Orphan Drugs Coverage In European Health Insurance Systems

Orphan Drugs Coverage In European Health Insurance Systems – The team of authors, together with Rita Banzi from the Italian Mario Negri Institute, analyzed the effects of the lack of evidence on orphan drugs at the European and national level. The result: the need for reform is great.

With the Regulation on drugs for rare diseases (orphan drugs) in 2000, the number of newly approved orphan drugs increased significantly. While 63 orphan drugs were approved in the first decade after

Orphan Drugs Coverage In European Health Insurance Systems


Orphan Drugs Coverage In European Health Insurance Systems

By regulation, the number rose to 133 in the second decade. In 2022 alone, 22 orphan drugs were approved.

Managing Rare Diseases: Examples Of National Approaches In Europe, North America And East Asia

) implies a superior therapeutic benefit (called “significant benefit”) over existing treatment options. This has negative effects on national policies: in Germany, for example, a superior therapeutic benefit (so-called “additional benefit”) is automatically assumed for orphan drugs when entering the market, regardless of the actual evidence available, meaning that this benefit is fictitious. But are new orphan drugs really better than existing treatments? In the article for

, a team led by Philip Kranz, Natalie McGauran and Thomas Kaiser of the Institute for Healthcare Quality and Efficiency

) in Germany, together with Rita Banzi from the Mario Negri Institute for Pharmacological Research in Italy, investigated whether the assumption of superiority is justified. They conclude: We often don’t know, because orphan drug status is equated with superior therapeutic benefit without sufficient evidence to prove it.

For this reason, the authors conclude that there is an urgent need to separate orphan drug status from the “superior therapeutic benefit” designation. Until now, this has been foreseen by law in order to create incentives for the development of orphan drugs. However, in

Why We Should Care About Ultra Rare Disease

In the article, the authors argue that this designation should only be granted if there is strong evidence of superior therapeutic benefit compared to the standard of care.

Health care systems across Europe are different. However, they all ultimately face the same problem: there is often not enough evidence to determine whether a new drug offers advantages over existing treatment options. In Germany, where almost all new drugs are reimbursed, this means that around half of all drugs enter the market without enough evidence to show whether they offer real improvement for patients. In many other countries where sufficient evidence is a prerequisite for reimbursement, the problem is primarily at another level: effective drugs may fail to pass the threshold in cost-effectiveness analyzes due to insufficient evidence and may not be reimbursed at all – thus depriving patients of an important treatment option.

Evidence gathering is also possible for orphan drugs. In order to improve the evidence base for orphan drugs in the future, international rare disease registries should be established to facilitate implementation

Orphan Drugs Coverage In European Health Insurance Systems

Director Thomas Kaiser notes: “Robust evidence comparing a new drug to the standard of care is essential for rational treatment and reimbursement decisions – early generation of this data, for example, in parallel with the approval process, should therefore be mandatory.” Currently, subsequent reimbursement is mainly promoted for orphan drugs. However, it is much more important to support the early generation of meaningful study results.”

The Shake Up Of Orphan And Pediatric Rules

In the European Union alone, about 30 million people suffer from more than 6,000 different rare diseases. The economic problem in orphan drug development is that there is a high demand among affected patients, but the markets are small. This led to legislation that encouraged the development of these drugs:

The level of orphan drug status is associated with a designation of superior therapeutic benefit. However, hard evidence is not needed to prove this. Philip Kranz, researcher at

The Department of Medicines Evaluation explains: “Due to the lack of evidence and the assumption of superiority, the actual superior therapeutic benefit remains unclear.” This makes it difficult to make informed decisions about clinical use, reimbursement and pricing.”

Authors’ conclusion: designation indicating that orphan drugs have a truly superior therapeutic benefit should be granted only on the basis of strong evidence and should be completely separate from the regulatory approval process. This would create incentives to demonstrate the superiority of a new drug over the standard of care early on, ultimately improving treatment outcomes for patients with rare diseases.

