Enhancing Transsectoral Interdisciplinary Patient-Centered Care for Patients With Rare Cancers: Protocol for a Mixed Methods Process Evaluation.

BACKGROUND: Rare cancers account for approximately 24% of all new cancers. The category of rare tumor diseases includes almost 200 different entities. In particular, the treatment of patients with extensive care needs requires cooperation between service providers, both between sectors (outpatient and inpatient) and within sectors (eg, between different medical disciplines). The treatment pathway is associated with a high need for coordination and information sharing between providers. When crossing sectoral boundaries in the German health care system, interface problems between the outpatient and inpatient sectors can lead to gaps in care delivery. The multicomponent program Trans-sectoral Personalised Care Concept for Patients with Rare Cancers aims to optimize transsectoral cooperation and coordination of care to enhance patient involvement and the medical care coordination of patients with rare cancers. OBJECTIVE: This process evaluation will contribute to answering questions about intervention fidelity and the implementation of transsectoral communication, identifying and describing the intended and nonintended effects of the intervention, and exploring the barriers to and facilitators of the implementation. METHODS: We will include patients who participate in the intervention phase; all persons and staff involved in the development and implementation of the intervention (Onco Coach, psychologists, physicians on the contact platform, IT staff, and staff of the Bavarian Association of Statutory Health Insurance Physicians); physicians from the Ludwig-Maximilians-University Hospital Munich and the hospital of the Technical University Munich who are involved in the treatment of patients during the course of the project; and participating office-based hematologists and oncologists. Data collection will be conducted at the beginning, during, and at the end of the intervention using mixed methods. Data will be collected from questionnaires, document analyses, semistructured interviews, and structured observations and will cover different aspects of process evaluation. These include examining the context to explore existing patterns, changes in patterns, attitudes, and interactions; analyzing the implementation of intervention elements; and exploring the complex causal pathways and mediators of the intervention. Qualitative data will be analyzed using thematic analysis. The data will then be combined using between-methods triangulation. RESULTS: This project received funding on March 1, 2022. The intervention phase and recruitment for the process evaluation began on March 1, 2023, and the recruitment is expected to end on September 30, 2025. At the time of protocol submission in June 2023, a total of 8 doctors from hematology and oncology practices were enrolled. Data collection began on March 14, 2023. CONCLUSIONS: The Trans-sectoral Personalised Care Concept for Patients with Rare Cancers project is a complex intervention that is to be implemented in an equally complex health care context. The process evaluation will help understand the influence of contextual factors and assess the mechanisms of change. TRIAL REGISTRATION: ISRCTN registry ISRCTN16441179; https://doi.org/10.1186/ISRCTN16441179. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/49731.

Changing treatment landscape associated with improved survival in advanced hepatocellular carcinoma: a nationwide, population-based study.

BACKGROUND AND AIMS: The treatment of hepatocellular carcinoma (HCC) is undergoing a historic transformation with the approval of several new systemic therapies in the last few years. This study aimed to examine the impact of this changing landscape on survival and costs in a Western nationwide, real-world cohort. METHODS: A nationwide representative claims database (InGef) was screened for HCC cases between 2015 and 2020. Survival in an era with only sorafenib (period A, January 2015 to July 2018) and after approval of lenvatinib and other systemic treatments (period B, August 2018 to December 2020) was analysed. Health care costs were assessed. RESULTS: We identified 2876 individuals with HCC in the study period. The proportion of patients receiving systemic therapy increased significantly over time, from 11.8% in 2015 to 15.1% in 2020 (p < 0.0001). The median overall survival in period B was 6.5 months (95% confidence interval [CI]: 4.9-8.9) and in period A was 5.3 months (95% CI: 4.5-6.3; p = 0.046). In period B, the median overall survival with lenvatinib was 9.7 months (95% CI: 6.3-18.4) versus 4.8 months with sorafenib (95% CI: 4.0-7.1, p = 0.008). Costs for prescription drugs per patient increased from €6150 in 2015 to €9049 in 2020 (p < 0.0001), and costs for outpatient care per patient increased from €1646 to €2149 (p = 0.0240). CONCLUSION: The approval of new systemic therapies resulted in a survival benefit in patients with HCC. The magnitude of the effect is modest and associated with a moderate increase in health costs.

Health technology assessment for cancer medicines across the G7 countries and Oceania: an international, cross-sectional study.

