Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective

2021 ◽  
Author(s):  
Giovanni Tafuri ◽  
Andrea Bracco ◽  
Jens Grueger
Keyword(s):  
European Union ◽  
Rare Diseases ◽  
The European Union

scholarly journals The latest orphan drug designations and the Commission Communication on Regulation (EC) 141/2000

2005 ◽  
Vol 11 (3) ◽  
Author(s):  
Rashmi R Shah
Keyword(s):  
European Union ◽  
Rare Diseases ◽  
European Medicines Agency ◽  
Biological Plausibility ◽  
The European Union ◽  
Medicinal Products ◽  
Market Exclusivity ◽  
Community Regulation ◽  
Orphan Medicinal Products ◽  
Patient Groups

The implementation of Community Regulation on orphan medicinal products in the European Union in April 2000 has resulted in a deluge of applications for designation of medicinal products as orphan for rare diseases. By April 2004, the Committee for Orphan Medicinal Products had already given positive opinion on 63 per cent of the 316 applications considered by them. A significant number of these positive designations have already matured into full marketing authorisations. Three major reasons – failure to meet prevalence or significant benefit criteria or provide evidence of biological plausibility – have equally contributed to either the negative opinion on or the applicants withdrawing the remaining applications. In July 2004, the European Commission issued a communication setting out its position on certain matters relating to the implementation of the designation and market exclusivity provisions. The Commission, the European Medicines Agency (EMEA) and the Committee for Orphan Medicinal Products (COMP) continue to be proactive and provide as much guidance and incentives as practical, engaging themselves with sponsors, patient groups and academia. As experience builds up and issues are clarified, there are expectations that the Community Regulation on orphan medicines will prove to be a spectacular success.

scholarly journals Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives

2014 ◽  
Vol 9 (1) ◽  
pp. 20 ◽  
Author(s):  
David Uguen ◽  
Thomas Lönngren ◽  
Yann Le Cam ◽  
Sarah Garner ◽  
Emmanuelle Voisin ◽  
...  
Keyword(s):  
European Union ◽  
Rare Diseases ◽  
Access To Medicines ◽  
The European Union ◽  
Adaptive Approaches

scholarly journals Evaluating the European Union Committee of Experts on Rare Diseases Joint Action

2015 ◽  
Vol 25 (suppl_3) ◽  
Author(s):  
V Gómez ◽  
A Machado ◽  
P Rama ◽  
C Furtado ◽  
D David ◽  
...  
Keyword(s):  
European Union ◽  
Rare Diseases ◽  
Joint Action ◽  
The European Union ◽  
Union Committee

Hungarian National Plan and Strategy for Rare Diseases

2014 ◽  
Vol 155 (9) ◽  
pp. 325-328 ◽  
Author(s):  
György Kosztolányi
Keyword(s):  
European Union ◽  
Rare Diseases ◽  
Present Report ◽  
Health Resources ◽  
The European Union ◽  
National Plan ◽  
European Union Policy ◽  
Lack Of Information ◽  
Health Authorities ◽  
Information Research

The rarity of low prevalence diseases and the lack of information, research, diagnosis, treatment and expert availability may mean that the people affected do not benefit from the health resources and services they need. Rare diseases are considered to have little impact on society as a whole, yet they pose serious difficulties for sufferers and their families. By the end of the last century, two robust achievements in science and technology, i.e. the biotechnological and informatics revolutions, have created a real base for global approach to rare diseases by coordinating the capacities for health care, biomedical research and drug development and pooling the very limited resources available both nationally and transnationally. The European Commission has taken a number of actions which help patients and professionals to share expertise and information across borders with the objective of reducing the number of people suffering from these types of diseases. These actions together form the legal basis of the European Union policy on rare diseases. Orphan or rare diseases are now one of the priorities in the public health programmes in European Union. In 2009, the document “European Union Council Recommendation on an action in the field of rare diseases” was released with the main goal to provide national health authorities with supporting tools for the development and implementation of national plans and strategies for rare diseases by the end of 2013. This recommendation adopted by European Union Member States, allows common policy guidelines to be shared everywhere in Europe. By September 2013 the Hungarian National Plan for Rare Diseases, a health policy strategy until 2020 was finalized. The present report gives a short view on the document. Orv. Hetil., 2014, 155(9), 325–328.

