Building Trust, Engaging Communities and Disseminating Results Ken Getz, Associate Professor and Director, Tufts CSDD Tufts University School of Medicine Founder and Chairman, CISCRP September 2016

Key Takeaways Each individual’s health journey begins before she or he is a patient The journey is dynamic and unique to the individual Patients want high-touch while embracing high tech Patient engagement is more than partnering with the patient alone The patient sees, and relies on the clinical research enterprise as a coordinated and open ‘system of care’ One bad clinical trial experience or questionable company practice reflects poorly on the entire clinical research enterprise

Engagement Friction Points Abrupt Termination Limited Transparency Past Participant Public Prospective Volunteer Study Volunteer Limited Connection Poor Relevance Participation burden Limited advocacy Limited HCP Facilitation Inconsistent & poor access

Public Willingness and Self-Reported General Knowledge of Clinical Research Source: CISCRP 2015; N=8,857 Who Have Never Participated in a Clinical Trial

Connection to the Clinical Research Enterprise Percent who believe they know where clinical research is conducted Percent who can name A living scientist Source: Research!America, 2010; N=1,000 – 2,000 Adults

Who makes a greater contribution to human health? Appreciation for Study Volunteer Contribution Who makes a greater contribution to human health? Source: CISCRP 2015; N=12,009

Top Perceived Risks and Benefits Which ONE of the following do you consider to be the greatest risk/benefit of participating in a clinical research study? (Top 5 Selected) BENEFITS RISKS Source: CISCRP 2015; N=12,009

Engagement Friction Points Past Participant Public Prospective Volunteer Study Volunteer Limited HCP Facilitation Inconsistent & poor access

Trusted Sources for Clinical Research Information 62% of patients rate their HCP as the top preferred source for information about clinical research 71% of patients say that they would speak with their physician prior to deciding to participate 83% of patients consider their physician’s recommendation a top factor influencing their decision to participate Percent of Total Source: Gallup Survey, 2014 (N=6,369 Americans) Source: CISCRP 2015 P&I study (N=12,009 Global Respondents)

Preferred and Actual Sources for Information about Clinical Research Studies Percent of Total Source: CISCRP 2015; N=12,009

Health Care Provider Experience and Comfort Level Making Referrals MDs (N=755) Nurses (N=1,255) Received special training on CT in school 40% 45% Attended lecture(s) on CT at society meetings 39% 21% Consider themselves familiar (SW/Very) with CTs 88% 69% Comfortable (SW/Very) providing CT info to patients 63% Comfortable (SW/Very) discussing CT opportunities with patients 91% 72% Have referred their patients to clinical trials 60% 17% Source: Tufts CSDD 2015

Health Care Provider Referral Behavior Physicians (N=465) Nurses (N=1,112) Percent of my patients who have asked to be referred 7% 1% Median number of patients referred annually 5 2 Median number of patients seen annually 3,100 5,560 Source: Tufts CSDD, 2015

Losing Ineligible Prospective Volunteers What did you do after finding out that you did not qualify for a clinical research study? Source: CISCRP 2015. Volunteers Who Wanted to Participate But Did Not Qualify (N=2,647)

Engagement Friction Points Past Participant Public Prospective Volunteer Study Volunteer Participation burden Limited advocacy

Complex and Demanding Protocols (Means) A Typical Phase III Protocol 2001 - 2005 2011-2015 Scientific Total Number of Endpoints 7 13 Total Number of Eligibility Criteria 31 50 Total Number of Procedures 110 187 Total Number of Procedures per visit 10 Proportion of Procedures that are ‘Non Core’ 18% 31% Operating Median Number of Countries* 4.7 9.5 Median Number of Investigative sites 124 196 Median Number of Patients Randomized 729 597 Total number of data points collected** 494,236 929,203 Source: Tufts CSDD ; *DRI; **Medidata Solutions

Study Enrollment Challenges Doubling Planned Timelines Enrollment Achievement Rates Increase in Planned Study Duration to Reach Target Enrollment Overall 94% Cardiovascular 99% CNS 116% Endocrine/Metabolic 113% Oncology 71% Respiratory 95% 1.9 Study timelines are typically extended to nearly double their original duration to meet desired enrollment levels for all therapeutic areas. Less than half of all patients screened complete clinical trials overall though there is wide variation by therapeutic area with endocrine studies having the lowest trial completion rates (34% of all patients screened). Oncology studies tend to have shorter timeline extensions to achieve desired enrollment levels compared to endocrine and CNS studies with the longest average study duration extensions. Source: Tufts CSDD, 2013

Most and Least Liked Clinical Trial Elements MOST LIKED Percent of Total LEAST LIKED Helping advance the treatment of disease 35% Not knowing whether I was getting the investigational treatment 30% Helping others who may suffer from my disease 28% Location of the research center 22% Compensation that I received 25% Study visits too time consuming 19% Care and attention from study staff 21% Compensation not enough given the demands of the study 16% Information I learned about my disease 17% Study procedures too cumbersome 15% Source: CISCRP, 2015: N= 2,849 Patients Who Completed Participation

Positive Volunteer Experiences Overall Source: CISCRP, 2015: N= 2,849 Patients Who Completed Participation

Engagement Friction Points Abrupt Termination Limited Transparency Past Participant Public Prospective Volunteer Study Volunteer

Information Shared Post-Participation What information would you be most interested in receiving after completing your study? What information did you receive after completing your study? Percentage that Report Receiving Summary of my Study Results 9% Whether I received a placebo or the study intervention 4% News about my study intervention being approved 7% CISCRP 2014 Study N=1,072 Source: CISCRP, 2015: N= 2,849 Patients Who Completed Participation

The Impact of Familiarity Non-Participants Past Participant Percent very willing to participate (again) 30% 68% Percent very confident in finding an (another) appropriate clinical trial 18% 46% Percent very comfortable speaking about clinical trials with others 39% 81% Source: CISCRP, 2015: N=8,857 non-participants and 3,152 past participants

CISCRP’s Model of Engagement CONNECT Community of Participants and Ambassadors EDUCATE General Education and Awareness ENABLE Access, Input, Network of Support Affiliation initiatives/Alumni Programs Post Trial Plain Language Communication Post Trial assessment Volunteer Ambassadors Global public and patient assessment Site/CTSA Support HC Provider Education and outreach Pharmacy-directed education Patient and care network boards Medical Community Outreach Search Clinical Trials ENGAGEMENT AWARE for All ‘Medical Heroes’ PSA Media Outreach Science Museum Exhibit Educational Outreach Speakers Bureau Legislation Gift of Participation CISCRP.ORG

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Thank You! Ken Getz Director, Sponsored Research Programs, Associate Professor Tufts CSDD, Tufts School of Medicine 617-636-3487, Kenneth.getz@tufts.edu