The development of drugs and medical devices follows well-established paths to make sure that they are safe and effective when they reach the public. From concept to approval and beyond, FDA:

o   Reviews research data and information about drugs and devices before they become available to the public
o   Watches for drug problems once drugs and devices are available to the public
o   Monitors drug information and advertising
o   Protects drug quality

The development processes for drugs and devices are similar – each involves five basic steps. However, the processes differ within those steps.

Step 1: Discovery/Concept
Research for a new drug or device begins in the laboratory.

Step 2: Preclinical Research
Drugs and devices undergo laboratory and animal testing to answer basic questions about safety.

Step 3: Clinical Research
Drugs and devices are tested on people to make sure they are safe and effective.

Step 4: FDA Review
FDA review teams thoroughly examine all of the submitted data related to the drug or device and make a decision to approve or not to approve it.

Step 5: FDA Post-Market Safety Monitoring
FDA monitors all drug and device safety once products are available for use by the public.

Speeding the availability of drugs that treat serious diseases are in everyone's interest, especially when the drugs are the first available treatment or if the drug has advantages over existing treatments. The Food and Drug Administration has developed four distinct and successful approaches to making such drugs available as rapidly as possible.

These include the following:

Fast Track
Fast track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions.

Determining whether a condition is serious is a matter of judgment, but generally is based on whether the drug will have an impact on such factors as survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress from a less severe condition to a more serious one. AIDS, Alzheimer’s, heart failure and cancer are obvious examples of serious conditions. However, diseases such as epilepsy, depression and diabetes are also considered to be serious conditions.

Breakthrough Therapy
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

Accelerated Approval
When studying a new drug, it can sometimes take many years to learn whether a drug actually provides a real effect on how a patient survives, feels, or functions. A positive therapeutic effect that is clinically meaningful in the context of a given disease is known as “clinical benefit”. Mindful of the fact that it may take an extended period of time to measure a drug’s intended clinical benefit, in 1992 FDA instituted the Accelerated Approval regulations. These regulations allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint. Using a surrogate endpoint enabled the FDA to approve these drugs faster.

Priority Review
Prior to approval, each drug marketed in the United States must go through a detailed FDA review process. In 1992, under the Prescription Drug User Act (PDUFA), FDA agreed to specific goals for improving the drug review time and created a two-tiered system of review times – Standard Review and Priority Review. A Priority Review designation means FDA’s goal is to take action on an application within 6 months (compared to 10 months under standard review).

A Priority Review designation will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

Because each of these approaches implies speed, there can be confusion about the specific meaning of each and the distinctions among them. 

To add the confusion, is the FDA's Compassionate Use (or Expanded Access) Program.

Compassionate Use (or Expanded Access) Program
Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

Expanded access may be appropriate when all the following apply:

o   Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition
o   There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition
o   Patient enrollment in a clinical trial is not possible
o   Potential patient benefit justifies the potential risks of treatment
o   Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication

Investigational drugs, biologics or medical devices have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.


Popular posts from this blog