Individual Assessments of Knowledge of the Expanded Access and Compassionate Use Programs Among Stakeholders

Expanded Access / Compassionate
Use stakeholder focus groups
MODERATOR GUIDE

CONFIDENTIAL

PRA compliance
Control
number and
expiration
date

Public
reporting
burden

OMB control number: 0910-0497
Expiration date: 10/31/2020

Public reporting burden for this collection of information is estimated to be 90 minutes,
including time for reviewing instructions, searching existing data sources, gathering and
maintaining the data needed, and completing and reviewing the collection of information.
Send comments regarding this burden estimate or any other aspect of this collection of
information, including suggestion for reducing burden to [email protected]. An
agency may not conduct or sponsor, and a person is not required to respond to, a
collection of information unless it displays a currently valid OMB control number. The
control number for this project is 0910-0497.

Your participation/nonparticipation is completely voluntary, and your responses will not
have an effect on your eligibility for receipt of any FDA services. In instances where
Voluntary
respondent identity is needed (e.g., for follow-up of non-responders), this information
participation
collection fully complies with all aspects of the Privacy Act and data will be kept private to
the fullest extent allowed by law.

PROPRIETARY

2

Contents

▪ Patient advocacy groups
▪ IRBs
▪ Health system administrators

PROPRIETARY

3

PATIENT ADVOCACY GROUPS

Agenda for patient/patient advocacy focus groups
Topics covered
Welcome and
A moderator
introduction

B

Participant
introduction

Structured discussion
C around potential pain
points

▪
▪
▪
▪
▪
▪
▪

Open discussion on
D successes/challenges
of existing programs

▪
▪
▪

General feedback
E
questions and wrap-up

▪
▪
▪

Purpose of focus group and agenda for the session

Time allocation [min]
5

How/why participants were chosen
Role of moderator (e.g., time management)
Names of participants and their patient advocacy groups

10

Number of members with EA/CU experience

Hypothesized pain points across identification, application, and
treatment

35

Role of advocacy group in EA/CU programs
Additional challenges and successes

25

Suggestions for improvement
Approach to educating potential patients about EA/CU
Likelihood of recommending the program to someone else

15

Other feedback not previously covered
Favorability of right to try legislation
90

Total

PROPRIETARY

4

PATIENT ADVOCACY GROUPS

A Welcome and moderator introduction

Moderator
instructions

This guide will be used to manage time effectively and ensure coverage of certain topics
of conversation. Participants should freely discuss topics presented by the moderator,
rather than following the question/answer cadence of an interview. Due to time
restrictions, it is possible that not every question will be asked, and it is the moderator’s
responsibility to decide when to transition to a new topic. Additionally, the moderator
should adapt questions based on the composition of each group and the group’s prior
responses. “Voting” sections should be completed using Mentimeter, and time estimates
for these questions are included in this guide.

PROPRIETARY

5

PATIENT ADVOCACY GROUPS

A Welcome and moderator introduction
Hi, my name is [fill in name], and I will be leading our discussion today on the FDA’s
Expanded Access and Compassionate Use programs for drugs/biologics and devices,
respectively.
I’d like to start by thanking each of you for your participation in this session, which will last
90 minutes. The input from this session will be reported back to FDA in aggregate to help
improve EA/CU programs, but all responses will be anonymized. This session will not be
recorded in any form, but I will take notes, as will [fill in name] in the back of the room.
Each of you represents a patient advocacy group with some degree of experience with
Moderator
introduction EA/CU. During this session, I will pose questions to the group and allow time for open
script
discussion. There are no right or wrong answers, and I would encourage everyone to
share their honest opinions.
To start the conversation, I’ll give everyone a chance to introduce themselves. Then, we’ll
transition to discussing specific challenges and successes with the existing EA/CU
programs. Lastly, I’ll leave time for general feedback not covered earlier in the session.
As moderator, it is my job to actively manage the time and make sure that we cover all of
our desired topics. Please do not be alarmed if I stop an on-going discussion in order to
move to a new topic.

