Delays in therapy, a lack of specialist knowledge and legal uncertainties in the assessment board threaten to make care for rare diseases more difficult.
Vienna (OTS) – The new evaluation board is intended to enable uniform access to specialized medications in hospitals throughout Austria. But criticism has been leveled at its design for months. It mainly refers to possible time delays, a lack of specific specialist knowledge and legal uncertainties. It is feared that this will worsen the care of patients, especially those with rare diseases. Where exactly adjustments are necessary so that the evaluation board can have a positive effect was discussed at the 15th Rare Diseases Dialogue of the PHARMIG ACADEMY.
“We welcome the creation of an evaluation board that regulates access to therapies for the whole of Austria. However, in our opinion, the clinically active experts have not yet been sufficiently taken into account. However, their relevant, indication-specific medical expertise is essential for a therapy recommendation. This is particularly true in the area of rare diseases. The integration of this specialist knowledge into the decision-making body would therefore have to be routine and binding,” explains Prim. Univ. Prof. Dr. Reinhold Kerbl, Secretary General of the Austrian Society for Pediatrics and Adolescent Medicine and head of the department of the same name at the Hochsteiermark State Hospital in Leoben. Clear wording in the legal text that ensures that the respective medical expertise must form the necessary basis for treatment decisions would provide clarity.
“As the Austrian alliance for rare diseases and an umbrella organization with more than 100 members, we have been calling for uniform access to innovative therapies across Austria for years. In addition to the mandatory involvement of medical experts, the current law on the evaluation board also lacks the involvement of patients for rare diseases,” explains Mag. Elisabeth Weigand, MBA, Managing Director of Pro Rare Austria. The design of the law and the rules of procedure should include the fact that when assessing the additional benefits of innovative therapies, the medical-professional assessment must always be carried out by the members of the European reference networks for rare diseases.
Weigand adds: “It is also mandatory to obtain the expertise of patient experts from the rare disease area. Because no one else can expertly report on the lived experience with a specific indication and on any additional benefits of the intervention being evaluated compared to the treatment standard currently used in Austria as a comparator. The patient advocacy group, which fulfills very important functions, cannot do this.” According to Weigand, there is also concern that there will be a delay in access to new therapies for those affected. To counteract this, Pro Rare Austria calls for the evaluation of new therapies to be initiated early, based on horizon scanning. This process serves to gain early insights into innovative health technologies.
“Time is a crucial factor, especially for people with rare diseases. It is important that decisions are made quickly and that the procedure does not cause delays in receiving therapies that could prevent irreparable damage from the disease,” agree Weigand and Kerbl.
From a legal point of view, decisions by the evaluation board will not bring the hoped-for clarity. Univ. Prof. Dr. Claudia Fuchs, professor at the Vienna University of Economics and Business, says: “It is unclear what binding effect the committee’s decisions should have. On the one hand, these are ‘recommendations’ that are to be ‘applied’, and on the other hand, they are expert reports. In addition, there is neither a right to prompt issuing of a recommendation nor an opportunity to object, especially against negative recommendations. Last but not least, representatives with a compulsory pharmacological or medical background are in the minority on the board. The possibility that they can be overruled in decisions exposes inconsistencies in the existing drug procurement system and provides an unstable basis for therapy recommendations, especially when it comes to rare diseases.”
While medical care in hospitals should be provided according to the latest scientific standards, the outpatient area is based on the criteria of necessity. Mag. Gunda Gittler, MBA, aHPh, head of the Barmfühlen Brüder pharmacy in Linz and vice president of the Austrian Society for Hospital Pharmacy, sees special challenges in care, especially in the case of drug specialties at the interface between the extramural and intramural areas: “The evaluation board means a fundamental change for medication purchasing in the hospital sector, which is already coordinated by the eleven hospital purchasing cooperatives. However, this new board creates even more uncertainty, particularly with the so-called interface products, as no corresponding budgets were planned for subsequent decisions that could potentially conflict with financing. For these reasons, hospital operators could delay the use of such new therapies until there is a recommendation from the board.”
With regard to the goals of the committee, Priv.-Doz. Dr. Robert Sauermann, deputy head of the “Medicines Contract Partner” department in the umbrella organization of Austrian social insurance providers, states: “The evaluation board is intended to close an important gap in Austria, as has long been a reality in most countries in Western and Northern Europe. Not only the doctors concerned, but also the healthcare system must deal with new medications in a structured and evidence-based manner in order to enable more uniform and well-founded access to therapies. Because well-regulated, comprehensible cost assumption is important in all sectors. Now it’s all about good implementation. In particular, early involvement of the board with new medications will be very important in practice.”
“Assessment boards for highly specialized medicines are among the international standards for good reasons. They serve transparency and ultimately contribute to fairness in the healthcare system. Access to medically innovative therapies must be equal for everyone in Austria. The board will make its recommendations based on scientific evidence, against the background of the sharp increase in the number of specialized therapies in the future. This pooling of expertise will support doctors with complex issues and relieve the burden on hospital operators in many ways,” explains ao. University Prof. Dr. Herwig Ostermann, managing director of the national research and planning institute in the healthcare system Gesundheit Österreich GmbH.
The pharmaceutical industry is open to medical evaluation of innovative medicines. This explains Dr. Ronald Pichler, Head of Public Affairs & Market Access at PHARMIG, the Austrian pharmaceutical industry association: “The primary goal in the healthcare system is to offer patients the best possible care and to enable them to receive the most suitable therapy in a timely manner. A uniform use of innovative medicines across Austria, which is based on the respective international state of science, is therefore welcomed. In order for the future evaluation board not to become a board that prevents medical innovations, fundamental modifications are necessary.” Because this new board also creates a lot of ambiguity regarding planning and liability for pharmaceutical companies. All of this also has an impact on the attractiveness of Austria as a market for innovations and thus also for the upstream implementation of clinical trials in Austria. “In this regard, it is important to make the appropriate adjustments quickly,” said Pichler.
You can access the PHARMIG ACADEMY newsroom here.
About PHARMIG: PHARMIG is the voluntary interest group of the Austrian pharmaceutical industry. The association currently has around 120 members (as of May 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.
About PHARMIG ACADEMY: The PHARMIG ACADEMY is the training and further education institute of PHARMIG, the Austrian pharmaceutical industry association. It offers seminars, courses and training on all healthcare topics. The offer is based on current developments and is aimed at everyone who is interested in or works in the health sector. The format of the Rare Diseases Dialogue offers all those affected, interested and relevant stakeholders an open discussion platform on current topics in the field of rare diseases.
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
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