Market and patient access to new oncology products in Europe: a current, multidisciplinary perspective.

To air challenging issues related to patient and market access to new anticancer agents, the Biotherapy Development Association--an international group focused on developing targeted cancer therapies using biological agents--convened a meeting on 29 November 2007 in Brussels, Belgium. The meeting provided a forum for representatives of pharmaceutical companies and academia to interact with European regulatory and postregulatory agencies. The goal was to increase all parties' understanding of their counterparts' roles in the development, licensure, and appraisal of new agents for cancer treatment, events guided by an understanding that cancer patients should have rapid and equitable access to life-prolonging treatments. Among the outcomes of the meeting were a greater understanding of the barriers facing drug developers in an evolving postregulatory world, clarity about what regulatory and postregulatory bodies expect to see in dossiers of new anticancer agents as they contemplate licensure and reimbursement, and several sets of recommendations to optimize patients' access to innovative, safe, effective, and fairly priced cancer treatments.

Treatment pathways, resource use and costs in the management of small cell lung cancer.

BACKGROUND: Small cell lung cancer (SCLC) represents about 20% of primary lung tumours and the costs associated with the management of SCLC can be significant. The main objective of this study was to obtain information on current patterns of care and associated resource use and costs for patients with SCLC from initial diagnosis and treatment phase, throughout disease progression and terminal care. METHODS: A 4 year retrospective patient chart analysis (1994-7) was conducted on a consecutive series of 109 patients diagnosed with SCLC in two Newcastle hospitals. For this consecutive series of patients all details about care received including tests and procedures, treatment, and medication from diagnosis till death were recorded. Pathways of care and forms were designed to enable resource use to be captured for different disease phases. Unit costs were determined from a variety of sources including the Newcastle Hospitals NHS Trust Finance Department and the British National Formulary. RESULTS: The average total cost per patient calculated for the full cohort of 109 patients was pound 11,556. Initial treatment was the most resource use intensive constituting 48.2% of the total cost. The major cost element throughout all disease phases was hospitalisation. Twenty eight percent of the total costs of care occur after recurrence of the disease until death, of which 73% are generated by terminal care. CONCLUSION: The results of this retrospective medical chart analysis show that the costs of care of SCLC are considerable, although the variability between patients in terms of the type and quantity of resource use is very high. Analyses such as this provide a useful insight into resources used in actual clinical practice.

[Adjuvant drug treatment of alcoholism with acamprosate: between sectoral budgets and disease management]. / Die adjuvante Arzneimitteltherapie der Alkoholkrankheit mit Acamprosa--Zwischen sektoraler Budgetierung und Disease Management.

OBJECTIVE: On the basis of several controlled clinical investigations the cost-effectiveness of acamprosate as adjuvant therapy of alcohol-dependent patients has yet been evaluated. These optimal conditions cannot be found in the daily ambulant practice and results in asking which of the alternatives, "standard plus acamprosate" or "standard without acamprosate", is more cost-effective in maintaining abstinence in alcohol dependent patients under realistic conditions. PATIENTS AND METHODS: In an open multi-centre study, medical care, costs and therapeutic outcome was prospectively documented. Prior to enrolling, all patients were obliged to undergo a detoxification procedure. At a mean age of 45 years the patients suffered an average of ten years from alcohol dependence. 521 patients were documented in the acamprosate cohort and 265 patients in the cohort "other therapy" over one year. Two thirds of the participating patients were male. RESULTS: At 33.6 % the rate of abstinence was remarkably higher in the acamprosate cohort in comparison to the cohort "other" at 21.1 % abstinent patients. The mean total costs per patient and year amounted to DM 3191 in the acamprosate-cohort and were significantly lower than in the cohort "other" with DM 4046. Effectiveness-adjusted costs of DM 9500 per successfully treated patient in the acamprosate-cohort were superior to the cohort "other", amounting to DM 19 148 per successfully treated patient. CONCLUSION: The described economic benefits may be utilised under conditions of an adequate disease management.

[Cost effectiveness of bisoprolol in treatment of heart failure in Germany. An analysis based on the CIBIS-II study]. / Wirtschaftlichkeit von Bisoprolol in der Behandlung der Herzinsuffizienz in Deutschland. Eine Analyse auf der Basis der CIBIS-II-Studie.

