USA – FDA seeks ways to promote individualized therapies

The US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) is looking for ways to facilitate the development of more products intended to treat individual or small groups of patients, including cell and gene therapies, antisense oligonucleotides and phage therapies.

At a workshop at FDA’s headquarters in Silver Spring, MD on Tuesday, CBER Director Peter Marks addressed some of the barriers to developing individualized therapies such as “customized products” with a single indication and a mode of action that are tailored to individual patients, or “created products” that could target different indications via different modes of action.

Marks noted that there are “probably some regulatory distinctions between these two classes.”
The workshop comes after the landmark use of an individualized antisense oligonucleotide therapy was documented in the New England Journal of Medicine last year. In an accompanying editorial, Marks and Center for Drug Evaluation and Research Director Janet Woodcock raised questions about how these so-called “n-of-1” therapies would be regulated.


According to Marks, one of the biggest challenges to developing gene therapies for small populations is in manufacturing. Marks noted that while second and third generation genome editing tools have been developed, vectors are being produced “much the same way that we made them at the turn of the millennium.”

“The setup costs, currently, to make a gene therapy for 20 people are very similar to the setup costs to make a gene therapy for 200 people, in terms of commercial process,” he said.

Marks also cited challenges in nonclinical and clinical development when considering products developed to treat a single patient or very few patients…