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  • 1
    Online Resource
    Online Resource
    Academy of Managed Care Pharmacy ; 2017
    In:  Journal of Managed Care & Specialty Pharmacy Vol. 23, No. 2 ( 2017-02), p. 206-213
    In: Journal of Managed Care & Specialty Pharmacy, Academy of Managed Care Pharmacy, Vol. 23, No. 2 ( 2017-02), p. 206-213
    Type of Medium: Online Resource
    ISSN: 2376-0540 , 2376-1032
    Language: English
    Publisher: Academy of Managed Care Pharmacy
    Publication Date: 2017
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  • 2
    In: Journal of Medical Economics, Informa UK Limited, Vol. 15, No. 3 ( 2012-01), p. 454-464
    Type of Medium: Online Resource
    ISSN: 1369-6998 , 1941-837X
    Language: English
    Publisher: Informa UK Limited
    Publication Date: 2012
    detail.hit.zdb_id: 2156786-4
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  • 3
    Online Resource
    Online Resource
    Health Affairs (Project Hope) ; 2016
    In:  Health Affairs Vol. 35, No. 9 ( 2016-09), p. 1588-1594
    In: Health Affairs, Health Affairs (Project Hope), Vol. 35, No. 9 ( 2016-09), p. 1588-1594
    Type of Medium: Online Resource
    ISSN: 0278-2715 , 1544-5208
    Language: English
    Publisher: Health Affairs (Project Hope)
    Publication Date: 2016
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  • 4
    Online Resource
    Online Resource
    American Society of Hematology ; 2014
    In:  Blood Vol. 124, No. 21 ( 2014-12-06), p. 2608-2608
    In: Blood, American Society of Hematology, Vol. 124, No. 21 ( 2014-12-06), p. 2608-2608
    Abstract: Cancer survival has improved much since the 1970s, particularly for non-Hodgkin's lymphoma, breast, and colon cancer (Pulte, Arch Int Med 2008; Espey, Cancer 2007; Breen JNCI 2001). Yet some argue that newer treatments offer minimal survival gains at high costs (Kantarjian, Blood 2014). This motivates rigorous evaluation of the clinical and overall economic impacts of new cancer therapies. A recent study (Yin, Am J Manag Care 2012) showed that the introduction of tyrosine kinase inhibitors for chronic myeloid leukemia led to significant survival gains in the community, a value that far exceeded the cost of therapy. Using the methods employed by Yin et al., this study estimated the value of survival gains in myelodysplastic syndromes (MDS) attributable to the introduction of 3 therapies: azacitidine (2004), lenalidomide (2005), and decitabine (2006). While survival gains in clinical settings are well established (Fenaux, Lancet Onc 2009; Lubbert, J Clin Onc 2011; Fenaux, Blood 2011), survival gains in the community are unknown. We conducted 3 distinct analyses. First, we used multivariate Cox proportional hazards models to estimate the increase in survival associated with the introduction of new MDS therapies for 38,085 patients diagnosed from 2001–2011 in the Surveillance, Epidemiology, and End Results Program registries. The key variable in the hazard model was the post-2006 indicator variable, which equaled 1 in all years following 2006, the first year when all 3 therapies were available. The coefficient on the post-2006 indicator reflected the decline in the hazard rate associated with the introduction of the 3 therapies, relative to the years prior to their introduction (2001–2003). We estimated that the introduction of these 3 therapies is associated with a hazard ratio of 0.901 (p 〈 0.10). Second, utilization of the 3 new therapies from 2006 to 2012 was estimated with the Optum Touchstone commercial claims database (Optum). Approximately 25% of MDS patients had claims for at least 1 of the 3 novel treatments consistently over this period, resulting in a hazard ratio of 0.901 and an overall increase in median survival from 33 to 57.5 months, conditional on treatment observed in the community (Fig. 1). Figure 1 Figure 1. Finally, we calibrated an existing economic model (Becker et al., Am Econ Rev 2005) to value survival gains. The annual value of survival gains associated with innovative treatmentsroughly $208,000 per yearequaled the estimated amount a patient would be willing to pay for the higher survival profile associated with the new therapies. Table 1 compares the value of survival gains to costs, where annual treatment costs of the new therapies from 2006–2012 are estimated using Optum. The value of survival gains is estimated to exceed the cost of therapy, with the annual and lifetime net benefit to patients of $68,200 and $238,700, respectively. The total present discounted value of the survival gains and costs considers future MDS incident cohorts together with the current cohort, and accounts for patent expiry (generic versions of azacitidine and decitabine became available in 2013). Assuming that the patent on lenalidomide expires in 2020, and that costs fall to 10% of current prices post expiry, we estimate that 85% (= $86.5B / $101.5B) of the total value accrues to MDS patients, and 15% accrues to manufacturers. Table 1.Value of Survival gain for MDS patientsAnnual Value per PatientLifetime PDV per PatientPDV for