PROPRIETARY

6

PATIENT ADVOCACY GROUPS

B Participant introduction
Prioritized questions in bold

To begin with, in order to facilitate good dialogue amongst ourselves, can everyone please
Participant
say their name, the group they represent, and how they first learned about EA/CU
introduction
programs? Wait for everyone to introduce themselves. Allow no more than one minute per
script
participant.
Thank you for those introductions. Now, I’ll ask you to use the iPad in front of you to
answer a question about your patient advocacy group’s experience with EA/CU programs.
Participant
introduction I’ll give everyone a minute to respond to the question, and please let me know if you’re
having any difficulty with the device.
script
Wait one minute.
1) How many members of your patient advocacy group or community have sought
or are seeking access to treatment through the FDA’s Expanded Access (EA) or
Compassionate Use (CU) programs?
None
1-2
Demographi
cs (voting)

3-5
6-8
>8
N/A
Don’t know

PROPRIETARY

7

PATIENT ADVOCACY GROUPS

C Structured discussion around potential pain points
Prioritized questions in bold

Pain point
script

In this part of the conversation, we’ll break the EA/CU application process into 3 parts. In
the first, identification, an EA/CU program is deemed appropriate for a patient by their
doctor, and the patient must decide whether to proceed. I’ll start by asking:
1) What do you think about EA/CU programs overall, and what do you tell members
of your patient advocacy group or community about them?
2) Do you connect members of your patient advocacy group or community to resources to
better educate them about these programs?

Identification

3) What would members of your patient advocacy group or community consider in
deciding whether to seek treatment through an EA/CU program, and who would they
consult for guidance?
4) What main challenges would be encountered in making this decision? Can give
examples if responses are going in a different direction than desired: high stress,
short timeline, lack of information, finding investigational products

PROPRIETARY

8

PATIENT ADVOCACY GROUPS

C Structured discussion around potential pain points
Prioritized questions in bold

Pain point
script

Next, we’ll talk about the application process, in which access to an investigational drug,
biologic, or device is sought from a manufacturer and documentation is submitted to
regulatory bodies. To begin with, I’d like to know:
5) How well have members of your patient advocacy group or community
understood the EA/CU application process, who needs to approve their requests
in order to begin treatment, which forms are required, and where these forms
should be submitted?

Application

6) Do you communicate directly with manufacturers, healthcare providers, IRBs, health
insurance companies, or the FDA on behalf of members of your patient advocacy group
or community? What causes you to engage with these parties, and how easy or
challenging are these interactions?
7) What other roles, if any, does your group play in the application process?
8) On average, how satisfied are members of your patient advocacy group or
community with the EA/CU application process?

Pain point
script

Lastly, we’ll talk about the treatment of patients under EA/CU programs. We are especially
interested in understanding how much financial hardship patients endure as a result of
treatment through EA/CU programs. To begin with:
9) To your knowledge, have members of your patient advocacy group or community
needed to pay out of pocket for treatment under EA/CU programs?

Treatment

10) In general, how challenging is it for members of your patient advocacy group or
community to determine whether health insurance covers some or all of their EA/CU
treatment? Does your patient advocacy group ever communicate directly with health
insurance companies on behalf of your members?
PROPRIETARY

9

PATIENT ADVOCACY GROUPS

D Open discussion on successes/challenges of existing programs
Prioritized questions in bold

Open
discussion
script

Now that we’ve covered specific difficulties associated with existing EA/CU programs, I’d
like to ask the group some more open-ended questions to fill in any gaps that we may
have missed. As time allows, ask participants to brainstorm answers to each question for
one minute separately before discussing as a group.

Challenges

1) Beyond the challenges that we have already discussed, what are the top 1-2
challenges that members of your patient advocacy group or community face with
EA/CU programs?

Suggestions

2) Based on these challenges and those described earlier, what suggestions do you
have for improvement?

Successes

3) Thinking about members of your patient advocacy group or community, are there
parts of existing EA/CU programs that function especially smoothly that you
would not want to see changed?

Education

4) What can be done to better educate members of your patient advocacy group or
community about the existence of these programs and how to best navigate the
application process?

PROPRIETARY

10

PATIENT ADVOCACY GROUPS

E General feedback and wrap-up
Prioritized questions in bold

General
feedback
script

Likelihood
of use

Right to try

General
feedback

Wrap-up
script

Finally, I’d like to gauge the group’s overall satisfaction with existing EA/CU programs and
cover any general feedback that we’ve missed to this point:

1) If medically appropriate, would you recommend treatment through the FDA’s
Expanded Access and Compassionate Use programs to a member of your
patient advocacy group or community? To a friend or family member? Why or
why not?
2) “Right to try” laws enable patients to try experimental therapies that have completed
Phase I testing without soliciting FDA authorization. Would members of your patient
advocacy group or community be in favor of right to try legislation?

