CTTI Overview One Decade of Impact. One Vision Ahead.

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Feb. 6, 2018 CTTI Overview One Decade of Impact. One Vision Ahead. Pamela Tenaerts, MD, MBA Executive Director

#CelebrateCTTI Discover how CTTI has influenced the clinical trials enterprise during the past decade. Hear from other organizations about the benefits of applying CTTI recommendations successfully. Learn how CTTI s current work will impact and shape the future of clinical trials.

Public-Private Partnership Co-founded by Duke University & FDA Involves all stakeholders

Clinical Investigators Government & Regulatory Agencies Industry (pharma, bio, device, CRO, & tech) Everyone Better must Streamlined have a Fit seat for purpose at the table Clinical Trials IRBs Trade & Professional Orgs Patients, Caregivers & Patient Advocacy Groups Academia MULTI-STAKEHOLDER TRANSFORMING CLINICAL TRIALS Multi-Stakeholder REAL-WORLD IMPACT EVIDENCE-BASED

EVIDENCE-BASED MULTI-STAKEHOLDER 2 TRANSFORMING CLINICAL TRIALS REAL-WORLD IMPACT Evidence-Based RESEARCH METHODS We use quantitative & qualitative research methods, selecting those best aligned with each project s objectives, to: Identify/describe what is going on to gain a better understanding of a particular phenomenon. Move beyond individual views to a more complete and objective understanding of the disincentives and motivators for change. Equipped with data, we then challenge assumptions, identify roadblocks, build tools and develop recommendations to change the way people think about and conduct clinical trials. STAKEHOLDER INTERVIEWS FOCUS GROUP DISCUSSIONS SURVEYS SYSTEMATIC LITERATURE REVIEWS EXPERT MEETINGS

EVIDENCE-BASED MULTI-STAKEHOLDER TRANSFORMING CLINICAL TRIALS REAL-WORLD IMPACT Examples of Real-World Impact within Organizations CTTI s Central IRB tools & recommendations are used by organizations such as the National Institute of Neurological Disorders and Stroke (NIH) and North Shore-LIJ Health System. CTTI s Quality by Design framework is used by organizations such as AstraZeneca, DCRI, The Medicines Company, PCORNET, Pfizer, Target Health Inc. and University of Oxford. The Cystic Fibrosis Foundation has applied CTTI s recommendations to improve its DMC operations. Eli Lilly is implementing CTTI s informed consent recommendations through their new e-consent model. UCB Bioscience is applying CTTI s recommendations to develop mechanisms for implementing patient engagement strategies across the drug development life cycle.

MULTI-STAKEHOLDER REAL-WORLD IMPACT TRANSFORMING CLINICAL TRIALS Real-World Impact at the Policy Level EVIDENCE-BASED CTTI and its work have been cited in: Several FDA guidance documents An EMA reflection paper A NIH draft policy Congressional efforts around 21st Century Cures legislation

CTTI Organization Executive Committee Oversight and strategic direction Steering Committee Input into strategy and project selection Conducts projects and develops strategies for implementation Staff Project support and organization in pursuit of mission

CTTI Membership *Version: Jan. 29, 2018

Patient Engagement Evolution Patient Engagement 1.0 Patient advocates on EC, SC, and project teams PLC established Jan 2013 Increased patient representative participation Patient reps began serving as team leads Fast-tracked PG&CT project Normed (no question) inclusion of patients as equal partners into every aspect of clinical trial (reform) Patient Engagement 2.0 PLC full integration into the Steering Committee in 2015 Additional activities being explored Individual patients reimbursed for time on CTTI activities incl. projects Patient/caregivers on project teams have played critical role in shaping many projects NEW: CTTI and FDA launch Patient Engagement Collaborative Almost all CTTI recommendations to date include a recommendation to involve all stakeholders particularly patients in the process

Evolution of CTTI Mission 2008 To identify practices that through broad adoption will increase the quality and efficiency of clinical trials 2013 To identify and promote practices that will increase 2016 To develop and drive adoption of practices that will increase

CTTI Projects

CTTI Methodology

PROJECT PORTFOLIO Areas of Strategic Focus: SYSTEMATIC EVIDENCE GENERATION PATIENTS AS EQUAL PARTNERS EFFICIENT & QUALITY TRIALS PUBLIC HEALTH CONCERN SAFE & ETHICAL TRIALS Active Projects: MCT Legal & Regulatory MCT Mobile Devices Patient Groups & Clinical Trials Investigator Qualification ABDD HABP/VABP Studies MCT Stakeholder Perceptions Real World Evidence State of Clinical Trials Complete Projects: Large Simple Trials MCT Novel Endpoints Registry Trials GCP Training Investigator Community Monitoring Quality by Design Recruitment Site Metrics ABDD Peds Trials ABDD Streamlining HABP/VABP Trials ABDD Unmet Need Long-Term Opioid Data Central IRB, Central IRB Adv DMCs Informed Consent Pregnancy Testing IND Safety, IND Safety Adv SAE Reporting

COLLABORATIONS PORTFOLIO Areas of Strategic Focus: SYSTEMATIC EVIDENCE GENERATION PATIENTS AS EQUAL PARTNERS EFFICIENT & QUALITY TRIALS PUBLIC HEALTH CONCERN SAFE & ETHICAL TRIALS Active Collaborations: Sentinel IMPACT-AFib Patient Engagement Collaborative ABDD PTN Complete Collaborations: Clinical Trials for Comparative Effectiveness Electronic Healthcare Data Patient Engagement Survey Clinical Trials Poll FDA Training Course Cardiovascular Endpoints

Road to Impact CTTI Recommendations, Resources, & Findings Awareness Adoption Impact

CTTI Recommendations & Tools Streamline Antibacterial Pediatric and HABP/VABP Trials Organize DMCs to ensure patients safety Move Recruitment planning upstream to reduce barriers to participation Develop a better IND Safety Reporting system Perform higher quality Informed Consent process Involve Patient Groups as equal partners Apply Quality by Design (QbD) principles to create better protocols Improve ethics review process via use of Central IRB Reduce inefficiencies of investigator GCP Training Use Registries to conduct more efficient clinical trials Identify the best pathways for developing Novel Endpoints generated by mobile technologies Create Pregnancy Testing plans for improved clinical trials Strengthen the Site Investigator Community

Engage with CTTI Today Participate in a project or driving adoption activity Attend an upcoming CTTI webinar Implement CTTI recommendations & resources Share recommendations with others Stay informed: Follow CTTI on Twitter and LinkedIn; share our news with your followers & friends Sign up for CTTI s monthly e-newsletter

THANK YOU. #CelebrateCTTI @CTTI_Trials www.ctti-clinicaltrials.org