Addressing Unmet Patient Needs

The Novartis Group has been present in Hungary for more than 25 years. Country President Matt Zeller, the head of its local subsidiary, Novartis Hungary Ltd. elaborates to Diplomacy&Trade on the company’s activities on the Hungarian market with special regard to medical research and development (R&D), an area where the Group is a leading global force.

Research collaborations in Hungary cover the full Novartis therapeutic spectrum. Each year, the company coordinates and oversees around a hundred clinical development programs, offering the most modern therapies for more than a thousand patients in Hungary every year. “We focus on five therapeutic areas: cardiovascular diseases, oncology, hematology, neurology and immunology. Our work is powered by a toolkit of five R&D approaches – two established platforms (chemistry and biotherapeutics) plus three advanced platforms (xRNA, radioligand therapy and gene and cell therapy) that will play an increasingly important role in making new medicines. We are looking for innovative solutions to problems that have a significant public health impact. One example is cardiovascular diseases which are the leading causes of death in Hungary,” the Country President highlights.
He adds that they also seek to unlock their potential to treat rare diseases that affect a small number of people but can change the entire lives of the families involved, such as children diagnosed with SMA, or spinal muscular atrophy, and gene therapies for patients with inherited retinal disease, a condition that threatens blindness.

Coordination center
In May of this year, the news came that Novartis was expanding its presence in Hungary by coordinating clinical operations across a cluster of countries. As Matt Zeller explains, the Group organizes its research & development activities worldwide into regional centers. The region to be overseen from Budapest will be a diverse group of approximately a dozen countries. “Hungary is an excellent location geographically and, more importantly for drug development, it is one of those places where we can collaborate with the best minds. We have a lot to build on: 8-12% of new trials are initiated in Hungary that has reached more than 10,000 patients in the last decade, with 65 clinical trials currently underway in the country. This was the main reason why we decided that Hungary was the best place for the coordination center. Our company's investments in the forthcoming period, in addition to strengthening international innovation, will help Hungarian patients gain access to state-of-the-art treatments and provide professional development for doctors, thus contributing to improving the functioning of the healthcare system. As a coordination center, we will also have access to earlier phases of research elsewhere, which will strengthen the research pipeline.” Novartis Hungary will not only overview, but also participate in the research it manages. This role will therefore provide the home country's professional community with more tasks and access to new ideas, methods and markets. Not only can this help doctors' professional development, but it also gives Hungarian patients faster access to a wider range of new therapies.

Addressing unmet patient needs
Personalization and digitalization are major trends in medical research globally. As a drug development company, Novartis – with its very comprehensive R&D portfolio – is looking for solutions for a wide range of diseases and disease groups. “Our current portfolio of therapies reaches 800 million people worldwide. Thus, we have to ask ourselves what impact we want to achieve with our products in the future. We are looking at the unmet patient needs that are present and will be present in the future, and how we can address them with specific therapeutic solutions. One example is gene therapy, which is one of the most prominent areas of science and innovation in Hungary and around the world,” the Country President notes.

PUBLIC-PRIVATE PARTNERSHIP

Novartis Hungary Ltd. has signed a cooperation agreement last year with Semmelweis University (SU) and the Ministry of Innovation and Technology to promote innovation in Hungarian public health through their unique public-private partnership. The three pillars it is built on are summarized by the Country President:
"Gene therapy is one of the most exciting areas of global science today, and one part of our agreement is to ensure pathway to Hungarian leadership in cell and gene therapies. Semmelweis University became a certified ophthalmology gene therapy center of excellence, one of the few leading centers in the world that can successfully use gene therapies to treat both spinal muscular atrophy (SMA) and inherited retinal diseases (IRDs). SU is also the largest laboratory involved in genetic researches. SU as an IRD center has focused intensively on building broad genetic testing/screening tools and architecture within Hungary to ensure that identified IRD patients have access to the most appropriate therapies available.
Building on the success of the program above, Novartis has also created a framework agreement with SU to enable execution of broad genetic testing services. The scope includes the most detailed Whole Exome Sequencing techniques as well. We have jointly launched a genetic testing program for children suffering from Acute Infectious Diseases (AIDs) to support patients and caregivers in getting a diagnosis faster than ever before.
The other is to accelerate scientific-clinical research by better harnessing artificial intelligence and machine learning-based methods. Our aim is to strengthen collaboration between the signatories to increase access to new innovation with SU as a key regional site and to accelerate innovation in research through new technologies like trial digitization, new patient recruitment, new digital interfaces, etc. For example, Semmelweis University has a uniquely rich and nationally representative CVD data on set to draw on. Based on this, AI can potentially be used to determine the extent of the problem and then to estimate who is likely to develop cardiovascular disease, the aim is to find the most effective intervention in the treatment.
The third is to prevent and treat cardiovascular disease (CVD) at an early stage by developing new types of collaborations. CVDs affect one in five people in Hungary and kill about 60,000 people each year – one in two deaths in the country, compared with one in three in the EU average. Novartis, in the framework of this collaboration, set out to determine which interventions would have the greatest impact on reducing CVD among patients in Hungary. We are approaching CVDs in a completely new way: using the latest research and innovative data analytics, artificial intelligence and machine learning-based solutions, as well as by educating patients and healthcare workers. The program aims to reach 50,000 patients in its first three years and find new ways to prevent and treat CVD on a national scale. After only six months, we are already seeing the first results of regional mapping of the population's lipid levels, and an experimental, first-of-its-kind lipid management center has been established at Semmelweis University to treat selected patients. We are proud to be part of this unique 'cardiovascular intervention' partnership, which clearly demonstrates Hungary's commitment to testing new public and private models to improve the health of its citizens."