Cost-effectiveness Of New Drugs Research Articles

Cost-effectiveness of hypothetical new cancer drugs in patients with advanced small-cell lung cancer: results of a markov chain model.

In the last decade, a number of new treatment modalities have been developed for patients with small cell lung cancer (SCLC). The clinical effects are encouraging, but little is known about the costs and cost-effectiveness of new drugs. A Markov chain model has been developed to project patient outcomes and costs for patients with advanced SCLC. All patients in the control group were treated with etoposide-cisplatin chemotherapy. Patients in the study group received a hypothetical new drug. The model consisted of four states: response, stable disease, progressive disease, and death. Estimates of transition probabilities were calculated using published data on survival and recurrence-free survival. For the cost analysis and utility calculation, published data and expert opinion were used as sources. The duration of the follow-up was maximal 2 years. The total treatment costs in the etoposide-cisplatin group amounted to euro16 038 and in the alternative treatment groups between euro16 644 and euro18 171. The number of life years and quality adjusted life years (QALYs) gained were very small, around 16 days. The cost-effectiveness ratio varied between euro22 208 and euro81 443 and the cost-utility ratio varied accordingly. Results of the sensitivity analysis showed that the results were robust in favor of etoposide-cisplatin treatment. SCLC is an illness with a poor prognosis which needed substantial healthcare resources to optimise patient survival and overall quality of life. New treatment modalities with better outcome and favourable cost-effective profiles can hopefully be developed.

Cost-effectiveness of new drugs: A systematic review of published evidence for new chemical entity drugs introduced on the Swedish market 1987–2000

The number of published economic evaluations has increased dramatically during the past decades, but the number of studies performed for different drugs varies substantially. The objective of this study was to analyze the amount of cost-effectiveness evidence for new drugs, by systematically reviewing the published evidence of cost-effectiveness. The study included 442 new chemical entities, approved in Sweden between 1987 and 2000. The amount of cost-effectiveness evidence was rated and analyzed together with information about sales and the therapeutic benefit of the drugs. Information about cost-effectiveness was obtained from the Health Economic Evaluation Database. The results showed that most cost-effectiveness evidence was published approximately 1 to 5 years after the approval year and that very few articles were published before or during the approval year. More than half of the drugs did not have any evidence of their cost-effectiveness. A total of 51 of the evaluated drugs were considered having much evidence of cost-effectiveness, 84 drugs were considered having some evidence, and the remaining 307 drugs had little evidence. The analyses indicated that drugs with improved effectiveness or safety compared with other marked drugs had more evidence of cost-effectiveness and that drugs with low sale were likely to have less evidence of cost-effectiveness than drugs with high sale. The study indicated that the publication of cost-effectiveness information for new drugs introduced between 1987 and 2000 may be considered rather rational, that is, the economic evaluations were performed for drugs for which this information was most important.

The lag between effectiveness and cost-effectiveness evidence of new drugs

A new drug is approved for use if its efficacy and safety have been demonstrated. However, healthcare decision makers may also require data on the cost-effectiveness of new drugs if they are to make informed decisions about their place in therapy. Cost-effectiveness evidence may lag behind the effectiveness data in terms of its availability. We explored the timeliness of delivering cost-effectiveness information about new drugs with established effectiveness and significant financial impact. Drugs were identified, based on guidance documents and reports published by the UK National Institute for Clinical Excellence (NICE), and the following data were collected: dates of publication of first effectiveness and cost-effectiveness evidence, methodology of the cost-effectiveness analysis, quality scores of the clinical studies. Eighteen guidance documents on the use of new drugs/drug groups published by NICE by October 2001 covered 30 health technologies, which were included in the analysis. The analysis of the evidence showed that their effectiveness had been demonstrated in the last 12 years, with only two exceptions. However, cost-effectiveness evidence had been published for 21 (70%) of the technologies. The cost-effectiveness was estimated in 52.4% of cases using models. The good quality effectiveness evidence lagged behind the first effectiveness evidence by 1.40 years (95% CI 0.57-2.23), while the mean lag between the first effectiveness evidence and the first cost-effectiveness publications was estimated as 3.20 years (95% CI 1.76-4.65). Cost-effectiveness evidence thus often lags behind the effectiveness evidence. As a result healthcare decision makers are sometimes in a position of having to take decisions without having adequate cost-effectiveness data at their disposal.

