Comparing Predicted vs. Real-World Spending in the United States for Single-Administration Cell and Gene Therapies
Author(s)
Natasha Kulkarni, BS1, John Jarvis, MBA2, Cheryl Steward, MPA2, Lufei Tu, MS2, Marjorie Crowell, MPA2, Alexa C. Klimchak, MA1, Kathy Gooch, PhD1;
1Sarepta Therapeutics, Inc., Cambridge, MA, USA, 2Medicus Economics, LLC, Milton, MA, USA
1Sarepta Therapeutics, Inc., Cambridge, MA, USA, 2Medicus Economics, LLC, Milton, MA, USA
Presentation Documents
OBJECTIVES: With a growing number of single-administration cell and gene therapies (CGTs) in development, there has been increasing focus on the US national spending for these treatments. US media outlets have expressed concern that this spending will overwhelm the US healthcare system, citing analytical models predicting high national spending for CGTs. To assess the accuracy of these models, the present analysis sought to identify published predictions of US spending across CGTs and compare with real-world spending to date.
METHODS: A targeted literature review (TLR) was conducted in June 2023 to identify analytical predictions of US national spending across all single-administration CGTs. Annual US net sales data were gathered from public financial records for the 11 single-administration CGTs approved by the US Food and Drug Administration as of December 2022.
RESULTS: Two published analytical models were identified which predicted US national spending across CGTs over multiple years. Models predicted spending between $13.6 billion and $15.4 billion in 2022 alone. Real-world US net sales for approved CGTs totaled $2.2 billion in 2022, approximately 15% of predictions. Analytical models predicted spending across many treatment areas (range: 57 to 176 treatment areas), including many rare and ultra-rare diseases. Model methods were reviewed, and four key factors were identified which may have contributed to overestimation of real-world spending: 1) fewer/delayed CGT approvals; 2) smaller eligible patient populations; 3) lower market penetration and/or slower uptake; and 4) discrepancies in treatment price. Despite predicting similar levels of spending, the models differed significantly on analytical decisions across these factors, highlighting the complexity of these prediction models.
CONCLUSIONS: Prediction models are important tools to estimate the potential budget impact of therapies on future healthcare spending. However, this analysis highlights model assumptions that were most likely to result in substantial differences between predicted and actual spending for CGTs.
METHODS: A targeted literature review (TLR) was conducted in June 2023 to identify analytical predictions of US national spending across all single-administration CGTs. Annual US net sales data were gathered from public financial records for the 11 single-administration CGTs approved by the US Food and Drug Administration as of December 2022.
RESULTS: Two published analytical models were identified which predicted US national spending across CGTs over multiple years. Models predicted spending between $13.6 billion and $15.4 billion in 2022 alone. Real-world US net sales for approved CGTs totaled $2.2 billion in 2022, approximately 15% of predictions. Analytical models predicted spending across many treatment areas (range: 57 to 176 treatment areas), including many rare and ultra-rare diseases. Model methods were reviewed, and four key factors were identified which may have contributed to overestimation of real-world spending: 1) fewer/delayed CGT approvals; 2) smaller eligible patient populations; 3) lower market penetration and/or slower uptake; and 4) discrepancies in treatment price. Despite predicting similar levels of spending, the models differed significantly on analytical decisions across these factors, highlighting the complexity of these prediction models.
CONCLUSIONS: Prediction models are important tools to estimate the potential budget impact of therapies on future healthcare spending. However, this analysis highlights model assumptions that were most likely to result in substantial differences between predicted and actual spending for CGTs.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR87
Topic
Health Policy & Regulatory
Topic Subcategory
Public Spending & National Health Expenditures
Disease
SDC: Rare & Orphan Diseases