FDA's Accelerated Approval Process - Benefits and Requirements
FDA's Accelerated Approval Process: Benefits and Requirements
The FDA has an accelerated approval mechanism for new drugs. Is accelerated FDA approval a good thing? Probably Yes. This mechanism of approval was put into place to help patients who suffer from certain serious diseases.
As the phrase suggests accelerated FDA approval involves a faster approval than for non-accelerated approvals. To get an accelerated approval sponsors must submit their application to the FDA. Before an application is accepted for approval, two major regulatory tests described below are applied.
THE DRUG HAD TO TARGET SERIOUS CONDITIONS
What is a serious condition? If you are a patient almost every disease would be a serious condition. The FDA has clarified this in a guidance document.
This is a disease or condition which has significant impacts on a person's day-to-day ability to function or even die.
Clinical judgment has to be made in determining whether a disease is serious or not.
The judgment is based on the impact of the disease on factors such as the patients' survival and day-to-day functioning. In addition, an assessment is made on the likelihood that the disease will progress to a more severe condition if no treatment is provided.
There are other three pathways for expedited approval of NDA which require establishment of serious condition. They include fast track, breakthrough therapy and priority review.
All diseases that are life threatening qualify as serious illnesses but not necessarily for accelerated FDA approval as we will see later. Some will qualify for fast track, breakthrough therapy and priority review but not for accelerated approval.
THE DRUG HAD TO BE FOR AN UNMET MEDICAL NEED
The term "unmet Medical Need" is used to refer to a medical condition that does not have adequate treatment or diagnosis. It may be for treatment of cancer or a method for better and faster diagnosis of a serious illness. The application of the term "unmet medical need" could be quite broad.
The term "unmet medical need" could be used where there is no available appropriate treatment to the patients. Sometimes a new therapy may be available but unmet medical need may still exist such as when the new reduces the symptoms but not the serious outcome such as progressive disability or progression of the illness.
WHAT THE SPONSOR SHOULD DO
Because many sponsors may want to use accelerated FDA approval, certain pre-qualification criteria have been put into place.
As the accelerated FDA approval application is based on surrogate endpoints. Sponsors should ordinarily discuss with the FDA of the intention to request for accelerated approval.
The request would help the sponsor to choose the best surrogate endpoints during the clinical trials. Surrogate endpoints are endpoints other than clinical endpoints which take longer to determine for diseases that take long to show a clinical endpoint.
For example, it might take long to show that a drug prolongs life. Prolongation of life is a clinical endpoint. Decrease in the blood in viral load such as in HIV would be a surrogate endpoint.
Surrogate endpoints include laboratory measurements and images among others. The use of clinical endpoints is however required in the confirmatory clinical trial reports.
By the time this application is being submitted the sponsor should be in a position to discuss the confirmatory clinical trials which should be in progress.
Without evidence of these confirmatory trials the FDA may be reluctant to approve the requested accelerated FDA approval. The risk to patients is exposure to expensive drugs which may offer no clinical benefits. The sponsor must provide evidence for commitment to complete the pending confirmatory trials.
Does Canada have a similar program to accelerated FDA approval? The Canadian equivalence is called priority review. In this program the sponsor is encouraged to request for priority review status early. The sponsor will get a notice of compliance status with conditions after the application when the only data available is from surrogate end-points.
The target review period is 200 days. Just as in USA, the sponsor has to submit confirmatory clinical trial data for full compliance without conditions.
