Background: Non-interventional studies (NIS) are observational studies that evaluate medicinal products and medical devices in routine care after market authorisation in their approved indications. For some years now, there has been a critical discussion about their role, whether they are a valuable source of knowledge or a marketing instrument. In Austria, NIS are not subject to approval, but they are subject to notification. The NIS must be registered in a study database (register), in which some (but not all) information is also made publicly accessible.

This systematic analysis addresses the questions of how many non-interventional studies are being conducted in Austria, who is responsible for them and what the knowledge interest of the registered NIS is.

Method: For the first research question, the BASG/AGES NIS database (https://www.basg.gv.at/gesundheitsberufe/klinische-studien/nicht-interventionelle-studien-nis) was systematically evaluated, as of 11th June 2021. To answer the second research question, it was necessary to refer to the summaries of the final reports that had to be deposited in the BASG/AGES database after the completion of a NIS. This was done with a cut-off date of 16th June 2021.

Result: As of 11th June 2021, 462 NIS with a planned number of patients of 757,948 were registered in the BASG/AGES NIS database. Of these, 55,708 persons were identified as Austrian patients. Most of the NIS were carried out by pharmaceutical companies themselves (292/462). The –in the conduct of NIS- 15 most active pharmaceutical companies are responsible for 164 of the 462 NIS (35.5%). To this end, a total of 119,238 patients (3,792 from Austria) were/are recruited, which corresponds to almost a doubling compared to the initial report/update (2016/2017: 59,438). A total of 31 organisations perform/performed ≥ 4 NIS or were responsible for 59.3% of the total number of NIS. For 161 completed NIS, 156 short versions of the final reports were available in the NIS database. These included three post-authorisation safety studies (PASS) and no post-authorisation efficacy studies (PAES). The most common research questions were about the efficacy under routine conditions, on tolerability and practicability, as well as on the long-term safety of the study medication.

Conclusions: In 2020, the (German) National Association of Statutory Health Insurance Physicians (KBV) listed four aspects according to which NIS should be evaluated critically. Three of these aspects (unusually high numbers of participants, studies on long established and well tested drugs, several different/very similar NIS on the same drug) could be derived from the Austrian NIS register. Regarding the fourth aspect (unusually high reimbursements), there is no publicly available information. Moreover, the formal quality of the register has not improved. As in the first report and in the first update, the conclusion is that the existing NIS register in its current form does not contribute to supporting the critical public (including patients) in identifying the research questions and the knowledge gain of non-interventional studies.