Assessing Disease Severity: How HTA Bodiea Evaluate Unmet Needs and Influemce Decision-Making
Author(s)
Anthony Hudzik, PharmD1, Denise Garner, PharmD, MS2, Er Chen, MPP3, Chad Patel, PharmD4, Thomas Butt, PhD5, Danny Yeh, PhD6;
1AESARA, Boston, MA, USA, 2AESARA, Chapel Hill, NC, USA, 3BioMarin, San Rafael, CA, USA, 4AESARA, Charlotte, NC, USA, 5BioMarin, London, United Kingdom, 6AESARA, Burlingame, CA, USA
1AESARA, Boston, MA, USA, 2AESARA, Chapel Hill, NC, USA, 3BioMarin, San Rafael, CA, USA, 4AESARA, Charlotte, NC, USA, 5BioMarin, London, United Kingdom, 6AESARA, Burlingame, CA, USA
OBJECTIVES: The HEOR community acknowledges that innovation value goes beyond cost and quality-adjusted life year (QALY), leading to methodologies like Proportional QALY Shortfall (PQS) or Absolute QALY Shortfall (AQS) to quantify disease severity. This review examines how HTA bodies have incorporated PQS/AQS in their assessments and its impact on decision-making.
METHODS: This study compared how severity of diseases is evaluated among HTA bodies using QALYs as a measure of health benefit. Specifically, whether the HTA bodies incorporate PQS/AQS, along with its subsequent impact on willingness to pay (WTP). The study also examined whether PQS/AQS assessment is associated with final assessment decision for the 2024 appraisals by National Institute of Clinical Excellence (NICE), an HTA body that formally integrates PQS/AQS into their decision process.
RESULTS: Eight HTA bodies were reviewed: Institute for Clinical and Economic Review (ICER, US), NICE (England and Wales), Canadian Agency for Drugs and Technologies in Health (Canada), Zorginstituut Nederland (ZIN, Netherlands), Swedish Dental and Pharmaceutical Benefits Agency (Sweden), Pharmaceutical Benefits Advisory Committee (Australia), Norwegian Institute of Public Health (Norway), and Scottish Medicines Consortium (Scotland). Among them, three agencies (ICER, NICE, and ZIN) incorporated PQS and/or AQS in their assessments, with two (NICE and ZIN) explicitly linking PQS/AQS’ influence on WTP threshold in decision-making. In 2024, approximately one-third (16/47) of NICE appraisals assessed technologies for diseases with high PQS/AQS, leading to increased WTP thresholds. Among appraisals with increased WTP thresholds, 81% of technologies were recommended, compared to 45% for non-severe diseases assessed at the standard threshold.
CONCLUSIONS: Rewarding technologies for severe diseases can incentivize innovation in areas with high unmet needs. Initial evidence suggests that technologies addressing high-severity diseases, as measured by PQS/AQS, often receive more favorable recommendations from NICE. Other HTA bodies have adopted PQS/AQS to quantify disease severity, but more guidance is needed on how these results integrate into decision-making.
METHODS: This study compared how severity of diseases is evaluated among HTA bodies using QALYs as a measure of health benefit. Specifically, whether the HTA bodies incorporate PQS/AQS, along with its subsequent impact on willingness to pay (WTP). The study also examined whether PQS/AQS assessment is associated with final assessment decision for the 2024 appraisals by National Institute of Clinical Excellence (NICE), an HTA body that formally integrates PQS/AQS into their decision process.
RESULTS: Eight HTA bodies were reviewed: Institute for Clinical and Economic Review (ICER, US), NICE (England and Wales), Canadian Agency for Drugs and Technologies in Health (Canada), Zorginstituut Nederland (ZIN, Netherlands), Swedish Dental and Pharmaceutical Benefits Agency (Sweden), Pharmaceutical Benefits Advisory Committee (Australia), Norwegian Institute of Public Health (Norway), and Scottish Medicines Consortium (Scotland). Among them, three agencies (ICER, NICE, and ZIN) incorporated PQS and/or AQS in their assessments, with two (NICE and ZIN) explicitly linking PQS/AQS’ influence on WTP threshold in decision-making. In 2024, approximately one-third (16/47) of NICE appraisals assessed technologies for diseases with high PQS/AQS, leading to increased WTP thresholds. Among appraisals with increased WTP thresholds, 81% of technologies were recommended, compared to 45% for non-severe diseases assessed at the standard threshold.
CONCLUSIONS: Rewarding technologies for severe diseases can incentivize innovation in areas with high unmet needs. Initial evidence suggests that technologies addressing high-severity diseases, as measured by PQS/AQS, often receive more favorable recommendations from NICE. Other HTA bodies have adopted PQS/AQS to quantify disease severity, but more guidance is needed on how these results integrate into decision-making.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HTA25
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas