Cost-benefit evaluation of advanced therapy lines in metastatic triple-negative breast cancer in Germany.

BACKGROUND: Triple-negative breast cancer (TNBC) is responsible for 10-20% cases of breast cancer and is resulting in rising healthcare costs. Thus, health-economic evaluations are needed to relate clinical outcomes and costs of treatment options and to provide recommendations of action from a health-economic perspective. METHODS: We investigated the cost-benefit-ratio of approved treatment options in metastatic TNBC in Germany by applying the efficiency frontier approach. These included sacituzumab-govitecan (SG), eribulin, vinorelbine, and capecitabine. Clinical benefit was measured as (i) median overall survival (mOS) and (ii) health-related quality of life (HRQoL) in terms of time to symptom worsening (TSW). To assess medical benefits, literature was systematically reviewed in PubMed for (i) and (ii), respectively. Treatment costs were calculated considering annual direct outpatient treatment costs from a statutory healthcare payer perspective. It was intended that both, (i) and (ii), yield an efficiency frontier. RESULTS: Annual direct outpatient treatment costs amounted to EUR 176,415.21 (SG), EUR 47,414.14 (eribulin), EUR 13,711.35 (vinorelbine), and EUR 3,718.84 (capecitabine). Systematic literature review of (i) and statistical analysis resulted in OS values of 14.3, 9.56, 9.44, and 7.46 months, respectively. Capecitabine, vinorelbine, and SG are part of the efficiency frontier including OS. The highest additional benefit per additional cost was determined for vinorelbine, followed by SG. Systematic review of (ii) revealed that no TSW data of TNBC patients receiving vinorelbine were available, preventing the presentation of an efficiency frontier including HRQoL. CONCLUSIONS: Vinorelbine is most cost-effective, followed by SG. Health-economic evaluations support decision-makers to assess treatment options within one indication area. In Germany, the efficiency frontier can provide decision support for the pricing of innovative interventions. Results of our analysis may thus guide reimbursement determination.

[Characteristics of Care and Outcome Quality within the Specialized Ambulatory Palliative Care (SAPV): Analysis of Patient Characteristics and Indicators of Care of a Palliative Care Team]. / Versorgungscharakteristika und Ergebnisqualität in der Spezialisierten Ambulanten Palliativversorgung (SAPV): Analyse und Verlauf von Patienten- und Versorgungsdaten am Beispiel eines SAPV-Teams.

BACKGROUND: The ambulatory specialized palliative care (SAPV) was implemented to enable patients with the need for end-of-life intensive care to die with accompanied professionality and in familiar surroundings. Studies on changes of patient characteristics as well as the contents of care within time are scarce. The aim of this study was the evaluation of care parameters and variations in time as well as a qualitative assessment of the dying process and the stress on the dependents. METHODS: Anonymized routine patient data sets from 2012 to 2016 were retrospectively analyzed and compared to assess the efforts, characteristics of care as well as development of work within time. On the basis of the palliative care problem severity score (PCPSS), we developed the instrument "score of concern" to describe the quality of the dying process and the burden on the dependents. RESULTS: In the complete time frame, 1806 cases were treated. The median age was 74 years, 82% had cancer, 36% were counselled, 14% had consultation, 50% had partial care. 98% died at their desired place. The patients of 2016 were older in comparison to those from 2012, had more non-oncological diagnoses, were more severely ill, were infrequently hospitalized and more intensely cared for. The dying process was associated with fewer signs of stress. CONCLUSION: Data relevant for care document the changes in complexity as well as density of the work within time. The instrument "score of concern", developed to describe the dying process, can be a helpful tool to assess the qualitative outcome of patient and dependents' care and may be helpful also for other SAPV teams.

[Readiness to Participate in Ambulatory Special Medical Care (ASV): A Critical Appraisal - Results of a Survey Amongst Office-Based Oncologists in Germany]. / Bereitschaft zur Teilnahme an der ambulanten spezialfachärztlichen Versorgung (ASV): eine kritische Bestandsaufnahme.

