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The "P" in PCOR: IRB Oversight and

the Role of the Patient

July 16, 2015

1:00-2:30 PM ET

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Jeremy Block, PhD, MPP

Icahn School of Medicine at Mount Sinai

Jeremy Corsmo, MPH, CIP, CHRC

Cincinnati Children’s Hospital Medical Center

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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

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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.

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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

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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.

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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.

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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

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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

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Impact of Multiple Roles

Patients as advisors or disease advocates

vs.

Patients as members of research team

(patient-researcher)

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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)

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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

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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

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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

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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

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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

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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.

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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

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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?

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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

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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?

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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

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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

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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

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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

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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

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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

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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

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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

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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.

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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

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Example: Apple ResearchKit

(researchkit.org)

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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

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Example: e-NABLE prosthetic limbs

A Global Network Of Passionate Volunteers Using 3D

Printing To Give The World A "Helping Hand”

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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.

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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.

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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

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This webinar is sponsored by:

The industry-leading independent IRB

in customer service and technology.

Learn more at www.sairb.com

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Questions and comments

To submit a question,

simply click on the Q & A menu

at the top of the screen.

webinars@primr.org

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Disclaimer

Please note that the presentations and

views are those of the individual speakers,

and do not represent the organizational

views of PRIM&R.

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Thank you!

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