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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.

  • List the steps in the drug approval process.

  • List the components of an Investigational New Drug Application (IND).

  • Describe the difference between commercial, treatment, emergency use, and individual investigator INDs.

  • 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 protocol assessments for use by the IRB or other review committees when they evaluate new human subject protocols.

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

    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

  • image The FDA is the federal agency that decides which drugs, biologics, and medical devices are safe and effective, determinations upon which the agency decides if a product can be marketed in the United States.

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

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

  • image The Investigational New Drug Application (IND) is the application submitted by the study sponsor to the FDA to begin clinical trials in humans.

  • image The IND should be amended as necessary. There are four types of documents used to amend the IND:

    1. Protocol amendments

    2. Information amendments

    3. IND Safety Reports

    4. IND Annual Reports

  • image 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.

  • image 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.

  • image Drug accountability records are mandated by law. They can be electronic or in paper form. Necessary components include the following:

    1. The date of the transaction

    2. The type of the transaction

    3. The receiving party of the transaction (if this is a drug dispensation, the subject initials and an identifying number are required)

    4. The number of units being used or received (for patient dispensation this should include the actual dose the patient will receive)

    5. The lot number ...

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