The National Institute for Health and Care Excellence (NICE) and NHS England have proposed a landmark revision to their cost-effectiveness evaluation framework, introducing a third explicit threshold specifically for ultra-orphan drugs. This development marks a significant shift in how the UK healthcare system values and funds different categories of medical treatments.
The evolving framework now encompasses three distinct thresholds, each serving different patient populations and treatment contexts. At its foundation, NICE maintains its long-standing baseline threshold of £20,000 to £30,000 per Quality Adjusted Life Year (QALY) for treatments targeting common diseases. This baseline has remained consistent since 2004 and was formally reinforced in the 2014 Pharmaceutical Price Regulation Scheme (PPRS).
For end-of-life treatments, NICE operates with an elevated threshold of approximately £50,000 per QALY, introduced in 2009. This higher threshold reflects a societal judgment that treatments for terminal patients warrant additional investment, challenging the traditional notion that all QALYs carry equal weight.
The most recent addition - and perhaps most controversial - is the £100,000 per QALY threshold for Highly Specialised Technologies (HSTs), commonly known as ultra-orphan drugs. This new threshold comes with a significant caveat: automatic funding is contingent upon the treatment not exceeding a budget impact of £20 million in any of the first three years post-launch.
Impact on Stakeholders
The threshold system has far-reaching implications across the healthcare landscape. For patients and clinicians, these thresholds determine access to new medicines through routine NHS funding. When treatments fall outside these thresholds, patients must navigate complex bureaucratic processes for individual funding requests or consider private payment options.
Pharmaceutical companies face significant commercial implications, as these thresholds effectively determine market access and revenue potential in the UK market. The system also impacts health economists' ability to optimize resource allocation within the NHS's limited budget.
Methodological Concerns
Critics have raised questions about the evidence base supporting these thresholds, particularly the newest £100,000 benchmark for ultra-orphan drugs. Unlike the original threshold, which evolved through empirical observation of decision patterns, or the end-of-life threshold, which emerged from societal values discussions, the ultra-orphan threshold appears to lack robust empirical or theoretical foundation.
System Implications
The introduction of multiple thresholds raises important questions about consistency and fairness in healthcare resource allocation. Some experts advocate for an independent threshold panel to reduce political influence in these crucial decisions. The system also prompts broader discussions about aligning health spending across different sectors, including preventive care and public health initiatives.
Future Considerations
As healthcare costs continue to rise and budgetary pressures intensify, the sustainability and appropriateness of these thresholds will likely face ongoing scrutiny. The upcoming renewal of the Pharmaceutical Price Regulation Scheme may further influence the evolution of these thresholds, though economic constraints make any upward adjustments unlikely in the near term.
