Telling the Rare Disease Story…

Rare diseases (also known as orphan diseases) are known to affect approximately 350 million people worldwide (Orphanet Journal of Rare Diseases, 2017). Such diseases may be considered uncommon, but for individuals experiencing and coping with these conditions, the general burden of illness and the day to day challenges are far from rare - impacting individuals and their quality of life at many levels. 

Today being Rare Disease Day, we at Self Care Catalysts recognize our patient advocacy and community partners who work tirelessly to build a network of support for patients, family and friends connecting and engaging them about not only the issues but also the avenues of support that build awareness about rare diseases. 

Previous and current partnerships have allowed us to harness our research methods utilizing our integrated system of patient support solutions, intelligence and behavior analytics. 

Project Zebra is one such campaign that engaged patients of the Carcinoid and Neuroendocrine Tumors (NETs) rare disease community (Carcinoid Cancer Foundation). Over a two-month period, patients were recruited and encouraged to capture data such as their daily moods, symptom severity, as well as regular journaling, utilizing the Carcinoid NETs Health Storylines mobile self-care management platform. 

Our unique approach of patient acquisition and engagement ensured ongoing adoption of a high tech, high touch approach which reflected the real life day-to-day carcinoid or NET experiences of patients over time. 

As a result of the study, the tracking of real life experiences allowed patients to gain a clearer understanding of their condition. The increased knowledge, awareness and attitudes towards Carcinoid and NETs patients enabled them to embrace new ways of managing their day to day symptoms, presenting new perspectives, better coping mechanisms and added support concerning health outcomes. 

Some insights from the study revealed: 

* 33% of patients with Carcinoid or NETs report between 4-9 symptoms
* 44% of patients reported diarrhea as one of the top symptom reported followed by fatigue and nausea/vomiting at 23% and 22% respectively.

Through our real world, real-time patient behavior intelligence and insights platform aggregated and anonymized data provided deeper patient insights and an outlet to share this rare disease experience. It is becoming more and more evident that medical research and stakeholders such as health care providers and payers seek efficiencies that can positively impact health outcomes including those of rare diseases. 

Advances in Real World Evidence, related to the burden of rare diseases are important in validating the experience of the rare disease community - especially since their daily challenges might often go unnoticed and misunderstood. 

On this Rare Disease Day, we recognize the efforts being made by many of our partners and empathize with patients as we continue to support not only on Rare Disease Day, but everyday to gain a deeper understanding and build awareness of conditions and day to day experiences which result in improved healthcare outcomes.

NewsLinda ObengComment