Yes, your assumption that an investigator initiated trial can be used for proof of concept is correct. This is often done for a marketed product when the commercial or medical affairs side of the company is exploring a new indication and doesn't want to involve an operations organization.
Keep in mind that anyone doing a study with an investigational agent anywhere in the world will need to obtain regulatory authority and ethics approvals. Such an investigator initiated trial is possible if the investigator submits an IND/CTA and IRB/EC applications in his/her own name and assumes legal responsibility for conduct of the study regardless of the source of funding for the trial. The investigator is responsible for conduct of the study under GCP, which any regulatory authority or ethics committee would require. There is no requirement that the company providing the investigational agent discuss the study with the regulatory agency. The company can design the trial, write the protocol, or provide whatever support is needed, including data collection and monitoring. All of the company's involvement should be detailed in a contract with the investigator as the basis for supplying the investigational agent. See the below definition of a Sponsor - the only requirement for being a Sponsor is taking responsibility and initiating a clinical trial.
Quoting 21 CFR 312.3 Definitions and interpretations:
Sponsor means a person who takes responsibility for and initiates a clinical investigation. The sponsor may be an individual or pharmaceutical company, governmental agency, academic institution, private organization, or other organization. The sponsor does not actually conduct the investigation unless the sponsor is a sponsor-investigator. A person other than an individual that uses one or more of its own employees to conduct an investigation that it has initiated is a sponsor, not a sponsor-investigator, and the employees are investigators.
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Glen Park PharmD
Vice President, Regulatory Affairs and Quality Assurance
Scynexis, Inc.
New York NY
United States
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Original Message:
Sent: 27-May-2021 18:55
From: Anonymous Member
Subject: Investigator Initiated Trials
This message was posted by a user wishing to remain anonymous
I know a company sponsor can use an IIT -- independent investigator initiated trial for an investigational agent as the proof of concept for the basis of a phase 3 trial design, correct? Drug is being supplied to the investigator. Does the company sponsor need to discuss this up front with the Agency if planning for use in designing a registration trial? Can a company assist in the protocol review or medical writing support? What expectation to the data access after completion is there and in what form should the sponsor recommend to the investigator with assurance the study was conducted under GCP.
Meaning if this is independent and the investigator is inclined to do it but seeks a bit of advice from the co, how does this work?
Thanks for any input from experience.