Orphan Drugs: understanding the rare disease market and its dynamics
Gespeichert in:
| Format: | Elektronisch E-Book |
|---|---|
| Sprache: | English |
| Veröffentlicht: |
Cambridge, UK
Biohealthcare Pub.
2012
|
| Schlagworte: | |
| Online-Zugang: | DE-1046 Volltext |
| Beschreibung: | This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patientsA source of updated information, news and trends for those who are already active in this fast-evolving fieldCovers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe |
| Beschreibung: | 334 pages |
| ISBN: | 1907568093 9781907568091 9781306177054 1306177057 |
Internformat
MARC
| LEADER | 00000nam a2200000zc 4500 | ||
|---|---|---|---|
| 001 | BV044383303 | ||
| 003 | DE-604 | ||
| 007 | cr|uuu---uuuuu | ||
| 008 | 170630s2012 xx o|||| 00||| eng d | ||
| 020 | |a 1907568093 |9 1-907568-09-3 | ||
| 020 | |a 9781907568091 |9 978-1-907568-09-1 | ||
| 020 | |a 9781306177054 |9 978-1-306-17705-4 | ||
| 020 | |a 1306177057 |9 1-306-17705-7 | ||
| 035 | |a (ZDB-33-ESD)ocn870964084 | ||
| 035 | |a (OCoLC)870964084 | ||
| 035 | |a (DE-599)BVBBV044383303 | ||
| 040 | |a DE-604 |b ger |e aacr | ||
| 041 | 0 | |a eng | |
| 082 | 0 | |a 615.19 |2 23 | |
| 245 | 1 | 0 | |a Orphan Drugs |b understanding the rare disease market and its dynamics |c Elizabeth Hernberg-Stahl and Miroslav Reljanovic |
| 264 | 1 | |a Cambridge, UK |b Biohealthcare Pub. |c 2012 | |
| 300 | |a 334 pages | ||
| 336 | |b txt |2 rdacontent | ||
| 337 | |b c |2 rdamedia | ||
| 338 | |b cr |2 rdacarrier | ||
| 500 | |a This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patientsA source of updated information, news and trends for those who are already active in this fast-evolving fieldCovers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe | ||
| 650 | 7 | |a Orphan drugs |2 fast | |
| 650 | 4 | |a Orphan drugs | |
| 700 | 1 | |a Hernberg-stahl, Elizabeth |e Sonstige |4 oth | |
| 700 | 1 | |a Reljanovic, Miroslav |e Sonstige |4 oth | |
| 856 | 4 | 0 | |u http://www.sciencedirect.com/science/book/9781907568091 |x Verlag |z URL des Erstveröffentlichers |3 Volltext |
| 912 | |a ZDB-33-ESD | ||
| 912 | |a ZDB-33-EBS | ||
| 943 | 1 | |a oai:aleph.bib-bvb.de:BVB01-029785524 | |
| 966 | e | |u http://www.sciencedirect.com/science/book/9781907568091 |l DE-1046 |p ZDB-33-ESD |q FAW_PDA_ESD |x Verlag |3 Volltext | |
Datensatz im Suchindex
| _version_ | 1816805427901366272 |
|---|---|
| adam_text | |
| any_adam_object | |
| building | Verbundindex |
| bvnumber | BV044383303 |
| collection | ZDB-33-ESD ZDB-33-EBS |
| ctrlnum | (ZDB-33-ESD)ocn870964084 (OCoLC)870964084 (DE-599)BVBBV044383303 |
| dewey-full | 615.19 |
| dewey-hundreds | 600 - Technology (Applied sciences) |
| dewey-ones | 615 - Pharmacology and therapeutics |
| dewey-raw | 615.19 |
| dewey-search | 615.19 |
| dewey-sort | 3615.19 |
| dewey-tens | 610 - Medicine and health |
| discipline | Medizin |
| format | Electronic eBook |
| fullrecord | <?xml version="1.0" encoding="UTF-8"?><collection xmlns="http://www.loc.gov/MARC21/slim"><record><leader>00000nam a2200000zc 4500</leader><controlfield tag="001">BV044383303</controlfield><controlfield tag="003">DE-604</controlfield><controlfield tag="007">cr|uuu---uuuuu</controlfield><controlfield tag="008">170630s2012 xx o|||| 00||| eng d</controlfield><datafield tag="020" ind1=" " ind2=" "><subfield code="a">1907568093</subfield><subfield code="9">1-907568-09-3</subfield></datafield><datafield tag="020" ind1=" " ind2=" "><subfield code="a">9781907568091</subfield><subfield code="9">978-1-907568-09-1</subfield></datafield><datafield tag="020" ind1=" " ind2=" "><subfield code="a">9781306177054</subfield><subfield code="9">978-1-306-17705-4</subfield></datafield><datafield tag="020" ind1=" " ind2=" "><subfield code="a">1306177057</subfield><subfield code="9">1-306-17705-7</subfield></datafield><datafield tag="035" ind1=" " ind2=" "><subfield code="a">(ZDB-33-ESD)ocn870964084</subfield></datafield><datafield tag="035" ind1=" " ind2=" "><subfield code="a">(OCoLC)870964084</subfield></datafield><datafield tag="035" ind1=" " ind2=" "><subfield code="a">(DE-599)BVBBV044383303</subfield></datafield><datafield tag="040" ind1=" " ind2=" "><subfield code="a">DE-604</subfield><subfield code="b">ger</subfield><subfield code="e">aacr</subfield></datafield><datafield tag="041" ind1="0" ind2=" "><subfield code="a">eng</subfield></datafield><datafield tag="082" ind1="0" ind2=" "><subfield code="a">615.19</subfield><subfield code="2">23</subfield></datafield><datafield tag="245" ind1="1" ind2="0"><subfield code="a">Orphan Drugs</subfield><subfield code="b">understanding the rare disease market and its dynamics</subfield><subfield code="c">Elizabeth Hernberg-Stahl and Miroslav Reljanovic</subfield></datafield><datafield tag="264" ind1=" " ind2="1"><subfield code="a">Cambridge, UK</subfield><subfield code="b">Biohealthcare Pub.