In most of the cancer drugs, the MCB threshold is not met at the time of approval when measured using both ESMO-MCBS scales. Since approval status does not translate into a MCB, stakeholders and decision makers should focus on the benefit/risk ratio of anticancer drugs to assure an appropriate allocation of resources in health care systems.

IntroductionLymphoedema is a chronic, debilitating condition caused by an affected lymphatic system. Supermicrosurgical techniques like lymphovenous anastomosis (LVA) have gained popularity because of its minimal invasiveness, better aesthetic outcome, and lower costs in comparison to physical medicine. This systematic review aims to evaluate the clinical effectiveness and safety of LVA in comparison to conservative or other surgical treatments for primary or secondary lymphoedema patients.Materials and MethodsA systematic literature search was performed in four databases in December 2017. We applied a methodological framework based on the HTA Core Model®. According to the grading of recommendations, assessment, development, and evaluation (GRADE) scheme, we synthesized the data on each selected outcome category. The studies were systematically assessed for risk of bias (RoB) using the Risk of Bias Assessment tool for Non‐randomized controlled studies (RoBANS) and the Institute of Health Economics (IHE) Risk of Bias checklist for case series.ResultsA total of 629 citations were identified and five studies were assessed eligible for final inclusion (one non‐randomized controlled trial and four prospective single‐arm studies). Across the studies, 217 patients were enrolled. All studies showed a moderate to high RoB. The strength of evidence for the effectiveness and safety of LVA is “very low.” Due to the methodological shortcomings of the available evidence, no conclusions can be made about the effectiveness of the procedure.ConclusionLVA might be a safe technique for patients with primary and secondary lymphoedema—particularly because no serious complications were reported. Furthermore, LVA may have a role in the prevention of lymphoedema.

The introduction provisional approval strategies increases the approval of anticancer drugs with ambiguous benefit-risk profiles. Thus, in many instances, there is lacking evidence about overall survival (OS) at the time of marketing authorisation. Our objective was to monitor and characterise therapies with ambiguous benefit-risk profiles and identify any postapproval updates on median OS after at least 3 years of approval by the European Medicines Agency (EMA). Methods: We included all originator anticancer drugs with initially ambiguous benefit-risk profiles that received marketing authorisation by the EMA between January 1, 2009 and May 31, 2015. Our monitoring timeframe was at least 3 years after EMA approval. To identify study updates, the following three sources were included: clinicaltrials.gov, European Public Assessments Reports and PubMed. Results: In total, we identified 102 eligible approval studies. Out of these, a negative difference in median OS or no information was available in 43 (42.2%) instances. During monitoring, 14 updates with accessible positive information on OS could be identified. Including monitoring results, there are still 29 remaining therapies (28.4%) where no or negative information (n Z 24 [23.5%] and n Z 5 [4.9%], respectively) regarding OS is present at least 3 years after EMA approval. Conclusion: One-third of oncology drugs with ambiguous benefit-risk profiles at the time of approval fail to demonstrate a survival benefit even after several years of marketing

Summary Background In the past decade, the Ludwig Boltzmann Institute for Health Technology Assessment (LBI-HTA) has introduced two programs: “Horizon Scanning in Oncology” (HSO) and extra medical services (“MELs”), which are to facilitate coverage decisions based on early assessments. This article aims to outline the general process and methods within these two programs. Methods A narrative-descriptive synthesis of the literature was performed to outline the general and LBI-HTA-specific processes and methods of early assessments. Results In total, 79 HSO assessments (2009–2018) and 95 MELs (2008–2018) have been conducted by the LBI-HTA. Recently, additional methods that contribute to European applicability have been introduced into these programs. Conclusions Overall, pre-coverage decisions based on early assessment reports are dependent on the existing evidence. However, the organisation of the health care system and the cross-linking between decision-makers and HTA institutions can have an impact.

Background: Metastatic spinal lesions are difficult-to-treat entities that are most commonly associated with pain and severely reduced health-related quality of life (HRQoL). Within the last 5 to 10 years, radiofrequency ablation (RFA) has emerged as an option in the palliative treatment of vertebral metastases. Objectives: Our review aims to evaluate the clinical effectiveness and safety of RFA, mostly in combination with vertebroplasty, in patients with painful vertebral metastases. Study Design: The design of this study is a systematic review. Methods: We conducted a systematic literature search and a manual search of 5 databases in December 2016. The review applied a methodological framework based on the HTA Core Model®. Data on each selected outcome category were synthesized according to the Grading of Recommendations, Assessment, Development and Evaluation (GRADE) scheme. Risk of bias was assessed using the Institute of Health Economics (IHE) Risk of Bias checklist for case series. Results: We identified 299 citations. After applying the inclusion criteria, a total of 9 studies (4 prospective and 5 retrospective studies) were determined to be eligible. These studies included a total of 583 patients with vertebral metastases who were treated with RFA and, in most cases, received an additional vertebroplasty treatment (n = 437). The studies were categorized as having a moderate to high risk of bias. The strength of evidence was found to be “very low” for safety outcomes and could not be assessed for efficacy outcomes. Current evidence suggests that RFA leads to significant pain reduction. Furthermore, no major complications occurred when using RFA. Limitations: A major concern is the low number of included patients and heterogeneity of study characteristics in most of the studies. The low number of patients also impeded comparison of the effectiveness of RFA alone to RFA in combination with vertebroplasty. Conclusion: According to the available evidence, RFA may be safe and effective, especially for patients with painful vertebral metastases who show contraindications or unresponsiveness to conventional therapies (e.g., radiation) or for those who are at risk of tumor progression. Key words: Radiofrequency ablation, vertebral metastases, metastatic spinal lesions, pain, clinical effectiveness, safety, palliative pain treatment