When the U.S. Food and Drug Administration (FDA) decides to approve a new drug, it does so with very good reason. A drug that has been approved by the FDA means that it has passed many requirements and has been deemed as safe and effective for its use as possible by the FDA’s standards.
While getting FDA approval isn’t easy and can take quite some time, this article will provide you with all the information you need to know about how the FDA decides to approve a new drug, and what steps you might have to take in order to get FDA approval yourself!
The Research and Development Phase
The FDA’s first step in reviewing an application for a new drug is to determine whether or not it has jurisdiction over that application.
This means they must consider:
1) Is it really a new drug?
2) If so, is it similar enough to an existing product that they can use information submitted by another company (i.e., one of its references listed drugs)?
3) How will they handle applications for generic drugs?
Once these questions are answered, they evaluate safety and effectiveness data. To prove safety, companies need data about adverse events associated with use of their product.
They usually rely on studies done in animals or humans; however, under some circumstances companies can use history of traditional use in addition to data from animal and human studies.
The Filing with the FDA
The United States Food and Drug Administration (FDA) approves drugs before they can be sold in pharmacies. This approval process is called licensing and it includes three steps: filing, reviewing, and licensing.
To get a drug licensed, you need to fill out a New Drug Application (NDA) Form FDA 1572. The first page of an NDA contains basic information about your company and lists any patents for your product that are going to expire during its projected 12-year lifespan.
These expired patents tell competitors that their medication won’t infringe on your patent—so once your patent expires, it’s not as likely that there will be a lot of generic competition immediately after launch.
For now, let’s focus on how to write up an NDA; we’ll touch on what happens when your application is approved later in our series. After preparing all sections of your NDA form FDA 1572, you submit it to the FDA’s Center for Drug Evaluation and Research (CDER).
At CDER, each section gets reviewed by people with different specialties such as pharmacology or bio-pharmaceutics. Afterward, people from each specialty meet to discuss a final recommendation on whether or not your drug should receive FDA approval.
If you follow good clinical practices while testing your treatment in humans, it’s very likely that your drug will make it through these three hurdles successfully.
The Review of New Drug Applications
The process begins when an application for approval is filed with FDA by an applicant, who must provide substantial evidence that shows:
(1) The product is safe and effective for its intended use;
(2) The labeling of promotional claims is truthful and not misleading; and
(3) The manufacturing practices used in producing it are adequate to preserve its identity, strength, quality, and purity.
In reviewing applications, FDA may consult experts outside of FDA on scientific or technical matters. The agency may also review existing scientific literature pertaining to a new drug application.
All written communications submitted by outside consultants are available to both FDA reviewers and applicants during review of applications. Information in these communications may be considered in making approval decisions.
Pre-Approval Clinical Trials
A small number of drugs don’t go through Phase I and II clinical trials. Instead, pharmaceutical companies start out by testing these drugs on people who suffer from certain conditions (such as cancer) in clinical trials that are conducted under an Investigational New Drug (IND) application.
The FDA issues an IND after reviewing data submitted by a company that indicates that a particular investigational drug appears safe and has potential therapeutic benefits.
In some cases, pre-IND meetings with FDA staff may take place before an IND is submitted so both parties can get on the same page regarding proposed trial protocols and objectives.
Once a drug has been approved by the FDA, it can still be taken off of store shelves if post-approval studies reveal dangerous side effects or other problems.
This can happen for several reasons: Patients are taking more than one medication and aren’t following directions properly, doctors are prescribing incorrect dosages, or there is faulty packaging (more about that here).
Not only that, but once drugs hit pharmacy shelves, they are prescribed in slightly different ways depending on patients. For example, one patient may take a daily dosage while another takes half as much every other day.
Or maybe they skip days or add time between doses. All of these factors can have serious consequences on efficacy and safety.
The FDA uses five criteria to determine whether or not to approve a medicine for sale.
First, there has to be data that shows how well it works.
Second, there needs to be information on what possible side effects are associated with its use.
Third, it must be demonstrated that it’s safe in humans–for example, animals might react differently than humans.
Fourth, doctors have to show they understand how to prescribe and use it.
Fifth, there should be guidelines available to make sure patients are aware of all the risks involved when taking a particular drug.
Finally, FDA looks at economic issues like competition between similar drugs already on market and pricing information provided by pharmaceutical companies. There is no formula; each medication follows its own path through FDA approval process.
A given drug might get approved without an additional study or without having been tested in children (although many do undergo further testing after initial FDA review).
For example, Plan B One-Step was approved on April 4th, 2013 based on data showing it was safe and effective in preventing pregnancy after unprotected sex. In January 2014, Plan B One-Step made history as one of very few emergency contraceptive medications available over-the-counter to people ages 15 years and older without needing a prescription from their healthcare provider.
After doing extensive research on how women were using emergency contraception products—including collecting feedback from women who were buying EC directly from Canadian pharmacies over state lines—FDA made a decision about approving Plan B One-Step for unrestricted sale with no prescription required.