Announcements

Worldwide, 82 particle therapy facilities are currently in clinical operation; further 37 centers are under construction or in planning. This means that the number of centres worldwide has more than doubled since 2013. In Europe alone, 27 centres are currently operational (2013: 14). With such a large number of treatment centers, it should be assumed that the underlying clinical evidence on patient benefit is convincing. The update of the LBI-HTA report 2013 has the task to give an overview of secured indications as well as indications that are only recommended under clinical research. Only a hand search for HTA reviews and systematic reviews was conducted. The identified reviews articulate in unison that the available - mostly retrospective - studies are of low quality and insufficient to make conclusive statements on the added value of proton or carbon ion therapy. Only a few indications are recommended for proton or carbon ion therapy (chordomas and chondrosarcomas, uveamelanoma, pediatric tumors in the base of the skull, brain and head and neck tumors) to avoid secondary tumors. Further indications, with curative intention and non-metastatic, are only recommended under conditions of prospective clinical studies.

 The establishment of a HSS/ Horizon Scanning System for Covid19 interventions has the intentions of informing health policy makers at an early stage which interventions (vaccinations and drugs) are currently undergoing clinical trials and to monitor them over the next few months in order to support evidence-based purchasing if necessary.

Based on international sources (information gathered by the WHO, preliminary work by other international institutions, publications) first selective searches by means of searches in study registries are carried out for information on clinical studies in humans and the state of research.  Thereafter “vignettes” (short descriptions) are developed for those products that are already in an "advanced" stage.  Subsequently, the products are monitored with regard to the status of the clinical studies up to approval and finally evaluated for their benefit and harm.

All work steps are conducted in close international (European) cooperation.

Real-world data can provide information on the quality of health care and can inform health care system decisions. The present data analysis was focusing on the health care at the end of life of cancer patients, based on Austrian data (2012 to 2016) from all nine federal states. In the period under review, 283,228 persons with cancer diagnoses were admitted to a publicly financed hospital. Of these, about 29% (80,818) persons died. The proportion of patients who died in the inpatient sector was homogeneous across Austria (~61%) with the exception of Vienna. About 13% of deceased patients were admitted to the hospital two days before their death. With regard to the analysis of special oncological treatments (radiotherapy, chemotherapy and monoclonal antibodies) within 30 days before death, slight variabilities could be observed across the Austrian federal states.

In June 2017, Nusinersen (Spinraza®) was approved broadly  – for all SMA Typologies (despite lack of evidence) –  by the European Medicines Agency (EMA) for the treatment of chromosome 5q13 (5q)-associated SMA. The question of the update presented here is which scientific evidence from clinical intervention or observational studies for the medium (or long-term) benefit of Nusinersen (Spinraza®) in children and adolescents > 6 years with SMA "late onset" (SMA 2 + 3) is available. Three publications on the question (SMA 2 + 3 with a total of 40 patients) were identified. The available evidence for medium-term (> 12 months) follow-up data comes from a single prospective sponsor-funded ISIS CS12 study, as well as from a small retrospective study (of poor quality). Even after more than two years after the approval of Nusinersen, the evidence for this patient population is of extremely low quality, lacking validity.

Experience in routine clinical practice may differ from that seen in a controlled clinical trial. Although randomised clinical trials (RCTs) are considered to be the standard for generating clinical evidence, they lack generalisability to real-world evidence due to selected patient populations (strict and complex enrolment criteria). Therefore, the use of real-world evidence (RWE) to evaluate the effectiveness and safety of medical interventions is gaining interest. As there is limited evidence regarding the real-world effectiveness of immunotherapy in Austria, we conducted a retrospective pilot study in six Austrian hospitals to present data on patient characteristics, effectiveness and safety from real-world practice in an NSCLC population treated with anti-PD-1/PD-L1 monotherapy.

A possible measure for quality assurance in day surgery is minimum volume standards (MVSs). While the majority of research in this context observes a positive relationship between volume and outcome in the form of statistical correlation, the data availability on surgery specific and evidence-based minimum volume thresholds is low. Aims of the present report were to show the fundamentals of the MVSs theory, to illustrate possible internationally implemented MVSs frameworks in day surgery, and (in the course of a systematic review) to examine whether MVVs for specific day surgeries lead to better results in terms of efficacy and safety.

Publication: LBI-HTA Project report No. 125: http://eprints.hta.lbg.ac.at/1225/
Contact: Michal Stanak, Christoph Strohmaier