Monthly dosing with ranibizumab (RBZ) is needed to achieve maximal visual gains in patients with neovascular ('wet') age-related macular degeneration (wAMD). In Sweden, dosing is performed as needed (RBZ PRN), resulting in suboptimal efficacy. Intravitreal aflibercept (IVT-AFL) every 2 months after three initial monthly doses was clinically equivalent to RBZ monthly dosing (RBZ q4) in wAMD clinical trials. We assessed the cost-effectiveness of IVT-AFL versus RBZ q4 and RBZ PRN in Sweden.
A Markov model compared IVT-AFL to RBZ q4 or RBZ PRN over 2 years. Health states were based on visual acuity in better-seeing eye; a proportion discontinued treatment monthly or upon visual acuity
To conduct an economic analysis comparing tamoxifen and anastrozole (Arimidex) in the adjuvant treatment of hormone receptor-positive (HR+), post-menopausal early breast cancer patients.
An economic model examined typical patients (64 years of age, HR+, 64% node negative) from the Arimidex, tamoxifen alone, or in combination (ATAC) trial over a lifetime horizon. Rates of events were derived from ATAC trial results. Post-trial event rates were drawn from the literature for tamoxifen; event rates for anastrozole were modified by the relative risks observed in the ATAC trial. Resource utilization was drawn from Statistics Canada's Population Health Model for breast cancer, supplemented by an expert panel. A public health care system perspective, 2004 Canadian prices and a 5% discount rate were employed.
Anastrozole-taking patients incurred additional hormonal treatment costs compared to tamoxifen-taking patients (incremental lifetime cost, 6,974 Canadian dollars per patient), partially offset by reduced downstream recurrences of breast cancer (1,143 Canadian dollars lifetime savings per patient) for a net incremental cost of 5,796 Canadian dollars per patient on anastrozole. The anastrozole-treated patients were projected to experience a 5.6% absolute risk reduction of first breast cancer recurrence and a 2.8% absolute risk reduction in breast cancer death. This corresponded to 30,000 Canadian dollars per life year gained and 28,000 Canadian dollars per quality-adjusted life year gained (95% confidence interval, 17,428 to 54,605 Canadian dollars). The results were affected by the duration and extent of anastrozole benefit under sensitivity analysis but remained cost-effective.
Compared to tamoxifen, anastrozole therapy is effective and cost-effective as initial adjuvant therapy in post-menopausal, HR+ early breast cancer patients.
To use data from a randomized controlled trial and update an earlier economic evaluation of surgery versus collagen injection for the treatment of female stress urinary incontinence (SUI).
A decision tree model was developed using probabilities of success and complications from a randomized controlled trial. Resource use and cost data were taken from the earlier economic evaluation. The primary outcome was treatment success, which was defined as a negative 24 hour PAD test given 1 year post-treatment. The evaluation was conducted from the 'healthcare system' perspective and separate analyses were undertaken for Ontario and Québec. Sensitivity analyses were used to examine uncertainty in probabilities and costs.
Surgery was generally more costly and more successful than collagen injection. Incremental cost effectiveness ratios indicated that the healthcare system would incur an additional cost of $121.08 to $341.35 per additional patient that was successfully treated with surgery. Sensitivity analyses showed that surgery would be less costly and more successful than collagen injection if the postoperative length of hospital stay was reduced to 1 day. Surgery might also be more cost effective than collagen injection if the number of injections used to treat patients were to increase beyond two for treatment successes and four for treatment failures.
Collagen injection is an outpatient procedure without risk of significant morbidity or complications. However, this does not readily translate into a clear cost effective advantage relative to surgery. In some cases, surgery may be more cost effective than collagen injection in the treatment of female SUI.
To provide context in oncology for the significance of the benefits and cost of combined androgen blockade (CAB) in the treatment of advanced prostate cancer.
Canadian drug costs for the survival benefit with CAB in advanced prostate cancer were compared with the costs of benefit with new treatments in advanced non-small-cell lung cancer (NSCLC), metastatic colorectal cancer, and metastatic breast cancer. Clinical toxicities were also compared.
The survival benefit with CAB in advanced prostate cancer appears to be approximately 3 months. The survival benefit with the addition of vinorelbine to cisplatin for the treatment of advanced NSCLC is approximately 2 months, and the survival benefit with the addition of irinotecan to fluorouracil (and leucovorin) for the treatment of metastatic colorectal cancer is approximately 2 to 3 months. The survival benefit with anastrozole or exemestane in advanced breast cancer, or with the addition of trastuzumab to standard chemotherapy in metastatic breast cancer that overexpresses human epidermal growth factor receptor 2 (HER2), is approximately 4 to 5 months. The calculated cost per month of survival benefit with bicalutamide in CAB for prostate cancer is 437 US dollars to 1107 US dollars. The cost per month of survival benefit with vinorelbine for NSCLC is 1241 US dollars and with irinotecan for colorectal cancer is 6812 to 11,214 US dollars. The calculated cost per month of survival benefit with anastrozole for breast cancer is 170 US dollars, for exemestane is 185 US dollars, and the cost per month with the addition of trastuzumab is 5230 US dollars. Vinorelbine and irinotecan are associated with severe grade 3 or 4 clinical toxicities, and an increased frequency of heart failure has been observed when trastuzumab is added to anthracyclines. Anastrozole, exemestane and nonsteroidal antiandrogens are associated with mild to moderate side effects.
The advantages offered by CAB (including the cost per month of survival benefit and minimal associated clinical toxicities) are comparable to the reported advantages of new treatments for other common cancers such as NSCLC, colorectal cancer, and breast cancer.
Value-based pricing has recently been discussed by international bodies as a means to estimate a drug price that is linked to the benefits it offers patients and society. The World Health Organization (WHO) has recommended using three times a country's per capita gross domestic product (GDP) as the threshold for economic value. Using the WHO criteria, pharmacoeconomic modelling was used to illustrate the application of value-based price towards bevacizumab, a relatively new drug that provides a 1.4-month survival benefit to patients with metastatic colorectal cancer (mCRC).
A decision model was developed to simulate outcomes in mCRC patients receiving chemotherapy ± bevacizumab. Clinical data were obtained from randomized trials and costs from Canadian cancer centres. Utility estimates were determined by interviewing 24 oncology nurses and pharmacists. A price per dose of bevacizumab was then estimated using a target threshold of $CAD117,000 per quality adjusted life year gained, which is three times the Canadian per capita GDP.
For a 1.4-month survival benefit, a price of $CAD830 per dose would be considered cost-effective from the Canadian public health care perspective. If the drug were able to improve patient quality of life or survival from 1.4 to 3 months, the drug price could increase to $CAD1560 and $CAD2180 and still be considered cost-effective.
The use of the WHO criteria for estimating a value-based price is feasible, but a balance between what patients/governments can afford to pay and the commercial viability of the product in the reference country would be required.
