Page 6 - HFA Dateline 2019-2020 Special Issue

A Look at the

Drug Approval Process

What is a drug?

U.S. Food and Drug Administration defines a drug as any product intended for the use in the diagnosis, cure,
mitigation, treatment or prevention of disease, and is intended to affect the structure or any function of the body.

PRE-CLINICAL: DRUG SPONSOR’S DISCOVERY AND SCREENING

Drug Developed
A sponsor develops a new drug compound and seeks approval by the FDA for sale in the U.S.

Animals Tested
1 Sponsor uses multiple species of animals to test new drug for toxicity, gathering basic information on the safety

and efficacy of the compound being investigated/researched.

Sponsor Submits Investigational New Drug Application
2 The sponsor submits an Investigational New Drug application to FDA. This application includes the drug’s

composition and manufacturing specifications and offers a plan for testing the drug on humans.

IND Review:
FDA reviews the application to verify there are adequate informed consent and human subject protections,
and assures that the proposed studies, generally referred to as clinical trials, do not place human subjects at
unreasonable risk of harm.

CLINICAL TRIALS: DRUG SPONSOR’S STUDIES AND TRIALS WITH PATIENT VOLUNTEERS

20 to 80 Healthy Volunteers

3 Sponsor Conducts Phase 1

The sponsor selects 20 to 80 healthy volunteers during this phase, which emphasizes safety. This phase determines
the most frequent side effects and how the drug is metabolized and excreted.

100s of Patients

4 Sponsor Conducts Phase 2

The sponsor selects hundreds of patients for this phase, which emphasizes effectiveness and gathers preliminary
data on whether the drug works in people who have a certain disease or condition. They continue to evaluate
safety and short-term side effects. For controlled trials, patients receiving the drug are compared with similar
patients receiving a different treatment—usually a placebo or a different drug.
At the end of Phase 2, FDA and sponsors discuss how large-scale studies in Phase 3 will be done.

6 HEMOPHILIA FEDERATION OF AMERICA < www.hemophiliafed.org