The Regulatory Landscape for Rare Pediatric Diseases

Pharmaceutical Executive previously covered FDA’s efforts to expand the range of data allowed in clinical trials, including the use of the Bayesian method. This style of data collection allows for clinical trial data to be examined alongside existing, relevant data.

This expansion of data is especially important for clinical trials in any rare disease space that have limited patient populations. While clinical trials in this area still present unique difficulties, regulatory changes have provided solutions to issues related to limited data-sets.

Pharmaceutical Executive spoke with Lisa Bollinger, chief medical officer at Polaryx. The clinical-stage biotech focuses on treatments for rare, pediatric lysosomal storage disorders. Due to its focus, Polaryx has been able to update its trial design thanks to the previously mentioned regulatory updates.

Bollinger discusses the regulatory landscape in this area, along with the style of trial design that the company is utilizing and some of the unique challenges they still face.

Pharmaceutical Executive: What is the regulatory landscape like at the moment for rare pediatric diseases?
Lisa Bollinger: The rare pediatric disease landscape with regulators (focusing on FDA and EU) is very robust. What we've really seen over the last couple of years is a commitment to developing therapies for rare diseases, and this is really evidenced by the formation of a rare disease review division within the centers at CBER, as well as some additional offices like the Rare Disease Hub and Rare Disease representation within the Office of the Commissioner, as well

The one reason why I say this provides evidence that the regulators are really rallying around rare disease is because when you start staffing up and putting offices together to ensure collaboration across different centers at the FDA and EMA as well, what you really see is the agencies leaning into making sure that they can adequately support rare disease drug development.

We definitely see this infrastructure at both FDA and EMA. They've advanced several guidances for industry to help rare disease companies go through the process of developing trials and studying drugs.