NICE updates approach to orphan technologies
In May 2025, the UK National Institute for Health and Care Excellence (NICE) published an updated guide to health technology assessment - Health Technology Evaluations Manual (PMG36)
This document defines which technologies and under what conditions can be recommended for funding in the NHS. Particular attention is paid to orphan drugs - for them, new approaches to assessing effectiveness, financing and managing uncertainty are proposed.
We've looked closely at the updates - and here are 3 points that stand out:
1. Increasing the ISER* cap to £300,000 per QALY
*ICER is an incremental measure of the cost of one additional unit of health, measured in QALYs (quality-adjusted life years).
According to the new document in the UK:
- Standard technologies: the threshold remains the same — £20,000–30,000 per QALY (≈ 2.1–3.2 million rubles)
- For orphan diseases in the Highly Specialised Technologies (HST) program — increased to £100,000 (≈ 10.7 million rubles)
- In the presence of modifying factors (rarity, severity of the disease, high unmet need) — an increase to £300,000 per QALY (≈ 32.1 million rubles) is permissible
In Russia The IPZE is calculated as three times the GDP per capita and is 4.1 million rubles — a single threshold for all technologies. This approach does not take into account the specifics of rare diseases, where therapy can be expensive but vital.
2. Reimbursement on terms (managed access agreements)
The Guidelines contain clear provisions for Managed Access Agreements (MAAs):
- the criteria for temporary funding are fixed,
- there is a mandatory collection of real-world clinical data,
- entry and exit conditions are defined,
- review of decisions and price adjustments are allowed,
- payment based on outcome may be used.
This is critical for high-cost orphan drugs entering the market with limited evidence.
3. Strengthening the role of real-world data (RWD/RWE)
The new document for the first time so clearly establishes the status of RWE as a decision-making tool.
This is especially important for orphan technologies, where:
- it is often not possible to conduct full-fledged RCTs (small samples, ethical restrictions),
- decisions are made under conditions of uncertainty,
- long-term observation is required,
- price and reimbursement conditions can be revised as new data accumulates.
Thus, NICE is relying on a flexible, step-by-step assessment model, where evidence accumulates in real practice, and not just at the start of registration.
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