A comparative analysis of assessment procedures for authorization of all European Union (EU) applications for advanced therapy medicinal products (ATMPs) shows that negative opinions were associated with a lack of clinical efficacy and identified severe safety risks. Unmet medical need was often considered in positive opinions and outweighed scientific uncertainties. Numerous quality issues illustrate the difficulties in this domain for ATMP development. Altogether, it suggests that setting appropriate standards for ATMP authorization in Europe, similar to elsewhere, is a learning experience. The experimental characteristics of authorized ATMPs urge regulators, industry, and clinical practice to pay accurate attention to post-marketing risk management to limit patient risk. Methodologies for ATMP development and regulatory evaluations need to be continuously evaluated for the field to flourish.
In: Auffray , C , Balling , R , Barroso , I , Bencze , L , Benson , M , Bergeron , J , Bernal-Delgado , E , Blomberg , N , Bock , C , Conesa , A , Del Signore , S , Delogne , C , Devilee , P , Di Meglio , A , Eijkemans , M , Flicek , P , Graf , N , Grimm , V , Guchelaar , H J , Guo , Y K , Gut , I G , Hanbury , A , Hanif , S , Hilgers , R D , Honrado , Á , Hose , D R , Houwing-Duistermaat , J , Hubbard , T , Janacek , S H , Karanikas , H , Kievits , T , Kohler , M , Kremer , A , Lanfear , J , Lengauer , T , Maes , E , Meert , T , Müller , W , Nickel , D , Oledzki , P , Pedersen , B , Petkovic , M , Pliakos , K , Rattray , M , i Màs , J R , Schneider , R , Sengstag , T , Serra-Picamal , X , Spek , W , Vaas , L A I , van Batenburg , O , Vandelaer , M , Varnai , P , Villoslada , P , Vizcaíno , J A , Wubbe , J P M & Zanetti , G 2016 , ' Making sense of big data in health research : Towards an EU action plan ' , Genome medicine , vol. 8 , no. 1 , 71 . https://doi.org/10.1186/s13073-016-0323-y
Medicine and healthcare are undergoing profound changes. Whole-genome sequencing and high-resolution imaging technologies are key drivers of this rapid and crucial transformation. Technological innovation combined with automation and miniaturization has triggered an explosion in data production that will soon reach exabyte proportions. How are we going to deal with this exponential increase in data production? The potential of "big data" for improving health is enormous but, at the same time, we face a wide range of challenges to overcome urgently. Europe is very proud of its cultural diversity; however, exploitation of the data made available through advances in genomic medicine, imaging, and a wide range of mobile health applications or connected devices is hampered by numerous historical, technical, legal, and political barriers. European health systems and databases are diverse and fragmented. There is a lack of harmonization of data formats, processing, analysis, and data transfer, which leads to incompatibilities and lost opportunities. Legal frameworks for data sharing are evolving. Clinicians, researchers, and citizens need improved methods, tools, and training to generate, analyze, and query data effectively. Addressing these barriers will contribute to creating the European Single Market for health, which will improve health and healthcare for all Europeans.
Medicine and healthcare are undergoing profound changes. Whole-genome sequencing and high-resolution imaging technologies are key drivers of this rapid and crucial transformation. Technological innovation combined with automation and miniaturization has triggered an explosion in data production that will soon reach exabyte proportions. How are we going to deal with this exponential increase in data production? The potential of "big data" for improving health is enormous but, at the same time, we face a wide range of challenges to overcome urgently. Europe is very proud of its cultural diversity; however, exploitation of the data made available through advances in genomic medicine, imaging, and a wide range of mobile health applications or connected devices is hampered by numerous historical, technical, legal, and political barriers. European health systems and databases are diverse and fragmented. There is a lack of harmonization of data formats, processing, analysis, and data transfer, which leads to incompatibilities and lost opportunities. Legal frameworks for data sharing are evolving. Clinicians, researchers, and citizens need improved methods, tools, and training to generate, analyze, and query data effectively. Addressing these barriers will contribute to creating the European Single Market for health, which will improve health and healthcare for all Europeans.
