Rare disease patients could be denied vital treatment under new rules from NICE

Various UK biotechs have stated the current system in England for assessing treatments for extremely rare diseases is “not fit for purpose” and needs to change.

The BioIndustry Association made the statement in a response to proposals to change the way highly specialised drugs and health technologies are evaluated for NHS use.

The 12-week consultation by NICE and NHS England requested comments on proposals for evaluating and funding drugs and other health technologies, appraised through NICE’s Technology Appraisal and Highly Specialised Technologies programmes.

The proposals included automatic funding from routine commissioning budgets, treatments for very rare conditions costing up to £100,000 per Quality Adjusted Life Year (QALY) – much greater than NICE’s upper threshold of £30,000 per QALY for more commonly used medicines and technologies.

Treatments for very rare diseases above the £100,000 range will be considered through NHS England’s process for prioritising other highly specialised technologies.

However, this process is “not fit for purpose”, the BIA said, as it fails to address issues such as unmet medical need, burden of illness and impact on patients and carers.

The BIA also stated that QALY thresholds are not appropriate for evaluating medicines for very rare diseases, due to the small patient numbers which often limited data and uncertainty in the figures produced.

Many very rare disease drugs produce QALY values far more than £100,000, the BIA said, meaning that many treatments will get caught in bureaucracy.

The BIA recently hosted a round table discussion at the House of Lords, bringing together MPs and Alexion, BioMarin, Sanofi, Shire and Vertex – companies which all market highly expensive rare disease treatments.

NICE found that Alexion’s Soliris which was approved for a rare liver disease, costs the NHS more than £340,000 annually per patient – although it did recommend reimbursement in atypical haemolytic uraemic syndrome two years ago.

BIA chief executive Steve Bates 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.”


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