CTTI OverviewOne Decade of Impact. One Vision Ahead.

Pamela Tenaerts, MD, MBAExecutive Director

Feb. 6, 2018

#CelebrateCTTIDiscover 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 PartnershipCo-founded by Duke University & FDA Involves all stakeholders

BetterStreamlined

Fit for purposeClinical Trials

Everyone must have a seat at the

table

ClinicalInvestigators

Industry (pharma,

bio, device, CRO, &

tech)

Academia

IRBsTrade &

Professional Orgs

Government &

Regulatory Agencies

Patients, Caregivers & PatientAdvocacy Groups

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TRANSFORMING CLINICAL TRIALS Multi-Stakeholder

RESEARCH METHODS

STAKEHOLDER INTERVIEWS

FOCUS GROUP DISCUSSIONS

SURVEYS

SYSTEMATIC LITERATURE REVIEWS

EXPERT MEETINGS

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.

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TRANSFORMING CLINICAL TRIALS Evidence-Based2

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TRANSFORMING CLINICAL TRIALS

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.

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

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TRANSFORMING CLINICAL TRIALS Real-World Impact at the Policy Level

CTTI Organization

• Oversight and strategic direction

Executive Committee

• Input into strategy and project selection• Conducts projects and develops strategies for

implementation

Steering Committee

• Project support and organization in pursuit of mission

Staff

CTTI Membership

*Version: Jan. 29, 2018

Patient Engagement EvolutionPatient 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

To identify practices that through broad adoption will increase the quality and efficiency of clinical trials

To identify and promote practices that will increase…

To develop and drive adoption of practices that will increase…

2008

2013

2016

CTTI Projects

CTTI Methodology

Areas of StrategicFocus:

SYSTEMATIC EVIDENCE

GENERATION

PATIENTS AS EQUAL PARTNERS

EFFICIENT & QUALITY TRIALS

PUBLIC HEALTH

CONCERN

SAFE & ETHICAL TRIALS

Active Projects:

MCT Legal & Regulatory

MCT Mobile Devices

MCT Stakeholder Perceptions

Real World Evidence

State of Clinical Trials

Patient Groups & Clinical Trials

Investigator Qualification

ABDD HABP/VABP Studies

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

PROJECT PORTFOLIO

Areas of StrategicFocus:

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

COLLABORATIONS PORTFOLIO

Road to Impact

CTTI Recommendations,

Resources, & Findings

Impact

Awareness

Adoption

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 Endpointsgenerated 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

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your followers & friends Sign up for CTTI’s monthly e-newsletter

www.ctti-clinicaltrials.org

THANK YOU.

#CelebrateCTTI@CTTI_Trials