scholarly journals Legal assessment of current situation on orphan patients in Lithuania

Medicina ◽  
2008 ◽  
Vol 44 (8) ◽  
pp. 571 ◽  
Author(s):  
Indrė Špokienė
Keyword(s):  
Health Care ◽  
European Union ◽  
Rare Diseases ◽  
Comparative Method ◽  
National Level ◽  
Practical Implementation ◽  
The European Union ◽  
Medicinal Products ◽  
Member States ◽  
Orphan Medicinal Products

After Lithuania joined the European Union, the Regulation (EC) No. 141/2000 on orphan medicinal products and Commission Regulation (EC) No. 847/2000 came into force as part of national legislation. Member States must adopt specific measures to increase knowledge on rare diseases and to improve their detection, diagnosis, and treatment. The aim of this article was to present and to assess the current legal situation on orphan patients and their treatment in Lithuania, to identify legislation gaps, and to propose some ideas how to facilitate the solution of the existing problems in this field. For this purpose, European Union and Lithuanian legal documents on rare medicinal products are examined using a comparative method. With reference to inventory of Member States’ incentives for rare diseases in national level, the most important issues, which orphan patients face to in Lithuania, are singled out. In Lithuania, the situation of orphan patients in terms of protection of patient rights is insufficiently determined. The access to effective health care services or approved therapies in some cases is restricted. Working relationships between genetic services and various clinical specialists as well as with those in primary care are not legally determined; the number of clinical trials aimed at orphan medicinal products is low. These results suggest a need for awareness raising among Lithuanian Government, health care specialists, patient organizations about the importance to improve practical implementation of European Union legislation and progressive experience of some European countries in this field.

The European Union Policy in the Field of Rare Diseases

2013 ◽  
Vol 16 (6) ◽  
pp. 268-277 ◽  
Author(s):  
A. Montserrat Moliner ◽  
J. Waligóra
Keyword(s):  
European Union ◽  
Rare Diseases ◽  
The European Union ◽  
European Union Policy ◽  
Union Policy

Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations

2017 ◽  
Vol 103 (5) ◽  
pp. 427-430 ◽  
Author(s):  
Andrea Ecker ◽  
Segundo Mariz ◽  
Frauke Naumann-Winter ◽  
Koenraad Norga ◽  
Ingeborg Barisic ◽  
...  
Keyword(s):  
European Union ◽  
Comparative Analysis ◽  
Rare Diseases ◽  
Market Forces ◽  
The European Union ◽  
Medicinal Products ◽  
Paediatric Investigation Plan ◽  
Pharmaceutical Development ◽  
Orphan Medicinal Products ◽  
Small Patient

BackgroundMarket forces may not be sufficient to stimulate research and development of medicines for small patient populations, such as children and patients with rare diseases. Both the European Union Orphan and Paediatric Regulations were introduced to address the unmet public health needs of these smaller patient populations through the use of incentives, rewards and obligations. Developers for new medicines for rare diseases must agree a paediatric investigation plan (PIP) or waiver with the European Medicines Agency’s (EMA) Paediatric Committee (PDCO), and can also apply for an orphan designation (OD) from the EMA’s Committee of Orphan Medicinal Products (COMP). The scope of both the OD and the PIP (or waiver) is defined by the agreed condition.ObjectivesThe aim of this study was to analyse the approach of PDCO and COMP in defining the appropriate condition for a PIP or OD, respectively, in order to investigate potential challenges in the paediatric development of orphan medicines which have to meet the requirements of both legislations.MethodsA comparative analysis of PIP conditions and OD conditions was performed for medicines that have been reviewed by both Committees.ResultsWe found that in the substantial majority of cases there is no divergence between the conclusions of COMP and PDCO with regard to the condition for which a medicine is to be developed.ConclusionThese findings demonstrate that a collaborative approach allows both Regulations to work synergistically to foster pharmaceutical development for rare diseases in childhood.

Current state of developing advanced therapies for rare diseases in the European Union

2020 ◽  
Vol 8 (10) ◽  
pp. 417-429
Author(s):  
Tingting Qiu ◽  
Yitong Wang ◽  
Monique Dabbous ◽  
Eve Hanna ◽  
Ru Han ◽  
...  
Keyword(s):  
European Union ◽  
Rare Diseases ◽  
The European Union ◽  
Current State ◽  
Advanced Therapies
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