Tagged with Orphan Drugs

Global The rise of niche therapies is shaping a more tailored engagement model, driven by data and AI, writes Florian Schnappauf, vice president of enterprise commercial strategy at Veeva Europe.   Last year, orphan drugs accounted for more than a third of Europe’s new drug approvals, and the number of gene…

Italy SIFI’s Fabrizio Chines outlines how the company is progressing in European ophthalmic markets, explains the added value it is bringing to the glaucoma field, and tells us about its orphan drug for acanthamoeba keratitisis.    … Germany and the UK are the next strategic targets, but in those markets we will…

Singapore Writing in the December edition of DIA’s Global Forum magazine, Agnes Ho, Cindy Zen, and Tan Kwee Lan of Boehringer Ingelheim give an overview of the current regulatory framework for orphan drug registration in four ASEAN countries and what more could be done to improve it.   Regulatory authorities such…

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 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…

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…

Czech Republic Ewopharma’s Jan Krejčí, Country Manager for the Czech affiliate for the last eight years, shares the achievements of the company in its two main business pillars, prescription drugs, and consumer healthcare, and the successful entry into the hospital segment through the challenging and rewarding journey launching Santhera’s orphan drug for…

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…

UAE The UAE ranks as a world leader in rapid drug approvals with its fast-track registration system that accelerates the process and eliminates long waiting times for innovative and orphan drugs.   If you bring innovation, the [UAE] government will support you on all levels Georg Schroeckenfuchs, Novartis In January 2018…

China Eric Bouteiller outlines the paradigm shift occurring in China around rare diseases and how the country has the potential to lead the global pharmaceutical industry in this vital area.   Through exchanges with public officials, academics, representatives of patient associations and industry experts, we sensed a paradigm shift The 8th…

Opinion Kevin Rufang Huang is the president and founder of the Chinese Organization for Rare Disorders (CORD). A rare disease patient himself, he is responsible for bringing“International Rare Disease Day” to China. Consequently, rare diseases now have much more visibility in the country. As an expert on the subject, Kevin breaks…

Rare Diseases Long regarded as a neglected backwater within the drug discovery landscape and the preserve of only a handful of niche players, the rare disease space has been undergoing an extraordinary turnaround in fortunes of late.   According to EvaluatePharma’s forecasts, orphan drug sales are set to grow 11 percent each…