Aside from being a spectacular thing to watch, the slam dunk is the highest percentage shot a basketball player can take. The likelihood of getting the ball in the basket is so high that the shot is almost a sure thing.
In the world of the FDA, sure things don’t exist. Approvals are never assured; however, in the right hands, they can get pretty close.
When developing a new drug that is believed to offer a strong benefit to society, sponsors sometimes feel that they should have a quick and assured approval from the FDA. These expectations are unrealistic FDA’s responsibility is to protect the American Public, and they have established very strict standards and processes that guide its efforts to determine the safety and efficacy of the drug or device.
The FDA Will Not Compromise Its Standards
Even if the brand-new drug is a life-saving product, the FDA will not compromise its standards. The Agency will always require a data-driven determination of the safety and efficacy of a drug. Furthermore, the sponsor and any of its contractors must also have followed good clinical practice (GCP) and good manufacturing practice (GMP) standards.
One of the most important considerations is the risk/benefit analysis that the FDA conducts when evaluating a drug. In the analysis, if the benefit is very significant, then the balance between risk and benefit will tend to shift to the side of the benefit and away from the risk. For example, imagine that there is a drug that has certain adverse effects, but it brings with it an outstanding benefit. In that case, assuming all other standards have been sufficiently met by the sponsor, the Agency may approve the drug because of the overwhelming benefit that the drug provides.
There Are No Shortcuts to Approval
Sometimes sponsors believe that because their drug has such an expected high benefit they can take a shortcut and obtain approval faster or with less data. Fortunately for the safety of the public, there are no such shortcuts.
Moreover, attempting to take shortcuts can cost a sponsor an enormous amount of time and money. One example is the sponsor that decides to conduct a small, relatively inexpensive clinical trial thinking that the FDA will approve their product based on the data derived from the trial. After reviewing the data, the Agency may decide that the study doesn’t sufficiently demonstrate efficacy. In this situation, the sponsor wasted time and money conducting the first clinical trial, and must now go back to the drawing board, potentially having to conduct a larger, more expensive, and time-consuming trial to get their drug approved.
Drug Development Designations
While sponsors should never expect a slam dunk, there are several drug development designations that can expedite the process without needing to take a shortcut. These designations are meant to encourage the development of certain drugs, especially those that may represent the first available treatment for an illness.
Each designation helps ensure that therapies for serious conditions are made available to patients as soon as FDA reviewers has concluded that their benefits justify their risks:
Fast Track is a review process that facilitates the development and advanced review of new drugs that treat serious conditions and fill an unmet need. The goal of the Fast Track designation is to get important new drugs to patients earlier.
Breakthrough Therapy designation is intended to expedite the development and review of drugs that are intended to treat a serious condition. Preliminary clinical data must indicate that the drug may demonstrate substantial improvement over currently available therapies.
Priority Review means that the FDA will act on an application within six months, rather than the standard review timeline of ten months. A Priority Review designation directs the Agency’s attention to the evaluation of applications for drugs that would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
The sponsor must make a specific request to receive one of these designations.
Let Us Coach You Towards Approval
At The Weinberg Group we understand the internal and external pressures that make some sponsors attempt to get a slam dunk approval. That’s why we take the approach of any good coach, focusing on the fundamentals. With our help, you can develop a realistic regulatory strategy that will move your product closer to approval as you reach and surpass each necessary milestone. Contact us today to learn more about our services and how we can help you.