Tagged with Rare Diseases

India Anay Shukla leads the pharmaceutical, life sciences, med-tech and healthcare practice at Nishith Desai Associates, a leading Indian law firm. In this article, he examines the benefit of the recent policy changes to price controls for innovative drugs in India.  Unlike many other countries, India controls prices of all drugs…

Korea This week, the Korean Ministry of Food and Drug Safety announced that it would enhance drug safety management and make a new, streamlined review process for advanced healthcare goods. The new process will tighten control on raw drug materials, expand patient compensation for side-effects, and prepare review systems for advanced…

China Davide Dalle Fusine, general manager for Chiesi China, shares the key milestones for the company in the past five years, their leadership position in neonatology and strength respiratory franchise in China, the importance of the Chinese market for global operations, as well as his strategic priorities for the next few…

Canada The research areas in which Canada has the potential to excel are numerous, one being the world of genomics. The stem cell itself having been discovered in Canada, Marc LePage, president and CEO of Genome Canada, identifies great potential to attract more fundamental research into that domain in the future.…

France Franck Puget, managing director for CSL Behring in France, discusses the blood plasma specialist’s commitment to France, building bridges with public authorities, and regulatory attitudes towards rare disease treatments.   Our neighbours envy the Temporary Authorization of Use (Autorisation Temporaire d’Utilisation / ATU) system that we have in France, and…

Opinion After her son was diagnosed with SYNGAP1 – an extremely rare gene mutation – Monica founded Bridge the Gap – SYNGAP Education and Research Foundation to support families of those suffering from the illness and to accelerate the path to better therapies. Here Monica discusses the incredibly low rate of treatment options for…

Spain Giuseppe Chiericatti of Chiesi Spain talks strategy, the development of the affiliate and shifting attitudes towards rare diseases in Spain.   Social responsibility and sustainability is a key part of our mission. Our goal is to integrate the concept of sustainability in whatever we do You’ve been leading the Spanish…

USA PJ Brooks PhD, program director in the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH) in the USA, highlights the importance of going beyond one disease at a time in clinical trials, some of the roadblocks that still remain…

Europe 125 medicines for rare diseases were approved by the EMA between 2004 and 2014, of which 71 were granted orphan drug status. This chart shows the availability of 112 of these drugs across European countries. The largest number of medicines for rare diseases were in continuous use in Germany and…

Turkey Levent Canyurt, CEO and founder of BIEM Ilac, shares how he transformed the company from a domestic pharmaceutical distributor into an international player, with an innovative portfolio for the treatment of rare diseases. He also highlights the importance of greater collaboration between stakeholders in the Turkish biotech industry and gives…

Turkey Abidin Gulmus, founder and chairman of Gen Ilaç, describes the company’s impressive journey to become Turkey’s principle orphan drug distributor and shares his vision for leveraging the company’s domestic expertise to expand their reach into foreign markets.   Gen Ilaç is one of the fastest growing Turkish success stories although…

Malaysia Sea cucumbers – odd-looking marine animals with leathery skins and elongated bodies – can be found on ocean floors worldwide. Although more famous as a culinary delicacy in East and Southeast Asia, one Malaysian company – Healwell – is using these primitive creatures to save lives.   Dr Hassan Yaakob,…