scholarly journals Orphan drugs

2013 ◽  
Vol 66 (9-10) ◽  
pp. 373-378
Author(s):  
Svetlana Golocorbin-Kon ◽  
Aleksandra Vojinovic ◽  
Mladena Lalic-Popovic ◽  
Nebojsa Pavlovic ◽  
Momir Mikov
Keyword(s):  
Rare Diseases ◽  
United States Of America ◽  
Turning Point ◽  
Life Quality ◽  
Orphan Drugs ◽  
The United States ◽  
Pharmaceutical Companies ◽  
Time Loss ◽  
Number Of Patients ◽  
The Right

Introduction. Drugs used for treatment of rare diseases are known worldwide under the term of orphan drugs because pharmaceutical companies have not been interested in ?adopting? them, that is in investing in research, developing and producing these drugs. This kind of policy has been justified by the fact that these drugs are targeted for small markets, that only a small number of patients is available for clinical trials, and that large investments are required for the development of drugs meant to treat diseases whose pathogenesis has not yet been clarified in majority of cases. The aim of this paper is to present previous and present status of orphan drugs in Serbia and other countries. The beginning of orphan drugs development. This problem was first recognized by Congress of the United States of America in January 1983, and when the ?Orphan Drug Act? was passed, it was a turning point in the development of orphan drugs. This law provides pharmaceutical companies with a series of reliefs, both financial ones that allow them to regain funds invested into the research and development and regulatory ones. Seven years of marketing exclusivity, as a type of patent monopoly, is the most important relief that enables companies to make large profits. Conclusion. There are no sufficient funds and institutions to give financial support to the patients. It is therefore necessary to make health professionals much more aware of rare diseases in order to avoid time loss in making the right diagnosis and thus to gain more time to treat rare diseases. The importance of discovery, development and production of orphan drugs lies in the number of patients whose life quality can be improved significantly by administration of these drugs as well as in the number of potential survivals resulting from the treatment with these drugs.

Farmaci orfani e malattie rare: un confronto internazionale delle normative di riferimento

2001 ◽  
Vol 2 (3) ◽  
pp. 185-193
Author(s):  
S. Martina ◽  
C. Denti ◽  
L. Garattini
Keyword(s):  
United States ◽  
United States Of America ◽  
Orphan Drugs ◽  
The United States ◽  
Pharmaceutical Companies ◽  
Economic Returns ◽  
Research Activity ◽  
Common Limit ◽  
Market Situation

Orphan drugs are defined as medicines with low economic returns, so that their production is not a profitable business far pharmaceutical companies. The present study analyses the main characteristics and the role of orphan drugs in four countries (United States of America, .Japan, Australia and European Union), by considering the regulation and the market situation of each State. All countries have introduced a specific legislation on orphan drugs to stimulate the research activity of pharmaceutical industry. The first law was the Orphan Drug Act of the United States of America in 1982. A common limit of all regulations is the strict correlation between “orphan drugs” and “rare diseases”. In fact, the term “orphan” does not refer only to rare disease, but also to other elements that can determine low economic returns for the industry (e.g. drugs with high cost of research and development, drugs that cannot be patented).

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.

Weighting of Votes in an International Assembly

1944 ◽  
Vol 38 (6) ◽  
pp. 1192-1203 ◽  
Author(s):  
Louis B. Sohn
Keyword(s):  
International Organizations ◽  
United States Of America ◽  
The United States ◽  
International Labor Organization ◽  
World Government ◽  
The Past ◽  
International Labor ◽  
Small Countries ◽  
World Organization ◽  
The Right

All the plans for future world organization, whether they envisage a world government or are limited to providing agencies for better collaboration between the peoples of the world, are built around two main conceptions—a small council and a larger assembly. But the different plans disagree widely upon the powers and the make-up of these bodies. The purpose of the present article is to analyze the difficulties relating to the structure of the larger body, the assembly, and to outline a tentative method for surmounting them.The structure of the different international organizations existing in the past was based on two principles: equality of representation and unanimity. That meant, first of all, that in the assemblies of nations the United States of America (population, 131 million) and Luxemburg (population, 300 thousand) had the right of equal representation. For instance, in the Conferences of the International Labor Organization, both countries have been equally entitled to appoint four delegates. Secondly, when an international assembly has tried to arrive at a Decision, not only the largest but also the smallest country could block such a Decision by casting a negative vote. While sometimes a little country has been forcibly persuaded to abandon its opposition, in many instances small countries have been able to frustrate the efforts of international assemblies and conferences otherwise unanimous.

