In a recent statement, Scott Gottlieb, the Commissioner of the US Food and Drug Administration, has re-affirmed the organisation’s commitment to modernising drug regulatory pathways. New changes are hoped to help the FDA’s processes keep up to date with recent medical developments, such as the growth of targeted therapies and drugs that address the underlying causes of disease (such as gene therapies). To read the statement in full, you can view it at the FDA’s website here.
In the statement, the FDA says they want to make the process of gathering evidence for an experimental drug “more modern, more scientifically rigorous, and more efficient.” As part of this aim, they say that they are developing regulatory frameworks that will be technology and disease-specific. This could help modern experimental drugs that don’t easily fit into existing FDA pathways. They also say that they want to encourage more competition with similar types of drugs, in the hope that this will bring down costs and offer more options to patients.
To support the development of new forms of drugs, such as targeted therapies, the FDA has made several changes. These include a recently announced pilot program, which is hoped to encourage drug developers to collaborate and use innovative trial designs, particularly when it comes to rare diseases and unmet patient needs. The FDA has also released two guidance documents that outline possible approaches to innovative trials.
In addition to this, in their recent statement, the FDA mentioned two new documents that drug developers can refer to when developing cutting-edge treatments. One is focused on haematologic malignancies, while the other is for targeted therapies.
This year, the FDA says that it has approved 45 drugs already – only one fewer than the 46 drugs it approved in 2017, and they anticipate more approvals before the end of the year. Newly approved drugs include treatments for several rare diseases, such as Dravet syndrome, Fabry disease, and polyneuropathy linked to hereditary transthyretin-mediated amyloidosis. These steps towards modernising the FDA’s regulatory pathways are hoped to bring more drugs to patients who urgently need them.