The FDA – Medical Review Process & Development Programs

What Is the FDA?

The U.S. Food and Drug Administration (FDA) is an agency that is a part of the U.S. Department of Health and Human Services. Specifically, the FDA can be broken down into five primary sections. This includes the Office of the Commissioner, as well as four directories that oversee the fundamental responsibilities of the FDA. The flour directories are in charge of Food, Medical Products and Tobacco, Operations, and Global Regulatory Operations and Policy. 

How Is the FDA Organized?

The U.S. Food and Drug Administration (FDA) is an agency that is a part of the U.S. Department of Health and Human Services. Specifically, the FDA can be broken down into five primary sections. This includes the Office of the Commissioner, as well as four directorates that oversee the fundamental responsibilities of the FDA. The four directorates are in charge of Foods, Medical Products and Tobacco, Operations, and Global Regulatory Operations and Policy.

Where Does the FDA Have Jurisdiction?

The scope of the FDA’s responsibilities extends beyond the immediate 50 United States. Additional territories under FDA regulation include Guam, Puerto Rico, American Samoa, the Virgin Islands, and other areas of jurisdiction within the United States. In addition, the FDA regulates the same products and scientific exploration within the District of Columbia.

When Was the FDA Founded?

The FDA has a long history of protecting consumers from illnesses, injuries, and diseases through ingestion of drugs and food. The FDA has changed since its first introduction in America along with the many social, economic, political, and legal changes.

The FDA’s functions of protecting the public began in 1906 with the passage of the Pure Food and Drugs Act. This prohibited interstate commerce in adulterated and misbranded foods and drugs. The FDA then began providing protection that consumers didn’t know they needed at that time. 

What Is the FDA Responsible For?

According to the U.S. Food and Drug Administration (FDA) website, the FDA is responsible for: 

• Regulation of tobacco products.
• Protecting the American public from electronic radiation from approved products. 
• Assuring the safety of and proper labeling of cosmetics. 
• Assuring the safety and proper labeling of dietary supplements. 
• Protecting public health through regulation and assurance that foods are wholesome, safe, sanitary, and properly labeled. 
• Protecting public health through regulation and assurance that vaccines, human drugs, medical devices, veterinary drugs, and other biological products that are approved for use within the United States are both effective and safe. 
• Helping the American public attain access to accurate scientific information that is required to use devices, medicines, and foods to maintain the best possible health. 
• Advancing American public health by aiding with the speed of product innovations.

What Does the FDA Regulate? 

With the FDA being around for a long time, a lot of products fall under its jurisdiction.

The different products that the FDA regulates include: 

• Drugs and biologics
• Medical devices
• Human cells and tissues
• Tobacco Products
• Animal medications
• Food
• Color additives
• Products that give off radiation

What Doesn’t the FDA Regulate?

With how much falls under the FDA, it’s easier to ask, “What doesn’t the FDA regulate?” The list is significantly shorter, but includes: 

• Cosmetics
• Medical foods
• Dietary supplements
• Baby formula

What Are the FDA Medical Review and Development Programs?

With the FDA being responsible for advancing American public health, the FDA agreed to a set list of goals to improve a medical product’s review time. The FDA Fast Track Development Program originated under the Prescription Drug User Act (PDUFA) in 1992. 

What Are the Three FDA Sub-Categories?

This FDA program evolved into three sub-categories: FDA Fast Track Program, FDA Accelerated Approval Program, and FDA Priority Review Program. These different programs work in unique ways towards a common goal: to make therapeutically important medical products available to the American public at an earlier time.

What Is the FDA Fast Track Program? 

The Fast Track program aims to expedite the development and review of new drugs or medical devices that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. 

How Does the FDA Decide That an Illness is Serious?

Determining whether a condition is serious is generally based on whether the drug will have an impact on survival, day-to-day functioning, or the likelihood of the condition becoming more serious if left untreated. 

Conditions that fall under this category include:

• AIDS
• Alzheimer’s
• Heart failure
• Cancer
• Epilepsy
• Depression
• Diabetes

What Happens to a Fast-Track Drug if It Meets a Medical Need?

The FDA defined that the medical product must offer a new and immediate benefit to the healthcare industry. Filling an unmet medical need means providing a treatment where none exists or providing a treatment that may be potentially better than available options. 

A drug being developed to treat or prevent a condition with no current treatment is immediately directed to be fast-tracked since there are no other treatment options available.

However, if there are already available treatments, a Fast Track drug must show an advantage such as: 

• Showing superior effectiveness
• Avoiding serious side effects of an available treatment
• Improving the diagnosis of a serious condition 
• Decreasing clinically significant toxicity of an available treatment 
• Ability to address emerging or anticipated public health needs

What Is a Drug with Fast Track Designation Eligible for?

A drug that receives Fast Track designation is eligible for some or all of the following: 

• More frequent FDA meetings to discuss a developmental plan and ensure the collection of appropriate data to support drug approval.
• More frequent communication from the FDA about clinical trials and the use of biomarkers.
• Eligibility for Accelerated Approval and Priority Review, if criteria are met.
• Rolling Review, which means that the drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review rather than waiting until the entire application is filled out. 

What Is the FDA Accelerated Approval Program? 

The Accelerated Approval Program allows drugs for serious conditions that fulfill a medical need to be approved based on an expedited benefit, rather than a benefit proven with clinical trials and studies. The company is still required to conduct studies and trials, but they can do so after approval in order to keep their drug or device on the market. 

What Are Surrogate and Intermediate Clinical Endpoints?

A surrogate endpoint used for accelerated approval is an indication in the form of a lab measurement, radiographic image, physical sign, or any other measure that predicts clinical benefit. This prediction is made to assume that, when the clinical studies and trials happen, this clinical benefit will be seen. 

Similarly, an intermediate clinical endpoint is a measure of a therapeutic effect that is considered likely to predict the clinical benefit of a drug, like an effect on irreversible morbidity and mortality (IMM).

How Does a Drug Get Approved Through the Accelerated Approval Program?

The FDA bases its decision to accept the surrogate or intermediate clinical endpoint on the scientific support for that endpoint. Instead of having to wait to learn if a drug actually has the predicted effect – since that could take years or decades to confirm – the FDA can approve a drug based on the evidence. The drug company will still need to conduct ongoing studies. 

The ongoing studies will determine if the drug or medical device stays on the market. If the product fails to show the clinical benefit that the surrogate or intermediate clinical endpoint predicted, the FDA can withdraw its approval of the product. Additionally, they can choose to take the product off the market. 

What Is the FDA Priority Review Program?

The FDA created a two-tiered system of view times – Standard Review and Priority Review. 

Standard Review is the suggestion for medical products that only offer minor improvements over preexisting treatments. Standard Review time is supposed to take no longer than 10 months for a new drug or medical device to be approved.

What Is Priority Review Designation?

Priority Review Designation is for drugs or medical devices that would provide significant improvements in an already existing treatment.

Significant improvements can look like: 

• Evidence of increased effectiveness in treatment, prevention, or diagnosis of the condition.
• Elimination or substantial reduction of a treatment-limiting drug reaction.
• Documented enhancement of patient compliance to use drug or medical device
• Evidence of safety and effectiveness of the new medical product

The FDA decides on the review designation for every application, but a company can request that their drug or medical device receive Priority Designation. Designation of a drug as “Priority” does not change the evidence needed to prove that the drug or medical device is effective and safe.