Tagged with Rare Diseases

Switzerland Pierrre Morneau of Takeda Switzerland explains the therapeutic areas at the core of the company, equitable access, the Swiss framework for rare diseases, and the major challenges he is dealing with today.   The environment is shifting at a more rapid speed than ever before. Past paradigms are obsolete Having…

USA Zogenix’s Dr Stephen Farr shares his remarkable career journey from academia to industry, the company’s transition from pain therapeutics into rare diseases, product launch strategies, access and affordability issues, and talent attraction challenges.   While it is true you can make great contributions in academia, I think you can make…

USA Takeda USA’s Ramona Sequeira outlines her expanded role as president of global portfolio commercialization, the crucial importance of investing in culture, and the company’s multifaceted response to the COVID-19 crisis.   In general, through the global COVID response, we’ve seen the walls between companies and across sectors come down as…

USA Brad Campbell, president and COO of US rare disease specialist Amicus, talks domestic and global product launches, the company’s first steps into the gene therapy space, and how a biotech success story like Amicus can be a guiding light in market access and pricing discussions.   Ultimately, we envision our…

Switzerland Biogen Switzerland’s Dr Katharina Gasser outlines how the company’s 100 percent commitment to neuroscience helps it to stand apart, why Switzerland will continue to play a vital role in Biogen’s global clinical research footprint, and some of the country’s market access and pricing challenges. Dr Gasser also highlights Biogen’s burgeoning…

USA Ahead of the 2020 US National Organization for Rare Disorders (NORD)’s Rare Diseases and Orphan Products Breakthrough Summit, NORD president and CEO Peter L. Saltonstall outlines his hopes for the Summit, the impact of COVID-19 on the US rare disease community, and touches on the current reality and potential problems…

USA Certara’s Ulrich Neumann looks at the challenges that payment models for cell and gene therapy (CGT) have faced, the multiple models now under development, and why an open and collaborative approach among US healthcare stakeholders is needed if the significant legal and practical barriers are to be overcome.   The…

USA Gary Zieziula, president & region head North America for Kyowa Kirin, a specialty care-focused Japan-based global pharma company, highlights the tremendous growth the affiliate has seen over the past three years on track to become a USD 500 million+ organization within the next year; the exciting products and assets that…

Belgium Kevin Houdenaert, general manager for Benelux and the Nordics at Italian rare disease, specialty and primary care specialist Recordati, gives an overview of the market access specificities of the markets he manages, the autonomy that Recordati country managers enjoy, and his main priorities for 2020.   I see a lot…

USA William H Lewis, chairman and CEO of serious and rare disease specialist Insmed, talks through the impact of recent product approvals in the US, preparations underway for international launches, the impact of COVID-19 on Insmed’s operations, and the firm’s highly promising clinical pipeline.   The most important element of any…

Global Speaking exclusively to PharmaBoardroom, Sanofi Genzyme Executive VP Bill Sibold outlines Sanofi’s strategy for its specialty care unit, the wealth of potential breakthrough assets in its pipeline, product launch strategies, and the post-COVID industry of tomorrow.   Since I joined in 2011, it feels as if my time with Sanofi…

USA French firm LFB is targeting the lucrative US market with its hemophilia products and recently received two FDA approvals. Although launching new products in the midst of a global pandemic is not without challenges, LFB USA CEO Jose Antonio Moreno Toscano is optimistic that the launches will be a success…