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Challenges in rare diseases:

Misplaced treatment expectations

Challenges to find the right treatment (if any):

  • Limited number of effective treatments
  • Lack of meaningful, real-world evidence or clear treatment guidelines
  • Treatment may only target symptoms, not cure

95% of rare conditions have no licensed treatment in the UK1


Treatments may be available but not accessible:

  • Reimbursement challenges for treatments that are considered too costly compared with perceived value of treatment
  • Challenging treatment regimens for patients
  • Limited access to treatment centres or specialists

Demonstration of real-life benefits

Reservations around initiating or continuing therapy:

  • Lack of real-world evidence on patient outcomes and improved quality of life
  • Concern for potential therapeutic side effects, leading to lower patient satisfaction compared with no treatment
  • No perceived benefit after treatment commencement
  • Perception of patient being ‘cured’ or no longer needing expensive ongoing treatment


1. Slade A, Isa F, Kyte D, et al. Patient reported outcome measures in rare diseases: a narrative review. Orphanet J Rare Dis 2018;13(1):61

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