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Therapies for Rare Diseases: How Regulatory Frameworks Keep Research and Development in Europe

Vienna, 22.02.2023, On average, it takes ten years for a drug to reach market maturity. In the area of orphan diseases, there have so far been coordinated framework conditions for research and development. Two major pieces of legislation, the Orphan Medicinal Product Regulation and the General Pharmaceutical Legislation, are currently undergoing significant revision at EU level, which could put the brakes on the development of medical products in the field of rare diseases. There’s a question of shortening the protection against imitation. On the occasion of Rare Disease Day 2023, AOP Orphan Pharmaceuticals GmbH (AOP Health), an Austrian pharmaceutical company specializing in therapeutic solutions in the field of rare diseases and intensive care, points out that research in the field of rare diseases takes place in a niche with special framework conditions. Regulatory and policy measures are important for supporting research here.

There are often only a handful of sufferers with rare diseases. Nevertheless, the performance of meaningful studies and the requirements for statistics often require a very high number of patients. Since it is often difficult to find patients, clinical trials and therefore the research and development of therapies take increasingly longer, which drives up costs. The high development costs can only be justified with the hope of successful marketing. If this is reduced by cutting protection against copying, there is a risk that less research will be carried out for patients with rare diseases and that therapies will only be available to a limited extent.

Keeping research and development in Europe

Another aspect of the innovations that are planned for the legislation is an assessment of clinical data planned for the first time at European level as an optional basis for national reimbursements of medicines. Up to now, different data and analyses are required in each country to determine national reimbursement.

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Dr. Martin Steinhart Co-Chief Executive Officer | AOP Health

“It’s important that the planned changes in legislation lead to simplified  processes, thereby  giving patients quicker access to new medicines. This would be the way for the European market to remain attractive for research and development.“

Security of supply

The pandemic as well as the current economic challenges have shown that joint efforts are also needed in Europe to ensure access to effective and safe medicines. AOP Health does its part by carefully building and controlling its production and supply chain and producing 80 percent of its stocks in Europe. Steinhart comments: “The short distances, but also the fact that we have opened our own packaging plant at our headquarters in Vienna, give us the flexibility to react quickly and unbureaucratically to the supply situation.”

About rare diseases

By definition, a rare disease is one that affects no more than 5 in 10,000 people. There are currently around 8,000 known rare diseases. More than half of these affect children.

Even if individual rare diseases only affect a few people, in total it amounts to a lot of people: in Austria alone, approximately 400,000 people, or six to eight percent of the population, suffer from rare diseases. There are an estimated 30 million people within the EU and around 300 million people worldwide.

Knowledge about the disease patterns is sometimes very limited, which means that the path to diagnosis is very lengthy in many cases. It can take up to 10 years for the correct diagnosis to be made, which is often very stressful for those affected because of their untreated symptoms, but also because of the uncertainty associated with them.

Rare Disease Day on 28 February

On 28 February, Rare Disease Day takes place worldwide with the aim of raising awareness about rare diseases. To mark the occasion, AOP Health invites you to a lecture evening followed by a panel discussion. Health experts, representatives of patient organizations, as well as researchers report from their individual perspectives, share coping strategies and inform the audience about new approaches in research and development. 
The evening talks and discussion will take place at Billrothhaus der Ärzte, Frankgasse 8, 1090 Vienna and will be live-streamed. Start: 5.00 p.m. 
For more information: https://www.aop-health.com/global_en/stories/rare-disease-day-2023
Please register at events@aop-health.com

About AOP Health
The AOP Health Group incorporates several companies including AOP Orphan Pharmaceuticals GmbH with its seat in Vienna, Austria (“AOP Health”). The AOP Health Group is the European pioneer for integrated therapies for rare diseases and in critical care. Over the past 25 years, the Group has become an established provider of integrated therapy solutions operating from its headquarters in Vienna, its subsidiaries and representative offices throughout Europe and the Middle East, as well as through partners worldwide. 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 the patients and their families as well as the doctors and healthcare professionals treating them. 

Press contact

DI Isolde Fally
Corporate Communications
Isolde.Fally@aop-health.com 
+43-676-500 4048