Pharmaceuticals, policy and law最新文献

{"title":"Challenges and priorities for patients with immune thrombocytopenic purpura and their physicians","authors":"J. George","doi":"10.3233/PPL-2009-0242","DOIUrl":"https://doi.org/10.3233/PPL-2009-0242","url":null,"abstract":"Immune thrombocytopenic purpura (ITP) is a disorder of too few platelets (termed thrombocytopenia), the smallest of the circulating blood cells. ITP is defined as isolated thrombocytopenia with no clinically apparent associated conditions or other causes of thrombocytopenia, such as congenital/hereditary thrombocytopenias, drug-induced thrombocytopenia, or autoimmune disorders such as systemic lupus erythematosus [1,2]. “Isolated” thrombocytopenia implies that the red blood cells and white blood cells are normal in number and appearance. The function of blood platelets is to provide initial hemostasis in response to vessel injury, creating a plug to prevent bleeding. Hemostasis is a term to describe prevention of bleeding. If blood vessel injury is small and superficial, the platelet plug is sufficient to stop bleeding. If the vessel injury is extensive, platelets can provide only an initial, temporary seal; permanent hemostasis of larger wounds requires plasma coagulation factors (such as antihemophilic factor and fibrinogen) to provide a strong fibrous matrix to strengthen the platelet plug [3]. Therefore the health problem of patients with ITP, and perhaps their only problem, is a risk for excessive bleeding. The normal platelet concentration in blood is 150,000–350,000 × 10 /L. Like many body functions, the normal number of platelets far exceeds the minimum requirement to provide effective hemostasis. A platelet count of 50,000 × 10 /L is sufficient to stop excessive bleeding following major trauma, surgery, or childbirth. A platelet count of 10,000–20,000× 10/L is sufficient to prevent spontaneous bleeding","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"38 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"124732540","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The global response to the threat of antimicrobial resistance and the important role of vaccines","authors":"E. Utt, C. Wells","doi":"10.3233/PPL-160442","DOIUrl":"https://doi.org/10.3233/PPL-160442","url":null,"abstract":"Antimicrobial resistance (AMR) has emerged as a significant threat to global health security and threatens the achievements of modern medicine. Research and successful development of new antibiotics, especially those with novel mechanisms of action vital to combat resistance, has slowed dramatically since the 1980s. Surveillance for AMR is highly variable globally with significant limitations in many countries impeding the ability to fully characterize the problem. Global efforts to control tuberculosis, malaria and HIV are facing increasing difficulties from the emergence of resistance. Similarly, bacteria causing some of the most common infections in communities or in hospitals such as Escherichia coli and Klebsiella pneumoniae have shown high levels of resistance to third generation cephalosporins requiring treatment with expensive carbapenems as last-resort. Additionally, Streptococcus pneumoniae has shown reduced susceptibility to penicillin in many regions, exceeding 50% in some settings. The cost in lives from AMR over the next 40 years could go as high as 10 million per year with the cost to economic development as high as $3 trillion per year if current trends continue. In addition to ensuring appropriate use of antibiotics and development of novel classes with new or enhanced mechanisms of action, many plans for the global response call for new vaccines as integral to the fight against AMR. Vaccines and antibiotics should be used together to produce synergistic gains in public health, and ultimately, vaccines will extend the clinical utility of antibiotics. The decrease in cases of invasive pneumococcal disease and decrease in prescriptions for antibiotics in some settings resulting from the introduction of broad access to, and utilization of conjugate vaccines for Streptococcus pneumoniae exemplifies the synergy that can be achieved in the fight against AMR.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"18 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"129904178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"TEDDY NoE project in the framework of the EU Paediatric Regulation","authors":"A. Ceci, P. Baiardi, F. Bonifazi, C. Giaquinto, M. J. M. Peña, P. Mincarone, A. Nicolosi, M. Sturkenboom, I. Wong","doi":"10.3233/PPL-2009-0206","DOIUrl":"https://doi.org/10.3233/PPL-2009-0206","url":null,"abstract":"The lack of good quality medicines with formulations tailored for children and supported by properly conducted clinical trials or high level clinical evidence is a longstanding problem in Europe and worldwide. The adoption of the new Paediatric Regulation (Reg. 1901/2006/EC), which forces pharmaceutical industries to conduct a paediatric investigation plan (PIP), is expected to increase the availability of properly tested and authorised medicines for paediatric use. In this framework, the Task-force in Europe for Drug Development for the Young (TEDDY) Network of Excellence was established in 2005 to promote cooperation among researchers and other important stakeholders (regulatory authorities, professionals, patients and consumers) in order to optimise the paediatric use of current drugs and promote the development of new drugs for children, thus actively supporting the implementation of the European Paediatric Regulation.