the p in pcor: irb oversight and the role of the patient€¦ · 16/7/2015 · o a patient that is...
TRANSCRIPT
7/15/2015
1
The "P" in PCOR: IRB Oversight and
the Role of the Patient
July 16, 2015
1:00-2:30 PM ET
7/15/2015
2
Jeremy Block, PhD, MPP
Icahn School of Medicine at Mount Sinai
Jeremy Corsmo, MPH, CIP, CHRC
Cincinnati Children’s Hospital Medical Center
7/15/2015
3
Webinar Objectives
Following this presentation you will:
Have an improved understanding of patient
involvement/roles in Patient Centered Outcomes
Research (PCOR)
Manage patient expectations about getting feedback from
physicians and researchers and obtaining access to
personal data gathered during PCOR
Have information to facilitate better interactions with
patients involved in PCOR
7/15/2015
4
What is PCOR?
Patient-Centered Outcomes Research, or PCOR, is an
approach to traditional comparative effectiveness research
(CER) focused on addressing the questions and concerns
most relevant to patients, and typically involves patients,
caregivers, clinicians, and other healthcare stakeholders,
along with researchers, throughout the entire research
process from identification of research questions, to study
design, execution and dissemination of results.
These collaborations are challenging many of the long-
accepted traditional norms of roles/responsibilities and the
boundaries between research and clinical care.
7/15/2015
5
What is PCOR?
Patient-Centered Outcomes Research, or PCOR, is an
approach to traditional comparative effectiveness research
(CER) focused on addressing the questions and concerns
most relevant to patients, and typically involves patients,
caregivers, clinicians, and other healthcare
stakeholders, along with researchers, throughout the
entire research process from identification of research
questions, to study design, execution and
dissemination of results.
These collaborations are challenging many of the long-
accepted traditional norms of roles/responsibilities and the
boundaries between research and clinical care.
7/15/2015
6
What is PCOR?
Patient-Centered Outcomes Research, or PCOR, is an
approach to traditional comparative effectiveness research
(CER) focused on addressing the questions and concerns
most relevant to patients, and typically involves patients,
caregivers, clinicians, and other healthcare stakeholders,
along with researchers, throughout the entire research
process from identification of research questions, to study
design, execution and dissemination of results.
These collaborations are challenging many of the long-
accepted traditional norms of roles/responsibilities and
the boundaries between research and clinical care.
7/15/2015
7
Background Statement
As patients assume new roles as “patients,”
“patient-researchers,” and “research participants,”
IRBs struggle to define the boundaries of their
responsibility around human subjects protections
and oversight of investigators and researchers.
7/15/2015
8
Defining the Roles
For the purpose of this presentation…
Patiento Person with a certain medical condition, at risk for the medical
condition or connected to the person with the medical condition
(e.g. parent, care giver, etc.)
o May be a recipient of medical care or advocate for medical care
Patient-Researchero A patient that is an active co-equal member of the a research team
o May contribute to some or all of study design, data collection,
analysis, consenting, etc.
Research Participanto A person participating in a research study as the subject
of the research
o Also may apply conventional FDA/OHRP regulatory definitions
7/15/2015
9
PCOR Related Challenges for IRBs
Understanding which patient roles are involved
by a given PCOR project
Ensuring that participants are properly informed
when they are filling multiple roles
Managing participant expectations regarding
access to data
Managing novel and innovative methods of data
collection
7/15/2015
10
Impact of Multiple Roles
Patients as advisors or disease advocates
vs.
Patients as members of research team
(patient-researcher)
7/15/2015
11
Patients in Advocacy Roles
Not new…
o Clearly emphasized in PCOR but not new…
Important to differentiate patients advising or
advocating to researchers in contrast to patients
being active co-equal members of research team
(patient-researcher)
7/15/2015
12
Patients in Advocacy Roles
Patients (or communities) advising/guiding
research programs or advocating for research in
certain disease areas is not new.
