The FDA approval process

The time it takes for new drugs to be approved takes many years. Let's take a closer look at the process the US Food and Drug Administration (FDA) generally follows to better understand how new potential medicines are evaluated.

THE FDA

The FDA's role is to evaluate data on an investigational drug and determine whether it should be provided to patients. The FDA aims to ensure that drugs work, and that their health benefits outweigh their known risks. It also provides doctors and patients the information they need to use medicines properly.

10%

of drugs that enter clinical
testing are ultimately approved

DRUG COMPANY

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Scientists discover a new compound with the potential to become a meaningful medicine.

Drug company tests the drug on animals to learn more about its safety and how it works.

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IND SUBMISSION

Drug company submits an Investigational New Drug (IND) application to the FDA with key information including:

  • What the drug is made of and how the company plans to manufacture it
  • Results from animal testing
  • Plans for testing the drug in humans (clinical trials)
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IND REVIEW

FDA works to ensure proposed clinical trials:

  • Do not place participants at extreme risk or harm
  • Provide informed consent and protection for participants
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If the FDA approves the IND,
clinical trials can begin.

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If the FDA approves the IND,
clinical trials can begin.

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Clinical trial phases

Phase 1

Testing with a small group
(generally 20-80 people)

  • WHO? Often, people who do not have the disease (healthy volunteers)
  • WHY? To better understand the safety profile, including side effects
Phase 2

Testing with a larger group
(generally several hundred people)

  • WHO? Patients who have the disease
  • WHY? To find out if the drug is providing the desired effect, how effective it is, and what dose works best. More safety information is also collected
Phase 3

Testing with an even larger group
(generally 300-3,000 people)

  • WHO? Patients who have the disease
  • WHY? To confirm the drug is effective, better understand the safety profile (including side effects), and sometimes compare it with other current or similar treatments

 

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NDA SUBMISSION

Company submits a New Drug Application (NDA), including the results of all trials, as well as manufacturing information, to the FDA.

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NDA ACCEPTANCE AND REVIEW

60 days later, the FDA decides if it will accept
the NDA application.

 

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  • 10 months to review the application for drugs that offer only minor improvements over current treatments

or

  • 6 months for drugs that offer major advances in treatment or provide a treatment where there was none


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Before approval, the FDA:

  • Reviews and approves the label for the drug to make sure it includes important information healthcare professionals and patients need to know
  • Inspects the facility where the drug will be manufactured

100,000pages

are typically in a
New Drug Application

 

10+years

the time it takes for
a drug to progress from
an idea to an approval

Can the approval process be shortened?

The FDA may be able to approve a drug more quickly if it is for rare or serious diseases. Available options include:

FAST TRACK
FAST TRACK

Fast track is a process designed to help shorten the time it takes to develop and review drugs that treat serious conditions and fill an unmet medical need. It allows early and frequent communication between the drug company and the FDA to help resolve questions and issues quickly.

BREAKTHROUGH THERAPY
BREAKTHROUGH THERAPY

The FDA can speed up the development and review of a drug if evidence from early clinical trials shows that it is a substantial improvement compared with other available treatments. The evidence can include the effect of the drug on serious symptoms, or how it provides life-saving benefits.

PRIORITY REVIEW
PRIORITY REVIEW

A priority review means that the FDA will aim to take action on an NDA within 6 months (compared with 10 months under standard review).

ACCELERATED APPROVAL
ACCELERATED APPROVAL

An FDA regulation that allows early approval for drugs that treat serious conditions or address an unmet medical need. Approval may be granted based on a surrogate endpoint — a measure that reasonably predicts how well a drug may work.