Tagged with Rare Diseases

Hong Kong K P Tsang, president of the Hong Kong Alliance for Rare Diseases (HKARD), shares the challenges faced by patients with rare diseases and their caregivers in Hong Kong, the areas of improvement when it comes to rare disease policy, the advocacy work that HKARD is undertaking, and his insights on…

UK Three years into his role as general manager of Sobi’s UK and Republic of Ireland operations, Neil Dugdale introduces the scope of operations of the Swedish rare disease player to our audience, highlighting the company’s unique positioning in working closely with patient groups to drive forward better understanding of unmet…

Spain Alba Ancochea of FEDER, the association that groups together Spain’s 337 rare disease patient organizations, explains how it interacts with both government and industry to the benefit of rare disease patients, the evolving situation for these patients in Spain, and her strategy for the future. Could you please start by…

Portugal Alnylam, pioneers in the RNA interference field, seek to bring their revolutionary medicine across Europe, and in particular Portugal before 2020. In this interview the new General Manager for Spain and Portugal, Alicia Folgueira Lopez, a rare diseases authority, discusses how to build an affiliate from scratch, the synergies across…

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…