New NICE ultra-rare disease proposals "not fit for purpose", says BIA

The BioIndustry Association (BIA) has accused new proposals put forward by the National Institute for Health and Care Excellence (NICE) of being "not fit for purpose" and potentially denying patients from accessing treatments for ultra-rare disease.

The proposals could fundamentally change the way NICE judges determine the cost-effectiveness of new drugs and devices for use on the NHS. Among the proposals was the concept of automatic funding from routine commissioning budgets and the delineation of ultra-rare disease treatments as costing up to £100,000 per Quality Adjusted Life Year (QALY) compared to £30,000 for regular treatments. For disease treatments costing £100,000 and beyond, the appraisal would be carried out through the Technology Appraisal and Highly Specialised Technologies programmes.

The BIA has spoken out against the proposals for using the inappropriate metric of QALY, which does not lend itself well to evaluating ultra-rare disease due to small patient populations and thin data, and for failing to tackle the issues of unmet medical need, burden of illness and impact on both carers and patients. Many treatments cost much more than £100,000 QALY a year.

BIA chief executive Steve Bates explained: "Rather than unlocking innovation in the NHS and delivering equity and access to all patients the proposed changes in the consultation will delay access to medicines and undermine confidence in the system for both patients and industry. It is vital that the industry collaborates with NICE and NHS England to ensure that ground breaking treatments can be made available to patients quickly and efficiently, wherever they live in the UK."

Matt Fellows