The roles of academia, rare diseases, and repurposing in the development of the most transformative drugs

Health Affairs

Volumn 34, Issue 2, 2015, Pages 286-293

  Kesselheim, Aaron S ^a   Tan, Yongtian Tina ^b   Avorn, Jerry ^a  

^a BRIGHAM AND WOMEN S HOSPITAL   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALGLUCERASE; BISPHOSPHONIC ACID DERIVATIVE; BOTULINUM TOXIN A; CENTOXIN; CLOZAPINE; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; DRUG; FLUOXETINE; GLUCOSYLCERAMIDASE; HUMAN IMMUNODEFICIENCY VIRUS PROTEINASE INHIBITOR; IMATINIB; INFLIXIMAB; INTERFERON BETA SERINE; LATANOPROST; METFORMIN; MEVINOLIN; NITISINONE; OMEPRAZOLE; PROPOFOL; PROSTACYCLIN; RECOMBINANT ERYTHROPOIETIN; REMIFENTANIL; RITUXIMAB; SEROTONIN UPTAKE INHIBITOR; SILDENAFIL; SUMATRIPTAN; TAMSULOSIN; TUMOR NECROSIS FACTOR INHIBITOR; ZIDOVUDINE; ZIMELDINE;

ARTICLE; DRUG DEVELOPMENT; DRUG INDUSTRY; DRUG REPOSITIONING; DRUG RESEARCH; FOOD AND DRUG ADMINISTRATION; FUNDING; GAUCHER DISEASE; GOVERNMENT; HUMAN; RARE DISEASE; DRUG MANUFACTURE; ECONOMICS; LEGISLATION AND JURISPRUDENCE; MANPOWER; PERSONNEL; PROCEDURES; RARE DISEASES; STANDARDS; UNITED STATES; UNIVERSITY HOSPITAL;

ACADEMIC MEDICAL CENTERS; DRUG INDUSTRY; DRUG REPOSITIONING; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES; RESEARCH PERSONNEL; UNITED STATES;

EID: 84922704898     PISSN: 02782715     EISSN: 15445208     Source Type: Journal    
DOI: 10.1377/hlthaff.2014.1038     Document Type: Article

References (36)

