Company: Sigma-Tau Pharmaceuticals
Liaison(s): Warren Holmes, Rebecca Moore, Brian Schreiber
Sigma-Tau Pharmaceuticals, Inc. (Sigma-Tau) is a research-based, privately held pharmaceutical company focused on the manufacturing and commercialization of novel medicines for patients with rare diseases. Sigma-Tau Pharmaceuticals was founded in 1957 by an entrepreneurial research chemist Claudio Cavazza, PhD. Intent on becoming an international player, Sigma-Tau began its US operations in 1980 by setting up a subsidiary located in Gaithersburg, Maryland. In 1984, Sigma-Tau became the fourth company in the world to obtain an Orphan Drug Designation in the US, signaling the company’s arrival as a major player in the rare disease market. Sigma-Tau is in the midst of Phase I development of a prophylactic for a rare disease called Necrotizing Enterocolitis (NEC). NEC is a devastating gastrointestinal disease which predominately affects prematurely born newborns. In order to demonstrate the value of the company’s therapeutic to the healthcare community, Sigma-Tau would like to investigate the entire lifetime costs associated with this rare disease. In undertaking this analysis, Sigma-Tau hopes to contribute to a more robust economic climate in which to introduce its NEC preventative therapy. The Sigma-Tau TMP is aimed at developing a unique pharmacoeconomic cost analysis to assess the lifetime economic burden of NEC including direct, indirect and intangible costs associated with this disease and its complications. Given this goal, a “cost of illness” study was identified as an appropriate pharmacoeconomic model to guide the efforts of the project. The team gained an understanding of the clinical process influencing the direct costs of NEC and a subsequent direct cost model was constructed. The team also developed a framework for possible indirect and intangible costs associated with the long-term complications of NEC. To establish the indirect and intangible cost inputs, the team utilized an interactive marketing approach to target individuals affected by NEC, including survivors, caregivers and the families of patients. Interviews were conducted to develop case studies, through which the indirect cost model was created. Through the integration of all direct, indirect and intangible costs, the team has quantified the economic impact of NEC and its complications. These results provide Sigma-Tau and their key stakeholder’s with valuable insights into the costs of this disease and the overwhelming need for a cure.