BACKGROUND: Outpatient specialized care (ASV) is intended to improve interdisciplinary, transsectoral care for patients with rare or complex diseases, especially cancer. Up to now, only very few such specialized care teams have been established. The aim of our study was to evaluate the current state of information and participation as well as reasons for the reluctance of office-based hematologists and oncologists. METHODS: We conducted a web-based survey amongst office-based hematologists and oncologists in Germany and received responses from 145 institutions comprising more then 350 doctors working there. The questions comprised 22 items concerning the state of information, implementation as well as obstacles to participation and evaluation of the ASV. RESULTS: The majority of the interviewed doctors was well informed about ASV. Only a minority was in principle against this concept. 6% of them had already treated patients within ASV, 8% had completed the preparation, 16% were in the preparation process, 26% were indecisive and 45% had decided not to participate. The main reasons for non-participation or barriers were high expenditure and a lack of benefit for patients, strategic positioning as well as reimbursement. CONCLUSIONS: Although implemented more than 5 years ago, ASV plays no relevant role in patient care. The reluctance seems not to be due to information deficits, but rather to obstacles in the participation procedure. Relevant advantages for patient care, interdisciplinary communication or reimbursement are not anticipated. As long as the current low acceptance to participate in ASV is not improved, it cannot unfold its potential.

Allogeneic hematopoietic stem cell transplantation (allo SCT) for chronic myeloid leukemia in the imatinib era: evaluation of its impact within a subgroup of the randomized German CML Study IV.

The role of allogeneic stem cell transplantation in chronic myeloid leukemia is being reevaluated. Whereas drug treatment has been shown to be superior in first-line treatment, data on allogeneic hematopoietic stem cell transplantation (allo SCT) as second-line therapy after imatinib failure are scarce. Using an interim safety analysis of the randomized German CML Study IV designed to optimize imatinib therapy by combination, dose escalation, and transplantation, we here report on 84 patients who underwent consecutive transplantation according to predefined criteria (low European Group for Blood and Marrow Transplantation [EBMT] score, imatinib failure, and advanced disease). Three-year survival after transplantation of 56 patients in chronic phase was 91% (median follow-up: 30 months). Transplantation-related mortality was 8%. In a matched pair comparison of patients who received a transplant and those who did not, survival was not different. Three-year survival after transplantation of 28 patients in advanced phase was 59%. Eighty-eight percent of patients who received a transplant achieved complete molecular remissions. We conclude that allo SCT could become the preferred second-line option after imatinib failure for suitable patients with a donor. The study is registered at the National Institutes of Health, http://clinicaltrials.gov: NCT00055874.

[Outpatient medical specialist care (ASV): A multiperspective study on status quo, challenges and perspectives]. / Ambulante Spezialfachärztliche Versorgung (ASV): eine multiperspektivische Studie zu Status quo, Herausforderungen und Perspektiven.

BACKGROUND: In 2012, the so-called ambulatory medical specialist care (ASV) was implemented in accordance with para. 116b of Book V of the German Social Code (SGB V), enabling physicians in outpatient practices and hospitals to treat patients with rare diseases or complex courses of disease in a uniform framework. The implementation, however, is slow. The Joint Federal Committee (G-BA) has therefore commissioned an evaluation of the ASV with the aim to examine the reasons for this and to provide recommendations for further development. METHODS: The health services research study "GOAL-ASV" (Innovation Fund, 01VSF19002) included a multi-perspective design with primary data collection as well as secondary data analyses. Data from the ASV service center and the central association of statutory health insurances and the notification forms of the extended state committees were analyzed. Data from the Robert Koch-Institute, the Federal Joint Committee, the National Association of Statutory Health Insurance Funds and a literature database analysis were used in order to estimate the proportion of insured persons qualifying for ASV. Care was examined by analyzing pseudonymized routine data from the statutory health insurances using selected indicators. Participating and not participating physicians were asked to complete an online survey. RESULTS: Since the start of ASV, 615,531 insured persons have been treated in this form of care. At the time of analysis, 509 teams were operating, with 26,540 physicians treating 102,898 patients by the end of March 2021 in all indications. This comprises less than 9.8 %. of all approx. 1.05 million eligible patients. Especially in the case of rare diseases, a low willingness of participation can be seen. In addition, there was a relevant proportion of multiple uses of physicians within and outside ASV at 31 percent as well as indications of passive participation of doctors. We found significant regional differences in type and scope of the notification procedure as well as the implementation of teams with 13.4 teams per 1 million inhabitants in Schleswig-Holstein and no team in Mecklenburg-Vorpommern. Patient benefits (84 %), interdisciplinary (82 %) and cross-sectoral cooperation (75 %) were cited as motivations for participation. The main barriers reported by the respondents were the complex and laborious notification procedure (60 %), the administrative and documentation effort during participation (50 %), insufficient billing figures (49 %), and a small proportion of patients (32 %) with a consecutively unfavorable assessment of the cost to income ratio due to the current reimbursement system. DISCUSSION: Nearly ten years after its introduction, the ASV has not become established nationwide. The reasons for this probably are the complex notification procedure and the reimburesement system for rare diseases. In the case of rare diseases, the risk of underuse is becoming apparent. CONCLUSION: Strategies to further develop the ASV should, in particular, simplify the notification procedure and reduce the obstacles during participation. The remuneration system should take more account of the specific care required.

