FDA commissioner says more patients can expect ability to arrest illness or achieve cure
Scientific and medical advances are giving the US Food and Drug Administration (FDA) more opportunities to address diseases and illness.
In a statement on the proposed modernising of the FDA’s drug review office, FDA commissioner Scott Gottlieb said that while at one time “all that patients may have hoped for was the ability to control a chronic disease, and perhaps slow its advance or lessen its insidious effects, more patients today can expect the ability to arrest the march of illness or achieve an outright cure.
However, with this progress comes more complexity. Not only are there challenges related to the science of how drugs are discovered, but also the manner in which they are developed. For very novel drugs targeting unmet needs, this often doesn’t follow the traditional three phases of clinical trials.”
The FDA has introduced advances in how it evaluates drugs for safety and effectiveness, as well as the manner in which clinical trials are guided. These include adaptive approaches to clinical development such as the introduction of trial designs, master protocols or tissue agnostic product approvals. “Each allow us to better marry the scientific prospect more closely to the approaches that can best unlock these opportunities,” said Scott Gottlieb.
He said the FDA wanted “to give our clinicians and scientists more time, better tools and greater support to advance the clinical and regulatory principles that the FDA uses to evaluate new drugs for safety and efficacy”.
One of the FDA’s goals is to issue more product-specific guidance documents. “We’ll develop hundreds of new clinical guidance documents and make sure they stay up-to-date to reflect the latest science,” he said.
Scott Gottlieb went on to say that the organisation is also looking to “engage the external community more closely in our work, especially patients who inform our patient-focused drug development, with the aim of advancing more modern regulatory principles.”