Medicine and healthcare are undergoing profound changes. Whole-genome sequencing and high-resolution imaging technologies are key drivers of this rapid and crucial transformation. Technological innovation combined with automation and miniaturization has triggered an explosion in data production that will soon reach exabyte proportions. How are we going to deal with this exponential increase in data production? The potential of "big data" for improving health is enormous but, at the same time, we face a wide range of challenges to overcome urgently. Europe is very proud of its cultural diversity; however, exploitation of the data made available through advances in genomic medicine, imaging, and a wide range of mobile health applications or connected devices is hampered by numerous historical, technical, legal, and political barriers. European health systems and databases are diverse and fragmented. There is a lack of harmonization of data formats, processing, analysis, and data transfer, which leads to incompatibilities and lost opportunities. Legal frameworks for data sharing are evolving. Clinicians, researchers, and citizens need improved methods, tools, and training to generate, analyze, and query data effectively. Addressing these barriers will contribute to creating the European Single Market for health, which will improve health and healthcare for all Europeans.
In: Auffray , C , Balling , R , Barroso , I , Bencze , L , Benson , M , Bergeron , J , Bernal-Delgado , E , Blomberg , N , Bock , C , Conesa , A , Del Signore , S , Delogne , C , Devilee , P , Di Meglio , A , Eijkemans , M , Flicek , P , Graf , N , Grimm , V , Guchelaar , H-J , Guo , Y-K , Gut , I G , Hanbury , A , Hanif , S , Hilgers , R-D , Honrado , Á , Hose , D R , Houwing-Duistermaat , J , Hubbard , T , Janacek , S H , Karanikas , H , Kievits , T , Kohler , M , Kremer , A , Lanfear , J , Lengauer , T , Maes , E , Meert , T , Müller , W , Nickel , D , Oledzki , P , Pedersen , B , Petkovic , M , Pliakos , K , Rattray , M , I Màs , J R , Schneider , R , Sengstag , T , Serra-Picamal , X , Spek , W , Vaas , L A I , van Batenburg , O , Vandelaer , M , Varnai , P , Villoslada , P , Vizcaíno , J A , Wubbe , J P M & Zanetti , G 2016 , ' Making sense of big data in health research : Towards an EU action plan ' Genome Medicine , vol 8 , no. 1 , pp. 71 . DOI:10.1186/s13073-016-0323-y
Medicine and healthcare are undergoing profound changes. Whole-genome sequencing and high-resolution imaging technologies are key drivers of this rapid and crucial transformation. Technological innovation combined with automation and miniaturization has triggered an explosion in data production that will soon reach exabyte proportions. How are we going to deal with this exponential increase in data production? The potential of "big data" for improving health is enormous but, at the same time, we face a wide range of challenges to overcome urgently. Europe is very proud of its cultural diversity; however, exploitation of the data made available through advances in genomic medicine, imaging, and a wide range of mobile health applications or connected devices is hampered by numerous historical, technical, legal, and political barriers. European health systems and databases are diverse and fragmented. There is a lack of harmonization of data formats, processing, analysis, and data transfer, which leads to incompatibilities and lost opportunities. Legal frameworks for data sharing are evolving. Clinicians, researchers, and citizens need improved methods, tools, and training to generate, analyze, and query data effectively. Addressing these barriers will contribute to creating the European Single Market for health, which will improve health and healthcare for all Europeans.