Mine drainage: Treatment technologies and rare earth elements.

The recent research and development on mine drainage published in 2018 was summarized in this annual review. In particular, this review was focused on two main aspects of mine drainage: (a) advances in treatment technologies and (b) rare earth elements in mine drainage and its recovery. The first section covers passive treatment technologies and active treatment options, including physiochemical treatment and biological treatment. The second section includes the characterization of rare earth elements in mine drainage and recovery technologies. Due to the importance of rare earth elements and the growing interest in their recovery from mine drainage, rare earth elements are reported as a separate section for the first time in this review. PRACTITIONER POINTS: Advances in treatment technologies for mine drainage are reviewed. Rare earth elements in mine drainage and its recovery are summarized. Reviewed technologies include passive, active, advanced and novel processes.

[Reimbursement differences in the ambulatory specialized care (ASV) compared to the previous care system. An exemplary analysis in medical oncology treating gastrointestinal malignancies]. / Vergütungsunterschiede bei der ambulanten spezialfachärztlichen Versorgung (ASV) im Vergleich zur bisherigen Behandlungsform.Eine explorative Analyse an drei Einrichtungen der Fachgruppe internistische Onkologie im Indikationsbereich gastrointestinale Tumoren.

INTRODUCTION AND AIM: The ambulatory specialized care (ASV) act (Sect. 116b of the Social Code Fifth Book [SGB V]) is intended to enable patients with a rare disease or a special course of disease or patients needing a highly specialized treatment to get access to outpatient care by office-based as well as hospital doctors. Data concerning care, service performance and fees - in comparison to the usual contract with the statutory insurance or the former Sect. 116b SGB V - are lacking. We explored the question whether differences in reimbursement between ASV and the previous system exist and which factors are influencing them. METHODS: We analyzed ICD-10 diagnoses, performance parameters as well as budgets and service fees in the former care system of medical oncologists in the institutions of three ASV participants of two federal countries treating gastrointestinal malignancies. We compared the results (fees, remuneration) to those from the statutory contract system and the former ambulatory care of hospitals and calculated the differences. Data were analyzed descriptively and analytically using SPSS. RESULTS: The analyses showed significant differences in the reimbursement rates between both office-based teams due to different budgets in the statutory contract system of the different federal countries. This led to additional remuneration of 12.5 to 49 % in ASV. The increase in fees of the hospital-guided team was exclusively due to the ASV-only fees of chapter 51 of EBM since there were no limitations of budgets even in the former system. DISCUSSION AND CONCLUSION: Exemplified with the ASV subgroup GIT, our study shows for the analyzed medical specialty that the difference in reimbursement in ASV is mostly due to the federal country-specific budgets and that the increase in honoraria can be substantial. Due to differences in budgets and quota systems, there may be different results in other ASV indications and specialist groups as well as in other federal states. Irrespective of these arguments, further aspects need to be taken into account when participation in ASV is considered.

[For discussion: Restructuring of medical specialist treatment using the example of ambulatory special medical care (ASV): Taking a critical look at its aims and implementation]. / Zur Diskussion: Neustrukturierung der fachärztlichen Behandlung am Beispiel der ambulanten spezialfachärztlichen Versorgung (ASV): ein kritischer Blick auf Ziele und Umsetzung.

