Expanded Access. to Investigational Drugs & Biologics. for Treatment Use

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1 SJMHS Research Compliance Office Guidance Document Expanded Access to Investigational Drugs & Biologics for Treatment Use May

2 Expanded Access to Investigational Drugs & Biologics for Treatment Use The purpose of this document is to help guide SJMHS researchers to information on a) what expanded access is in regard to investigational drugs and biologics, b) which SJMHS IRB form & FDA forms to submit in the process, c) where to find more information on the FDA website, and d) provide a grid summary of the FDA requirements. See separate guidance for devices. A. Expanded Access Use Definition & Types As per the FDA website: Expanded access (some people refer to this as "compassionate use") is the use of an investigational drug outside of a clinical trial (i.e., one that has not been approved by FDA). Wherever possible, use of an investigational drug by a patient as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of products and, consequently, to wider availability. However, when patient enrollment in a clinical trial is not possible (e.g., a patient is not eligible for any ongoing clinical trials, or there are no ongoing clinical trials), patients may be able to receive the product, when appropriate, through expanded access. 21 CFR part 312 subpart I provides general requirements, describes criteria that must be met to authorize expanded access, lists requirements for expanded access submissions, and describes safeguards that will protect patients and preserve the ability to develop meaningful data about the use of the investigational drug. Types of Access Under FDA s current regulations for investigational drugs (including biologics), there are three categories of expanded access: Expanded access for individual patients, including for emergency use; Expanded access for intermediate-size patient populations; and Expanded access for widespread use. Types of FDA Submissions For each of the above: The medical product company can amend its existing IND to include an access protocol or The physician may submit for a new access IND. See Expanded Access Grid for different types of access. In some cases the physician will need to consult with the FDA to determine best course of action. 2

3 B) Forms for Expanded Access Use 1) Emergency Expanded access for individual patient: a) Contact the drug manufacturer and obtain permission to use the drug. The FDA cannot make the drug manufacturer provide the investigational drug. b) Contact the FDA-- In an emergency the FDA may authorize the expanded access use to begin without a written submission to the FDA. The FDA reviewing official may authorize the emergency use by telephone. Refer to the FDA website for further FDA requirements: Emergency Investigational New Drug Application Eligibility Tool (PDF - 45KB) 7 Emergency Investigational New Drug Application Eligibility Tool (text version) 8 For the difference between emergency and non-emergency see: Physician's Checklist for an IND Application for Emergency Treatment (PDF - 102KB) 5 Emergency IND Application Timeline (PDF - 30KB) 6 Investigator-Initiated IND Applications 9 c) If you are seeking expanded access for an individual patient and it meets the criteria for an emergency (and you are authorized for an emergency from the FDA), then complete and submit to the IRB the Permission for Emergency Use form located on the IRB forms page. Please also call us so we are aware of your emergency: Please note: this expanded access use will not be considered research (by DHHS) and will be acknowledged by the IRB, rather than reviewed. The consent will not say "research". It may be appropriate to use treatment consent (should state that this is an investigational drug or biologic, no standard treatment available, and state known risks and that there are unknown risks), given the emergency and a typical research protocol is not normally available. Drug administration is typically submitted to give guidance on how to suspend the investigational drug or biologic. d) Emergency use must be reported to the IRB within 5 working days of its use. An emergency access IND or access protocol must be submitted to the FDA within 15 days of FDA authorization of the use. See steps at end of this document. 2) Expanded access for individual patient: If you are seeking expanded access for an individual patient and your situation is urgent, but not yet an emergency (and you are seeking an individual access IND from or an access protocol from the FDA and, generally, that you have more than a few days before you will need to use the drug or biologic and it is feasible to undergo a full board IRB review in the time frame before use): a) Contact the manufacture of the drug and obtain permission to use the drug. The FDA cannot make the manufacture allow access to the drug. b) Complete necessary paperwork for the FDA. See FDA steps for access INDs, below. Refer to the FDA website for further FDA requirements: 3

