Page 7 - HFA Dateline 2019-2020 Special Issue

1,000s of Patients

5 Sponsor Conducts Phase 3
Thousands of patients are selected for this phase, which gathers more information about safety and effectiveness.
This study includes different populations, different dosages, and uses the drug in combination with other drugs.
To have a statistically-relevant sample size to draw conclusions about the drug being studied, the FDA requires the
number of patients to conduct a study/trial be changed based on the size of the patient population being studied.
Example: 200 trial participants studying hemophilia is one percent of the U.S. hemophilia population, whereas one
percent of the U.S. diabetic population in a diabetes study trial would equal 291,000 participants.

FDA’S NEW DRUG APPLICATION REVIEW

6 Review Meeting

FDA meets with a drug sponsor prior to submission of a New Drug Application.

7 Sponsor Submits NDA Application
The drug sponsor formally asks FDA to approve a drug for marketing in the U.S. by submitting an NDA, which includes
all animal and human data, analyses of the data, how the drug behaves in the body, and how it is manufactured.

8-9 FDA Reviews Application
The FDA has 60 days to decide whether to file the application for review. If the FDA files the NDA, a review team is
assigned to evaluate the sponsor’s research.

10 FDA Reviews Drug Labeling
FDA reviews the drug’s professional labeling and assures appropriate information is communicated to health care
professionals and consumers.

11 Facility Inspection
FDA inspects the facilities where the drug will be manufactured.

12 Drug Approval
FDA reviewers approve the application or issue a complete response letter, which will describe the specific
deficiencies that the agency has identified in an application.

CONTINUED SAFETY MEASURES: FDA’S RISK ASSESSMENT SYSTEMS

Phase 4
Because it’s not possible to predict all of a drug’s effects during clinical trials, post-approval and post-marketing
safety monitoring is critical. The FDA’s safety system is meant to detect unexpected adverse events, and take
definitive action when needed, after the drug has been approved and marketed. The sponsor (typically the
manufacturer) is required to submit periodic safety updates to FDA.

FDA’s MedWatch voluntary system allows physicians and consumers to report adverse events. When new risks are
discovered, they are added to the drug’s labeling and the public is informed through letters, public health advisories
and other educational means. In some cases, the use of the drug might be substantially limited and in rare cases,
the drug might be withdrawn from the market. S
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