No, manufacturers or Sponsors accepting requests for single-patient investigational new drug applications INDs or protocol exceptions including for emergency use should provide only one expanded access record.
When sponsors or their representatives register to become PRS data providers, they will be given information on using PRS, including instructions for creating additional user accounts.
Can registration and results information be uploaded electronically to ClinicalTrials. You must use ClinicalTrials. FDAAA applies to any clinical study that meets the definition of an Applicable Clinical Trial and that was initiated after September 27, , or that was initiated on or before that date and was still ongoing as of December 26, FDAAA does not distinguish between types of sponsors or funding sources in establishing requirements for registration and results submission.
Am I required to submit to ClinicalTrials. Under the Voluntary Submissions PDF provision, a Responsible Party who submits results for such a clinical trial must submit complete clinical trial results information and must also submit results for each Applicable Clinical Trial that is required to be submitted to FDA under Sections , k , , and m of the Federal Food, Drug, and Cosmetic Act or Section of the Public Health Service Act for the same use studied.
How do I submit results information if the trial is terminated that is, stopped prematurely and no data were collected for one or more Outcome Measures? If no participants were ever enrolled in the trial, set the Overall Recruitment Status to Withdrawn, and no further results information will need to be submitted.
For a trial that was terminated after participants were enrolled, provide any available data. In this case, provide an explanation in the Analysis Population Description for why zero participants were analyzed and, if appropriate, provide information in the Limitations and Caveats module.
Even if data are not entered for Outcome Measures, submit the available data for the enrolled participants in the Participant Flow, Baseline Characteristics, and Adverse Events modules. How do I determine if my study is an applicable clinical trial? Beyond their primary purpose, the ACT Checklist and Elaboration may also be useful to assist in evaluating whether a clinical trial or study that was initiated before January 18, , and which is not subject to the final rule requirements, is an ACT under section j of the Public Health Service Act.
We note that Responsible Parties or other users of the ACT Checklist and Elaboration are responsible for using accurate data about a clinical trial or study and for properly evaluating whether the trial or study must be registered and, if so, which results must be submitted. The outcome generated from the ACT Checklist will not be retained by the National Institutes of Health NIH and will not be binding on either the user or any government agency in any future action s.
If a clinical trial is not an applicable clinical trial ACT at study initiation because it is conducted entirely outside the United States, but the trial subsequently opens a U. If a sponsor of a clinical trial in a foreign country that does not meet the definition of an ACT, and has an initiation date after the effective date of the regulations in 42 CFR Part 11, decides to add a location in the U.
The requirements set forth in the regulation would need to be met, beginning with registration of the ACT not later than 21 days after the enrollment of the first participant at the U. Per 42 CFR Clinical trial registration information must include information applicable to the entire trial, as is the case with all multi-site trials with information in ClinicalTrials.
What is the definition of a drug, biological, or device product under investigation being "manufactured" in the United States? A clinical trial or study initiated on or after January 18, , that meets certain conditions in 42 CFR One of these conditions is whether the drug, biological, or device product "under investigation is a Product Manufactured in and Exported from the U.
The agency explained that the term "manufacture" is used as a "short-hand for all device [or drug] activities within FDA's jurisdiction.
In addition, the drug, biological, or device product "under investigation" as described in 42 CFR If a drug, biological, or device product is tested in conjunction with, or compared to, one or more other drug, biological, or device products including a placebo or sham , then the products would be considered "under investigation" for purposes of this ACT condition.
How do I know if my clinical trial "Studies a U. Specifically, for a trial conducted entirely outside the U. The regulation defines both "U. FDA-regulated device product" and "U. The regulation further defines these specific concepts as data elements in 42 CFR FDA-regulated Device Product" and The Final Rule preamble states: "[A] clinical study of a device product that is being conducted entirely outside of the United States i.
Therefore, a study record that 1 does not list "United States" or a U. For such a study, the responsible party would list "No" for the Studies a U.
FDA-regulated Device Product data element and the study would not be considered an applicable device clinical trial. Note that even if the device product being studied had previously been approved or cleared by the U.
The Final Rule preamble states: "[A] clinical investigation of a drug product including a biological product that is being conducted entirely outside of the United States i.
For such a study, the responsible party would answer "No" to the Studies a U. FDA-regulated Drug Product data element and the study would not be considered an applicable drug clinical trial. Note that even if the drug or biologic product being studied had previously been approved by the U. Example: For a clinical study conducted entirely outside of the United States or its territories, in which the drug, biological, or device product is not studied under an IND or IDE, and the studied drug, biological, or device product is manufactured outside of the United States or its territories, then the studied product would not be considered "FDA-regulated" under the relevant condition in 42 CFR The responsible party of such a study would select "No" for the data elements of Studies a U.
Is a clinical trial that uses a radiation-emitting product considered to be a trial that "Studies a U. To assess whether a clinical trial using a radiation-emitting product "Studies a U.
In explaining the "applicable device clinical trial" definition in 42 CFR In addition, for the clinical trial to be an "applicable device clinical trial," the trial must meet the definition at 42 CFR For example, magnetic resonance diagnostic devices and medical charged-particle radiation therapy systems are designated in 21 CFR The Final Rule preamble also explains that radiation-emitting products can be subject to the requirements in 42 CFR Part "For example, a clinical trial for which a responsible party indicates the Intervention Type is 'radiation,' 'genetic,' or 'procedure' could in fact be an applicable device clinical trial studying a device product subject to section k , , or m of the FDC Act e.
In addition, the Final Rule preamble explains that when considering whether a clinical trial "studies" a device product, the responsible party should consider whether a the study is designed to examine the effect or performance of an FDA-regulated device product or differences in the intended use, for example, variations in frequency of use, method of administration, design specifications, and other characteristics e. Example: A clinical trial that assesses the safety or efficacy of different radiation doses emitted from a device product previously approved or cleared by the U.
For such a study, the responsible party would list "Yes" for the Studies a U. FDA-regulated Device Product data element. The trial would be an "applicable device clinical trial," so long as it is interventional, the primary purpose is not a feasibility study, and it also meets one or more of the following conditions: 1 at least one study facility is located in the U.
The regulations in 42 CFR For those applicable device clinical trials that were initiated on or after January 18, that meet the conditions specified in 42 CFR The determination of whether the study of a specific device product is an applicable device clinical trial does not depend on the product's device classification in 21 CFR Is a study coordinating center located in the United States considered to be a "Facility Location" within the United States for evaluating whether a study is an applicable clinical trial?
A clinical trial enrolling participants only at locations outside the United States, but using a study coordinating center in the United States with no participants enrolled at the coordinating center location itself , would not be considered to have a "Facility Location" in the United States. One of these criteria is met if the clinical trial has "at least one Facility Location … within the United States or one of its territories" 42 CFR It refers to the city, state, country, and Zip Code for U.
Whether a site is considered a "participating facility" is dependent on whether participants can enroll at that site; this is reflected in 42 CFR A study coordinating center that may provide study oversight or data management and analysis support, but that does not enroll participants at that location, would not be considered a "participating facility" as described in Facility Location.
Therefore, the coordinating center would not be considered a Facility Location for the purposes of 42 CFR Are "pilot" drug or device studies considered to be an "applicable drug clinical trial" or "applicable device clinical trial," respectively, under the regulation? It depends. The terms "pilot" drug or device study are not interchangeable with the terms "phase 1" drug study or "feasibility" device study, respectively.
The regulation does not identify "pilot" studies in defining "applicable drug clinical trial" and "applicable device clinical trial" in 42 CFR Therefore, the characteristics of each individual clinical trial of a drug, biological, or device product must be evaluated to determine whether it meets the applicable clinical trial definition, independent of whether the responsible party considers the trial to be a "pilot" study. We note that the definition of applicable drug clinical trial in 42 CFR The regulation states that phase 1 has the meaning given in 21 CFR In summary, a phase 1 trial includes the initial introduction of an investigational new drug into humans and is designed to determine the metabolism and pharmacologic actions of the drug in humans, the side effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness.
Phase 1 trials also include studies of drug metabolism, structure-activity relationships, and mechanism of action in humans, as well as studies in which investigational drugs are used as research tools to explore biological phenomena or disease processes. We note that these provisions in the regulation do not use the term "pilot. Similarly, the definition of "applicable device clinical trial" in 42 CFR The regulations at 42 CFR The regulations specify that the sponsor of the trial will be considered the responsible party unless and until a principal investigator has been designated the responsible party in accordance with 42 CFR For clinical trials not conducted under an IND or IDE, the sponsor is considered to be the person or entity who initiated the trial and would be identified as follows: Where the clinical trial is being conducted by an entity under a research assistance funding agreement such as a grant or sponsored research agreement, the funding recipient generally is considered to be the initiator of the clinical trial, and therefore, the sponsor.
