Wales announces new process for appraisal of treatments for rare diseases

From September 2015 the All Wales Medicines Strategy Group (AWMSG) will be changing its process for appraising orphan, ultra‐orphan and medicines developed specifically for rare diseases to enable even greater involvement of patients and clinicians in Wales.

Where the cost per QALY is above the normal thresholds applied, additional criteria for appraising these medicines will be considered. These will include, but will not be limited to:

  • The degree of severity of the disease as presently managed, in terms of survival and quality of life impacts on patients and their carers
  • Whether the medicine addresses an unmet need (e.g. no other licensed medicines)
  • Whether the medicine can reverse or cure, rather than stabilise the condition 3 May 2015
  • Whether the medicine may bridge a gap to a “definitive” therapy (e.g. gene therapy) and that this “definitive” therapy is currently in development
  • The innovative nature of the medicine
  • Added value to the patient which may not adequately be captured in the QALY (e.g. impact on quality of life such as ability to work or continue in education/function, symptoms such as fatigue, pain, psychological distress, convenience of treatment, ability to maintain independence and dignity)
  • Added value to the patient’s family (e.g. impact on a carer or family life)

There is no mention of whether treatments in the NICE highly specialised technology process will be also be evaluated by the Welsh HTA.

Full detail from AWMSG is available in this document

Published 4. June 2015 in News UK