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 Analysis of patient access to orphan drugs in Turkey

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Güvenç Koçkaya ◽  
Sibel Atalay ◽  
Gülpembe Oğuzhan ◽  
Mustafa Kurnaz ◽  
Selin Ökçün ◽  
...  
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Anatomical Therapeutic Chemical ◽  
Descriptive Analysis ◽  
Orphan Drugs ◽  
Orphan Drug Status ◽  
European Medicines Agency ◽  
Drug Status ◽  
The European Union ◽  
Early Access

Abstract Background Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals. Methods The drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey. Results Based on the EMA, 105 pharmaceuticals were approved with “orphan drug” status except for drugs that have lost orphan drug status, decommissioned in the European Union and withdrawn from the European Community Register by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. 17 (74%) of licensed products and 17 (35%) of unlicensed products were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L—Antineoplastic and Immunomodulatory” agents. Conclusion An orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. Considering the capacity and prices for orphan drugs in Turkey, it can be said that many patients with rare diseases have difficulty in their treatment. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.

scholarly journals Analysis of Patient Access to Orphan Drugs in Turkey

2020 ◽  
Author(s):  
GÜVENÇ KOÇKAYA ◽  
Sibel Atalay ◽  
Gulpembe Oguzhan ◽  
Mustafa Kurnaz ◽  
Selin Okcun ◽  
...  
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Anatomical Therapeutic Chemical ◽  
Descriptive Analysis ◽  
Orphan Drugs ◽  
European Medicines Agency ◽  
Drug Status ◽  
Early Access ◽  
Life Threatening ◽  
Low Prevalence

Abstract Background: Rare diseases are life-threatening, serious, and chronic genetic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals. Methods: The drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey.Results: Based on the EMA, 105 pharmaceuticals were approved with “orphan drug” status by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. Seventeen licensed products (74%) and 17 unlicensed products (35%) were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L –Antineoplastic and Immunomodulatory” agents.Conclusion: An orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.

scholarly journals The role of globalization in drug development and access to orphan drugs: orphan drug legislation in the US/EU and in Latin America

F1000Research ◽  
2015 ◽  
Vol 4 ◽  
pp. 57 ◽  
Author(s):  
Renée J.G. Arnold ◽  
Lida Bighash ◽  
Alejandro Bryón Nieto ◽  
Gabriela Tannus Branco de Araújo ◽  
Juan Gabriel Gay-Molina ◽  
...  
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Latin American ◽  
Rare Diseases ◽  
Market Access ◽  
Orphan Drugs ◽  
The United States ◽  
Drug Status ◽  
Drug Legislation ◽  
The Us

Compared to a decade ago, nearly three times as many drugs for rare diseases are slated for development. This article addresses the market access issues associated with orphan drug status in Europe and the United States in contrast to the legislation in five Latin American (LA) countries that have made strides in this regard--Mexico, Brazil, Colombia, Chile and Argentina. Based on the success of orphan drug legislation in the EU and US, LA countries should strive to adopt similar strategies with regard to rare diseases and drug development. With the implementation of new targeted regulations, reimbursement strategies, and drug approvals, accessibility to treatment will be improved for people afflicted with rare diseases in these developing countries.

OP65 Pharmacoeconomic Evaluation Of Orphan Drugs: Impact Of Extra Criteria?

2019 ◽  
Vol 35 (S1) ◽  
pp. 16-16
Author(s):  
Orla Maguire ◽  
Laura McCullagh ◽  
Cara Usher ◽  
Michael Barry
Keyword(s):  
Cost Effectiveness ◽  
Orphan Drug ◽  
Societal Perspective ◽  
Evaluation Process ◽  
Orphan Drugs ◽  
Pharmacoeconomic Evaluation ◽  
Qaly Gain ◽  
Minor Effect ◽  
Orphan Medicinal Products ◽  
A Minor

IntroductionThere is ongoing debate as to whether conventional pharmacoeconomic evaluation (PE) methods are appropriate for orphan medicinal products (OMPs). The National Centre for Pharmacoeconomics (NCPE) in Ireland has a well-defined process for conducting pharmacoeconomic evaluations of pharmaceuticals, which is the same for OMPs and non-OMPs. The objective of this study was to identify whether supplementary criteria considered in the pharmacoeconomic evaluation of OMPs would affect final reimbursement recommendations.MethodsA literature search was conducted to identify criteria. Orphan drug pharmacoeconomic evaluations completed by the NCPE between January 2015 and December 2017 were identified and supplementary criteria, where feasible, were applied.ResultsFourteen pharmacoeconomic evaluations were included in the study. Three criteria that could feasibly be applied to the NCPE evaluation process were identified, all three of which essentially broadened the economic perspective of the pharmacoeconomic evaluation. Higher cost-effectiveness threshold: Despite being arbitrarily raised from EUR 45,000/QALY to EUR 100,000/QALY, only one orphan drug demonstrated cost-effectiveness at this higher threshold. Weighted QALY gain: here, a weighted gain of between one and three is applied to drugs demonstrating QALY gains between 10 and 30, respectively. No OMPs included in the study showed a QALY gain of more than 10. Thirteen demonstrated QALY gains less than 10 and one could not be evaluated. Societal perspective: six submissions incorporated societal perspective as a scenario analysis. Despite incremental cost-effectiveness ratios (ICERs) being reduced between 4 percent and 58 percent, only two OMPs demonstrated cost-effectiveness at the higher threshold (EUR 100,000/QALY).ConclusionsApplication of supplementary criteria to the pharmacoeconomic evaluation of OMPs had a minor effect on three products assessed. However, for the majority, the final cost-effectiveness outcomes remained the same. The study highlights that other criteria are being considered in the decision to reimburse.

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.

Orphan Drugs: The Question of Products Liability

1985 ◽  
Vol 10 (4) ◽  
pp. 491-513
Author(s):  
Susan F. Scharf
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Tort Law ◽  
Orphan Drugs ◽  
Products Liability ◽  
Liability Law ◽  
Design Defect ◽  
Liability Costs ◽  
Orphan Drug Development

AbstractOrphan drugs, essential for die treatment of persons widi rare diseases, generally are unprofitable for manufacturers to develop and market. While congressional and administrative efforts to promote die development of orphan drugs have met widi modest success, application of products liability doctrine to orphan drug sponsors could subvert those efforts. This Note describes die provisions of die Orphan Drug Act and analyzes products liability law with respect to orphan drug litigation. It argues that die goals of tort law support the imposition of liability for design defect, failure to warn and negligence in testing. Finally, die Note acknowledges diat liability costs create disincentives for orphan drug development and suggests mechanisms for reducing manufacturers’ liability concerns.

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

2012 ◽  
Vol 69 (4) ◽  
pp. 1009-1024 ◽  
Author(s):  
Roberta Joppi ◽  
Vittorio Bertele’ ◽  
Silvio Garattini
Keyword(s):  
Orphan Drug ◽  
Orphan Drugs ◽  
Orphan Diseases ◽  
Drug Legislation ◽  
The Eu
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