scholarly journals International Cooperation as a Prerequisite for Improvement of Access to Orphan Biologic Medicines

2021 ◽  
Vol 14 (2) ◽  
Author(s):  
Zbigniew Więckowski
Keyword(s):  
International Cooperation ◽  
Rare Diseases ◽  
Patent Protection ◽  
Positive Impact ◽  
Mutual Recognition ◽  
Orphan Drugs ◽  
Economic Incentives ◽  
Regulatory Agencies ◽  
The Usa ◽  
The Eu

Rare diseases constitute a global problem. Worldwide, 350 million people suffer from such diseases. The number of diagnosed cases are on the rise. Only a small percentage of those suffering have the opportunity to be treated with modern therapies. Medicines used to treat rare diseases are called orphan drugs. Biologic medicines developed for orphan drug indications, besides patent protection, have a period of regulatory and market exclusivity. After this period of time has elapsed, access to orphan drugs could be improved by the introduction of biosimilar medicines. The biggest challenge is to develop effective legal, tax and economic incentives to stimulate the development of biosimilar medicines for orphan indications. The regulatory agencies - EMA in the EU and the FDA in the USA - play a key role in increasing access to orphan biologics. Undoubtedly, the international cooperation, especially the mutual recognition of registration procedures between countries, and the creation of a common vocabulary and the unification of incentives for the pharmaceutical industry would have the positive impact on access to modern therapies.

scholarly journals The Hypothesis of Jobless Economic Growth in the Global Triad Countries

Equilibrium ◽  
2014 ◽  
Vol 9 (3) ◽  
pp. 109-129
Author(s):  
Piotr Misztal
Keyword(s):  
Economic Growth ◽  
International Finance ◽  
Positive Impact ◽  
Ordinary Least Squares ◽  
Research Association ◽  
Literature Study ◽  
Econometric Methods ◽  
The Usa ◽  
Annual Frequency ◽  
The Eu

The aim of the study is to analyze the hypothesis of jobless economic growth in economic theory and in the Global Triad countries (U.S. , EU-15, Japan, China , India). In the article the research method based on the literature study in the field of macroeconomics and international finance were used, as well as econometric methods (Ordinary Least Squares). All the statistics used in the study had an annual frequency and covered the period from 1990 to 2012. These data came from the statistical database of the Business Membership and Research Association – The Conference Board Total Economy Database. On the basis of the study the phenomenon of jobless economic growth in China and India was revealed. However, in the case of the USA, the EU-15 and Japan the positive impact of economic growth on changes in employment was confirmed.

scholarly journals TRANSATLANTIC INTEGRATION AND DEVELOPED COUNTRIES COMPETITIVENESS PROBLEMS

2016 ◽  
pp. 249-257
Author(s):  
A. A. Sidorov
Keyword(s):  
Mutual Recognition ◽  
Developed Countries ◽  
High Tech ◽  
Transatlantic Trade ◽  
Regulatory Cooperation ◽  
The Usa ◽  
History Of ◽  
Tariff Liberalization ◽  
General Business ◽  
The Eu

Factors and possible consequences of transatlantic integration are elaborated in the article. An overview of the history of transatlantic cooperation is provided. The author highlights the paramount goal of Transatlantic Trade and Investment Partnership (TTIP) - strengthening the positions of its parties in the world economy against the backdrop of global competition. Stalemate in Doha round of WTO trade negotiations as well as depressed state of the European economy also contributed to transatlantic integration. Validity of the EU Commission conclusion on TTIP benefits is examined. Results of TTIP econometric modelling are critically assessed. Problems of the EU-US non-tariff liberalization are analyzed. Efficiency of the EU and US labor markets is compared. Low competitiveness of the EU in comparison to the USA and underlying risks for TTIP economic growth and employment are outlined. High unemployment, difficulties of manufacturing (including high-tech industries thereof) recovery, adverse general business situation in the EU are among such risks. Various modes of regulatory cooperation and possibility of their adoption in TTIP are considered. Harmonization, erga omnes mutual recognition of regulations, bilateral mutual recognition of regulations, mutual recognition of conformity testing are distinguished. Possible implications of the modes of regulatory cooperation on TTIP members competitiveness, competition with emerging economies and global standard setting are examined. Conflict of TTIP goals and motivations is revealed. The existence of economic factors of transatlantic integration as well as overestimation of TTIP benefits (primarily for the EU economy) is concluded.

