Protein replacement therapies for rare diseases: A breeze for regulatory approval?

Science Translational Medicine

Volumn 5, Issue 178, 2013, Pages

  Gorzelany, Jennifer A ^a   De Souza, Mark P ^b  

^a Laurelwood Biopartners   (United States)

^b Shire Human Genetic Therapies   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR; LYSOSOME ENZYME; ORPHAN DRUG;

COST EFFECTIVENESS ANALYSIS; DRUG APPROVAL; DRUG CONTROL; DRUG COST; DRUG INDICATION; DRUG INDUSTRY; DRUG LEGISLATION; DRUG MARKETING; DRUG PROGRAM; HUMAN; PRIORITY JOURNAL; PROTEIN REPLACEMENT THERAPY; QUALITY OF LIFE; RARE DISEASE; REIMBURSEMENT; SHORT SURVEY; SUBSTITUTION THERAPY;

DRUG APPROVAL; ENZYME REPLACEMENT THERAPY; HUMANS; ORPHAN DRUG PRODUCTION; PROTEINS; RARE DISEASES; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84876115251     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3005007     Document Type: Short Survey

References (10)

1. H. Hyde, D. Dobrovolny, Orphan drug pricing and payer management in the United States: Are we approaching the tipping point? Am. Health Drug Benefits. 3, 15-23 (2010).
2. M. Schlander, M. Beck, Expensive drugs for rare disorders: To treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI. Curr. Med. Res. Opin. 25, 1285-1293 (2009).
3. D. A. Hughes, B. Tunnage, S. T. Yeo, Drugs for exceptionally rare diseases: Do they deserve special status for funding? QJM 98, 829-836 (2005).
4. M. Rohrbach, J. T. Clarke, Treatment of lysosomal storage disorders: Progress with enzyme replacement therapy. Drugs 67, 2697-2716 (2007).
5. M. Connock, A. Burls, E. Frew, A. Fry-Smith, A. Juarez-Garcia, C. McCabe, A. Wailoo, K. Abrams, N. Cooper, A. Sutton, A. O'Hagan, D. Moore, The clinical eff ectiveness and cost-eff ectiveness of enzyme replacement therapy for Gaucher's disease: A systematic review. Health Technol. Assess. 10, iii-iv, ix-136 (2006).
6. T. Watt, A. P. Burlina, C. Cazzorla, D. Schönfeld, M. Banikazemi, R. J. Hopkin, A. M. Martins, K. Sims, D. Beitner-Johnson, F. O'Brien, U. Feldt-Rasmussen, Agalsidase beta treatment is associated with improved quality of life in patients with Fabry disease: Findings from the Fabry registry. Genet. Med. 12, 703-712 (2010).
7. J. A. DiMasi, L. Feldman, A. Seckler, A. Wilson, Trends in risks associated with new drug development: Success rates for investigational drugs. Clin. Pharmacol. Ther. 87, 272-277 (2010).
8. Tufts Center for the Study of Drug Development, U.S. orphan product designations more than doubled from 2000-02 to 2006-08. Impact Report. 12 (2010); available at http://csdd.tufts.edu/files/uploads/2010-jan-feb-summary. pdf.
9. K. I. Kaitin, J. A. DiMasi, Pharmaceutical innovation in the 21st century: New drug approvals in the fi rst decade, 2000-2009. Clin. Pharmacol. Ther. 89, 183-188 (2011).
10. S. M. Paul, D. S. Mytelka, C. T. Dunwiddie, C. C. Persinger, B. H. Munos, S. R. Lindborg, A. L. Schacht, How to improve R&D productivity: The pharmaceutical industry's grand challenge. Nat. Rev. Drug Discov. 9, 203-214 (2010).