o Community Based Participatory Research (CBPR)
programs are usually guided by community advisors
o Disease advocacy groups (e.g., American Heart
Association) are made up of patients and family
members focused on a specific disease
7/15/2015
13
Patients in Advocacy Roles
No new or special challenges with PCOR
associated with patients serving in
advocacy roles
IRBs should not be overly concerned about
potential research participants also being in
advisory or advocacy roles
7/15/2015
14
The Patient-Researcher
Patients as members of research teams
(patient-researcher)
Grants often include patient(s) in this role without the
research team thinking through the logistics
Researcher tend to struggle with the patient in the
patient-researcher role more than the patient does
7/15/2015
15
The Patient-Researcher
Patient-researcher is a active, contributing
member of the research team
IRBs need to determine:
o Whether the patient is a patient-researcher or in an
advocacy/advisory role
o If patient-researcher, whether they are engaged in
research such that they need to be on the IRB-
reviewed research team
7/15/2015
16
Is the patient-researcher a member
of the IRB-reviewed research team?
Questions to ask…
Will the patient-researcher have access
to study data?
o Contribute to analysis
o Evaluate study end points
o Directly influence study design
7/15/2015
17
Is the patient-researcher a member
of the IRB-reviewed research team?
Questions to ask…
Will the patient-researcher interact with research
participants?
o In an educational role
o In a recruitment capacity
o Obtaining consent
o Facilitate focus groups/interviews (data collection)
More natural role of the patient-researcher.
7/15/2015
18
How should IRBs handle the patient-
researcher during IRB review?
Do not automatically apply a different standard
to patient-researcher vs. other researchers
Flexibility may be needed:
o Human subjects training: modality/context may
need to be modified
o Conflicts of interest: areas of potential bias or conflict
may be different; assessments may need to refined
7/15/2015
19
The research protocol
Protocol should describe the role of the
patient-researcher
It is common to publish/report on the experience
of patient-researcher
o If applicable, IRBs should understand this plan
o Determine whether it is an aim of the protocol, a
separate protocol, what data is being collected and
how, and whether consent is needed
How should IRBs handle the patient-
researcher during IRB review?
7/15/2015
20
The Multiple Roles of the Patient
What if the patient-researcher will also be a
research participant?
Patients that want to be part of research teams likely
also have strong interest in participating in research
o Not likely to be aware of traditional firewalls between
researcher and research participant
o May not understand the potential for bias and how
that might impact the overall research
7/15/2015
21
The Multiple Roles of the Patient
What should IRBs consider?
May not be appropriate for a patient-researcher
to also be a research participant in all cases
IRBs should consider:
o Is the protocol vulnerable to bias?
• Are there a lot of subjective data collection measures?
(QOL, preference scores, etc.)
• Are the subjective measures central to the primary aims?
• How many participants will be enrolled?
7/15/2015
22
The Multiple Roles of the Patient
What should IRBs consider?
Are certain aspects (aims) of the protocol better
suited to allowing the patient-researcher to also
be a research participant?
o Protocol says the patient-researcher will facilitate focus groups or
interviews… probably cannot also be a participant
o Large observation studies and/or studies with objective
data collection or using medical record data are probably
best suited for the patient-researcher to also be a
research participant
7/15/2015
23
The Multiple Roles of the Patient
What should IRBs consider?
IRB may have existing policies that restricting
members of research teams from participating
in their own studies
Need to revisit these policies within the context of
PCOR as well as other types of research
(e.g., CBPR) that are likely to have patients
or others serving in multiple roles
7/15/2015
24
The Multiple Roles of the Patient
What should IRBs consider?
Informed consent: consent may need to be
supplemented for a research participant that is
also a patient-researcher
o Increased risk for undue influence.
• Patient-researcher knowledge of slow recruitment or poor
retention may create perceived pressure for them to
participate or continue participating.
o Limitation on access to their own data over the
course of the study
7/15/2015
25
The Multiple Roles of the Patient
What should IRBs consider?
Informed consent: provisions for protecting
confidentiality presented during the consenting
process may need to be modified
o Need to understand that they will have both a research
participant-researcher relationship as well as a collegial
research team member relationship
o The reality of confidentiality will be different given the
research team member relationship
7/15/2015
26
The Multiple Roles of the Patient
Summary: What IRBs should consider
Special attention to elements of consent:
o Voluntariness, right to withdraw, confidentiality, limitation
on access to data
Protocols should contain:
o Plans for the patient-researcher role and provisions for
managing them as a research participant
Research teams (and IRBs) may need additional
education regarding the unique circumstances
associated with the patient-researchers
7/15/2015
27
Managing Patient-Researcher
ExpectationsBecause the role of the patient-researcher is more complex,
there must be a more robust plan!
