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First-In-Human (FIH) studies represent a pivotal milestone in the development of new drugs and medical treatments. These initial trials, conducted after extensive preclinical testing, involve administering a new treatment to humans for the first time. The primary goal of FIH studies is to evaluate the safety profile, pharmacokinetics, and pharmacodynamics of a new intervention in a small group of participants. This phase is crucial for determining the treatment’s feasibility for further development and larger clinical trials.

What is the Importance of FIH Studies in pharmaceutical development?

FIH studies are foundational in the realm of medical advancements, bridging the gap between theoretical research and practical, life-saving treatments. They mark the transition from laboratory research to actual patient care, serving several critical purposes:

Safety Evaluation: The foremost objective of FIH studies is to assess the safety and tolerability of a new treatment in humans, which cannot be fully predicted by preclinical models

Dosage Determination: These studies help in identifying the appropriate dosage range and delivery method for the new treatment, ensuring maximum efficacy with minimal side effects.

Early Efficacy Signals: While primarily focused on safety, FIH studies also provide early insights into a treatment's effectiveness, guiding future research directions.

These initial trials are vital for ensuring that only safe and potentially effective treatments proceed to more extensive testing, safeguarding patient welfare and resource allocation.

How do FIH studies impact Medical Research and Development?

FIH studies serve several key roles in the broader landscape of medical research and development:

Ethical and Regulatory Compliance: They ensure that new treatments are introduced to humans ethically and in compliance with regulatory standards, based on robust preclinical evidence.

Scientific Advancement: By validating hypotheses from preclinical research in a human setting, FIH studies contribute to the scientific understanding of diseases and treatments.

Innovation Acceleration: Successful FIH trials can accelerate the development of innovative treatments by providing the necessary data to proceed with confidence in further clinical phases.

These roles underscore the critical position of FIH studies at the intersection of science, ethics, and regulatory compliance, driving forward the development of new medical interventions.

Guidelines surrounding FIH studies

Regulatory services agencies have developed and implement guidelines to provide sponsors with guidance on FIH studies. For example, the European Medicines Agency’s (EMAs) guideline on “Strategies to identify and mitigate risks for first-in-human and early clinical trials with investigational medicinal products” provides relevant stakeholders with assistance in transitioning from non-clinical to early clinical development. It also helps in identifying factors that can affect the risk for new IMPs, or investigational medicinal products. The guideline highlights key considerations related to quality, non-clinical, and clinical testing as well as study designs & trial conduct.

On the other hand, an example of a guidance implemented by the United States Food and Drugs Administration (US FDA) is the “Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics”. This document specifically covers key aspects that trial sponsors should consider when it comes to the design and conduct of FIH which are intended to expedite clinical development of drugs used for cancer treatment, diagnosis, prevention, and cure by way of multiple expansion cohort trial designs.


First-in-human (FIH) trials are an important aspect in pharmaceutical product development as they assess the safety, dosage, and efficacy in human subjects. These trials are critical for bridging the gap between pre-clinical research and wider clinical studies to ensure that new therapy options are evaluated in a robust and rigorous manner before they reach a larger & wider patient population.

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