New Drug Approval
What must be accomplished to qualify a drug candidate for clinical studies?
FDA’s Center for Drug Evaluation and Research (CDER) has developed a small business-oriented website (link provided below) that describes in more lay terms the U.S. regulatory requirements for development and approval of pharmaceutical products. This site has definitions and interactive charts that provide basic information for small businesses and others who are unfamiliar with the new drug development and approval process.
Most visitors to this site will likely be in the earlier stages of drug development, possibly involved in pre-clinical (animal) testing and interested in the regulatory requirements governing the transition to clinical (human) testing.
FDA's role begins when...
“When the drug’s sponsor or development partner (having screened the candidate for pharmacological activity and acute toxicity in animals) wants to test it in humans, the drug molecule changes in legal status. Under the Federal Food, Drug, and Cosmetic Act, the candidate molecule then becomes a new drug subject to specific requirements of the regulatory system.”
One of the most basic laws affecting drug development involves the restrictions associated with transport of an unapproved drug candidate across state lines, regardless of your intention.
Investigational New Drug (IND) Exemption
Because a sponsor will want the flexibility to ship an investigational drug to clinical investigators in any state, one must seek an exemption from the legal ban on such transport. The IND is the means through which the sponsor technically obtains this exemption from the FDA. Of course, in so doing, the sponsor provides a great deal of information about the drug candidate, along with the rationale for testing it in a specific patient population.
In addition to the standard commercial IND, there are research IND categories. An example of the latter is an Investigator IND, which can be submitted by a physician independent of any company, and under whose immediate direction the investigational drug will be administered or dispensed.
Regardless of which type IND is submitted, the application must contain adequate information in each of the following categories to allow expert reviewers to answer important questions:
- Animal Pharmacology and Toxicology Studies
Is the product reasonably safe for initial testing in humans? Has there been any previous experience with the drug in humans (e.g., foreign use)?
- Drug Manufacturing Information
Does information pertaining to the composition, manufacturing, stability, and controls used in producing the drug substance ensure that the company can adequately supply consistent batches of the drug?
- Clinical Protocols and Investigator Information
Will protocols for proposed clinical studies expose subjects to unnecessary risks? Are the clinical investigators and study administrators adequately experienced to conduct the study under IND regulations?
Once the IND is submitted, the sponsor must wait 30 calendar days before initiating any clinical trials.
For more information on the IND process and for a detailed overview of the entire drug development and New Drug Approval (NDA) review process, visit this FDA link for small business drug developers.