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Special report

What might have happened: the impact of interrupting entry of innovative drugs on disease outcomes in the United States

, , , , &
Pages 529-541 | Received 04 Oct 2021, Accepted 25 Jan 2022, Published online: 21 Feb 2022

Figures & data

Figure 1. Ischemic heart disease.

Abbreviations: IHD, ischemic heart disease; TPA, tissue plasminogen activator; US, United States; YLL, years of life lost.*Innovations were identified from publicly available information and the significance of their impact on outcomes was confirmed through interviews with clinical specialists.†YLLs per individual with IHD in the United States derived from the Global Burden of Disease database [Citation20].‡Age-adjusted IHD mortality rates per 1,000 persons (standardized U.S. population) from the Atherosclerosis Risk in Communities study [Citation31]. Other studies identified in the literature review are presented in Appendix B.
Figure 1. Ischemic heart disease.

Figure 2. Lung cancer.

Abbreviations: EGFR, epidermal growth factor receptor; SEER, surveillance, epidemiology, and end results; US, United States; YLL, years of life lost.*Innovations were identified from publicly available information and the significance of their impact on outcomes was confirmed through interviews with clinical specialists.†YLLs per individual with lung cancer in the United States derived from the Global Burden of Disease database [Citation20].‡5-year survival rates for invasive lung and bronchus cancer at diagnosis relative to the age-adjusted general population from SEER Cancer Statistics [Citation32]. Other studies identified in the literature review are presented in Appendix B.
Figure 2. Lung cancer.

Figure 3. Breast cancer.

Abbreviations: HER2, human epidermal growth factor 2; US, United States; YLL, years of life lost.*Innovations were identified from publicly available information and the significance of their impact on outcomes was confirmed through interviews with clinical specialists.†YLLs per individual with breast cancer in the US derived from the Global Burden of Disease database [Citation20].‡Median overall survival estimates for de novo and recurrent metastatic breast cancer from a multicountry systematic literature review and meta-regression analysis [Citation33]. Other studies identified in the literature review are presented in Appendix B.
Figure 3. Breast cancer.

Figure 4. Human immunodeficiency virus.

Abbreviations: HAART, highly active antiretroviral therapy; HIV, human immunodeficiency virus; US, United States; YLL, years of life lost.*Innovations were identified from publicly available information and the significance of their impact on outcomes was confirmed through interviews with clinical specialists.†YLLs per individual with HIV infection in the United States derived from the Global Burden of Disease database [Citation20].‡Remaining life expectancy at age 20 years for HIV-infected and HIV-uninfected individuals estimated using age-adjusted mortality rates from Kaiser Permanente data [Citation34]. Other studies identified in the literature review are presented in Appendix B.
Figure 4. Human immunodeficiency virus.

Figure 5. Type 2 diabetes mellitus.

Abbreviations: ESRD, end-stage renal disease; GLP-1, glucagon-like peptide 1; TZD, thiazolidinediones; T2DM, type 2 diabetes mellitus; US, United States; YLL, years of life lost.*Innovations were identified from publicly available information and the significance of their impact on outcomes was confirmed through interviews with clinical specialists.†YLLs per individual with T2DM in the United States derived from the Global Burden of Disease database [Citation20].‡Age-adjusted incidence rates for diabetes-related ESRD among those with diabetes from the U.S. Renal Data System, the National Health Interview Survey (1990–2006), and the Behavioral Risk Factor Surveillance System (2000–2014) [Citation35,Citation36]. Other studies identified in the literature review are presented in Appendix B.
Figure 5. Type 2 diabetes mellitus.

Figure 6. Rheumatoid arthritis.

Abbreviations: HAQ, health assessment questionnaire; QALY, quality-adjusted life-year; RA, rheumatoid arthritis; TNF, tumor necrosis factor; US, United States; YLL, years of life lost.*Innovations were identified from publicly available information and the significance of their impact on outcomes was confirmed through interviews with clinical specialists.†Mean HAQ score for individuals with RA in the United States from the Arthritis, Rheumatism and Aging Medical Information System database [Citation29].‡Discharges per 100,000 people with RA for total knee and hip arthroplasty using data from the California Office of Statewide Health Planning [Citation37]. Other studies identified in the literature review are presented in Appendix B.
Figure 6. Rheumatoid arthritis.
Supplemental material

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Data availability statement

RA functional status from: Krishnan E, Lingala B, Bruce B, et al. Disability in rheumatoid arthritis in the era of biological treatments. Ann Rheum Dis. 2012;71:213–218.

GBD data from: Global Health Data Exchange. Global burden of disease study 2017 (GBD 2017) data resources. BD results tool. 23 July 2019[cited 23 April 2021]. Available from: http://ghdx.healthdata.org/gbd-2017