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Biomedical subjects

Mark J C Nuijten

Publications and source records attributed to Mark J C Nuijten.

11 recordsLinked to original sources

Pricing matrix model: dealing with uncertainty.

A previous publication in this journal showed that the pricing matrix model (PMM) allows an assessment of the pricing potential of a new innovative product. When the PMM is going to be used for the determination of a drug price for a new drug in the strategic product planning process, it is important that this methodology is reliable. In the initial paper the PMM only yielded an expected price for the new antidepressant without generating an estimate of the probability that the new drug would indeed be listed at this expected price. In this manuscript we present various methodologies to deal with uncertainty in the PMM. We introduce the concept of price acceptability curves. The conclusion of this paper is that the incorporation of uncertainty into the PMM will lead to a more accurate assessment of the pricing potential of a new drug.

Antidepressive Agents↗

Cost effectiveness of palivizumab for respiratory syncytial virus prophylaxis in high-risk children: a UK analysis.

OBJECTIVE: To assess the cost effectiveness of palivizumab (a preventative treatment against severe respiratory syncytial virus [RSV] infection) in children at high risk of hospitalisation, i.e. preterm infants < or = 35 weeks gestation, children with bronchopulmonary dysplasia (BPD) and children with congenital heart disease (CHD). METHODS: A decision tree model was developed employing data sources from the published literature, palivizumab clinical trials, official UK price/tariff lists and national population statistics. The comparator was no prophylaxis. The primary perspective of the study was that of the UK NHS. In a societal perspective scenario analysis, the future lost productivity of a child resulting from RSV-related mortality (indirect costs) was also included. The cost of administration of palivizumab, hospital care for RSV infections and the cost of asthma treatment were included. The analysis was based on a lifetime follow-up period in order to capture the impact of palivizumab on long-term morbidity and mortality resulting from an RSV infection. The primary efficacy outcome in the palivizumab clinical trials was the number of RSV hospitalisations avoided, which was extrapolated to effectiveness outcomes, i.e. number of life-years gained and number of QALYs. Costs and effects were discounted by 3.5%. RESULTS: In preterm infants and children with BPD, prophylaxis with palivizumab compared with no prophylaxis had an incremental cost-effectiveness ratio (ICER) of 7042 pounds/QALY without discounting outcomes, increasing to 16,720 pounds/QALY after discounting. In babies with CHD, the use of palivizumab resulted in an ICER of 2427 pounds/QALY without discounting outcomes and 6664 pounds/QALY after discounting. One-way sensitivity analyses and probabilistic sensitivity analyses confirmed the robustness of the model. A scenario analysis showed that the inclusion of indirect costs leads to further improvement in the cost-effectiveness outcomes for palivizumab. CONCLUSION: This study suggests that palivizumab prophylaxis against severe RSV infection in children at high risk may be cost effective from the NHS perspective (vs no prophylaxis), and that the positive clinical and economic benefits may persist beyond one RSV season.

Antibodies, Monoclonal↗

Pricing of pharmaceuticals. Assessing the pricing potential by a pricing matrix model.

Pricing and reimbursement of new pharmaceuticals have been based until recently on the traditional clinical trial outcomes (efficacy, safety, and quality parameters) used for registration. Now we can distinguish various additional data requirements which relate to the use of the drug in real daily practice. The most important new data requirements are effectiveness, cost-effectiveness, and budgetary impact. A main question is how much the impact is of the various types of data in the pricing and reimbursement process. The objective of this contribution is to present a method for quantifying this type of uncertainty in order to develop a more solid pricing and reimbursement strategy for a new innovative drug. The concepts are illustrated for a new hypothetical antidepressant drug in The Netherlands. This method is based on the analytic hierarchy process (AHP) concept which measures decision makers' preferences for the critical success factors. This study shows that the AHP concept may be applied to the pricing and reimbursement environment. The method may be used to assess the pricing potential of a new drug, considering the various data requirements in the reimbursement process.

Cost-Benefit Analysis↗

Incorporation of statistical uncertainty in health economic modelling studies using second-order Monte Carlo simulations.

Health economic modelling studies are of interest to many parties with different responsibilities and diverging interests. Therefore, it is obvious that recognising the relevance of statistical uncertainty and dealing with it appropriately are required to obtain unbiased results from health economic modelling studies, especially when those data are being used for reimbursement decisions. In this manuscript we explore the relevance of the incorporation of statistical uncertainty in a health economic model and identify various types of statistical uncertainty. The concepts were applied to a hypothetical Markov model for a hypothetical antiparkinsonian (AP) product. The method was based on the incorporation of probability distributions in the input variables using a second-order Monte Carlo simulation and the definition of minimum relevant differences for clinical and economic input variables and outcomes. Our paper shows that the outcomes of a health economic model might be severely biased when statistical uncertainty is not taken into account, which justifies the need for the incorporation of statistical uncertainty in a health economic model.

Antiparkinson Agents↗

Importance of nondrug costs of intravenous antibiotic therapy.

