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 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.

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.

PP106 Twenty Years Of Orphan Medicines Regulation: Have Treatments Reached Patients In Need Across Europe And Canada?

2021 ◽  
Vol 37 (S1) ◽  
pp. 19-19
Author(s):  
Nadine Henderson ◽  
Phill O'Neill ◽  
Martina Garau
Keyword(s):  
European Union ◽  
Western Europe ◽  
European Countries ◽  
Marketing Authorization ◽  
European Medicines Agency ◽  
The European Union ◽  
The Public ◽  
Orphan Medicinal Products ◽  
Medicines Regulation ◽  
Canadian Provinces

IntroductionThe European Union regulation for orphan medicinal products (OMPs) was introduced to improve the quality of treatments for patients with rare conditions. To mark 20 years of European Union OMP regulation, this study compared access to OMPs and the length of their reimbursement process in a set of European countries and Canadian provinces. Access refers to their full or partial reimbursement by the public health service.MethodsData were collated on European Medicines Agency orphan designation and marketing authorizations, health technology assessment (HTA) decisions and reimbursement decisions, and the respective dates of these events for all the OMPs centrally authorized in 14 European countries (Belgium, England, France, Germany, Hungary, Italy, the Netherlands, Norway, Poland, Scotland, Slovakia, Spain, Sweden, and Switzerland) and four Canadian provinces (Alberta, British Columbia, Ontario, and Quebec).ResultsSince the implementation of the OMPs Regulation in 2000, 215 OMPs obtained marketing authorization. We found that Germany had the highest level of coverage, with 91 percent of OMPs being reimbursed. The three countries with the lowest reimbursement rates were Poland, Hungary, and Norway (below 30%). We observed that Germany had the quickest time to reimbursement following marketing authorization, followed by Switzerland and Scotland. We observed that Poland, Hungary, and Slovakia consistently had the longest time to reimbursement.ConclusionsWe observed substantial variation in the levels and speed of national reimbursement of OMPs, particularly when comparing countries in Eastern and Western Europe, which suggests that an equity gap between the regions may be present. The data also indicated a trend toward faster times to reimbursement over the past 10 years.

scholarly journals Biosimilars: what clinicians should know

Blood ◽  
2012 ◽  
Vol 120 (26) ◽  
pp. 5111-5117 ◽  
Author(s):  
Martina Weise ◽  
Marie-Christine Bielsky ◽  
Karen De Smet ◽  
Falk Ehmann ◽  
Niklas Ekman ◽  
...  
Keyword(s):  
European Union ◽  
Working Party ◽  
The United States ◽  
European Medicines Agency ◽  
Clear Understanding ◽  
The European Union ◽  
Medicinal Products ◽  
Regulatory Guidance ◽  
Appropriate Treatment ◽  
Scientific Principles

Abstract Biosimilar medicinal products (biosimilars) have become a reality in the European Union and will soon be available in the United States. Despite an established legal pathway for biosimilars in the European Union since 2005 and increasing and detailed regulatory guidance on data requirements for their development and licensing, many clinicians, particularly oncologists, are reluctant to consider biosimilars as a treatment option for their patients. Major concerns voiced about biosimilars relate to their pharmaceutical quality, safety (especially immunogenicity), efficacy (particularly in extrapolated indications), and interchangeability with the originator product. In this article, the members and experts of the Working Party on Similar Biologic Medicinal Products of the European Medicines Agency (EMA) address these issues. A clear understanding of the scientific principles of the biosimilar concept and access to unbiased information on licensed biosimilars are important for physicians to make informed and appropriate treatment choices for their patients. This will become even more important with the advent of biosimilar monoclonal antibodies. The issues also highlight the need for improved communication between physicians, learned societies, and regulators.

scholarly journals EMEA and Gene Therapy Medicinal Products Development in the European Union

2003 ◽  
Vol 2003 (1) ◽  
pp. 3-8 ◽  
Author(s):  
Marisa Papaluca Amati ◽  
Francesco Pignatti ◽  
Alexis Nolte ◽  
Nirosha Amerasinghe ◽  
Daniel Gustafsson ◽  
...  
Keyword(s):  
Gene Therapy ◽  
European Union ◽  
Clinical Trials ◽  
Good Clinical Practice ◽  
Evaluation Agency ◽  
The European Union ◽  
Medicinal Products ◽  
Regulatory Processes ◽  
National Competent Authorities ◽  
Practice Implementation

The evaluation of quality, safety, and efficacy of medicinal products by the European Medicines Evaluation Agency (EMEA) via the centralized procedure is the only available regulatory procedure for obtaining marketing authorization for gene therapy (GT) medicinal products in the European Union. The responsibility for the authorization of clinical trials remains with the national competent authorities (NCA) acting in a harmonized framework from the scientific viewpoint. With the entry into force of a new directive on good clinical practice implementation in clinical trials as of 1 May 2004, procedural aspects will also be harmonized at EU level. Scientifically sound development of medicinal products is the key for the successful registration of dossiers and for contributing to the promotion and protection of public health. The objective of this paper is to introduce the EMEA regulatory processes and scientific activities relevant to GT medicinal products.

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