Expanded Access Data Element Definitions

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 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 BDCTR ID 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).
• EU Trial (CTIS) Number: Identifier assigned by the European Union (EU) and European Economic Area (EEA).
• 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.

  • 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

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 the U.S. Food and Drug Administration.

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.

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.

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. 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.)

Study Description

Brief Summary *

Definition: A short description of the availability of expanded access, including the procedure for requesting the investigational product.
Limit: 5,000 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.

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.

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.

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: 1,000 characters.

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.

• 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.

• 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.

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 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:

• 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 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

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

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: 2,000 characters.

• PubMed Identifier

  Definition: PMID for the citation in MEDLINE

• Citation

  Definition: A bibliographic reference in NLM's MEDLINE format
  Limit: 2,000 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 BMU CTR.

• URL

  Definition: Complete URL, including http:// or https://
  Limit: 3,999 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.)

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.

January 9, 2024: Made formatting changes by replacing the section numbers with a left-sided menu for navigation. This was done as part of the process of moving the data element definitions pages to the modern BMU CTR site.

June 17, 2024: Added 'EU Trial (CTIS) Number' to options for Secondary ID. Added 'No symbol' to the requirements legend to indicate data elements that are Optional. Corrected typographical errors.