NICE and NHS England consultation could stop access to new medicines for patients with rare diseases

A consultation by NICE and NHS England on changes to the arrangements for evaluating and funding medicines could potentially stop the flow of new medicines reaching patients with rare and complex diseases.

NICE and NHS England proposed introducing a £100,000 quality-adjusted life year (QALY) threshold for medicines evaluated by NICE’s Highly Specialised Technologies (HST) programme, designed to assess treatments for very rare conditions.

Treatments for very rare conditions are currently funded by NHS England. These treatments, which include particular ones chosen by NICE’s HST programme, have costs per QALY in excess of £500,000.

The reduced threshold will mean that any medicines which exceed the £100,000 QALY limit, will instead be evaluated via NHS England’s prioritisation process. This process is regarded as being insufficient, as it doesn’t take into account unmet medical needs, burden of illness and the impact on patients and carers.

In addition, the new changes to the evaluation process will create inequalities within the UK, due to the restrictions placed on patients’ ability to access treatments.

The changes will mean that patients in England will be able to access treatments evaluated under the old system, but will be denied or have significantly delayed access to medicine assessed under the proposed new system.

74% of the public agrees that patients with a very rare disease should have the same access to treatment as patients with common diseases. More so, 67% of the public agrees that the NHS should ensure patients with very rare diseases have the same access to treatment as patients with common diseases, even if it means savings have to be made elsewhere in the NHS.

In response to the consultation, the UK BioIndustry Association held a roundtable to discuss the issues raised by the proposed changes. The roundtable was attended by MPs, peers and patient groups.

Steve Bates, 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.”