Skip to main content
Erschienen in: Applied Health Economics and Health Policy 2/2017

01.04.2017 | Original Research Article

Estimating Population Health Benefits Associated with Specialty and Traditional Drugs in the Year Following Product Approval

verfasst von: James D. Chambers, Teja Thorat, Colby L. Wilkinson, Mark Salem, Prasun Subedi, Sachin J. Kamal-Bahl, Peter J. Neumann

Erschienen in: Applied Health Economics and Health Policy | Ausgabe 2/2017

Einloggen, um Zugang zu erhalten

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.
Anhänge
Nur mit Berechtigung zugänglich
Literatur
1.
Zurück zum Zitat Gleason PP, Alexander GC, Starner CI, Ritter ST, Van Houten HK, Gunderson BW, et al. Health plan utilization and costs of specialty drugs within 4 chronic conditions. J Manag Care Pharm. 2013;19(7):542–8.PubMed Gleason PP, Alexander GC, Starner CI, Ritter ST, Van Houten HK, Gunderson BW, et al. Health plan utilization and costs of specialty drugs within 4 chronic conditions. J Manag Care Pharm. 2013;19(7):542–8.PubMed
2.
Zurück zum Zitat Goldman DP, Joyce GF, Lawless G, Crown WH, Willey V. Benefit design and specialty drug use. Health Aff (Millwood). 2006;25(5):1319–31.CrossRef Goldman DP, Joyce GF, Lawless G, Crown WH, Willey V. Benefit design and specialty drug use. Health Aff (Millwood). 2006;25(5):1319–31.CrossRef
3.
Zurück zum Zitat Sullivan SD. The promise of specialty pharmaceuticals: are they worth the price? J Manag Care Pharm. 2008;14(4 Suppl):S3–6.PubMed Sullivan SD. The promise of specialty pharmaceuticals: are they worth the price? J Manag Care Pharm. 2008;14(4 Suppl):S3–6.PubMed
5.
Zurück zum Zitat Chambers JD, Thorat T, Pyo J, Chenoweth M, Neumann PJ. Despite high costs, specialty drugs may offer value for money comparable to that of traditional drugs. Health Aff (Millwood). 2014;33(10):1751–60.CrossRef Chambers JD, Thorat T, Pyo J, Chenoweth M, Neumann PJ. Despite high costs, specialty drugs may offer value for money comparable to that of traditional drugs. Health Aff (Millwood). 2014;33(10):1751–60.CrossRef
6.
Zurück zum Zitat Cutler DM, McClellan M. Is technological change in medicine worth it? Health Aff (Millwood). 2001;20(5):11–29.CrossRef Cutler DM, McClellan M. Is technological change in medicine worth it? Health Aff (Millwood). 2001;20(5):11–29.CrossRef
7.
Zurück zum Zitat Cutler DM, Long G, Berndt ER, Royer J, Fournier AA, Sasser A, et al. The value of antihypertensive drugs: a perspective on medical innovation. Health Aff (Millwood). 2007;26(1):97–110.CrossRef Cutler DM, Long G, Berndt ER, Royer J, Fournier AA, Sasser A, et al. The value of antihypertensive drugs: a perspective on medical innovation. Health Aff (Millwood). 2007;26(1):97–110.CrossRef
8.
Zurück zum Zitat Lichtenberg FR. The quality of medical care, behavioral risk factors, and longevity growth. Int J Health Care Finance Econ. 2011;11(1):1–34.CrossRefPubMed Lichtenberg FR. The quality of medical care, behavioral risk factors, and longevity growth. Int J Health Care Finance Econ. 2011;11(1):1–34.CrossRefPubMed
9.
Zurück zum Zitat Lichtenberg FR. Contribution of pharmaceutical innovation to longevity growth in Germany and France, 2001–7. Pharmacoeconomics. 2012;30(3):197–211.CrossRefPubMed Lichtenberg FR. Contribution of pharmaceutical innovation to longevity growth in Germany and France, 2001–7. Pharmacoeconomics. 2012;30(3):197–211.CrossRefPubMed
10.
Zurück zum Zitat Lichtenberg FR. The effect of using newer drugs on admissions of elderly Americans to hospitals and nursing homes: state-level evidence from 1997 to 2003. Pharmacoeconomics. 2006;24(Suppl 3):5–25.CrossRefPubMed Lichtenberg FR. The effect of using newer drugs on admissions of elderly Americans to hospitals and nursing homes: state-level evidence from 1997 to 2003. Pharmacoeconomics. 2006;24(Suppl 3):5–25.CrossRefPubMed
11.
