Challenges and Hurdles to Business as Usual in Drug Development for Treatment of Rare Diseases

Clinical Pharmacology and Therapeutics

Volumn , Issue , 2016, Pages 339-341

  Swinney, D C ^a  

^a Institute for Rare and Neglected Diseases Drug Discovery   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMMERCIAL PHENOMENA; DRUG APPROVAL; DRUG DESIGN; DRUG INDUSTRY; DRUG RESEARCH; NOTE; PHARMACEUTICAL CARE; PRIORITY JOURNAL; RARE DISEASE;

EID: 84986612831     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1002/cpt.422     Document Type: Note

References (9)

1. Lanthier, M., Miller, K.L., Nardinelli, C. & Woodcock, J. An improved approach to measuring drug innovation finds steady rates of first-in-class pharmaceuticals, 1987–2011. Health Aff. (Millwood) 32, 1433–1439 (2013).
2. Pariser, A.R. & Gahl, W.A. Important role of translational science in rare disease innovation, discovery, and drug development. J. Gen. Intern. Med. 29 Suppl 3, S804–S807 (2014).
3. Munos, B. Lessons from 60 years of pharmaceutical innovation. Nat. Rev. Drug Discov. 8, 959–968 (2009).
4. Scannell, J.W., Blanckley, A., Boldon, H. & Warrington, B. Diagnosing the decline in pharmaceutical R&D efficiency. Nat. Rev. Drug Discov. 11, 191–200 (2012).
5. Swinney, D.C. Biochemical mechanisms of drug action: what does it take for success? Nat. Rev. Drug Discov. 3, 801–808 (2004).
6. Swinney, D.C. & Anthony, J. How were new medicines discovered? Nat. Rev. Drug Discov. 10, 507–519 (2011).
7. Swinney, D.C. & Xia, S. The discovery of medicines for rare diseases. Future Med. Chem. 6, 987–1002 (2014).
8. Buttoo, A.S., Israuels, M.C. & Wilkinson, J.F. Hypocholesterolaemia and orotic aciduria during treatment with 6-azauridine. Br. Med. J. 1, 552–554 (1965).
9. Collins, F.S. & Varmus, H. A new initiative on precision medicine. N. Engl. J. Med. 372, 793–795 (2015).