In a rush? That's your Bulletin summary done, or find the full story below.
Amanda Cole, Director
Power, Policy and Patients.
From April 2026, the National Institute for Health and Care Excellence (NICE) will raise its cost-effectiveness threshold, potentially enabling more medicines to be recommended on the NHS. However, the change — linked to a UK–US trade deal — signals a shift in decision-making power toward ministers. Clear, evidence-based and consultative processes will be essential to protect NICE’s independence and public trust.
Marked each year on the last day of February, last Saturday was Rare Disease Day and the focus this year was equity. Persistent inequities face rare disease patients worldwide. In ourinsight published today, we examine how policy reforms in the UK, EU, and US are reshaping incentives for developing and accessing rare disease treatments.
The common thread? Policy design matters. Getting the policy architecture right today is essential to ensuring tomorrow’s breakthroughs reach the patients who need them most.
Understanding the challenges in orphan drug development and patient care
Global rare‑disease policy shifts: what are the implications for equitable access to orphan medicines?
Rare diseases affect millions globally, but only a small fraction have approved treatments. This report examines the barriers to research and access, the impact of regulatory incentives, and recent UK, EU, and US policy developments. It highlights the need for coordinated efforts to improve access to orphan medicines.
NICE’s new cost-effectiveness threshold: Significance, consequences, and unanswered questions
In April 2026, NICE will increase its cost-effectiveness threshold for the first time in 20 years, from £20,000–£30,000 to £25,000–£35,000 per QALY. This change could lead to more medicines being recommended each year, but questions remain about its impact on NHS spending, innovation incentives, and how thresholds will be applied across different health technologies.
The Importance of Diversity of Supply in Rare Diseases Markets
Rare disease medicine markets often rely on a single supplier, leaving patients vulnerable to shortages and limiting treatment options. OHE looks at how increasing supplier diversity can make healthcare systems more resilient, encourage innovation, and offers actionable recommendations for policymakers.
The World Evidence, Pricing and Access Congress is Europe’s largest conference focused on market access, pricing and evidence. The event brings together 300 speakers across multiple streams and welcomes more than 1,500 attendees from pharma, biotech and payer/HTA organisations. The congress will take place on 3–4 March 2026 at the RAI Congress Centre in Amsterdam.
