CERSI Compounding PRA SSA 2019

U.S. Food & Drug Administration
Obtaining Information for Evaluating Nominated Bulk Drug Substances for Use in
Compounding Drug Products Under Section 503B of the Federal Food, Drug, and Cosmetic Act.
OMB Control No. 0910-NEW
SUPPORTING STATEMENT—Part A: Justification
1. Circumstances Making the Collection of Information Necessary
This information collection supports Food and Drug Administration (FDA, us or we) research.
Section 503B of the Federal Food, Drug and Cosmetic Act (FD&C Act) describes the conditions
under which outsourcing facilities, a new type of drug compounder established in 2013, may be
exempt from the following requirements of the FD&C Act: requirements for FDA approval of
drugs in section 505 of the FD&C Act (21 U.S.C. 355), labeling with adequate directions for use
under section 502(f)(1) of the FD&C Act (21 U.S.C. 352(f)(1)), and drug supply chain security
requirements under section 582 (21 U.S.C. 360eee-1).
One of the conditions that must be met for a drug product compounded by an outsourcing facility
to qualify for these exemptions is that the outsourcing facility may not have compounded a drug
using a bulk drug substance unless (1) the bulk drug substance appears on a list established by
the Secretary identifying bulk drug substances for which there is a clinical need (“bulks list”); or
(2) the substance appears on the drug shortage list in effect under section 506E of the FD&C Act
at the time of compounding, distribution, and dispensing.
FDA has solicited nominations for the 503B bulks list and has received over 200, but these
these nominations often do not contain substantive information on the current and historical use
of these substances in clinical practice to inform FDA’s evaluation. Developing the 503B bulks
list is a significant priority for the agency. To inform our evaluation of bulk drug substances for
inclusion on the 503B bulks list, we have entered into research studies with the University of
Maryland (UMD) Center of Excellence in Regulatory Science and Innovation (CERSI) and the
Johns Hopkins University (JHU) CERSI. We intend to seek input from the UMD-CERSI and
JHU-CERSI on the use of these bulk drug substances in clinical practice by examining their
current and historical use in compounding.
We therefore request OMB approval of the information collection as discussed in our supporting
statement parts A and B.
2. Purpose and Use of the Information
Information regarding the historical and current use of the substances in compounding obtained
by this research will help inform our assessments as to the clinical need for outsourcing facilities
to compound drug products using nominated bulk drug substances. We intend to use a two-part
analysis in evaluating substances nominated for placement on the 503B bulks list to determine

whether there is a clinical need. The collaboration with UMD-CERSI and JHU-CERSI pertains
to Part 2 of the analysis, which applies to bulk drug substances that are not components of FDAapproved drug products, as well as certain bulk drug substances that are components of FDAapproved drug products and that have gone through Part 1 and warrant further evaluation under
Part 2 of the analysis. One of the factors that FDA considers under Part 2 is “current and
historical use of the substance in compounded drug products, including information about the
medical condition(s) that the substance has been used to treat and any references in peerreviewed medical literature.”
Information regarding the historical and current use in clinical practice of compounded drugs
containing these bulk drug substances obtained by UMD-CERSI and JHU-CERSI will help
inform FDA’s assessments as to the clinical need for outsourcing facilities to compound drug
products using the bulk drug substances.
3. Use of Information Technology and Burden Reduction
Researchers may use interviews, focus groups, and questionnaires, as appropriate, to obtain
information concerning the use of compounded drug(s) from medical experts, outsourcing
facilities, and other stakeholders. Web-based data collection tools will be used for questionnaires
to minimize respondent burden.
4. Efforts to Identify Duplication and Use of Similar Information
We are unaware of a similar data collection.
5. Impact on Small Businesses or Other Small Entities
We expect most respondents will be private individuals.
6. Consequence of Collecting the Information Less Frequently
The proposed data collection is a discrete endeavor relevant to the specific study described.
7. Special Circumstances Relating to the Guidelines of 5 CFR 1320.5
There are no special circumstances for this collection of information.
8. Comments in Response to the Federal Register Notice and Efforts to Consult Outside Agency
In accordance with 5 CFR 1320.8(d), FDA published a 60 day notice for public comment in the
Federal Register of September 17, 2018 (83 FR 46957). One comment was received, however it
pertained to a different docket and we have posted the comment to the appropriate docket
accordingly.

