For decades, developing a new CF treatment followed a familiar script. Patients in trials had progressive lung disease and few alternatives. Placebo-controlled studies were the standard. Improvement in lung function was how success was measured. That script no longer fits, and the European Medicines Agency has finally rewritten it. The revised EMA guideline on the […]
Month: July 2026
The first Joint Clinical Assessment under the EU HTA Regulation: what it means for people with CF
On 22 June 2026, a milestone arrived quietly but with significant implications for people living with rare diseases across Europe: the publication of the first Joint Clinical Assessment (JCA) under the EU HTA Regulation. The webinar marking this moment brought together patient organisations, medical societies, industry and health technology assessment (HTA) bodies, organised by the […]
Believing. Building. Becoming: CF Europe’s Annual General Meeting in Lisbon 2026
Lisbon in early June has a particular quality to it: warm evenings, long light, and a city that feels like it has been waiting for you. It turned out to be the perfect backdrop for two days that were, by any measure, full. CF Europe members and partners came together from across the continent for […]