Individualized therapies and the integration of the patient’s perspective are becoming increasingly important.
Vienna (OTS) – February 29th is Rare Disease Day. On this occasion, the Austrian pharmaceutical company AOP Orphan Pharmaceuticals GmbH (“AOP Health”) takes a look at the challenges that people with rare diseases face and highlights the trends in their treatment.
With over 30 million Europeans affected, including 400,000 Austrians, rare diseases are becoming an increasingly pressing health issue. What is alarming is that there are still no specific treatment options for 95 percent of these diseases. AOP Health, specializing in the research and development of therapies for rare diseases and in intensive care medicine, has been committed to this market segment for over 25 years in order to be able to offer medical solutions to those affected.
Research is the key to new therapies
In order to create new treatment options, intensive research programs are necessary, which are currently inconceivable without private initiatives. In Austria, 50.4 percent of all research is carried out by private companies(1). In the area of drug development, this proportion is even higher, because the approval or marketing of drugs is applied for or carried out exclusively by pharmaceutical companies and biotech companies.
In order for new therapies to be developed, framework conditions that promote research and development are necessary, as Martin Steinhart, CEO of AOP Health, emphasizes: „If we want to ensure that the supply of medicines in Europe remains guaranteed, we need regional research programs, a strong pharmaceutical industry and clear regulations that promote the development of new therapies and make research attractive for a wide range of actors.
Steinhart is therefore critical of the planned EU pharmaceutical legislation, because the draft includes a shortening of document protection and market exclusivity. This means that companies have less time to recoup the cost-intensive developments through sales. The CEO of AOP Health continued: „Shorter protection periods during which pharmaceutical companies can exclusively market their developments make it even more difficult for those companies that want to get involved in the already challenging field of rare diseases. The planned legislation could make the situation in Europe more difficult for patients and weaken the business location.
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Trend towards individualized therapy
The trend towards personalized medicine is also clearly noticeable in the area of rare diseases. Improved diagnostic procedures enable the clinical pictures to be determined more precisely. This means that clinical pictures can be divided into subgroups with specific characteristics and treatment needs, and particularly rare diseases or forms of disease can be identified. Treatment has also changed significantly in recent years: Newly discovered therapeutic approaches are bringing individualized therapy concepts to the fore. Molecular testing and targeted therapies can improve prognosis and treatment. The “one size fits all” approach is a thing of the past. AOP Health is integrating these new insights into its development programs.
Bianca Tan, Director of Therapeutic Area Cardiology & Pulmonology at AOP Health explains: „Our goal is to find answers to previously open medical questions for our patients. In our research, we therefore try to understand the mechanisms of the disease at a molecular genetic level in order to perhaps even be able to modify the disease. In addition, we are examining whether synergies caused by combination therapies can be of benefit to patients.
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Treatment in specialized centers brings better results.
In addition to individualized therapy, prompt referral of patients to specialist centers is also crucial. Prof. Irene Lang, specialist in pulmonary vascular diseases at MedUni Vienna explains: “Patients with rare diseases benefit enormously from treatment in an expertise center. On the one hand, there are experts there who deal with those affected on a daily basis and therefore have a lot of experience in diagnosing and selecting therapies. On the other hand, they offer the necessary diagnostic and therapeutic infrastructure.
“ She therefore recommends that colleagues outside of specialist centers immediately refer patients to an EU-approved center if rare diseases are suspected. Long continue: „It is crucial for those affected to receive the correct diagnosis and treatment quickly. Treatments for pulmonary hypertension, for example, are expensive and time-consuming, and it causes damage to our healthcare system if the wrong patients receive the expensive medication.
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Let’s talk with Patients, not above she
Another trend that can be observed throughout medicine is the increasing inclusion of the patient perspective in treatment, but also in research work. Represented by so-called patient advocacy groups, those affected not only get involved in the selection of therapies, but also in the creation of study designs. However, they still face hurdles in their work.
Claas Röhl, chairman and founder of the patient advocacy group NF Kinder and board member of Pro Rare Austria, says: „The current regulations, which are actually intended to protect patients, make it difficult for us to be involved in the development of medications and therapies.
” It is essential that patients are seen as experts in their illness by all players in the healthcare system. He adds: „We want our concerns and needs to be included because it is our lives that are at stake here. Our perspective is unique and cannot be covered by scientists. We rely on advanced training programs, some of which are at university level, which enable us to speak with the treatment teams, but also with pharmaceutical companies, on an equal footing in order to be heard and to communicate the needs of the patients so that research is geared towards them.
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Those:
1) Austrian Research and Technology Report 2023
About AOP Health
The AOP Health Group includes several companies, including AOP Orphan Pharmaceuticals GmbH based in Vienna, Austria (“AOP Health”). The AOP Health Group is the European pioneer in integrated therapies for patients with rare diseases and in intensive care medicine. Over the last 25 years, the group has developed into an established provider of integrated therapy solutions, operating from its headquarters in Vienna, its subsidiaries and representative offices throughout Europe and the Middle East, and through partners worldwide. With the claim “Needs. Science. Trust.”, the basis of success is summed up: trust through continuously high investments in research and development and a very consistent and pragmatic focus on the needs of all stakeholders – in particular the patients and their relatives as well as the treating doctors* Inside.
Questions & Contact:
BY Isolde Fally
Isolde.Fally@aop-health.com
+43-676-500 4048