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Complex Issues of Self-management in Rare Disease

Friday, 10 June 2016

Rare Disease InfographicAuckland/London/Cranford, NJ – Treatment nonadherence doesn’t discriminate by illness, and rare disease presents additional challenges to patients’ ability to effectively self-manage.  Atlantis Healthcare, global leaders in treatment adherence and self-management programs, joined stakeholders in pharma, biotech, patient advocacy and academia at the World Orphan Drug Congress (WODC) recently to discuss best practices in supporting individuals with rare diseases.

“The management of rare disease presents unique challenges for the healthcare community, patients and their families,” said Kate Perry, MSc, PsychD, MNZPB, Director, Behavioral Science at Atlantis Healthcare, a sponsor of the WODC. “Unlike more common chronic diseases, physicians may have limited experience in a particular rare disease, and it isn’t always possible to turn to the literature or textbook for guidance. Very often, the patient is the primary source of communicating the physical and emotional aspects of the condition.  It is crucial, therefore, that effective self-management interventions acknowledge their voice.”

At WODC, Dr. Kate Perry led a 4-hour Workshop entitled, “Addressing the Why Behind Nonadherence in Rare Disease.” She also moderated a panel discussion on “Success Measures in the Launch and Lifecycle of Orphan Drugs.”

Rare diseases are serious, chronic conditions that generally start in early childhood, are life-limiting and poorly understood.  There are currently over 6,000 recognized rare diseases, including cystic fibrosis and Huntington's disease. According to Dr. Perry, treatments exist for only approximately 5% of rare diseases, and are designed to manage symptoms or slow down the progression of the disease, not to cure the condition.

“Health psychology frameworks provide us with tools to understand the unique characteristics of rare disease that impact treatment adherence,” said Dr. Perry.  “For Gaucher’s disease, we need to acknowledge that often people living with this rare disease don’t even feel unwell. If you aren’t feeling unwell it can be hard to understand the necessity for treatment or other self-management tasks.  For conditions like Hypophosphatasia, there are incredibly complicated dosing regimens that make adherence practically challenging.  And, because most rare diseases start in childhood, almost all individuals experience issues during the transition from adolescent to adult, as responsibility shifts from parent to patient.”

Challenges inherent in rare disease also offer opportunity for pharma, said Dr. Perry.  At the WODC, there was an emergence of pharma/biotech partnerships that leverage digital technology to transcend geographic borders to create patient communities and build digital registers.


About Atlantis Healthcare Group

Atlantis Healthcare leverages health psychology to develop and deliver uniquely personalized healthcare solutions that drive sustained improvement in patient outcome through self-management and treatment adherence across chronic diseases.

With the largest team of globally recognized health psychology experts in a commercial setting, supported by digital creative experts, Atlantis Healthcare uses clinically proven models and academic research to develop award-winning behavior change programs, helping patients achieve better health outcomes. The group provides solutions and services across the globe from offices in UK, Germany, US, Australia and New Zealand.


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