Investigator-initiated studies: Challenges and solutions

Area of Science:

• Clinical Research
• Pharmacovigilance
• Health Services Research

Background:

• Investigator-initiated studies (IISs) are vital for real-world drug effectiveness and safety data.
• These studies address clinical questions beyond initial drug development phases (Phases I-III).
• IISs are managed by researchers, not pharmaceutical companies.

Purpose of the Study:

• To review the literature on IISs.
• To share personal experiences conducting IISs over two decades.
• To describe challenges and propose solutions for IISs.

Main Methods:

• Literature review of investigator-initiated studies.
• Personal narrative of conducting multiple IISs.
• Analysis of challenges and potential solutions in IISs.

Main Results:

• IISs provide valuable real-world data on drug efficacy and safety.
• Common challenges include financial constraints, regulatory hurdles, and data management.
• Despite difficulties, IISs are rewarding and can impact healthcare policy.

Conclusions:

• Investigator-initiated studies are essential for expanding drug knowledge beyond clinical trials.
• Addressing challenges in funding, regulation, and expertise is key to successful IISs.
• IISs have the potential to significantly contribute to medical evidence and policy changes.