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Exclusive Life Science Feature CASE BY CASE, TO EVERY CASE Vivaldi was instrumental in winning early access to Ceredase in Brazil and ultimately in many other countries, driven personally by experience with his first Gaucher patient in 1992. An M.D., he specialized in diabetes care, and a friend in academic research asked him to take care of a patient who was just beginning ERT. "That was a turning point — for the patient and for me," he says. "The patient had his life transformed, but my life was transformed at the same time because I saw how the treatment was changing the natural history of that disease. It was a dramatic effect rarely seen before in any therapy, for any disease." Once Vivaldi learned about Ceredase, which had just gained approval in the United States the previous year, he wanted to make it available to as many patients as possible. But his initial hurdle was paying for the drug. The therapy then cost about $200,000 per year, and it had to be administered every other week for life. "Soon I probably had more Gaucher patients than the majority of documented cases in the world at that time, and several years later, Genzyme asked me to help the company make Ceredase available to more patients because I did have the first ones, but they were paying for the therapy out-of-pocket." In Brazil, the payment challenge, though still daunting, was simplified by the universal healthcare mandate of its national health system. Vivaldi went straight to the top, appealing directly to the Minister of Health, who subsequently agreed to cover Ceredase and later listed it on the national formulary — the first and only time Brazil, or likely any country, paid for a product that had not yet received market authorization from the regulatory authority. A key point in Vivaldi's argument for national government reimbursement was the need for pan-Brazilian access to Gaucher treatment: "I told the Minister that with a rare disease, a state-bystate approach would never work because you would have people migrating to different states just to stay alive." Vivaldi also persuaded the Minister to make the government coverage permanent and build the infrastructure necessary to maintain steady delivery of the medicine and supportive care for all Gaucher patients anywhere in Brazil. Since then, he and the company applied the same logic globally, winning reimbursement and logistical support wherever possible, so that any Gaucher patient in any location can be treated. Vivaldi visited every hospital in Brazil, asking three questions: "Do you know what Gaucher disease is? Do you know Gaucher disease has a treatment? And do you know that the treatment is available Achieve better models, candidates & markers with metabolomics. Innovative diagnostics and research solutions for discovery, clinic and bioprocess. Learn more at metabolon.com. January 2014 LifeScienceLeader.com 35