3) What other feedback or suggestions for improvement would you like to share?
Save no more than five minutes for this question.

Again, I want to thank everyone for taking the time to participate in this session. Your input
is incredibly valuable in informing our assessment of EA/CU programs.

PROPRIETARY

11

Contents

▪ Patient advocacy groups
▪ IRBs
▪ Health system administrators

PROPRIETARY

12

IRBS

Agenda for IRB focus groups
Topics covered
Welcome and
A moderator
introduction

B

Participant
introduction

Structured discussion
C around potential pain
points

▪
▪
▪
▪
▪
▪
▪
▪

Open discussion on
D successes/challenges
of existing programs

▪
▪
▪

General feedback
E
questions and wrap-up

▪
▪
▪

Purpose of focus group and agenda for the session

Time allocation [min]
5

How/why participants were chosen
Role of moderator (e.g., time management)
Names of participants and their IRBs

10

How participants first learned about EA/CU programs
Frequency with which IRB deals with EA/CU requests
Hypothesized pain points across identification, application, and
follow-up

35

Role of IRB in EA/CU programs
Additional challenges and successes

25

Suggestions for improvement
Approach to educating stakeholders about EA/CU
Likelihood of recommending the program to someone else

15

Other feedback not previously covered
Favorability of right to try legislation
90

Total

PROPRIETARY

13

IRBS

A Welcome and moderator introduction

Moderator
instructions

This guide will be used to manage time effectively and ensure coverage of certain topics
of conversation. Participants should freely discuss topics presented by the moderator,
rather than following the question/answer cadence of an interview. Due to time
restrictions, it is possible that not every question will be asked, and it is the moderator’s
responsibility to decide when to transition to a new topic. Additionally, the moderator
should adapt questions based on the composition of each group and the group’s prior
responses. “Voting” sections should be completed using Mentimeter, and time estimates
for these questions are included in this guide.

PROPRIETARY

14

IRBS

A Welcome and moderator introduction
Hi, my name is [fill in name], and I will be leading our discussion today on the FDA’s
Expanded Access and Compassionate Use programs for drugs/biologics and devices,
respectively.
I’d like to start by thanking each of you for your participation in this session, which will last
90 minutes. The input from this session will be reported back to FDA in aggregate to help
improve EA/CU programs, but all responses will be anonymized. This session will not be
recorded in any form, but I will take notes, as will [fill in name] in the back of the room.
Each of you represents an institutional review board with some degree of experience with
Moderator
introduction EA/CU. During this session, I will pose questions to the group and allow time for open
script
discussion. There are no right or wrong answers, and I would encourage everyone to
share their honest opinions.
To start the conversation, I’ll give everyone a chance to introduce themselves. Then, we’ll
transition to discussing specific challenges and successes with the existing EA/CU
programs. Lastly, I’ll leave time for general feedback not covered earlier in the session.
As moderator, it is my job to actively manage the time and make sure that we cover all of
our desired topics. Please do not be alarmed if I stop an on-going discussion in order to
move to a new topic.

PROPRIETARY

15

IRBS

B Participant introduction
Prioritized questions in bold

To begin with, in order to facilitate good dialogue amongst ourselves, can everyone please
say their name, the IRB they represent, and how they first learned about EA/CU
Participant
introduction programs?
Wait for everyone to introduce themselves. Allow no more than one minute per participant.
script
Thank you for those introductions. Now, I’ll ask you to use the iPad in front of you to
answer a question about how often your IRB handles EA/CU requests. I’ll give everyone a
Participant
introduction minute to respond to the question, and please let me know if you’re having any difficulty
with the device.
script
Wait one minute.
1) How often does your IRB receive Expanded Access (EA) / Compassionate Use
(CU) requests?
Never
Demographi
cs (voting)

Rarely (once or twice in the past few years)
Occasionally (one or twice per year)
Frequently (multiple times per quarter)
N/A
Don’t know

PROPRIETARY

16

IRBS

C Structured discussion around potential pain points
Prioritized questions in bold

Pain point
script

Identification

In this part of the conversation, we’ll look at 3 parts of the EA/CU application process. In
the first, identification, an EA/CU program is deemed appropriate for a patient by their
doctor, and the patient must decide whether to proceed. I’ll start by asking:
1) How easy or challenging are your interactions (if any) with healthcare providers
and patients prior to receiving an EA/CU application?
2) Would you feel prepared and empowered to provide guidance on IRB requirements at
this early stage? Why or why not?