4 Investigator-Initiated IND Applications c) Complete and submit to the IRB the New Study Application form located on the IRB forms page. All expanded access use must receive full board review and is considered research. It may be appropriate for your application to be accepted past the deadline to get it to the next IRB meeting, so please contact us to make us aware of your urgent situation: You will also need to meet several FDA requirements - refer to the FDA website. 3) Expanded access for intermediate-size patient populations and 4) Expanded access for widespread use: a) Contact the manufacture of the drug and obtain permission to use the drug. The FDA cannot make the manufacture allow access to the drug. b) Complete necessary paperwork for the FDA. Refer to the FDA website for further FDA requirements: c) Complete and submit to the IRB the New Study Application form located on the IRB forms page. All expanded access use must receive full board review and is considered research. You will also need to meet several FDA requirements - refer to the FDA website. Refer to the FDA website for further requirements: Learn more about expanded access for intermediate-size patient populations and expanded access for widespread treatment use. Investigator-Initiated IND Applications 11 FDA Steps for Access INDs: 1) The physician should ask the medical product company for a Letter of Authorization (LOA), if applicable. An LOA from a company allows the physician submitting the single patient IND to satisfy some of the submission requirements by relying on information in the company s existing IND. It also authorizes FDA to refer to the company s IND when reviewing the single patient IND. 2) Note that the party who (1) submits a request to open an emergency IND application and (2) receives FDA s authorization to use the investigational product is considered the sponsor of the emergency IND application. In the absence of any other sponsor (e.g. pharmaceutical company), the treating physician is the sponsor of the emergency IND application. 3) FDA Forms: Currently, Form FDA 1571 is the appropriate FDA form for submission of all INDs, including individual patient expanded access submissions. Note that the FDA is in the process of developing an alternative form, draft Form FDA 3926, that licensed physicians will be able to use for submitting requests for individual patient expanded access to investigational drugs, including in emergencies. 4

5 C. Additional FDA Guidance on all types of expanded access are located at: IND Applications for Clinical Treatment* (Expanded Access) Overview Contents and Format Treatment of a Single Patient in Emergency Setting Treatment of a Single Patient in Non-emergency Setting Treatment of a Group of Patients * Form FDA 1571 This is the currently approved form for submitting requests for an individual patient expanded access to investigational drugs (including biologics). FDA's Draft Guidance: Expanded Access to Investigational Drugs for Treatment Use - Qs (PDF - 75KB) 21 CFR 312 Subpart I Learn more about FDA s current expanded access regulations for investigational drugs (including biologics). D. Expanded Access to Investigational Drugs (including Biologics) for Treatment Use (starts on the next page) 5

6 SJMHS Research Compliance Office Guidance Document Expanded Access to Investigational Drugs (including Biologics) for Treatment Use 21 CFR (Subpart I) This grid reflects a summary of the FDA requirements for the use of investigational new drugs and approved drugs where availability is limited by a risk evaluation and mitigation strategy (REMS) & when the primary purpose is to diagnose, monitor, or treat a patient's disease or condition. The aim is to facilitate the availability of such drugs to patients with *serious diseases or conditions when there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the patient's disease or condition. See full regulations for more info. Note: the term Compassionate Use has been used by some to refer to expanded access. Expanded Access Type Expanded Access for Individual Patient (Single Patient Access) Reg 21 CFR CFR CFR Criteria for Use- FDA & Physician must determine (1) *Serious or immediately lifethreatening disease or condition, & there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; (2) The potential patient benefit justifies the potential risks of the treatment use & those potential risks are not unreasonable in the context of the disease or condition (probable risk to the person from the investigational drug is not greater than the probable risk from the disease or condition); and (3) Providing the investigational drug will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval or otherwise compromise the potential development of the expanded access use. (4) FDA must determine that the patient cannot obtain the drug under another IND or protocol. Note: Treatment is generally limited to a single course of therapy for a specified duration unless FDA expressly authorizes multiple courses or chronic therapy. FDA Approval Needed? Yes: (1) The medical product company must agree to provide the investigational drug for expanded access use. FDA cannot require the company to do this. (2) A sponsor may satisfy the submission requirements by amending its existing IND to include a protocol for individual patient expanded access. (3) A licensed physician may satisfy the submission requirements by obtaining from the sponsor permission for FDA to refer to any information in the IND that would be needed to support the expanded access request (right of reference) and by providing any other required information not contained in the IND (usually only the information specific to the individual patient). IRB Review & Approval Needed? Yes; Submit a New Project Application form plus FDA approval letter, sponsor acknowledgement letter, and consent with HIPAA- see below. FDA Follow-up Submit IND safety reports promptly, and annual reports if the protocol continues for one year or longer. (2) At the conclusion of treatment, the licensed physician or sponsor must provide FDA with a written summary of the results of the expanded access use, including adverse effects. (3) FDA may require sponsors to monitor an individual patient expanded access use if the use is for an extended duration. (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 or

