§312.315. Intermediate-size patient populations. — Inbound Citations

(c) A sponsor shall submit a protocol amendment when a new investigator is added to carry out a previously submitted protocol, except that a protocol amendment is not required when a licensed practitioner is added in the case of a treatment protocol under § 312.315 or § 312.320. Once the investigator is added to the study, the investigational drug may be shipped to the investigator and the investigator may begin participating in the study. The sponsor shall notify FDA of the new investigator within 30 days of the investigator being added.

The criteria, submission requirements, safeguards, and beginning treatment information set out in this section apply to all expanded access uses described in this subpart. Additional criteria, submission requirements, and safeguards that apply to specific types of expanded access are described in §§ 312.310 through 312.320.

(4) When a significant number of similar individual patient expanded access requests have been submitted, FDA may ask the sponsor to submit an IND or protocol for the use under § 312.315 or § 312.320.

(2) For expanded access to an investigational drug for treatment use under §§ 312.315 (intermediate-size patient populations) and 312.320 (treatment IND or treatment protocol), in addition to the direct costs described in paragraph (d)(1)(i) of this section, a sponsor may recover the costs of monitoring the expanded access IND or protocol, complying with IND reporting requirements, and other administrative costs directly associated with the expanded access IND.

(i) An indication of whether there is expanded access to the investigational drug product (including a biological product) under section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) for those individuals who do not qualify for enrollment in the applicable clinical trial, under one or more of the following types of expanded access programs: for individual patients, including for emergency use, as specified in 21 CFR 312.310; for intermediate-size patient populations, as specified in 21 CFR 312.315; or under a treatment IND or treatment protocol, as specified in 21 CFR 312.320; and

(c) If expanded access is available, as specified in 21 CFR 312.315 (for an intermediate-size patient population) or 21 CFR 312.320 (under a treatment IND or treatment protocol), for an investigational drug product (including a biological product) studied in an applicable drug clinical trial, and the data elements set forth in paragraphs (c)(1) through (4) of this section have not been submitted in an expanded access record for that investigational product, the responsible party, if both the manufacturer of the investigational product and the sponsor of the applicable clinical trial, must submit the clinical trial information specified in paragraphs (c)(1) through (4) of this section to ClinicalTrials.gov in the form of an expanded access record. If expanded access is available only as specified in 21 CFR 312.310 (for individual patients, including for emergency use) for an investigational drug product (including a biological product) studied in an applicable drug clinical trial, and the data elements set forth in paragraphs (c)(1)(i), (iii), (iv), (vi), (ix), (x), (c)(2)(iv), (c)(3), (c)(4)(i), (iii),(iv), and (v) of this section have not been submitted in an expanded access record for that investigational product, the responsible party, if both the manufacturer of the investigational product and the sponsor of the applicable clinical trial, must submit the clinical trial information specified in those paragraphs to ClinicalTrials.gov in the form of an expanded access record.

(i) A short title identifying the expanded access, written in language intended for the lay public. If an acronym or abbreviation is used publicly to identify the expanded access, it must be provided.

(ii) The title, if any, of the expanded access program corresponding to the title that has been submitted to FDA for that program

(iii) A short description of the availability of expanded access, including the procedure for requesting the investigational drug product (including a biological product).

(iv) The nature of the investigation or investigational use for which clinical trial information is being submitted, i.e., “expanded access”.

(v) The name(s) of the disease(s) or condition(s) for which expanded access to the investigational drug product (including a biological product) is available. Use, if available, appropriate descriptors from NLM's MeSH-controlled vocabulary thesaurus, or terms from another vocabulary, such as the SNOMED CT, that has been mapped to MeSH within the UMLS Metathesaurus.

(vi) A brief descriptive name used to refer to the investigational drug product (including a biological 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.

(vii) Any other current and former name(s) or alias(es), 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.

(viii) 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 trials.

(ix) For each investigational drug product (including a biological product) for which expanded access is available, the general type of intervention, e.g., drug.

(x) The type(s) of expanded access for which the investigational drug product (including a biological product) is available, as specified in § 11.10(b)(28).

(i) A limited list of criteria for determining who is eligible to receive the investigational drug product (including a biological product) through expanded access, provided in terms of inclusion and exclusion criteria and suitable for assisting potential patients in identifying investigational drug products (including biological products) of interest for which expanded access is available.

(ii) The sex and gender (if applicable) of the patients for whom expanded access is available.

(iii) The minimum and maximum age of patients for whom expanded access is available, provided in relevant units of time.

(iv) The status of availability of the investigational drug product (including a biological product) through expanded access.

(ii) The official name of the entity.

(iii) The name or official title, toll-free telephone number, and email address of a person to whom questions concerning expanded access can be addressed.

(i) Any unique identifier assigned by the sponsor to refer to the availability of its investigational drug product (including a biological product) for expanded access use or to identify the expanded access record.

(A) Any identifier(s) other than the Unique Protocol Identification Number or the NCT number that is assigned to the expanded access record, including any unique identifiers assigned by other publicly available clinical trial or expanded access registries.

(iii) An indication of whether there is an IND and, if so, each of the following elements:

(A) Name or abbreviation of the FDA center with whom the IND is filed (i.e., CDER or CBER), if applicable;

(B) IND number (assigned by the FDA center) under which the investigational drug product (including a biological product) is being made available for expanded access, if applicable; and

(C) as defined in 21 CFR 312.23(e), if any, assigned to the expanded access.

(iv) The date on which the responsible party last verified the information in the expanded access record, even if no additional or updated information was submitted at that time.

(v) Administrative information sufficient to identify and allow communication with the responsible party entering the clinical trial information into the expanded access record by telephone, email, and regular mail or delivery service. Responsible Party Contact Information includes the name, official title, organizational affiliation, physical address, mailing address, phone number, and email address of the individual who is the responsible party or of a designated employee of the organization that is the responsible party.