Announcements

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.

Until a few years ago, there were no specially designed rehabilitation centres for children and adolescents in Austria, which differ from adult rehabilitation programmes with regard to the supply structure (e.g.; structural requirements or rehabilitation measures). In 2010, a need for a total of 343 beds for 11 indicator groups and an additional 50 beds for the relatives was indicated within the scope of the planning phase of the child and adolescent rehabilitation centres in four Austrian supply zones by 2020. The six newly built centres will now be evaluated on a regular basis. Therefore, the present report provides an overview of international evaluation methods and (standardised) evaluation instruments that can be used for future evaluations.

In recent years, payments made by the pharmaceutical industry to doctors and medical institutions have increasingly been questioned critically. In a systematic analysis, the websites of all PHARMIG member companies (113) were once again searched for information on financial contributions to Austrian healthcare professionals and institutions in 2018. In 2018, a total of 138.3 million euros flowed from PHARMIG member companies to doctors and medical institutions as well as to clinical research and observational studies in Austria. In 2015, the total amounted to 104.1 million euros.

Although the number of Pharmig member companies declaring grants increased only slightly compared to 2015 (2015: 69/113, 2018: 76/115), in 2018 almost one third (32.8%) more grants were made - an increase of 34.2 million euros. In 2018, the share of donations to physicians in the total amount was 7% lower than in 2015. Since this figure corresponds exactly to the increase in the category research and development, a re-declaration can be assumed here. The actual willingness to disclose financial contributions, especially from doctors, is still low - the individual disclosure rate (by name) averaged only 21.1% (2015: 21.9%), which corresponds to 18.5% (2015: 17%) of the amounts paid out. In contrast, the willingness to disclose payments to medical institutions by name (institution) was still significantly higher on average at 49.4% (2015: 50.2%).