What Is an Investigational New Drug (IND)?

An Investigational New Drug (IND) is a drug developed by a pharmaceutical or biotech company or other organization that is ready for clinical trials on humans. When a drug reaches this point, the pharma company submits an application to get the consent of the Food and Drug Administration (FDA) to begin these trials. This step is important as approval for the application allows a sponsor to ship the drug across the country to begin testing.

Key Takeaways

  • An Investigational New Drug (IND) is a drug developed by a sponsor that is ready for clinical trials on humans.
  • The IND application is submitted by the company or research group responsible for developing the drug to the FDA.
  • The FDA reviews IND applications and decides whether they are safe for companies to progress to the clinical trial stage.

Understanding an Investigational New Drug (IND)

When a company develops a new drug, it must get approval from the FDA before it can sell it to the general public to ensure its safety. The company must go through a series of steps and applications before it can get to this point. It's up to the company, which is also called the drug sponsor, to run the required tests, gather data, and make sure patients aren't exposed to unnecessary risks when they take the drug. The FDA reviews the results after each phase and makes a determination of whether the drug is safe for the public.

One of the application steps is the Investigational New Drug (IND) stage. An IND is not an application for marketing approval. Instead, it is the way a sponsor gets an exemption to the federal law that prohibits an unapproved drug from being transported across state borders from the FDA. This exemption is a requirement since, in most cases, the sponsor must ship an investigational drug to investigators in other states. To obtain the exemption, the sponsor must submit sufficient data through the IND, documenting the safety of the drug for use in human testing.

The Investigational New Drug (IND) Application

The primary requirements for the IND application are as follows:

  • Animal Pharmacology and Toxicology Studies: Companies need to collect enough data from preclinical studies to establish whether the drug is reasonably safe for initial tests in humans, as well as any previous experience involving human use of the drug, such as any usage in foreign markets. This step involves testing on animals to determine the drug's safety and efficacy.
  • Manufacturer Information: Information needs to be included about the manufacturer to ensure that the company can manufacture sufficient batches of the drug and has the proper controls in place to do so safely.
  • Clinical Protocols and Investigator Information: Detailed protocols are needed to determine if the initial trials will expose human subjects to needless risks. This also includes the qualifications of the clinical investigators who will oversee the administration of the compound.

An IND is submitted after the sponsor determines that the proposed drug is reasonably safe for initial use in humans and that it shows sufficient promise as a treatment to justify commercial development.

The FDA reviews the IND application and decides whether it is safe for the company to progress to the next stage. This is the clinical trial stage; the point at which the drug is tested on humans. The sponsor has to wait for 30 calendar days after submitting the IND before commencing any clinical trials.

It can cost hundreds of millions of dollars—and many years—to undertake clinical trials to bring a new drug to market. The IND application signifies that the sponsor is willing to make this huge investment. As such, investor reaction to an IND application, which is merely the first step in a long and arduous process for drug approval, is typically neutral.

Types of Investigational New Drugs (INDs)

Investigational New Drugs (INDs) fall into two categories: commercial and research. The big difference between these two categories is who does the application filing. As the name suggests, the commercial IND category is sought by a company that wants to test a drug in order to bring it to market. Any company can apply for this IND, whether it's a large pharma or biotech company, as well as a nonprofit organization (NPO), such as a cancer research group. The application process for a commercial IND can be fairly lengthy and complicated. That's because data is often collected at multiple locations and involves many investigators.

The research or non-commercial IND is the step investigators require to run tests on an existing drug. Researchers require approval when they want to test approved drugs that are already on the market. Testing may include new dosages or new applications for these drugs. The majority of INDs are filed for non-commercial research and are of three main types: Investigator IND, Emergency Use IND, and Treatment IND. The application process is generally simpler than that of a commercial IND because testing is normally done by a smaller group of people and in one location.