Addressing Prohibitive Costs for Rare 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: How can prohibitive costs for treatments be addressed?
Lisa Bollinger: These are oftentimes very expensive therapies, especially if you think about things like gene therapy. The problem is that there is a substantial cost to developing these drugs, and with these lysosomal storage diseases, there are so many challenges.

For example, the drugs have to get into the brain, so how do you make a drug that can cross what's known as the blood brain barrier? It's a protective mechanism in our bodies that prevents a lot of foreign things from getting into a very protected site, our brain. A lot of these therapies don't have the ability to penetrate into the brain, especially when you think about things like enzymes.

When you think about gene therapy, it has to be delivered directly to the brain. So, number one, there's a substantial development cost that comes with developing these drugs. There's a small patient population, so sometimes you also must price your drug high to recoup some of that R&D expense.

At Polaryx, we're working with a repurposed drug. We're reformulating it and trying to reach a broader patient population. Maybe one of the ways to get cost down is to be able to have a therapy that can help across lysosomal storage diseases, rather than just going for a single lysosomal storage disease.

No matter what we do initially, the cost is going to be expensive, as these are small patient populations and the therapies that we're developing are pretty advanced therapies, and they have a substantial R&D cost with them.