Alpharmaxim full lockup logo


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

At Alpharmaxim, we have extensive experience in helping speciality healthcare companies across the world communicate with healthcare professionals and patients, particularly in rare diseases. We are passionate about helping our clients tell their stories and fulfil their promises, and we aim to make a real difference to patients, families and healthcare professionals.

If you would like to know more, please visit our website, or contact Sophie Jones on +44 (0)161 929 0400.