PROPRIETARY

17

IRBS

C Structured discussion around potential pain points
Prioritized questions in bold

Pain point
script

Next, we’ll talk about the application process, in which access to an investigational drug,
biologic, or device is sought from a manufacturer and documentation is submitted to
regulatory bodies. Please start by using the iPad in front of you to answer a second voting
question, which asks you to rate the importance of a list of factors (from 1-10) in
determining whether or not to approve an application for treatment through an EA/CU
program. I’ll give everyone a couple of minutes to respond to the question, and please let
me know if you’re having any difficulty with the device. Wait three minutes.
3) How relevant would each of these factors be in deciding whether to approve an
application for treatment through an EA/CU program? Please rate from 1-10 with
1 being completely insignificant and 10 being extremely significant.
1 2 3 4 5 6 7 8 9 10 N/A Don’t
know

Product / device safety
Product / device efficacy
Application
(voting)

Risk / benefit analysis of using investigational product
Sufficiency of informed consent, including awareness
of out of pocket costs
Prior experience with the sponsor
Emergent / non-emergent nature of the request
Patient population (e.g., adult vs. pediatric vs. elderly)
Proposed monitoring protocol
Data to be collected
PROPRIETARY

18

IRBS

C Structured discussion around potential pain points
Prioritized questions in bold

Pain point
script

Thank you for answering that question. Continuing with the application process:
4) What is your standard turnaround time on EA/CU applications, and what
challenges does this timeframe impose? Do you have special procedures in
place to handle EA/CU requests?

Application

5) As part of FDA’s effort to simplify the EA/CU application process, only one
member of an IRB, either the chair or another appropriate person, now needs to
approve a healthcare provider’s use of an investigational product to treat a
patient. How has this change in regulation impacted the manner in which your
IRB reviews EA/CU applications?
6) Are members of your IRB in favor of this change in regulation? Why or why not?
7) What would cause your IRB to take longer than the standard amount of time to
approve a request? To not approve a request?
8) How easy or challenging are your interactions with healthcare providers while
processing EA/CU applications?
9) Does your IRB ever speak with other IRBs about how they handle EA/CU requests?
Why or why not?

Pain point
script

Lastly, we’ll talk about follow-up after patients have begun treatment under EA/CU
programs:

Follow-up

10) How easy or challenging are your interactions (if any) with patients, healthcare
providers, and the FDA after treatment has begun?
PROPRIETARY

19

IRBS

D Open discussion on successes/challenges of existing programs
Prioritized questions in bold

Open
discussion
script

Now that we’ve covered specific difficulties associated with existing EA/CU programs, I’d
like to ask the group some more open-ended questions to fill in any gaps that we may
have missed. As time allows, ask participants to brainstorm answers to each question for
one minute separately before discussing as a group.

Challenges

1) Beyond the challenges that we have already discussed, what are the top 1-2
challenges that your IRB faces with EA/CU programs? ? Can you identify any
bottlenecks in the approval process?

Suggestions

Successes

Education

2) Based on these challenges and those described earlier, what suggestions do you
have for improvement?

3) Reflecting on your experience, are there parts of existing EA/CU programs that
function especially smoothly that you would not want to see changed?

4) What can be done to better educate IRBs about EA/CU programs and how to best
handle requests? Who else should education efforts target?

PROPRIETARY

20

IRBS

E General feedback and wrap-up
Prioritized questions in bold

General
feedback
script

Finally, I’d like to gauge the group’s overall satisfaction with existing EA/CU programs and
cover any general feedback that we’ve missed to this point:

Likelihood
of use

1) If medically appropriate, would you recommend treatment through the FDA’s
Expanded Access and Compassionate Use programs to a healthcare provider
with whom you’ve worked? To a friend or family member? Why or why not?

