June 7, 2013
by Mark Dunning
Clinical trials and studies on retinitis pigmentosa usually exclude people with Usher syndrome, at least in the early stages. The reason given is often that people with Usher syndrome not only have RP, but they also have hearing loss. What is left unspoken is that the hearing loss is believed to make communication with people with Usher syndrome more difficult. So people with Usher are excluded.
I am not going to give specific examples of trials and studies for retinitis pigmentosa that have excluded Usher. First, I don’t want to call out any particular researcher or study. But more importantly, I understand that screening candidates for clinical trials is an extremely difficult process, especially in early phases of trials. Candidates need to meet certain age, disease progression, clinical, and proximity requirements. You could be the perfect candidate in every way but live too far away to visit the clinic with the optimal frequency and be excluded.
These strict selection criteria are important to accurate and successful trials. They ensure that the data gathered is consistent and without bias across all subjects. So it makes sense to include only people with the same genetic form of RP in early trials. When a treatment appears to work for subject A but not for subject B, researchers want to figure out what was different between them. It is easier to determine the cause and effect when every subject in the pool is as similar as possible.
So the argument here is not that people with Usher syndrome should be included in every study and trial for RP treatments. No, the argument is that people with Usher syndrome should be considered without bias toward their hearing loss.
Look, if hearing loss as a clinical condition is something that would impact the study, then that makes sense. As a researcher, you don’t want to have to account for that data point. But the sad fact is that people with Usher are excluded from many of these trials because of misconceptions about hearing loss.
It is believed in some quarters, for instance, that communication with people that use ASL is difficult, especially when complex scientific subject matter is involved. Though this can be addressed with interpreters, the added cost of sign language interpreters may be considered to have a significant impact on a trial’s budget. So researchers find it easier to exclude people who use ASL, both because it will make communication easier but also because they don’t have to seek more funding or allocate resources differently.
Now bear with me on this because the whole idea that people using ASL are difficult to work with or somehow are unable to understand complex subject matter makes my blood boil. But let’s assume for a moment that this is actually the case. Well, there are still more than enough people with Usher syndrome that use oral communication to fill out a trial. Digital hearing aids and cochlear implants are widely used these days. Aside from some issues with background noise and communication, there is very little difference between oral deaf and people with hearing these days when it comes to basic communication. Any issues with comprehension can be easily addressed with some simple staff training that would add very little to the overall cost of a trial.
Yet hearing loss is still a barrier to inclusion in clinical trials. What we’re left to conclude is that there is an unfair bias toward people with Usher syndrome either because of an outdated understanding of the nature of hearing loss and communication or because of the type of budgetary decision making that would set off sirens at the ADA. Either way, this is wrong.
It has been twenty five years since the Deaf President Now student protests at Gallaudet. As I. King Jordan, the President of Gallaudet, said after those protests: "Deaf people can do anything hearing people can do, except hear." That includes participate in clinical trials.
This is no small thing. Studies that exclude people with Usher limit our understanding of Usher vis a vis other RP diseases. That only furthers the likelihood that other RP diseases will be included in trials while people with Usher are excluded because it is easier to work with diseases that are well documented. Every time one disease is studied and Usher is excluded, Usher research falls behind.
There is the very real potential that treatments will be approved for certain types of RP but not for people with Usher because people with Usher were not part of the trial. Even if the delay between acceptance of a treatment for people with some types of RP and acceptance of a treatment for people with Usher is a couple of years, that can have a significant impact. This is a degenerative disease. A couple of years is a big deal to people with Usher.
The real impact is that these biases delay research, and ultimately treatments, that could help people with Usher syndrome. That’s a high price to pay for ignorance about hearing loss. I hope my researcher friends consider this and listen when I steal from I. King Jordan:
“People with Usher syndrome can do anything hearing people can do, except hear.”