Tagged with Rare Disease

USA Peter L Saltonstall, president and CEO of the US National Organization for Rare Disorders (NORD) shares the organization’s key priorities, what makes NORD a unique patient advocacy group in the US, prevailing access and affordability issues for American rare disease patients, and how the country should continue to incentivize a…

Belgium Geert Van Hoof, founding general manager of Chiesi Belgium, shares the exciting milestones of the company’s first decade in Belgium, including their stunning year-on-year double-digit growth, their market leadership in respiratory as well as neonatology, and their growing presence in rare diseases, as well as the attractiveness of the Chiesi…

Czech Republic A cross-industry push for better and broader access to innovative treatments is underway in the Czech Republic. Ripe for reform is the country’s orphan drug legislation as no standard pathway for their assessment, pricing and reimbursement currently exists.   [For cancer treatment the country has been] quite successful in moving…

Mexico Chiesi Mexico’s Marco Ruggiero discusses his initial impressions of the Mexican pharma market, his strategic priorities for the affiliate, and the increasing importance of the company’s respiratory portfolio in Mexico.   With respiratory diseases, there is sometimes the erroneous perception that they are not as severe or urgent as other…

Mexico David López García, Mexico country manager of global rare genetic therapy player Biomarin, outlines the challenging situation around patient access to orphan drugs in Mexico and the importance of building awareness of rare diseases.   The challenge for orphan drugs is the price. It is not always easy to explain…

Italy Italian mid-cap firm Chiesi is continuing its expansion in the USA market with the launch of a Boston-based subsidiary focusing on rare and ultra-rare diseases.   We are very excited to put Chiesi’s decades of experience in drug development and dedication to patients to work to make a positive difference…

Czech Republic Patrik Zachar, Vice President Central Europe of Ipsen, shares how the company has built a strong presence in oncology and neuroscience in the CE region and the Czech Republic, quickly establishing market-leading positions in the indications they compete in. Whilst in neuroscience, Zachar still sees significant growth potential for its…

UAE Genpharm is a Dubai-based regional company providing market access and marketing solutions for rare disease products in the MENA region. Co-Founders Karim Smaira and Kamel Ghammachi provide insights into the rare disease space in the region, explain how the company is positioning itself in the gene therapy arena and why…

France These are worrying times for iconic French mid-cap Ipsen. Trials for palovarotene, a treatment for rare bone disorders, have been plagued by setbacks, perhaps contributing to erstwhile CEO David Meek’s decision to quit the firm in December 2019. The company’s share price has since plummeted.   Meek’s resignation to join…

Hungary Tamás Demeter, Country Manager for AOP Orphan shares his insights on the footprint of the company in Hungary, the areas of therapeutic need that AOP caters to, the performance of 2019 and its key achievements, the ecosystem of rare diseases in Hungary, and patient awareness while highlighting the role of…

UAE Myriam Hakim, the recently appointed GM for Japanese specialty pharma firm Kyowa Kirin’s UAE affiliate, discusses the strategic significance of the UAE and the Gulf region as a whole to the global group.   I appreciate all the efforts the [UAE] authorities have been putting into fast-tracking registration for innovative…

Canada Pamela Fralick of Innovative Medicines Canada (IMC) advocates for a collaborative multi-stakeholder approach to better tackle rare diseases in Canada.   Especially in smaller provinces, it’s virtually impossible to predictably manage the affordability and accessibility of drugs necessary to treat rare disease Around the world, rare diseases are becoming easier…