Development and validation of COMPASS: Clinical evidence of orphan medicinal products - An assessment tool

Orphanet Journal of Rare Diseases

Volumn 8, Issue 1, 2013, Pages

  Picavet, Eline ^a   Cassiman, David ^b   Aertgeerts, Bert ^a,c   Simoens, Steven ^a  

^a UNIVERSITY OF LEUVEN   (Belgium)

^b UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^c Belgian Center for Evidence Based Medicine   (Belgium)

Author keywords

Quality assessment; Quality assessment tool; Quality of reporting

Indexed keywords

ARTICLE; CLINICAL ASSESSMENT TOOL; CLINICAL EVIDENCE OF ORPHAN MEDICINAL PRODUCT TOOL; CONSULTATION; DRUG INDUSTRY; EVIDENCE BASED MEDICINE; HUMAN; PHARMACIST; PHYSICIAN; REIMBURSEMENT;

DECISION MAKING; HUMANS; ORPHAN DRUG PRODUCTION; OUTCOME ASSESSMENT (HEALTH CARE); RARE DISEASES;

EID: 84885150620     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-8-157     Document Type: Article

References (23)

1. Regulation (EC) No 141/2000 of the European parliament and of the council of 16 December 1999 on orphan medicinal products. Commission E, Off J Euro Comm 2000 L18 1 5
2. Orphanet-The portal for rare diseases and orphan drugs http://www.orpha.net
3. Rare diseases and the assessment of intervention: what sorts of clinical trials can we use? Wilcken B, J Inherit Metab Dis 2001 24 291 298 10.1023/A:1010387522195
4. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Tambuyzer E, Nat Rev Drug Discov 2010 9 921 929 10.1038/nrd3275 21060315
5. European Medicines Agency http://www.ema.europa.eu/ema/
6. Role of carglumic acid in the treatment of acute hyperammonemia due to N-acetylglutamate synthase deficiency. Haberle J, Ther Clin Risk Manag 2011 7 327 332 21941437
7. Evidence-based medicine and rare diseases. Day S, Rare Dis Epidemiol 2010 686 41 53 10.1007/978-90-481-9485-8-3
8. GRADE guidelines: 3. Rating the quality of evidence. Balshem H, Helfand M, Schunemann HJ, Oxman AD, Kunz R, Brozek J, Vist GE, Falck-Ytter Y, Meerpohl J, Norris S, Guyatt GH, J Clin Epidemiol 2011 64 401 406 10.1016/j.jclinepi. 2010.07.015 21208779
9. Which clinical studies provide the best evidence? The best RCT still trumps the best observational study. Barton S, BMJ 2000 321 255 256 10.1136/bmj.321.7256.255 10915111
10. Clinical research for rare disease: opportunities, challenges, and solutions. Griggs RC, Batshaw M, Dunkle M, Gopal-Srivastava R, Kaye E, Krischer J, Nguyen T, Paulus K, Merkel PA, Mol Genet Metab 2009 96 20 26 10.1016/j.ymgme.2008.10.003 19013090
11. The role of evidence-based medicine and clinical trials in rare genetic disorders. Kruer MC, Steiner RD, Clin Genet 2008 74 197 207 10.1111/j.1399-0004. 2008.01041.x 18657147
12. Undertaking systematic reviews of research on effectiveness http://www.york.ac.uk/inst/crd/pdf/Systematic-Reviews.pdf
13. Assessing the quality of reports of randomized clinical trials: is blinding necessary? Jadad AR, Moore RA, Carroll D, Jenkinson C, Reynolds DJ, Gavaghan DJ, McQuay HJ, Control Clin Trials 1996 17 1 12 10.1016/0197-2456(95) 00134-4
14. Evaluating non-randomised intervention studies. Deeks JJ, Dinnes J, D'Amico R, Sowden AJ, Sakarovitch C, Song F, Petticrew M, Altman DG, Health Technol Assess 2003 7 ii-173
15. Reviews assessing the quality or the reporting of randomized controlled trials are increasing over time but raised questions about how quality is assessed. Dechartres A, Charles P, Hopewell S, Ravaud P, Altman DG, J Clin Epidemiol 2011 64 136 144 10.1016/j.jclinepi.2010.04.015 20705426
16. Scales to assess the quality of randomized controlled trials: a systematic review. Olivo SA, Macedo LG, Gadotti IC, Fuentes J, Stanton T, Magee DJ, Phys Ther 2008 88 156 175 10.2522/ptj.20070147 18073267
17. EUdraCT http://eudract.ema.europa.eu/
18. ClinicalTrials.gov clinicaltrials.gov/
19. Gaining reimbursement of orphan products in Europe: challenges Due to wide variations in evidence requirements and processes. Heron L, Laurenson S, Costello J, Anderl C, Knospe J, Value in Health 2012 15 309
20. Orphan drug pricing and access-current situation and future trends in Eu. Jeffery M, White R, Value in Health 2012 15 309
21. Food and Drug Administration, Guidance for Industry: Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics http://www.fda.gov/ downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071590.pdf
22. European Medicines Agency, Guideline on the evaluation of anticancer medicinal products in man http://www.ema.europa.eu/docs/en-GB/document-library/ Scientific-guideline/2013/01/WC500137128.pdf
23. Improving the quality of reporting of randomized controlled trials-The CONSORT statement. Begg C, Cho M, Eastwood S, Horton R, Moher D, Olkin I, Pitkin R, Rennie D, Schulz KF, Simel D, Stroup DF, JAMA 1996 276 637 639 10.1001/jama.1996.03540080059030 8773637