Right to try

2) “Right to try” laws enable patients to try experimental therapies that have completed
Phase I testing without soliciting FDA authorization. Would you be in favor of right to try
legislation?

General
feedback

Wrap-up
script

3) What other feedback or suggestions for improvement would you like to share?
Save no more than five minutes for this question.

Again, I want to thank everyone for taking the time to participate in this session. Your input
is incredibly valuable in informing our assessment of EA/CU programs.

PROPRIETARY

21

Contents

▪ Patient advocacy groups
▪ IRBs
▪ Health system administrators

PROPRIETARY

22

HEALTH SYSTEMS

Agenda for health systems focus groups
Topics covered
Welcome and
A moderator
introduction

B

Participant
introduction

Structured discussion
C around potential pain
points

▪
▪
▪
▪
▪
▪
▪
▪

Open discussion on
D successes/challenges
of existing programs

▪
▪
▪

General feedback
E
questions and wrap-up

▪
▪
▪

Purpose of focus group and agenda for the session

Time allocation [min]
5

How/why participants were chosen
Role of moderator (e.g., time management)
Names of participants and their health systems

10

How participants first learned about EA/CU programs
Frequency with which health system deals with EA/CU requests
Hypothesized pain points across identification and treatment

35

Role of health system in EA/CU programs

Additional challenges and successes

25

Suggestions for improvement
Approach to educating health system admins about EA/CU
Likelihood of recommending the program to someone else

15

Other feedback not previously covered
Favorability of right to try legislation
90

Total

PROPRIETARY

23

HEALTH SYSTEMS

A Welcome and moderator introduction

Moderator
instructions

This guide will be used to manage time effectively and ensure coverage of certain topics
of conversation. Participants should freely discuss topics presented by the moderator,
rather than following the question/answer cadence of an interview. Due to time
restrictions, it is possible that not every question will be asked, and it is the moderator’s
responsibility to decide when to transition to a new topic. Additionally, the moderator
should adapt questions based on the composition of each group and the group’s prior
responses. “Voting” sections should be completed using Mentimeter, and time estimates
for these questions are included in this guide.

PROPRIETARY

24

HEALTH SYSTEMS

A Welcome and moderator introduction
Hi, my name is [fill in name], and I will be leading our discussion today on the FDA’s
Expanded Access and Compassionate Use programs for drugs/biologics and devices,
respectively.
I’d like to start by thanking each of you for your participation in this session, which will last
90 minutes. The input from this session will be reported back to FDA in aggregate to help
improve EA/CU programs, but all responses will be anonymized. This session will not be
recorded in any form, but I will take notes, as will [fill in name] in the back of the room.
Moderator
Each of you represents a health system with some degree of experience with EA/CU.
introduction During this session, I will pose questions to the group and allow time for open discussion.
script
There are no right or wrong answers, and I would encourage everyone to share their
honest opinions.
To start the conversation, I’ll give everyone a chance to introduce themselves. Then, we’ll
transition to discussing specific challenges and successes with the existing EA/CU
programs. Lastly, I’ll leave time for general feedback not covered earlier in the session.
As moderator, it is my job to actively manage the time and make sure that we cover all of
our desired topics. Please do not be alarmed if I stop an on-going discussion in order to
move to a new topic.

PROPRIETARY

25

HEALTH SYSTEMS

B Participant introduction
Prioritized questions in bold

To begin with, in order to facilitate good dialogue amongst ourselves, can everyone please
say their name, the health system they represent, and how they first learned about EA/CU
Participant
introduction programs?
Wait for everyone to introduce themselves. Allow no more than one minute per participant.
script
Thank you for those introductions. Now, I’ll ask you to use the iPad in front of you to
answer a question about how often your health system handles EA/CU requests. I’ll give
Participant
introduction everyone a minute to respond to the question, and please let me know if you’re having
any difficulty with the device.
script
Wait one minute.
1) How often does your health system handle Expanded Access (EA) /
Compassionate Use (CU) requests?
Never
Demographi
cs (voting)

Rarely (once or twice in the past few years)
Occasionally (one or twice per year)
Frequently (multiple times per quarter)
N/A
Don’t know

PROPRIETARY

26

HEALTH SYSTEMS

C Structured discussion around potential pain points

Pain point
script

In this part of the conversation, we’ll focus on two parts of the EA/CU application process.
In the first, identification, an EA/CU program is deemed appropriate for a patient by their
doctor, and the patient must decide whether to proceed. Please start by using the iPad in
front of you to answer a second voting question, which asks you to rate the importance of
a list of factors (from 1-10) in evaluating whether or not a provider in your health system
should apply to treat a patient through an EA/CU program. I’ll give everyone a couple of
minutes to respond to the question, and please let me know if you’re having any difficulty
with the device. Wait three minutes.

