Time delays, a lack of specialist expertise and funding endanger treatments for those affected by rare diseases. Integration of expertise centers necessary.
When making therapy recommendations in specific areas such as rare diseases, medical expertise must be taken into account. If it is missing, an assessment is insubstantial and there is a risk that economic considerations rather than medical considerations will be in the foreground.
Mag. Alexander Herzog, Secretary General of PHARMIG
Vienna (OTS) – The assessment board passed by law last year is intended to standardize access to innovative therapies in the Austrian hospital sector. A detailed analysis of the text of the law has provoked loud criticism from various disciplines. Time delays, but also the lack of specific specialist knowledge, give rise to fears that patient care will not be improved. Particularly when it comes to the area of rare diseases, where new therapies are constantly coming onto the market, it would be important to involve centers of expertise in the decision-making process of this committee.
Alexander Herzog, Secretary General of PHARMIG, takes the sum of these concerns as an opportunity to warn on today’s World Day for Rare Diseases: “When making therapy recommendations in specific areas such as rare diseases, medical expertise must be taken into account. If it is missing, an assessment is insubstantial and there is a risk that economic considerations rather than medical considerations will be in the foreground.“
The draft law states that only 3 of the 25 planned members of the evaluation board should come from the pharmacological or medical field. Specialists can be called in, but they don’t have to be. “As if that wasn’t enough, the perspective of the patients on the board is also neglected. “A single member of the patient advocacy group is planned, but without voting rights,” says Herzog, taking up the criticism that was leveled across the board at the end of last year with regard to the evaluation board initiated by Minister Rauch.
In addition, access to vital therapies could be delayed due to up to additional five-month post-approval assessment periods and a pre-existing recommendation based on Health Technology Assessments (HTAs). This period can also be extended without any limit. Finally, there are no provisions for uniform nationwide financing for such therapies, which not only calls consistent treatment into question, but actually endangers it.
“The challenge with rare diseases lies in the small number of people affected by each disease and the therefore limited knowledge about these diseases. This makes the research and development of medical therapies in this area so challenging and requires the bundling and international networking of this expertise. In the so-called expertise centers for rare diseases, medical experts with the necessary knowledge work to carry out a precise and subject-specific assessment of new, specialized drugs,” explains Herzog.
As highly specialized clinical facilities, expertise centers for rare diseases make an essential contribution to the treatment of those affected. They act as supra-regional, central contact points for defined groups, are closely networked with each other and share the necessary knowledge about this disease area and the treatment experiences and findings based on it. Austria already has numerous institutions nationwide that are either associated centers or full members of the 24 so-called European Reference Networks (ERN) and would make their expertise on rare diseases available in the assessment board. The current list of all centers is available on the health portal.
“A clear formulation in the legal text that ensures that the respective medical expertise must form the necessary basis for treatment decisions would create clarity for all those affected and ensure that quick and correct recommendations are made before they are applied throughout Austria,” says Herzog with reference to the valuable contribution of the expertise centers.
The extent to which the evaluation board will serve as a barrier or bridge for access to therapies in the future will be the focus of a discussion by experts from the health sector as part of the 15th Rare Diseases Dialogue of the PHARMIG ACADEMY on May 7, 2024. Further information is available available here.
About PHARMIG: About PHARMIG: PHARMIG is the voluntary interest group of the Austrian pharmaceutical industry. The association currently has around 120 members (as of February 2024), who cover a good 95 percent of the medication market. PHARMIG and its member companies stand for the best possible security of supply of medicines in the healthcare system and ensure social and medical progress through quality and innovation.
Questions & Contact:
PHARMIG – Association of the Austrian Pharmaceutical Industry Peter Richter, BA MA MBA Head of Communications & PR +43 664 8860 5264 peter.richter@pharmig.at www.pharmig.at
