Presentation by Craig Lipset at Precision Medicine World Congress (Palo Alto CA, 24 January 2020). This presentation shares a "top 10 list" of places where patient participation in research is facing radical change for the better.

1. Craig H Lipset
@craiglipset
linkedin.com/in/lipset
Evolution in the Role of Patient as
Participant in Clinical Research
Confidential: Clinical Innovation Partners LLC
24 January 2020

2. My Affiliations
Confidential: Clinical Innovation Partners LLC
Managing Partner, Clinical Innovation Partners LLC
Board of Directors, People Centered Research Foundation (PCORnet)
Board of Directors, Foundation for Sarcoidosis Research
Board of Directors, MedStar Health Research Institute
Board of Directors, Circuit Clinical / TrialScout
Venture Partner, Boston Millennia Partners

3. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

4. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

5. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

6. 2011
Drug trial (tolterodine tartrate)
Fully remote/virtual
REMOTE Trial
REMOTE:
Research on Electronic Monitoring of
OAB Treatment Experience
Safety & efficacy of Detrol LA
for overactive bladder
600 patients from 10 states
California, Texas, Illinois, Florida,
Pennsylvania, Michigan, Georgia,
North Carolina, New Jersey & Virginia
2 lab assessments
5 mobile based e-diary
assessments
© Clinical Innovation Partners LLC 2019

7. 2014
REMOTE Trial
© Clinical Innovation Partners LLC 2019

8. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

9. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

10. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

11. Research Around the Patient
© Clinical Innovation Partners LLC 2019
Master
Protocols
All-Comer
Big-Data
Observational
Studies
One app for multiple studies
One set of tasks across all studies
Access and share health data across studies
Apple Research app

12. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

13. Stakeholder Expectations
© Clinical Innovation Partners LLC 2019
 90% of patients want to know the results of their clinical trials(1)
 93% of patients never receive the results from the study staff or sponsor(1)
 98% of study staff would like to provide results to study participants(2)
 Recruitment and retention rates continue to worsen; public and patient trust in the research
enterprise has eroded sharply
 Declaration of Helsinki obligates sponsors and research professionals to provide study
results (2008; Guideline 33)
 FDAAA 2007 results disclosure required on CT.gov
 March 2001 Informed Consent Reform
 Test Act of 2012 (H.R. 6272)
 EMA Requiring Return of Study Results to Patients
Patients
Research
Professionals
Regulatory
Agencies /
Policymakers
Source: (1) Shalowitz, D. Miller, F. Communicating the Results of Clinical Research to Participants: Attitudes, Practices and Future Directions. PLoS Medicine 2008;5:714-720;
(2)Dixon-Woods, M. Jacson, C. Windridge, K., Kenyon, S. Receiving a Summary of the Results of a Trial: Qualitative Study of Participant Views. British Medical Journal 2006; 332: 206-210.

14. Returning Data
© Clinical Innovation Partners LLC 2019
Source: Multi-Regional Clinical Trial (MRCT) Center Return of Individual Results to Participants Recommendations Document, 2017

15. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

16. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

17. Confidential: Clinical Innovation Partners LLC

18. Confidential: Clinical Innovation Partners LLC
Trial Design Input from physician KOLs Inconsistent patient input & insights Study co-development
Trial Awareness Newspaper, radio & TV Social media & community Clinical research as a care option
Trial Access Patient travels to the site Some use of digital/video for remote
visits
Location-flexibility allowing patient choice
Participant
Support
Access to study coordinator Incremental support programs (ex:
travel)
Digital concierge support
(“Hey Siri…”)
Endpoints Scientific instruments Increasingly patient-focused Digital, passive,
patient-centered
Centricity Patient in a trial Patient in a trial Research around the patient
Deliverables to
Patients
None Still limited studies sharing patient-
friendly summaries of results
Access to results and individual data
from trial
Registries Physician manual-entry Some physician-entered,
some patient-entered,
Some EHR-sourced
Patient mediated access to rich
electronic health data
RWD Aggregated deidentified data for
epidemiology and study planning
Aggregated deidentified data used for
regulatory submissions
Permissioned data used with patient
awareness & engagement
Compassionate
Use
Burdensome request & access
process
Burdensome request & access
process
Exchange access for outcomes data
10 Points of Evolution in Research Participation
Yesterday Today Tomorrow

19. Patients in full control of their
PERSONAL HEALTH DATA
will be a
KEY DRIVER
of the future of
medicine development.
© Clinical Innovation Partners LLC 2019

20. We must ensure that
PRECISION MEDICINE and
PATIENT-CENTRICITY do not
pull in opposite directions in a
SURVEILLANCE ECONOMY
© Clinical Innovation Partners LLC 2019

21. Craig H Lipset
@craiglipset
linkedin.com/in/lipset
Evolution in the Role of Patient as
Participant in Clinical Research
Confidential: Clinical Innovation Partners LLC
24 January 2020