1. Pammolli F, Magazzini L, Riccaboni M. The productivity crisis in pharmaceutical R&D. Nat Rev Drug Discov. 2011;10(6):428-38.
2. Arrowsmith J. A decade of change. Nat Rev Drug Discov. 2012;11(1): 17-8.
3. Lincker H, Ziogas C, Carr M, Porta N, Eichler HG. Regulatory watch: where do new medicines originate from in the EU? Nat Rev Drug Discov. 2014;13(2):92-3.
4. Kesselheim AS, Avorn J. The most transformative drugs of the past 25 years: a survey of physicians. Nat Rev Drug Discov. 2013;12(6):425-31.
5. Kesselheim AS, Wang B, Avorn J. Defining "innovativeness" in drug development: a systematic review. Clin Pharmacol Ther. 2013;94(3): 336-48.
6. Garber AM, Clarke AE, Goldman DP, Gluck ME. Federal and private roles in the development and provision of alglucerase therapy for Gaucher disease [Internet]. Washington (DC): Office of Technology Assessment; 1992 Oct [cited 2014 Dec 5]. Available from: https://www.princeton.edu/~ota/disk1/1992/9214/9214.PDF
7. Easton J. Eugene Goldwasser, biochemist behind blockbuster anemia drug, 1922-2010. UChicagoNews [serial on the Internet]. 2010 Dec 22 [cited 2014 Dec 8]. Available from: http://news.uchicago.edu/article/2010/12/22/eugene-goldwasserbiochemist-behind-blockbusteranemia-drug-1922-2010
8. Lin FK, Suggs S, Lin CH, Browne JK, Smalling R, Egrie JC, et al. Cloning and expression of the human erythropoietin gene. Proc Natl Acad Sci U S A. 1985;82(22):7580-4.
9. Frances MD, Valent DJ. Historical perspectives on the clinical development of bisphosphanates in the treatment of bone diseases. J Musculoskelet Neuronal Interact. 2007; 7(1):2-8.
10. Caine M, Raz S, Zeigler M. Adrenergic and cholinergic receptors in the human prostate, prostatic capsule, and bladder neck. Br J Urol. 1975;47(2):193-202.
11. Takenaka T. Legends in urology. Can J Urol. 2010;17(5):5344-5.
12. Carlsson A. A historical note on the development of zimelidine, the first selective serotonin reuptake inhibitor. Eur Psychiatry. 1996;11(Suppl 4):S235-6.
13. Wong DT, Perry KW, Bymaster FP. Case history: the discovery of fluoxetine hydrochloride (Prozac). Nat Rev Drug Discov. 2005;4(9):764-74.
14. Nussbaum B. Good intentions: how big business and the medical establishment are corrupting the fight against AIDS. New York (NY): Atlantic Monthly Press; 1990.
15. Dronsfield A, Ellis P. Drug discovery: metformin and the control of diabetes. Education in Chemistry. 2011; Nov:185-7.
16. Mueller H. Tumor necrosis factor as an antineoplastic agent: pitfalls and promises. Cell Mol Life Sci. 1998; 54(12):1291-8.
17. Qiu P, Cui X, Barochia A, Li Y, Natanson C, Eichacker PQ. The evolving experience with therapeutic TNF inhibition in sepsis: considering the potential influence of risk of death. Expert Opin Investig Drugs. 2011;20(11):1555-64.
18. Schoemaker HJ, Schoemaker AF. The three pillars of bioentrepreneurship. Nat Biotech. 1998; 16(Suppl):13-5.
19. Feldmann M, Maini R. Discovery of TNF-alpha as a therapeutic target in rheumatoid arthritis: preclinical and clinical studies. Joint Bone Spine. 2002;69(1):12-8.
20. Van Dullemen HM, van Deventer SJ, Hommes DW, Bijl HA, Jansen J, Tytgat GN, et al. Treatment of Crohn's disease with anti-tumor necrosis factor chimeric monoclonal antibody (cA2). Gastroenterology. 1995;109(1):129-35.
21. Francis MD, Valent DH. Historical perspectives on the clinical development of bisphosphonates in the treatment of bone diseases. J Musculoskelet Neuronal Interact. 2007; 7(1):2-8.
22. McKiernan PJ. Nitisinone in the treatment of hereditary tyrosinaemia type 1. Drugs. 2006;66(6):743-50.
23. Kim LA, D'Amore PA. A brief history of anti-VEGF for the treatment of ocular angiogenesis. Am J Pathol. 2012;181(2):376-9.
24. Ghofrani HA, Osterloh IH, Grimminger F. Sildenafil: from angina to erectile dysfunction to pulmonary hypertension and beyond. Nat Rev Drug Discov. 2006;5(8): 689-02.
25. Ting PT, Freiman A. The story of Clostridium botulinum: from food poisoning to Botox. Clin Med. 2004;4(3):258-61.
26. Stevens AJ, Jensen JJ, Wyller K, Kilgore PC, Chatterjee S, Rohrbaugh ML. The role of public-sector research in the discovery of drugs and vaccines. N Engl J Med. 2011;364(6): 535-41.
27. Reichert JM, Milne CP. Public and private sector contributions to the discovery and development of "impact" drugs. Am J Ther. 2002;9(6): 543-55.
28. Holmer AF. Testimony by President and CEO of PhRMA before the Senate Committee on Health, Education, Labor, and Pensions. Congressional Record, S. Hrg. 106-651 (13 June 2000) [Internet]. Washington (DC): Government Printing Office; 2000 [cited 2015 Jan 2]. p. 91. Available from: https://ia601407.us.archive.org/12/items/drugsafetypricin00unit/drugsafetypricin00unit.pdf
29. Pharmaceutical Research and Manufacturers of America. 2013 profile: biopharmaceutical research industry [Internet]. Washington (DC): PhRMA; 2013 Jul [cited 2014 Dec 8]. Available from: http://www.phrma.org/sites/default/files/pdf/PhRMA%20Profile%202013.pdf
30. Light DW, Lexchin J. Foreign free riders and the high price of US medicines. BMJ. 2005;331(7522): 958-60.
31. Ramamoorthi R, Graef KM, Krattiger A, Dent JC. WIPO Re: Search: catalyzing collaborations to accelerate product development for diseases of poverty. Chem Rev. 2014 Sep 17. [Epub ahead of print].
32. Editorial Board. NIH tries a new approach to speed drug development. Washington Post. 2014 Feb 8.
33. Allison M. NCATS launches drug repurposing program. Nat Biotechnol. 2012;30(7):571-2.
34. National Center for Advancing Translational Sciences. Discovering new therapeutic uses for existing molecules [Internet]. Bethesda (MD): NCATS; 2014 May [cited 2014 Dec 8]. Available from: http://www.ncats.nih.gov/files/factsheettherapeutics-rev.pdf
35. Braun M, Frag-El-Massah S, Xu K, Cote T. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nat Rev Drug Discov. 2010;9(7):519-22.
36. Kesselheim AS, Myers JA, Avorn J. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer. JAMA. 2011;305(22):2320-6.