The value of survival gains in myelodysplastic syndromes.

Objectives: To measure the value of survival gains attributable to the introduction of 3 novel therapies for myelodysplastic syndromes (MDS).

Study Design: Retrospective study of patients diagnosed with MDS in the Surveillance, Epidemiology and End Results Program (SEER) registry, clinical trial evidence for MDS therapies, and claims data.

Methods: We used multivariate Cox proportional hazards models to estimate the increase in survival associated with the introduction of the 3 new therapies for patients diagnosed with MDS from 2001 to 2011 in the SEER cancer registry.

The Value of Survival Gains in Pancreatic Cancer from Novel Treatment Regimens.

Background: Metastatic pancreatic cancer (mPC) is associated with low survival, with less than 10% of patients surviving 5 years. Recent therapies improve survival outcomes where few alternative therapies exist, but few economic analyses measure the value of survival gains attributable to new therapies.

Objective: To estimate the value of survival gains in advanced or mPC attributable to the introduction of novel treatment regimens.

Orphan Drug Expenditures In The United States: A Historical And Prospective Analysis, 2007-18.

The Orphan Drug Act of 1983 established incentives for the development of drugs that treat rare, or orphan, diseases. We used the IMS Health MIDAS database of audited biopharmaceutical sales to measure US annual spending on orphan drugs in the period 2007-13, and we estimated spending on the drugs for the period 2014-18. We identified 356 brand-name orphan drugs that were approved by the Food and Drug Administration in the period 1983-2013.

Pharmaceutical expenditure on drugs for rare diseases in Canada: a historical (2007-13) and prospective (2014-18) MIDAS sales data analysis.

Background: Health Canada has defined rare diseases as life-threatening, seriously debilitating, or serious chronic conditions affecting a very small number of patients (~1 in 2,000 persons). An estimated 9 % of Canadians suffer from a rare disease. Drugs treating rare diseases (DRDs) are also known as orphan drugs.

Nab-paclitaxel, docetaxel, or solvent-based paclitaxel in metastatic breast cancer: a cost-utility analysis from a Chinese health care perspective.

Background: Paclitaxel and docetaxel are commonly used for metastatic breast cancer in the People's Republic of China. To improve the safety and efficacy of paclitaxel, an albumin-bound formulation (nab) is now available in the People's Republic of China (Abraxane(®)). To provide health economic data for the key stakeholders, a cost-utility analysis comparing nab-paclitaxel to docetaxel, both as alternatives to paclitaxel, was conducted.

Impact of patient programs on adherence and persistence in inflammatory and immunologic diseases: a meta-analysis.

Objectives: Patient adherence and persistence is important to improve outcomes in chronic conditions, including inflammatory and immunologic (I&I) diseases. Patient programs that aim at improving medication adherence or persistence play an essential role in optimizing care. This meta-analysis assessed the effectiveness of patient programs in the therapeutic area of I&I diseases.

Abraxane® versus Taxol® for patients with advanced breast cancer: A prospective time and motion analysis from a Chinese health care perspective.

Background: Abraxane® and Taxol® are both effective drugs for the treatment of advanced stage breast cancer. However, each agent possesses unique drug delivery characteristics with the former not requiring premedication and having a considerably shorter recommended infusion time (i.e.

Denosumab versus zoledronic acid for treatment of bone metastases in men with castration-resistant prostate cancer: a cost-effectiveness analysis.

Objective: Denosumab has been approved in the US for skeletal-related event (SRE) prevention in bone-metastatic prostate cancer on the basis of a phase III clinical trial in which denosumab reduced SREs relative to zoledronic acid. Overall survival, disease progression, and serious adverse events did not differ significantly between groups. This analysis assessed the cost-effectiveness of denosumab vs zoledronic acid in bone-metastatic prostate cancer from a US payer perspective.

Cost-effectiveness of denosumab versus zoledronic acid in the management of skeletal metastases secondary to breast cancer.

Background: Denosumab has been approved in the United States for the prevention of skeletal-related events (SREs) in metastatic breast cancer. In a Phase III trial in patients with bone-metastatic breast cancer (N = 2033), denosumab was associated with a significantly delayed time to first SRE (by 18%; P < 0.001 noninferiority; P = 0.

Evaluation of the clinical benefit of long-term (beyond 2 years) treatment of skeletal-related events in advanced cancers with zoledronic acid.

Background: Skeletal complications of malignant bone disease are common among patients with both solid tumors and multiple myeloma (MM). Zoledronic acid (ZOL; Zometa*) is an intravenous bisphosphonate with proven efficacy in reducing the incidence of skeletal complications and delaying the time to a first skeletal complication. This study was designed to assess the continued benefit of ZOL treatment over a prolonged period.

Pharmacoeconomics of bisphosphonates for skeletal-related event prevention in metastatic non-breast solid tumours.

Bisphosphonates reduce the risk of skeletal-related events (SREs; i.e. spinal cord compression, pathological fracture, radiation or surgery to the bone, and hypercalcaemia) in patients with metastatic cancer.

Persistency with zoledronic acid is associated with clinical benefit in patients with multiple myeloma.