BACKGROUND: Criticisms have emerged that cancer medicines offer modest benefits at increasingly high prices. Reimbursement decisions made by health technology assessment (HTA) agencies have become a complex endeavour for cancer medicines. Most high-income countries (HICs) use HTA criteria to identify high-value medicines for reimbursement under public drug coverage plans. We compared HTA criteria specific for cancer medicines in economically similar HICs, to understand how these criteria contribute to reimbursement decisions. METHODS: We did an international, cross-sectional analysis in collaboration with author investigators across eight HICs, from the Group of Seven (known as G7; Canada, England, France, Germany, Italy, and Japan) and Oceania (Australia and New Zealand). Publicly available data from HTA agency reports and official documentation were extracted and analysed between Aug 15, 2021, and July 31, 2022. We collected data pertaining to the decision-making criteria used by the national HTA agency; HTA reimbursement status for 34 medicine-indication pairs corresponding to 15 unique US top-selling cancer medicines; and HTA reimbursement status for 18 cancer medicine-indication pairs (13 unique medicines) with minimal clinical benefit (score of 1 on the European Society of Medical Oncology Magnitude of Clinical Benefit Scale). Descriptive statistics were used to compare HTA decision criteria and drug reimbursement recommendations (or for Germany and Japan, final reimbursement status) across the eight countries. FINDINGS: Therapeutic impact related to clinical outcomes of the new medicine was a uniform criterion across the eight countries, whereas quality of evidence (under the remit of therapeutic impact assessment) and equity were infrequently cited criteria. Only the German HTA agency mandated that surrogate endpoints be validated in therapeutic impact assessment. All countries except Germany included formal cost-effectiveness analyses within HTA reports. England and Japan were the only countries that specified a cost-effectiveness threshold. Of the 34 medicine-indication pairs corresponding to US top-selling cancer medicines, Germany reimbursed the maximum (34 [100%]), followed by Italy (32 [94%] recommended for reimbursement), Japan (28 [82%] reimbursed), Australia, Canada, England, and France (27 [79%] recommended for reimbursement), and New Zealand (12 [35%] recommended for reimbursement). Of the 18 cancer medicine-indication pairs with marginal clinical benefit, Germany reimbursed 15 (83%) and Japan reimbursed 12 (67%). France recommended nine (50%) for reimbursement, followed by Italy (seven [39%]), Canada (five [28%]), and Australia and England (three [17%] each). New Zealand did not recommend any medicine-indications with marginal clinical benefit for reimbursement. Considering the overall cumulative proportion across the eight countries, 58 (21%) of 272 indications for the US top-selling medicines and 90 (63%) of 144 marginally beneficial medicine-indications were not recommended for reimbursement or reimbursed. INTERPRETATION: Our findings indicate discordance in public reimbursement decisions across economically similar countries, despite overlapping HTA decision criteria. This suggests a need for improved transparency around the nuances of the criteria to ensure improved access to high-value cancer medicines, and deprioritisation of low-value cancer medicines. Health systems have opportunities to improve their HTA decision-making processes by learning from the systems in other countries. FUNDING: None.

Profile of the multicenter cohort of the German Cancer Consortium's Clinical Communication Platform.

Treatment concepts in oncology are becoming increasingly personalized and diverse. Successively, changes in standards of care mandate continuous monitoring of patient pathways and clinical outcomes based on large, representative real-world data. The German Cancer Consortium's (DKTK) Clinical Communication Platform (CCP) provides such opportunity. Connecting fourteen university hospital-based cancer centers, the CCP relies on a federated IT-infrastructure sourcing data from facility-based cancer registry units and biobanks. Federated analyses resulted in a cohort of 600,915 patients, out of which 232,991 were incident since 2013 and for which a comprehensive documentation is available. Next to demographic data (i.e., age at diagnosis: 2.0% 0-20 years, 8.3% 21-40 years, 30.9% 41-60 years, 50.1% 61-80 years, 8.8% 81+ years; and gender: 45.2% female, 54.7% male, 0.1% other) and diagnoses (five most frequent tumor origins: 22,523 prostate, 18,409 breast, 15,575 lung, 13,964 skin/malignant melanoma, 9005 brain), the cohort dataset contains information about therapeutic interventions and response assessments and is connected to 287,883 liquid and tissue biosamples. Focusing on diagnoses and therapy-sequences, showcase analyses of diagnosis-specific sub-cohorts (pancreas, larynx, kidney, thyroid gland) demonstrate the analytical opportunities offered by the cohort's data. Due to its data granularity and size, the cohort is a potential catalyst for translational cancer research. It provides rapid access to comprehensive patient groups and may improve the understanding of the clinical course of various (even rare) malignancies. Therefore, the cohort may serve as a decisions-making tool for clinical trial design and contributes to the evaluation of scientific findings under real-world conditions.

[Patient access - access to innovations in oncology]. / Patient access ­ Zugang zu Innovationen in der Onkologie.

INNOVATIVE ONCOLOGICAL DIAGNOSTICS AND THERAPIES: Compared to other European countries, Germany has a large number of innovative therapy options for the treatment of patients with cancer. Currently, the main challenge in care is to be able to offer these options at the right time to all patients, regardless of their place of residence and treatment setting, who could benefit from innovative therapies. ACCESS VIA CLINICAL TRIALS, MOLECULAR TUMOR BOARDS: Clinical trials are often the first opportunity for controlled access to oncology innovation. Reducing bureaucratic processes and increasing transparency about currently recruiting trials is imperative to allow more patients early access across sectors. The concept of decentralized clinical trials and (virtual) molecular tumor boards is also appropriate to allow more patients potential trial inclusion. COSTS OF INNOVATIVE ONCOLOGICAL THERAPY: The best possible use of a growing number of innovative and cost-intensive diagnostic and therapeutic options for a wide variety of patient-specific situations requires low-threshold transsectoral exchange, i.e., communication between (certified) oncological competence centers and physicians across the broad spectrum of medical care, who are expected to simultaneously treat the large number of German cancer patients in everyday care and cover the entire range of the increasingly complex oncological therapy landscape. INNOVATIVE THERAPIES: DIFFERENT ACCESS IN THE REGIONS: The overdue implementation of digital options for cross-sector collaboration is an absolute prerequisite for giving patients who live farther away from a competence or study center access to innovations that are not available at their place of residence or treatment. NEW FORMS OF CARE: OPTIMIZED ACCESS TO INNOVATIVE CARE: The development and testing of new forms of care requires the participation of all those responsible for the care process in order to jointly improve structural conditions, create sustainable incentives and provide the necessary capacities. The basis for this is an ongoing, concerted provision of evidence on the care situation, e.g. in the context of statutory cancer registration and clinical registries at oncology centers.