5 Expanded Access DED & PRS Forms

Attachment 3

26 Oct 2022

Attachment 3 - Expanded Access Record Data Element Definitions and Data Entry Forms

ClinicalTrials.gov Registration Data Element Definitions
for Expanded Access
October 1, 2020
This document describes the definitions for registration data elements submitted to ClinicalTrials.gov for
expanded access records. Such records provide information about investigational products that are made
available through expanded access for patients who do not qualify for enrollment in a clinical trial. For
investigational drug products (including biological products) this includes all expanded access types
under section 561 of the Federal Food, Drug, and Cosmetic Act: (1) for individual patients, including
emergency use; (2) for intermediate-size patient populations; and (3) under a treatment IND or treatment
protocol.
Only one expanded access record should be created for any given investigational product, even if the
investigational product is being made available for individual patient expanded access (that is, the
responsible party should not create an expanded access record for each instance of individual patient
access).These definitions for expanded access records are mostly adapted from 42 CFR Part 11.
Data element entries are annotated with symbols to indicate generally what information is required to be
submitted (and under which circumstances). The responsible party must ensure that the information
provided complies with any applicable laws, regulations, or policies. For more information about some of
the relevant requirements, see Support Materials.

* Required
*§ Required if initial submission date is on or after January 18, 2017
[*] Conditionally required
1. Study Identification
Unique Protocol Identification Number *
Definition: Any unique identifier assigned by the sponsor to refer to the availability of its
investigational product for expanded access use or to identify the expanded access record.
Limit: 30 characters.
Brief Title *
Definition: A short title identifying the expanded access, written in language intended for the lay
public.
Limit: 300 characters.
Acronym [*]
Definition: An acronym or abbreviation used publicly to identify the expanded access, if any.
Limit: 14 characters.
Official Title [*] (Optional if Expanded Access Type is "Individual Patients")
Definition: The title of the expanded access program corresponding to the title that has been

1

Attachment 3

26 Oct 2022

submitted to the U.S. Food and Drug Administration (FDA) for that program, if any.
Limit: 600 characters.
Secondary ID [*] (Optional if Expanded Access Type is "Individual Patients")
Definition: An identifier(s) (ID), if any, other than the organization's Unique Protocol Identification
Number or the NCT number that is assigned to the expanded access record. This includes any unique
identifier assigned by other publicly available clinical trial or expanded access registries.
Limit: 30 characters.
If there is a Secondary ID, then the following information must be provided:
Limit: 119 characters.
Secondary ID Type [*]
Definition: A description of the type of Secondary ID. Select one.
•

•

•

•
•

U.S. National Institutes of Health (NIH) Grant/Contract Award Number: In the
Secondary ID field, include activity code, institute code, and 6-digit serial
number. Other components of the full award number (type code, support year and
suffix, if applicable) are optional.
Other Grant/Funding Number: Identifier assigned by a funding organization
other than the U.S. NIH; also required to enter the name of the funding
organization.
Registry Identifier: Number assigned by a clinical trial registry (for example, a
registry that is part of the World Health Organization [WHO] Registry Network);
also required to enter the name of the clinical trial registry.
EudraCT Number: Identifier assigned by the European Medicines Agency
Clinical Trials Database (EudraCT).
Other Identifier: Also required to enter a brief description of the identifier (for
example, name of organization that issued the identifier).

Description [*]
Definition: If a Secondary ID Type of "Other Grant/Funding Number," "Registry Identifier,"
or "Other Identifier" is selected, provide the name of the funding organization, clinical trial
registry, or organization that issued the identifier.
Study Type *
Definition: The nature of the investigation or investigational use for which clinical study information
is being submitted. Select the "Expanded Access" menu item. (For more information on data
requirements for Interventional or Observational Study Types, see Protocol Registration Data
Element Definitions).
Interventional: Participants are assigned prospectively to an intervention or interventions
according to a protocol to evaluate the effect of the intervention(s) on biomedical or other
health related outcomes.
Observational: Studies in human beings in which biomedical and/or health outcomes are
assessed in pre-defined groups of individuals. Participants in the study may receive
diagnostic, therapeutic, or other interventions, but the investigator does not assign specific
interventions to the study participants. This includes when participants receive interventions
as part of routine medical care, and a researcher studies the effect of the intervention.

