Recently, the All Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions published a report which showed that the UK Strategy for Rare Diseases is not being implemented effectively in England. You can read the full report online on the Rare Disease UK website. The four nations of the UK have until 2020 to implement the 51 commitments outlined in the plan., While the departments of health in Wales, Scotland and Northern Ireland have all developed implementation plans, the Department of Health in England has not.
The APPG’s report found that:
New threat to rare disease patients
In the Strategy, NICE is responsible for implementing Commitment 13 to “ensure that there are appropriate procedures for evaluating the costs and benefits of treatments for patients”. However, NICE has recently announced its decision to implement changes to the programme for evaluating medicines for rare diseases, despite widespread condemnation from the rare disease community. This conflicts with Commitment 13 in the Strategy. In future, a measure called the Quality Adjusted Life Year (QALY) will be used in NICE’s evaluation rare disease medicines. QALYs have not previously been used to evaluate rare disease medicines because they do not fully capture the benefit a treatment can offer to patients and families affected by a rare condition. A new upper limit has been introduced in relation to the cost of the medicine. The upper-limit will vary according to the life-long impact of the medicine on the patient. NICE has so far approved 4 rare disease medicines that bring tremendous value to the patients eligible to receive them. Under the new rules, none of these treatments would have been approved.
The plans represent a risk to rare and genetic disease patients and their ability to access life-changing treatment.
Please join the TSA and the Genetic Alliance UK and act today, ahead of a Westminster debate on Tuesday 28th March.
Write to your MP
Become a case study
The Genetic Alliance UK are looking for people that may be impacted by these changes. For example, patients with experience of: