The proposed changes could stop the flow of new medicines reaching patients with very rare and complex diseases.
BIA members recently held a roundtable to discuss the issues raised by the consultation that was attended by MPs, Peers and patient groups with an interest in medicines for rare diseases.
Following the discussion the BIA’s Chief Executive, Steve Bates OBE, 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.”
Alastair Kent OBE, Director of Genetic Alliance UK, said: “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. Rather than the current situation of two complementary routes for funding approval for rare disease medicines, this proposal would compound an already existing bottleneck.”
The consultation proposes introducing a £100,000 quality adjusted life year (QALY) threshold for medicines evaluated via NICE’s Highly Specialised Technologies (HST) programme, which was developed to assess treatments for very rare conditions. If introduced, the threshold will effectively stop the flow of new medicines reaching patients with very rare and complex diseases.
Many treatments for very rare conditions that are currently funded by NHS England have costs per QALY in excess of £500,000 including the three medicines that have been approved by NICE’s HST process to date.
It is also widely acknowledged in the industry that QALY thresholds are not appropriate for evaluating medicines for very rare diseases, owing to the small patient populations, often limited data and uncertainty in the figures produced. Under the proposed changes, medicines which exceed the £100,000 QALY threshold will be evaluated via NHS England’s prioritisation process, which is not fit for purpose as it takes little account of unmet medical need, burden of illness and impact on patients and carers.
Introducing such dramatic changes to the evaluation process in England will create inequalities within the UK in patients’ ability to access treatments. The UK Rare Disease Strategy, published in 2013, commits each of the four nations to “equity of access.”
However, the changes proposed in the consultation will result in patients in England being able to access treatments evaluated under the old system but denied or significantly delayed access to medicines assessed under the proposed new system.
There is strong public support for treating patients with very rare diseases as part of the comprehensive offer of the NHS. Almost three quarters (74%) of the public agrees that patients with a very rare disease should have the same access to treatment based on clinical need as patients with common diseases and over two thirds (68%) 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.