Evaluation of Orphan Products by the U.S. Food and Drug Administration

1992 ◽  
Vol 8 (4) ◽  
pp. 647-657 ◽  
Author(s):  
Marlene E. Haffner ◽  
John V. Kelsey
Keyword(s):  
Drug Administration ◽  
Rare Diseases ◽  
Food And Drug Administration ◽  
Orphan Drugs ◽  
Marketing Approval ◽  
Drug Products ◽  
Governmental Agencies ◽  
Number Of Patients ◽  
Life Threatening ◽  
The U.S

AbstractOrphan drug products generally are used in treating or preventing rare diseases. The small number of patients available for study may create special problems in the evaluation of these products. This paper examines some of the special problems that are associated with the design and implementation of studies to evaluate the safety and efficacy of orphan drugs. The U.S. Food and Drug Administration (FDA) has not established special criteria for evaluating orphan drugs per se, but the FDA has been flexible in evaluating drug products that present special problems, especially when these products are for treatment of serious of life-threatening illnesses. The FDA and other U.S. governmental agencies also have taken steps to promote the development and availability of drugs for rare diseases, including making these products available to patients who are in need, even before the drugs have full FDA marketing approval.

scholarly journals Perfil biográfico de Juan Byron Markholz.

2013 ◽  
Vol 6 (1) ◽  
Author(s):  
Alfonso MONTAGNE V
Keyword(s):  
United States ◽  
History Of Medicine ◽  
United States Of America ◽  
The United States ◽  
Medical Society ◽  
History Of ◽  
The Right

The life of Dr. Juan Byron fills of pride the history of medicine of our nation. Peruvian by birth, he lived in Lima during the second half of the IXI century. Survivor of the war against Chile where his knowledge saved many lives, he was the founder of the medical society “Union Fernandina” and of its journal “Crónica Médica”. Journalist, author of dramas, meteorologist, poliglot, bacteriologist and epidemiologist, researcher and teacher of great prestige in the United States of America and a martyr of medicine. None the less this has not been enough spread. Being close to the centennial of his dead (8th May 1,995), I believe it is the right time to make known the most important aspects of his life.

scholarly journals The right to medical care and its economic consequences: an American dilemma

1972 ◽  
Vol 6 (4) ◽  
pp. 317-327
Author(s):  
José Duarte de Araújo
Keyword(s):  
United States ◽  
Health Care ◽  
United States Of America ◽  
The United States ◽  
Right To Health ◽  
Economic Consequences ◽  
Right To Health Care ◽  
Modern Times ◽  
The Right

The concepts of "rights" and of "right to health care" including its evolution in modern times are discussed. The consequences of implementing this right are discussed in economic terms, regarding the situation in the United States of America. A discussion is also included on the limitations of the role of Health Insurance as a measure to solve the problem of providing health care for all individuals.

scholarly journals Home clinical nutrition and prospects for its development

2020 ◽  
Vol 1 (2) ◽  
pp. 92-98
Author(s):  
K. Yu. Krylov ◽  
M. V. Petrova ◽  
A. E. Shestopalov ◽  
S. V. Sviridov ◽  
V. V. Zozulya ◽  
...  
Keyword(s):  
United States ◽  
United States Of America ◽  
Patient Monitoring ◽  
Prior Experience ◽  
The United States ◽  
Clinical Nutrition ◽  
Routine Practice ◽  
Energy Needs ◽  
Number Of Patients ◽  
The Russian Federation

There is an increase in the number of patients worldwide who require special home feeding after discharge from the hospital Home clinical nutrition (HCN). The yearly prevalence of home clinical nutrition in 2017 was at 463 cases per million in the United States of America whereby in the 90s it was around 163360 per million in the United States of America and Europe. There isnt an established practice of HCN in the Russian Federation and thereby no data on the amount of patients who require HCN and the new risks associated with it because there isnt prior experience in conducting it. To study the routine practice and identify patients who need HCN, we planned a multicenter observational monitoring programme. The goal of the study is patient monitoring who are unable to completely cover their energy needs with the help of conventional nutrition.

