France’s Patient Advocacy Power
France’s patient advocacy network is one of Europe’s most well-developed and formalised. Patient advocacy groups – including the three featured below – fill in research, care, and support gaps left…
Patients and carers with lived experience of a disease have valuable insights to share with physicians and drug developers alike. This is especially true of rare diseases, defined in the US as those that affect fewer than 200,000 people nationwide…
France’s patient advocacy network is one of Europe’s most well-developed and formalised. Patient advocacy groups – including the three featured below – fill in research, care, and support gaps left…
France has long been held up as Europe’s reference point for rare disease care – the first EU country with a national plan, a pioneer of coordinated expert networks, and…
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…
Global advances in rare blood disorders increasingly hinge on where evidence is generated, how patients are followed, and whether innovation reaches beyond a limited few. Khaled Musallam describes how clinical reality at Burjeel informs international research, trial design, and guideline development, while pressing for models that balance scientific ambition with global accessibility. From building integrated research platforms within Burjeel to…
Dr Said Ismail, geneticist and professor of genomics at Hamad Bin Khalifa University, reflects on his role as the founding director and one of the architects of the Qatar Genome…
Craig Martin, CEO of the Orphan Therapeutics Accelerator (OTXL), discusses the urgent need for a new business model to rescue abandoned clinical-stage programs in the rare disease sector. He details…
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…
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…
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.…
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…
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,…
France is considered among the leading rare disease markets worldwide, both scientifically and organisationally. The country has cultivated a fully-fledged supporting ecosystem for rare conditions that is today regarded as…
See our Cookie Privacy Policy Here