PHP20: THE LAG BETWEEN EFFECTIVENESS AND COST-EFFECTIVENESS EVIDENCE OF NEW DRUGS AND DECISION-MAKING IN HEALTH CARE

BACKGROUND: A new drug is approved for use if its effectiveness has been demonstrated. Recently decision-makers in a number of countries have begun to account for both the effectiveness and cost-effectiveness of new drugs. However, cost-effectiveness evidence lags behind the effectiveness data. OBJECTIVE: To explore the timeliness of delivering cost-effectiveness information about new drugs with established effectiveness and significant financial impact. METHODS: New drugs were identified, based on guidance documents and reports published by the National Institute for Clinical Excellence of England and Wales, and the following data were collected: dates of publication of first effectiveness and cost-effectiveness evidence, methodology of the cost-effectiveness analysis, funding of the research, etc. RESULTS: Guidance documents for the following new drugs/drug groups have been published by NICE by the end of 2000: taxanes for ovarian and breast cancer, proton pump inhibitors in the treatment of dyspepsia, glycoprotein IIb/IIIa inhibitors, methylphenidate for hyperactivity in childhood, zanamivir, and rosiglitazone for type II Diabetes Mellitus. The analysis of the evidence shows that the effectiveness of these drugs has been demonstrated in the last 12 years. However, cost-effectiveness evidence has been published for 70% of the drugs with an average delay of 3 years (range 0–10). The cost-effectiveness of those, introduced after 1995 (80% of all included drugs/drug groups), has been demonstrated using models only, if at all. CONCLUSIONS: Cost-effectiveness evidence is produced with a lag behind the effectiveness evidence. As a result, decision-makers are in a position of awaiting sound evidence while issuing guidance based on current inconclusive research results. The cost to society is discussed, and establishing the cost-effectiveness of new drugs alongside RCTs at an earlier stage of their development is suggested.

Clinical governance.

Clinical governance.

Newer atypical antipsychotic medication versus clozapine for schizophrenia

Clozapine is an atypical antipsychotic drug, which is claimed to have superior efficacy and to cause fewer movement disorders. However, clozapine carries a significant risk of serious blood disorders. Newer atypical antipsychotics are safer alternatives that might share the benefits of clozapine. It is thus of interest to compare the effectiveness of newer atypical antipsychotics with the effectiveness of clozapine. To evaluate the clinical effects of newer atypical antipsychotic drugs in comparison to clozapine for schizophrenia. Publications in all languages were searched from the following databases: Biological Abstracts/BIOSIS (1980-1999), The Cochrane Schizophrenia Group's Register of Trials (1998), The Cochrane Library CENTRAL Register (Issue 4, 1999), EMBASE (1980-1998), MEDLINE (1966-1999), LILACS/CD-ROM (1998), and PsycLIT/PsycINFO (1974-1999). Trials were also sought from recent conference proceedings and reference lists of included papers. Authors of recent trials and the manufacturers of clozapine, iloperidone, olanzapine, quetiapine, remoxipride, risperidone, sertindole, ziprasidone and zotepine were contacted. All randomised controlled trials comparing clozapine with newer atypical antipsychotic drugs were included by independent assessment by two reviewers. Data were extracted independently by two reviewers. Relative risks (RR) and 95% confidence intervals (CI) of homogenous dichotomous data were calculated. A random effects model was used for heterogeneous dichotomous data. Where possible the number needed to treat (NNT) statistic with 95%CI were also calculated. Weighted or standardised means were calculated for continuous data. Due to the small number of included studies, sensitivity analyses or funnel plot statistics were not undertaken for this version of the review. The current review includes eight studies (22 papers), of which three studies are 4-6 weeks in duration and only one study is of more than 12 weeks' duration. Newer atypical drugs seemed to be broadly similar to clozapine using a clinical global index or trialists' definitions of improvement, but this result was obtained from a relatively small number of studies. Due to the small number of studies and patients, wide confidence intervals were seen when their effectiveness as measured by symptom rating scales was compared. Social functioning was better in patients on newer atypical medication (risperidone) than in those on clozapine, but this finding is based on a single underpowered trial and has to be interpreted with caution. Clozapine and newer atypical drugs showed their adverse effect profile to be dissimilar: while clozapine produced more fatigue, hypersalivation, nausea, and orthostatic dizziness, new atypical drugs, with the exception of olanzapine, produced more extrapyramidal symptoms. The impact of these drugs and their effects on patients' day-to-day quality of life, service use, hospital admission, and pharmacoeconomics was not measured. The equal effectiveness and tolerability of new atypical drugs in comparison with clozapine is not yet demonstrated. Lack of statistical power to determine the comparative efficacy and effectiveness of newer atypical drugs makes it difficult to judge whether newer drugs are more effective, less effective or equivalent. Trials of sufficient power, with longer duration, measuring clinically important outcomes, are needed to assess the true comparative clinical effectiveness, tolerability and cost effectiveness of newer drugs in relation to clozapine.

