Tagged with Rare Disease

USA Founded to tackle one of the most persistent blind spots in rare disease policy, the Rare Access Action Project (RAAP) focuses on what happens after FDA approval, when therapies meet the realities of coverage, reimbursement and real-world access. Led by Executive and Founder Michael Eging, RAAP brings together patient organisations,…

Taiwan Taiwan, an early international market for Kyowa Kirin, now serves as a strategic hub within a Korea-Taiwan-Australia cluster. With a lean team, close government engagement, and clinical trial initiatives, Taiwan General Manager Tatsuru Iwamoto outlines his affiliate’s portfolio prioritisation, collaboration, and life-changing value delivery while leveraging Taiwan’s policy strengths and…

MEA As global pharmaceutical strategies continue to shift, Ali Sleiman reflects on how Genesys Pharmaceuticals is being built as a disciplined execution platform for the Middle East and Africa. The interview explores how distributor-led models, portfolio selectivity, and strong infrastructure shape access to innovation, including in rare diseases, against an increasingly…

USA At a moment when rare disease policy, diagnostics, and patient-driven research are converging, Danielle Carnival sets out a clear vision for the role of the Undiagnosed Diseases Network Foundation. Drawing on her experience across neuroscience, federal health policy, and patient-led organisations, she explains how UDNF operates upstream of diagnosis, addressing…

MEA Rare diseases in the Middle East and Africa are no longer an afterthought. As regulatory frameworks mature and governments demand deeper partnership, access, science and local commitment are being redefined. In this interview, Sherif Basha explains how Versalya Pharma is building a rare diseases platform designed for long-term impact, translating…

USA Adriana Herrera, CEO of Pierre Fabre Pharmaceuticals Inc, discusses Pierre Fabre’s dedicated expansion into the US market and the unique strategies guiding its next phase of growth. Drawing on more than two decades of leadership experience across global biopharma organizations, Herrera reflects on building a purpose-driven organization, establishing credibility beyond…

USA Dr Carlos Prada, Director of the Edwards Family Division of Genetics and Rare Diseases at Ann & Robert H. Lurie Children’s Hospital of Chicago, discusses how earlier diagnosis, coordinated care pathways, and emerging therapeutic modalities are reshaping outcomes for children and families. From newborn screening and multidisciplinary follow-up to the…

LatAm Fernando Almeida, EVP China & International Markets, discusses his global pharma career and Chiesi’s evolution into a high-performing, innovation-led group. He highlights strong double-digit growth, disciplined investment, and expansion across China and Latin America, underpinned by access-driven strategies, talent mobility, and long-term patient impact.   To become a strong and…

UAE Adeeb Al Attar leads Genpharm at a point where rare and advanced therapies in the Middle East face a more exacting test. Scale and ambition are no longer enough. What matters now is execution, credibility, and the ability to move innovation through complex healthcare systems without losing focus. In this…

USA Neil F. McFarlane, President and CEO of Zevra Therapeutics, reflects on how his experiences in clinical practice, military service, and leadership roles across biotech and mid-sized pharma come together and led him to this moment. Under McFarlane, Zevra is driven by the urgency of patient needs and engaging with patient…

France Kyowa Kirin’s new head for France and Benelux steps into the role at a time when rare diseases, regulatory expectations and scientific modalities are all shifting at speed. In this interview, Aurelie-Anne Chausse reflects on the recent strategic transition toward a fully rare-disease-focused organisation, the growing pressure to identify patients…

Taiwan With Takeda preparing for global leadership change and Taiwan rapidly upgrading its innovation and access frameworks, General Manager Alexis Lin is navigating one of the most pivotal periods in the affiliate’s 64-year history. Drawing on a career that began far from pharma, she has reshaped Takeda Taiwan into a regional…