According to a story from Pharma Voice, Dr. Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA) has recently released a statement in regards to efforts by the agency to promote the development of targeted therapies, modernize the drug development process, and improve efficiency.
New Drug Technologies
In his statement, Scott mentions the rising impact of new technologies that are becoming increasingly more popular as drug platforms, such as gene therapies, cell therapies, and small molecules. Such mechanisms often involved intervening in the genetic basis of a disease, and therefore are more adept at addressing the underlying causes of disease. These new drugs have the potential to slow, halt, or even reverse the progress of many rare, long term, progressive, debilitating disorders and illnesses. Scott elaborates that these new technologies will also require changes to the regulatory structure so that they can be assessed and tested accurately.
Changes to these regulatory mechanisms, Scott claims, are essential for ensuring that drugs utilizing these new technologies are as effective as possible, as well as ensuring that they are safe for use. Part of this process is taking into account the specific characteristics of different diseases and technologies and implementing regulatory frameworks that take into account these characteristics. Many of these technologies have not had an effective, efficient, or well structured developmental and regulatory pathway in the past.
Rare Disease Innovations and a Lot of Other Drugs Too
Scott, perhaps somewhat regrettably, has also touted the fact that 45 novel drugs or therapies have been approved so far in 2018; last year, the FDA approved 46. He refers to these high numbers as a “strong year,” but it is worth noting that the FDA approving more therapies per year than in the past is not necessarily a completely good thing, as it could be a sign of a less strenuous and scientifically rigorous regulatory process, or even conflicts of interest within the agency. Regardless, the agency has approved some important rare disease treatments in 2018 for diseases like polyneuropathy caused by hereditary transthyretin-mediated amyloidosis, Dravet syndrome, and Fabry disease. These innovations are not to be taken lightly.