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Background: The Dutch Committee for the Evaluation of Oncological Agents (cieBOM) assesses the clinical benefit of systemic anti-cancer treatments (SACTs). For SACTs tested in non-randomized trials (NRTs), cieBOM primarily utilizes response-related thresholds as assessment criteria. As sufficiency of NRT-based evidence for benefit assessments is questionable, this study investigated whether and how NRTs can be used to assess the clinical benefit of new SACTs initially appraised by cieBOM based on randomized controlled trials (RCTs)., Methods: Using the RCTs underpinning cieBOM recommendations issued between 2015 and 2017, we searched for matching NRTs and applied the NRT-related assessment criteria by cieBOM to them. We then compared the assessment outcomes to the respective RCT-based cieBOM recommendations. Further, we investigated how the assessments would change when applying different response-related thresholds and adding a progression-free survival (PFS) threshold., Results: For 13 of the 37 eligible recommendations, a matching NRT was found. Two treatments were assessed positively and six negatively; five treatments were non-assessable. Two positive recommendations matched a positive NRT-based assessment; one matching negative assessment was found, and one treatment could not be assessed based on either trial results. Adding a > 6 months PFS threshold decreased the number of non-assessable NRTs (five to two)., Conclusions: Limited publications and inconsistent data reporting hampered the viability of NRTs for clinical benefit assessments of SACTs beyond the scope of rare indications. Further, response-related assessment criteria alone might not fully grasp the clinical benefit of novel SACTs. NRT-based assessments should be considered with caution due to uncertainty of the trial results., Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2024 The Authors. Published by Elsevier Ltd.. All rights reserved.)

Aim: Capecitabine and bevacizumab (CAP-B) maintenance therapy has shown to be more effective compared with observation in metastatic colorectal cancer patients achieving stable disease or better after six cycles of first-line capecitabine, oxaliplatin, bevacizumab treatment in terms of progression-free survival. We evaluated the cost-effectiveness of CAP-B maintenance treatment., Methods: Decision analysis with Markov modelling to evaluate the cost-effectiveness of CAP-B maintenance compared with observation was performed based on CAIRO3 study results (n = 558). An additional analysis was performed in patients with complete or partial response. The primary outcomes were the incremental cost-effectiveness ratio (ICER) defined as the additional cost per life year (LY) and quality-adjusted life years (QALY) gained, calculated from EQ-5D questionnaires and literature and LYs gained. Univariable sensitivity analysis was performed to assess the influence of input parameters on the ICER, and a probabilistic sensitivity analysis represents uncertainty in model parameters., Results: CAP-B maintenance compared with observation resulted in 0.21 QALYs (0.18LYs) gained at a mean cost increase of €36,845, yielding an ICER of €175,452 per QALY (€204,694 per LY). Varying the difference in health-related quality of life between CAP-B maintenance and observation influenced the ICER most. For patients achieving complete or partial response on capecitabine, oxaliplatin, bevacizumab induction treatment, an ICER of €149,300 per QALY was calculated., Conclusion: CAP-B maintenance results in improved health outcomes measured in QALYs and LYs compared with observation, but also in a relevant increase in costs. Despite the fact that there is no consensus on cost-effectiveness thresholds in cancer treatment, CAP-B maintenance may not be considered cost-effective., (Copyright © 2017 Elsevier Ltd. All rights reserved.)

The aim of the present study was to identify trends in numbers of European patients treated with autologous and allogeneic haematopoietic stem cell transplantation (HSCT) as well as to provide anticipated transplant rates for the upcoming years. The following indications were considered: haematological malignancies (acute leukaemias, myeloproliferative disorders, lymphoproliferative disorders and multiple myeloma), solid tumours and non-malignant diseases. Numbers of patients treated from 1990 to 2004 were extracted from the European Group for Blood and Marrow Transplantation database, extrapolated to 2012 using mathematic models and adjusted to the literature study and expert opinion. In Europe, a 13% raise in HSCT utilisation is to be expected from 2005 to 2010, mostly due to the growing application of reduced-intensity conditioning regimens followed by allogeneic HSCT. Growing transplant rates are likely to exert health expenditure budgets and put pressure on health care providers and health insurers in Europe. Therefore, the rapid expansion would ideally imply a simultaneous increase in HSCT budgets.