2

Attachment 3

26 Oct 2022
Patient Registry
Definition: An observational study that is also considered to be a Patient Registry.
This type of study should only be registered once in the PRS, by the sponsor
responsible for the primary data collection and analysis.
Note: The Agency for Healthcare Research and Quality (AHRQ) defines a Patient
Registry as including an organized system that uses observational methods to collect
uniform data (clinical and other) prospectively for a population defined by a
particular disorder/disease, condition (including susceptibility to a disorder), or
exposure (including products, health care services, and/or procedures) and that serves
a predetermined scientific, clinical, or policy purpose. Patient registries may be single
purpose or on-going data collection programs that address one or more questions.

Expanded Access: An investigational drug product (including biological product) available
through expanded access for patients who do not qualify for enrollment in a clinical trial.
Expanded Access includes all expanded access types under section 561 of the Federal Food,
Drug, and Cosmetic Act: (1) for individual patients, including emergency use; (2) for
intermediate-size patient populations; and (3) under a treatment IND or treatment protocol.
Expanded Access Type *§
Definition: The type(s) of expanded access for which the investigational drug product
(including a biological product) is available, as specified in U.S. Food and Drug
Administration (FDA) regulations. Select all that apply.
•

•
•
•

Not Applicable: Expanded access is for a product other than an
investigational drug product (for example, device product) covered by
FDA expanded access regulations (21 CFR 312)
Individual Patients: For individual participants, including for emergency
use, as specified in 21 CFR 312.310
Intermediate-size Population: For intermediate-size participant
populations, as specified in 21 CFR 312.315
Treatment IND/Protocol: Under a treatment IND or treatment protocol,
as specified in 21 CFR 312.320

2. Study Status
Record Verification Date *
Definition: The date on which the responsible party last verified the information in the entire
expanded access record, even if no additional or updated information is being submitted.
Expanded Access Status *
Definition: The status of availability of the investigational drug product (including a biological
product) through expanded access. Select one.
•
•
•

Available: Expanded access is currently available.
No longer available: Expanded access was available previously but is not currently
available and is not expected to be available in the future.
Temporarily not available: Expanded access was previously available, is not currently
available, but is expected to be available in the future.

3

Attachment 3
•

26 Oct 2022
Approved for marketing: Expanded access was available previously, but is not currently
available because the product has been approved, licensed, or cleared by the U.S. Food
and Drug Administration.

3. Sponsor/Collaborators
Responsible Party, by Official Title *
Definition: An indication of whether the responsible party is the sponsor, the sponsor-investigator, or a
principal investigator designated by the sponsor to be the responsible party. Select one.
•
•
•

Sponsor: The entity (for example, corporation or agency) that initiates the study
Principal Investigator: The individual designated as responsible party by the sponsor (see Note)
Sponsor-Investigator: The individual who both initiates and conducts the study

Note: The sponsor may designate a principal investigator as the responsible party if such principal
investigator meets all of the following requirements: is responsible for conducting the study; has access to
and control over the data from the study; has the right to publish the results of the study; and has the
ability to meet all of the requirements for submitting and updating clinical study information.
Investigator Information [*]
If the Responsible Party, by Official Title is either "Principal Investigator" or "Sponsor-Investigator,"
the following is required:
•
•

•

Investigator Name: Name of the investigator, including first and last name
Investigator Official Title: The official title of the investigator at the primary organizational
affiliation
Limit: 254 characters.
Investigator Affiliation: Primary organizational affiliation of the individual
Limit: 160 characters.