Zurück zum Zitat Lichtenberg FR. The impact of pharmaceutical innovation on premature cancer mortality in Switzerland, 1995–2012. Eur J Health Econ. 2016;17(7):833–54.CrossRefPubMed Lichtenberg FR. The impact of pharmaceutical innovation on premature cancer mortality in Switzerland, 1995–2012. Eur J Health Econ. 2016;17(7):833–54.CrossRefPubMed
12.
Zurück zum Zitat Lichtenberg FR. The impact of new (orphan) drug approvals on premature mortality from rare diseases in the United States and France, 1999–2007. Eur J Health Econ. 2013;14(1):41–56.CrossRefPubMed Lichtenberg FR. The impact of new (orphan) drug approvals on premature mortality from rare diseases in the United States and France, 1999–2007. Eur J Health Econ. 2013;14(1):41–56.CrossRefPubMed
13.
Zurück zum Zitat Lichtenberg FR. Has medical innovation reduced cancer mortality? CESifo Econ Stud. 2014;60(1):135–77.CrossRef Lichtenberg FR. Has medical innovation reduced cancer mortality? CESifo Econ Stud. 2014;60(1):135–77.CrossRef
14.
Zurück zum Zitat Chambers JD, Thorat T, Chenoweth MD, Neumann PJ. Fast-tracking innovation: a scorecard for the FDA’s expedited review process. Value Health. 2015;18(7):A517.CrossRefPubMed Chambers JD, Thorat T, Chenoweth MD, Neumann PJ. Fast-tracking innovation: a scorecard for the FDA’s expedited review process. Value Health. 2015;18(7):A517.CrossRefPubMed
15.
Zurück zum Zitat Caremark CVS. Comprehensive specialty pharmacy drug list. Woonsocket: CVS Caremark; 2015. Caremark CVS. Comprehensive specialty pharmacy drug list. Woonsocket: CVS Caremark; 2015.
16.
Zurück zum Zitat Scripts Express. Specialty drug list by disease state. St. Louis: Express Scripts Holding Company; 2015. Scripts Express. Specialty drug list by disease state. St. Louis: Express Scripts Holding Company; 2015.
17.
Zurück zum Zitat Bell CM, Urbach DR, Ray JG, Bayoumi A, Rosen AB, Greenberg D, et al. Bias in published cost effectiveness studies: systematic review. BMJ. 2006;332(7543):699–703.CrossRefPubMedPubMedCentral Bell CM, Urbach DR, Ray JG, Bayoumi A, Rosen AB, Greenberg D, et al. Bias in published cost effectiveness studies: systematic review. BMJ. 2006;332(7543):699–703.CrossRefPubMedPubMedCentral
18.
Zurück zum Zitat Garattini L, Koleva D, Casadei G. Modeling in pharmacoeconomic studies: funding sources and outcomes. Int J Technol Assess Health Care. 2010;26(3):330–3.CrossRefPubMed Garattini L, Koleva D, Casadei G. Modeling in pharmacoeconomic studies: funding sources and outcomes. Int J Technol Assess Health Care. 2010;26(3):330–3.CrossRefPubMed
21.
22.
Zurück zum Zitat Board of Governors of the Federal Reserve System. Foreign exchange rates—H.10. 10-3-2016. Accessed 5 Oct 2016. Board of Governors of the Federal Reserve System. Foreign exchange rates—H.10. 10-3-2016. Accessed 5 Oct 2016.
24.
Zurück zum Zitat Kent DM, Hayward RA. Limitations of applying summary results of clinical trials to individual patients: the need for risk stratification. JAMA. 2007;298(10):1209–12.CrossRefPubMed Kent DM, Hayward RA. Limitations of applying summary results of clinical trials to individual patients: the need for risk stratification. JAMA. 2007;298(10):1209–12.CrossRefPubMed
25.
Zurück zum Zitat Garjon FJ, Azparren A, Vergara I, Azaola B, Loayssa JR. Adoption of new drugs by physicians: a survival analysis. BMC Health Serv Res. 2012;12:56.CrossRefPubMedPubMedCentral Garjon FJ, Azparren A, Vergara I, Azaola B, Loayssa JR. Adoption of new drugs by physicians: a survival analysis. BMC Health Serv Res. 2012;12:56.CrossRefPubMedPubMedCentral
26.
Zurück zum Zitat Chambers JD, Thorat T, Pyo J, Neumann PJ. The lag from FDA approval to published cost-utility evidence. Expert Rev Pharmacoecon Outcomes Res. 2015;15(3):399–402.CrossRefPubMed Chambers JD, Thorat T, Pyo J, Neumann PJ. The lag from FDA approval to published cost-utility evidence. Expert Rev Pharmacoecon Outcomes Res. 2015;15(3):399–402.CrossRefPubMed
27.