all Current and Future Cohorts(1)(2)(3)Value of survival gain$208,000$728,000$101.5 billionCost$139,100$489,300$15.0 billionPatient net benefit$68,200$238,700$86.5 billion Notes: MDS indicates myelodysplastic syndromes. All values reported in 2013$. Column (1) reports the per-patient annual value, cost, and net benefit of treatment to patients. Column (2) reports the present discounted value (PDV) over a patient's lifetime (estimated at 3.5 years on average). Column (4) reports PDV for all current and future incident cohorts. We estimate the incidence cohort size is 14,650, and assume a 3% annual discount rate. This study measured the value of survival gains attributable to the introduction of 3 novel therapies for MDS and found significant benefits from improved survival. For current and future MDS patients, these 3 therapies will generate over $100B in value due to survival gains, of which the vast majority85%will accrue to patients. Disclosures MacEwan: Celgene: Consultancy, Research Funding. Yin:Celgene: Consultancy, Research Funding. Khan:Celgene: Employment, Equity Ownership, Leadership - Vice President Other. Kaura:Celgene: Employment, Equity Ownership.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
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    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2014
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    detail.hit.zdb_id: 80069-7
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  • 5
    In: Blood, American Society of Hematology, Vol. 114, No. 22 ( 2009-11-20), p. 3881-3881
    Abstract: Abstract 3881 Poster Board III-817 Background For MM patients with malignant bone lesions (BM), SREs including pathologic fracture, spinal cord compression, hypercalcemia of malignancy, and radiotherapy and/or surgery to bone are associated with significant morbidity and mortality and reduced quality of life. ZOL is an IV bisphosphonate (BP) proven to reduce and delay incidence of SREs in several tumor types. This study was designed to assess the benefit of long-term ZOL use in a real-life setting. Methods Claims-based analysis of commercial and Medicare data from a large US managed care plan and a 45 health-plan database was used to evaluate SRE rates, time from BM to 1st SRE, and mortality in patients treated with ZOL or no IV BP therapy. Patients older than 18 years with MM and BM diagnosed between Jan 2001 and Dec 2006 were included. Treatment Persistency was defined as the absence of a 〉 45 day gap between ZOL administrations. Continuous enrollment in the health plan for 6 months before and no prior evidence of BM or IV BP use were required. When assessing mortality, patients with a date of death less than 30 days following index date were excluded. Patients were followed until they disenrolled from the plan or to the end of the study's follow-up period. In this study, SREs were defined as evidence of pathologic fracture, spinal cord compression, and radiotherapy and/or surgery to bone. Results The study sample included 1,655 Patients with a mean age of 61.7 ± 11.9 years; approx. 64% were treated with ZOL and 36% with no IV BP. Incidences of SREs and mortality rates were both greater in the no IV BP group (incidence rate ratio [IRR] = 1.58; p-value 〈 0.001 and mortality rate = 1.71; p-value=0.0234) vs. the ZOL groups. Longer persistency with ZOL was associated with a lower risks of first SRE compared with no IV BP (trend test p-value=0.0025) [TABLE 1]. Conclusions This study showed that in MM Patients with BM, ZOL use was associated with a lower risk of SREs, including fractures, and lower mortality rates. In addition, longer persistence was found to be associated with lower risk of SRE. Disclosures: Kaura: Novartis: Employment, Equity Ownership. Perez:Novartis: Employment, Equity Ownership.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
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    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2009
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    detail.hit.zdb_id: 80069-7
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  • 6
    Online Resource
    Online Resource
    American Society of Clinical Oncology (ASCO) ; 2015
    In:  Journal of Clinical Oncology Vol. 33, No. 15_suppl ( 2015-05-20), p. e19536-e19536
    In: Journal of Clinical Oncology, American Society of Clinical Oncology (ASCO), Vol. 33, No. 15_suppl ( 2015-05-20), p. e19536-e19536
    Type of Medium: Online Resource
    ISSN: 0732-183X , 1527-7755
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    Language: English
    Publisher: American Society of Clinical Oncology (ASCO)
    Publication Date: 2015
    detail.hit.zdb_id: 2005181-5
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  • 7
    Online Resource
    Online Resource
    Informa UK Limited ; 2012
    In:  Current Medical Research and Opinion Vol. 28, No. 7 ( 2012-07-01), p. 1119-1127
    In: Current Medical Research and Opinion, Informa UK Limited, Vol. 28, No. 7 ( 2012-07-01), p. 1119-1127
    Type of Medium: Online Resource
    ISSN: 0300-7995 , 1473-4877
