1. Rethinking recruitment and retention through patient engagement Saturday, March 2 Petra Kaufmann, M.D. Director, Office of Clinical Research National Institute of Neurological Disorders and Stroke/NIH American Society for Experimental NeuroTherapeutics | 15 th Annual Meeting

2. Disclosure Name of Commercial Interest none American Society for Experimental NeuroTherapeutics | 15th Annual Meeting Type of Financial Relationship none Full-time employee of the NIH

3. Learning Objectives Better understand barriers to recruitment and retention Examine the opportunities and challenges of engaging patients more fully in the trial enterprise American Society for Experimental NeuroTherapeutics | 15th Annual Meeting

4. Crisis in US Trials Clinical trials are challenging because of increasingly high cost and lengthy delays Many trials are now being conducted in other countries Many factors contribute to this crisis, but one of the greatest challenges is low rates of enrollment of people in clinical trials Weisfeld N, English R, Claiborne A. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020. Washington, DC: Institute of Medicine of the National Academies, The National Academies Press; 2012. (Rapporteurs) Workshop Summary. www.nap.edu.www.nap.edu.

5. IOM Workshop “Envisioning a transformed clinical trials enterprise in the US” Lack of awareness of the benefits of engaging in trials Lack of availability of relevant trials to participate in Clinical trials designed without patient input and therefore lacking acceptability Patient preferences for one treatment over another, or the availability of a treatment outside of the trial setting Logistic hassles in participation (such as frequent visits, lab tests, questionnaires) Inadequate reimbursement (Medicare has paid for routine care in trials since 2000, but private payers may not) Weisfeld N, English R, Claiborne A. Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020. Washington, DC: Institute of Medicine of the National Academies, The National Academies Press; 2012. (Rapporteurs) Workshop Summary. www.nap.edu., pp 38-39www.nap.edu., pp 38-39

6. © 2013 The Neuropathy Association, Inc. N=539 ©2010 The Neuropathy Association, Inc. With permission from Tina Tockarshewsky President and CEO The Neuropathy Association The patient perspective Example: Neuropathy

7. © 2013 The Neuropathy Association, Inc. N=539 ©2010 The Neuropathy Association, Inc.

8. © 2013 The Neuropathy Association, Inc. Barriers to Participation: Access Lack physical, financial, support strengths to participate: Mobility: because of neuropathy/neuropathy feet, they are not mobile, cannot drive, walking is a problem, so can’t go/travel too far; they are dependent on others or on public transportation to get to locations…and often cannot depend on these resources. “Travel is difficult for me on the best of days.” Do not have physical/financial/personal support to travel any great distance. “Afraid of traveling alone” Access to trials are primarily in major urban areas, difficult for patients to get to from outside of the immediate urban core.

9. © 2013 The Neuropathy Association, Inc. Barriers to Participation: Costs Concerns about costs—real/hidden, costs and burden of travel to sites on top of financial burden they already have with neuropathy treatment/management. – Many are already on disability and struggling due to job loss. Personal costs: time away from work, loss of work time for dependents/family due to inability to travel alone. – “Very difficult to leave my work and spend more money during the process;” “Just too poor and have children at home;” “Not enough compensation for the risks we run;” – “I miss too much work now…to be out even more, for a study that may not yield results, is difficult to arrange;” “I run a business, so it’s got to be convenient and cannot take too much time;” “Time away from work/family would be a concern.” With permission from Tina Tockarshewsky

10. Barriers to Participation: Awareness Physicians may lack education or time to be stewards in encouraging patient participation – “I have asked my doctor and he does not say anything about it…he ignores my questions about clinical trials;” “I keep bugging my neurologist about clinical trials, and she just gives me this look and says that she will look into it, but never does” Less than five percent of Americans know where to find information about relevant clinical trials. (Getz, Philadelphia Survey) More than 70% of those who have participated in clinical trials] are likely to do so again. (Sun, "Sometimes Bumpy Road". 2008) With permission from Tina Tockarshewsky The Neuropathy Association The Center for Information & Study on Clinical Research Participation http://www.ciscrp.org/professional/facts_pat.html

11. How can patient engagement help? Awareness – Patients participate in concept development Making sure that the question matters Making sure that the outcome matters Two-way street: patients bring important insight, and take information back to their communities – Patients help develop communications plan Making sure the material is user-friendly Choosing the best strategy (advocacy groups, web, social media, meetings)

12. How can patient engagement help? Logistics and Trial Implementation – Patients participate in protocol development Making sure that the plan is feasible Making sure that the burden is acceptable Planning for reimbursement of expenses Recruitment and retention plan Two-way street: patients bring important insight, and take information back to their communities

13. How can patient engagement help? Safety concerns – Patients participate on DSMBs Making sure that the patient perspective on acceptable risks is heard Increased trust in the trial enterprise if patients are represented

14. NINDS experience Lay members on DSMBs NeuroNEXT (new Phase 2 trials network) has patient representative on protocol working group Challenges – Identifying volunteers – Providing information and training so that patient volunteers feel comfortable communicating in the context of protocol groups or DSMBs

15. Approaches Example: Parkinson’s Disease Parkinson’s Disease Foundation (PDF) Learning Institutes – three-day trainings – preparing people living with Parkinson’s and care partners for work as Research Advocates – Regional model – Patients learn from experts in the field about the science of Parkinson’s and the process that brings new treatments to market – Goals are to gain leadership and advocacy skills to confidently interact with the scientific community DSMB training: NIAID http://www.niaid.nih.gov/LabsAndResources/resources/toolkit/guidance/Documents/DSMB.pdf www.pdf.org

16. Conclusion Greater patient engagement in the trial enterprise can increase Awareness Protocol feasibility Recruitment Retention Patient advocacy groups can support education, training and communications efforts Professional organizations can increase awareness and provide education and training opportunities to clinicians and patients