The public strongly agree that patients living with a rare disease should be able to equally access medicines on the NHS, new report reveals

The BIA has today launched a report Public attitudes to Rare Diseases: The case for equal access which presents the findings of a survey commissioned by the BIA on public attitudes towards rare diseases and access to medicine.

The survey, conducted by YouGov, revealed that the public strongly believe that patients living with a rare disease should be able to access medicines through the NHS on the same basis as people living with more common conditions. Further, the majority agreed that the NHS should ensure access on the basis of clinical need to these patients, even if it would be more costly to the NHS.

The findings follow recent assertions made by England’s HTA body, NICE, during a review of their methods and processes that there isn’t appetite or interest among the general public for specific measures to tackle rarity as an issue.  

The report recommends that NICE revise their position on this issue and considers the value of a rarity modifier as part of the HTA process to people with rare diseases and the general public.

Key findings:

  • 79% of respondents agreed that patients living with a rare disease should be able to access medicines on the same basis as people living with more common conditions.
  • 78% of respondents agreed that the NHS should ensure access on the basis of clinical need even if this would be more costly to the NHS.
  • 46% agreed that the cost threshold for medicines for rare diseases should be raised to ensure equitable access to medicines for all.

Steve Bates OBE, Chief Executive of the BIA said: “As scientific discovery delivers new and extraordinary treatments, it is essential that patients are able to benefit from them and that must include people living with rare diseases. Securing patient access to rare diseases face very specific issues, including small patient populations, relatively poor understanding of the epidemiology of these conditions, a significant diagnostic odyssey and the absence of a ‘standard of care’ to act as a benchmark. This survey once again demonstrates that there is broad public support for measures to ensure access to medicines for rare diseases based on clinical need even if that would entail higher costs. We would therefore encourage NICE and the Department of Health and Social Care to revisit their assumptions on public appetite for rarity specific measures.”

ENDS

Notes to Editors

  1. To read Public attitudes to Rare Diseases: The case for equal access please follow this link.
  2. The field work took place between 7 and 8 April 2021 with 2,023 respondents in Great Britain (no responses were received from Northern Ireland)
  3. Read more about the BIA’s recommendations for improving access to medicines for rare diseases here
  4. Read more about the NICE Methods Review here