The BioIndustry Association is warning that proposals to change to the way medicines and other health technologies are evaluated for NHS use could actually stop the flow of new therapies reaching patients with very rare and complex diseases.

The National Institute for Health and Care Excellence and NHS England launched a consultation last year on planned changes to the arrangements for evaluating and funding health technologies appraised through NICE's Technology Appraisal and Highly Specialised Technologies programmes.

Among the proposals is to automatically fund, from routine commissioning budgets, treatments for very rare conditions up to £100,000 per QALY (five 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.

However, the BIA argues that NHS England's prioritisation process "is not fit for purpose as it takes little account of unmet medical need, burden of illness and impact on patients and carers", and that QALY thresholds "are not appropriate for evaluating medicines for very rare diseases, due to the small patient populations, often limited data and uncertainty in the figures produced".

Given that many treatments for very rare conditions currently funded by NHS England have costs per QALY in excess of £500,000, introducing the new £100,000 per QALY threshold would interrupt access to rare disease therapies, it claims.

"NHS England's prioritisation process desperately needs improvements to ensure equitable decision-making for rare disease therapies. Before these improvements are made, we must reject proposals to link NICE's rare disease process with NHS England's in the manner proposed," commented Alastair Kent OBE, Director of Genetic Alliance UK.

"Rather than the current situation of two complementary routes for funding approval for rare disease medicines, this proposal would compound an already existing bottleneck".

Also rejecting the proposals, Steve Bates, the BIA's chief executive, said: "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."

Other plans announced by NICE and NHS England are 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, and to 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 cost watchdog.