Apr 25 -27, 2018
Board member Jennifer Silvia and president Peter Mansbach represented Circadian Sleep Disorders Network at the World Orphan Drug Conference at National Harbor, Washington, DC, on April 25 - 27. "Orphan Drugs" are drugs for rare diseases, which therefore are unlikely to have blockbuster sales and often go looking for a company to sponsor them: hence the "orphan" designation. As the conference title indicates, there were a lot of pharmaceutical companies represented. There is a lot of attention now on soliciting patient involvement in drug development, and we and other patient advocacy groups had been invited to attend.
The most interesting sessions for me (Peter) were the four roundtable discussions I attended. In each one I was able to raise awareness a little by presenting information on our disorders tailored to each particular topic. Being a rare disease conference, our concerns were well received. For example, one discussion was Best Practices and Tips...to Tell a Rare Disease Story, and I emphasized the difficulty we have in getting an audience even to believe that we really can't fall asleep at a desired time no matter how tired we feel. For the Integrating the Patient Perspective I brought up how treatments have been based on lab measurements of melatonin onset, but we were more concerned with how tired we feel and how well we can function.
I attended a number of talks of interest. One was about patient registries for drug development. Among other things, these are useful for indicating the variability in people's experience with a particular disorder, and learning what endpoints (treatment goals) are important to the patients. [Aside: if you haven't yet, please join our registry and take the survey, to help jump-start research on Circadian Rhythm Sleep Disorders.]
One participant at the roundtable on real-world data used the term "free-range humans" to describe participants in a patient registry, because their answers may not hew to rigorous definitions of diseases or symptoms. I think I persuaded him of the value of registries like ours, to collect real-world patient data and to stimulate further research. I had a lot of support on this point from other participants.
I was again aware of how many of the activists in other patient advocacy groups are the parents of patients, since the patients themselves often don't have enough energy to pursue awareness and advocacy in addition to just surviving. With circadian disorders, we still often have difficulty persuading our parents that our disorders are real and serious, so we haven't seen enough such support.
Possibly the most useful interaction for me at the conference was a long discussion I had with a woman who works for Montgomery County, MD (where I live) helping small businesses. We discussed some of the issues that Circadian Sleep Disorders Network faces. She said she will refer me to a non-profit organization that helps other non-profits start and grow. This will be a long-term project.
Our registry and survey is hosted by a health company that assures its privacy and security, and guarantees that it meets HIPAA (Health Information Portability and Accountability Act [U.S.]) requirements. This company was originally called PatientCrossroads, then changed its name to AltaVoice. Last year AltaVoice was bought by Invitae. Now they have switched the branding of the registry and survey to Invitae.
Your old links should continue to work. Our own website links have been redirected to the new site at invitae.com. Any previous survey answers have been carried over to the new site. Your login should continue to work. Let us know (at ) if you encounter any problems.
And if you haven't yet: please Take the Survey!
The (US) National Institutes of Health (NIH) is launching an unprecedented study, called All of Us, that will attempt to enroll a whopping one million participants from across the country. The goals:
The NIH wants input from all of us: which research questions should this vast study help answer? This is where we owls come in! We want more research on what makes our body clocks tick to the beat of a different drummer—and how to reset our clocks. We can also study sleep hours and habits, both for connections with other disorders and illnesses, and to attempt to get a handle on the incidence of CRDs.
Submit your research idea/s here: https://allofusresearchpriorities.ideascale.com/?new-idea=1
More info here:
CSD-N's Board of Directors held elections for officers to serve for the next two years (through Jan 2020). The previous officers all agreed to run again, and no new people were nominated. The officers are: