TABLE OF CONTENTS

‍

Introduction

     General

     Investigator Initiated Trials

     The Sponsor-Investigator

Key Elements of an IIT

     Application/Study Design

     Research Protocol

     Study Management

     Publication

Reference materials

Introduction

General

Ethical collaboration between practitioners and industry is essential to advances in medicine.  However, laws and institutional policies sharply circumscribe the ways in which these two groups can interact.

Real-world studies represent an important medium through which such interaction can properly and effectively occur.  As is the case with traditional RCT’s, industry can support investigators who are conducting independent and properly designed studies through study fees, honoraria and product discounts.

RegenMed provides the clinical grade yet cost-effective tools and processes intermediating the funding and execution of real-world studies.

Investigator Initiated Trials

An Investigator Initiated Trial (“IIT”) is a common example of real-world study ethically accommodating the objectives of the independent provider, as well as a third party with related objectives which can provide financial support.

An IIT is a study where the study investigator is also the study sponsor. In other words, the Investigator is not only responsible for execution of the study, but also for its design and oversight.  In an IIT, the investigator is therefore called the Sponsor-Investigator.

Many medical product manufacturers, foundations, governmental and non-governmental organizations have programs to help fund IIT’s.  

The Sponsor-Investigator

The Sponsor-Investigator may be an individual clinician, small clinic, medical society, or a hospital department.

Although each category differs markedly in its capacity to design and manage an ITT, it is unlikely any will be able properly to do so without third-party support. (The only exception is a large academic/medical center, which has in place a Clinical Trial Unit or similar infrastructure.)

Although real-world studies can be executed efficiently even by individual clinicians, the expectations of IIT funding sources usually demand a more robust study design and management infrastructure.  Typical elements are described in the following section.

Key Elements of an IIT

An IIT usually involves the following important elements:

Application/Study Design

An IIT Sponsor-Investigator will apply to a suitable funding source.  This process typically begins with a “concept note” which, if approved, will be fleshed out into a more thorough study design.  Many IIT funding sources articulate their IIT criteria and evaluation processes on their websites. The concept note of course should be drafted in accordance with these guidelines.

It is important to distinguish between two types of “audiences”: clinical/ scientific and business.  Usually, both groups will be involved in evaluating an IIT application.  Sometimes, however, the evaluation may be made solely by the clinical/scientific group.  This factor obviously dictates the emphasis of certain elements of the application.

Important components of an IIT application include:

• Study Rationale/Unmet Need. Why might the results of the study make a difference? For which patient population? Study rationale criteria will often include relevance to the funder’s products or therapeutic area of interest.
• Purpose of the Study. Examples include (i) test the safety/efficacy of two or more products; (ii) test a product in different population subsets; (iii) test a non-approved use for the product; (iv) combine the product with other technologies to improve its safety and/or efficacy; (v) develop a new product.
• Type of study. (Prospective randomized double blind controlled, in vitro, animal, observational, surveys, clinical, pragmatic, “n of 1”, other.)
• Summary of relevant prior literature.
• Identity and credentials of team members. (Principal and sub-investigators. Researchers. Laboratories.   Statisticians. Medical writers. Support staff.)
• Experience of Team Members, especially with similar studies.
• Ability of Team Members to adhere to Good Clinical Practice.  
• Research Protocol. (See below.)
• Approach to assessing clinical and statistical significance.
• Software and other technical support.
• Legal/Regulatory Compliance. (Including IRB/Medical Ethics Committee processes.)
• Endpoints
• Data ownership and rights.
• Budget and Financial Resources.

Research Protocol

These are the parameters pursuant to which the study will be carried out.  Key components include:

• Population size.
• Inclusionary and exclusionary criteria.
• Blinding or other approaches to remove bias.
• Specific comparators. (Dosages, products, patient cohorts, etc.)
• Outcomes measures, and timing of measurement reports.
• Type(s) of statistical analysis, including methods for determining statistical significance as well as clinical significance.)
• Reports (type, illustrative, frequency).
• Adverse Events Reporting.
• Planned regulatory submissions, and coordination with regulators.

Study Management

Important items here include:

• Legal/Regulatory (Consent forms, data privacy, allocation of liability, data ownership and rights, anti-corruption, anti-bribery and anti-kickback laws.)
• Institutional Review Board/Medical Ethics Committee identification and processes.
• Quality control and assurance. (More complex in context of multi-center studies.)
• Allocation of financial/legal liabilities. (An IIT funding source will not accept legal liability arising from the study.)
• (E.g., Part 11 compliance if in the U.S., database management, source data review/verification, HIPAA/GDPR compliance, electronic Case Report Forms.)

Publication

This element includes medical writing, diagrams and tables, citations, journal selection.  Some funding sources may look for publication in peer-reviewed print journals; others may be satisfied with publication through on-line journals or presentations of abstracts at conferences

Reference materials

F.D.A.

Important F.D.A. links relating to ITT’s include:

Good Clinical Practice.

Good Laboratory Practice.

IIT IND’s.

ICH

The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH)

World Health Organization

WHO Consent Form Templates

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