Navigating The Icer Value Assessment Framework For Rare Disease Treatments

• By: YU, Tianzhou (University of Southern California, United States)
• Co-author(s): Mr Tianzhou Yu (Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, United States)
  Ms Ishita Vettiyadan (Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, United States)
  Mr Yuantong Li (Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, United States)
  Mr Xinyuan Gao (Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, United States)
  Ms Jaime DuFauchard (Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, United States)
  Ms Lakshmi Palivela (Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, United States)
  Mr John Stofko (Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, United States)
  
• Abstract:

  Background Information:
  Cost-effectiveness analysis (CEA) is a primary decision tool used to assess the value of new medical tests, treatments, devices, and public health programs. It has been noted that the Institute for Clinical and Economic Review (ICER), the leading health technology assessment (HTA) body in the US, tends to recommend substantial discounts for rare disease treatments. As ICER gains prominence in the US, this could potentially lead to delayed or denied access to treatment for rare disease patients. In addition, this could result in spillover effects and discourage future research and development efforts in rare diseases.
  
  Purpose:
  To review the current Institute for Clinical and Economic Review (ICER) Value Assessment Framework (VAF) used for biopharmaceutical health technology assessments, identify its limitations, and propose changes and improvements to inform the design and implementation of VAFs for rare diseases worldwide.
  
  Method:
  We conducted a systematized review to explore the criticisms and proposed modifications to ICER’s current VAF. Four databases, including PubMed, EconLit, CINAHL, and ABI/INFORM were searched on February 7-9th, 2023. Research articles were screened by six independent reviewers in two stages - title and abstract, and full-text screening. 2449 articles were retrieved for review after deduplication and 232 records remained after title and abstract screening. We also conducted an analysis of ICER’s health technology assessment reports published between January 2020 and April 2023 to compare ICER’s appraisal of rare and non-rare disease treatments. Primary research with two field experts supplemented our study.
  
  Results:
  1.ICER’s use of a narrow healthcare system perspective for its base-case CEA omits potentially important elements and therefore underestimates the value of treatments for rare diseases
  2.ICER uses 10,000 patients as the threshold for special consideration in its rare disease framework, far lower than that needed for Orphan Drug designation in the U.S. and stricter than the definition in most countries.
  3.ICER is more likely to report a dominant cost-effectiveness ratio for non-rare disease treatments compared to rare disease treatments.
  4.At least an 80% discount from the manufacturer list price is suggested for 35% of the rare disease treatments reviewed, compared to 10% for non-rare diseases.
  5.ICER’s quality ratings of the supporting evidence to assess comparative clinical effectiveness were comparable for non-rare and rare diseases.
  
  Conclusion:
  ICER’s value assessment framework (VAF) is inadequate when it is used to evaluate rare disease treatments. Emerging decision tools such as multi-criteria decision analysis (MCDA) and generalized risk-adjusted cost-effectiveness (GRACE) model address some of the fundamental issues of the traditional CEA. When designing an improved HTA model, stakeholders should:
  1) Heighten patient engagement and incorporate disease-specific patient-reported outcomes (PROs); 2) Consistently re-evaluate the treatment post-approval and update the value assessment when new evidence becomes available; and 3) Develop and incorporate more diverse scenario analyses, such as healthy years in total (HYT) as a health outcome, alternative discount rates, and post-launch price changes.
  
  Topic area: Social and administrative pharmacy