Zoledronic acid (ZOL), an intravenous bisphosphonate, has been shown to reduce and delay the incidence of skeletal-related events (SREs) in multiple myeloma (MM) patients with bone disease. A retrospective claims-based analysis was conducted that used two distinct US managed care databases to examine the relationship between persistency with ZOL and clinical benefit. Patients >18 years, diagnosed with MM, and with at least one claim for ZOL (or a claim for malignant bone disease and ZOL initiation within 30 days) between 1/1/2001 and 12/31/2006 were included.

Cost-effectiveness of zoledronic acid vs clodronic acid for newly-diagnosed multiple myeloma from the United Kingdom healthcare system perspective.

Objective: In the Medical Research Council Myeloma IX Study (MMIX), zoledronic acid (ZOL) 4 mg 3-4/week reduced the incidence of skeletal-related events (SREs), increased progression free survival (PFS), and prolonged overall survival (OS), compared with clodronic acid (CLO) 1600 mg daily, in 1970 patients with newly-diagnosed multiple myeloma (MM).

Methods: An economic model was used to project PFS, OS, the incidence of SREs and adverse events and expected lifetime healthcare costs for patients with newly-diagnosed MM who are alternatively assumed to receive ZOL or CLO. The incremental cost-effectiveness ratio [ICER] of ZOL vs CLO was calculated as the ratio of the difference in cost to the difference in quality-adjusted life years (QALYs).

Retrospective evaluation of the clinical benefit of long-term continuous use of zoledronic acid in patients with lung cancer and bone metastases.

Background: For patients with bone metastases, skeletal-related events including fracture are common, can cause considerable morbidity, and may reduce overall survival (OS). This retrospective analysis assessed the effect of Zometa (zoledronic acid, ZOL), an intravenous bisphosphonate (IV-BP), on fracture risk and OS in patients with bone metastases from lung cancer (LC). (Zometa is a registered trademark of Novartis Pharmaceuticals Corporation, USA.

Assessment of zoledronic acid treatment patterns and clinical outcomes in patients with bone metastases from genitourinary cancers.

Background: Patients with bone metastases secondary to genitourinary (GU) cancer are at risk for skeletal-related events (SREs), including bone pain requiring palliative radiotherapy, fractures or surgery to bone, spinal cord compression, and hypercalcemia of malignancy. These SREs can be debilitating and potentially life-limiting. This study examined treatment practices and the association of treatment patterns with Zometa (zoledronic acid, ZOL), an intravenous bisphosphonate (IV-BP), with SREs and fractures.

Retrospective database analysis of the effect of zoledronic acid on skeletal-related events and mortality in women with breast cancer and bone metastasis in a managed care plan.

Background: Bone metastases are common in patients with advanced breast cancer, and place patients at risk for skeletal-related events (SREs) including pathologic fracture, spinal cord compression, hypercalcemia of malignancy, and the need for radiotherapy and/or surgery to bone. These SREs are associated with reduced survival and quality-of-life. The nitrogen-containing bisphosphonates Zometa (zoledronic acid, ZOL) and Aredia (pamidronate disodium, PAM) reduce SRE risk in patients with bone metastases from breast cancer.

Cost-effectiveness of zoledronic acid in the management of skeletal metastases in patients with lung cancer in France, Germany, Portugal, the Netherlands, and the United kingdom.

Background: Zoledronic acid (ZOL) significantly reduces the risk of new skeletal-related events (SREs) in patients with non-small cell lung cancer (NSCLC) who have bone metastases.

Objective: The purpose of this study was to assess the cost and cost-effectiveness of ZOL in the management of skeletal metastases in this population across 5 European countries (France, Germany, United Kingdom, Portugal, and the Netherlands) from the perspective of national health care.

Methods: This cost-effectiveness analysis was based on a subset of patients with NSCLC who were enrolled in a Phase III trial of patients with bone metastases secondary to a variety of solid tumors.

Cost-effectiveness of zoledronic acid plus endocrine therapy in premenopausal women with hormone-responsive early breast cancer.

Purpose: The aim of this study was to estimate the cost-effectiveness of adding zoledronic acid 4 mg intravenously every 6 months to endocrine therapy in premenopausal women with hormone receptor-positive early breast cancer from a US health care system perspective.

Materials And Methods: A Markov model was developed to predict disease progression, mortality, and costs of breast cancer care for premenopausal women with hormone receptor-positive early breast cancer receiving up to 3 years of (1) endocrine therapy (goserelin plus tamoxifen or anastrozole); or (2) endocrine therapy plus zoledronic acid. Model parameters were obtained from ABCSG-12 (Austrian Breast and Colorectal Cancer Study Group Trial-12) and the literature.

Risk-factor clustering and cardiovascular disease risk in hypertensive patients.

Background: Patients with hypertension often have other major risk factors for cardiovascular disease (CVD). Little is known, however, about the extent of risk-factor clustering in these patients and its importance in CVD risk and medical-care costs.

Methods: Study subjects were selected from the electronic medical records system of Kaiser Permanente Northwest, a large health maintenance organization, and included all patients aged > or =35 years with hypertension who were free of CVD in 1998.