Duchenne UK participates in the recent NICE/ NHS England consultation January 2017 Duchenne UK were pleased to participate in the recent NICE and NHS England consultation on changes to the arrangements for evaluating and funding drugs and other health technologies appraised through NICE’s Technology Appraisal (TA) and Highly Specialised Technologies (HST) programmes. NICE and NHS England propose to work together more closely to better manage access to new drugs and medical technologies (devices and diagnostics) by simplifying and speeding up some appraisals, and by making the arrangements for funding others more clear. The proposed changes are intended to benefit patients by providing access to the most effective and cost-effective new treatments more quickly and also to help the life sciences industry by increasing the opportunities for companies to help manage the introduction of their new technologies into the NHS. The consultation covers all new therapies and technologies and is not specific to Duchenne muscular dystrophy (DMD) although our responses were from the perspective of those living with DMD. The main proposals, alongside the Duchenne UK response, are summarised below: Proposal 1 To introduce a ‘fast track’ NICE technology appraisal process for the most promising new technologies, which fall below an incremental cost-effectiveness ratio of £10,000 per QALY (quality adjusted life year), to get these treatments to patients more quickly. Duchenne UK response: We agree that this is a good idea in principle but it is only likely to be relevant to low cost interventions in prevalent conditions. £10,000/QALY is an unrealistic figure for any treatment for Duchenne. Proposal 2 Operate a ‘budget impact threshold’ of £20 million, set by NHS England, to signal the need for a dialogue with companies to agree special arrangements to better manage the introduction of new technologies recommended by NICE. This would apply to a small number of technologies that, once determined as cost effective by NICE, would have a significant impact on the NHS budget. Duchenne UK response: A budget impact threshold of £20m appears an arbitrary figure, and some more information on how this was reached as a proposal would be helpful. It is also unclear how this will be calculated and subsequently measured, and further explanation is required to make informed comment. It is also unclear how a new treatment for disease where there was previously no treatment would be managed, as there would be a prevalent group of patients requiring treatment in the first year, skewing the costs. Upcoming treatments for Duchenne could be expected to be above the proposed budget impact threshold set at £20m/annum. It is not clear how NHS England will respond to paying for these treatments once the threshold is reached, other than negotiating lower costs from the companies, phased introduction or patient access schemes. Proposal 3 To vary the timescale for the funding requirement when the budget impact threshold is reached or exceeded, and there is therefore a compelling case that the introduction of the new technology would risk disruption to the funding of other services. Duchenne UK response: We do not agree that the timescale should be varied. NHS England and NICE need to look to the long term social, financial and health benefits of introducing new treatments for Duchenne. Short term analysis of the benefits and costs of the new treatments for Duchenne set against cuts to other services is a policy that could further delay the possibilities to treat and cure Duchenne muscular dystrophy. Proposal 4 To automatically fund, from routine commissioning budgets, treatments for very rare conditions (highly specialised technologies) up to £100,000 per QALY (5 times greater than the lower end of NICE’s standard threshold range), and provide the opportunity for treatments above this range to be considered through NHS England’s process for prioritising other highly specialised technologies. Duchenne UK response: We do not agree with the imposition of a threshold in HST. If a threshold is implemented, and medicines exceed it, then we would suggest that a designated budget to meet the needs of new Duchenne medicines would need to be in place.