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HTA Appraisal Framework Suitable for Rare Disease Treatments

K Facey, A Whittal, M Drummond, S Upadhyaya, T Junghans, E Nicod
May 2021

Clinical- or cost-effectiveness- based HTA appraisals of rare disease treatments (RDTs) are often challenging due to small patient populations, uncontrolled trials and limited disease knowledge. Following consultation with HTA leaders from various country pricing and reimbursement (P&R) systems, this paper seeks to overcome these difficulties by developing recommendations for a RDT appraisal process that extends beyond traditional economic evaluations to better capture RDT specificities. The resulting IMPACT HTA Appraisal Framework, based on the tenets of leniency, flexibility and consistency, offers detailed guidance for HTA’ bodies to conduct fair and consistent evaluations of RDTs.

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