As companies turn to rare disease as primary targets for drug development, they’re starting to hit a snag: It’s hard to find a sufficient patient population to test their drugs and, interestingly, must vie against one another to get access to these communities.
The Wall Street Journal ran an interesting piece on this dilemma, centering on the fatal genetic disease Niemann-Pick Type C. While there’s no approved therapy for NPC, there are three companies developing therapies – but there are only about 500 known cases of the disease worldwide. So the question is, can a rare disease community support more than one clinical trial at a time? The WSJ rightly points out:
As companies compete for participants, some scientists and families are worried that they may siphon patients away from each other, and the result will be that no trial has the heft to get a drug approved.
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This could be a real blow for patient communities, who have been the driving force behind bringing rare disease to the attention of biopharma companies.
“This is a critical juncture for our community,” Nadine Hill, executive director of the National Niemann-Pick Disease Foundation, told the WSJ. “To lose out on having an approved treatment is our worst-case scenario.”
These high-cost drugs can, after all, completely change the lives of these small patient pools. And drug companies are chasing the rare disease model, of course – of the 41 new drugs approved last year by the Food and Drug Administration, 17 were for rare diseases. The WSJ continues:
But even with more common conditions like cancer, it isn’t always easy to get patients onto trials. Patients worry they will get a placebo, and trials can require travel and time off work or school. Trials also require patients with very specific characteristics such as a particular age range or stage of disease, which limits the pool of potential participants—a problem exacerbated in rare diseases.
The article centers on three early stage companies: Maryland startup Vtesse, Florida-based CTD Holdings, and Danish startup Orphazyme. It points out the critical relationship between the startups and their patient populations – and how Vtesse, in particular, has won over the support of its patient population. It requires 51 patients for a clinical trial, and parents of NPC kids at a recent conference wore “51 and Done” pins to support the trial, the WSJ says.
CTD Holdings, to drum up support for its therapeutic Trappsol, is offering to donate $1 million worth of its drug as part of a compassionate use program – treating some 40 patients in a year.
And these three companies, unlike the concerned parents and patient advocates, seem to think it’s possible for companies to coexist in a crowded rare disease patient pool. However, as Orphazyme CEO AndersMørkeberg Hinsby told the WSJ, “it has to be coordinated well.”
[IMAGE: Flickr user Lisa Williams]
