0746 Supporting Statement Part A 2016

Evaluation of the Program for Enhanced Review Transparency and Communication
for New Molecular Entity New Drug Applications and Original Biologics License Applications
in Prescription Drug User Fee Acts 
OMB Control No. 0910-0746
SUPPORTING STATEMENT Part A

A. Justification
1.

Circumstances Making the Collection of Information Necessary
Pursuant to the requirements of the Paperwork Reduction Act (44 USC 35) and Public
Law, No. 112-144, 126 Stat. 993 (also known as the Food and Drug Administration Safety
and Innovation Act, or FDASIA), the Food and Drug Administration (FDA) is requesting
Office of Management and Budget (OMB) approval of the information request that FDA
committed to in Section II.B of the Prescription Drug User Fee Act (PDUFA)
Reauthorization Performance Goals and Procedures Fiscal Years 2013 Through 2017 letter
(“Commitment Letter”). In accordance with earlier sections of the Commitment Letter,
FDA established a new review model for enhanced review transparency and
communication for new molecular entity (NME) new drug applications (NDAs) and
original biologics license applications (BLAs) for fiscal years (FYs) 2013 to 2017; this new
review model is called “the Program.” Section II.B of the Commitment Letter states that
the Program will be assessed by an independent contractor and that the assessment will
“include interviews of the sponsor.”
Since 1992, FDA has reviewed and acted on NME NDAs and original BLAs in accordance
with timeliness and other requirements established in PDUFA. Each 5-year reauthorization
of PDUFA has incorporated additional provisions to enhance and improve the process for
the review of human drugs and biological products.  As part of its commitments in PDUFA
V, FDA established the Program, a new review model to promote greater transparency and
increased communication between the FDA review team and the applicant on the most
innovative products reviewed by the agency. The Program applies to all NME NDAs and
original BLAs that are received from October 1, 2012, through September 30, 2017.
The goals of the Program are to increase the efficiency and effectiveness of the first review
cycle and decrease the number of review cycles necessary for approval so that patients have
timely access to safe, effective, and high quality new drugs and biologics. A key aspect of
the Program is an interim and final assessment that will evaluate how well the parameters
of the Program have achieved the intended goals. The PDUFA V Commitment Letter
specifies that the interim and final assessments must be conducted by an independent
contractor and that they must include interviews of pharmaceutical manufacturers who
submit NME NDAs and original BLAs to the Program in PDUFA V. The independent
contractor for the Program assessments is Eastern Research Group, Inc. (ERG), and the
statement of work for the assessments is available at
http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM304793.pdf.

In accordance with the PDUFA V Commitment Letter, FDA proposes to have ERG
conduct independent interviews of applicants after FDA issues a first-cycle action for
applications reviewed under the Program. The purpose of these interviews is to collect
feedback from applicants on the success of the Program in increasing review transparency
and communication during the review process, including any best practices or challenges
experienced in the Program. ERG will not ask applicants for any confidential business
information, nor will ERG ask applicants to evaluate the performance of FDA staff.
Instead, interviews will focus on applicant experiences with the new review process under
the Program. Some applicants might perceive their individual feedback about the new
review process to be sensitive given that they might interact with FDA about other
NDAs/BLAs in the future. To address these potential concerns, ERG will not share any
identifying information with FDA and will include only anonymized, aggregated interview
results in the interim and final assessment reports and presentations at public meetings held
regarding each assessment. FDA will publish ERG’s assessments (with interview results
and findings) for public comment.
The independent assessments, including information from applicant interviews, will be of
great interest to FDA’s stakeholders, including the regulated industry, patient and consumer
groups, healthcare professionals, and Congress. Equally importantly, these assessments
will be critical in determining whether and how to refine Program implementation to
improve the likelihood of success of the Program.
2.

