Rare diseases collectively exert a global public health burden in the severity of their manifestations and the total number of people they afflict. For many patients, considerable barriers exist in terms of access to appropriate care, delayed diagnosis and limited or non-existing treatment options. Motivated by these challenges, the rare disease patient community has played a critical role, elevating the patient voice and mobilizing legislation to support the development of programs that address the needs of patients with rare diseases.The US Orphan Drug Act of 1983 served as a key milestone in this journey, providing a roadmap for other countries to introduce and implement similar orphan drug legislation; more recently, the European Union (EU) has gone further to encourage the widespread adoption and implementation of rare disease plans or strategies designed to more adequately address the comprehensive needs of patients with rare diseases. Despite these legislative efforts and the growing contributions of patient advocacy groups in moving forward implementation and adoption of rare disease programs, gaps still exist across the policy landscape for several countries. To gain deeper insights into the challenges and opportunities to address key needs of rare disease patients, it is critical to define the current status of rare disease legislation and policy across a geographically and economically diverse selection of countries. We analyzed the rare disease policy landscape across 11 countries: Germany, France, the United Kingdom, Canada, Bulgaria, Turkey, Argentina, Mexico, Brazil, China, and Taiwan. The status and implementation of policy was evaluated for each country in the context of key patient needs across 5 dimensions: improving coordination of care, diagnostic resources, access to treatments, patient awareness and support, and promoting innovative research. Our findings highlight the continuing role of the patient community in driving the establishment and adoption of legislation and programs to improve rare disease care. Further, we found that while national rare disease plans provide important guidance for improving care, implementation of plans is uneven across countries. More research is needed to demonstrate the effect of specific elements of rare disease plans on patient outcomes.

provide RWD on the use of BV in the treatment of HL in a private healthcare setting in Brazil. Methods: This retrospective analysis collected data from Evidencias-Kantar Health claims database (Auditron, which covers 3+ million lives, ~7% of Brazilian private sector) between March/2013-March/2016 on demographic, diseaserelated and treatment-related parameters regarding patients diagnosed with HL. Data were summarized using descriptive statistics. Results: We found 147 HL patients, being 51.7% female, with median age of 32 years (IQR:25-41). The number of patients that received each therapy line was: 147, 42, 29, 20, 9, 5, 3, 2 and 1 for first to ninth-line respectively. From the third-line forward, BV was present among the chosen therapies. Of the 15 patients treated with BV (1 patient received it in 3 lines), 60% were male, 43% on stage IV disease, had a median age of 30 years (IQR:28-39) and a median number of cycles of 3 (IQR:3-4). Distribution of patients (number of patients/total number of patients per line) that received BV in each line was: thirdline (1/20); fourth-line (7/20, with one patient receiving BV in combination with gemcitabine+liposomal doxorubicin); fifth-line (4/9); sixth-line (3/5); seventh-line (1/3); eighth-line (1/2). Overall, 10.2% of the patients with HL were treated with BV and only one received SCT (heterologous) in fourth-line. ConClusions: RWD from this cohort of HL from Auditron demonstrates 20% of patients with HL received at least 3 lines of treatment. The rate of patients treated with BV increased, as the lines of therapy advanced and 10.2% received the drug at some point in treatment.

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