Appraising Ultra-Orphan Drugs: Is Cost-Per-QALY Appropriate? A Review of the Evidence

Towse, A. and Garau, M.

Consulting Report
March 2018

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The report addresses the implications of NICE appraising treatments for very rare diseases using a cost-per-QALY gained decision rule of the type used by NICE in its Technology Appraisal Programme to appraise therapies for more common conditions.  
 
Given the importance of non-QALY elements in the assessment of HSTs, such as treatment impact on the process of care and on the patients’ or their carers’ ability to go to school or to work respectively, and issues in measuring quality of life when the population affected are infants or young children, it is inappropriate to focus the appraisal of treatments for very rare diseases solely on a cost-per-QALY measure. Given the lack of empirical basis, the new £100,000 cost per QALY threshold and its further possible uplift up by a factor of three seem arbitrary.