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Learning Objectives

After completing this chapter, the reader will be able to

  • List the major legislative acts that led to the current system of drug evaluation, approval, and regulation used in the United States (U.S.).

  • List the steps in the drug approval process.

  • List the components of an investigational new drug application (IND).

  • Recognize the difference between a commercial IND, a treatment IND, an emergency use IND, and an individual investigator IND.

  • Define orphan drug status and list the advantages of classifying a drug as an orphan drug.

  • List all of the requirements (as specified by the Office of Human Research Protections [OHRP]) for an institutional review board (IRB).

  • Prepare appropriate reviews of protocols for use by the IRB or other review committees when they evaluate new protocols.

  • Describe the type of support that is necessary for clinical research, including (but not limited to):

    1. Ordering drug supplies for ongoing clinical trials

    2. Maintaining drug accountability records as required by the Food and Drug Administration (FDA)

    3. Preparing drug and protocol data sheets for use by health care personnel in the hospital

    4. Preparing pharmacy budgets for sponsored clinical research

    5. Aiding investigators in designing and conducting clinical trials in their institution

    6. Assisting investigators in initiating and conducting clinical trials (including emergency use INDs)

Key Concepts

  1. The Food and Drug Administration (FDA) is the federal agency that decides which drugs, biologics, and medical devices are safe and effective and, therefore, can be marketed in the United States.

  2. In addition to review by the FDA, research protocols are also reviewed for ethical appropriateness by IRBs.

  3. The drug approval process in the United States is standardized by FDA review. It consists of preclinical testing and Phase I through IV of clinical testing.

  4. The investigational new drug application (IND) is the application by the study sponsor to the FDA to begin clinical trials in humans.

  5. The IND should be amended as necessary. The four types of documents used to amend the IND include:

    1. Protocol amendments

    2. Information amendments

    3. IND safety reports

    4. IND annual reports

  6. After sufficient evidence is obtained regarding the drug’s safety and effectiveness, the sponsor will submit a new drug application/biologics licensing application to the FDA requesting approval of the agent for marketing.

  7. The FDA allows the manufacturer to charge for an investigational drug under certain conditions.

  8. An orphan drug is one that is used for the treatment of a rare disease, affecting fewer than 200,000 people in the United States, or one that will not generate enough revenue to justify the cost of research and development.

  9. Drug accountability records are mandated by law. They can be computerized or in paper form. Necessary components include:

    1. Transaction date

    2. Transaction type

    3. The receiving party (for patient dispensing this should include patient initials/identifying number)

    4. The dose/number of units dispensed/received

    5. The lot number dispensed/received

    6. The initials of the person who performed the transaction



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