Capturing Real-world Experience with Pitolisant
If you intend to try pitolisant for your child, would you consider documenting your experience online with TREND Community?
TREND (https://trend.community) is an invitation-only network of consented patients and caregivers living with rare disease who are sharing stories & data using leading-edge, clinical-trial quality tools. Please email firstname.lastname@example.org to request an invitation.
The new 21st Century Cures Act has increased the Food and Drug Administration’s (FDA’s) receptivity to the use of real world evidence (http://blogs.fda.gov/fdavoice/index.php/2016/12/21st-century-cures-act-making-progress-on-shared-goals-for-patients/). The Commissioner of the FDA, Robert M. Califf, M.D. wrote in his blog that, “Cures provides support for continued exploration of the use of real world evidence in the regulatory context.”
TREND provides an opportunity to collect this data in a systematic and meaningful way. It is our hope that, using TREND, we can gather enough real world data to convince the FDA that pitolisant is safe and effective for the treatment of sleepiness associated with PWS. Such an indication may increase the likelihood that insurance companies will pay for pitolisant for our children.
To document an effect, we would need to collect at least one week’s worth of baseline data (but preferably much more) before you begin pitolisant. We will ask that you spend approximately five minutes a day recording your child’s body weight, morning body temperature, and dreams (dream journal). Each weekend we will ask for you to fill out a simple questionnaire that will include questions on sleep, hunger, and cognition. The questionnaire should take approximately 15 minutes. TREND will also provide an opportunity for you to journal/record your child’s challenges and any changes that you see (both positive and negative) with pitolisant. We will also ask you to upload as many supporting documents that you have on hand. These can include:
Sleep study results
Occupational therapy assessments
Blood work (specifically fasting lipids)