This is because, as a general rule, when a clinical trial is funded in this manner, the funding recipient "initiates" the clinical trial process by, for example, submitting a funding proposal and designing the clinical trial. Where the clinical trial is being conducted by an entity under a procurement funding agreement such as a contract, the party obtaining the goods or services for its direct benefit or use the funder generally is considered to be the initiator of the trial, and therefore, the sponsor.
This is because, as a general rule, when a clinical trial is funded in this manner, it is the funder of the clinical trial that initiates the clinical trial process by, for example, contracting with another entity for that entity to conduct a clinical trial meeting the specifications of the funder. The sponsor of the clinical trial, whether an individual or entity, is the responsible party, unless the principal investigator has been designated the responsible party in accordance with 42 CFR The principal investigator may be designated by a sponsor, so long as the principal investigator is responsible for conducting the trial, has access to and control over the data from the clinical trial, has the right to publish the results of the trial, and has the ability to meet all of the requirements under the regulation for submitting and updating of clinical trial information.
If the principal investigator does not meet the specified conditions for serving as the responsible party, the sponsor cannot designate the principal investigator as the responsible party, and the sponsor must remain the responsible party. The format for designation of the principal investigator as the responsible party by the sponsor is specified in ClinicalTrials.
The acknowledgement is reflected by having the principal investigator list their name as the responsible party and indicate that they were designated as the responsible party by the sponsor. If and when a designated principal investigator no longer meets or is no longer able to meet all of the requirements of a responsible party, 42 CFR This might occur if, for example, a principal investigator dies, retires, changes jobs, or turns control of the clinical trial data over to the sponsor.
The final rule clarifies that expanded access EA use of a drug, biological, or device product is not considered an "applicable clinical trial" ACT under the definition in 42 CFR Thus, the submission of clinical trial registration and results information for EA use would not be required by 42 CFR However, for "applicable drug clinical trials" that are required to submit the registration information specified in 42 CFR In addition, an expanded access record must be submitted as required under 42 CFR More information about the expanded access submission requirements is available in the final rule preamble.
Who is the responsible party for a pediatric postmarket surveillance of a device product that is not a clinical trial? If my study is an applicable clinical trial ACT , am I required to register? What registration information must I submit if my applicable clinical trial is required to be registered?
As outlined in the table, required registration information is determined by the study initiation date. The initiation date is the date on which the trial is initiated i.
When must I submit the required clinical trial registration information? The final rule in 42 CFR For an applicable device clinical trial that is a pediatric postmarket surveillance of a device product and is not a clinical trial, the registration information specified in section j 2 A ii of the PHS Act or 42 CFR How are examinations by telephone call or other electronic means considered in determining when an applicable clinical trial reaches its "primary completion date" or "study completion date" under the regulation?
At what point is a human subject considered to be "enrolled" in an applicable clinical trial? The regulation explains that, for the purposes of this part, potential subjects who are screened for the purpose of determining eligibility for a trial, but do not participate in the trial, are not considered enrolled, unless otherwise specified by the protocol. The Enrollment data element is defined in 42 CFR That regulation further explains that once the trial has reached the primary completion date, the responsible party must update the Enrollment data element to reflect the actual number of human subjects enrolled in the clinical trial.
The first scenario involves the use of a separate informed consent document for screening. In this situation, there are two distinct informed consent documents: one for trial screening for eligibility and if eligible, one for trial participation. Under this first scenario, the signing of the second separate informed consent document for trial participation would mean the subject is enrolled in the clinical trial.
In the second scenario, there is only one informed consent document for both trial screening and trial participation. In this scenario, the Final Rule preamble explains that a participant would not be considered enrolled until he or she met all the eligibility criteria assessed during screening, unless the participant is considered enrolled as outlined specifically in the protocol.
Based on this clarification, when there is only one informed consent document for both trial screening and trial participation, whether a human subject "participates" in a study, and is therefore considered "enrolled" under the definition in 42 CFR This determination may vary across clinical trial protocols.
For example, assignment to a study arm may be considered the beginning of trial participation based on a particular study protocol. In this example, if the study was halted prematurely before any subjects were assigned to a study arm i. Also, if a human subject signs the informed consent document but then withdraws his or her informed consent before participation begins, the subject would not be considered "enrolled" in the clinical trial under the definition.