Purpose and Use of the Information Collection
The contractor for the independent assessment has prepared a draft protocol and script for
scheduling and conducting interviews with applicants after their NDA/BLA receives a first
review cycle action from FDA. The protocol ensures that ERG is aware of all applicants
who are candidates for interviews and schedules and conducts post-action interviews in a
timely, consistent manner using good interview practices. The script includes rating-scale
questions and open-ended questions aimed at obtaining a thorough understanding of
applicants’ experiences and insights about the review process for their application under
the Program.
To design and prepare for this information collection, FDA consulted with the contractor,
ERG, which has program evaluation and interview experts on staff. Similarly, ERG
consulted corporate experts outside its independent assessment project team to obtain input
and feedback on this information collection.
The contractor will analyze rating-scale question responses to determine the extent to
which applicants as a group believe that Program practices contribute to review process
transparency, efficiency, and predictability. ERG will analyze open-ended responses to
identify practices that applicants perceive as enhancing review process transparency,
practices that applicants perceive as increasing the efficiency of the review process, and
aspects of Program implementation that might benefit from improvement. In addition,
ERG will consider how interview responses might explain or provide context for results
from other parts of the independent assessment, such as metrics pertaining to presubmission meetings, mid-cycle communications, late-cycle meetings, and other Program
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elements. In this way, ERG will use interview responses to complement and supplement
data on Program parameters obtained through other means. ERG will synthesize and
interpret the results to develop a set of findings and recommendations for the Program to be
included in interim and final assessment reports and presentations.
In turn, FDA will use the independent assessment results, findings, and recommendations
to:

3.



Determine the success of the Program in achieving established goals.



Determine whether and how to refine implementation of the Program during the
remainder of PDUFA V.



Demonstrate compliance with the commitment to conduct the independent assessments
and publish them for public comment.



Share information about the Program with the regulated community, the public health
community, Congress, and the general public.

Uses of Improved Technology and Burden Reduction
ERG will not employ any web-based resources in soliciting sponsor and FDA feedback.
While online surveys can be distributed quickly and easily to a large volume of
respondents, they are not the best vehicle for discussing the nuances of respondent’s
individual experiences, insights, and reasoning. Even surveys with open-ended questions
do not permit immediate follow-up exchanges to clarify or elucidate responses; any followup requires additional contact with respondents, increasing burden. Interviews provide the
more detailed and nuanced feedback needed for this independent assessment in a way that
minimizes burden.
ERG will also minimize the burden by offering to conduct interviews by telephone or
videoconference if meeting in person is burdensome. Finally, the interview instrument has
been designed to elicit the desired feedback in as short an interview time as possible.

4.

Efforts to Identify Duplication and Use of Similar Information
No other Agency or program is interviewing sponsors of NME NDAs/original BLAs as
part of the required assessments of the Program in PDUFA V. Members of FDA’s Center
for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and
Program Advisory Group have met to discuss/identify any similar efforts. This effort is
expected by the regulated industry based on the requirements specified in the PDUFA V
Commitment Letter.

5.

Impact on Small Businesses or Other Small Entities
The contractor for the independent assessment will not ask small businesses or entities to
travel, pay for telephone charges, or incur other unusual expenses. To avoid such expenses
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and minimize burden, ERG will conduct interviews by telephone if the applicant will not
be in or near Silver Spring, MD during the desired interview timeframe.
6.

Consequences of Collecting the Information Less Frequently
There would be several consequences of not collecting the data as proposed. First, the
requirements of the PDUFA V agreement between FDA and the regulated industry would
not be met. Second, FDA would lack applicant perspectives on best practices and potential
improvements for the Program to help ensure the success of the Program during PDUFA
V. Third, the regulated industry, FDA’s other stakeholders, and the public would lack a
key piece of information about the effectiveness of the Program in achieving its stated
goals. Finally, applicants would not have the opportunities they expect to share their
opinions about the review process under the Program.

7.

Special Circumstances Relating to the Guidelines of 5 CFR 1320.5
There are no special circumstances for the collection of information.

8.

Comments in Response to the Federal Register Notice and Efforts to Consult Outside the
Agency
In accordance with 5 CFR 1320.8(d), FDA published a 60 day notice for public comment
in the Federal Register of December 10, 2015 (Vol. 80 FR 76699). No comments were
received.

9.

Explanation of Any Payment or Gift to Respondents
Respondents will not receive any payments or gifts for participating in this information
collection.

10.

Assurance of Confidentiality Provided to Respondents
While there is no express assurance of confidentiality that cannot be supported by law, the
design of the data collection will allow responses to be anonymous. Interviewees will be
assured of the privacy, to the extent available under law, of their responses through
language placed prominently on all interview materials as well as introductory comments
made by the interviewer. Interviewers will be trained on the privacy of responses and will
be prepared to describe the policy in detail, provide examples, and respond to any related
questions from participants. For example, the interviewer will explain that each
individual’s answers will be combined with those of others and presented in summary form
only, and that FDA will not have access to the names of participants.
All responses that could identify specific sponsors will be kept only by the contractor,
ERG, for use in analysis. Any data received by FDA will not contain personal identifiers,
thus precluding individual identification. Public use data files produced at the end of the
study will follow the current OMB checklist on confidentiality to ensure that they can be
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distributed to the general public for analysis without restrictions and without identification
of interviewees.
11.

