Assessment of Physician Applicants' Experiences with FDA's Expanded Access and Compassionate Use Programs

Customer Satisfaction Survey
OMB No. 0910-0360
Exp. Date: 9/30/2020

Paperwork
Reduction
Act
Statement

Public reporting burden for this collection of information is estimated to be 20 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 to Food and Drug Administration (FDA) Office Operations, 3WFN,
11601 Landsdown Street, North Bethesda, MD 20852. 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 0910-0360.
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 respondent identity is needed (e.g., for follow-up
of non-responders), this information collection fully complies with all aspects of the Privacy Act and data will be
kept private to the fullest extent allowed by law.

Instructions

Please answer the following questions based on your experience with Expanded Access (EA) and
Compassionate Use (CU) programs and relevant stakeholders (e.g., patients, drug and device manufacturers,
health system administrators, health insurance companies, institutional review boards, FDA)

1

A Survey respondent demographics

1) In which state do you practice?

State

2) In which medical sub-specialties do you practice? Select all that apply.

3) What is your practice affiliation? Select all that apply.
Demographics

Private – solo

Private – group

Academic

Hospital affiliated

Other

4) How did you get information about EA/CU programs? Select all that apply.
A colleague

FDA website (FDA.gov)

A patient

ClinicalTrials.gov (ClinicalTrials.gov)

Literature search

Reagan-Udall Foundation Expanded Access
Navigator (navigator.reaganudall.org)

Medical society/conference
Patient advocacy group
Other (explain)

2

A Survey respondent demographics
5) Over the course of your career, how many times have you contacted a drug or device manufacturer to
request access to a product through the EA/CU program?
times
6) Of those initial requests to manufacturers, how many were formally submitted to the FDA for review?
requests

Demographics

When was the first time?

When was the most recent time?

Year

Year

7) What was the most common reason that requests submitted to manufacturers were not submitted to the
FDA?
Patient withdrew
Patient died
Manufacturer did not agree to provide investigational product
Institutional Review Board (IRB) did not approve
Health Care Administrators did not approve
Paperwork was too cumbersome
Process was too time-intensive
Treatment wasn't covered by insurance
Unknown
Other (explain):

3

B Ease and satisfaction
8) How satisfied are you with the entirety of the existing EA/CU program, from identifying appropriate patients
through to treatment and follow-up? Please explain.
Very unsatisfied
Unsatisfied
Neutral
Satisfied
Very satisfied
NA / don’t know
0
1
2
3
4
5
Overall
satisfaction

Explain:
9) Would you encourage a colleague to consider an EA/CU program if appropriate for a patient?
Please explain.
Yes

No

Explain:

4

B Ease and satisfaction
10a) During your most recent application for treatment through an EA/CU program, how would you rate the
following:

▪

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪

Finding appropriate products for that patient

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪
Ease /
difficulty of
program

Identifying that the EA/CU program would be appropriate for a patient

Making decisions based on preliminary/incomplete data

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪

Coordinating and timing contact with manufacturers, IRBs, and the FDA

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪

Working with the manufacturer to request access

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪

Working with an IRB to obtain approval

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5
5

B Ease and satisfaction
10b) During your most recent application for treatment through an EA/CU program, how would you rate the
following:

▪

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪

Understanding the roles and responsibilities of all stakeholders throughout the process

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪
Ease /
difficulty of
program

Working with the FDA to obtain authorization to treat with an investigational product

Receiving drugs/devices from the manufacturer

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪

Understanding who would pay for the treatment

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪

Covering your expenses (e.g., your time, your staff’s time, medical supplies)

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪

Completing documentation during and after treatment (as appropriate)

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5

▪

Attributing relatedness of adverse events to the treatment

Very difficult

Difficult

Neutral

Easy

Very Easy

NA / don’t know

0

1

2

3

4

5
6

B Ease and satisfaction
11) Was there anything else that was especially challenging?
Challenge
identification

Was there anything else that was especially easy that you would not want to see changed?

7

C Process evaluation
12a) On average, how much time per patient did:
You personally
spend
Your staff spend
communicating
communicating
with:
with:

Time
estimation

You personally
spend completing
paperwork for:

Your staff spend
completing
paperwork for:

Patients

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

Manufacturers

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

Health
system
administrators

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know
8

C Process evaluation
12b) On average, how much time per patient did:
You personally
spend
Your staff spend
communicating
communicating
with:
with:

Time
estimation

You personally
spend completing
paperwork for:

Your staff spend
completing
paperwork for:

Insurance
Companies

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

IRBs

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

FDA

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know

No time at all
0-15 minutes
15-30 minutes
30-60 minutes
1-2 hours
>2 hours
Don’t know
9

C Process evaluation
13) How much time did each of the following take?

▪

Gaining access from a drug or device manufacturer (inclusive of time to submit the request and gain
access)
Less than a day

▪
Time
estimation

1-2 weeks

2-4 weeks

> 4 weeks

1-2 days

3-7 days

1-2 weeks

2-4 weeks

> 4 weeks

Gaining approval from an IRB after submitting a request (inclusive of time to submit the request and
gain approval)
Less than a day

▪

3-7 days

Gaining authorization from FDA after submitting an application (inclusive of time to submit the request
and gain approval)
Less than a day

▪

1-2 days

1-2 days

3-7 days

1-2 weeks

2-4 weeks

> 4 weeks

Receiving a drug/device from a manufacturer after FDA authorization (inclusive of time to submit the
request and receive the drug/device)
Less than a day

1-2 days

3-7 days

1-2 weeks

2-4 weeks

> 4 weeks

10

D Suggestions for improvement
14) Which of the following would substantially improve the EA/CU program? Please rank the top five
improvements.
Increased availability of academic literature
More information from patient advocacy groups
Less paperwork for requesting access from manufacturer
Less paperwork for FDA application
Less paperwork for IRB approval
Option to chat online with FDA medical officers
FDA call center to answer questions
Suggestions for
improvement

More responsive communication from manufacturers
More responsive communication from FDA
More responsive communication from IRBs
Transparency on policies for receiving access from drug or device manufacturers
Transparency on standards for FDA authorization of EA/CU
Transparency on requirements for IRB approval
Other (explain):
15) Is there anything else you would like to add about past experiences with EA/CU programs or suggestions
for improvement?

11

E Right to try
16) "Right to try" laws enable patients to try experimental therapies that have completed Phase I testing
without soliciting FDA authorization. Are you in favor of “right to try” laws being passed?
Right to try

Strongly against

Against

Neutral

In favor

Strongly in favor

NA / don’t know

0

1

2

3

4

5

12