The Role of IND-enabling Studies

IND-enabling studies are crucial for advancing a New Chemical Entity (NCE) from preclinical evaluation to its first-in-human clinical trial. These studies comprehensively assess the NCE’s safety, pharmacology, and manufacturing processes, leading to the submission of an Investigational New Drug (IND) application to regulatory agencies like the FDA.

Successful planning and execution of IND-enabling studies are vital for navigating the drug development process efficiently and increasing the chances of IND approval. This involves assembling a multidisciplinary team and collaborating with an experienced Clinical Research Organization (CRO) to optimize the process.

In this blog post, we explore the intricacies of IND-enabling studies, their strategic importance in planning Phase 1 clinical trials, the roles of pharmacological and toxicological studies, considerations in chemistry, manufacture, and control (CMC) studies, regulatory aspects, and the benefits of partnering with an experienced CRO like BioPharma Services. 

Before a New Chemical Entity (NCE) can be tested in clinical trials, its safety profile and pharmacological properties should be elucidated in preclinical studies. In the United States, this process of in vitro and in vivo preclinical characterization of an NCE is concluded with the filing of an Investigational New Drug (IND) application with the United States Food and Drug Administration (US FDA). An IND application contains pharmacological, toxicological, and manufacturing data on the NCE as well as a protocol for the planned clinical trial and investigator information. Once an IND application is approved by the FDA, a first-in-human clinical trial can be initiated.

Comprehensively planning preclinical studies that will be included in an IND application (IND-enabling studies) can help study sponsors avoid delays in the drug development process and increase the likelihood of approval of their IND application. However, the successful design and execution of IND-enabling studies requires the efforts of a multidisciplinary team with know-how in the fields of pharmacology, pharmacokinetics, toxicology, chemistry, regulatory science, and biostatistics. Working with an experienced clinical research organization (CRO) with expertise in all these areas can aid study sponsors to streamline and optimize their IND-enabling studies.

Using IND-enabling Studies to Strategically Plan for Phase 1 Clinical Trials

IND-enabling studies should be clinically relevant and should collect preclinical data aligned with the rationale of future clinical trials. Therefore, it is important to clearly define the therapeutic target of an NCE early on in IND-enabling studies. In addition, the intended disease indications in planned future clinical trials should be kept in mind when selecting the most appropriate preclinical models to acquire data for an IND application.

Overall, IND-enabling studies help determine whether an NCE is reasonably safe for the initiation of a first-in-human clinical trial and whether its pharmacological activity supports further development. By the time an IND-enabling study is completed, study sponsors should demonstrate that the use of an NCE in a first-in-human clinical trial will not expose study volunteers to an unreasonable risk.