Treatment

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

Access

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

References

 
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

Talk to us about how we can transform your communications

Alpharmaxim in the press

Alpharmaxim in the press

With many organisations beginning to invest in behavioural science, we’ve been shouting in the press about the benefits for pharma companies.
Ten ways that internal communications can boost your business

Ten ways that internal communications can boost your business

Employee engagement, brand alignment, sharing expertise to solve problems: these are all things that internal communications can achieve.
It's time for behavioural science to take centre stage in health communications

It's time for behavioural science to take centre stage in health communications

Progress brings new challenges, and help is needed. It might be time for behavioural science to play a bigger part than ever before.