A review of the challenges and proposals for improving patient access to advanced therapeutic medicinal products in Austria

Executive Summary

RARE IMPACT is a multi-stakeholder initiative working to improve patient access to advanced therapy medicinal products (ATMPs). This patient-focused initiative aims to assess challenges and propose actionable solutions to concerns regarding access to these transformative rare disease treatments in Europe. Through engagement with health technology assessment (HTA) agencies, regulatory bodies, payers, patient groups, clinicians, manufacturers and other experts across Europe, RARE IMPACT partners identified challenges and have proposed solutions for better access to ATMPs in Europe.

This report aims to stimulate multi-level stakeholder discussions on patient access to ATMPs and is not intended to capture all challenges to patient access to ATMPs. The RARE IMPACT initiative was launched at the European Conference on Rare Diseases and Orphan Products in 2018.

Patient access to orphan products in Austria has been relatively good with a >90% access rate to EMA approved orphan products, similar to that seen in France. There are currently no specific assessment or reimbursement pathways for ATMPs in Austria. While there are some aspects of the current country processes that help manage barriers to ATMP access, there is recognition that changes are required in order to ensure sustainable access

This recognition is borne out in the legislative changes taking place in Austria which are intended to create a more standard pathway of assessment and negotiation. However, it is possible that this will not include ATMPs. Within the current system, ATMPs are considered hospital products, which do not go through HTA assessment. Hospital products are instead assessed and negotiated directly between the manufacturer and the hospital, which places a large burden on the hospitals, is inconsistent, and time and resource demanding. Given that there is ongoing reform in Austria, there is an opportunity for some of these challenges to be addressed.

Under the current system, funding for ATMPs is most likely to be via reformed security funds (health insurance), but this would pose challenges, such as how to allocate funds between hospitals/treatment centres. For now, affordability does not seem to be an issue in Austria, although as the number of ATMPs on the market increases, it may represent a greater barrier, creating the need for innovative methods to reduce financial burden on regional budgets. ATMPs are also associated with higher treatment costs, and there is a lack of clarity as to what exactly these costs might include.

However, since affordability is not an immediate issue, Austria is in a good position to anticipate and prepare in advance for potential future challenges

The availability of ATMPs for rare disease in Austria is relatively good and does not pose a significant challenge under the current process. Patients have received access to treatment via cross-border initiatives, which work both ways in the country, and hospital exemption policies are in place and clearly defined.

There is recognition that future access to products for rare disease (including ATMPs) is uncertain in Austria. In response, the Austrian government has established the National Centre for Coordination of Rare Diseases (NKSE) to focus on improving access to products for rare disease. Accessibility challenges would be best managed with more standardised assessment and decision-making processes, which may not be straightforward to implement but would help in creating a sustainable pathway for patient access. These solutions are feasible with minimal interruption to current patient access procedures.

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