7 Expanded Access Type Reg Criteria for Use- FDA & Physician must determine FDA Approval Needed? IRB Review & Approval Needed? FDA Follow-up Emergency Expanded Access for Individual Patient 21 CFR CFR CFR CFR (d) (1) Emergency use means the use of a test article on a human subject in a life-threatening situation in which no standard acceptable treatment is available, and in which there is not sufficient time to obtain IRB approval [review at a convened meeting is not feasible]. (2) *Serious or immediately lifethreatening disease or condition, & there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; (3) Potential patient benefit justifies the potential risks of the treatment use & those potential risks are not unreasonable in the context of the disease or condition (4) Probable risk to the person from the investigational drug is not greater than the probable risk from the disease or condition); (5) Providing the investigational drug will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval or otherwise compromise the potential development of the expanded access use. (6) FDA must determine that the patient cannot obtain the drug under another IND or protocol. (7) One use per institution, but there is some leeway**. See note at end of document. Yes: 1) Contact sponsor to obtain permission to use and obtain test article. 2) You will need to gain an emergency IND from the FDA or amendment to existing IND. In an emergency the FDA may authorize the expanded access use to begin without a written submission to the FDA. The FDA reviewing official may authorize the emergency use by telephone. For investigational biological drug products under Center for Biologics Evaluation and Research, physician contacts the FDA's Office of Communication, Outreach & Development, or , ocod@fda.hhs.gov tell them it is an emergency For all other investigational drugs, physician contacts the Division of Drug Info: , druginfo@fda.hhs.gov tell them it is an emergency. After hours (8 a.m. to 4:30 p.m.), contact FDA Emergency Call Center: , emergency.operations@fda.hhs.gov (2) The physician or sponsor must explain how the expanded access use will meet the requirements of and and must agree to submit an expanded access submission within 15 working days of FDA's authorization of the use. Emergency Use is exempt from IRB review per FDA reg, 'provided that such emerg. use is reported to the IRB within 5 working days. Any further use of the drug at SJMHS is subject to IRB review.' Emergency use is not research under 45 CFR 46***. However, the IRB must still acknowledge the use & assess whether or not the conditions for the emergency use were satisfied (misuse will be evaluated for noncompliance). Submit the Permission for Emergency Use form to the IRB within 5 working days after the use. You may report prior to use, as well. See consent options at end of grid. It may be appropriate to use treatment consent, given the emergency and a typical research protocol is not normally available. Drug administration is typically submitted to give guidance on how to suspend the investigational drug or biologic. FDA: Physician or sponsor must submit a new emergency access IND, or amendment to an existing IND (access protocol), within 15 working days of FDA s authorization of the use. Clearly mark Emergency IND on top of Form 1571 (new form coming soon). (21 CFR )] IRB: Report within 5 days of use: consent process or exemption criteria, adverse events, and outcome of use. 7

8 Expanded Access Type Expanded Access for Intermediate Size Patient Population Reg 21 CFR CFR CFR Criteria for Use- FDA & Physician must determine (1) *Serious or immediately lifethreatening disease or condition, & there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; (2) The potential patient benefit justifies the potential risks of the treatment use & those potential risks are not unreasonable in the context of the disease or condition; and (3) Providing the investigational drug will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval or otherwise compromise the potential development of the expanded access use. FDA Approval Needed? IRB Review & Approval Needed? FDA Follow-up Yes: The submission may be a new access IND or a protocol amendment to an existing IND (access protocol). Information required for a submission may be supplied by referring to pertinent information contained in an existing IND if the sponsor of the existing IND grants a right of reference to the IND. See (b)2&3 for detailed list of submission requirements. Yes; Submit a New Project Application form plus FDA approval letter, sponsor acknowledgement letter, and consent with HIPAA Submit IND safety reports promptly, and annual reports if the protocol continues for one year or longer. (4) There is enough evidence that the drug is safe at the dose & duration proposed for expanded access use to justify a clinical trial of the drug in the approximate # of patients expected to receive the drug under expanded access; and (5) There is at least preliminary clinical evidence of effectiveness of the drug, or of a plausible pharmacologic effect of the drug to make expanded access use a reasonable therapeutic option in the anticipated patient population. 8