Name of the Sponsor *
Definition: The name of the entity that is the sponsor of the expanded access
Limit: 160 characters.
Note: When a clinical study is conducted under an investigational new drug application (IND) or
investigational device exemption (IDE), the IND or IDE holder is considered the sponsor.
Collaborators
Definition: Other organizations (if any) providing support, including funding, design, implementation,
data analysis and reporting. The responsible party is responsible for confirming all collaborators before
listing them.
Limit: 160 characters.
4. Oversight
Investigational New Drug Application (IND)/Investigational Device Exemption (IDE)
Information

4

Attachment 3

26 Oct 2022

Definition: Complete the following information regarding an IND or IDE for the expanded access as
defined under U.S. Food and Drug Administration (FDA) regulations in 21 CFR 312.3 or 21 CFR
812, respectively.
U.S. Food and Drug Administration IND or IDE *
Definition: Indicate whether there is an IND or IDE for the expanded access. Select Yes/No.(Will not
be made public - for administrative purposes only)
If the investigational product is being made available for expanded access under an IND or IDE, the
following are required:
FDA Center [*]
Definition: The name or abbreviation of the FDA center with which the IND or IDE is filed.
Select one. (Will not be made public - for administrative purposes only.)
▪
▪
▪

CDER: Center for Drug Evaluation and Research
CBER: Center for Biologics Evaluation and Research
CDRH: Center for Devices and Radiological Health

IND or IDE Number [*]
Definition: IND or IDE number assigned by the FDA center. (Will not be made public - for
administrative purposes only.)
IND Serial Number [*]
Definition: For an IND, the IND serial number, as defined in 21 CFR 312.23(e), if any,
assigned to the expanded access. (Will not be made public - for administrative purposes
only.)
5. Study Description
Brief Summary *
Definition: A short description of the availability of expanded access, including the procedure for
requesting the investigational product.
Limit: 5000 characters.
Detailed Description
Definition: Extended description of the expanded access, including more technical information (as
compared to the Brief Summary), if desired. Do not duplicate information recorded in other data
elements, such as Eligibility Criteria.
Limit: 32,000 characters.
6. Conditions and Keywords
Conditions or Focus of Study [*] (Optional if Expanded Access Type is "Individual Patients")
Definition: The name(s) of the disease(s) or condition(s) for which expanded access to the
investigational product is available. Use, if available, appropriate descriptors from NLM's Medical
Subject Headings (MeSH)-controlled vocabulary thesaurus, or terms from another vocabulary, such
as the Systematized Nomenclature of Medicine—Clinical Terms (SNOMED CT), that has been
mapped to MeSH within the UMLS Metathesaurus.

5

Attachment 3

26 Oct 2022

Keywords
Definition: Words or phrases that best describe the expanded access. Keywords help users find
studies in the database. Use NLM's Medical Subject Heading (MeSH)-controlled vocabulary terms
where appropriate. Be as specific and precise as possible. Avoid acronyms and abbreviations.
7. Interventions
Interventions
Definition: Specify the investigational product available for expanded access.
Intervention Type *
Definition: For the investigational product for which expanded access is available, the general
type of intervention. Select one.
•
•
•
•
•
•
•
•
•
•
•

Drug: Including placebo
Device: Including sham
Biological/Vaccine
Procedure/Surgery
Radiation
Behavioral: For example, psychotherapy, lifestyle counseling
Genetic: Including gene transfer, stem cell and recombinant DNA
Dietary Supplement: For example, vitamins, minerals
Combination Product: Combining a drug and device, a biological product and
device; a drug and biological product; or a drug, biological product, and device
Diagnostic Test: For example, imaging, in vitro
Other

Intervention Name(s) *
Definition: A brief descriptive name used to refer to the investigational product that is
available through expanded access. A non-proprietary name of the intervention must be used,
if available. If a non-proprietary name is not available, a brief descriptive name or identifier
must be used.
Limit: 200 characters.
Other Intervention Name(s) [*] (Optional if Expanded Access Type is "Individual
Patients")
Definition: Other current and former name(s) or alias(es), if any, different from the
Intervention Name(s), that the sponsor has used publicly to identify the intervention,
including, but not limited to, past or present names such as brand name(s), or serial numbers.
Limit: 200 characters.
Intervention Description [*] (Optional if Expanded Access Type is "Individual Patients")
Definition: Details that can be made public about each intervention, other than the
Intervention Name(s) or Other Intervention Name(s), sufficient to distinguish the intervention
from other, similar interventions that are available through expanded access or in clinical
studies.
Limit: 1000 characters.
8. Eligibility