scholarly journals Salud, género y clima en migrantes internos e internacionales por Sonora, México

2019 ◽  
Vol 8 (2) ◽  
pp. 171
Author(s):  
José Eduardo Calvario Parra ◽  
Rolando Enríque Díaz Caravantes
Keyword(s):  
High Temperatures ◽  
United States Of America ◽  
Qualitative Method ◽  
Turning Point ◽  
The United States ◽  
The People ◽  
Northern Border ◽  
Semistructured Interviews ◽  
Internal Migrants ◽  
Weather Risks

Introducción: La experiencia migratoria tanto interna como internacional está compuesta por un conjunto de situaciones de peligros y riesgos que ponen en jaque la integridad física y emocional de las personas. El objetivo de este artículo es documentar las experiencias de los/as migrantes ante altas temperaturas y su salud tanto de quienes se dirigen de México o Centroamérica hacia los Estados Unidos de América como los que se desplazan al interior de México rumbo a los campos agrícolas del noroeste.Método: Este trabajo se basa en un estudio de corte cualitativo por medio de entrevistas semiestructuradas sobre el riesgo climático y la salud en migrantes internacionales e internos en la frontera norte de México, particularmente en el estado de Sonora.Resultados: La migración es un punto de inflexión en la vida de las personas entrevistadas. Existe una asocación cualitativa entre la masculinidad y el hecho de emigrar; la noción de proveduria e idea de la aventura son cruciales para entender el proceso migratorio. El afrontar distintos peligros como la violencia y los factores medioambientales generan daños físicos y emocionales.Discusión y/o Conclusión: Existe un proceso de relativización del riesgo climático, en este caso, la minimización de los daños en la salud por las altas temperaturas. La idea de la autosuficiencia e independencia es parte de las identidades de género en los varones migrantes entrevistados. Introduction: The migrational experience, both internal and international is composed by a combination of hazardous and risk situations that threaten the physical and emotional integrity of individuals. The goal of this article is to document the experiences and the health of migrants facing high temperatures when heading from Mexico or Central America towards the United States of America, as well as those that transit through the interior of Mexico towards the agricultural fields of the Northwest.Method: These findings are based on a qualitative method study that used semistructured interviews that delve into weather and health risks in international and internal migrants on the Mexican northern border, particularly in the state of Sonora.Results: Migration is a turning point in the lives of the people interviewed. There exists a qualitative association between masculinity and the act of migrating; the notion of being a provider and the idea of adventure are crucial for understanding the migrational process. Facing different hazards like violence and environmental factors generates physical and emotional damage.Conclusion: There exists a relativization process of weather risks, on this case, the minimization of the damage to health due to high temperatures. The idea of selfsufficiency and independence is part of the gender identities of the migrant males interviewed.

35 United States of America

2018 ◽  
Author(s):  
Cohn Joshua
Keyword(s):  
United States ◽  
New York ◽  
United States Of America ◽  
New York State ◽  
The United States ◽  
Cross Border ◽  
Security Interest ◽  
Bankruptcy Code ◽  
The Right ◽  
The U.S

This chapter examines the most common aspects of the right of set-off in the United States, focusing on the State of New York. It also considers the U.S. Bankruptcy Code and its implications for the right of set-off. The chapter first considers contractual and statutory set-off outside bankruptcy proceedings and whether set-off can be considered a security interest before discussing set-off against insolvent parties. It explains how the right of set-off is affected by the automatic stay provision in section 362 of the Bankruptcy Code, the prohibition of creditor preferences, and fraudulent transfers. It also analyses choice of law issues arising in cross-border set-off, taking into account the relevant provisions of the New York State law and Chapter 15 of the Bankruptcy Code. Finally, it reviews the applicable rules for non-U.S. parties participating in a debtor's plenary Bankruptcy Code proceeding in the absence of a Chapter 15 ancillary proceeding.

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