The dilemma of new drugs. Are costs rising faster than effectiveness?

This century has seen a phenomenal growth in the development, understanding and use of pharmaceuticals. Additionally, this period has seen the balance of the treatment needs of patients shift dramatically from infectious to cardiovascular and cancer diseases, and from infancy and middle-age to old age. The value of modern pharmaceuticals to society is undoubted. However, the benefits in terms of health gain, when adequately evaluated, are often more modest than first supposed, and are often achieved at considerable costs and sometimes considerable risks to patients. Data are seldom adequate to attempt a robust evaluation of the cost effectiveness of drugs and compare how cost effectiveness may have changed over time. Rapid increases in development costs coupled with the increasing focus on chronic and old-age diseases make it probable that the cost effectiveness of new drugs is broadly declining, but such a conclusion should be interpreted with caution. The big challenge for the next century with its likely genetic and biotechnological discoveries, bringing as yet unforeseen benefits, risks and costs, is to radically improve the manner in which new drugs are evaluated and diffuse into health systems.

Cancer Treatment in Sweden - Costs of Drugs, Inpatient and Outpatient Care from 1985 to 1996 and Cost Effectiveness of New Drugs

This study was carried out to investigate the direct costs for treatment of patients with cancer from 1985 to 1996 in Sweden, and to examine health economic effects of changes in treatment pattern. Material for the study was collected from official statistics and from published health economic evaluations of cancer treatment. Costs for inpatient care decreased during the period, while costs for outpatient care and drugs increased. In total, the direct health care costs for cancer treatment decreased from 1985 to 1996. New drugs registered on the market are often more expensive than the drugs they replace. From a health economic perspective it is not clear, however, that higher drug costs necessarily increase total costs. Further health economic research is needed because many treatment alternatives have not yet been evaluated, and furthermore, because a treatment option can be cost effective in one specific indication but not in another.

What is Direct Evidence-Based Policy-Making? Experience from the Drug Benefits Program for Seniors in British Columbia

AbstractDrug benefits policy-making for seniors in British Columbia has been increasingly influenced by research since 1993. The "evidence-based medicine" paradigm, which emphasizes the primacy of direct evidence from randomized control trials, inspired key policy-makers and influenced policy concerning coverage of new and existing drugs. New drugs, if more expensive than existing similar drugs, are not covered unless published randomized control trials show superior effectiveness. Indirect evidence of effectiveness, based on surrogates, is given less weight, and non-randomized studies are rarely considered. Evidence of cost-effectiveness of new drugs is reviewed separately. For existing drugs, a new reimbursement policy, Reference Based Pricing (RBP), was introduced, based on both direct and indirect evidence of comparative effectiveness of drugs. Implementation of RBP was complex and necessarily rapid, which meant that independent systematic review of evidence relevant to implementation issues was infeasible, particularly in regard to rapid prior authorization of exemptions to RBP. Contrasts between the processes for new and existing drugs provide insights into the difficulties of applying the idea of direct evidence-based policy-making in practice.