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The pool of patients suffering from an individual rare disease is very small. Drug development in this area is complex and the risk of failure is much higher than for other diseases. Basic research and the search for treatment options is neglected and often not deemed worth the investment. Ninety-five percent of all rare diseases are still without any treatment options. On October 1, 2021, panelists at the European Health Forum Gastein took a closer look at this issue and discussed potential future approaches to form new partnerships and collaborations to change this dramatic situation for patients.

Vienna, October 6, 2021. Why should a research institute or a pharmaceutical company invest time and money in therapy that may not pay off in the long run? Funding is hard to set up and payers may not be willing to cover the expensive treatment, looking at individual costs per patient. Are rare disease patients not worth being cared for?

This multi-dimensional topic was discussed by a panel at the European Health Forum Gastein last Friday. During the session “New Partnership Models for Rare Diseases”, organized by AOP Orphan, representatives from patient organizations, research institutions, pharmaceutical companies, and authorities discussed potential future flagship financing and R&D solutions for accessible care.

Participants agreed that a major problem in Europe is the fragmented access to drugs and treatments across the different countries. As Dimitrios Athanasiou, Board Member of the European Patients' Forum and father of a rare disease boy with Duchenne bluntly put it: “You may just live a few kilometers on the wrong side of the border but you will die because you don’t have access to the medication you need.” “Unfortunately, drugs for rare diseases are not always available in all European countries. Incentives for marketing authorizations are not coupled with access across markets” said Christa Wirthumer-Hoche, Head of the Austrian Medicines and Medical Devices Agency (AGES).

“Access to rare disease innovation for patients across Europe remains a challenge” added Labrini Papageorgiou, Global Access Policy Leader at Roche. She stressed that a positive regulatory environment is just one piece of the puzzle and that it is necessary to set policy frameworks that support sustainable and equitable access to advancement management and care for people affected by rare diseases. There is a need to address infrastructure and capacity gaps, enhance across the life cycle of interventions the coordination and harmonization of evidence generation, and to evolve access procedures in a way that they are able to recognize specificities of rare diseases and reward innovation. Plus, all stakeholders involved should work in close collaboration and strong partnership to build better healthcare systems.

One out of eighty drugs might be successful

At the same time, European countries offer different availability of funding for basic research and drug development. Günther Krumpl, Member of Governing Board at AOP Orphan International AG, who has a strong research background, pointed out that developing an orphan drug takes years and is full of risks. One out of eighty drugs might be successful. Even if you have a good result, the committee may decide that the treatment doesn’t qualify as an orphan drug making subsequent reimbursement discussions very difficult. “You think twice about investing in orphan drugs. Especially if an organization cannot be sure that in the end an orphan drug designation will be granted.”

Christa Wirthumer-Hoche was optimistic: “Let´s use the momentum! New legislations including the Orphan Drug Regulation, with incentives for drug development and provisions for marketing activities are currently under review.”

Equal access for patients: What are new approaches to reward innovation for rare diseases?

Michael Freissmuth, Chairman at the Medical Institute of Pharmacology at the University of Vienna: “I encourage trans-European solutions for equitable access in Europe. We need a fair price. This can be solved with risk sharing models.”

Michael Freissmuth proposed a bold initiative: “We need a European fund that all health systems pay into with a European agency in charge of distributing the money. Just as important is a translational research center to incentivize the transfer of knowledge coming out of laboratories. Research can be much more successful with the exchange of data organized in a European patient registry. This will allow payers to make evidence-based decisions.”

Let’s hope for miracles but let’s work for them

Based on this idea Günther Krumpl proposes unifying the regulation and reimbursement process as well as forming a European body for financing trials. “Make it obligatory for countries to develop rare disease drugs to create healthy competition.” Labrini Papageorgiou pointed out the importance of fostering collaboration with partners and early engagement with stakeholders as well as evidence-based solutions.

Jo de Cock, a former CEO of the Belgian reimbursement authority (NIHDI) and session moderator supports the idea of bringing workstreams together to equalize access and pricing. “An equalization fund will eliminate the issue of differential pricing and parallel trade in all European countries.”

When the issue of data privacy came up, Dimitrios Athanasiou pointed out: “If you have a life expectancy of one and a half years, you don’t care about your data”, adding: “Let’s hope for miracles but let’s work for them.”

The European Health Forum Gastein is an independent, non-partisan organization founded in 1998 as a European health policy platform with the aim to provide a neutral and inclusive platform for the discussion and advancement of health, solidarity and equity in the EU and beyond. Their founding principle is the equal representation of all stakeholders. Since its inception, the EHFG has developed into a key platform for health policy exchange, bringing together stakeholders and decision-makers from the public and private sector, civil society, as well as science and academia.

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.

Media Contact:
AOP Orphan Pharmaceuticals GmbH
Leopold-Ungar-Platz 2, 1190 Vienna, Austria
Nina Roth
+43 676 3131509
nina.roth@aoporphan.com