The application of pharmacoeconomic modelling to estimate a value-based price for new cancer drugs.

RATIONALE, AIMS AND OBJECTIVES: 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). METHODS: 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. RESULTS: 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. DISCUSSION: 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.

Economic analysis of albumin-bound paclitaxel for the treatment of metastatic breast cancer.

BACKGROUND: An albumin-bound formulation of paclitaxel (nab-paclitaxel) was developed to provide additional efficacy and to overcome the safety drawbacks of paclitaxel and docetaxel in patients with metastatic breast cancer. To provide health economic data for the Canadian setting, an economic analysis comparing each of nab-paclitaxel and docetaxel, both as alternatives to paclitaxel in metastatic breast cancer was conducted. METHODS: The clinical and safety data were obtained from a meta-analysis of randomized trials comparing either nab-paclitaxel (260 mg/m(2) q3wk) or docetaxel (100 mg/m( 2) q3wk), to standard paclitaxel (175 mg/m(2) q3wk). Health care resource use for the delivery of chemotherapy and the management of grade III/IV toxicity was collected from the oncology literature. Treatment preferences and health state utilities were obtained from 24 female oncology nurses and pharmacists using the time trade-off technique. RESULTS: Nab-paclitaxel had the lowest incidence of grade III/IV toxicity. This translated to lower overall costs for managing the grade III/IV events relative to both docetaxel and paclitaxel ($597 vs. $2626 vs. $1227). Using the median number of cycles administered and the cost impact of grade III/ IV toxicity, the overall cost for nab-paclitaxel would be $15,105 compared to $15,268 for docetaxel and $3557 for paclitaxel. When treatment preferences were assessed, 20 of 24 (83.3%) respondents selected nab-paclitaxel as their preferred choice compared to only 4 who selected docetaxel. These corresponded to a gain of 0.203 and 0.016 QALYs for nab-paclitaxel and docetaxel, respectively. With these utility benefits, the incremental cost per QALY gained was more favorable for nab-paclitaxel than docetaxel ($56,800 vs. $739,600). CONCLUSIONS: Nab-paclitaxel would be an economically reasonable alternative to docetaxel in MBC patients. As an alternative to paclitaxel, formulary committees must decide if the $56,800 cost per QALY represents good value in their health care setting.

Severe diarrhea in patients with advanced-stage colorectal cancer receiving FOLFOX or FOLFIRI chemotherapy: the development of a risk prediction tool.

BACKGROUND: FOLFOX (oxaliplatin/leucovorin/5-fluorouracil) and FOLFIRI (irinotecan/leucovorin/5-fluorouracil) are important regimens for the treatment of advanced-stage colorectal cancer (CRC). However, both are associated with severe diarrhea, leading to hospitalization, dose reductions/delays, and even death. In this study, the development of a prediction model for severe diarrhea is described. PATIENTS AND METHODS: The records of 200 patients with CRC who had received FOLFOX or FOLFIRI in 3 Canadian cancer centers were reviewed. Clinical and biochemistry parameters potentially associated with diarrhea were abstracted. Logistic regression analysis was applied to develop the final risk model. A risk scoring system, ranging from 0 to 15, was then created from the regression parameters. A receiver operative characteristic curve analysis was done to measure the accuracy of the scoring system. RESULTS: Important predictors for severe diarrhea included existing comorbidity, patient performance status, an increased baseline bilirubin level, resection of the primary tumor, FOLFOX chemotherapy, metastatic or advanced locoregional versus resected stage IV disease, and the initiation of treatment in the summer months. The receiver operative characteristic analysis had an area under the curve of 0.80 (95% confidence interval, 0.74-0.87). An overall risk score of > or = 7 for a given patient was identified as being the optimal cutoff to maximize the sensitivity (61.4%) and specificity (89.6%) of the prediction tool. CONCLUSION: We developed a prediction tool for severe diarrhea in patients with CRC receiving FOLFOX or FOLFIRI chemotherapy. To make the model available for easy use and access, we have incorporated it on to our risk prediction Web site: www.PredictPatientEvents.com. Prospective external validation is also being planned.