A prospective randomised phase III study in patients < or =65 years old with previously untreated multiple myeloma (MM), intensive chemotherapy followed by myeloablative chemotherapy and autologous stem-cell rescue was compared with intensive chemotherapy alone. This economic evaluation was based on detailed data from patient charts and hospital information systems. In the intention-to-treat analysis, mean total treatment and follow-up costs of the myeloablative treatment arm were 81,643 euros compared to 68,802 euros for the chemotherapy arm (P=0.09). Costs per quality-adjusted life year were 51,357 euros versus 37,328 euros. In the clinical study, no significant differences were found in overall survival after a median follow-up of 33 months from randomisation. Intensive chemotherapy is regarded as standard therapy for younger patients with previously untreated MM. Cost-effectiveness of myeloma therapy after 3 years of follow up seems not to be favoured by myeloablative treatment with autologous stem-cell rescue.

We retrospectively calculated the costs of head and neck oncology for reimbursement purposes. This analysis was based on 854 head and neck cancer patients treated between 1994 and 1996 in two major Dutch university hospitals. To anticipate future care costs, costs of required improvements in the quality of care were added. Costs of diagnosis, treatment and 2 years of follow-up of patients with a primary tumour were (euro) 21 858. For patients with a recurrent tumour, this amount was (euro) 27 629. The costs of 10 years of follow-up were (euro) 423 after discounting and correction for survival. In total, average costs per new patient were (euro) 31 829, which covered discounted costs of treating the primary tumour, costs of treating recurrent tumours in 40% of all patients and the costs of 10 years of follow-up. Costs of improving the quality of care were estimated to be (euro) 1598 per new patient.

The cost-effectiveness of autologous peripheral blood stem cell transplantation (PBSCT) compared with autologous bone marrow transplantation (ABMT) for refractory or relapsed non-Hodgkin's lymphoma (NHL) or Morbus Hodgkin (MH) was assessed. Costs were determined from the induction chemotherapy regimen up to 3 months after discharge from hospital following the transplantation. Quality of life was measured by the EuroQol, the Rotterdam Symptom Checklist (RSCL) and the SF-36. Patients were randomised according to a 2:1 ratio to undergo either PBSCT or ABMT. 62 patients underwent PBSCT and 29 ABMT. Costs of the transplantation period were significantly lower in the PBSCT group (15008 Euros) than in the ABMT group (19000 Euros). Significant differences in quality of life were all in favour of PBSCT and emerged using the RSCL, both on 14 days after the transplantation and three months after discharge. We conclude that PBSCT is associated with lower costs and a better quality of life than ABMT for patients with refractory or relapsed NHL or MH.

Emesis (nausea and vomiting) is one of the most important toxicities associated with chemotherapy. Although it is not life threatening, it has a major impact on a patient's health-related quality of life (HRQL) and overall response to chemotherapy. New antiemetics are expensive and well-conducted comparative health economic studies are rare. The aim of the study was to review the literature in the area of chemotherapy-induced emesis in cancer patients and to offer recommendations for the inclusion of these outcomes in the design of clinical trials for new antiemetic therapies. The economic literature was reviewed based on methodological standards for economic evaluation. Many studies did not comply with standards, specifically with regard to the choice of alternatives, chosen perspective, setting, type of emesis, measurement of costs and defining outcomes (including health-related quality of life). These issues are described for each study and recommendations for trial design are presented. The role of economic data is to support decision making in choosing between competing antiemetic therapies. It is the combination of clinical outcomes, costs and health-related quality of life, which will allow treating physicians to comprehensively assess the relative value of antiemetic therapies and to provide the most cost-effective therapy for their patients.

The aim of this study was to determine the effect of the reduction of the length of hospital stay after surgery for breast cancer on the rate of care consumption and the cost of care. Patients with operable breast cancer were randomised to a short or long postoperative hospital stay. Data on care consumption were collected for a period of 4 months in diaries administered by patients, and socioeconomic status was evaluated by questionnaires. A cost minimisation analysis using the 'societal' perspective was performed and savings were compared with the savings of hospital charges. The use of professional home care was higher for the short stay group during the first month (7.2 versus 1.3 h, P < 0.0001). The number of out-patient consultations, the intensity of informal home care and patient's expenses did not increase after early discharge. The total cost of care was reduced by US$1320 by introducing the short stay programme (P = 0.0007), but the savings were substantially lower than the savings in hospital charges (US$2680).