</subfield><subfield code="c">2012</subfield></datafield><datafield tag="300" ind1=" " ind2=" "><subfield code="a">334 pages</subfield></datafield><datafield tag="336" ind1=" " ind2=" "><subfield code="b">txt</subfield><subfield code="2">rdacontent</subfield></datafield><datafield tag="337" ind1=" " ind2=" "><subfield code="b">c</subfield><subfield code="2">rdamedia</subfield></datafield><datafield tag="338" ind1=" " ind2=" "><subfield code="b">cr</subfield><subfield code="2">rdacarrier</subfield></datafield><datafield tag="500" ind1=" " ind2=" "><subfield code="a">This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patientsA source of updated information, news and trends for those who are already active in this fast-evolving fieldCovers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe</subfield></datafield><datafield tag="650" ind1=" " ind2="7"><subfield code="a">Orphan drugs</subfield><subfield code="2">fast</subfield></datafield><datafield tag="650" ind1=" " ind2="4"><subfield code="a">Orphan drugs</subfield></datafield><datafield tag="700" ind1="1" ind2=" "><subfield code="a">Hernberg-stahl, Elizabeth</subfield><subfield code="e">Sonstige</subfield><subfield code="4">oth</subfield></datafield><datafield tag="700" ind1="1" ind2=" "><subfield code="a">Reljanovic, Miroslav</subfield><subfield code="e">Sonstige</subfield><subfield code="4">oth</subfield></datafield><datafield tag="856" ind1="4" ind2="0"><subfield code="u">http://www.sciencedirect.com/science/book/9781907568091</subfield><subfield code="x">Verlag</subfield><subfield code="z">URL des Erstveröffentlichers</subfield><subfield code="3">Volltext</subfield></datafield><datafield tag="912" ind1=" " ind2=" "><subfield code="a">ZDB-33-ESD</subfield></datafield><datafield tag="912" ind1=" " ind2=" "><subfield code="a">ZDB-33-EBS</subfield></datafield><datafield tag="943" ind1="1" ind2=" "><subfield code="a">oai:aleph.bib-bvb.de:BVB01-029785524</subfield></datafield><datafield tag="966" ind1="e" ind2=" "><subfield code="u">http://www.sciencedirect.com/science/book/9781907568091</subfield><subfield code="l">DE-1046</subfield><subfield code="p">ZDB-33-ESD</subfield><subfield code="q">FAW_PDA_ESD</subfield><subfield code="x">Verlag</subfield><subfield code="3">Volltext</subfield></datafield></record></collection> |
| id | DE-604.BV044383303 |
| illustrated | Not Illustrated |
| indexdate | 2024-11-26T17:04:34Z |
| institution | BVB |
| isbn | 1907568093 9781907568091 9781306177054 1306177057 |
| language | English |
| oai_aleph_id | oai:aleph.bib-bvb.de:BVB01-029785524 |
| oclc_num | 870964084 |
| open_access_boolean | |
| owner | DE-1046 |
| owner_facet | DE-1046 |
| physical | 334 pages |
| psigel | ZDB-33-ESD ZDB-33-EBS ZDB-33-ESD FAW_PDA_ESD |
| publishDate | 2012 |
| publishDateSearch | 2012 |
| publishDateSort | 2012 |
| publisher | Biohealthcare Pub. |
| record_format | marc |
| spelling | Orphan Drugs understanding the rare disease market and its dynamics Elizabeth Hernberg-Stahl and Miroslav Reljanovic Cambridge, UK Biohealthcare Pub. 2012 334 pages txt rdacontent c rdamedia cr rdacarrier This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patientsA source of updated information, news and trends for those who are already active in this fast-evolving fieldCovers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe Orphan drugs fast Orphan drugs Hernberg-stahl, Elizabeth Sonstige oth Reljanovic, Miroslav Sonstige oth http://www.sciencedirect.com/science/book/9781907568091 Verlag URL des Erstveröffentlichers Volltext |
| spellingShingle | Orphan Drugs understanding the rare disease market and its dynamics Orphan drugs fast Orphan drugs |
| title | Orphan Drugs understanding the rare disease market and its dynamics |
| title_auth | Orphan Drugs understanding the rare disease market and its dynamics |
| title_exact_search | Orphan Drugs understanding the rare disease market and its dynamics |
| title_full | Orphan Drugs understanding the rare disease market and its dynamics Elizabeth Hernberg-Stahl and Miroslav Reljanovic |
| title_fullStr | Orphan Drugs understanding the rare disease market and its dynamics Elizabeth Hernberg-Stahl and Miroslav Reljanovic |
| title_full_unstemmed | Orphan Drugs understanding the rare disease market and its dynamics Elizabeth Hernberg-Stahl and Miroslav Reljanovic |
| title_short | Orphan Drugs |
| title_sort | orphan drugs understanding the rare disease market and its dynamics |
| title_sub | understanding the rare disease market and its dynamics |
| topic | Orphan drugs fast Orphan drugs |
| topic_facet | Orphan drugs |
| url | http://www.sciencedirect.com/science/book/9781907568091 |
| work_keys_str_mv | AT hernbergstahlelizabeth orphandrugsunderstandingtherarediseasemarketanditsdynamics AT reljanovicmiroslav orphandrugsunderstandingtherarediseasemarketanditsdynamics |