6

Attachment 3

26 Oct 2022

Sex/Gender [*] (Optional if Expanded Access Type is "Individual Patients")
Definition: The sex and, if applicable, gender of the patients for whom expanded access is available.
Sex [*]
Definition: The sex of the patients who may obtain expanded access to the investigational
product. Select one.
Note: "Sex" means a person's classification as male or female based on biological
distinctions.
•
•
•

All: Indicates that expanded access is not limited based on the sex of patients
Female: Indicates that only female patients may obtain expanded access
Male: Indicates that only male patients may obtain expanded access

Gender Based [*]
Definition: If applicable, indicate whether expanded access availability is based on gender of
the patient. Select one.
Note: "Gender" means a person's self-representation of gender identity.
•
•

Yes: Expanded access availability is based on gender
No: Expanded access availability is not based on gender
Gender Eligibility Description [*]
Definition: If expanded access availability is based on gender, provide descriptive
information about gender criteria.

Age Limits [*] (Optional if Expanded Access Type is "Individual Patients")
Definition: The minimum and maximum age of patients eligible for the expanded access, provided in
relevant units of time.
Minimum Age [*]
Definition: The numerical value, if any, for the minimum age a patient must meet to be
eligible for the expanded access.
Unit of Time [*]
Select one.
•
•
•
•
•
•
•

Years
Months
Weeks
Days
Hours
Minutes
N/A (No limit)

Maximum Age [*]
Definition: The numerical value, if any, for the maximum age a patient can be to be eligible
for the expanded access.

7

Attachment 3

26 Oct 2022

Unit of Time [*]
Select one.
•
•
•
•
•
•
•

Years
Months
Weeks
Days
Hours
Minutes
N/A (No limit)

Eligibility Criteria [*] (Optional if Expanded Access Type is "Individual Patients")
Definition: A limited list of criteria for determining who is eligible to receive the investigational
product through expanded access, provided in terms of inclusion and exclusion criteria and suitable
for assisting potential patients in identifying investigational products of interest for which expanded
access is available.
Limit: 20,000 characters.
9. Contacts, Locations, and Investigator Information
Central Contact Person *
Definition: The name or official title, toll-free telephone number, and email address of a person to
whom questions concerning expanded access can be addressed. Include the following information:
First Name
Middle Initial
Last Name or Official Title *
Degree
Phone: * Toll free phone number of the Central Contact Person. Use the format 800-5555555 within the United States and Canada. If outside the United States and Canada, provide
the full phone number, including the country code.
Ext: Phone extension, if needed
Email: * Electronic mail address of the central contact person
Central Contact Backup
Definition: Person to contact if Central Contact is not available. Include the following information:
First Name
Middle Initial
Last Name or Official Title
Degree
Phone: Toll free phone number of the Central Contact Backup. Use the format 800-555-5555
within the United States and Canada. If outside the United States and Canada, provide the full
phone number, including the country code.
Ext: Phone extension, if needed
Email: Electronic mail address of the contact person
Overall Study Officials
Definition: Person(s) responsible for the overall scientific leadership of the protocol, including study
principal investigator. Include the following information:

8

Attachment 3

26 Oct 2022

First Name
Middle Initial
Last Name
Degree
Organizational Affiliation: Full name of the official's organization. If none, specify
Unaffiliated.
Limit: 255 characters.
Official's Role: Position or function of the official. Select one.
▪
▪
▪

Study Chair
Study Director
Study Principal Investigator

Facility Information
Definition: For each facility participating in providing expanded access to an investigational product,
the following information:
Facility Name: *§ Full name of the organization where expanded access is available
Limit: 254 characters.
City: *
State/Province: * Required for U.S. locations (including territories of the United States)
ZIP/Postal Code: *§ Required for U.S. locations (including territories of the United States)
Country *
Facility Contact
Definition: For each facility participating in in providing expanded access to an investigational drug
product, the following information:
First Name
Middle Initial
Last Name or Official Title
Degree
Phone: Office phone of the facility contact person.
Ext: Phone extension, if needed
Email: Electronic mail address of the facility contact person
Facility Contact Backup
Definition: Person to contact if Facility Contact is not available (that is, a second contact person).
Investigators (at the expanded access location). Including the following information:
First Name
Middle Initial
Last Name
Degree
Role: Select one.
▪
▪

