Tagged with Rare Diseases

Saudi Arabia Kamel Ghammachi and Karim Smaira are the co-founders of Genpharm, an entrepreneurial success story that now provides market access and marketing solutions for rare disease products across the MENA region. They discuss the impact of COVID-19 on their business; the evolving dynamics of the Saudi pharma market; and the crucial…

Turkey Few Turkish companies are experiencing such a transformational moment as GEN. Just in 2021, the Ankara-based organization made its public debut at the Istanbul Stock Exchange and signed an exclusive collaboration agreement with a Dutch biotech company for the development and commercialization of a therapy against Alzheimer’s and other neurodegenerative…

Denmark Industry veteran Patrik Forsell oversees Takeda’s operations in Denmark. Here, Forsell talks through how sizeable shifts in the company’s global portfolio and focus over the past four years have played out at the Danish level, the increasingly challenging market access situation for rare disease treatments in the country, and how…

Saudi Arabia Rodrigo Rodriguez, general manager for Takeda’s Middle East cluster, discusses the company’s ambitions in the collective Saudi pharma market, which is expected to reach a size of USD 10 billion by 2025, the need for tailor-made programs for different countries in the Middle East, and how clinical trials and registry…

Global Bill Sibold, Executive Vice President and Head of Sanofi Genzyme, highlights the achievements of the company’s Rare Humanitarian Program over the last 30 years, bringing treatments to patients in developing countries with lysomal storage disorders who would otherwise not have access. Sibold outlines the persistent obstacles facing people with rare…

Turkey Takeda’s general manager for Turkey, Şeyda Atadan Memiş, reflects on the Japanese giant’s ambitions in Turkey at a pivotal moment when the Shire integration has been completed. Atadan discusses how the organization has seized on Takeda’s revamped portfolio, focusing on rare diseases, haematology, immunology, gastroenterology, and oncology, to move up…

Switzerland Biogen Switzerland’s Dr med Katharina Gasser outlines how her affiliate navigated the COVID-19 pandemic from an organisational and communication perspective, the increasing importance being given to mental health by the Swiss government, and the reimbursement and market challenges facing Biogen in 2021 and beyond.   2020 saw a lot of…

Canada Writing in the March edition of DIA’s Global Forum magazine, Judith Glennie examines the findings of Health Canada’s consultation with Canadians on what a national strategy for high-cost rare disease drugs could look like.   On January 27, 2021, Health Canada launched a consultation on the development of a National…

China Kevin Rufang Huang, president and founder of the Chinese Organization for Rare Disorders (CORD), outlines the findings of the first ever analysis into rare disease public insurance coverage across key cities in China.   On May 30, 2020, the China Organization for Rare Disorders (CORD) and the management consulting team…

Switzerland Dipharma SA is a Swiss specialty pharmaceutical company, developing improved generics medicines for rare metabolic diseases. Founder & CEO Marc-Olivier Geinoz discusses why the company has chosen to compete in the largely unexplored rare diseases generics space, its direct presence in the German market, and the opportunities inherent in metabolic…

USA With Rare Disease Day coming up on February 28th, one of the leading organizations dedicated to serving affected patients in the US, the National Organization for Rare Disorders (NORD), is making a push to put the spotlight on the limited access to diagnosis, treatment and care suffered by rare disease…

USA Following on from our profile on five of the most promising clinical-stage biotech companies developing orphan therapies in Europe, here we move to the world’s biggest biopharmaceutical market and R&D hub, shining the spotlight on five of the most important and innovative rare-disease start-ups in the USA.   The US…