Tagged with Rare diseases

India As rare disease regulation moves from the margins to the mainstream of global health policy, India faces both a challenge and an opportunity: how to align with international best practices while designing solutions suited to its vast population and limited resources. This article — authored by experts from Power In…

France With only a handful of patients affected by each condition, rare diseases present a unique set of scientific, medical, and economic challenges. At the heart of this complex landscape is Recordati Rare Diseases, whose European headquarters and manufacturing footprint are anchored in France. General Manager Jean-Claude Roche shares his perspective…

Europe Affordability is not necessarily the main barrier to better rare disease therapy access in Europe. Across the continent, orphan availability correlates poorly with GDP per capita, but strongly with health technology assessment methods, speed of pricing and reimbursement, and central vs regional decision-making. Better system design – rather than simply…

Italy Banking on three key therapeutic areas, including a newly established rare disease franchise, the Italian midcap Chiesi has pursued a sweeping transformation in recent years. In its reboot efforts, the company has not only made significant R&D investments, but pursued key M&A and partnership deals, and aimed for global expansion.…

Puerto Rico Glorimar Torres, with over 33 years in the pharmaceutical industry, has had a diverse career, starting as a sales representative in Puerto Rico and progressing through key roles at companies like Merck & Co. (MSD), Bristol Myers Squibb, Pfizer, and GlaxoSmithKline (GSK) before joining Amgen. At Amgen, she played a…

Saudi Arabia Rare diseases, by definition, only affect small patient populations. However, when the more than 7,000 known rare conditions are taken together, their impact on health, societies, and economies is enormous. Moreover, over 90 percent of rare diseases have no known treatment, and rare disease patients often face delayed diagnoses and…

Saudi Arabia Osama Braiwish leverages over 26 years of industry experience across the Gulf region and Saudi Arabia. He joined Bristol Myers Squibb (BMS) in 2024, drawn to the company’s commitment to addressing significant unmet medical needs in oncology, hematology, immunology, cardiovascular and neuroscience. In his first few months at BMS, Braiwish…

Europe Dr Carolyn H. Rogers, Rushikesh S. Dasoondi and Dr Michael A. Roberts from UK and European intellectual property law firm Reddie & Grose LLP examine the intersection of personalised medicine and rare diseases and its implications for IP law.   Personalised medicine, also referred to as precision medicine, is an…

Mexico Rare diseases, by definition, only affect small patient populations. However, when the more than 7,000 known rare conditions are taken together, their impact is enormous. Rare disease patients, for example, often face delayed diagnoses, limited treatment options, and a lack of specialised care compared to those suffering from more common…

Saudi Arabia AstraZeneca has made remarkable progress over the past three years in Saudi Arabia, gaining significant leadership in therapeutic areas like oncology and respiratory care, and expansion into rare diseases. Sameh ElFangary, Country President of AstraZeneca GCC & Pakistan shares insights into the company’s strategy and strong alignment with Saudi Arabia’s…

China There are over 7,000 different rare diseases globally, many of which affect only a handful of patients. However, given the scale of China’s population, rare diseases in the country take on an increased significance, something that Chinese policymakers have worked to address in recent years.   “China now introduces new…

China Deng Haoqing, President and General Manager of Chiesi China, explores the company’s strategic growth in one of the world’s most dynamic healthcare markets. Deng discusses the alignment of Chiesi’s “AIR, CARE, and RARE” focus with China’s evolving healthcare priorities, highlighting efforts to bring innovation through local and global collaborations, including…