Abstract
Objective
Compared to traditional drugs, specialty drugs tend to be indicated for lower prevalence diseases. Our objective was to compare the potential population health benefits associated with specialty and traditional drugs in the year following product approval.
Methods
First, we created a dataset of estimates of incremental quality-adjusted life-year (QALY) gains and incremental life-year (LY) gains for US FDA-approved drugs (1999–2011) compared to standard of care at the time of approval identified from a literature search. Second, we categorized each drug as specialty or traditional. Third, for each drug we identified estimates of US disease prevalence for each pertinent indication. Fourth, in order to conservatively estimate the potential population health gains associated with each new drug in the year following its approval we multiplied the health gain estimate by 10% of the identified prevalence. Fifth, we used Mann–Whitney U tests to compare the population health gains for specialty and traditional drugs.
Results
We identified QALY gain estimates for 101 drugs, including 56 specialty drugs, and LY gain estimates for 50 drugs, including 34 specialty drugs. The median estimated population QALY gain in the year following approval for specialty drugs was 4200 (IQR = 27,000) and for traditional drugs was 694 (IQR = 24,400) (p = 0.245). The median estimated population LY gain in the year following approval for specialty drugs was 7250 (IQR = 39,200) and for traditional drugs was 2500 (IQR = 58,200) (p = 0.752).
Conclusions
Despite often being indicated for diseases of lower prevalence, we found a trend towards specialty drugs offering larger potential population health gains than traditional drugs, particularly when measured in terms of QALYs.
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Funding source
The study was funded by Pfizer. Researchers at Tufts Medical Center retained full control over question formulation, study selection, data extraction, data analyses, and interpretation of results.
Conflicts of interest
JC, TT, CW, MS, and PJN report receiving grant funding from Pfizer. PS and SKB report employment, including stock ownership/options, by Pfizer.
Author contributions
James Chambers: concept and design, analysis and interpretation of data, drafting of the manuscript, obtaining funding. Teja Thorat: statistical analysis, analysis and interpretation of data, drafting of the manuscript. Mark Salem: acquisition of data, administrative support, analysis and interpretation of data, drafting of the manuscript. Colby Wilkinson: acquisition of data, analysis and interpretation of data, statistical analysis. Prasun Subedi: concept and design, critical revision of the manuscript for important intellectual content, analysis and interpretation of data. Sachin Kamal-Bahl: concept and design, critical revision of the manuscript for important intellectual content, analysis and interpretation of data. Peter Neumann: supervision, obtaining funding, critical revision of the manuscript for important intellectual content.
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Chambers, J.D., Thorat, T., Wilkinson, C.L. et al. Estimating Population Health Benefits Associated with Specialty and Traditional Drugs in the Year Following Product Approval. Appl Health Econ Health Policy 15, 227–235 (2017). https://doi.org/10.1007/s40258-016-0291-9
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DOI: https://doi.org/10.1007/s40258-016-0291-9