Tagged with Rare Disease

Rare Diseases In the run-up to Rare Disease Day 2019, David H. Crean, PhD, Managing Director for Objective Capital Partners, reviews the investment and deal activity for orphan drugs in 2018 and delves into future forecasts for the rare disease space.   With drugs currently available for only about 5% of rare diseases,…

Canada Durhane Wong-Rieger is the President & CEO of the Canadian Organization for Rare Disorders (CORD). In her first article for PharmaBoardroom, Durhane explains the changing landscape for access to rare disease drugs in Canada and the actors involved in getting there.   The irony of the government processes, which, on…

Opinion After her son was diagnosed with SYNGAP1 – an extremely rare gene mutation – Monica Weldon 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 shares her organisations secret to success in the…

China It has been announced by China’s State Council that as of March 1st 2019, the government will reduce the VAT on a “first batch” of 21 rare disease drugs and four active pharmaceutical ingredients (API’s) by more than 80% in a bid to get new treatments to market faster. Historically, China has…

GW Pharma Founded in 1998, GW Pharmaceuticals is a British biopharmaceutical company, best known for being the only company with a license to cultivate cannabis in the UK. True pioneers, they are the producers of Sativex and Epiodiolex, the former being the world’s first prescription medicine derived from the cannabis plant —…

Korea ISU Abxis, a subsidiary of the ISU conglomerate, is a biotech company with a focus on orphan drugs and rare disease. CEO Seok Joo Lee explains the rationale behind his decision to transform ISU Abxis’ international strategy from targeting developing markets to aiming for developed Western markets, such as the…

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.…