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.
{"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}
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.
{"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}
The drug, as a technical product, has been created in order to control and dominate nature. Thus, the pharmaceutical industry acts against infections and diseases in nature, as well as other attacks on our health that can weaken it or bring it to a complete end. Unfortunately, these technologies are not innocuous; they eliminate “threats” but create “risks”, even for the health-related object of protection (suffice to say the side effects of drugs like Vioxx or Thalidomide). Not surprisingly, from its earliest origins the word “drug” has referred to a substance that can be harmful, as well as ultimately, healing. These products are not safe. Therefore, the Legislator has created an assumption of risk: it is supposed
{"title":"Drugs labelling, leaflets and packing in European pharmaceutical law with special reference to the Spanish and Italian cases","authors":"Francisco Bombillar","doi":"10.3233/PPL-2010-0298","DOIUrl":"https://doi.org/10.3233/PPL-2010-0298","url":null,"abstract":"The drug, as a technical product, has been created in order to control and dominate nature. Thus, the pharmaceutical industry acts against infections and diseases in nature, as well as other attacks on our health that can weaken it or bring it to a complete end. Unfortunately, these technologies are not innocuous; they eliminate “threats” but create “risks”, even for the health-related object of protection (suffice to say the side effects of drugs like Vioxx or Thalidomide). Not surprisingly, from its earliest origins the word “drug” has referred to a substance that can be harmful, as well as ultimately, healing. These products are not safe. Therefore, the Legislator has created an assumption of risk: it is supposed","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"219 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":"115067997","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}
Summary: The greatest challenges for pharmaceutical policies in Latin American countries are related to the objective of promoting access for their populations. Around two thirds of the spending on medicine in the region is financed by household incomes, with a strong regressive effect on their financing. For these reasons, the economic regulation tools for controlling spending and prices of medicines take on singular importance. The present article examines a set of measures implemented by the countries in the region for the economic regulation of medicines centred on ensuring access to them, emphasizing those intended to control the prices of the pharmaceutical products and those oriented to moderating the spending on medicines in the health systems.
{"title":"Economic tools for ensuring access to medicines in Latin American countries","authors":"F. Tobar, Evangelina Martich","doi":"10.3233/PPL-140385","DOIUrl":"https://doi.org/10.3233/PPL-140385","url":null,"abstract":"Summary: The greatest challenges for pharmaceutical policies in Latin American countries are related to the objective of promoting access for their populations. Around two thirds of the spending on medicine in the region is financed by household incomes, with a strong regressive effect on their financing. For these reasons, the economic regulation tools for controlling spending and prices of medicines take on singular importance. The present article examines a set of measures implemented by the countries in the region for the economic regulation of medicines centred on ensuring access to them, emphasizing those intended to control the prices of the pharmaceutical products and those oriented to moderating the spending on medicines in the health systems.","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":"114536309","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}
List of the new health (professional) and social (administrative) services provided by pharmacy according to new Italian law and National Health Service Authority; weak and strength points, examination of all the aspects. New health services are important to give effort to the pharmacist role, on the other hand the social ones need time and human resources to run. Some pharmacy provides a wide range of services for free already and small pharmacies cannot compete due to problems dealing with the surface of the shop or with the number of employees. New strategies oriented to help and protect small pharmacies are vital, but the most important thing is to create a network of pharmacies that will offer new services; everyone must join it, we do not want pharmacies lack in knowledge in the future and not professional.
{"title":"How is Italian pharmacy changing and what is the new direction: A modern health post or a drug-store?","authors":"P. Cicconetti","doi":"10.3233/PPL-2010-0306","DOIUrl":"https://doi.org/10.3233/PPL-2010-0306","url":null,"abstract":"List of the new health (professional) and social (administrative) services provided by pharmacy according to new Italian law and National Health Service Authority; weak and strength points, examination of all the aspects. New health services are important to give effort to the pharmacist role, on the other hand the social ones need time and human resources to run. Some pharmacy provides a wide range of services for free already and small pharmacies cannot compete due to problems dealing with the surface of the shop or with the number of employees. New strategies oriented to help and protect small pharmacies are vital, but the most important thing is to create a network of pharmacies that will offer new services; everyone must join it, we do not want pharmacies lack in knowledge in the future and not professional.","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"76 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":"114882245","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":"Pharmacoeconomic evidence and policies to promote use of generic medicines in Jordan","authors":"F. El‑Dahiyat","doi":"10.3233/PPL-170448","DOIUrl":"https://doi.org/10.3233/PPL-170448","url":null,"abstract":"","PeriodicalId":348240,"journal":{"name":"Pharmaceuticals, policy and law","volume":"123 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":"125118182","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}
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
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.
{"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}
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.
{"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}
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.
{"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}
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:
{"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}
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