How the “Hard-to-Find Patient” Can Cost a Trial

How the “Hard-to-Find Patient” Can Cost a Trial

Rare disease drugs are growing in attractiveness because (a) they can benefit from the more favorable development environment put forth by the Orphan Drug Act and (b) the R&D invested into these niche opportunities are increasingly being found to pave the way toward therapies for more common diseases. That said, when it comes to clinical trial recruitment, the rare disease can prove to be particularly challenging because finding the prospective recruits can be akin to finding a “needle in a haystack.”

Rare disease patients are often widely dispersed due to the simple statistical nature of their condition—they are one of less than 200,000 throughout the country. What’s more, many rare diseases are subject to internal variance. For instance, while a disease may be linked to a mutation in a single gene, there may be dozens or even hundreds of mutation possibilities within that gene that affect therapy development.

These logistical and physiological barriers create challenges to finding and connecting with the target population for both clinical trial recruitment and subsequent product promotional campaigns and patient support programs.

A prime example is the failed 2011 Phase Ib clinical trial recruitment for StemCells’ investigational treatment for neuronal ceroid lipofuscinosis (more commonly called Batten disease). A fatal, inherited disorder that develops at an early age, NIH estimates it affects only 2-4 of every 100,000 births in the US – a statistic that unfortunately proved too overwhelming for StemCells.

VP Stephen Huhn stated, “Out of 22 initial prospects, not one patient has met the entry criteria.” This was an immense disappointment for the company and closed window of opportunity for those affected by the disease.

Fortunately, social media has opened up new possibilities for reaching patients with rare diseases. Combined with the right content—such as peer-to-peer videos that are transparent and compelling—we have helped rare disease companies to connect with the right patients on their terms and their turf. Knowing where to look and how to engage potential participants is a critical first step toward clinical trial success. Searching for your own needle in a haystack? We can help.