PROPRIETARY

27

HEALTH SYSTEMS

C Structured discussion around potential pain points
Prioritized questions in bold

1) How relevant would each of these factors be in evaluating whether a provider in
your health system should apply to treat a patient through an EA/CU program?
Please rate from 1-10 with 1 being completely insignificant and 10 being
extremely significant.
1 2 3 4 5 6 7 8 9 10 N/A Don’t
know

Product / device safety
Product / device efficacy
Sufficiency of informed consent
Identificatio
n (voting)

Effect on hospital reputation, including potential
media backlash
Cost to health system, including but not limited to
medical supplies and staff time
Provider’s experience with EA/CU
Provider’s experience with other clinical trials
Provider’s reputation within health system
Health system’s experience with EA/CU
Whether health system is seen as a leader in the
therapeutic area of the EA/CU request
Legal risk

PROPRIETARY

28

HEALTH SYSTEMS

C Structured discussion around potential pain points
Prioritized questions in bold

Pain point
script

Thank you for answering that question. Continuing with our discussion of the identification
step:
2) In general, what resources does your health system grant to providers dealing with
especially challenging cases, and what is the process for handling these cases? Does
your health system convene groups of healthcare providers to facilitate discussion
(e.g., tumor boards)?

Identification

3) What resources (e.g., educational materials) does your health system grant to
providers considering EA/CU programs?
4) What procedures and resources (if any) does your health system have in place to
enable patients to be treated through an EA/CU program?
5) How closely do health system administrators work with providers who are
interested in applying for one of these programs? What role(s) do they play?
6) Do you feel prepared and empowered to engage with other relevant stakeholders
(including IRBs) at this stage? Why or why not?

Pain point
script

Treatment

Secondly, we’ll talk about treatment of patients under EA/CU programs:

7) Once treatment through an EA/CU program has begun, how prepared is your health
system to deal with adverse events related to treatment?
8) What costs does your health system incur from administering treatments
through an EA/CU programs, and how significant are these costs?
PROPRIETARY

29

HEALTH SYSTEMS

D Open discussion on successes/challenges of existing programs
Prioritized questions in bold

Open
discussion
script

Challenges

Suggestions

Successes

Education

Now that we’ve covered specific difficulties associated with existing EA/CU programs, I’d
like to ask the group some more open-ended questions to fill in any gaps that we may
have missed. As time allows, ask participants to brainstorm answers to each question for
one minute separately before discussing as a group.
1) Beyond the challenges that we have already discussed, what are the top 1-2
challenges that your health system faces with EA/CU programs?

2) Based on these challenges and those described earlier, what suggestions do you
have for improvement?

3) Reflecting on your experience, are there parts of existing EA/CU programs that
function especially smoothly that you would not want to see changed?

4) What can be done to better educate health system administrators about the
existence of these programs and how to best approach requests within their
health system? Who else should education efforts target?

PROPRIETARY

30

HEALTH SYSTEMS

E General feedback and wrap-up
Prioritized questions in bold

General
feedback
script

Finally, I’d like to gauge the group’s overall satisfaction with existing EA/CU programs and
cover any general feedback that we’ve missed to this point:

Likelihood
of use

1) If medically appropriate, would you recommend treatment through the FDA’s
Expanded Access and Compassionate Use programs to a provider in your health
system? To a friend or family member? Why or why not?

Right to try

2) “Right to try” laws enable patients to try experimental therapies that have completed
Phase I testing without soliciting FDA authorization. Would you be in favor of right to try
legislation?

General
feedback

Wrap-up
script

3) What other feedback or suggestions for improvement would you like to share?
Save no more than five minutes for this question.

Again, I want to thank everyone for taking the time to participate in this session. Your input
is incredibly valuable in informing our assessment of EA/CU programs.

PROPRIETARY

31