Patients receiving chemotherapy frequently develop fever and neutropenia. Haematopoietic growth factors (HGFs) may decrease the duration of such episodes or may prevent a febrile neutropenic episode. In this study we introduce a Markov type economic model for the hospital which calculates all relevant direct costs and savings of HGF therapy and may support decisions on HGF administration. A distinction is made between patients receiving intensive and standard chemotherapy schedules. Our results indicate that HGFs can induce savings in intensive chemotherapy and standard chemotherapy following neutropenic fever. Prophylactic administration of HGF is cost-effective if the risk of infection is considerable. The risk of infection depends on underlying malignancy, corresponding treatment modalities and the health condition of the patient. The model is meant as an analytical framework and should be used carefully, as not all benefits (e.g. benefits to the patients) are considered. These benefits may be balanced against the additional costs or savings resulting from the economic model.

In a retrospective study, we calculated the treatment costs of 26 patients, who received either high dose melphalan combined with granulocyte colony-stimulating factor (G-CSF; filgrastim)(n = 7) or without G-CSF (n = 11) or alternatively, peripheral blood progenitor cell reinfusion (PBPC) mobilised by G-CSF following high dose melphalan. In comparison with the control group, a shortening of the pancytopenic period and platelet recovery was noticed in the PBPC group. This resulted in a reduction in hospital costs, diagnostics, laboratory services, total parenteral nutrition and transfusions. The average costs per treatment in the PBPC group amounted to about US$ 17,908 as compared to US$ 32,223 in the control group, implying a cost reduction of 44% when changing to PBPC reinfusion.

In the economic evaluation (EE) of technologies in cancer treatment at least three endpoints are relevant: costs, survival and quality of life (QoL). This article is focused on QoL. EE requires the use of generic and valuation QoL instruments at a disease non-specific level, but the inclusion of cancer-specific instruments may be advisable, particularly for reasons of explanation if changes in dimensions are small or conflicting. Given the pros and cons of the available questionnaires, we advocate the use of the Nottingham Health Profile, the EuroQol and the Rotterdam Symptom Checklist. In our experience the QoL issue in EE linked with cancer trials is associated with practical problems like questionnaire composition, follow-up time, interviewing schedule, patients' compliance and doctors' acceptance. These problems are discussed and some practical guidelines for the design of QoL measurement in cancer trials are given.

In a retrospective study, we calculated the treatment costs of 63 patients who received either autologous bone marrow transplantation (ABMT) with recombinant human granulocyte colony-stimulating factor (r-metHuG-CSF) (filgrastim) (n = 13) or without r-metHuG-CSF (n = 22) or alternatively, peripheral blood progenitor cell (PBPC) transplantation mobilised by r-metHuG-CSF (n = 28). The recovery of granulocytes, platelets and reticulocytes after PBPC was markedly accelerated as compared with ABMT with or without r-metHuG-CSF. The accelerated haematopoietic recovery was associated with a reduction in platelets and red blood cell transfusion requirements, with a reduction in episodes of fever and with earlier discharge from the hospital. This resulted in the average cost per treatment of the PBPC group being almost 30% lower than the treatment costs in the ABMT groups.

A recent extension of clinical evaluation is "economic evaluation", which seeks to characterise each relevant alternative health care strategy in terms of a summary measure incorporating the costs and benefits of such strategies. In an economic evaluation, separate measurements of resource volumina and resource prices on the cost side, and separate measurements of survival and quality of life effects and valuation of these outcome effects on the benefit side are required. From these effect parameters, which should be calculated for all competing strategies considered in the analysis, the relative cost-effectiveness of one strategy as against the other can be derived. The degree of generalisability of the study results determines the validity of economic evaluation in decision-making. This depends on the generalisability of the clinical findings, and in this respect the so-called "piggyback" economic evaluation, which is added to a clinical trial, has its limitations. In the field of cancer, specific attention should be given to costs and effects occurring after non-mortality endpoints, to patient and family costs and to variations in treatments between settings of care. It is argued that conventional clinical trials and economic evaluations will integrate further in the future.