Rare disease insights
Reports, articles and conversations on launching into rare disease systems.
Article · By Patrick de Boer · 29 October 2026
Rare disease drug development is evolving. What does this mean for evidence strategies?
In 2025, half of the FDA's novel drug approvals were for rare diseases. How can sponsors turn evolving evidence approaches into successful development and patient access across international markets?
Article · By Jochem Bossenbroek · 19 October 2026
EU pharma package: orphan exclusivity and HTA evidence
Under the Council's text, orphan exclusivity would be nine years, or eleven for breakthrough medicines. What this means for HTA evidence teams.
Article · By Patrick de Boer · 15 October 2026
FDA's new rare disease framework: What it means for drug developers
The FDA wants to make rare disease drug development more predictable by involving patients and disease experts earlier and tailoring evidence requirements to individual diseases. We examine what could change, where challenges remain and why understanding the wider disease ecosystem matters for pharmaceutical and biotechnology companies.
Article · By Hilde Gerris-Krol · 6 July 2026
The Joint Clinical Assessment and rare disease
One EU-wide review of clinical value. Twenty-seven national decisions still to win. For rare and orphan products, the JCA is the hardest test yet — and 2028 is closer than it looks.
Article · By Jochem Bossenbroek · 1 June 2026
Why we focus on product-system fit
Product-market fit is necessary in healthcare, but it is not enough. Strong products can still fail to reach patients if the surrounding system is not ready to adopt and act on them. That missing piece is product-system fit.
Article · By Jochem Bossenbroek · 1 June 2026
How we measure ecosystem readiness
Readiness sounds soft. We make it a structured read of where the system stands and where it is weak.
Working through a launch readiness question? Let's talk.
Tell us the disease area, the priority markets and the decision in front of you, and we will come back to you.