Site Principal Investigator
Site Sub-Investigator

9

Attachment 3

26 Oct 2022

Contact information character limits:
First Name: 62 characters
Last Name: 62 characters
Degree: 30 characters
Phone: 30 characters
Phone Ext: 14 characters
Email: 254 characters
10. References
Citations
Definition: Citations to publications related to the expanded access: background and/or results.
Provide either the PubMed Unique Identifier (PMID) of an article or enter the full bibliographic
citation.
Limit: 2000 characters.
PubMed Identifier
Definition: PMID for the citation in MEDLINE
Citation
Definition: A bibliographic reference in NLM's MEDLINE format
Limit: 2000 characters.
Results Reference?
Definition: Indicate if the reference provided reports on results from this expanded access.
Select Yes/No.

Links
Definition: A web site directly relevant to the expanded access may be entered, if desired. Do not
include sites whose primary goal is to advertise or sell commercial products or services. Links to
educational, research, government, and other non-profit web pages are acceptable. All submitted links
are subject to review by ClinicalTrials.gov.
URL
Definition: Complete URL, including http:// or https://
Limit: 3999 characters.
Description
Definition: Title or brief description of the linked page.
Limit: 254 characters.
Responsible Party Contact Information *§ (Provided as part of User Information or Organization
Information in a PRS Account)
Definition: Administrative information to identify and enable communication with the responsible
party by telephone, email, and regular mail or delivery service. Responsible Party Contact
Information is for the individual who is the responsible party or of a designated employee of the
organization that is the responsible party. (Will not be made public - for administrative purposes
only.)
10

Attachment 3

26 Oct 2022

Note: "Responsible party" means with respect to a clinical study, the sponsor of the clinical study, as
defined in 21 CFR 50.3; or the principal investigator of such clinical study if so designated by a
sponsor, grantee, contractor, or awardee, so long as the principal investigator is responsible for
conducting the study, has access to and control over the data from the clinical study, has the right to
publish the results of the study, and has the ability to meet all of the requirements for the submission
of clinical study information. For a pediatric postmarket surveillance of a device product that is not a
clinical trial, the responsible party is the entity who FDA orders to conduct the pediatric postmarket
surveillance of the device product.
Name of Individual *
Official Title *
Physical Address *
Name of Organizational Affiliation *
Street Address *
City *
State/Province *
ZIP/Postal Code *
Country *
Mailing Address * (If different from Physical Address)
Name of Organizational Affiliation *
Street Address *
City *
State/Province *
ZIP/Postal Code *
Country *
Phone: * Use the format 800-555-5555 within the United States and Canada. Otherwise,
provide the full number, including the country code.
Ext: Phone extension, if needed
Email: * Electronic mail address
History of Changes
January 18, 2017: Document updated with data element changes per the FDAAA 801 final rule (42
CFR Part 11).
April 18, 2017: Formatting and typographical errors were corrected.
October 1, 2020: Increased field lengths.

11

Attachment 3

26 Oct 2022

OMB NO: 0925-0586
EXPIRATION DATE: 02/28/2023
Burden Statement
Public reporting burden for this collection of information is estimated to vary from 2.0 to 8.0
hours per response for registration, 10.0 to 45.0 hours per response for results information
submissions, and 15 minutes to 2 hours for other submissions including certifications for delay,
extension requests, and expanded access. These estimates include the time for reviewing
instructions, searching existing data sources, gathering the data needed, and completing and
reviewing the collection of information. 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. Send comments regarding this burden estimate or any other aspect of this
collection of information, including suggestions for reducing this burden, to: NIH, Project
Clearance Branch, 6705 Rockledge Drive, MSC 7974, Bethesda, MD 20892-7974, ATTN: PRA
(0925-0586). Do not return the completed form to this address

12

Attachment 3

26 Oct 2022

13

Attachment 3

26 Oct 2022

14

Attachment 3

26 Oct 2022

15

Attachment 3

26 Oct 2022

16

Attachment 3

26 Oct 2022

17

Attachment 3

26 Oct 2022

18

Attachment 3

26 Oct 2022

19

Attachment 3

26 Oct 2022

20

Attachment 3

26 Oct 2022

21