Equal Access To Care For Rare Diseases

Subscribe to daily updates on projects, calls for tenders and events, as well as our press releases. Analysis of EU and National Orphan Drug Regulation Reforms to Improve Outcomes for Patients with Rare Diseases 2023; 381 doi: https://doi.org/10.1136/-2022-072796 (published 9 May 2023) Cite this as: 2023;381:e072796

Philip Kranz and colleagues argue that the alleged superior therapeutic benefit attributed to orphan drugs is often not supported by evidence from clinical trials and propose reform of EU and national orphan drug regulations

About 30 million people in the European Union (EU) are affected by over 6000 different rare diseases, 1 which makes the lack of available and affordable treatments for rare diseases a major public health problem. The development of drugs for rare diseases (orphan drugs) was commercially less attractive due to small markets.1 This led to legislation that encouraged the development of orphan drugs, such as the EU Orphan Regulation of 2000.2 Since then, the number of orphan drugs approved in EUs have grown rapidly—from 63 in the first decade after the regulation came into force to 133 in the second decade; 22 were approved in 2022, indicating that this trend will continue.3 The pharmaceutical industry has characterized the increase in new orphan drug approvals as “a true success story in the EU”4, and patient organizations as “a true success, which should be celebrates with pride from all.”5 European Commission …Self-assessment of physical fitness and health versus the motivational value of physical activity goals in people involved in fitness, football, martial arts and wheelchair rugby

Orphan Drugs Coverage In European Health Insurance Systems

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Germany: Approval Of The Cost Containment Bill

Comparison of Orphan Drug Prices in the US and the EU in the Perspective of Considered Modifications to the US Orphan Drug Act and Considered Adjustments to Price Regulation Mechanisms in the US and the European Union

Author Paveł Zelevski Paveł Zelevski Scilit Preprints.org Google Scholar 1, * , Michał Vojna Michał Vojna Scilit Preprints.org Google Scholar 1, 2, Katarzina Sigit Katarzina Sigit Scilit Preprints.org Google Scholar 3, Cipora El. Scholar 4, Izabela Gaska Izabela Gaska Scilit Preprints.org Google Scholar 4, Mateusz Niemiec Mateusz Niemiec Scilit Preprints.org Google Scholar 4, Mateusz Kaczmarski Mateusz Kaczmarski Scilit Preprints.org Google Scholar 4, Tomasz Niemiec Scilit Preprints.org , 6, Beata Karakievicz Beata Karakievicz Scilit Preprints.org Google Scholar 7, Artur Kotvas Artur Kotvas Scilit Preprints.org Google Scholar 7, Paulina Zabielska Paulina Zabielska Scilit Preprints.org Google Scholar 7, Olga Partika Olga Partis2 Scilit Scholar , 8, Monika Pajevska Monika Pajevska Scilit Preprints. org Google Scholar 8, Edita Krzich-Fałta Edita Krzich-Fałta Scilit Preprints.org Google Scholar 9, Eva Bandurska Eva Bandurska Scilit Preprints.org Google Scholar 10, Veronika Cilitcieć S Veronika Cilitcieć Preprints. org Google Scholar 10 and Aleksandra Czerv Aleksandra Czerv Scilit Preprints.org Google Scholar 2, 8

Department of Health Economics and Medical Law, Medical University of Warsaw, Zwirk i Viguri 81 St., 02-091 Warsaw, Poland

Orphan Drugs Coverage In European Health Insurance Systems

Department of Social Medicine and Public Health, Department of Social Medicine, Pomeranian Medical University in Szczecin, 70-204 Szczecin, Poland

Reforming Eu And National Orphan Drug Regulations To Improve Outcomes For Patients With Rare Diseases

Received: 25 July 2022 / Revised: 16 September 2022 / Accepted: 20 September 2022 / Published: 24 September 2022

Global sales of orphan drugs in 2019 were estimated at $136 billion, which accounted for 16% of the global pharmaceutical prescription market and are expected to grow by 12% over the next 5 years. A better understanding of orphan drug pricing can inform ongoing discussions about adjustments to the Orphan Drug Act (ODA) in the US or modifications to pricing mechanisms in the EU. The aim of the study was to compare and analyze the prices of orphan drugs in the USA and the EU. All drugs with orphan drug status were compared in the US and the EU. For now

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