INTRODUCTION: Costs are one of the factors determining physicians' choice of medication to treat patients in specific situations. However, usually only the drug acquisition costs are taken into account, whereas other factors such as the use of disposable materials, the drug preparation time and the staff workload are insufficiently taken into consideration. We therefore decided to assess true overall costs of intravenous (IV) antibiotic administration by performing an activity-based costing approach. METHODS: A prospective survey on costs and workload by means of a time and motion analysis and activity-based costing was performed in a 605-bed secondary referral centre with 20 intensive care unit beds. The subjects were 50 consecutive patients admitted to our hospital with community-acquired pneumonia or intra-abdominal infections requiring treatment with IV antibiotics. A time and motion analysis of 103 routine acts of preparing and administering IV antibiotics was performed in the intensive care unit and in the Department of Internal Medicine. To measure the entire process an inventory and work flowchart were made using detailed questionnaires completed by members of the nursing staff, the medical staff and the pharmacy staff. In addition, questionnaires were distributed to management and secretarial staff to determine additional overhead costs. The average costs for different methods of IV antibiotic administration were then compared by timing all steps in the process. Four different methods of drug administration were used: administration by volumetric pump, administration by syringe pump, administration by 'unaided' infusion bag, and administration by direct IV injection. RESULTS: The average times required for each of these procedures, including preparation and administration of the drug, were 4:49 +/- 2:37, 4:56 +/- 2:03, 5:51 +/- 3:33 and 9:21 +/- 2:16 min (mean minutes:seconds +/- standard deviation), respectively. When the costs for expended staff time and materials (not including drug costs) were calculated this resulted in average costs of 5.65, 7.28, 5.36 and 3.83, respectively, for administration of each dose of antibiotics. These costs represent between 11% and 53% of the total daily costs of antibiotic therapy. Compared with the acquisition costs, these indirect costs ranged from 13% to 113%. Not included in this comparison is the time required for insertion of an IV catheter, which was found to be 10:15 +/- 6:31 min with an average calculated cost of 9.17. CONCLUSIONS: Total costs of IV antibiotic administration are formed not only by the costs of the drugs themselves, but also, to a substantial degree, by the time expended by medical and nursing staff, costs of disposable materials and overhead costs. Physicians making decisions regarding the use of specific medications in intensive care unit patients should take these factors into account. Use of IV antibiotics is associated with considerable workload and additional costs that can exceed the acquisition costs of the medications themselves.

Adult↗

Health care reform in six Central European countries. A focus on health economic requirements in the drug pricing and reimbursement processes.

This study gives an overview of the health care reform in six Central European countries after the transition from a central planning system to a regulated market economy. We focused on cost containment policies for drugs, especially the requirements for submitting health economic data in the pricing and/or reimbursement processes. The literature review was supplemented with a survey with decision makers at national health authorities in each country. The study covered Croatia, Czech Republic, Hungary, Poland, Slovakia, and Slovenia. All countries had in common that health economic information was used in reimbursement rather than in pricing processes. Differences between the six countries were mainly variations in the relative importance of health economic data and the presence of explicit requirements and guidelines. Published health economic guidelines exist in two countries and one of the six countries applies a mandatory submission system for a selected range of new drugs. In most of the Central European countries it is more typical that authorities issue a brief list of required data for reimbursement submissions that include health economic information among other data. There is a generally widespread expectation towards more systematic and formalized requirements for health economic and outcomes research data appearing within the next 3-5 years in the region.

Journal Article↗

The incorporation of potential confounding variables in Markov models.

OBJECTIVE: To improve the quality of the methods used in Markov modelling studies by increasing the external validity by means of the incorporation of confounding variables. STUDY DESIGN: The concepts were illustrated using a hypothetical Markov model for Parkinson's disease. METHODS: The methodology consisted of incorporation of an extra explanatory variable in the Markov health states by means of health state-specific relationships between this explanatory variable and costs as well as time-dependent values of the extra explanatory variable. In addition, we determined the relevance of the incorporation of an extra explanatory variable by means of various sensitivity analyses. RESULTS: The results showed that the outcomes of a health economic model may be severely biased, when a confounding effect of an extra explanatory variable is not taken into account. Hence the external validity of Markov models may be limited, and consequently the results of the model are not an accurate reflection of reality. CONCLUSION: This study proves the need for the incorporation of all relevant explanatory variables in a health economic model.

Antiparkinson Agents↗

Cost effectiveness of treatment of Parkinson's disease with entacapone in the United States.