Justification for Sensitive Questions
The interview script contains no questions of a sensitive nature. Some interview questions
ask applicants for frank assessments of the review process under the Program, which might
be perceived as sensitive to some applicants. This information is crucial to understanding
the extent to which the Program achieves its goals of improving communication and
transparency in application reviews, what practices contribute to transparent review, and
what practices can be refined to improve review transparency and communication. The
contractor will keep private the identity of individual applicants, as well as each applicant’s
responses to interview questions; all interview materials will emphasize this. No individual
applicant will be identified as an interviewee to FDA.

12.

Estimates of Annualized Burden Hours and Costs
12a.

Annual Burden Estimate

FDA estimates the total annual burden of this information collection to be 210 hours, as
outlined in Table 1.
Table 1. Estimated Annual Reporting Burden
Portion of Study
Pre-test
Interviews
Total

12b.

No. of
Respondents
5
135

No. of
Responses
per
Respondent
1
1

Average
Burden per
Response
1.5
1.5

Total Hours
7.5
202.5
210.0

Annualized Cost Burden Estimate

FDA estimated the annualized cost to respondents to be the burden hours estimate
multiplied by the median hourly wage estimate (Source: Occupational Employment
Statistics, Bureau of Labor Statistics). FDA used the median wage estimate ($90.00) for
Physicians and Surgeons, All Others, Standard Occupational Code (SOC) 29-1069,
because this wage falls in the middle of the range of wages for NDA/BLA managers in
the pharmaceutical industry. FDA multiplied this median wage by 1.4 to capture
benefits, resulting in a loaded hourly median wage rate of $126.00.

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Table 2. Annualized Cost to Respondents
Type of Respondent

Total Burden Hours

Hourly Wage Rate

Total Respondent
Costs

NME NDA / Original
BLA Sponsors

210.0

$126.00

$26,460.00

13.

Estimates Total Annual Cost Burden to Respondents and Record Keepers
There are no capital, start-up, operating, or maintenance costs associated with this
information collection.

14.

Annualized Cost to the Federal Government
This 5-year independent assessment of the Program encompasses several evaluation
methodologies, including interviews with sponsors of NME NDAs and original BLAs
under the Program. The interview effort involves development of an interview protocol
and script, implementation of the interviews, and analysis of results to develop findings and
recommendations about FDA’s Program. The annualized cost to the Federal government is
estimated to be $50,000, which is the total contractor cost of conducting the interview
portion of the independent assessment project. Of the $265,000 allotted over five years to
this interview process, approximately $15,000 was budgeted for interview development
costs (e.g., instrument development, implementation design, etc.) and $250,000 was
budgeted for interview implementation and analysis. There are no other costs to the
Federal government for implementation.

15.

Explanation for Program Changes or Adjustments
This is renewal of an existing data collection wherein the burden remains unchanged.

16.

Plans for Tabulation and Publication and Project Time Schedule
The contractor conducting the independent assessment will present results, including
interview results, in two reports:


Assessment of the Program for Enhanced Review Transparency and Communication
for NME NDAs and Original BLAs in PDUFA V: Interim Report



Assessment of the Program for Enhanced Review Transparency and Communication
for NME NDAs and Original BLAs in PDUFA V: Final Report

FDA will publish these reports, or links to the reports, in the Federal Register.
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Table 3. Schedule for Project Tasks
Tasks
Draft post-action interview protocol and script
Finalize post-action interview protocol and script

Schedule
January 25, 2013
May 8, 2013
Approximately 2 weeks after applications receive a
first review cycle action from FDA (throughout
PDUFA V)
Quarterly throughout PDUFA V
February 15, 2015 (PREPARED 3-27-2015)
November 15, 2016

Conduct post-action interviews
Analyze interview information
Prepare interim assessment report
Prepare final assessment report

17.

Reason(s) Display of OMB Expiration Date is Inappropriate
Not applicable. The collection instrument will display the expiration date.

18.

Exceptions to Certification for Paperwork Reduction Act Submissions 
There are no exceptions to the certification.

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