scholarly journals Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases

2021 ◽  
pp. 1-16
Author(s):  
Yoshitsugu Aoki ◽  
Matthew J.A. Wood
Keyword(s):  
Rare Diseases ◽  
Neuromuscular Diseases ◽  
Exon Skipping ◽  
Rare Condition ◽  
Regulatory Agencies ◽  
Ribonucleic Acids ◽  
Rna Transcripts ◽  
The Usa ◽  
Target Rna

Research and drug development concerning rare diseases are at the cutting edge of scientific technology. To date, over 7,000 rare diseases have been identified. Despite their individual rarity, 1 in 10 individuals worldwide is affected by a rare condition. For the majority of these diseases, there is no treatment, much less cure; therefore, there is an urgent need for new therapies to extend and improve quality of life for persons who suffer from them. Here we focus specifically on rare neuromuscular diseases. Currently, genetic medicines using short antisense oligonucleotides (ASO) or small interfering ribonucleic acids that target RNA transcripts are achieving spectacular success in treating these diseases. For Duchenne muscular dystrophy (DMD), the state-of-the-art is an exon skipping therapy using an antisense oligonucleotide, which is prototypical of advanced precision medicines. Very recently, golodirsen and viltolarsen, for treatment of DMD patients amenable to skipping exon 53, have been approved by regulatory agencies in the USA and Japan, respectively. Here, we review scientific and clinical progress in developing new oligonucleotide therapeutics for selected rare neuromuscular diseases, discussing their efficacy and limitations.

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.

Scenario-Patent Protection Compared to Climate Change

2013 ◽  
Vol 4 (3) ◽  
pp. 61-70
Author(s):  
Araken Alves de Lima ◽  
Patricia Carvalho dos Reis ◽  
Julio César Moreira Reis Castelo Branco ◽  
Rodrigo Danieli ◽  
Cibele Cristina Osawa ◽  
...  
Keyword(s):  
Climate Change ◽  
United States ◽  
South Korea ◽  
International Cooperation ◽  
Patent Protection ◽  
Positive Impact ◽  
Environmental Benefits ◽  
Green Technologies ◽  
Industrial Property ◽  
The Government

The United Nations Framework on Climate Change (UNFCCC) took effect as a treaty in 1994 to promote international cooperation in the fight against global warming. Currently, nearly 190 countries are signatories of the UNFCCC, which has had successive additions as the Kyoto Protocol (1997). In 1995, the Climate Technology Initiative was established within the UNFCCC to encourage international cooperation in the accelerated development and diffusion of environmentally Sound Technologies - EST. Such technologies are also capable of protection provided by patents, and this kind of protection is a valuable tool for the industrial production inventions to become a worthwhile investment, contributing to economic development. Many patent applications claim advantages relative to efficiency, waste reduction, or even the costs of operation/manufacturing. However, the difficulty of accurately distinguishing the EST’s technologies among others, which are those that only claim environmental benefits, compared to those who actually have a higher potential to promote a more positive impact on the environment directed. This study aims to report some performance initiatives in relations between technologies, focusing on the so-called “GREEN”, and the effects of climate change. Some initiatives have already been started in countries such as Australia, Canada, United States, United Kingdom, Spain, Japan, South Korea and Israel. These nations are constituted in the form of their industrial property offices, as entities that have implemented regulations regarding the patentability of requests for green technologies or EST’s such requests are known as “green patents” applications. In this context, it is highlighted that the definition of “green patents” differs from country to country and this leads to greater uncertainty in this designation, with the codes of the International Patent Classification (IPC) should be prioritized. This study observed that, in the case of South Korea, green patents are technologies classified in accordance with the interests of the Government, or, according to designations of environmental laws. Moreover, it still shows that South Korea, Australia, United States, Japan, Israel already have programs to promote accelerated examination of “green patents” applications with different criteria.