    Language: English
    Publisher: Informa UK Limited
    Publication Date: 2012
    detail.hit.zdb_id: 2034331-0
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  • 8
    In: ClinicoEconomics and Outcomes Research, Informa UK Limited
    Type of Medium: Online Resource
    ISSN: 1178-6981
    Language: English
    Publisher: Informa UK Limited
    Publication Date: 2015
    detail.hit.zdb_id: 2520698-9
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  • 9
    In: Blood, American Society of Hematology, Vol. 126, No. 23 ( 2015-12-03), p. 3294-3294
    Abstract: Introduction. A previous study of patients (pts) with newly-diagnosed multiple myeloma (NDMM) showed that initial high monthly total costs declined over time until returning near initial levels upon pts advancing to later lines of treatment (Tx) at disease progression (Arikian, CMRO 2015). However, the longer-term economic implications of the choice of first-line Tx, regardless of subsequent Tx regimens, have not been described. We utilized an intent-to-treat approach to further evaluate and compare the total costs of NDMM patient cohorts initiated on Lenalidomide (LEN) or Bortezomib (BORT)-based Tx and followed these patients over 54 months. Methods: A retrospective analysis was performed using a large US medical and pharmacy claims database, covering 〉 25 million commercial and Medicare lives annually. NDMM patients were defined as having ≥ 2 outpatient claims or ≥ 1 inpatient medical claim with a diagnosis of MM (ICD-9-CM code 203.0X), with the first such claim used to define the index date. Inclusion criteria required ≥ 12 months' pre-index and ≥ 6 months' post-index continuous enrollment from 2006 - 2013, and evidence of initiation of a subsequent line of therapy. Pts with claims for stem cell transplantation (SCT) or receiving concurrent LEN plus BORT as first-line Tx were excluded. The analysis focused on cohorts of NDMM pts receiving LEN or BORT-based first line Tx, who progressed to one or more subsequent lines of Tx, using time to next therapy (TTNT) as a proxy for progression. Pts receiving LEN or BORT-based first line Tx were randomly matched 1:1 on age (+/- 3 years at index), sex, baseline Charlson comorbidity score (+/- 1), and presence of renal disease. Using methods similar to those described by Gaultney(J Clin Pharm Ther, 2013), pts' average monthly costs and standard errors were determined, including medical and pharmacy costs, and total cost patterns were calculated from first line Tx initiation until the end of patient eligibility or 54 months. Results: 1,181 NDMM pts receiving LEN (N=444) or BORT-based (N=737) first line Tx with complete data available through initiation of subsequent line of Tx were identified. After matching, 856 NDMM patients remained (428 LEN, 428 BORT). Monthly total direct medical plus pharmacy costs for these pts were in excess of $12,000 at initiation. Total monthly costs declined quarterly for each cohort until the median TTNT was reached (20 months for BORT; 37 months for LEN). As increasing numbers of pts in each cohort then advanced to subsequent lines of therapy, costs of those patients began to offset continued cost declines for pts still on first line of therapy. Over the full 54 month follow-up period, total monthly costs averaged $8,324 (SE = $370.30) for pts initiated on LEN vs. $10,728 (SE = $500.55) for pts initiated on BORT (p-value 〈 0.001). Medical costs represented 54-57% of total monthly costs for each cohort. In each cohort, the most common Tx option upon disease progression was to switch to the other regimen. Conclusions: For a matched population of NDMM pts followed continuously for 54 months, initiation on LEN- versus BORT-based Tx without SCT was associated with an average of $2,404 lower monthly direct total costs. Following initiation of first-line therapy, total monthly costs per pt declined quarterly until initiation of the next line of Tx. Cumulative costs over the 54-month period were over $120,000 lower per pt in favor of the LEN cohort, influenced by more rapid advancement to subsequent Tx in the BORT cohort, and the associated higher costs. Based on second-line Tx patterns, this analysis also suggests that a sequence of LEN in first line followed by BORT in second line is associated with lower costs over time compared to the reverse sequence. Due to the nature of retrospective claims, some underlying differences may remain between the two cohorts even after matching. Figure 1. Direct monthly costs (medical and pharmacy) for LEN- and BORT-initiated patients and savings attributed to LEN-initiated patients Figure 1. Direct monthly costs (medical and pharmacy) for LEN- and BORT-initiated patients and savings attributed to LEN-initiated patients Disclosures Arikian: Genesis Research: Consultancy. Binder:Celgene Corporation: Employment, Equity Ownership. Gibson:Celgene Corporation: Employment, Equity Ownership. Hu:Celgene Corporation: Employment, Equity Ownership, Patents & Royalties. Nagarwala:Celgene Corporation: Employment. Hussein:Celgene Corporation: Employment, Equity Ownership. Corvino:Genesis Research LLC: Consultancy. Lee:Genesis Research LLC: Consultancy. Surinach:Genesis Research LLC: Consultancy. Kaura:Celgene Corporation: Employment. Usmani:Onyx: Consultancy, Honoraria, Research Funding; Celgene Corporation: Consultancy, Honoraria; Janssen: Research Funding.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
