NICE has put forward key changes to the way they assess medicines and treatments, which should simplify the appraisal process for new rare disease treatments. Given the pace at which new neuromuscular treatments are being developed, these changes are coming at an opportune time, and we hope will reduce unnecessary delays people with a muscle-wasting disease have often experienced in the past.
The National Institute of Care and Health Excellence (NICE) is the public body that provides national guidance and advice to improve health and social care across England, Wales and Northern Ireland. It also reviews new treatments before they are approved to market.
NICE has been using the same appraisal since 2009. Over the last few years (there was a few delays because of COVID-19), NICE has been reviewing their process and methods for reviewing medicines. The goal is to ensure the methods are till fit-for-purpose, and to give NICE greater flexibility to decide which medicines represent value for money, and should be paid for by the NHS in England, Wales and Northern Ireland.
In our response to their proposed changes, we:
- welcomed the introduction of a severity modifier. (Modifiers are factors that are considered within NICE's decision-making process.) The introduction of a severity modifier means that NICE is prepared to pay more for a medicine if it treats patients with a severe disease.
- were pleased to see a greater emphasis on the consideration of uncertainty in decision-making. (Uncertainty is a common barrier to access to rare disease treatments, especially if there is a low number of participants in clinical trials.) We were disappointed it wasn't put forward as an official modifier, but we acknowledge this is already a big step forward.
- were surprised to note that NICE decided not to reduce the discount rate. (Discounting is an economic method used in assessing benefit versus costs. For example, offering people the opportunity to purchase a product and pay for it through installments rather than paying for it in one go.) Without the reduced discount rate, pharmaceutical companies will have to pay more at the start of the appraisal, while they continue to collect evidence to demonstrate the benefit in the longer term. If the upfront cost is large, which is often the case for rare disease treatments, then the pharmaceutical company may decide to delay submitting the treatment to NICE for appraisal. This, in turn, could lead to patients having to wait longer to access new treatments.
- welcomed the increased use of real-world evidence, which has been essential in organising managed access agreements in the past. The hope is that this will lead to an easier way to demonstrate the benefits of a treatment.
Robert Burley, MDUK Director of Care, Communications and Support, said:
MDUK is pleased with the proposed changes put forward in the NICE methods review, as we believe these changes will transofrm and widen access to new neuromuscular treatments as they emerge. We will continue to engage with NHS England, NICE, individuals, families, clinicians, and other patient groups to ensure people with muscle-wasting conditions access the treatments they need as soon and as safely as possible.
We're committed to campaigning for access to treatments for people with muscle-wasting conditions, and we'll update you on this review process as soon as NICE announces further details. If you would like to find out more about this and other campaigns, please do get in touch with us at firstname.lastname@example.org.