OBJECTIVES: Economic analysis of bisoprolol plus standard therapy versus placebo plus standard therapy in the treatment of chronic heart failure in Germany. MATERIALS AND METHODS: Prospective analysis of resource use and costs by way of integration into the international, randomized, double-blind CIBIS (Cardiac Insufficiency Bisoprolol Study)-II clinical trial, which treated 1,327 patients with bisoprolol and 1,320 with placebo. Two hundred and fifteen German patients were included in CIBIS-II (bisoprolol: 112, placebo: 103). The German health economic subpopulation comprised 97 patients (bisoprolol: 52, placebo: 45). The economic base analysis valued the resource use of every single patient of this subpopulation in monetary terms, from the perspective of Germany's third party payer (statutory sick funds). RESULTS: Mean observation time was 1.3 years. During this time hospitalization costs of DM 783.--were saved in the bisoprolol group. Total direct medical costs amounted to DM 7,651.--in the bisoprolol group and DM 8,905.--in the placebo group. This means savings of DM 1,254.--per patient, or a 14.1% cost reduction. If mean data of all German CIBIS-II patients are used as a broader basis, bisoprolol therapy saves DM 1,203.--per patient. Bisoprolol therapy induced a mortality rate reduction from 17% to 12% in the overall clinical CIBIS-II population (n = 2,647). This difference is statistically highly significant (p < 0.0001). Altogether 74 lives could be saved by bisoprolol therapy. Saved life years amounted to 0.03 per patient after 65 weeks of therapy (460 days), and to 0.12 per patient after 130 weeks (30 months). As bisoprolol therapy leads to net savings, a formal cost-effectiveness analysis, which would relate incremental clinical efficiency to additional costs, is not needed. CONCLUSION: The use of bisoprolol in the therapy of chronic heart failure is not only clinically effective, it also saves net costs.

A cost minimisation analysis of cardiac failure treatment in the UK using CIBIS trial data. Cardiac Insufficiency Bisoprolol Study.

The clinical benefits of beta-blockers in heart failure are currently subject to intense debate and are being investigated. The economic impact of beta-blockade, however, has largely remained unexplored. The Cardiac Insufficiency Bisoprolol Study (CIBIS), while failing to show statistically significant reduction in mortality over conventional therapy, demonstrates that the administration of bisoprolol adjuvant to standard therapy leads to a significant reduction in hospital admission. The present study is a cost minimisation analysis based on CIBIS data for the UK and is restricted to direct costs only. The costs of bisoprolol medication and inpatient treatment of heart failure are considered. The 'base case' analysis and the sensitivity analyses carried on all cost driver parameters show that administering bisoprolol to heart failure patients adjuvantly to the standard therapy is at least cost neutral. Additional drug costs incurred by bisoprolol are compensated by the inpatient treatment costs of heart failure avoided. All other non-quantifiable clinical benefits such as improvement of New York Heart Association functional class are positive extras to patients and the National Health Service.

Economic evaluation of the Cardiac Insufficiency Bisoprolol Study for the Federal Republic of Germany.

The Cardiac Insufficiency Bisoprolol Study (CIBIS) demonstrates that, for patients with heart failure of different aetiologies, administration of the beta 1-adrenoceptor blocker bisoprolol as an adjuvant to the standard therapy leads to a significant avoidance of hospital admissions. A pharmacoeconomic analysis of the results of the CIBIS was conducted for the Federal Republic of Germany, and was restricted to direct costs only. The costs of bisoprolol medication and inpatient treatment of heart failure were considered, the latter forming the major part of costs incurred. Per 1000 patient-years, adjuvant bisoprolol therapy resulted in overall cost savings of Deutschmarks (DM)157,272. Statutory Health Insurance had a net saving of DM186,719 in 1000 patient-years, while patients experienced additional net expenses of DM17,760 over 1000 patient-years. The economic advantage of adjuvant bisoprolol treatment was also borne out in the sensitivity analysis. Adjuvant therapy with bisoprolol was not only clinically beneficial for the patient with heart failure but was also economically advantageous.

[Cost effectiveness of bisoprolol in heart failure. Economic evaluation of the Cardiac Insufficiency Bisoprolol Study (CIBIS) for Germany]. / Wirtschaftlichkeit von Bisoprolol bie Herzinsuffizienz. Okonomische Bewertung der Cardiac Insufficiency Bisoprolol Study (CIBIS) für die Bundesrepublik Deutschland.

BACKGROUND: The Cardiac Insufficiency Bisoprolol Study (CIBIS) demonstrates that, for patients with heart failure of different etiologies, the administration of the beta(1)-adrenoceptor blocker bisoprolol adjuvant to the standard therapy leads to a significant avoidance of hospital admissions. PHARMACOECONOMIC EVALUATION: The results of the CIBIS were evaluated pharmacoeconomically for the Federal Republic of Germany, and were restricted to direct costs only. The costs of bisoprolol medication and in-patient treatment of heart failure were considered, the latter forming the major part of costs incurred. CONCLUSION: Adjunctive therapy with bisoprolol is not only clinically beneficial to the patient with heart failure, but also economically advantageous.