Tagged with Rare Diseases

Greece IFET is a unique institution within Greece that acts as the country’s mechanism for securing access to rare disease therapies not available in the market. The organisation also works to combat drug shortages in the country and played a key role in Greece’s COVID-19 pandemic response as the contracting authority…

Germany A roundup of some of the biggest recent stories from German pharma, including the latest on beleaguered Bayer CEO Werner Baumann, Merck KGaA’s latest investments in France and Germany, and STADA securing the first authorized treatment in Europe for the rare kidney disease immunoglobulin A nephropathy (IgAN).   Bayer’s hunt…

Americas Boehringer Ingelheim’s managing director for South America, Dirk van Niekerk, provides an overview of the healthcare climate in South America, the challenges tied to political and economic instability, future opportunities in specialty care, and the company’s strategy to continue a decades-long legacy. With many new governments in the region, we…

Italy Dompé’s Chief Operating Officer, Eriona Gjinukaj, responsible for creating the Italian mid-cap’s US affiliate, reveals the challenges and rewards of that experience and explains how she saw the company go from a primary care-focused Italian company to an internatonal biotech with an EMA and FDA-approved breakthrough therapy. I was lucky…

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…

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 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…

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…