Pharmaceuticals, policy and law最新文献_第6页

Policy considerations for originator and similar biotherapeutic products 原研药和类似生物治疗制剂的政策考虑

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-160438

G. Grampp, R. Kozak, Thomas Schreitmueller

{"title":"Policy considerations for originator and similar biotherapeutic products","authors":"G. Grampp, R. Kozak, Thomas Schreitmueller","doi":"10.3233/PPL-160438","DOIUrl":"https://doi.org/10.3233/PPL-160438","url":null,"abstract":"Biotherapeutic products (BTPs), also known as biotherapeutic medicines, contain structurally complex active substances produced by living organisms. Due to their complexity and method of manufacture BTPs require distinct regulatory approval standards relative to chemically-synthesized small molecule medicines. This is also relevant for licensing copied versions of a BTP, or similar biotherapeutic products (SBPs) made by a different manufacturer where regulatory concepts developed for generics should not have been applied. In all these licensing scenarios regulators need to evaluate the results of comparability exercises, including sensitive head-to-head analytical, pre-clinical and clinical comparisons with the original product as a basis for approval. SBPs do not contain chemically identical active substances, and may have slightly different benefit-risk profiles, therefore it is necessary to monitor post-approval safety on a product-specific basis. Policymakers may therefore emphasize the need for product-specific identification in patient records and safety reports using either a unique trade name or a distinguishable non-proprietary naming system. The unique nature of BTPs also informs the nature and degree of interchangeability between the originator and SBPs versions. Many policymakers also emphasize that switching between SBPs should only occur with the involvement of the prescriber. It is recommended that pharmacy substitution would only be appropriate when there is a robust framework for a competent authority to assess product-specific evidence of interchangeability. Another challenge is posed by the historical existence in some jurisdictions of copy BTPs that were not assessed according to current regulatory standards. To address this situation the World Health Organization has proposed a regulatory assessment framework wherein the status of such products can be normalized via the orderly submission and review of supplementary data.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"59 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":"134269446","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}

引用次数: 4

Designation, plausibility, protocol assistance, clinical benefit, similarity, reassessment: Rules and experiences 名称，合理性，方案援助，临床效益，相似性，重新评估:规则和经验

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-2010-0269

Chris Walker

{"title":"Designation, plausibility, protocol assistance, clinical benefit, similarity, reassessment: Rules and experiences","authors":"Chris Walker","doi":"10.3233/PPL-2010-0269","DOIUrl":"https://doi.org/10.3233/PPL-2010-0269","url":null,"abstract":"Orphan legislation provides incentives to industry to investigate rare conditions that otherwise would be unlikely to be investigated. These incentives include, free access to scientific advice during the development of their clinical programs (‘protocol assistance’); reduction in fees payable to EMA for review of the marketing authorisation and subsequent licence maintenance fees; sustained market protection in the form of market exclusivity in addition to a range of national incentives. To qualify for these incentives companies must apply for orphan designation confirming the seriousness of the condition which has inadequate alternative therapeutic options (for the diagnosis, prevention or treatment) and that the condition qualifies in terms of low prevalence in the European community. The company must also provide evidence of medical plausibility including likely significant medical benefit for patients to be treated with the product for the proposed orphan indication. If an application for orphan designation is not accepted then a company is able to appeal providing detailed grounds for reassessment and will receive a second opinion from the PDCO. The period of market exclusivity awarded by orphan designation will require that future applications received by the EMA for the same condition have to undergo an assessment of similarity to ensure that no variations or new applications for ‘similar’ medicinal products are granted licences during this ten year period. Products gaining orphan designation provide regular/annual reports regarding their development status, status of global regulatory submissions and any change likely financial returns predicted for the indication.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"104 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":"133790985","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}

引用次数: 0

Drugs labelling, leaflets and packing in European pharmaceutical law with special reference to the Spanish and Italian cases 药品标签，传单和包装在欧洲药法，特别参考西班牙和意大利的情况

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-2010-0298

Francisco Bombillar

引用次数: 2

Economic tools for ensuring access to medicines in Latin American countries 确保拉丁美洲国家获得药品的经济工具

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-140385

F. Tobar, Evangelina Martich

引用次数: 1

How is Italian pharmacy changing and what is the new direction: A modern health post or a drug-store? 意大利的药房正在发生怎样的变化?新的发展方向是什么?是现代保健站还是药店?

