We are delighted that research we conducted for The Bioindustry Association has recently been published as part of their report examining the social values of treating rare diseases, which looks into public opinion of how rare diseases should be funded.
During their recent large-scale review of its methods and processes, NICE concluded that there was no robust evidence that society values more highly health benefits in rare diseases. Following this conclusion, we were commissioned by the BIA to carry out qualitative research to support NICE’s requirement for usable and robust evidence. Building on deliberative public engagement approaches previously used by NICE, the research consisted of focus groups among a representative sample of the UK population and was supported by an online survey of 1,000 people.
Rare insights: examining the social values of treating rare diseases presents the findings of primary research into public opinion on how treatments for rare diseases should be evaluated and funded within the NHS.
The key findings as stated by the BIA are as follows:
- The research indicates that the UK public feel that equitable access is a central tenet of the NHS and that it would be unfair for people with rare diseases to not have equitable access to treatments, with 93% of focus group participants agreeing that people with rare diseases should have equitable access to treatments, even if this means additional costs for the NHS.
- The research also indicates that the public believe that the cost-effectiveness thresholds used for rare diseases should reflect the challenges associated with developing these treatments, relative to treatments for larger and smaller patient populations. In the focus groups, 75% of participants felt that the NHS should make funding decisions for medicines to treat patients with rare diseases using cost-effectiveness thresholds that fall in between those for treatments for very rare treatments and those for treatments for more common diseases.
- The research found that there is a range of factors that the public deems to be important in deciding how much the NHS should be willing to pay for rare disease treatments. Factors that a majority of participants felt to be important included the severity of the disease (71%), whether the disease is life threatening (64%), whether alternative treatments are available (64%), and whether the disease predominantly affects children (61%).
The research suggests that NICE is basing its methods and processes for evaluating medicines for rare diseases on outdated evidence. The findings signal the need for fresh research to determine the social values that should underpin the methods and processes used by NICE to make decisions about the NHS funding of treatments for rare diseases.
Click here to read the report.