Lorrin M. Melanson, Associate in the Health Care & Life Sciences practice, in the firm’s Washington, DC, office, was quoted in Law360 Healthcare Authority, in “FDA Rare-Disease Guidance Brings Both Hope and Questions,” by Dan McKay. (Read the full version – subscription required.)
Following is an excerpt:
A year ago, an infant in Philadelphia received the first in a series of infusions that would help save his life.
It was a medical breakthrough: "Baby KJ" was the first person to be successfully treated with a personalized CRISPR gene-editing therapy.
Federal regulators say they want to see similar treatments take off and are taking concrete steps to make it happen.
The U.S. Food and Drug Administration is touting what it calls a transformational regulatory framework that will accelerate approval of new therapies for extremely rare diseases. They include KJ's metabolic disorder, which can cause a toxic buildup of ammonia in the blood and lead to brain damage or even death.
In a 23-page guidance document last week, the FDA outlined how it proposes to evaluate the safety and efficacy of gene-editing tools and similar treatments targeting diseases so rare that a randomized controlled trial isn't feasible. This "plausible mechanism framework" is meant to allow researchers to present evidence that an intervention works based on the experience of a small sample of patients rather than a large clinical study. ...
Lorrin M. Melanson, a healthcare attorney at Epstein Becker Green, said the document makes clear the proposed framework isn't an entirely new approval pathway. Drugmakers would still have to eventually seek approval through the accelerated or traditional regulatory process; the framework outlines how the company and FDA would evaluate the drug's impact in the absence of a large trial.
"This pathway notably does not lower standards for safety and efficacy," Melanson said. "Just as with any other drug, individualized therapies proceeding through this framework will need to provide substantial evidence of effectiveness for intended use and sufficient information to conclude that the drug is safe for use as prescribed or suggested in labeling." ...
"While the guidance leaves some details to be determined in future publications, the possibilities that this guidance raises has already generated excitement from voices in the industry," said Melanson of Epstein Becker.