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-2010-0306

P. Cicconetti

引用次数: 0

Pharmacoeconomic evidence and policies to promote use of generic medicines in Jordan 促进约旦仿制药使用的药物经济学证据和政策

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-170448

F. El‑Dahiyat

引用次数: 3

Activity of Ethics Committees in Europe on issues related to clinical trials in paediatrics: Results of a survey 欧洲伦理委员会在儿科临床试验相关问题上的活动:一项调查结果

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-2009-0208

A. Altavilla, C. Giaquinto, D. Giocanti, C. Manfredi, J. Aboulker, F. Bartoloni, E. Cattani, M. L. Giudice, M. J. M. Peña, R. Nagler, C. Peterson, O. Vajnerová, F. Bonifazi, A. Ceci

{"title":"Activity of Ethics Committees in Europe on issues related to clinical trials in paediatrics: Results of a survey","authors":"A. Altavilla, C. Giaquinto, D. Giocanti, C. Manfredi, J. Aboulker, F. Bartoloni, E. Cattani, M. L. Giudice, M. J. M. Peña, R. Nagler, C. Peterson, O. Vajnerová, F. Bonifazi, A. Ceci","doi":"10.3233/PPL-2009-0208","DOIUrl":"https://doi.org/10.3233/PPL-2009-0208","url":null,"abstract":"The rights and well-being of children involved in clinical research in Europe should be assured by the respect of ethical considerations and legal rules. Specific provisions are included in the Directive 2001/20/EC (CT-Dir) aimed at providing a homogeneous ethical and legal context to perform clinical trials in Europe. The TEDDY Network of Excellence carried out a “Survey on the ethical and legal frameworks existing in Europe for paediatric clinical trials” to examine the measures enforced by Member States to implement the CT-Dir and other relevant ethical norms. The results showed that many differences exist in the protection of children enrolled in clinical trials. Such differences are especially due to a non-coordinated implementation of the Directive’s Article 4 and a lack of public awareness on ethical issues in this field. The recently approved ‘Ethical considerations for clinical trials on medicinal products conducted with the paediatric population’ should speed up the implementation of an ‘ad hoc set of ethical rules’ and increase the level of minors’ protection. Nevertheless, in lack of ‘binding rules’, a coordination at European level is still needed. Public initiatives aiming at promoting in-depth debates have to be supported in order to encourage this process of coordination.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"11 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":"129324294","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}

引用次数: 9

A public health perspective on the effectiveness of European pharmaceutical regulation 从公共卫生角度看欧洲药品管制的有效性

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-2011-0316

Rafael Bauschke

{"title":"A public health perspective on the effectiveness of European pharmaceutical regulation","authors":"Rafael Bauschke","doi":"10.3233/PPL-2011-0316","DOIUrl":"https://doi.org/10.3233/PPL-2011-0316","url":null,"abstract":"After more then four decades, the regulation of pharmaceuticals still is an evolving and much discussed topic within Europe from a political and economic perspective. Even though the benefits of regulatory harmonization remain uncontested, the constant review and evaluation of the sector may raise public concerns regarding regulatory performance. If regulatory 0experts still see the need for improving existing regulatory provisions, there is reason to believe that the current system does not live up to its expectations. An assessment of regulatory effectiveness, depicting the degree of goal attainment, provides a systematic answer to such questions. Drawing on the regulatory framework, the governance and the outcomes of the regulatory process the development of regulatory effectiveness since the beginnings of European regulation in the sector is discussed. While a continuous improvement from the perspective of effectiveness is traceable throughout time, some issues impeding the performance of the regulatory regime remain. Even though the pharmaceutical package may help to mitigate some of the identified problems, additional efforts will be necessary to finally strengthen the effectiveness of European pharmaceutical regulation and public health.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"13 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":"129481927","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}

