scholarly journals Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit–The EU Regulator's Perspective

2021 ◽  
Vol 8 ◽  
Author(s):  
Maria E. Sheean ◽  
Frauke Naumann-Winter ◽  
Giuseppe Capovilla ◽  
Maria Elisabeth Kalland ◽  
Eva Malikova ◽  
...  
Keyword(s):  
Treatment Options ◽  
Significant Benefit ◽  
European Medicines Agency ◽  
Medicinal Products ◽  
Orphan Medicinal Products ◽  
Satisfactory Method ◽  
Clinical Practise ◽  
Number Of Treatment ◽  
Methods Of Treatment ◽  
The Eu

Since the implementation of the EU Orphan Regulation in 2000, the Committee for Orphan Medicinal Products at the European Medicines Agency has been evaluating the benefits of proposed orphan medicines vs. satisfactory treatment methods. This type of evaluation is foreseen in the Orphan Regulation as the orphan designation criterion called the “significant benefit.” In this article, based on 20 years of experience, we provide a commentary explaining what is considered a satisfactory method of treatment in the context of the EU Orphan Regulation and for the purpose of the assessment of significant benefit. We discuss the challenges posed by continuously changing clinical practise, which is associated with the increasing number of treatment options, evolving nature of medicinal therapeutic indications and our understanding of them.

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 European and Italian regulation on orphan medicinal products

2013 ◽  
Vol 14 (2) ◽  
pp. 89-98
Author(s):  
Roberta Joppi
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Potential Benefit ◽  
Orphan Drugs ◽  
Pharmaceutical Companies ◽  
Drug Status ◽  
Medicinal Products ◽  
Eu Legislation ◽  
Orphan Medicinal Products ◽  
The Eu

The paper presents an overview of the European and Italian Regulation on Orphan Medicinal Products (OMPs), along with some data on the OMPs licensed in the EU from 2000 to 2012. The EU legislation encourages pharmaceutical companies to develop drugs for rare diseases, so-called “orphan drugs”. The European Medicine Agency recognizes orphan drug status mainly on the basis of the prevalence of the disease (≤ 5/10,000), and potential benefit. Orphan status implies incentives for pharmaceutical companies. From 2000 up to 2012 890 candidate orphan drug designations received a positive opinion and the marketing authorization was granted to 72 OMPs corresponding to 80 different indications. Currently, 59 OMPs are available to Italian patients either because licensed to the market by the AIFA or included in the list of the L. 648/96. Despite of an encouraging regulation nearly all the currently estimated rare diseases still await treatments.

scholarly journals Presence of nitrosamine impurities in medicinal products

2021 ◽  
Vol 72 (1) ◽  
pp. 1-5
Author(s):  
Ilijana Sedlo ◽  
Teo Kolonić ◽  
Siniša Tomić
Keyword(s):  
European Medicines Agency ◽  
Nitrogen Compounds ◽  
Medicinal Products ◽  
Different Types ◽  
Therapeutic Alternatives ◽  
The Eu

Abstract In 2018, some sartan medicinal products were reported to be contaminated with nitrosamine compounds, which are potent mutagenic carcinogens. Two nitrosamines received particular attention: N-nitrosodimethylamine (NDMA) and N-nitrosodiethylamine (NDEA). These have since been confirmed in different types of medicinal products, including ranitidine and metformin. Consequently, the European Medicines Agency (EMA) started an investigation into the cause of contamination and an assessment of the risk to patients taking contaminated medicinal products. The main source of contamination were changes in production, which involves combinations of amines and nitrogen compounds and the use of specific catalysts and reagents. Withdrawals of medicinal products that took place in Croatia did not lead to a shortage of sartan- or metformin-containing medicines. Moreover, ranitidine had been preventively withdrawn all over the EU, including Croatia, creating shortages at the time, but was subsequently replaced with therapeutic alternatives.

Determinants for successful marketing authorisation of orphan medicinal products in the EU

2012 ◽  
Vol 17 (7-8) ◽  
pp. 352-358 ◽  
Author(s):  
Michelle Putzeist ◽  
Harald E. Heemstra ◽  
Jordi Llinares Garcia ◽  
Aukje K. Mantel-Teeuwisse ◽  
Christine C. Gispen-De Wied ◽  
...  
Keyword(s):  
Marketing Authorisation ◽  
Medicinal Products ◽  
Orphan Medicinal Products ◽  
The Eu

In-block lateralization as a new technique for mobilization of the inferior alveolar nerve: a technique case series

2020 ◽  
Author(s):  
Marcos Augusto Tomazi ◽  
Alexandre da Silveira Gerzson ◽  
Angelo Menuci Neto ◽  
André Luciano Pasinato da Costa
Keyword(s):  
Treatment Options ◽  
Inferior Alveolar Nerve ◽  
Case Series ◽  
New Technique ◽  
Autogenous Bone ◽  
Minimal Risk ◽  
Short Implants ◽  
Bone Atrophy ◽  
A New Technique ◽  
Number Of Treatment

The edentulous atrophic posterior mandible is often a great challenge for implant rehabilitation. Although a number of treatment options have been proposed, including the use of short implants and surgical grafting techniques, in cases of severe bone atrophy, techniques for mobilization of the inferior alveolar nerve (IAN) have been shown to be efficient, with good results. Four female patients underwent IAN lateralization for prosthetic rehabilitation of the posterior mandible from 2013 to 2019, with 1 year to 5 years and 4 months of follow-up. This case series describes a new technique for mobilization of the IAN, named in-block lateralization, to facilitate access to the IAN and to reduce nerve manipulation. The implant is immediately installed (allowing nerve lateralization in unitary spaces) and the original mandibular anatomy is restored with autogenous bone from the original bed during the same surgical procedure. When well indicated and well performed, this new approach provides better and easier visualization of the IAN as well as safer manipulation aiming to achieve good results for implant stability and minimal risk of neurosensory disturbances, allowing rehabilitation even in unitary spaces.

scholarly journals The Biosimilar Landscape: An Overview of Regulatory Approvals by the EMA and FDA

Pharmaceutics ◽  
2020 ◽  
Vol 13 (1) ◽  
pp. 48
Author(s):  
Ioana Gherghescu ◽  
M. Begoña Delgado-Charro
Keyword(s):  
Drug Administration ◽  
Action Plan ◽  
European Medicines Agency ◽  
Patient Access ◽  
Clinical Evaluations ◽  
The Past ◽  
The Us ◽  
Improve Patient ◽  
The Eu

Biosimilar medicines expand the biotherapeutic market and improve patient access. This work looked into the landscape of the European and US biosimilar products, their regulatory authorization, market availability, and clinical evaluation undergone prior to the regulatory approval. European Medicines Agency (EMEA, currently EMA) and Food and Drug Administration (FDA) repositories were searched to identify all biosimilar medicines approved before December 2019. Adalimumab biosimilars, and particularly their clinical evaluations, were used as a case study. In the past 13 years, the EMA has received 65 marketing authorization applications for biosimilar medicines with 55 approved biosimilars available in the EU market. Since the first biosimilar approval in 2015, the FDA has granted 26 approvals for biosimilars with only 11 being currently on the US market. Five adalimumab biosimilars have been approved in the EU and commercialized as eight different medicines through duplicate marketing authorizations. Whilst three of these are FDA-approved, the first adalimumab biosimilar will not be marketed in the US until 2023 due to Humira’s exclusivity period. The EU biosimilar market has developed faster than its US counterpart, as the latter is probably challenged by a series of patents and exclusivity periods protecting the bio-originator medicines, an issue addressed by the US’s latest ‘Biosimilar Action Plan’.

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