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"1 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"121408977","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative effectiveness research in the United States and primary immunodeficiency diseases","authors":"Emily Hovermale, C. Scalchunes, M. Boyle","doi":"10.3233/PPL-2011-0323","DOIUrl":"https://doi.org/10.3233/PPL-2011-0323","url":null,"abstract":"The United States enacted legislation in 2010 that will promote the use of comparative effectiveness research in the making of healthcare decisions. Of concern with this relatively new mandate is the possibility of using comparative effectiveness research as a means only to mitigate costs rather than to focus on quality of care. This is of particular concern for patients with rare and chronic conditions, such as primary immunodeficiency diseases. The Immune Deficiency Foundation (IDF) uses their survey research to advocate for the needs of patients with primary immunodeficiency diseases to ensure that their unique medical concerns are not overlooked but instead, integrated within an overall healthcare emphasis on personalized medicine and differences in patient treatment response. IDF research shows the efficacy of treatment with immunoglobulin replacement therapy (IG therapy) for many patients with primary immunodeficiency diseases, that IG therapy is underused in the primary immunodeficient community, and that IG therapies are unique and not interchangeable.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"86 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"121928157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Rare diseases treated by plasma proteins","authors":"B. O’Mahony","doi":"10.3233/PPL-2009-0229","DOIUrl":"https://doi.org/10.3233/PPL-2009-0229","url":null,"abstract":"When plasma is separated or recovered from a blood donation or when plasma is collected by plasmapheresis the end result is not just a fluid which can be used in the treatment of many medical conditions but a raw material which can be used for the production of specific life saving or life enhancing medications for a variety of conditions. These conditions include Haemophilia A and Haemophilia B, von Wille- brands Disease a whole range of Primary Immunodeficiencies, Alpha 1 Antitrypsin deficiency, Guillain-BarrSyndrome, Hereditary Angioedema, Idiopathic Thrombo- cytopenic Purpura and Kawasaki Disease among others. Haemophilia is an inherited bleeding disorder caused by the absence or lower levels of coagulation Factor VIII or Factor IX. Haemophilia is caused by a defect on one of the X-chromosomes and therefore the condition is carried by females and presents almost exclusively in males. Some 30% of children with haemophilia are spontaneous mutations with no family history. The most common form of Haemophilia is Haemophilia A or Factor VIII deficiency. This occurs in 105 per million males and constitutes approximately 80% of the cases of Haemophilia. Haemophilia B or factor IX deficiency is less common and occurs in 28 per million males or 20% of cases. Haemophilia can be classified as severe, moderate or mild. An individual would be classified as having severe Haemophilia if their factor level is less than 1% of the normal factor level. A person with moderate haemophilia would have 1-5% of normal factor VIII level and mild would have 5-40%. Severe haemophilia is characterised by spontaneous bleeds into joints and muscles with the initial bleed often occurring when the child is less than 2 years of age. Life threatening bleeds such as bleeds into the central nervous system, intracranial bleeding and bleeding into the throat or internal bleeding can also occur. Initial treatment for haemophilia in the 1950's and 1960's consisted of whole blood or plasma. In the 1960's Cryoprecipitate was used to treat Factor VIII deficiency and from the 1970's Factor VIII and Factor IX deficiency have been treated with coagulation factor concentrates. Haemophilia A has been treated with Factor VIII concentrates since the early 1970's and haemophilia B has been treated with Pro- thrombin Complex Concentrates since the 1970's and with Prothrombin Complex Concentrates or specific Factor IX concentrates since the early 1990's. Treatment can be on-demand where bleeding episodes are treated as they occur or