By Wayne Koberstein, executive editor
Rogerio Vivaldi’s experience with rare disease therapies teaches that drug development is never finished until simple and certain access for patients is ensured.
Rogerio Vivaldi may be the most unique pharmaceutical executive I have ever met. His background, history in the industry, and longtime mission all sound more like an adventure than a career, as if he slayed monsters and rescued fair humans to get where he is. And in a way, he did. If you see Gaucher Disease as monstrous and its sufferers as the humans in dire distress, Vivaldi will appear as the hero discovering his fate: delivering life-saving and life-redeeming therapies to those who need them.
Vivaldi’s saga began in Brazil, where he won universal access to Genzyme’s enzyme replacement therapy (ERT) Ceredase (imiglucerase). In doing so, his travels took him from the urban streets of Sao Paulo to the third-world regions of the Amazon. His odyssey progressed as he followed the same calling in many other countries, then went on to take command of a global business in treatments for Gaucher and other extremely rare diseases where patients previously had no hope.
Prior to his current position as CEO of Minerva Neurosciences, Vivaldi was the head of Genzyme’s Rare Diseases Unit, which is an integrated commercial organization, one of only two independent Genzyme businesses remaining after the Sanofi merger; the other concentrates on multiple sclerosis. (Vivaldi moved to his new company just as this article went to press.)
I come not to praise Vivaldi but to understand what makes him tick. Overall, Vivaldi’s story may enlarge the idea of precommercial product development to include a factor normally perceived as marketing: patient access. “There is no development without access,” he says succinctly. Identifying new patients, guiding doctors through diagnosis and treatment, and building a sustainable supply chain are all essential to development before the market and delivery to the market.
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.”