References
- Rare diseases. [online]. Available from: https://ec.europa.eu/health/non_communicable_diseases/rare_diseases_en. Accessed April 12, 2020.
- The UK strategy for rare diseases; 2013. Available from: https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/260562/UK_Strategy_for_Rare_Diseases.pdf. Accessed April 12, 2020.
- Initial draft discussion document for a Canadian orphan drug regulatory framework 2012. Available from: http://www.orpha.net/national/data/CA-EN/www/uploads/Initial-Draft-Discussion-Document-for-A-Canadian-Orphan-Drug–Regulatory-Framework.doc. Accessed April 12, 2020.
- Orphan drug act—relevant excerpts; 2016. Available from: https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/orphan-drug-act-relevant-excerpts. Accessed April 12, 2020.
- Giannuzzi V, Conte R, Landi A, et al. Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen. Orphanet J Rare Dis. 2017;12(1):64. doi:10.1186/s13023-017-0617-1
- Fonseca DA, Amaral I, Pinto AC, Cotrim MD. Orphan drugs: major development challenges at the clinical stage. Drug Discov Today. 2019;24(3):867–872. doi:10.1016/j.drudis.2019.01.005
- Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care. 2007;23(1):36–42. doi:10.1017/S0266462307051550
- Pearson I, Rothwell B, Olaye A, Knight C. Economic modeling considerations for rare diseases. Value Health. 2018;21(5):515–524. doi:10.1016/j.jval.2018.02.008
- Hyry HI, Stern AD, Cox TM, Roos JC. Limits on use of health economic assessments for rare diseases. QJM. 2014;107(3):241–245. doi:10.1093/qjmed/hcu016
- Rawson NSL, Donna New Patented Medicine Regulations in Canada: Updated Case Study of a Manufacturer’s Decision-Making About a Regulatory Submission for a Rare Disorder Treatment. Canadian Health Policy; January 2020.
- Board TPMPR. PMPRB draft guidelines 2020; 2020. Available from: https://www.canada.ca/content/dam/pmprb-cepmb/documents/consultations/draft-guidelines/2020/PMPRB-Guidelines2020-en.pdf. Accessed June 21, 2020.
- Study on orphan drugs; 2005. Available from: https://ec.europa.eu/health//sites/health/files/files/orphanmp/doc/pricestudy/final_final_report_part_1_web_en.pdf. Accessed April 12, 2020.
- Hughes-Wilson W, Palma A, Schuurman A, Simoens S. Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet J Rare Dis. 2012;7:74. doi:10.1186/1750-1172-7-74
- Schuller Y, Hollak CE, Biegstraaten M. The quality of economic evaluations of ultra-orphan drugs in Europe - a systematic review. Orphanet J Rare Dis. 2015;10:92. doi:10.1186/s13023-015-0305-y
- Richter T, Janoudi G, Amegatse W, Nester-Parr S. Characteristics of drugs for ultra-rare diseases versus drugs for other rare diseases in HTA submissions made to the CADTH CDR. Orphanet J Rare Dis. 2018;13(1):15. doi:10.1186/s13023-018-0762-1
- Annemans L, Ayme S, Le Cam Y, et al. Recommendations from the European Working Group for value assessment and funding processes in rare diseases (ORPH-VAL). Orphanet J Rare Dis. 2017;12(1):50. doi:10.1186/s13023-017-0601-9
- Gammie T, Lu CY, Babar ZU. Access to orphan drugs: a comprehensive review of legislations, regulations and policies in 35 countries. PLoS One. 2015;10(10):e0140002. doi:10.1371/journal.pone.0140002
- Drummond MF. Challenges in the economic evaluation of orphan drugs. Eurohealth. 2008;14(2):16–17.
- Chambers JD, Silver MC, Berklein FC, Cohen JT, Neumann PJ. Orphan drugs offer larger health gains but less favorable cost-effectiveness than non-orphan drugs. J Gen Intern Med. 2020. doi:10.1007/s11606-020-05805-2
- DRUG REIMBURSEMENT RECOMMENDATION Burosumab (Crysvita); May 27, 2020. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0602%20Crysvita%20-%20CDEC%20Final%20Recommendation%20May%2029%2C%202020_For%20posting.pdf. Accessed June 25, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Tafamidis Meglumine (Vyndaqel); February 19, 2020. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0625%20Vyndaqel%20-%20CDEC%20Final%20%20Recommendation%20February%2020%2C%202020%20for%20posting.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Inotersen (Tegsedi); December 18, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0603%20Tegsedi%20-%20Final%20CDEC%20Recommendation%20December%2020%2C%202019%20for%20posting.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Teduglutide (Revestive) 2019; November 19, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0606%20Revestive%20-%20CDEC%20Final%20Recommendation%20November%2021%2C%202019_For%20posting.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION lanadelumab (Takhzyro) 2019; November 19, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0618%20Takhzyro%20-%20CDEC%20Final%20Recommendation%20November%2022%2C%202019_for%20posting.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW Pharmacoeconomic Review Report for lanadelumab (Takhzyro); November 19, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/sr0618-takhzyro-pharmacoeconomic-review-report.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Telotristat ethyl (Xermelo) 2019; June 19, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0580%20Xermelo%20-%20CDEC%20Final%20Recommendation%20July%203%2C%202019_For%20Posting.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Cysteamine (Cystadrops) 2019; June 18, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0595%20Cystadrops%20-%20CDEC%20Final%20Recommendation%20June%2020%2C%202019_for%20posting.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Cerliponase alfa (Brineura) 2019; April 10, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0574%20Brineura%20-%20Final%20CDEC%20Recommendation%20May%2027%2C%202019_for%20posting.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION nusinersen (Spinraza); February 27, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0576-Spinraza-Resubmission-Mar-1-19.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Sebelipase alfa (Kanuma); September 26, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0544%20Kanuma%20-%20CDEC%20Final%20Recommendation%20September%2028%2C%202018.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Nitisinone (Nitisinone Tablets). August 21, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0554_Nitisinone_Aug_23_18.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Nitisinone (MDK-Nitisinone) 2018; April 25, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0538_cdr_complete_MDK_Nitisinone_Apr_27_18_e.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION tocilizumab (Actemra) 2018; February 21, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0534_Actemra_GCA_complete_Mar-27-18.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION nitisinone (Orfadin) 2018; February 21, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0531_Orfadin_complete_Feb-23-18.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Migalastat (Galafold) 2018; January 24, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0522_Galafold_complete_Jan-26-18.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Cysteamine Delayed-Release (Procysbi); December 13, 2017. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0526_Procysbi_complete_Jan-26-18.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Eliglustat (Cerdelga); July 26, 2017. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0511_complete_Cerdelga_Jul_28_17_e.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Obeticholic Acid (Ocaliva) 2017; 21 June 2017. