Commissioning for rare diseases: View from the frontline

British Medical Journal

Volumn 331, Issue 7523, 2005, Pages 1019-1021

  Burls, Amanda ^a   Austin, Daphne ^b   Moore, David ^a  

^a UNIVERSITY OF BIRMINGHAM   (United Kingdom)

^b West Midlands Specialised Services Agency   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; IMIGLUCERASE; LARONIDASE; ORPHAN DRUG;

COST EFFECTIVENESS ANALYSIS; DECISION MAKING; DRUG COST; ENZYME REPLACEMENT; FABRY DISEASE; FINANCIAL MANAGEMENT; GAUCHER DISEASE; HEALTH CARE COST; HEALTH ECONOMICS; HUMAN; HURLER SYNDROME; LYSOSOME STORAGE DISEASE; PRIMARY MEDICAL CARE; PRIORITY JOURNAL; RARE DISEASE; SHORT SURVEY;

EID: 27644487456     PISSN: 09598146     EISSN: None     Source Type: Journal    
DOI: 10.1136/bmj.331.7523.1019     Document Type: Short Survey

References (13)

1. McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? BMJ 2005;331:1016-9.
2. Department of Health. Guidance on commissioning arrangements for specialised services. London: DoH, 2003.
3. Department of Health. National Specialist Commissioning Advisory Group (NSCAG). London: DoH, 2005.
4. McIver S. The views of the public about funding enzyme replacement therapy. Birmingham: West Midlands Specialised Services Agency, 2004.
5. Sheehan MP. Enzyme replacement therapy, resource allocation and distributive justice. Birmingham: West Midlands Specialised Services Agency, 2004.
6. Aggressive Research Intelligence Facility. Recombinant human alpha-L-iduronidase for the treatment of patients with mucopolysaccharidosis type 1. Birmingham: West Midlands Specialised Services Agency, 2004.
7. Aggressive Research Intelligence Facility. Recombinant human alpha-galactosidase for the treatment of patients with Fabry's disease. Birmingham: West Midlands Specialised Services Agency, 2004.
8. Connock MJ, Burls A, Frew E, Fry-Smith A, Juarez-Garcia A, McCabe C, et al. The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease. Health Technol Assess (in press).
9. Connock MJ, Frew E, Juarez-Garcia A, Mans A, Dretzke J, Fry-Smith A, Moore D. The clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1. Health Technol Assess (in press).
10. National Specialised Commissioning Advisory Group. Lysosomal storage disorders: NSCAG's policy on the funding of enzyme replacement therapy and substrate reduction therapy. London: DoH, 2005.
11. Department of Health. Mechanisms and funding of NSCAG. www.advisorybodies.doh.gov.uk/nscag/how.htm (accessed 2 Oct 2005).
12. Canadian Coordinating Office for Health Technology Assessment. Laronidase CEDAC Final recommendations and reasons for recommendation. Ottawa: CCOHTA, 2005.
13. National Institute for Clinical Excellence. NICE citizen's council report on ultra orphan drugs. London: NICE, 2004.