Thursday, January 12, 2017

Interviews about pediatric treatment goals

The medical community is listening to patients more and more, and now the XLH community is in particular demand for sharing our thoughts and experiences as the time approaches for Ultragenyx Pharmaceutical to apply for early approval of KRN23 for pediatric use in 2017. Ultragenyx is looking to talk to a few pediatric patients and their parents to better understand whether their original goals for the clinical trials were correctly targeted and to get better insight into what children and caregivers think are the most significant ways in which better treatment might be meaningful. 

If you're interested in participating, please contact Adelphi Values, the research company contracted by Ultragenyx to do the interviews. You can reach Holly Spencer at or 617-720-0001. Participants will be compensated for time and/or travel. More details are in the official flyer from Ultragenyx below.

Some members of the community have already offered feedback in other settings, like the Burden of Illness survey and the recent joint-replacement survey. We (and the researchers) appreciate everyone's contribution to science, but there's so much more to be learned. Potential new medical therapies like KRN23 require significant clinical research to make sure they're safe and effective and they're meeting the needs of patients and their families. Clinical trials, however, are only part of how the research community determines how best to treat patients living with chronic illnesses. Another important method consists of interviewing patients (and caregivers). 

Eventually, the Network's Natural History Study  (launching in 2017) will collect answers to a broad range of questions about experiences with phosphate-wasting disorders, so future research won't have to start from scratch. That will take time, though, and some answers are needed about the patient experience in the very short term, while the application for KRN23 approval undergoes review by the FDA and comparable governmental agencies throughout the world. Much of this information is related to whether or not the original goals for clinical trials were correctly targeted, and the only way to determine that is to ask further questions of patients. 

As always, The XLH Network, Inc. does not endorse or critique specific clinical trials, and does not counsel individual patients either for or against participation in any specific clinical trial. Prospective volunteers should always carefully review the clinical trial's informed consent documentation, and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.

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