Defining a strategy to increase disease awareness and diagnosis amongst important stakeholders involved in ATTP

Abstract

Within the specialty care unit, Sanofi has a franchise that focusses on rare blood disorders. Their latest drug, caplacizumab, Cablivi®, launched on September 1st, 2019. This is the first available nanobody® that is indicated specifically for adults experiencing an episode of aTTP, which is a rare and life-threatening blood disorder. First symptoms are often very non-specific which makes it more difficult to diagnose this disorder. This makes that many different specialists and stakeholders are involved in the patient journey, especially in the phase between the start of symptoms and the diagnosis. Moreover, the management of aTTP in Belgium is decentralized, meaning that basically any hospital with the capability of performing plasma exchange (PEX) can manage these patients. All these factors described above often make it very challenging to diagnose these patients early in the disease and start appropriate treatment. As this disease can become life-threatening very fast, an early start of treatment is crucial to improve patients’ outcomes. The research question, based on the problem statement, is formulated as follows: "Defining a strategy to increase disease awareness and diagnosis amongst important stakeholders involved in aTTP management in Belgium."