Tagged with Rare Diseases

USA Monica Weldon draws on her own experience as a rare disease patient advocacy group CEO to outline how such groups can work collaboratively with other stakeholders in drug development to better target funding and create a more patient-centric drug development process that ultimately gets better drugs to rare disease patients…

Spain Kyowa Kirin’s head of the South Europe cluster, Norberto Villarrasa, explains the Japanese company’s presence in Spain, its focus on rare diseases and onco-haematology, and comments on the challenges around access to rare disease treatments. In addition, the executive provides the company’s view on the potential and advantages of risk-sharing…

Europe In Europe, patient access to Orphan Medicinal Products (OMP) for the treatment of rare diseases remains inequitable. With the common goal of ensuring broader and faster access to OMPs across Europe, EURORDIS and EFPIA have joined forces to propose a series of solutions. According to a 2020 study, fragmentation of…

Italy Speaking exclusively to PharmaBoardroom, Pierluigi Antonelli outlines Angelini Pharma’s progress in transforming from a traditional, Italy-focused, and family-owned firm into a true European innovator. Antonelli outlines the potential impact of Angelini’s USD 960 million Arvelle acquisition, why he is optimistic about success in the challenging brain health space, and how…

Pharma Legal Handbook The key facts about orphan drugs & rare diseases in Brazil. Prepared in association with Trench, Rossi e Watanabe one of Brazil’s most prestigious law firms, this is an extract from The Pharma Legal Handbook: Brazil, which can be purchased for GBP 119, here.   1. What is the definition of Rare Diseases…

Italy Claudia Coscia outlines how the geographic and portfolio transformation of Kyowa Kirin has played out in Italy in recent years. Coscia also touches on access challenges for rare disease therapies in the Italian pharma market, rising levels of rare disease awareness, and the digitalisation status of the country’s healthcare system. …

Spain Sobi’s VP and general manager for Spain and Portugal, Pablo De Mora, comments on his experience during the pandemic, the Swedish company’s haematology and immunology portfolio, including its EMA-approved COVID-19 treatment, and the reimbursement challenges faced by rare disease players in Spain. Moreover, De Mora highlights Sobi’s vast clinical trials…

Spain The Spanish Association of Orphan and Ultra Orphan Medicinal Products Laboratories (AELMHU) is a non-profit organisation working to improve awareness about rare diseases and access of Orphan Drugs. Maria Jose Sanchez Losada, president of the association, comments on the current status of diagnosis, access and reimbursement of rare diseases and…

Saudi Arabia The general manager for Recordati Rare Diseases in MENA, Tony Zbeidy, explains why awareness of rare disorders has vastly improved in the region, which has one of the largest incidence rates in the world, the particular importance of the Saudi Arabian market to Recordati, the need for a more collaborative…

Saudi Arabia Jean Paul Scheuer, General Manager for Sanofi Genzyme and MCO Lead for Greater Gulf, highlights the company’s expanding footprint in the region, the organizational changes brought about by global CEO Paul Hudson, and how Sanofi has managed to launch 20 new products in the last year. Sanofi has been quick…

Saudi Arabia Myriam Hakim outlines the progress that Japanese rare disease and oncology specialist Kyowa Kirin has made in the GCC region in recent years; the importance of the Saudi market to Kyowa Kirin and its customised strategy there; as well as the main game-changers for patients in the region, namely the…

Saudi Arabia Few stakeholders are better placed to assess the landscape for rare disease therapies across the Middle East and North Africa (MENA) region than Karim Smaira and Kamel Ghammachi of Genpharm, an entrepreneurial success story that now provides market access and marketing solutions for rare disease products across the region.  …