引用次数: 1

Scientific advice in EU for products for treatment of rare diseases 欧盟对治疗罕见疾病产品的科学建议

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-2010-0270

Katrin Rupalla

{"title":"Scientific advice in EU for products for treatment of rare diseases","authors":"Katrin Rupalla","doi":"10.3233/PPL-2010-0270","DOIUrl":"https://doi.org/10.3233/PPL-2010-0270","url":null,"abstract":"The national scientific advice procedure can vary from country to country, with some national regulatory agencies not giving advice at all. Usually it is a face-toface meeting, for which there are no pre-specified dates. It can be requested at any time during the development process, also while concerned studies are in progress. The national procedure timelines are flexible and depend upon a local procedure, and can last from two to four months. The procedure is coordinated by a group of national staff, which often includes CHMP and SAWP members of the Agency, but can involve external experts as well. Essentially, the national scientific advice is a national opinion around issues concerning pre-clinical development, clinical development (trial design, statistical methods, RMP, etc), and quality aspects. Any deviation from the advice has to be justified. The advantages of asking a national scientific advice revolve around the flexibility of the procedure, with the possibility of having face-to-face meetings and ask general questions, as well as addressing specific national issues. On the other hand, timelines for the procedures are very variable, in particular when external experts are required. In addition, the national agency may ask the sponsor to go to CHMP for scientific advice. Finally it is important to consider that the national advice is the opinion from a single country and may diverge from the EU-wide position. In fact, the national advice is perceived as non-binding by some EU regulators.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"32 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":"129592054","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}

引用次数: 0

The concerns and problems facing Alpha-1 Antitrypsin Deficiency patients α -1抗胰蛋白酶缺乏症患者的担忧和问题

Pharmaceuticals, policy and law Pub Date : 1900-01-01 DOI: 10.3233/PPL-2009-0228

L. Warren

{"title":"The concerns and problems facing Alpha-1 Antitrypsin Deficiency patients","authors":"L. Warren","doi":"10.3233/PPL-2009-0228","DOIUrl":"https://doi.org/10.3233/PPL-2009-0228","url":null,"abstract":"Alpha-1 Antitrypsin Deficiency (AATD) is probably the most common life threatening inherited disease in Europe. It is estimated that in excess of 100,000 individuals on the Continent suffer from it. It fatally affects the lungs (emphysema) and liver (cirrhosis). As the only therapy for liver AATD is transplantation and as lung disease is far more common, I shall confine my remarks to lung related AATD for which replacement (augmentation) therapy has been developed and is fairly widely used. This is a plasma derived product and is IV delivered. An inhaled product is currently being developed also. However, most patients do not know that they have the condition and most doctors do not know that some of their patients have the condition either. This is true even with symptomatic patients. They are usually diagnosed with Asthma or COPD. This in turn, means that they are being inappropriately treated with therapies that do little for their condition of Alpha-1 Antitrypsin Deficiency. It has been estimated in the USA that it takes an average of five doctors, over a seven year period, to diagnose the condition in a patient. Even after that long delay there is no guarantee that an Alpha-1 patient will be diagnosed. To date, only c. 5% have been diagnosed in the countries that are looking for them. AATD is a deficiency of a vital lung protecting protein. Therefore, it would seem that the obvious therapy is to replace or augment that which is lacking. This therapy has been developed and is now being widely used in the USA and in Europe. In Europe it is licensed and prescribed in the following countries:","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"87 S74","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"120854675","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}

引用次数: 0