Investigator-Initiated Clinical Studies (IICS) Toolbox

The definition of the research question is key to research design. All research must have a primary question, clearly stated in advance, and founded on a systematic review of what is already known. Researchers who plan studies without reviewing what has been done, risk performing research for which the answer is already known or exposing participants to ineffective or an inferior treatment.

The ICH GCP E6 (R3) (International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use- Good Clinical Practice) guideline defines the protocol as “A document that describes the objective(s), design, methodology, statistical considerations and organisation of a trial. The protocol usually also gives the background and rationale for the trial, but these could be provided in other protocol-referenced documents”

The ICH for Good Clinical Practice guidelines E6 (R3) and the Clinical Trials Regulation (536/2014), define a sponsor as “an individual, company, institution or organisation which takes responsibility for the initiation, for the management and for setting up the financing of the clinical trial”

Industry-initiated clinical trials are financially supported by the industry. The principal investigator (PI) salary and the costs associated with running the trial are all covered by the pharmaceutical company that conceived the clinical trial. In investigator-initiated trials (IIT), however, usually is the PI who applies for funding through research programs and government grants to fund their conceived research project.

Risk assessment is a systematic process for identifying and evaluating events that could affect the achievement of clinical study´s objectives related to quality, safety, timelines and budget, positively or negatively.  

The purpose of a Project Management Plan (PMP) in a clinical trial is to define the scope, outline responsibilities and describe key steps of the clinical trial process.

DMP is a written document that describes the plans for collection and management of data throughout the lifecycle of a clinical trial. The DMP describes which clinical data will be acquired and how it will be handled, stored, checked for consistency and plausibility, and made available for the final analysis and further research after the end of the project.

Trial management is the process of ensuring that a trial is run effectively and within budget and timelines.

Prior to initiating a clinical trial, researchers must obtain approval from National Competent Authorities (NCA) and ethics committees.

The sponsor should implement a system to manage quality throughout all stages of the trial process, in particularly on trial activities essential to ensuring human subject protection and the reliability of trial results.  

The sponsor is responsible for the ongoing safety evaluation of the Investigational Medicinal Product(s) used in a Clinical Trial

A process that begins with conception and design of the clinical trial, continues through data capture and analysis to publication, data archiving and data sharing with the broader scientific community. The Data Management Plan (DMP) describes the procedures for data collection and management  throughout the lifecycle of a clinical trial. 

An investigational product (IP), as defined by the ICH is a pharmaceutical form of an active ingredient or placebo being tested or used as a reference in a clinical trial, including a product with a marketing authorization when used or assembled (formulated or packaged) in a way different from the approved form, or when used for an unapproved indication, or when used to gain further information about an approved use.

The analysis of samples collected from subjects participating in clinical trials forms a key part of the clinical trials process. Sample analysis or evaluation provides important data on a range of endpoints which is used, for example, to assess the pharmacokinetic profile of investigational medicinal products and to monitor their safety and efficacy.

The SAP is intended to be a comprehensive document that contains a detailed and technical description of the principal features of the  statistical analysis outlined in the protocol including detailed procedures for executing the statistical analysis of the primary and secondary endpoints and other data.

A Clinical Study Report (CSR) is a is a key document that describes the methodology and results of a clinical trial in drug development.

The documents which individually and collectively permit evaluation of the conduct of a clinical trial and the quality of the data produced are defined as essential documents according to the ICH Good Clinical Practice.

After each clinical trial finishes, the trial sponsor will compile a detailed clinical study report (CSR), which follows a format laid down by the regulatory authorities. Access to the complete CSR is usually limited to the sponsor and the regulatory authorities that are assessing the marketing authorisation application.