prophylac- tically with Factor VIII three times a week or with Factor IX twice a week. This prevents the bleeds from occurring, minimises joint damage and allows the person","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"9 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"122236542","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Illegal medicines as threats to public health","authors":"J. Valverde","doi":"10.3233/PPL-170449","DOIUrl":"https://doi.org/10.3233/PPL-170449","url":null,"abstract":"","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"58 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"131948836","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Counterfeit medicines: Threat to patient health and safety","authors":"Rubie Mages, Thomas T. Kubic","doi":"10.3233/PPL-160441","DOIUrl":"https://doi.org/10.3233/PPL-160441","url":null,"abstract":"Counterfeit medicines are, first and foremost, a matter of patient health and safety. Counterfeit medicines pose a threat to patients because of the conditions under which they are manufactured, in unlicensed, unregulated, uninspected and often unsanitary sites. The “medicines” themselves pose a threat to patient health and safety because their contents are not regulated and they may not contain the correct active pharmaceutical ingredient (API) to deliver the therapeutic benefit for which they were prescribed, or even ingredients that are themselves harmful such as heavy metals or pesticides. To mitigate that threat, and ensure that their patients receive safe and effective medicines, pharmaceutical companies have incorporated anti-counterfeiting technologies into their packaging and implemented campaigns to detect and disrupt those counterfeiters who place greed above patient safety. Although counterfeiting presents a global threat from which no company, therapeutic area, region or country is immune; gauging the true scope of the problem has remained a challenge. There are hopeful signs, however, as we have seen improved reporting and greater transparency by enforcement and regulatory agencies.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"26 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"132050823","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The practice of pharmaceutical patents","authors":"P. P. Cervantes, G. King, Ramón Soto Vázquez, S. Castello, C. L. López, Erika Alejandra Díaz Moreno","doi":"10.3233/PPL-140387","DOIUrl":"https://doi.org/10.3233/PPL-140387","url":null,"abstract":"Patricia Parra Cervantesa,∗, Gloria Benitez Kingb, Ramon Soto Vazqueza, Samuel Romero Castelloc, Carlos Labastida Lopezd and Erika Alejandra Diaz Morenoa aTecnologia Farmaceutica, FES Zaragoza, Universidad Nacional Autonoma de Mexico, Mexico bLaboratorio Neurofarmacologia, Instituto Nacional de Psiquiatria “Ramon de la Fuente”, Mexico cDireccion general, Research Pro S.C., Mexico dDepartamento Analisis Tecnologico, Research Pro S.C. Mexico","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"412 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"132521041","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Data protection and market exclusivities for pharmaceuticals in the EU","authors":"T. Cook","doi":"10.3233/PPL-140373","DOIUrl":"https://doi.org/10.3233/PPL-140373","url":null,"abstract":"Non-patent protection for pharmaceuticals in the EU dates back to 1987, when a formal system for the protection of the regulatory data used to secure the authorisation of pharmaceuticals was first established. Although often termed “data exclusivity” in recognition of the exclusive rights that it confers over the use of that data for a fixed period, it is only generally in practice that it confers market exclusivity during that period, as others can in theory replicate the generation of such data. Over time regulatory data protection has grown in importance, and has not only been extended in scope, to protect data used to secure a significant new indication, a paediatric use marketing authorisation, or a change of legal status, but it has been supplemented by a true market exclusivity regime in the form of orphan drug protection. This has thus resulted in the current framework of non-patent exclusivities conferred by the regulatory system for medicinal products in the EU as reviewed in this paper, consisting of the following such regimes:","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"117 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"117318299","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Introduction - Lessons for developing a sustainable life sciences eco-system in MICs and LICs","authors":"T. Wilsdon, A. Haderi, Lilian Li","doi":"10.3233/PPL-160428","DOIUrl":"https://doi.org/10.3233/PPL-160428","url":null,"abstract":"","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"67 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"133435064","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}