News

New (pharmaceutical) products are getting increasingly more expensive. As a result of this trend accessibility of pharmaceuticals decreases. However, not all innovation can be attributed to private companies. Subsequently, the role of public institutions like universities, university spin-offs, and publicly funded biotech start-ups is largely being disregarded at the price negotiations. Public funding in the form of research grants, tax incentives, utilisation of clinical infrastructure or regulatory measures make a major contribution.

Article 57 of the proposed Pharmaceutical Legislation (PL, Directive) will require market authorization applicants to publicly declare any direct financial support for R&D received from public authorities. The aim of our research (funded by EU Horizon Europe Grant Agreement Number 101095593) is to increase transparency in (pharmaceutical) R&D to mitigate the risk of market failure associated with information asymmetry by analyzing pharmaceutical R&D expenditures of bringing a new medicine to the market and which factors influence these expenditures and to identify the categories needed to capture direct or indirect public contributions to R&D, to provide a framework for standardized reporting of public contributions and to reduce ambiguity in the interpretation of “direct” and “indirect” public contributions.

The report is the first output of the project HI-PRIX (WP2, sub-deliverable 2.2).

Registries are systems for collecting observational data on patients in a database. They can be used by decision-makers to improve healthcare. However, the number and type of existing registries in Austria are unknown. Therefore, the aim of this report was to provide an overview of the Austrian registry landscape and to present the quality and characteristics of selected registries. Furthermore, it was a goal to examine the utilisation of Austrian registries and whether some areas need further development.

Publication: HTA Project Report No. 157: https://eprints.aihta.at/1489/
Contact: Christoph Strohmaier

For children, adolescents and adults with overweight or grade 1 obesity, group programmes are offered which include content on nutrition, exercise and behaviour. However, it is a challenge to reach the respective target groups and to retain the participants in the programme for the entire duration. Based on the reviewed literature, numerous strategies for recruiting target groups and improving programme adherence were identified. In addition, an overview of existing programmes in German-speaking countries was provided.

Since 2017, three therapies have been approved by the FDA and EMA for the treatment of SMA patients: The results show clinically relevant improvements in motor skills in SMA type 1 patients, especially in patients with early treatment initiation and >=2 SMN2 copies, as well as stabilisation of health status in SMA type 2 to 4 patients. In all studies reporting these outcomes, no significant improvements in respiratory and nutritional function were recorded regardless of SMA type and therapy used, with most studies reporting no change or an increase in the need for ventilation and nutritional support.

20 observational studies and 1 RCT were included in the synthesis, reporting on a total of 1,374 patients. 15 studies investigated nusinersen in 948 patients, one study was identified on onasmenogene abeparvovec and investigated 12 patients, and two studies investigated risdiplam in 221 patients. 193 patients received combination therapy.