Form 5 Expanded Access Record Data Element Definitions (referen

Attachment 3

21 Dec 2016

ClinicalTrials.gov Registration Data Element Definitions
for Expanded Access (DRAFT)
January 18, 2017

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

[*]

Conditionally required

Required if initial submission Date is on or after January 18, 2017

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
Defintion: A short title identifying the expanded access, written in language intended for the

1

Attachment 3

21 Dec 2016

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
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), 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 ID).

Description [*]
Definition: If a Secondary ID Type of "Other Grant/Funding Number," "Registry

2

Attachment 3

21 Dec 2016

Identifier," or "Other Identifier" is selected, provide the name of the funding
organization, clinical trial registry, or organization that issued the identification
number.

*

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
ClinicalTrials.gov Protocol Registration Data Element Definitions for Interventional and
Observational Studies).
•

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.
•

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 ongoing 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
3

Attachment 3

21 Dec 2016

(including a biological product) is available, as specified in FDA regulations. Select all that
apply.
•
•
•
•

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
Not applicable: Expanded access is for a product (for example, device product)
other than an investigational drug product covered by FDA expanded access
regulations (21 CFR 312)

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.
Approved for Marketing: Expanded access was available previously, but is not
currently available because the product has been approved, licensed, or cleared by
FDA.

3. Sponsor/Collaborators

*

Responsible Party, by Official Title
Definition: An indication of whether the responsible party is the sponsor, the sponsorinvestigator, or a principal investigator designated by the sponsor to be the responsible party.
Select one.

4

Attachment 3

•
•
•

21 Dec 2016

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 "SponsorInvestigator," 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
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
Definition: Complete the following information regarding an IND or IDE for the expanded
access as defined under U.S. FDA regulations in 21 CFR 312.3 or 21 CFR 812, respectively.

5

Attachment 3

21 Dec 2016

*

U.S. Food and Drug Administration IND or IDE Number
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 is 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.)
o
o
o

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
Primary Disease or Condition [*] (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

6

Attachment 3

21 Dec 2016

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.
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.

7

Attachment 3

21 Dec 2016

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
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.
•
•
•

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

Gender [*]
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

8

Attachment 3

21 Dec 2016

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.
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

9

Attachment 3

21 Dec 2016

products of interest for which expanded access is available.
Limit: 15,000 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
800-555-5555 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 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 800555-5555 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:
First Name
Middle Initial
Last Name
Degree

10

Attachment 3

21 Dec 2016

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: [*]
ZIP/Postal Code: [*]
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
11

Attachment 3

21 Dec 2016

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
Affiliation: 160 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: PubMed Unique Identifier (PMID) for the citation in MEDLINE
Citation
Definition: A bibliographic reference in NLM's MEDLINE format
Results Reference?
Definition: Indicate if the reference provided reports on results from this expanded
access.

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.

12

Attachment 3

21 Dec 2016

*

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.)
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

*

*

*

*
• ZIP/Postal Code *
• Country *
Mailing Address * (If different from Physical Address)
• Name of Organizational Affiliation *
• Street Address *
• City *
•

•

*

State/Province

13

Attachment 3

21 Dec 2016

•

*
• 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:

•

State/Province

* Electronic mail address

History of Changes
•

2017-01-18: Document updated with data element changes per the FDAAA 801 final
rule (42 CFR Part 11).

14

Attachment 3

21 Dec 2016

15

Attachment 3

21 Dec 2016

16

Attachment 3

21 Dec 2016

17

Attachment 3

21 Dec 2016

18

Attachment 3

21 Dec 2016

19

Attachment 3

21 Dec 2016

20