In: Auffray , C , Balling , R , Barroso , I , Bencze , L , Benson , M , Bergeron , J , Bernal-Delgado , E , Blomberg , N , Bock , C , Conesa , A , Del Signore , S , Delogne , C , Devilee , P , Di Meglio , A , Eijkemans , M , Flicek , P , Graf , N , Grimm , V , Guchelaar , H J , Guo , Y K , Gut , I G , Hanbury , A , Hanif , S , Hilgers , R D , Honrado , Á , Hose , D R , Houwing-Duistermaat , J , Hubbard , T , Janacek , S H , Karanikas , H , Kievits , T , Kohler , M , Kremer , A , Lanfear , J , Lengauer , T , Maes , E , Meert , T , Müller , W , Nickel , D , Oledzki , P , Pedersen , B , Petkovic , M , Pliakos , K , Rattray , M , i Màs , J R , Schneider , R , Sengstag , T , Serra-Picamal , X , Spek , W , Vaas , L A I , van Batenburg , O , Vandelaer , M , Varnai , P , Villoslada , P , Vizcaíno , J A , Wubbe , J P M & Zanetti , G 2016 , ' Erratum to : Making sense of big data in health research: Towards an EU action plan [Genome Med., 8 (2016) (71)] ' , Genome medicine , vol. 8 , no. 1 , 118 . https://doi.org/10.1186/s13073-016-0376-y
The published article [1] has two points of confusion in the section entitled "Technical challenges related to the management of electronic health records". Firstly, the International Rare Diseases Research Consortium (IRDiRC) has developed policies and guidelines on approaches to data sharing meant to enable and improve the development of diagnoses and therapies for rare diseases. However, at present, IRDiRC has not developed best practices for the management of electronic health records (EHRs). Secondly, RARE-Bestpractices is a European Commission 7th Framework Programme (FP7) funded initiative, independent of IRDiRC. RARE-Bestpractices contributes to IRDiRC goals and objectives; however the initiative itself is not sponsored nor connected to IRDiRC.
Medicine and healthcare are undergoing profound changes. Whole-genome sequencing and high-resolution imaging technologies are key drivers of this rapid and crucial transformation. Technological innovation combined with automation and miniaturization has triggered an explosion in data production that will soon reach exabyte proportions. How are we going to deal with this exponential increase in data production? The potential of "big data" for improving health is enormous but, at the same time, we face a wide range of challenges to overcome urgently. Europe is very proud of its cultural diversity; however, exploitation of the data made available through advances in genomic medicine, imaging, and a wide range of mobile health applications or connected devices is hampered by numerous historical, technical, legal, and political barriers. European health systems and databases are diverse and fragmented. There is a lack of harmonization of data formats, processing, analysis, and data transfer, which leads to incompatibilities and lost opportunities. Legal frameworks for data sharing are evolving. Clinicians, researchers, and citizens need improved methods, tools, and training to generate, analyze, and query data effectively. Addressing these barriers will contribute to creating the European Single Market for health, which will improve health arid healthcare for all Europearis. ; Funding Agencies|European Union [115568, 603160, 282510, 664691, 115749, 305033, 305397, 288028, 242189, 211601]; European Molecular Biology Laboratory; Wellcome Trust [WT098051]; [115372]; [257082]; [291814]; [291728]; [321567]; [262055]; [115446]; [602552]; [644753]; [634143]; [261357]; [305280]; [115525]; [2011 23 02]; [270089]; [278433]; [602525]; [201418]; [242135]; [260558]; [223411]; [305626]; [115621]; [611388]; [306000]; [354457]; [305564]; [115010]; [269978]
Medicine and healthcare are undergoing profound changes. Whole-genome sequencing and high-resolution imaging technologies are key drivers of this rapid and crucial transformation. Technological innovation combined with automation and miniaturization has triggered an explosion in data production that will soon reach exabyte proportions. How are we going to deal with this exponential increase in data production? The potential of "big data" for improving health is enormous but, at the same time, we face a wide range of challenges to overcome urgently. Europe is very proud of its cultural diversity; however, exploitation of the data made available through advances in genomic medicine, imaging, and a wide range of mobile health applications or connected devices is hampered by numerous historical, technical, legal, and political barriers. European health systems and databases are diverse and fragmented. There is a lack of harmonization of data formats, processing, analysis, and data transfer, which leads to incompatibilities and lost opportunities. Legal frameworks for data sharing are evolving. Clinicians, researchers, and citizens need improved methods, tools, and training to generate, analyze, and query data effectively. Addressing these barriers will contribute to creating the European Single Market for health, which will improve health and healthcare for all Europeans.