scholarly journals Independent Registries Are Cost-Effective Tools to Provide Mandatory Postauthorization Surveillance for Orphan Medicinal Products

2020 ◽  
Author(s):  
Sandra M. Sirrs ◽  
Marie-Francoise Arthus ◽  
Daniel G. Bichet ◽  
Cheryl Rockman-Greenberg ◽  
Kaye LeMoine ◽  
...  
Keyword(s):  
Cost Effective ◽  
Medicinal Products ◽  
Orphan Medicinal Products

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 Orphan drug designation in Europe-Procedural guidance and challenges

2019 ◽  
Vol 7 (3) ◽  
pp. 1-7
Author(s):  
Christina Nicolodi
Keyword(s):  
Orphan Drug ◽  
Early Stage ◽  
Specific Treatment ◽  
Development Program ◽  
Orphan Drug Designation ◽  
Medicinal Products ◽  
Strategic Decisions ◽  
Stage Of Development ◽  
Orphan Medicinal Products ◽  
Legislative Framework

The European legislative framework on orphan medicinal products was implemented to stimulate the development of medicinal products against rare diseases and to ensure the patient’s adequate access to qualitative and specific treatment methods. Between 2000 and 2018 3210 orphan drug designation applications were submitted in Europe of which 2121 orphan designations have been issued by the European Commission. (1) Though the definitions for orphan medicinal products and the regulatory procedures are well defined, a high degree of regulatory knowledge is needed and strategic decisions on the development program must be considered at a very early stage of development: in fact, only 164 of the 2121 designated orphan development products have resulted in authorised orphan medicinal products since the orphan legislation was implemented. In this article, the requirements and procedures for the orphan designation application and maintenance at the time of marketing authorisation application are discussed in the context of the European Regulation.

scholarly journals Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs

BMJ Open ◽  
2017 ◽  
Vol 7 (9) ◽  
pp. e017358 ◽  
Author(s):  
Viviana Giannuzzi ◽  
Annalisa Landi ◽  
Enrico Bosone ◽  
Floriana Giannuzzi ◽  
Stefano Nicotri ◽  
...  
Keyword(s):  
Marketing Authorisation ◽  
Development Process ◽  
Developmental Process ◽  
Clinical Stage ◽  
Phase Iii ◽  
Marketing Approval ◽  
Medicinal Products ◽  
Safety Issues ◽  
Stage Of Development ◽  
Orphan Medicinal Products

ObjectivesThe research and development process in the field of rare diseases is characterised by many well-known difficulties, and a large percentage of orphan medicinal products do not reach the marketing approval.This work aims at identifying orphan medicinal products that failed the developmental process and investigating reasons for and possible factors influencing failures.DesignDrugs designated in Europe under Regulation (European Commission) 141/2000 in the period 2000–2012 were investigated in terms of the following failures: (1) marketing authorisation failures (refused or withdrawn) and (2) drugs abandoned by sponsors during development.Possible risk factors for failure were analysed using statistically validated methods.ResultsThis study points out that 437 out of 788 designations are still under development, while 219 failed the developmental process. Among the latter, 34 failed the marketing authorisation process and 185 were abandoned during the developmental process. In the first group of drugs (marketing authorisation failures), 50% reached phase II, 47% reached phase III and 3% reached phase I, while in the second group (abandoned drugs), the majority of orphan medicinal products apparently never started the development process, since no data on 48.1% of them were published and the 3.2% did not progress beyond the non-clinical stage.The reasons for failures of marketing authorisation were: efficacy/safety issues (26), insufficient data (12), quality issues (7), regulatory issues on trials (4) and commercial reasons (1). The main causes for abandoned drugs were efficacy/safety issues (reported in 54 cases), inactive companies (25.4%), change of company strategy (8.1%) and drug competition (10.8%). No information concerning reasons for failure was available for 23.2% of the analysed products.ConclusionsThis analysis shows that failures occurred in 27.8% of all designations granted in Europe, the main reasons being safety and efficacy issues. Moreover, the stage of development reached by drugs represents a specific risk factor for failures.

scholarly journals Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties

2018 ◽  
Vol 13 (1) ◽  
Author(s):  
Caridad Pontes ◽  
Juan Manuel Fontanet ◽  
Roser Vives ◽  
Aranzazu Sancho ◽  
Mònica Gómez-Valent ◽  
...  
Keyword(s):  
Decision Making ◽  
Medicinal Products ◽  
Regulatory Decision ◽  
Orphan Medicinal Products

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 Evolution of AIFA Registries to Support Managed Entry Agreements for Orphan Medicinal Products in Italy

2021 ◽  
Vol 12 ◽  
Author(s):  
Entela Xoxi ◽  
Karen M Facey ◽  
Americo Cicchetti
Keyword(s):  
Pharmaceutical Companies ◽  
Medicinal Products ◽  
Value For Money ◽  
Orphan Medicinal Product ◽  
Collection System ◽  
Web Based ◽  
Best Value ◽  
Orphan Medicinal Products ◽  
Managed Entry Agreements ◽  
Equal Split

Italy has a well-established prominent system of national registries to support managed entry agreements (MEAs), monitoring innovative medicinal products (MPs) with clinical as well as economic uncertainties to ensure appropriate use and best value for money. The technological architecture of the registries is funded by pharmaceutical companies, but fully governed by the national medicines agency (AIFA). A desktop analysis was undertaken of data over a 15-year timeframe of all AIFA indication-based registries and associated EMA information. The characteristics of registries were evaluated, comparing orphan MPs vs. all MPs exploring cancer and non-cancer indications. OMP (orphan medicinal product) registries’ type vs. AIFA innovation status and EMA approval was reviewed. Of the 283 registries, 182 are appropriateness registries (35.2% relate to OMPs, with an almost equal split of cancer vs. non-cancer for OMPs and MPs), 35 include financial-based agreements [20% OMPs (2 non-cancer, 5 cancer)], and 60 registries are payment by result agreements [23.3% OMPs (4 non-cancer, 10 cancer)]. Most OMPs (53/88) came through the normal regulatory route. With the strengthening of the system for evaluation of innovation, fewer outcomes-based registries have been instigated. AIFA has overcome many of the challenges experienced with MEA through developing an integrated national web-based data collection system: the challenge that remains for AIFA is to move from using the system for individual patient decisions about treatment to reviewing the wealth of data it now holds to optimize healthcare.

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