USA Pamela Gavin, CEO of the National Organization for Rare Disorders, discusses NORD’s origins in the advocacy movement that led to the Orphan Drug Act and its current mission to improve diagnosis, care, research, and policy for rare disease patients. She highlights the growing role of patient-led research, the need for…
Europe Childhood cancer survival rates in high-income countries now exceed 80 percent, but that masks a more uneven picture: paediatric drug development lags far behind adult oncology, follow-up and psychosocial care remain patchy across Europe, and survivors can face life-altering side effects that surface decades after treatment ends. Carina Schneider and…
Ireland The first thing I’d say to innovators is to ask the patients. Patient groups are ready to support innovation, make sure we’re measuring what matters, and delivering what’s meaningful Avril Daly brings a rare dual perspective to Europe’s rare disease and ophthalmology landscape: as CEO of Retina International, she represents…
Europe A 2025 collection of perspectives from over 30 biopharmaceutical industry stakeholders finds that while patient centricity has made real progress — moving from an afterthought to an expectation in the US and Europe — implementation remains patchy and often superficial. As Matthew Reaney, writing in ISPOR’s Value & Outcomes Spotlight…
MEA Majid Jafar, Co-Founder of the Loulou Foundation, discusses the strategic evolution of the patient research organisation that he co-founded with his wife following their daughter Alia’s diagnosis with CDKL5 Deficiency Disorder (CDD). By moving beyond traditional advocacy into “philanthropharma”, Jafar has spearheaded the development of a global translational toolkit, contributing…
USA When Nasha Fitter’s daughter, Amara, was diagnosed with the ultra-rare FOXG1 syndrome at just seven months old, the prognosis was stark: no speech, no independent movement, severe intellectual disability, and lifelong epilepsy. For many families, such a diagnosis marks the beginning of a long wait – for care, for answers,…
USA Nasha Fitter, a tech entrepreneur and CEO of the FOXG1 Research Foundation, discusses the radical shift in drug development led by a new generation of sophisticated parent-entrepreneurs. Following her daughter’s diagnosis with the ultra-rare neurodevelopmental disorder FOXG1 syndrome, Fitter co-founded the FOXG1 Research Foundation and Citizen Health. Her mission: to…
France 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 by private industry and the state while elevating the concerns of the most important people within healthcare: patients and their…
USA 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 and which collectively impact over 30 million Americans. For these…
USA Founded to tackle one of the most persistent blind spots in rare disease policy, the Rare Access Action Project (RAAP) focuses on what happens after FDA approval, when therapies meet the realities of coverage, reimbursement and real-world access. Led by Executive and Founder Michael Eging, RAAP brings together patient organisations,…
USA At a moment when rare disease policy, diagnostics, and patient-driven research are converging, Danielle Carnival sets out a clear vision for the role of the Undiagnosed Diseases Network Foundation. Drawing on her experience across neuroscience, federal health policy, and patient-led organisations, she explains how UDNF operates upstream of diagnosis, addressing…
France Born from personal tragedy, Imagine for Margo has evolved into a pan-European force reshaping how childhood cancers are researched, funded, and brought to the policy agenda. Drawing on a parent’s perspective, Patricia Blanc explains how precision medicine, cross-border collaboration, and sustained advocacy can compress timelines and reduce inequality in rare…
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