OBJECTIVE: To determine the cost effectiveness of adjunctive therapy with entacapone versus standard treatment (levodopa) without entacapone for patients in the US with Parkinson's disease (PD) who experience 'off-time' (re-emergence of the symptoms of PD) while receiving levodopa. STUDY DESIGN: A Markov model was used to estimate 5-year costs and effectiveness of standard treatment with and without entacapone. METHODS: Probabilities, unit costs, resource utilisation data and utilities were obtained from published literature, clinical trial reports, a national database, and clinical experts. PD disability was measured using the daily proportion of off-time and Hoehn and Yahr scale scores. The analysis measured costs from a societal and third-party payer perspective, and effectiveness as gains in quality-adjusted life-years (QALYs) and years without progression to >25% off-time. RESULTS: From a societal perspective, entacapone therapy resulted in an incremental cost of US dollars 9327 per QALY gained compared with standard treatment. Treatment with entacapone also provided an additional 7.6 months with < or =25% off-time/day compared with standard treatment. Sensitivity analyses indicated that the model is sensitive to changes in rates of improvement/deterioration of off-time, and to the number of doses per day of levodopa with adjunctive entacapone. CONCLUSIONS: The addition of entacapone to standard treatment for patients receiving levodopa who experience off-time provides additional QALYs and gain in time with minimal fluctuations. Results of this modelling exercise suggest that therapy with entacapone may be cost effective when compared with standard treatment for PD.

Aged↗

Combining a budgetary-impact analysis and a cost-effectiveness analysis using decision-analytic modelling techniques.

BACKGROUND: Reimbursement of new drugs is usually based on the budgetary impact of a new drug but there is also increasing demand for cost-effectiveness data on new drugs. OBJECTIVE: To present a modelling technique (methodology) for an appropriate assessment of the budgetary impact of a new drug, which can simultaneously be used for a traditional cost-effectiveness analysis. DESIGN AND PERSPECTIVE: To illustrate the methodology, a model was constructed for a new hypothetical drug in Parkinson's disease, which allowed us to determine the budgetary impact and the cost effectiveness of this new antiparkinsonian drug from a societal perspective. The methodology consisted of two steps: (i) a simple population model (Markov model) was constructed to validate the epidemiological data by proving the consistency between the prevalence and incidence of Parkinson's disease for the Dutch population; (ii) this model was extended to a more complex model (semi-Markov model) by incorporation of disease progression for Parkinson's disease and all relevant economic and clinical measures. These included all drug utilisation associated with Parkinson's disease, as well as other resource utilisation patterns associated with outpatient and inpatient care for the treatment of Parkinson's disease. RESULTS: The study showed that the difference in epidemiological data between a simple model and a complex model are substantial, which justifies the development of a complex model with a higher external validity. The complex model allowed an assessment of all potential candidates for the new drug and simultaneously allowed the assessment of the cost effectiveness of the new drug versus usual care. CONCLUSION: One model can be used for an appropriate assessment of the budgetary impact and the cost effectiveness of a new drug.

Antiparkinson Agents↗

Cost-effectiveness of enoxaparin as thromboprophylaxis in acutelly ill medical patients from the Italian NHS perspective.

OBJECTIVE: To determine the cost-effectiveness of thromboprophylaxis with enoxaparin versus no thromboprophlaxis in patients with acute medical illness in Italy from the NHS perspective. METHODS: Markov process analysis techniques were used to model the health economic outcomes. Clinical data were derived mainly from the MEDENOX trial, while health care utilization was derived from Delphi panels. RESULTS: An analysis over the MEDENOX trial period shows that the cost per event avoided is [symbol: see text] 2450.99, while the cost per life saved is [symbol: see text] 8395.89. The lifetime model, which assumes no higher risk for recurrence of VTE and mortality in asymptomatic patients, shows that the use of enoxaparin leads a cost per event avoided of [symbol: see text] 2243.20, and cost per life year gained of [symbol: see text] 605.38. The lifetime model, which assumes a higher risk for recurrence of VTE in asymptomatic patients, shows that enoxaparin is dominant over no thromboprophylaxis. CONCLUSION: The results showed that the favourable clinical benefit of enoxaparin as thromboprophylaxis in patients with acute medical illness, which was observed in the MEDENOX trial, results in a positive health economic benefit on the short-term and long-term in the health care setting of Italy.

Acute Disease↗

Cost-effectiveness analysis of interferon beta in multiple sclerosis: a Markov process analysis.

OBJECTIVE: The objective of this study was to examine the cost-effectiveness of preventive treatment with interferon beta (IFNB) versus no preventive treatment in patients with multiple sclerosis. METHODS: The setting for this study was the United Kingdom. A lifetime Markov process model was constructed to model the average quality-adjusted life years (QALYs) and the costs of both treatment strategies. Data for the construction of the model came from published literature, including large multicenter randomized clinical trials in relapsing-remitting and secondary progressive multiple sclerosis. Costs were obtained from published sources. RESULTS: The results of the baseline analysis from the National Health Service (NHS) perspective showed that the use of interferon beta as preventive treatment for MS increased the total average discounted cost from 51,214 Pounds to 221,436 Pounds per patient. The undiscounted effectiveness increased from 24.9 QALYs to 28.2 QALYs, resulting in an incremental cost-effectiveness ratio of 51,582 Pounds per QALY. Sensitivity analyses showed the robustness of this model for other interferons. CONCLUSION: The study showed that preventive treatment with interferon beta in patients with multiple sclerosis may not be fully justified from a health-economic perspective, although interferon beta is associated with an improved effectiveness compared with no preventive treatment.

Adjuvants, Immunologic↗