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.

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.

Development of Orphan Drugs under European Regulatory Incentives and Patent Protection

2017 ◽  
Vol 24 (3) ◽  
pp. 239-263 ◽  
Author(s):  
Mari Minn
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Patent Protection ◽  
Market Access ◽  
Orphan Drugs ◽  
Patient Access ◽  
Generic Market ◽  
European Regulatory ◽  
Orphan Drug Development ◽  
Regulatory Incentives

AbstractThis article analyses how the regulatory incentives provided by Regulation 141/2000 affect orphan drug development and generic market entry. It seeks to find out how the regulatory incentives work in combination with patent protection, and whether in combination they foster orphan drug innovation and overall patient access, or rather hinder the latter. The article argues that even though the regulatory incentives are targeted to fostering innovation and early generic market access, when combined with patent protection, the generic entry is likely blocked or delayed.

scholarly journals Regulatory Strategies for Orphan Drug Development in Canada – Australia

2020 ◽  
Vol 5 (2) ◽  
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Integrated Approach ◽  
Orphan Drugs ◽  
The United States ◽  
Regulatory Policies ◽  
Regulatory Strategies ◽  
United States Food ◽  
Pharmaceutical Enterprise ◽  
States Food

Canada, Australia have evaluated how their governments can facilitate the improvement of scientific merchandise to deal with uncommon issues. Each has hooked up programs and/or policies to help the improvement of merchandise to deal with unmet clinical wishes in small populations and to ensure their citizens get right of entry to such important medicines. Australia’s software, initiated in 1998, changed into evolved in collaboration with the United States Food and Drug Administration to facilitate the alternate and evaluation of facts on orphan tablets. Canada’s evaluation, posted in 1996, determined that a standalone orphan drug software became now not presently warranted, as present regulation and regulatory policies permit early get right of entry to critical medicinal merchandise. The incidences of such diseases were increasing at an extra pace than the speed with which drugs are researched and developed to treat such diseases. One of the fundamental motives is that the pharmaceutical enterprise is not very keen to research the improvement of orphan capsules as those capsules do no longer capture a larger market. This is the modern situation in-spite of the various incentives furnished in the orphan drug act. However, in this article, we’ve tried to focus on present regulatory framework, Current principles of rare sickness, Regulatory Challenges for Rare Disease Drug Development, Regulatory Integrated approach for the improvement and approval of orphan drugs in Canada & Australia.

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 Letter from the Editors

2020 ◽  
Vol 2 ◽  
pp. 1
Author(s):  
Editorial Office
Keyword(s):  
Clinical Trials ◽  
Medical Journal ◽  
Open Access ◽  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Case Reports ◽  
Orphan Drugs ◽  
Original Research ◽  
Orphan Drug Development

Last year we successfully introduced a new journal: The Journal of Rare Diseases and Orphan Drugs (JRDOD) is a peer-reviewed open-access medical journal that publishes original research, reviews, case reports, and letters covering a broad field of its specialty. We intend to publish articles stimulating to read, educate, and inform readers with the most up-to-date research in genetics, rare diseases, and new orphan drug development in different stages of clinical trials. Journal topics are centered on patients living with undiagnosed rare diseases, the importance of a diagnosis, individual approaches to treatments. We hope that this journal will increase awareness of many difficult to diagnosed and treat medical conditions.

Potential Interactions of the Orphan Drug Act and Pharmacogenomics: A Flood of Orphan Drugs and Abuses?

2005 ◽  
Vol 31 (2-3) ◽  
pp. 365-380 ◽  
Author(s):  
David Loughnot
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
The United States ◽  
New Drugs ◽  
United States Government ◽  
Orphan Drug Act ◽  
The Government ◽  
Effective Drugs ◽  
Potential Interactions

To overcome the unattractiveness of small markets, the United States government provides financial aid and incentives for drug manufacturers to create cures for rare diseases under the Orphan Drug Act (“the Act”). Recent research integrating genetic information and pharmacology holds promise for creating more effective drugs targeted at smaller populations than ever before. In the near future, it seems that a flood of new drugs targeted at small disease populations could take advantage of the government benefits under the Act. Drug applicants will include true orphan drugs along with “Trojan” applicants that seek to co-opt the benefits for drugs that should not qualify as orphans. Currently, the FDA appears ill prepared to discern between the two types of applicants and prevent abuse of the system.In 1983, the federal government passed the Act. Congress designed the Act and subsequent modifications to provide incentives for companies to bring drugs for rare diseases to market.

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.

Geo-Economic Competition in Latin America: Brazil, Venezuela, and Regional Integration in the 21st Century

2018 ◽  
Vol 33 ◽  
Author(s):  
Guilherme Casarões
Keyword(s):  
Latin American ◽  
Regional Integration ◽  
Free Trade Area ◽  
The United States ◽  
Economic Competition ◽  
Integration Model ◽  
Trade Area ◽  
Complex Processes ◽  
The Us ◽  
New Institutions

The institutional framework of Latin American integration saw a period of intense transformation in the 2000s, with the death of the ambitious project of the Free Trade Area of the Americas (FTAA), spearheaded by the United States, and the birth of two new institutions, the Union of South American Nations (UNASUR) and the Community of Latin American and Caribbean States (CELAC). This article offers a historical reconstruction of regional integration structures in the 2000s, with emphasis on the fault lines between Brazil, Venezuela and the US, and how they have shaped the institutional order across the hemisphere. We argue that the shaping of UNASUR and CELAC, launched respectively in 2007 and 2010, is the outcome of three complex processes: (1) Brazil’s struggle to strengthen Mercosur by acting more decisively as a regional paymaster; (2) Washington’s selective engagement with some key regional players, notably Colombia, and (3) Venezuela’s construction of an alternative integration model through the Bolivarian Alliance (ALBA) and oil diplomacy. If UNASUR corresponded to Brazil’s strategy to neutralize the growing role of Caracas in South America and to break apart the emerging alliance between Venezuela, Argentina, and Bolivia, CELAC was at the same time a means to keep the US away from regional decisions, and to weaken the Caracas-Havana axis that sustained ALBA.

Comparison and analysis of the fda approved orphane drugs registerand the vital and essential drugs list of the Russian Federation

2020 ◽  
pp. 4-16
Author(s):  
D.S. Yurochkin ◽  
◽  
A.A. Leshkevich ◽  
Z.M. Golant ◽  
I.A. NarkevichSaint ◽  
...  
Keyword(s):  
United States ◽  
Russian Federation ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
The United States ◽  
Orphan Diseases ◽  
Essential Drugs ◽  
The Russian Federation ◽  
Comparison And Analysis ◽  
The Government

The article presents the results of a comparison of the Orphan Drugs Register approved for use in the United States and the 2020 Vital and Essential Drugs List approved on October 12, 2019 by Order of the Government of the Russian Federation No. 2406-r. The comparison identified 305 international non-proprietary names relating to the main and/or auxiliary therapy for rare diseases. The analysis of the market of drugs included in the Vital and Essential Drugs List, which can be used to treat rare (orphan) diseases in Russia was conducted.

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