9 Expanded Access Type Expanded Access for Large Population/ Wide-Spread Use Under a Treatment IND or Treatment Protocol Reg Criteria for Use- FDA & Physician must determine FDA Approval Needed? 21 CFR CFR CFR (1) *Serious or immediately life-threatening disease or condition, & there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; (2) The potential patient benefit justifies the potential risks of the treatment use & those potential risks are not unreasonable in the context of the disease or condition; and (3) Providing the investigational drug will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval or otherwise compromise the potential development of the expanded access use. (4) Trial status (i) The drug is being investigated in a controlled clinical trial under an IND designed to support a marketing application for the expanded access use, or (ii) All clinical trials of the drug have been completed; and (5) Marketing status. The sponsor is actively pursuing marketing approval of the drug for the expanded access use with due diligence; and (6) Evidence. (i) When the expanded access use is for a serious disease or condition, there is sufficient clinical evidence of safety and effectiveness to support the expanded access use. Such evidence would ordinarily consist of data from phase 3 trials, but could consist of compelling data from completed phase 2 trials; or (ii) When the expanded access use is for an immediately lifethreatening disease or condition, the available scientific evidence, taken as a whole, provides a reasonable basis to conclude that the investigational drug may be effective for the expanded access use and would not expose patients to an unreasonable and significant risk of illness or injury. This evidence would ordinarily consist of clinical data from phase 3 or phase 2 trials, but could be based on more preliminary clinical evidence. Yes: The submission may be a new access IND or a protocol amendment to an existing IND (access protocol). Information required for a submission may be supplied by referring to pertinent information contained in an existing IND if the sponsor of the existing IND grants a right of reference to the IND. See (b)2&3 for detailed list of submission requirements. IRB Review & Approval Needed? Yes; Submit a New Project Application form plus FDA approval letter, sponsor acknowledgement letter, and consent with HIPAA FDA Follow-up Submit IND safety reports promptly, and annual reports if the protocol continues for one year or longer. Note: The sponsor is responsible for monitoring the treatment protocol to ensure that licensed physicians comply with the protocol and the regulations applicable to investigators. 9

10 Informed Consent Options for all Expanded Access: Informed consent (and HIPAA Authorization) must be obtained from the subject (or the legally authorized representative), unless the requirements of an exception from the informed consent requirement [21 CFR 50.23(a)] are satisfied, see below. Exception from Informed Consent Requirement [21 CFR 50.23(a)] FDA regulations [21 CFR 50.23] permit emergency use of a test article without informed consent where the investigator and an independent physician, who is not otherwise participating in the clinical investigation, certify in writing: a) The patient is confronted by a life-threatening or severely debilitating situation, necessitating the use of the test article b) Informed consent cannot be obtained from the patient (because the patient cannot communicate or is incompetent to give consent) c) Time is not sufficient to obtain consent from the patient s legally authorized representative d) No alternative method of approved or generally recognized therapy is available that provides an equal or greater likelihood of saving the patient s life. If, in the investigator's opinion, immediate use of the test article is required and if time is not sufficient to obtain the independent physician determination, the investigator should make the determination and, within 5 working days after the use of the article, have the determination reviewed and evaluated in writing by an independent physician. *FDA Definitions: Immediately life-threatening disease or condition means a stage of disease in which there is reasonable likelihood that death will occur within a matter of months or in which premature death is likely without early treatment. Serious disease or condition means a disease or condition associated with morbidity that has substantial impact on day-to-day functioning. Short-lived and self-limiting morbidity will usually not be sufficient, but the morbidity need not be irreversible, provided it is persistent or recurrent. Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one. Emergency use means the use of a test article on a human subject in a life-threatening situation in which no standard acceptable treatment is available, and in which there is not sufficient time to obtain IRB approval. See the FDA Q & A document for more explanation: Expanded Access to Investigational Drugs for Treatment Use - Qs (PDF - 75KB) **One Emergency Use per Investigational Drug or Biologic: The FDA regulations [21 CFR (c)] allows for one emergency use of an investigational drug or biologic at an institution. Any subsequent use of the investigational drug or biologic at the institution is subject to prospective IRB review and approval. However, the FDA acknowledges that it would be inappropriate to deny emergency treatment to a second individual if the only obstacle is that the IRB has not had sufficient time to convene a meeting to review the issue (FDA Information Sheet, 2003 Update). ***Emergency Use is Not Considered Research under DHHS Regulations The FDA regards emergency use of an investigational drug or biologic as a clinical investigation and may require data from an emergency use to be reported in a marketing application. However, the DHHS states, emergency care may not be claimed as research, nor may the outcome of such care be included in any report of a research activity. Therefore, a person receiving an emergency use of a drug or biologic is not considered a research participant by DHHS regulation, and such emergency use is not research as covered under 45 CFR

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