The levonorgestrel-releasing intrauterine system (IUS) is a long-acting, fully reversible method of contraception. It is one of the most effective forms of contraception available, and combines the advantages of both hormonal and intrauterine contraception. The levonorgestrel-releasing IUS also gives the users many non-contraceptive benefits: the amount of menstrual bleeding and the number of days of menstrual bleeding are reduced, which makes it suitable for the treatment of menorrhagia (heavy menstrual blood loss). Dysmenorrhoea (painful menstruation) and premenstrual symptoms are also relieved. In addition, the levonorgestrel-releasing IUS provides protection for the endometrium during hormone replacement therapy. The local release of levonorgestrel into the uterine cavity results in a strong uniform suppression of the endometrial epithelium as the epithelium becomes insensitive to estradiol released from the ovaries. This accounts for the reduction in menstrual blood loss. All possible patterns of bleeding are seen among users of the levonorgestrel-releasing IUS; however, most of the women who experience total amenorrhoea continue to ovulate. The first months of use are often characterised by irregular, scanty bleeding, which in most cases resolves spontaneously. The menstrual pattern and fertility return to normal soon after the levonorgestrel-releasing IUS is removed. The contraceptive efficacy is high with 5-year failure rates of 0.5-1.1 per 100 users. The absolute number of ectopic pregnancies is low, as is the rate per 1000 users. The levonorgestrel-releasing IUS is equally effective in all age groups and the bodyweight of the user is not associated with failure of the method. In Western cultures continuance rates among users of the levonorgestrel-releasing IUS are comparable with those of other long-term methods of contraception. Premature removal of the device is most often associated with heavy menstrual bleeding and pain, as with other long-term methods of contraception, and is most common in the youngest age group. When adequately counselled about the benign nature of oligo- or amenorrhoea, most women are very willing to accept life without menstruation. The risk of premature removal can be markedly diminished with good pre-insertion counselling, which also markedly increases user satisfaction. User satisfaction is strongly associated with the information given at the time of the levonorgestrel-releasing IUS insertion. Thus, the benefits of the levonorgestrel-releasing IUS make it a very suitable method of contraception for most women.
The goal of this paper is to evaluate the comparative value of economic methodologies in assessing the benefit of a new cytoprotective agent (amifostine) delivered before cisplatin/cyclophosphamide in the treatment of ovarian cancer.
Data from a randomized controlled multinational multicenter ovarian cancer trial were used as the basis for a retrospective pharmacoeconomic analysis. Trial results demonstrated amifostine had no significant effect on oncolytic efficacy, but side effect profiles improved for febrile neutropenia (absolute risk reduction [ARR] 11%), neurotoxicity (ARR 11%), and nephrotoxicity (ARR 26%). Although, the methodology most commonly used in this type of analysis is cost utility (CU), we investigated a "willingness to pay" (WTP) approach. RESEARCH DESIGN SUBJECTS: Four pharmacy managers were given an informal telephone interview to assess their preferences. Two managers understood, and preferred, the CU data. The others, who had no education or training on CU principles, preferred WTP data. All managers understood the outputs from WTP studies. RESULTS/MEASURES: WTP for reductions in febrile neutropenia, neurotoxicity, and nephrotoxicity were collected from 50 healthy volunteers and measured against the cost of delivering the new therapy. Results revealed WTP values of $141 per year for reductions in febrile neutropenia, $86 per year for reductions in neurotoxicity, and $71 per year for reductions in nephrotoxicity. The base case analysis showed that amifostine was cost neutral ($350 net cost, CI = -850 to +1551).
The results were well-received by decision-makers. This manager survey illustrates that the methodologic choice should be determined not only by the nature of the comparison, but also by the stakeholder the data is meant to influence.
An ideal palliative therapy for bone metastases would successfully reduce skeletal complications in several thousands of breast cancer patients. Second- and third-generation bisphosphonates are effective in reducing the overall skeletal complication rate and the time to first skeletal complication. Nevertheless, not enough evidence supports their benefit on quality of life. Furthermore, bisphosphonates are expensive (up to 775 US dollars per month, 2002 value) and cost-effectiveness evaluations have been limited to pamidronate (pamidronic acid). In economic evaluations of pamidronate, resulting incremental dollar per quality-adjusted life year gained ranged from cost savings to 108,000 US dollars per quality-adjusted life year. The data were quite sensitive to quality-of-life estimates and country-specific cost values. Because of the wide range of the cost-effectiveness ratio, it is uncertain whether the universal prescription of bisphosphonates in this setting represents an efficient use of healthcare resources. Probably, country- and drug-specific policies might increase the efficiency of this treatment. Further outcomes research is required to assess these agents more fully.