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0509_complete_Ocaliva_Jul_27_17_e.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION glycerol phenylbutyrate (Ravicti) 2017; March 21, 2017. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0497_complete_Ravicti_Mar-23-17.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Selexipag (Uptravi); October 28, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0497_Ravicti_PE_Report.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Sapropterin dihydrochloride (Kuvan); October 26, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0472_complete_Kuvan-Oct-28-16.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Tesamorelin (Egrifta); August 24, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0477_complete_Egrifta_Aug-26-16.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Teduglutide (Revestive); July 29, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0459_Revestive_complete_Jul-29_16.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Canakinumab (Ilaris); June 21, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0463_complete_Ilaris_sJIA_June_21_16_e.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION elosulfase alfa (Vimizim); May 26, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0456_complete_Vimizim_May-26_16.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION adalimumab (Humira); May 24, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0455_complete_Humira-HS_May-24-16_e.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Sodium phenylbutyrate (Pheburane); April 25, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0452_complete_Pheburane-Apr-25_16-e.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Asfotase alfa (Strensiq). April 4, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0443_complete_Strensiq-Apr-4-16_e.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Tolvaptan (Jinarc); February 26, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0435_complete_Jinarc-Feb_26_16_e.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION taliglucerase alfa (Elelyso); October 30, 2015. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0390_Elelyso_Oct-30-15.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION lomitapide (Juxtapid); April 21, 2015. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/cdr_complete_SR0386_Juxtapid-Apr-21_15.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION AbobotulinumtoxinA (Dysport Therapeutic); July 28, 2017. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0512_complete_Dysport_Therapeutic_Jul_28_17_e.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION macitentan (Opsumit); January 30, 2015. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/cdr_complete_SR0364_Opsumit_Jan-30-15.pdf. Accessed April 12, 2020.
- DRUG REIMBURSEMENT RECOMMENDATION Riociguat (Adempas); December 17, 2015. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0438_complete_Adempas-Dec-21-15_e.pdf. Accessed August 6, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic review report for tafamidis meglumine (Vyndaqel); February 19, 2020. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/sr0625-vyndaqel-pharmacoeconomic-review-report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic review report for inotersen (Tegsedi); December 18, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/sr0603-tegsedi-pharmacoeconomic-review-report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic review report for teduglutide (Revestive); November 19, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/sr0606-revestive-pharmacoeconomic-review-report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic review report for patisiran (Onpattro); July 25, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/sr0598-onpattro-pharmacoeconomic-review-report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic review report for telotristat ethyl (Xermelo); June 19, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/sr0580-xermelo-pharmacoeconomic-review-report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic review report for cysteamine (Cystadrops). June 18, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/sr0595-cystadrops-pharmacoeconomic-review-report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW Pharmacoeconomic Review Report for Cerliponase alfa (Brineura); April 10, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/sr0574-brineura-pharmacoeconomic-review-report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic review report for nusinersen; February 27, 2019. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/sr0576-spinraza-resubmission-pharmacoeconomic-report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic review report for kanuma; September 26, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0544_Kanuma_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic review report for nitisinone tablets; August 21, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0554_Nitisinone_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for MDK-nitisinone; April 25, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0538_MDK_Nitisinone_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Actemra; February 21, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0534_Actemra_GCA_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for nitisinone (Orfadin); February 21, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0531_Orfadin_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Galafold; January 24, 2018. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0522_Galafold_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Procysbi; December 13, 2017. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0526_Procysbi_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Ocaliva; July 25, 2017. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0509_Ocaliva_PE_Report_e.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Ravicti; March 21, 2017. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0497_Ravicti_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Uptravi; October 28, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0482_Uptravi_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Kuvan; October 26, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/complete/SR0472_complete_Kuvan-Oct-28-16.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Egrifta; August 26, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0477_Egrifta_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Revestive; July 29, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0459_Revestive_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Ilaris; June 21, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0463_Ilaris_sJIA_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Vimizim; MAy 26, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0456_Vimizim_Resubmission_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Humira; May 24, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0455_HumiraHS_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Strensiq; April 4, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0443_Strensiq_PE_Report.pdf. Accessed April 12, 2020.
- CADTH COMMON DRUG REVIEW pharmacoeconomic report for Jinarc; February 26, 2016. Available from: https://www.cadth.ca/sites/default/files/cdr/pharmacoeconomic/SR0435_Jinarc_PE_Report.pdf. Accessed April 12, 2020.