1. P atients’ E xperiences in E mergency R esearch Neal Dickert, MD, PhD Emory Program in Cardiovascular Outcomes Research and Epidemiology (EPICORE) Department of Medicine, Division of Cardiology

2. Study Partners PI- Rebecca Pentz Sponsor- NINDS Site Co-Investigators Michelle Biros Jill Baren Prasha Govindarajan Art Pancioli Emory NETT team NETT/RAMPART Leadership

3. EFIC Research- Ethical Challenges Well-known and numerous Interpreting “unsatisfactory and unproven.” What is the goal of community consultation and how can that be achieved? Many of the toughest challenges are only magnified by the absence of consent EFIC research exposes an over-reliance on consent in general

4. The PolyHeme Experience “Traumas Create Unwitting Test Subjects” USA Today 2006 “ makes the inhabitants of 32 communities in 18 states, and anyone living or traveling near these communities, potential ‘guinea pigs’ without their consent and, absent consent, without full awareness of the risks and benefits.” Senator Charles Grassley, 2006

5. Patients’ Perceptions- Need for Data Good reasons to care about the views of enrolled patients. Conceptual- problematic if patients feel exploited/violated. Practical- may offer ways to improve the experience Existing data sources are inadequate Community consultation General Public Patients with similar conditions

6. EFIC Research- Public Views Surveys of general public Often patients in ED waiting rooms Less than half supportive of EFIC research Greater willingness (70%) to be included personally, particularly if risks are minimal McClure et al, Acad Emer Med, 2003 “Protocolization” of treatment may have negative effect. Abboud et al, J Med Ethics, 2006 Reports from community consultation Generally positive Greater willingness to “approve” the study in the community (82%) vs acceptance of being enrolled (64%) Longfield et al. Crit Care Med. 2008

7. EFIC Research- Public Views Methodologic challenges Responses highly sensitive to wording of questions Unclear whether participants understand Complicated, unfamiliar topic- no pre-formed opinions Surveys don’t allow clarification Nature of objections unclear Not the views of people actually included Attempts to target people at risk, to use non-closed-ended interview techniques.

8. EFIC Research- SCD Patients 19 survivors of Sudden Cardiac Death In-depth interviews Greater acceptance than previous surveys. Nobody said EFIC is unacceptable. Emphasis on risk/benefit Comparisons of existing treatment widely accepted Greatest concern about new interventions Studies posing some risk w/o prospect of benefit. Dickert and Kass, Soc Sci Med, 2009

9. Resuscitation Research- SCD Patients Case Description ARUN 1) New intervention for treatment of SCD87116 2) RCT- New Intervention v. std for SCD74213 3) RCT- 2 existing treatments for SCD 7 1012* 4) RCT- Blood substitute v. saline for shock 13 1119* 5) Public Access Defibrillator (PAD) Study 10 0113** 6) No prospect of direct benefit- blood draw 18 00 7) No prospect of direct benefit- contrast posing 1/10,000 chance of death 12 3217 *4 participants’ understanding insufficient. **2 participants’ understanding insufficient.

10. The PEER Study- Aims Assess views of enrolled patients regarding acceptability of EFIC and RAMPART enrollment Provide baseline/benchmark data Identify patient and study factors that predict satisfaction/acceptance: Conversations w research staff, understanding of research, prior experience, demographics Risk level, randomization, alternatives Assess whether Community Consultation elicits concerns of enrollees. Will link to RAMPART CC research.

11. The PEER Study- Methods Structured, interactive interviews In-person or telephone Series of open-ended questions followed by closed-ended questions Facilitates quantitative and qualitative aims Allows participants space to develop views Allows insight into participants’ understanding of questions/issues

12. The PEER Study- Domains Prior research experience & attitude toward research Knowledge of the RAMPART study Views on having been included in RAMPART Views on acceptability of EFIC in research- in RAMPART and in general Views on randomization Views on conversations with investigators/study staff Views on community consultation Impact of public disclosure Trust in researchers and in clinical research Demographics and medical background

13. The PEER Study Participating Sites 5 sites- UCSF, UC, Emory, UM, and UPenn 1 trained interviewer per site Sample English or Spanish speaking RAMPART enrollees or surrogate/LAR 20 subjects per site (n=100)

14. The PEER Study- Status IRB-approved at all participating sites Interview guide pre-tested at Emory Interviewer training in progress All sites to begin formal enrollment shortly Key challenge- Successful re-contact

15. The PEER Study- PROTECT Phase Sponsor- Greenwall Foundation Similar in scope and design Target= 100 participants (patients and decision-makers) Includes anyone initially enrolled using EFIC Target interview time = 3 months post- enrollment Key differences Interviews conducted by central firm (APCO Insight) All NETT sites invited to participate by referring participants.

16. PEER Studies- Critical Comparisons RAMPART Comparison of existing interventions Relatively low mortality/morbidity Many patients with pre-morbid illness PROTECT III Placebo-controlled trial of a new intervention High mortality/morbidty Most patients with no pre-morbid illness

17. Summary Unique opportunity to gather important data on patients’ experiences in EFIC research Ability to connect with existing work on CC Addition of PROTECT Phase offers important cross-study comparative data Illustrates commitment of NETT Improving understanding of ethical challenges Implementing data-driven approaches