Tagged with Rare Disease

Switzerland Jane Griffiths, global head of Actelion – acquired in 2017 by Johnson & Johnson – discusses her priorities heading up an iconic rare disease-focused biotech and gives her take on a wide range of hot-button industry issues; from the future of M&As to patient-centricity, women in leadership and digital disruption.…

USA 80 orphan drugs (those drugs intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions) were approved in the USA in 2017, a record year and more than double the 40 approved in 2016.   Orphan drug spending grew from 5 to 10%…

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 Our contributor at large, David Crean, Managing Director for Objective Capital Partners, attended J.P. Morgan’s 2019 Health Care Investor Conference, one of the biggest events in the life sciences calendar and a fantastic indicator of what is to come in healthcare for the year ahead. Here, Crean gives us the inside scoop. …

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…