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    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2015
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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  • 10
    Online Resource
    Online Resource
    American Society of Hematology ; 2014
    In:  Blood Vol. 124, No. 21 ( 2014-12-06), p. 736-736
    In: Blood, American Society of Hematology, Vol. 124, No. 21 ( 2014-12-06), p. 736-736
    Abstract: Introduction: The Orphan Drug Act (ODA) of 1983 established incentives for the development of drugs that treat rare or orphan diseases. The ODA targets therapies for diseases affecting fewer than 200,000 patients in the US. As a consequence, orphan drug development was stimulated and therapies have been developed to treat a variety of previously underserved conditions including acute myeloid leukemia, chronic lymphocytic leukemia, multiple myeloma, myelodysplastic syndromes, Gaucher disease and Hunter syndrome. Between 1973 to 1983, prior to the ODA, less than 10 drugs for orphan conditions were available to patients, while in 2013 the FDA website listed more than 400 approved orphan drugs (this includes both new molecular entities and supplemental applications/indications). Due to the perceived high therapy costs and therefore potential impact on payer's drug budgets, orphan drugs are facing increasing scrutiny from payers. The objective of this study was to estimate the total annual expenditures on orphan drugs in the US from 2007 to 2013, and to evaluate orphan drug expenditures as a percentage of total pharmaceutical expenditures. Methods: A list of drugs which have been approved by the FDA and given orphan status in the US since the establishment of the ODA through the end of 2013 was generated from the US FDA Orphan Drug Product designation website (N=366 unique products). Only branded products (N=357) were considered in this analysis. The IMS MIDAS database, the gold standard for audited biopharmaceutical sales, was used to assess orphan drug and total drug expenditures. The MIDAS database integrates and extends the IMS National Prescription Audits, which detail estimated sales by product and therapy class through retail and non-retail channels at the unit of the product pack level. The analysis focused on orphan drugs with expenditures measured between 2007 and 2013 in the MIDAS database. Only products with a single orphan indication or with multiple indications which were all orphan were included in the analysis (N=303). Total orphan drug expenditures were calculated annually from 2007 to 2013 and further reported as a proportion of total annual pharmaceutical drug expenditures. All expenditures were adjusted to 2014 USD using the Bureau of Economic Analysis GDP deflator. Expenditures for orphan drugs with cancer indications were evaluated as a subset of all orphan drugs. Results: Between 2007 and 2013, expenditures were measured for a final N=266 branded orphan drugs in the MIDAS database; 37 out of 303 were either not present or had no sales in MIDAS. Orphan drug expenditures totaled $15.0 billion (B) in 2007 and $25.6B in 2013, representing 4.8% to 7.6% of total U.S. pharmaceutical drug expenditures in 2007 and 2013, respectively (Figure 1). There were 74 (27.8%) orphan drugs identified with an orphan cancer indication with expenditures representing 26.7% (2007) and 34.8% (2013) of total orphan drug expenditures. Over the same period (2007–2013), a total of 145 orphan drugs were granted approval (both new molecular entities and supplemental applications/indications). Conclusions: Although the ODA has led to an increase in the number of approved orphan drugs and the annual expenditures on orphan drugs, the overall budget impact of orphan drugs is relatively small and has remained fairly stable as a proportion of total pharmaceutical expenditures. Concerns that growth in orphan drug expenditures may lead to unsustainable drug expenditures do not appear to be justified. Figure 1 Proportion of Total US Drug Expenditures, 2007-2013: Orphan and Non-Orphan Drugs Figure 1. Proportion of Total US Drug Expenditures, 2007-2013: Orphan and Non-Orphan Drugs Disclosures Divino: IMS Health: Employment. Dekoven:IMS Health: Employment. Wang:MKTXS: Employment. Kleinrock:IMS Health: Employment. Harvey:Celgene, Millennium, Onyx and Novartis: Research Funding. Wade:IMS Health: Employment. Kaura:Celgene: Employment, Equity Ownership.
    Type of Medium: Online Resource
    ISSN: 0006-4971 , 1528-0020
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    Language: English
    Publisher: American Society of Hematology
    Publication Date: 2014
    detail.hit.zdb_id: 1468538-3
    detail.hit.zdb_id: 80069-7
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