Zurück zum Zitat Wisloff T, Hagen G, Hamidi V, Movik E, Klemp M, Olsen JA. Estimating QALY gains in applied studies: a review of cost-utility analyses published in 2010. Pharmacoeconomics. 2014;32(4):367–75.CrossRefPubMedPubMedCentral Wisloff T, Hagen G, Hamidi V, Movik E, Klemp M, Olsen JA. Estimating QALY gains in applied studies: a review of cost-utility analyses published in 2010. Pharmacoeconomics. 2014;32(4):367–75.CrossRefPubMedPubMedCentral
28.
Zurück zum Zitat Neumann PJ, Greenberg D, Olchanski NV, Stone PW, Rosen AB. Growth and quality of the cost-utility literature, 1976–2001. Value Health. 2005;8(1):3–9.CrossRefPubMed Neumann PJ, Greenberg D, Olchanski NV, Stone PW, Rosen AB. Growth and quality of the cost-utility literature, 1976–2001. Value Health. 2005;8(1):3–9.CrossRefPubMed
29.
Zurück zum Zitat Neumann PJ, Fang CH, Cohen JT. 30 years of pharmaceutical cost-utility analyses: growth, diversity and methodological improvement. Pharmacoeconomics. 2009;27(10):861–72.CrossRefPubMed Neumann PJ, Fang CH, Cohen JT. 30 years of pharmaceutical cost-utility analyses: growth, diversity and methodological improvement. Pharmacoeconomics. 2009;27(10):861–72.CrossRefPubMed
31.
Zurück zum Zitat Garau M, Shah KK, Mason AR, Wang Q, Towse A, Drummond MF. Using QALYs in cancer: a review of the methodological limitations. Pharmacoeconomics. 2011;29(8):673–85.CrossRefPubMed Garau M, Shah KK, Mason AR, Wang Q, Towse A, Drummond MF. Using QALYs in cancer: a review of the methodological limitations. Pharmacoeconomics. 2011;29(8):673–85.CrossRefPubMed
32.
Zurück zum Zitat Williams A. QALYS and ethics: a health economist’s perspective. Soc Sci Med. 1996;43(12):1795–804.CrossRefPubMed Williams A. QALYS and ethics: a health economist’s perspective. Soc Sci Med. 1996;43(12):1795–804.CrossRefPubMed
33.
Zurück zum Zitat Bach PB, Pearson SD. Payer and policy maker steps to support value-based pricing for drugs. JAMA. 2015;314(23):2503–4. Bach PB, Pearson SD. Payer and policy maker steps to support value-based pricing for drugs. JAMA. 2015;314(23):2503–4.
34.
Zurück zum Zitat Neumann PJ, Cohen JT. Measuring the value of prescription drugs. N Engl J Med. 2015;373(27):2595–7. Neumann PJ, Cohen JT. Measuring the value of prescription drugs. N Engl J Med. 2015;373(27):2595–7.
35.
Zurück zum Zitat Najafzadeh M, Andersson K, Shrank WH, Krumme AA, Matlin OS, Brennan T, et al. Cost-effectiveness of novel regimens for the treatment of hepatitis C virus. Ann Intern Med. 2015;162(6):407–19.CrossRefPubMed Najafzadeh M, Andersson K, Shrank WH, Krumme AA, Matlin OS, Brennan T, et al. Cost-effectiveness of novel regimens for the treatment of hepatitis C virus. Ann Intern Med. 2015;162(6):407–19.CrossRefPubMed
36.
Zurück zum Zitat Schulman KA, Balu S, Reed SD. Specialty pharmaceuticals for hyperlipidemia-impact on insurance premiums. N Engl J Med. 2015;373(17):1591–3.CrossRefPubMed Schulman KA, Balu S, Reed SD. Specialty pharmaceuticals for hyperlipidemia-impact on insurance premiums. N Engl J Med. 2015;373(17):1591–3.CrossRefPubMed
37.
Zurück zum Zitat Brennan TA, Wilson JM. The special case of gene therapy pricing. Nat Biotechnol. 2014;32(9):874–6.CrossRefPubMed Brennan TA, Wilson JM. The special case of gene therapy pricing. Nat Biotechnol. 2014;32(9):874–6.CrossRefPubMed
Metadaten
Titel
Estimating Population Health Benefits Associated with Specialty and Traditional Drugs in the Year Following Product Approval
verfasst von
James D. Chambers
Teja Thorat
Colby L. Wilkinson
Mark Salem
Prasun Subedi
Sachin J. Kamal-Bahl
Peter J. Neumann
Publikationsdatum
01.04.2017
Verlag
Springer International Publishing
Erschienen in
Applied Health Economics and Health Policy / Ausgabe 2/2017
Print ISSN: 1175-5652
Elektronische ISSN: 1179-1896
DOI
https://doi.org/10.1007/s40258-016-0291-9

Weitere Artikel der Ausgabe 2/2017

Applied Health Economics and Health Policy 2/2017 Zur Ausgabe