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Literature DB >> 25203033

The special case of gene therapy pricing.

Abstract

Gene therapy companies that pursue high, one-time payments for their products risk a backlash from payors. A better solution may lie in a pay-for-performance model.

Mesh：

Year: 2014 PMID： 25203033 DOI： 10.1038/nbt.3003

Source DB: PubMed Journal: Nat Biotechnol ISSN： 1087-0156 Impact factor: 54.908

10 in total

1. Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.

Authors: George Buchlis; Gregory M Podsakoff; Antonetta Radu; Sarah M Hawk; Alan W Flake; Federico Mingozzi; Katherine A High
Journal: Blood Date: 2012-01-23 Impact factor: 22.113

2. Vaccine advance-purchase agreements for low-income countries: practical issues.

Authors: Ernst R Berndt; John A Hurvitz
Journal: Health Aff (Millwood) Date: 2005 May-Jun Impact factor: 6.301

Review 3. Lessons learned from the clinical development and market authorization of Glybera.

Authors: Laura M Bryant; Devin M Christopher; April R Giles; Christian Hinderer; Jesse L Rodriguez; Jenessa B Smith; Elizabeth A Traxler; Josh Tycko; Adam P Wojno; James M Wilson
Journal: Hum Gene Ther Clin Dev Date: 2013-06-29 Impact factor: 5.032

Review 4. An empirical review of major legislation affecting drug development: past experiences, effects, and unintended consequences.

Authors: Aaron S Kesselheim
Journal: Milbank Q Date: 2011-09 Impact factor: 4.911

Review 5. Gene therapy through autologous transplantation of gene-modified hematopoietic stem cells.

Authors: Donald B Kohn; Sung-Yun Pai; Michel Sadelain
Journal: Biol Blood Marrow Transplant Date: 2012-09-29 Impact factor: 5.742

Review 6. Recombinant adeno-associated virus: clinical application and development as a gene-therapy vector.

Authors: Ping-Jie Xiao; Thomas B Lentz; R Jude Samulski
Journal: Ther Deliv Date: 2012-07

7. New expensive treatments for hepatitis C infection.

Authors: Troyen Brennan; William Shrank
Journal: JAMA Date: 2014-08-13 Impact factor: 56.272

8. Safety and efficacy of gene transfer for Leber's congenital amaurosis.

Authors: Albert M Maguire; Francesca Simonelli; Eric A Pierce; Edward N Pugh; Federico Mingozzi; Jeannette Bennicelli; Sandro Banfi; Kathleen A Marshall; Francesco Testa; Enrico M Surace; Settimio Rossi; Arkady Lyubarsky; Valder R Arruda; Barbara Konkle; Edwin Stone; Junwei Sun; Jonathan Jacobs; Lou Dell'Osso; Richard Hertle; Jian-xing Ma; T Michael Redmond; Xiaosong Zhu; Bernd Hauck; Olga Zelenaia; Kenneth S Shindler; Maureen G Maguire; J Fraser Wright; Nicholas J Volpe; Jennifer Wellman McDonnell; Alberto Auricchio; Katherine A High; Jean Bennett
Journal: N Engl J Med Date: 2008-04-27 Impact factor: 91.245

9. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Authors: Amit C Nathwani; Edward G D Tuddenham; Savita Rangarajan; Cecilia Rosales; Jenny McIntosh; David C Linch; Pratima Chowdary; Anne Riddell; Arnulfo Jaquilmac Pie; Chris Harrington; James O'Beirne; Keith Smith; John Pasi; Bertil Glader; Pradip Rustagi; Catherine Y C Ng; Mark A Kay; Junfang Zhou; Yunyu Spence; Christopher L Morton; James Allay; John Coleman; Susan Sleep; John M Cunningham; Deokumar Srivastava; Etiena Basner-Tschakarjan; Federico Mingozzi; Katherine A High; John T Gray; Ulrike M Reiss; Arthur W Nienhuis; Andrew M Davidoff
Journal: N Engl J Med Date: 2011-12-10 Impact factor: 176.079

10. Musings on genome medicine: enzyme-replacement therapy of the lysosomal storage diseases.

Authors: David G Nathan; Stuart H Orkin
Journal: Genome Med Date: 2009-12-09 Impact factor: 11.117

10 in total

22 in total

The special case of gene therapy pricing.

1. Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.

2. Vaccine advance-purchase agreements for low-income countries: practical issues.

Review 3. Lessons learned from the clinical development and market authorization of Glybera.

Review 4. An empirical review of major legislation affecting drug development: past experiences, effects, and unintended consequences.

Review 5. Gene therapy through autologous transplantation of gene-modified hematopoietic stem cells.

Review 6. Recombinant adeno-associated virus: clinical application and development as a gene-therapy vector.

7. New expensive treatments for hepatitis C infection.

8. Safety and efficacy of gene transfer for Leber's congenital amaurosis.

9. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

10. Musings on genome medicine: enzyme-replacement therapy of the lysosomal storage diseases.

1. The payers' perspective on gene therapies.

Review 2. What Is Next for Retinal Gene Therapy?

Review 3. Chromatin regulation at the frontier of synthetic biology.

Review 4. Genome editing and the next generation of antiviral therapy.

5. The business case for cell and gene therapies.

6. Glybera's second act: the curtain rises on the high cost of therapy.

7. Public biotech in 2014--the numbers.

8. $1-million price tag set for Glybera gene therapy.

9. Beating the organ clock.

10. Biopharmaceutical benchmarks 2014.