Amyotrophic Lateral Sclerosis Behavioral variant, Frontotemporal Dementia, Granulin mutation Cystic fibrosis Duchenne muscular dystrophy Down Syndrome Fabry disease Gaucher disease Growth hormone insensitivity syndrome (Laron Syndrome) Hemophagocytic lymphohistiocytosis Pemphigus Pompe Disease Idiopathic pulmonary fibrosis Idiopathic thrombocytopenic purpura Juvenile idiopathic arthritis Lambert Eaton myasthenic syndrome Mucopolysaccharidosis I (Hurler syndrome) Mucopolysaccharidosis II (Hunter syndrome) Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) Niemann Pick type C Pulmonary arterial hypertension (PAH) Sickle cell disease Hemophilia B Thrombotic microangiopathy: Thrombotic thrombocytopenic purpura (TTP) Von Willebrand disease Velocardiofacial syndrome, psychosis Helsnoortel-Van der Aa syndrome Worldwide Observations Approximately 60 studies Novel and repurposed products including ATMP Permissive science, innovative technology Evolving regulatory and commercial climate Innovative, efficient program designs Diverse Measures Unique operational solutions Participatory research models Exploit geographical areas where the healthcare system and patient practice/referral patterns promote efficiencies in recruitment and study execution 14

DEVELOPMENT TO AN INFLECTION POINT WORLDWIDE INTEGRATED SOLUTIONS Prior Art Special Assessments Facility Orphan Clinical Development Bioanalysis Patient Related Strategic program support (IND,

15 DEVELOPMENT TO AN INFLECTION POINT WORLDWIDE INTEGRATED SOLUTIONS Prior Art Special Assessments Facility Orphan Clinical Development Bioanalysis Patient Related Strategic program support (IND, pre-ind, EOP2, Accelerated - Fast Track - Breakthrough ) Early phase/ late phase integration Staffing Appropriate to Phase Sample Acquisition (matrices and management) Special clinical assessments for PK/PD Cohort Management & Medical Oversight Enabling Technology Strategic Support 15

16 WORLDWIDE APPROACH WITH THE END IN MIND CREATING AN EVIDENCE-BASED VALUE PROGRAM Physician adoption, payer coverage, pricing and thus patient access Regulatory and peer- reviewed publication mix? What s the population likely to use the product? Did you include patients in the deep end of the swimming pool? How will physicians actually use it? Outcomes (not measures)? Interaction effects with current treatment How will it affect the health care system? methodological rigor, not funding source, determines if data will be acknowledged 16

17 IDEAL STRATEGIC PARTNER Experience Strategic & Operational capabilities Robust processes Fully Integrated Technology Transparent Culture of Communication and Visibility Flexible Ability to Expand alongside growing pipelines and optimize resources 17

18 THANK YOU Angelico Carta, MD President, Worldwide Clinical Trials Global United Kingdom Tel:

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