Sanctions of the USA and the Policy of Bank of Russia:double Blow to the National Economy

2014 ◽  
pp. 13-29 ◽  
Author(s):  
S. Glazyev
Keyword(s):  
National Security ◽  
Economic Sanctions ◽  
Capital Flight ◽  
Practical Advice ◽  
The Us ◽  
Monetary Authorities ◽  
The Usa ◽  
Simultaneous Adoption ◽  
The Eu

This article examines fundamental questions of monetary policy in the context of challenges to the national security of Russia in connection with the imposition of economic sanctions by the US and the EU. It is proved that the policy of the Russian monetary authorities, particularly the Central Bank, artificially limiting the money supply in the domestic market and pandering to the export of capital, compounds the effects of economic sanctions and plunges the economy into depression. The article presents practical advice on the transition from external to domestic sources of long-term credit with the simultaneous adoption of measures to prevent capital flight.

Assessment of Poland's Economic Policy During te Crisis

2013 ◽  
Vol 8 (3) ◽  
pp. 195-210
Author(s):  
Stefan Krajewski
Keyword(s):  
Economic Policy ◽  
Pension System ◽  
Fiscal Sustainability ◽  
Economic Policies ◽  
Role Of The State ◽  
The Usa ◽  
Reducing Costs ◽  
Polish Economy ◽  
Favorable Conditions ◽  
The Eu

The rapid weakening of economic activity, covering most states in the world, gives rise to a lively discussion on the choice of methods to tackle the crisis, the legitimacy and effectiveness of various economic policies, the role of the state and the scope of its intervention in the economy. The paper evaluates the Polish economic policy in recent years. This refers to the situation prevailing in the EU and the USA. I conclude that the Polish economy during the crisis remained relatively stable, without having to provide the emergency aid from the outside. The development of such a situation has been affected by different reasons, including: - The benefits of the so-called "backwardness rent", which resulted, among others, in the inflow of EU funds (Poland was in 2007-2013 and in will be in 2014-2020 the biggest beneficiary of the EU budget); - The effects of decisions on changes in the tax and social security, taken for political reasons (before the crisis); - The controversial withdrawal from the funded pension system, reducing the budget deficit and public debt; - The prudent monetary policy and anti-inflation policy pursued over many years. Actions taken in Poland are primarily focused on reducing costs, which differs quite significantly from the economic policy dominant in the U.S. and the "old" EU countries which generally pursue expansionary fiscal policy and a policy of cheap money. Polish solution facilitates the achievement of short-term fiscal sustainability, but does not create favorable conditions for the development in the long-term (insufficient investment, petrification of economic structure, lack of innovation). 

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.

Naldemedine: Peripherally Acting Opioid Receptor Antagonist for Treating Opioid-induced Adverse Effects

2020 ◽  
Vol 20 (31) ◽  
pp. 2830-2842
Author(s):  
Masanao Inagaki ◽  
Toshiyuki Kanemasa ◽  
Takaaki Yokota
Keyword(s):  
Adverse Effects ◽  
Adverse Events ◽  
Severe Pain ◽  
Lead Compound ◽  
Inhibitory Effects ◽  
Pharmacokinetic Profiles ◽  
Structure Activity ◽  
The Usa ◽  
Relationship Study ◽  
The Eu

Opioids are widely used for pain management in moderate-to-severe pain. However, opioids are associated with adverse events, such as constipation and emesis/vomiting. To reduce these undesired effects, a structure–activity relationship study of morphinan derivatives was conducted, and a promising lead compound with inhibitory effects on opioid receptors was obtained. Further improvement in the potency and pharmacokinetic profiles of the lead compound led to the discovery of naldemedine, which showed anti-constipation and anti-emetic effects against these adverse events that were induced by morphine without influencing morphine’s analgesic effect. Naldemedine was launched in Japan and the USA in 2017 and in the EU in 2019, for treating opioid-induced constipation.

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