FDA to provide clarity on drug development


The U.S. Food and Drug Administration (FDA) said it will be taking steps to clarify new drug development in the age of individualized medicine.

Given advances in scientific knowledge and drug development technology have provided new opportunities to approach drug development, the FDA said it has approved more than twice as many drugs for rare diseases, sometimes called “orphan drugs” in the past eight years as it had in the previous eight years.

“For genetic diseases, recent approaches to testing and molecular diagnosis have allowed us to pinpoint, in some cases, the exact cause of a patient’s disease,” the FDA wrote. “For a patient with a very rare genetic disease, development of a drug product that is tailored to that patient’s specific genetic variant may be possible. This is an important advance in treatment for those with very rare genetic diseases, especially those for which there are no adequate therapies available to treat the disease. Often, these very rare diseases are rapidly progressing, debilitating, and in many cases, can lead to premature death if left untreated.”

The FDA said the development of individualized genetic drug products is most advanced for antisense oligonucleotide (ASO) products. The agency said it would be taking steps to clarify its guidance on this area of individualized drug development.

The draft guidance will help those developing ASO products with how to approach obtaining feedback from the FDA and what the expectations and process for making regulatory submissions to the agency are, as well as make recommendations about the requirement for Institutional Review Board review of the protocols within, and how to obtain informed consent.
“The FDA understands that we’ll need to work together with the developers of these drug products to bring them safely to patients, and we are willing to engage as needed to address the challenges. For example, for those developing these drug products, it will be important to further understand the required data and information that must be submitted to the FDA so that clinical testing can begin. The FDA is continuing to consider and further develop policy to address some of these issues,” the agency wrote.