What Are Phase 3 Clinical Trials? The Pivotal Studies That Support FDA Approval

Phase 3 clinical trials are often considered the most important stage of clinical development. These are much larger scale trials compared to previous phases. The data and results obtained in Phase 3 clinical trials are what regulatory agencies use when deciding if a treatment should be approved for public use.

What Is a Phase 3 Clinical Trial?

Phase 3 trials are designed for further confirmation of the safety and efficacy of the investigational product (IP). A risk-benefit profile for the IP is also developed during Phase 3 trials. This have builds on previous studies by demonstrating the treatment performs consistently in larg populations. 

Phase 3 clinical trials are often called “pivotal trials” as they directly support any regulatory submissions. The data from this phase is used in the application submitted to FDA for approval. This data can be part of the:

• New Drug Applications (NDA) 

• Biologics License Applications (BLA) 

• Certain medical device submissions 

Successful Phase 3 results can ultimately lead to market approval.

Typical Characteristics of Phase 3 Studies

Most Phase 3 trials are larger in scope to previously trials and can have between 300-1000 or more participants. With such large participant numbers these can be done across dozes of research sites in multiple geographic regions. These trials are also longer with extended follow-up periods, some lasting for several years. 

Phase 3 trial also broaden the eligibility criteria which allows for inclusion of a more diverse patient population. This includes expanded age groups and comorbidities. The goal in this phase is to understand how the treatment performs in a broader range of patients who better resemble the populations seen in everyday clinical practice.

Continued Use of Randomization, Blinding, and Long-Term Safety Monitoring

Phase 3 clinical trials typically remain randomized and doble-blinded. The study designs are controlled and researchers may compare how the IP’s efficacy compared to a placebo. They would also compare the IP to the standard thereby and compare different types of treatment regimes. The goals is to generate the strongest possible evidence of how the IP works in a broad population. 

As a larger number of participants are included, researchers are able it identify additional adverse effects that are less commo and not identified in previous phases. With larger populations, researchers are better able to identify rare side effects, long-term risks, and any population specific concerns. This information is all crucial for regulatory review. 

Open-Label Extension Studies

A unique aspect of many Phase 3 trials is the inclusion of an open-label extension periods. At the end of the blinded portion of the study, individuals that participated in trial may be offered continued access to the IP. 

The open-label period help researchers to continue evaluations the long-term safety of the IP. This also provides additional insights such a the durability of teh treatment and patient adherence. It also provides them the opportunity to evaluate quality-of-life comes that may result from the IP. 

Why Phase 3 Studies Matter to Clinical Sites

Phase 3 trials are often attractive to providers because they provide opportunitties for larger enrollment across a broader patient population. This can increase clinical research opportunities and provide the opportunity for a substantial clinical experince. Part of this can include bringing new innovative therapies for patients.

For established research sites, Phase 3 studies frequently become a core part of their portfolio. Though they can be difficult as they also require additional expertise and personnel. In phase 3 trials Sites must be aware of an manage operational complexity that includes:

• Regulatory documentation 

• Recruitment campaigns 

• Monitoring visits 

• Data entry requirements 

• Protocol compliance 

• Audit readiness 

Strong operational systems become increasingly important and out team at TriStar Clinical Research Consulting, LLC helps provide sustainable clinical trial research programs through:

• Study startup support 

• Regulatory management 

• SOP development 

• Staff training 

• Recruitment assistance 

• Sponsor and CRO communication 

Whether you need full-service support or specific operational assistance, we help reduce barriers to participation.