The Food and Drug Administration (FDA) has reduced the paperwork involved in the process of obtaining an investigational drug for patients with rare life-threatening diseases. According to Richard A. Moscicki, MD, the FDA’s Deputy Center Director for Science Operations, Center for Drug Evaluation and Research on the FDA Voice, investigational drug access has now been streamlined and simplified. The FDA has issued a new application form with only 11 questions compared to the 26 questions in the previous form.
“FDA has recently made significant changes to streamline and simplify the process for single patient expanded access requests,” said Dr Richard Moscicki.
FDA approval is the final step before a drug can be officially launched for which drug companies gain approval for their experimental drugs by taking part in the Investigational New Drug (IND) application, which carries out human clinical trials to properly test the drugs. When the drug is being tested to determine all possible side effects, it is called an investigational drug.
An investigational drug can be defined as a drug with the potential to treat a fatal medical condition, but it is still being studied and does not have legal FDA approval for widespread marketing across the US.
According to the FDA, a rare disease can be defined as an affliction or disorder which affects less than 200,000 US citizens. Unusual forms of cancer, Alzheimer, AIDS etc. can be classified as rare diseases.
Although a myriad of drugs are now available for the treatment of many critical conditions, the FDA still has no focused therapies for a few rare diseases. There is a possibility that patients with such untreatable disorders can benefit from investigational drugs. Such patients can gain access to unapproved investigational drugs with the help of a physician.
In a process called expanded access to investigational drugs, the patient submits an application to the FDA. The process of ‘Expanded Access’ was designed by the FDA for those few patients who had exhausted all other treatment options. In the last six years, the FDA approved a vast majority of the forms submitted and had a 99% approval rate for drug applications. In 2015 alone, out of 1,430 only 14 drug access applications were rejected by the FDA, but the process of gaining drugs was still criticized by a few.
“We live in an era of unprecedented innovation in pharmaceuticals, diagnostics, medical devices, and food and nutrition science. Such innovation offers unprecedented opportunities to advance and improve public health. It is essential that we at FDA keep pace with these changes,” said Stephen Ostroff, MD, Acting Commissioner of Food and Drugs, at the 2015 FDLI Annual Conference in Washington, D.C.
Dr Moscicki shared that while the FDA has been helping patients gain access to new drugs some hurdles still remain. For one, the process had been very time consuming and sometimes difficult to understand for some patients which raised concerns. Therefore the new application form called the Form FDA 3926 has been issued which only requires answering 11 questions and one attachment while the previous form called Form 1571 had a total of 26 questions and required seven attachments. Comparatively, the new form can be filled out easily within 45 minutes.
“With this streamlined format, we estimate that physicians will be able to complete the form in just 45 minutes, as compared to the more difficult and time-consuming effort required previously,” said Dr Moscicki.
To ensure the simplification of the drug-accessing process, the FDA and the Reagan-Udall Foundation held a meeting on May 16, 2016. During the meeting all present stakeholders reflected on ideas which could help patients with rare disorders and in need of new drugs. The focus was to make sure both the patients and the doctor understood the protocol steps easily.
In the end, the FDA and the Reagan-Udall Foundation decided to start the forum on FDA Voice so that people could express their views on how the process could be made simpler and more efficient for them. The forum is still generating comments to date. One major decision of the meeting was in the acknowledgment of the need of a central repository or clearinghouse for storing all relevant information.
Dr Moscicki explained it “as a sort of “one-stop-shop” for physicians and patients to seek information about the expanded access process.”
The forum will also host a one-hour long webinar for patients and physicians who wish to gain more information on accessing an investigational drug, on July 12, 2016 at 1:00 PM EDT. The educational webinar will impart knowledge on how to use the new application form with ease. For further information on the Form FDA 3926, the forum has outlined guidance in the form of Questions and Answers.