Tagged with Rare Diseases

Belgium RaDiOrg aims to support rare disease patients and patient organisations in Belgium, defend their interests, and raise awareness of what can be devastating and little-understood conditions. Director Eva Schoeters – who joined the patient advocacy field following the diagnosis of her son with a rare condition – outlines some of…

UAE Following the diagnosis of her daughter with a rare and debilitating condition eight years ago, Youmna Ouraybi Ghaziri has dedicated herself to advocating on behalf of her and other rare disease patients. Two years ago, she founded Ana Fareed Health Consultancy; a patient advocacy group that gives voice to rare…

Rare Diseases With February’s Rare Disease Day behind us, it can be easy to forget the impact of these diseases on individuals, their families and care givers, and our communities throughout the other 11 months of the year. But if we – in healthcare, in government and in academia – are to…

Greece Takeda’s Cornelia Zanetti, a recent implant into Greece following stints in Germany, Switzerland, and Singapore, shares her first impressions of the Greek pharma market, and how the Japanese giant is making a difference for Greek patients in oncology, gastroenterology, plasma-derived therapies, and rare diseases. Zanetti also highlights some of the…

Americas Maria Gabriela Pittis, Head of SAM (South Cone, Andean Region, Mexico & Central America and Caribbean) at Takeda, outlines the company’s significant presence in the cluster, its double digit growth and plans to launch 14 new products there by 2025, most importantly its European Medicines Agency-approved dengue vaccine. She also…

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…