HTA Appraisal Framework Suitable for Rare Disease Treatments
WP10 – Guidance to support consistent HTA appraisal for orphan medicinal products (OMPs)
HTA Appraisal Framework Suitable for Rare Disease Treatments K Facey1, A Whittal2, M Drummond2‘3, S Upadhyaya4, T Junghans1– E Nicod2 University of Edinburgh – Scotland, 2Bocconi University – Italy, 3University of York – England, 4National Institute for Health and Care Excellence – England 12 May 2021 |
This appraisal framework with the enactment of the detailed guidance will support consistency of flexibility in appraising RDTs to ensure fairness, within a framework of accountability for reasonableness (Daniels and Sabin 2008). A fair process is one that ensures the inclusion of all relevant evidence and knowledge, consistently throughout the process, to allow for the best decision possible given the unique circumstances of the disease.Executive Summary Background
Orphan Medicinal Products or Rare Disease Treatments (RDTs) are challenging to appraise as the limited numbers for study often lead to small and/or uncontrolled trials of short duration. In addition to the limited clinical evidence base, there is often a paucity of knowledge about the disease and how patients progress. Furthermore, RDTs may have a high price to seek a return on investment from a small treatable population and strong demand given high unmet needs. These issues lead to major uncertainties in HTA appraisal of clinical and/or cost-effectiveness. Some HTA bodies address this by using special processes to appraise RDTs; others add special features, or decision modifiers, to standard processes; others give no leniency to RDTs. The aim of this research has been to create an appraisal process beyond conventional economic evaluation that is suitable for RDTs within the context of fair resource allocation and equitable service provision for all people in a health system.
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