FDA Takes Steps to Provide Clarity on Developing New Drug Products in the Age of Individualized Medicine Advances in scientific knowledge and drug development technology have provided an opportunity for new approaches to drug development, including the development of drugs for the treatment of rare diseases. These advances have contributed to an increase in development and approval of drugs for the treatment of rare diseases in recent years. In fact, in the past eight years, the U.S. Food and Drug Administration has approved more than twice as many drugs for rare diseases, often referred to as orphan drugs, as 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. 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. Developing these products – also referred to as "n of 1" therapies by some because they are designed for a patient population of one person – brings a set of challenges and considerations not seen with the typical drug development process. First, as noted above, the disease is often rapidly progressing, requiring prompt medical intervention. Therefore, development needs to proceed very quickly to have a chance at helping the individual. Second, drug discovery and development for these drug products may be carried out by academic investigators, rather than by biopharmaceutical or pharmaceutical companies. These investigators may be less familiar with FDA's regulations, policies and practices, and less experienced in interacting with the FDA. At this time, development of individualized genetic drug products is most advanced for antisense oligonucleotide (ASO) products. Therefore, we are taking the first steps in bringing clarity to this emerging area of individualized drug development by releasing a new draft guidance on investigational new drug (IND) submissions for individualized ASO drug products. The guidance was developed to advise those developing ASO products on an approach to interacting with, and making regulatory submissions to, the FDA. The guidance addresses the following points: - The approach to obtaining feedback from the FDA,
- The expectations and process for making regulatory submissions to the FDA,
- Recommendations about the requirement for Institutional Review Board (IRB) review of the protocols within, and
- How to obtain informed consent.
| 
No comments:
Post a Comment