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9. Explanation of Any Payment or Gift to Respondents
No payments or gifts will be provided to respondents.
10. Assurance of Confidentiality Provided to Respondents
Information provided by respondents will be kept private and anonymous, except as otherwise
required by law. All information that can identify individual respondents will be kept by FDA’s
UMD CERSI and JHU CERSI partners in files that are separate from information provided to
FDA.
Respondents also will be advised of the following: the nature of the activity; the intended
purpose and use of the data collected; FDA sponsorship; and the fact that participation is
voluntary at all times.
The UMD CERSI research study was found to be exempt from the “Regulations for the
Protection of Human Subjects” requirements in accordance with paragraph b(2) of 45 CFR Sec.
46.101 by the Research Involving Human Subjects Committee (RIHSC). As necessary, the JHU
CERSI study methods will be evaluated by RIHSC prior to information collection.
11. Justification for Sensitive Questions
This data collection will not include sensitive questions. The complete list of questions is
Included with our supporting statement part B.
12. Estimates of Annualized Burden Hours and Costs
12a. Estimated Annualized Hourly Burden
UMD-CERSI researchers will study the current and historical use of 75 bulk drug
substances per year. FDA estimates that each bulk drug substance may have uses that relate to,
on average, two different medical specialties, yielding 150 bulk drug substance-specialty
combinations. We estimate a total of 15 different specialties will be implicated and therefore that
10 bulk drug substance uses will be relevant to each specialty on average (150/15).
For focus groups and interviews, UMD-CERSI will seek to include around 10 experts
from each of the 15 specialty groups, yielding a total of 150 respondents. Each respondent will
opine on each of the 10 bulk drug substance uses that are relevant to their specialty, yielding an
estimated 1,500 annual responses. The average burden per response is 2 hours, for a total of
3,000 annual burden hours.
For the expert questionnaire, we estimate UMD-CERSI will obtain responses from 50
experts from each of the 15 specialty groups, yielding a total of 750 respondents. Each
respondent will respond to a questionnaire on each of the 10 bulk drug substance uses that are
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relevant to their specialty, yielding an estimated 7,500 annual responses. The average burden per
response is 30 minutes, for a total of 3,750 annual burden hours.
JHU-CERSI will study the use of six bulk drug substances in patients with autism
spectrum disorder (ASD) and will use a questionnaire to obtain information from parents on the
use of these substances. Researchers will seek responses from 1,000 parents of children with
ASD. The average burden per response is 30 minutes, for a total of 500 annual burden hours.
FDA therefore estimates the burden of this collection of information as follows:

Information
Collection
UMD-CERSI
Expert focus
groups and
interviews
UMD-CERSI
Expert
questionnaire
JHU-CERSI
Parent
questionnaire
TOTAL

Table 1. Estimated Annual Reporting Burden
Number of
Number of
Total Annual
Average Burden
Total
Respondents Responses per
Responses
per Response
Hours
Respondent
150
10
1500
2
3,000

750

10

7,500

.5

3,750

1,000

1

1,000

.5

500

1,900

10,000

7,250

12b. Annualized Cost Burden Estimates
We estimate no capital costs to respondents for the collection of information.
13. Estimates of Other Total Annual Cost Burden to Respondents or Recordkeepers
There are no capital, start-up, operating or maintenance costs associated with this information
collection.
14. Annualized Cost to the Federal Government
The total estimated cost to the Federal Government for the collection of data is $737,198; with
an annual cost to FDA of $29,000. This includes the costs paid to UMD CERSI and JHU CERSI
grantees to obtain the information described in this collection, assess results, and prepare a report
($650,198). The cost also includes FDA staff time to provide information and feedback to UMD
and JHU CERSI, including identifying specific bulk drug substances for inclusion in the study
($87,000; 4 hours per week for 3 years).

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15. Explanation for Program Changes or Adjustments
This is a new information collection.
16. Plans for Tabulation and Publication and Project Time Schedule
Assessment of the information collected will be primarily qualitative (for example, content
analysis for in-depth interviews); some quantitative analysis will be conducted for information
obtained through questionnaire instruments.
FDA will receive summarized research results from grantees on a rolling basis beginning 10
months after project initiation. Research results will be made publicly available. The UMDCERSI research is expected to last three years and the JHU-CERSI research is expected to last
one and a half years.
Please see Supporting Statement B for additional description of research methods.
17. Reason(s) Display of OMB Expiration Date is Inappropriate
The OMB expiration date will be displayed on the collection instruments.
18. Exceptions to Certification for Paperwork Reduction Act Submissions
There are no exceptions to the certification.

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