Tagged with Rare Diseases

Spain Jose Manuel Rigueiro, general manager of the Spanish affiliate of Actelion, discusses the lack of trust between health authorities and the industry and the challenges of launching new orphan drugs in such a fragmented healthcare system. He highlights the successful launch of Uptravi® and his commitment to avoid business disruption…

Portugal Carla Benedito, general manager of Shire Portugal, provides useful insight into the rare diseases landscape in Portugal. Carla discusses market access models for innovative and rare treatments, encouraging dialogue between industry and government, and the touching nature of working for a pharmaceutical company that knows all of its patients by…

UK With an appetite to take the next step in its rapid development, EUSA Pharma has already established itself as a leading rare-disease and oncology biotech company in just a short period of time. Currently active in both the UK and Europe, EUSA is set to further expand internationally, notably in…

India Swati Jajodia, managing director of Swati Spentose discusses her commitment to tackling underserved diseases and the company’s approach to bringing pentosan polysulfate to global markets while working with Key Opinion Leaders and CMEs in raising awareness about interstitial cystitis (IC).    Could you please introduce Swati Spentose to our international…

Switzerland Jürgen Pohle provides both a fascinating and open account of the latest movements of Neovii; a company that possesses the ability to make decisions at the speed of a startup but also has a heritage dating back to the 1970s. After an outstanding career spanning 25 years with Big Pharma…

Canada Marc LePage, president and CEO of Genome Canada describes the early days of the organization and talks modern day plans, such as personalized diagnostics for rare diseases, international alliances and genomic data collection and sharing.   Marc, you helped pioneer the creation of Genome in 2000 and led Génome Québec…

Poland Mirosław Zieliński, president of the Polish National Forum for the Therapy of rare diseases (ORPHAN), the representative voice for 35 Polish rare disease associations, discusses the implications of the introduction of a national rare disease plan and the need to improve rare disease diagnostics. Furthermore, he highlights the importance of…

Japan Makoto Suematsu, president of the Japan Agency for Medical Research and Development (AMED), discusses AMED’s successful unification of Japan’s previously disparate systems for medical research funding; the strategic significance of the country’s strong footprint in regenerative medicine and rare diseases; and the organization’s internationalization strategy, propelled by the push to…

Austria Rudolf Widmann, CEO and founder of AOP Orphan, the first rare disease-specific European company, discusses the overriding goal to ensure rare disease patients are matched with the correct treatments and the importance of open dialogue with specialists and patient groups. Furthermore, he highlights the need to differentiate health technology assessments…

Austria Dr. Wolfgang Schnitzel, general manager of Shire Austria, and Karl-Heinz Hofbauer, site lead Vienna and managing director for the Technical Operations business, discuss the huge impact of Shire’s 32 billion USD acquisition of Baxalta that has grown the workforce from a few Shire employees in Austria to 3,500. Furthermore, they…

Colombia Gianfranco Biliotti, recently appointed General Manager for Alexion Colombia, shares his insights on the particularities of operating in the ultra-rare disease segment, while highlighting the importance of managerial flexibility, the company’s unmatched patient-centricity and close ties with doctors, the role of education in improving patients’ lives and his strategic priorities…

Ireland James Lennertz, Group VP and GM EUMEA for BioMarin, discusses the key highlights of the past few years for BioMarin, their unique positioning as a company offering innovative therapies for ultra-rare diseases, the main challenge he sees for BioMarin’s international operations, and his priorities for the next few years. James,…