With the GKV-VStG act in 2012, the former section 116b SGB V was transformed into ambulatory special medical care (ASV). This system comprises the diagnosis and therapy of rare or complex diseases which are difficult to treat, in need of a special qualification of doctors, an interdisciplinary cooperation and/or special facilities. For the first time, a uniform regulatory framework was established for the participation of office- as well as hospital-based doctors in ambulatory care. There is no planning of demand or licensed permission. The Federal Joint Committee's instructions regulate the extent of care, the prerequisites of participation, scope of clinical services and reimbursement. The implementation is complex and has so far been very slow. The process is impeded by the legislative conflict between incentives for care providers and concerns about the uncontrolled evolution of expenditure. The procedure of furnishing the requested proof that the conditions of participation have been fulfilled is burdensome, creating yet another barrier to broad participation of doctors and institutions. Differences in competition between office-based physicians and hospitals have been reduced but not eliminated. Sectorial barriers are only partially reduced, existing structures provide alternative care. Improvement of care in rural areas, active involvement of patients and a genuine evaluation do not take place.

Quantification of circulating tumour cells for the monitoring of adjuvant therapy in breast cancer: an increase in cell number at completion of therapy is a predictor of early relapse.

Treatment efficiency of adjuvant therapy in breast cancer is revealed after several years by statistical evaluation, but this gives no answer for the individual patient. Having shown that circulating epithelial tumour cells (CETC) respond to neoadjuvant therapy in exactly the same way as the tumour, we monitored adjuvant therapy in 25 non-metastatic breast cancer patients. Nineteen patients with a decline or no change in number of CETC showed no relapse whereas six patients with a more than ten-fold increase had five distant and one local relapse, indicating that the dynamic of CETC in the individual patient is predictive of outcome.

Tumor Boards from the Perspective of Ambulant Oncological Care.

BACKGROUND: Cancer therapy requires the cooperation of physicians from different disciplines. At the core of the collaboration are 'tumor boards' (also known as 'multidisciplinary team meetings' and 'tumor conferences'). However, there is limited health care research that addresses the multiple facets of such meetings. MATERIAL AND METHODS: Data were analyzed from 3 routinely conducted surveys comprising information from (i) office-based hematologists and oncologists, (ii) their patients, and (iii) quality indicators. RESULTS: Most office-based oncologists stated that they participate in tumor boards taking place in hospitals. Although tumor boards are viewed as time intensive and lack financial compensation, they are considered as beneficial for patient care. Less than half of patients knew that their cases were discussed at a tumor board; those who knew rated the collaboration between their treating doctors as more positive, but also experienced slightly higher distress levels. The quality indicators showed that tumor board meetings were documented in three-quarters of the patient charts, but only half included the tumor board's recommendation. CONCLUSIONS: Participation in tumor boards is perceived as beneficial by oncologists and not just considered as a fulfillment of societies' recommendations or guidelines. The higher distress levels in patients who knew that their case was discussed at a tumor board may have occurred because more difficult cases are discussed at tumor boards and since the oncologists spend more time explaining the therapy process in complex cases, including the tumor board results.

An increase in cell number at completion of therapy may develop as an indicator of early relapse: quantification of circulating epithelial tumor cells (CETC) for monitoring of adjuvant therapy in breast cancer.

PURPOSE: Treatment efficiency of adjuvant therapy in breast cancer is only revealed after several years by statistical evaluation and gives no answer for the individual patient. We here present a method to analyze the response to adjuvant chemotherapy online in individual patients. METHODS/RESULTS: In 25 consecutive non-metastatic primary breast cancer patients adjuvant fluorouracil/epirubicin/cyclophosphamid (FEC) or EC followed by taxane (EC-T) or cyclophosphamid/methotrexate/fluorouracil (CMF) therapy were given. Circulating epithelial tumor cells (CETC) were quantified before and after each second cycle of the therapy regimen, between the anthracycline and the taxane block of the regimen and in some cases repeatedly during CMF treatment. Independent of the initial cell number CETC numbers showed a decline, no change or a minor increase in 15 patients of which 14 remained in complete remission and 1 suffered local relapse. Ten patients showed an increase at the end of therapy of which 4 have relapsed during the observation time of between 2 months and up to 54 months. This patient group was compared to a previously published group of 25 patients who have all reached a follow-up of 4.5 years or until relapse. CONCLUSION: As in the previous report, Kaplan-Meier analysis revealed a high correlation between the response of CETC to therapy and relapse (p < 0.0001) and curves of both patient groups were super imposable. Multivariate analysis revealed the response of CETC to therapy to be an independent predictive marker for relapse.