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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

Troponin and D-dimer point of care tests can be used to aid the diagnosis of patients with symptoms suggestive of suspected acute coronary syndrome and venous thromboembolism respectively. Potential advantages of point of care tests include faster turnaround time, reduced length of stay and reduced unnecessary hospitalisation/further testing. This report aimed at evaluating the clinical benefit of these two point of care diagnostics in various settings. Two overview of reviews, two guideline synopsis and interviews with experts were hereby carried out.

Cervical cancer screening has been revolutionized by the discovery that nearly all cervical cancer diseases are caused by persistent infection with human (high-risk) papillomaviruses (HPV). Along with this, the development of new HPV-based test systems internationally led to a change in existing cervical carcinoma screening strategies. The purpose of this report was to provide a basis for the decision to implement the HPV test in cervical carcinoma screening in Austria.