医師主導治験（IIT：Investigator Initiated Trial）：製薬会社による活用

Abstract

Establish and perfect internal controls to capitalize on IIT opportunities

Investigator-initiated clinical trials (IITs) provide companies with the clinical data to further scientific understanding of their drugs. IIT findings help to expand on- and off-label usage, reach new patient populations, generate data for publication and solidify relationships with key opinion leaders. Once considered an afterthought, IITs are now often regarded as critical pieces of product development strategy.

Reflecting this different mindset, many companies have established centralized IIT management groups and processes. While such efforts mark a giant step forward, all organizations still routinely confront challenges that range from slowdowns in the IIT-approval process to issues with trial oversight.

Investigator-Initiated Trials addresses such obstacles and provides solutions by outlining steps to refine IIT management efforts. Use this report to accomplish the following goals:

• - Develop strategic thinking to align IITs with corporate strategy
• - Compare timelines and best practices for IIT proposal evaluation and approval
• - Improve ongoing IIT oversight
• - Discover IIT management trends
• - Benchmark structures and investments
• - Examine profiles of 16 companies' IIT efforts

Table of Contents

Charts and Graphics

Chapter 1: Strategic Role of IITs

• Self-Assessment of IIT Performance
• Share of Company-Sponsored Versus Investigator-Initiated Trials
• Departments Involved in Use of IIT Study Findings
• Average Share of Approved IITs that Generate Medical Publications
• Share of Approved IITs that Generate Medical Publications (by Company)
• Average Usefulness of IITs in Accomplishing Key Objectives
• Average Share of IITs Conducted per Development Phase
• Share of IITs Conducted per Development Phase (by Company)
• Self-Identified Improvement Areas for IIT Management Groups

Chapter 2: Overview of the IIT Selection Process

• IIT Proposal Evaluation Process Diagram
• IIT Approval Pipeline: Share of Proposals that are Evaluated, Approved and Published
• Number of Proposals that are Received, Evaluated and Approved (by Company)
• IIT Proposal Receipt Process Diagram
• Share of Companies with Established Process for Collecting and Accepting IIT Proposals
• Share of Companies with an Established Process for Tracking IIT Proposals
• Percentage of Received Proposals that Reach Final Approval
• IIT Approval Timeline Breakdowns
• IIT Proposal Screening Process
• Time Spent from Proposal Receipt to Decision-Making Body
• Departments that Sit on the Formal IIT Proposal Committee
• Time Spent from Decision-Making Body to Final Approval
• Meeting Frequency Versus Average Approval Time
• Frequency of Meetings to Review IIT Proposals
• Challenges in the IIT Evaluation Process
• Time Spent from Final Approval to Notifying Investigator
• Share of Companies with an Established Process for Managing IITs
• IIT Management Process Diagram
• Contract Negotiations & Ongoing Management
• Most Problematic Aspects of the IIT Management Process

Chapter 3: Building an IIT Infrastructure

• Companies with Dedicated IIT Groups
• Breakdown of Companies without Dedicated IIT Groups
• FTEs per Dedicated IIT Management Function: Company-Wide Groups
• FTEs per Dedicated IIT Management Function: Therapeutic-Area Focused Groups
• Companies with a Formal Committee to Receive, Review and Approve IIT Proposals
• Departments that Sit on the Formal IIT Proposal Committee
• Number of Departments Sitting on the Formal Review Committee
• Level of Executive that Leads IIT Initiatives
• Single Executive Versus Committee Decision for Final Approval of IIT Proposal
• Departments Overseeing IIT Outcomes
• Number of Departments Overseeing IIT Outcomes
• Number of Departments Involved in Determining Use of IIT Study Findings
• Departments Involved in Evaluating Use of Trial Findings
• Self-Identified Improvement Areas for IIT Management Groups
• Percentage of Companies with a Dedicated IIT Budget
• Departments that Provide Funding for IIT Initiatives
• Percentage of Budget Allocated to Salaries and Overhead Expenses
• IIT Groups' Dedicated Budgets: Company-Wide
• IIT Groups' Dedicated Budgets: Therapeutic-Area Focused
• Budget Per IIT Proposal Approved
• Funds Spent on IITs by Brands with Less than $1b in Sales
• Funds Spent on IITs by Brands with More than $1b in Sales
• Forms of Support Companies Provide for IIT Investigators

Chapter 4: IIT Program Profiles

This chapter contains profiles of 16 companies' IIT efforts. The findings are presented in a series of charts and graphs for each company. Each profile follows the same structure:

• Figure 1: Management Infrastructure
  • Dedicated IIT management: budget and staffing for the company' s IIT group, if such a group exists
  • Proposal review committee: review committee membership, leadership and meeting frequency
• Figure 2: IIT Volume
  • IIT proposals: Number of proposals received, evaluated and approved
  • IITs by development stage: IIT breakdown by clinical development
• Figure 3: Productivity and oversight
  • Proposal process and timelines: average duration of IIT evaluation stages
  • Oversight and data management: groups involved in overseeing IITs and determining uses for completed studies.
• Figure 4: Impact and improvement
  • Value to objectives: impact of IITs on different company goals
  • Key improvement areas: improvement opportunities for proposal evaluation and ongoing study management