We also note that the definition of "enrolled" is important for determining the Study Start Date. The Primary Completion Date is the date that the final study participant was examined or received an intervention for the purpose of the final collection of data for the primary outcome. Similarly, the Study Completion Date is the date that the final study participant was examined or received an intervention for the purpose of the final collection of data for the primary and secondary outcome measures and adverse events see the definitions in 42 CFR The date that the final study participant was examined or received an intervention for the purpose of the final collection of data is the date of the examination or the administration of the intervention itself, not the date of any later assessment, analysis, or interpretation of the collected outcome or adverse event data.
For example, if a participant was examined with a magnetic resonance imaging MRI scan for the primary outcome, the Primary Completion Date is the date that the last participant underwent the MRI, and not when the MRI was subsequently assessed using a central reading process or other review procedure.
More information on this point is available in the Final Rule preamble. Am I required to submit results information for my applicable clinical trial ACT? The regulation at 42 CFR Am I required to submit results information for my applicable clinical trial ACT if the primary completion date was before January 18, the effective date of the Final Rule? If so, when? For ACTs subject to FDAAA's registration and results submission requirements that have a primary completion date before January 18, the effective date of the Final Rule , results information must be submitted as follows: If the ACT studied a drug, biological, or device product that was approved, licensed, or cleared by FDA before the ACT's primary completion date, the responsible party generally must submit the results information specified in section j 3 C and section j 3 I of the Public Health Service PHS Act no later than 1 year after the study's primary completion date; however, results information submission for these ACTs may be delayed under certain conditions as specified in section j 3 E v seeking approval, licensing, or clearance of a new use for the drug, biological, or device product and section j 3 E vi requesting an extension for good cause of the PHS Act.
Pursuant to the Federal Court decision in Seife et al. HHS et al. If the results submission deadline has already passed for an ACT affected by the Federal Court decision in Seife et al. The term "participant" is used to refer to human subjects. Limit: 30 characters. The title should include, where possible, information on the participants, condition being evaluated, and intervention s studied. Limit: characters. Limit: 14 characters. This includes any unique clinical study identifiers assigned by other publicly available clinical trial registries.
If the clinical study is funded in whole or in part by a U. Federal Government agency, the complete grant or contract number must be submitted as a Secondary ID. If there is a Secondary ID, then the following information must be provided:. Study Identification. Select one. Other components of the full award number type code, support year, and suffix are optional. 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.
Other Identifier: Also required to enter a brief description of the identifier for example, the name of organization that issued the identifier. Study Status.
If at least one facility in a multi-site clinical study has an Individual Site Status of "Recruiting," then the Overall Recruitment Status for the study must be "Recruiting. Definition: A brief explanation of the reason s why such clinical study was stopped for a clinical study that is "Suspended," "Terminated," or "Withdrawn" prior to its planned completion as anticipated by the protocol.
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 Affiliation : Primary organizational affiliation of the individual; Limit: characters. Studies a U. Yes: At least one studied FDA-regulated device product has not been previously approved or cleared by FDA No: All studied FDA-regulated device products have been previously approved or cleared by FDA Note : Full posting of registration information will be delayed if "Yes" is selected and the study is an applicable clinical trial that is required to be registered under 42 CFR FDA Approval or Clearance data element.
Post Prior to U. FDA Approval or Clearance Definition: Authorize NIH to post publicly clinical trial registration information for a clinical study of a device product that has not been previously approved or cleared that would otherwise be subject to delayed posting.
FDA-regulated device product is a pediatric postmarket surveillance of a device product ordered under section of the Federal Food, Drug, and Cosmetic Act. Will not be made public - for administrative purposes only. Expanded Access for investigational drug products including biological products includes all expanded access types under section of the Federal Food, Drug, and Cosmetic Act: 1 for individual participants, including emergency use; 2 for intermediate-size participant populations; and 3 under a treatment IND or treatment protocol.
If there is no existing expanded access record, the responsible party who is both the manufacturer of the investigational drug product including a biological product and the sponsor of the ACT is required to create an expanded access record. Product Manufactured in and Exported from the U. Required if U.
