Tagged with Rare Diseases

Europe With Rare Disease Day falling on 28 February, PharmaBoardroom dedicates this month to exploring the global rare disease landscape, highlighting key trends and developments as well as profiling the most innovative global players in this space. We start the party off with five of the most promising clinical-stage biotech companies…

Switzerland A trained physician with several years of practice under her belt, Katharina Gasser then moved into clinical research as an investigator before joining the pharma industry in 2005. Now head of Biogen’s Swiss affiliate and chair of the executive committee of industry association Interpharma, Gasser cites her diverse background as…

Global The Top 10 rare disease companies globally, ranked by worldwide orphan drug sales for 2018. Celgene takes top spot, followed by Roche and Novartis. Made with Visme Infographic Maker

Global In a wide-ranging and exclusive interview with PharmaBoardroom, Ipsen CEO David Loew looks back on an atypical first 100 days at the helm of the iconic French mid-cap; its EUR 3 billion war chest for bolt-on acquisitions; planned expansions in rare- and ultra-rare diseases; and the niche oncology segments where…

USA Rare disease – defined in the US as a disease with a maximum patient population of 200,000 – is perhaps the most salient issue at the nexus of innovation, access, and affordability in the country.   As FDA CDER director Dr Janet Woodcock indicates, “rare diseases have been rising as…

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…