New Approaches for Fostering Rare Disease Research
Although 30 million people in Europe are affected by a rare disease, there are currently no therapies available for 95% of the 7,000 known rare diseases.1. Leading experts from the healthcare and business sectors discussed ideas for successfully bringing innovative therapies to patients at a round table hosted by the pharmaceutical company AOP Orphan.
Vienna, 18 October 2021. In Austria, 400,000 people suffer from a rare disease, for the majority of which there are no effective therapies. Particularly from a patient's point of view, it is important to “invest in research for missing treatment options and to keep the patient's needs in mind during the research process” as Werner Zinkand, Chair of the MPN Advocates Network, pointed out.
This important topic was addressed at a round table in Vienna organized by AOP Orphan. Over the last 25 years, the international pharmaceutical company has become an established provider of integrated therapies with a focus on rare and complex diseases. High-level representatives from the business and healthcare sectors, along with members of AOP Orphan’s management, discussed new approaches to bring innovative therapies to patients.
Daria Julkowska, from the European Joint Programme on Rare Diseases, confirmed that the EU's efforts are also aimed at utilizing more national capacities for rare disease research. “We are working on strengthening national groups. In Austria, the Austrian Science Fund, the Austrian Institute of Technology (AIT) and the Ludwig Boltzmann Society are making valuable contributions.”
Medical research comes with high financial risks
“We reinvest up to 20% of our turnover in research and development every year and employ more than 70 highly qualified scientists in Vienna. We produce 80% of our drugs in Europe,” said Georg Fischer, CEO of AOP Orphan. Austria as a research location offers advantages for pharmaceutical companies, but also challenges, as Fischer pointed out at the round table: “Pharmaceutical research is a high-risk business. Prior to a drug being authorized, we incur high costs while dealing with a lot of setbacks. Only one in 80 drug candidates to treat rare forms of cancer is approved by authorities.” “In order to take on this high risk and offer patients innovations, we need a new common understanding of research and innovation” Fischer explained. For instance, the company depends on close collaborations with partner companies, especially in rare disease research, “while we do conceptual research in-house, we still require external resources for preclinical studies or the chemical engineering of our drugs needed to generate the data we require.”
More supply security through targeted funding
Alexander Herzog, General Secretary of PHARMIG, the Association of the Austrian Pharmaceutical Industry, sees opportunities for Austria as a location for innovation. For example, an allowance for certain investments, tax relief for manufacturing companies and the funding of domestic research are important measures for increasing Austria's competitive advantage and supply security. “Especially in these times, which are challenging for everyone, there is a strong focus on health. The quality of patient care of course also depends on the economic situation. Therefore, factors impacting supply cannot be isolated from economic factors, and vice versa,” according to Herzog.
A separate funding pot for specialized life sciences?
“Targeted funding and cooperation initiatives are needed to incentivize research in Austria and support companies,” says Dr. Florian Moosbeckhofer, Head of the Innovation and Digitalization Department of the Austrian Economic Chamber. “Existing funding instruments, such as the research premium, are essential and need to be easier to use for contract research. In addition, digitalization offers a wide range of opportunities for pharmaceutical companies, particularly in terms of data.” He can picture a separate funding pot for specialized ‘life science calls’ and is also asking for an appropriate assessment of the “in-house” factor in the research premium.
Patients across Europe have the right to optimized care
200 drugs are approved under the European Union's “orphan designation” for rare diseases. At the same time, with a total of 7,000 different rare diseases, there is still a lot of work to be done. “Patients across Europe have the same rights and have a right to optimized care. The networking of member states and companies is also important to facilitate new findings. We are currently working on this in the European “big data initiative,” says Christa Wirthumer-Hoche, head of the Austrian medical market regulator AGES.
Martin Schaffenrath, member of the Administrative Board of the Austrian Health Insurance Fund and rapporteur for the EU Pharmaceutical Strategy on the European Economic and Social Committee, explains: “The COVID-19 pandemic has not only highlighted the pharmaceutical industry's capacity for innovation and the importance of functioning and resilient supply chains, it has also shown how important it is that medicines get to where they are needed: to the patients.” To achieve this, it is essential that we “find solutions that strike a balance between funding medical innovation, avoiding the abuse of the incentive system, and providing safe, high-quality yet affordable drugs. At the same time, the financial sustainability of national healthcare systems must be ensured and sovereignty respected.”
A call for a new understanding of innovation
To further increase the appeal of Austria as a research location for the pharmaceutical sector, Georg Fischer believes that we need a “common understanding among all stakeholders involved in research and licensing. This is the only way we can deal with the high risks associated with developing therapies and develop new, creative approaches.”
Round table participants:
- Georg Fischer, CEO, AOP Orphan Pharmaceuticals GmbH
- Alexander Herzog, General Secretary of Pharmig, the Association of the Austrian Pharmaceutical Industry
- Daria Julkowska, representative of the European Joint Programme on Rare Diseases
- Sonja Kato, moderator
- Petra Lanz, CCO, AOP Orphan Pharmaceuticals GmbH
- Florian Moosbeckhofer, Head of the Innovation and Digitalisation Department of the Austrian Economic Chamber
- Nina Roth, Head of Corporate Communications, AOP Orphan Pharmaceuticals GmbH
- Martin Schaffenrath, member of the Administrative Board of the Austrian Health Insurance Fund and rapporteur for the EU Pharmaceutical Strategy on the European Economic and Social Committee
- Andreas Steiner, Group CEO, AOP Orphan International AG
- Barbara Weitgruber, Head of Innovation, Austrian Federal Ministry of Education, Science and Research
- Christa Wirthumer-Hoche, head of the Austrian medical market regulator AGES.
- Werner Zinkand, Chair of the MPN Advocates Network
AOP Orphan Pharmaceuticals GmbH is an international pharmaceutical company focused on rare and special diseases. Headquartered in Vienna, Austria, with subsidiaries and representative offices across Europe and the Middle East, the company has become an established provider of integrated therapy solutions over the past 25 years. This development has been made possible by a continually high level of investment in research and development on the one hand and a highly consistent and pragmatic orientation towards the needs of all its stakeholders on the other – especially patients and their families, but also the doctors and care professionals treating them.
AOP Orphan Pharmaceuticals GmbH
Leopold-Ungar-Platz 2, 1190 Vienna
+43 676 3131509