A study may be submitted for registration prior to approval by the review board so long as the study is not yet recruiting participants. Request not yet submitted: Review board approval is required but has not yet been requested Submitted, pending: Review board approval has been requested but not yet granted Submitted, approved: Review board approval has been requested and obtained Exempt: An exemption in accord with applicable law and regulation has been granted Submitted, denied: Review board has denied the approval request Submission not required: Review board approval is not required because the study is not subject to laws, regulations, or applicable institutional policies requiring human subjects review If the study is not an applicable clinical trial that is required to be registered under 42 CFR Part 11, is not funded in whole or in part by the U.
May be omitted if status is anything other than approved. Address : Mailing address for the board, including street address, city, State or province, postal code, and country. Data Monitoring Committee Definition: Indicate whether a data monitoring committee has been appointed for this study. The data monitoring committee board is a group of independent scientists who are appointed to monitor the safety and scientific integrity of a human research intervention, and to make recommendations to the sponsor regarding the stopping of the trial for efficacy, for harms or for futility.
The composition of the committee is dependent upon the scientific skills and knowledge required for monitoring the particular study. Food and Drug Administration regulation under section of the Public Health Service Act or any of the following sections of the Federal Food, Drug, and Cosmetic Act: , k , , m , and Section Clinical Trial Definition: If this study includes an FDA regulated intervention, indicate whether this is an applicable clinical trial as defined in U.
Study Description. Detailed Description Definition: Extended description of the protocol, including more technical information as compared to the Brief Summary , if desired. Do not include the entire protocol; do not duplicate information recorded in other data elements, such as Eligibility Criteria or outcome measures. Limit: 32, characters. For Patient Registries: Also describe the applicable registry procedures and other quality factors for example, third party certification, on-site audit.
In particular, summarize any procedures implemented as part of the patient registry, including, but not limited to the following: Quality assurance plan that addresses data validation and registry procedures, including any plans for site monitoring and auditing. Data checks to compare data entered into the registry against predefined rules for range or consistency with other data fields in the registry.
Source data verification to assess the accuracy, completeness, or representativeness of registry data by comparing the data to external data sources for example, medical records, paper or electronic case report forms, or interactive voice response systems.
Data dictionary that contains detailed descriptions of each variable used by the registry, including the source of the variable, coding information if used for example, World Health Organization Drug Dictionary, MedDRA , and normal ranges if relevant. Standard Operating Procedures to address registry operations and analysis activities, such as patient recruitment, data collection, data management, data analysis, reporting for adverse events, and change management.
Sample size assessment to specify the number of participants or participant years necessary to demonstrate an effect. Plan for missing data to address situations where variables are reported as missing, unavailable, non-reported, uninterpretable, or considered missing because of data inconsistency or out-of-range results.
Statistical analysis plan describing the analytical principles and statistical techniques to be employed in order to address the primary and secondary objectives, as specified in the study protocol or plan. Conditions and Keywords. Keywords Definition: Words or phrases that best describe the protocol. Keywords help users find studies in the database. Be as specific and precise as possible. Avoid acronyms and abbreviations. Study Design. Treatment: One or more interventions are being evaluated for treating a disease, syndrome, or condition.
Prevention: One or more interventions are being assessed for preventing the development of a specific disease or health condition. Diagnostic: One or more interventions are being evaluated for identifying a disease or health condition.
Supportive Care: One or more interventions are evaluated for maximizing comfort, minimizing side effects, or mitigating against a decline in the participant's health or function. Screening: One or more interventions are assessed or examined for identifying a condition, or risk factors for a condition, in people who are not yet known to have the condition or risk factor.
Health Services Research: One or more interventions for evaluating the delivery, processes, management, organization, or financing of healthcare.
Basic Science: One or more interventions for examining the basic mechanism of action for example, physiology or biomechanics of an intervention. Device Feasibility: An intervention of a device product is being evaluated in a small clinical trial generally fewer than 10 participants to determine the feasibility of the product; or a clinical trial to test a prototype device for feasibility and not health outcomes.
Such studies are conducted to confirm the design and operating specifications of a device before beginning a full clinical trial. Other: None of the other options applies. Select only one. Early Phase 1 Formerly listed as "Phase 0" : Exploratory trials, involving very limited human exposure, with no therapeutic or diagnostic intent e. Phase 2: Includes controlled clinical studies conducted to evaluate the effectiveness of the drug for a particular indication or indications in participants with the disease or condition under study and to determine the common short-term side effects and risks.
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