Re-anchoring the Value of Innovative Therapies in NICE Decision Making When Comparators are Cost Ineffective: A Case Study of Late-Onset Pompe Disease

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Abstract
National Institute for Health and Care Excellence (NICE) technology appraisal processes assume that the standard of care (SoC) is itself cost effective. However, many treatments in use in the UK National Health Service (NHS), particularly in rare diseases, were historically commissioned without formal value assessment and are priced without reference to cost-effectiveness thresholds. Cost-ineffective comparators distort how value is ascribed to new technologies, undermining the coherence of NICE’s decision-making framework, and imposing substantial opportunity costs on the NHS. Using late-onset Pompe disease (LOPD) as an exemplar, we demonstrate the implications of a cost-ineffective comparator in assessments of innovative therapies. A clinically superior enzyme replacement therapy (ERT) may command a lower value-based price than current ERTs, whilst a hypothetical curative gene therapy is valued at over £4 million against current ERT, but just £629,392 when re-anchored against best supportive care. Here, value is driven by displacement of costs rather than health gain, raising affordability concerns that may limit access to genuine innovation. The 2025 NHS 10-Year Plan grants new NICE statutory powers to withdraw access to cost-ineffective therapies, presenting an opportunity to reform technology appraisal. We propose several policy responses, including comprehensive reassessment of active guidance with decisions made with respect to a standard cost-effectiveness frontier, reviews triggered by new comparators, and use of flexible decision rules within existing frameworks. These changes could allow the evolving value of medicines to be reflected in NHS practice, redefining NICE as a body that takes a dynamic, whole-lifecycle view of value. Deliberative public and stakeholder engagement is essential for success, given the potential consequences for manufacturers and patients.
Funding Information
  • Evidence Synthesis Programme (NIHR153779)

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