Areas that commonly come up and merit greater detail in
the submitted protocol include:
Access to data
o Who, what, where, when, how, and for how long
Control over study endpoints
o Clearly indicate in the protocol and guide the subjects through
this, denoting the boundaries of their input
o This is a teaching moment: when investigators disagree,
there is an obligation to explain
7/15/2015
28
Managing Patient-Researcher
Expectations
Because the role of the patient-researcher is more
complex, there must be a more robust plan!
IRBs should encourage investigators to plan specific
meetings or discussions about patient-researcher roles at
different intervals throughout the study
Reinforce an understanding of the role
Guide the transition between patient-researcher
Explain and co-create endpoints
Define and identify boundaries
7/15/2015
29
Patient-Researcher Impact on IRB
Review Process
Expertise, experiences, and perspectives of patients
as researchers
Informed consent
Assessment of risks and benefits
Inform best practices for all phases of study design
and conduct
o This is especially helpful in new areas such as research
involving smartphone apps or gadgets where experience is
more limited
7/15/2015
30
Apps, Gadgets, etc.
Technological advances and development of
applications and gadgets (e.g., Fitbits, Apple
ResearchKit, RedCap, etc.) result in new media
for participant reporting.
Important for investigators to teach the IRB about
the new capabilities and engage in a dialogue
around how to best incorporate them into studies.
7/15/2015
31
Apps, Gadgets, etc.
This is a new area for researchers, so best
practices are still being worked out
An opportunity for PCOR research where the patient-
researchers input on the best ways to design studies
might really be useful not just for the research, but
also for product development
Critically important to be very clear about the
who/what/where/when/why/how of data collection
methods and sharing when using apps and gadgets
7/15/2015
32
Example: Apple ResearchKit
(researchkit.org)
7/15/2015
33
Example: Apple ResearchKit
(researchkit.org)
Asthma Health:
Entire project done without ever seeing a subject
Uses combination of location-based data and user-
reported content
• Accrued 5k+ people in under a month
• “The Block Rule”: at least three different types of
multi-media consenting techniques used
(graphics, interactive Q&A, video etc.)
• Data accessible to user
7/15/2015
34
Example: e-NABLE prosthetic limbs
A Global Network Of Passionate Volunteers Using 3D
Printing To Give The World A "Helping Hand”
7/15/2015
35
Example: e-NABLE FAQ Tidbit
Is it medically tested and approved? (from www.enablingthefuture.org)
Our current designs have been approved for testing through Creighton
University. We recommend careful observation while using these devices that
involve your family physician’s input and guidance.
If any redness or discomfort occurs, please stop using the device you have
created, until you can consult a licensed medical professional who can make an
informed decision whether the device is appropriate for you and possibly help
you improve the fit of your device with medical orthoplastic or padding.
This need for observation is why we discourage the use of these devices with
exceptionally young children (<4) Young children do not always express when
they are uncomfortable, so the possibility of injury is higher.
Please do not use these devices without consulting a physician prior to use and
consult with them as to the best fit and use for you or the person you have
created it for.
7/15/2015
36
Example: e-NABLE Research Study
Online information only denotes one study, conducted under
IRB approval at Creightonwww.enablingthefuture.org/2014/03/10/research-volunteers-needed/
The various volunteers are designing this, implementing it,
and distributing the devices as around the world in an open-
source fashion.
As far as can be ascertained from the website, none of the devices are FDA approved.
7/15/2015
37
Protocol Recommendations
Explicitly define the roles, timelines for those roles,
and how the team will manage/guide the transition
between roles
Describe the decision-making process concerning
study endpoints
Describe where and how patient-researcher
input will be used
Describe in tabular and graphical form where
different data is to be collected, how, by whom/what,
and how it will be shared/stored
7/15/2015
38
This webinar is sponsored by:
The industry-leading independent IRB
in customer service and technology.
Learn more at www.sairb.com
7/15/2015
39
Questions and comments
To submit a question,
simply click on the Q & A menu
at the top of the screen.
7/15/2015
40
Disclaimer
Please note that the presentations and
views are those of the individual speakers,
and do not represent the organizational
views of PRIM&R.
7/15/2015
41
Thank you!
Please complete the evaluation