ALTERNATIVE MEDICINE;
ARTICLE;
CONTROLLED STUDY;
COST;
DRUG APPROVAL;
DRUG INDICATION;
DRUG THERAPY;
FOOD AND DRUG ADMINISTRATION;
HEALTH CARE ACCESS;
HEALTH CARE UTILIZATION;
HEALTH INSURANCE;
HOSPITAL PHARMACY;
HUMAN;
OUTPATIENT DEPARTMENT;
PATIENT SELECTION;
RARE DISEASE;
DRUG MANUFACTURE;
ECONOMICS;
HEALTH CARE DELIVERY;
INSURANCE;
RARE DISEASES;
UNITED STATES;
COST SHARING;
HEALTH INSURANCE EXCHANGES;
HEALTH SERVICES ACCESSIBILITY;
HUMANS;
INSURANCE COVERAGE;
INSURANCE, PHARMACEUTICAL SERVICES;
ORPHAN DRUG PRODUCTION;
RARE DISEASES;
UNITED STATES;
Designation of an Orphan Drug, 21 CFR 316.20 (2012). Available at: http://www.ecfr.gov/cgi-bin/text-idx?rgn=div5&node=21:5.0.1.1.6# se21.5.316_120. Accessed August 28, 2014.
National Center for Advancing Translational Sciences. FAQ about rare diseases. Available at: http://www.ncats.nih.gov/about/faq/rare/rare-faq.html. Accessed August 17, 2014.
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National Institutes of Health. NIH announces new program to develop therapeutics for rare and neglected diseases. Press release. May 20, 2009. Available at: https://rarediseases.info.nih.gov/files/TRND%20Press%20 Release.pdf. Accessed August 17, 2014.
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Orphan-Drug Exclusive Approval, 21 CFR 316.31 (2012). Available at: http://www.ecfr.gov/cgi-bin/text-idx?rgn=div5&node=21:5.0.1.1.6# se21.5.316_131. Accessed August 28, 2014.
Faden L, Huskamp H. Appendix C: Medicare Part D coverage and reimbursement of orphan drugs. In: Field MJ, Boat TF, eds. Rare Diseases and Orphan Products: Accelerating Research and Development. Washington, DC: National Academies Press; 2010:309-43. Available at: http://www.ncbi.nlm. nih.gov/books/NBK56190/. Accessed August 17, 2014.
Institute of Medicine. Rare diseases and orphan products: accelerating research and development. Report brief. October 4, 2010. Available at: http:// www.iom.edu/~/media/Files/Report%20Files/2010/Rare-Diseases-and-OrphanProducts- Accelerating-Research-and-Development/Rare%20Disease%20 Research%202010%20Report%20Brief.pdf. Accessed August 17, 2014.
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Coverage of Essential Health Benefits, 45 CFR 156.140 (2012). Available at: http://www.ecfr.gov/cgi-bin/text-idx?SID=06591a443a74a17909f2f9db0d023efe&node=se45.1.156_1140&rgn=div8. Accessed August 28, 2014.
The Patient Protection and Affordable Care Act, Public Law 111-148. March 23, 2010. Available at: http://www.gpo.gov/fdsys/pkg/PLAW111publ148/ pdf/PLAW-111publ148.pdf. Accessed September 8, 2014.
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Coverage of Essential Health Benefits, 45 CFR 147.150 (2012). Available at: http://www.ecfr.gov/cgi-bin/retrieveECFR?gp=&SID=1787c2cea700807b70c897eacafd3eb0&r=PART&n=45y1.0.1.2.62#se45.1.147_1150. Accessed August 28, 2014.
Prescription Drug Benefit, 45 CFR 156.122 (2012). Available at: http:// www.ecfr.gov/cgi-bin/retrieveECFR?gp=&SID=7a1068a2d7e1d3583388896e3601d4c0&n=sp45.1.156.b&r=SUBPART&ty=HTML#se45.1.156_1122. Accessed August 28, 2014.
United States Pharmacopeia. Medicare Model Guidelines Version 5.0. 2011. Available at: http://www.usp.org/sites/default/files/usp_pdf/EN/healthcare Professionals/2011-03-11uspmgcatclass.pdf. Accessed August 17, 2014.
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Center for Consumer Information & Insurance Oversight (CCIIO). Letter to issuers on federally-facilitated and state partnership exchanges. April 5, 2013. Available at: http://www.cms.gov/CCIIO/Resources/ Regulations-and-Guidance/Downloads/2014_letter_to_issuers_04052013. pdf. Accessed August 17, 2014.
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Center for Consumer Information & Insurance Oversight (CCIIO). Additional information on proposed state essential health benefits benchmark pans. Available at: http://www.cms.gov/cciio/resources/data-resources/ ehb.html. Accessed August 17, 2014.
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Office of Rare Diseases Research. Rare diseases with FDA-approved medical products. Available at: http://rarediseases.info.nih.gov/gard/diseaseswith- medical-products/A. Accessed August 17, 2014.
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U.S. Food and Drug Administration. Search orphan drug designations and approvals. Available at: http://www.accessdata.fda.gov/scripts/opdlisting/ oopd/. Accessed August 17, 2014.
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Soliris (eculizumab) prescribing information. Alexion Pharmaceuticals, Inc. Revised April 2014. Available at: http://soliris.net/sites/default/files/ assets/soliris_pi.pdf. Accessed August 17, 2014.
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Ceredase (alglucerase injection) prescribing information. Genzyme Corporation. February 9, 2006. Available at: http://www.accessdata.fda.gov/ drugsatfda_docs/label/2006/020057s034lbl.pdf. Accessed August 17, 2014.
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Cerezyme (imiglucerase for injection) prescribing information. Genzyme Corporation. 2002. Available at: http://www.cerezyme.com/~/media/ CerezymeUS/Files/pdf/cerezyme_pi.pdf. Accessed August 17, 2014.
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Elelyso (taliglucerase alfa) prescribing information. Pfizer Labs. Revised May 2014. Available at: http://www.elelyso.com/pdf/ELELYSO_Prescribing_ Information.pdf. Accessed August 17, 2014.
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Vpriv (velaglucerase alfa for injection) prescribing information. Shire Human Genetic Therapies, Inc. Revised August 2013. Available at: http:// www.vpriv.com/app_presentation/vpriv/images/pdf/VPRIV_PI_Prescribing_ Information.pdf. Accessed August 17, 2014.
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Unpublished data from Avalere Health Enrollment Model. Scenario 2 (assumes 24 states opt out of the Medicaid expansion). December 2013.
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The Kaiser Family Foundation. Focus on health reform. Patient cost- sharing under the Affordable Care Act. April 2012. Available at: http:// kaiserfamilyfoundation.files.wordpress.com/2013/01/8303.pdf. Accessed August 17, 2014.
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U.S. Department of Health and Human Services. Office of the Assistant Secretary for Planning and Evaluation. Health insurance marketplace: summary enrollment report for the initial annual open enrollment period. ASPE Issue Brief. May 1, 2014. Available at: http://aspe.hhs.gov/health/ reports/2014/MarketPlaceEnrollment/Apr2014/ib_2014Apr_enrollment.pdf. Accessed August 17, 2014.
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Cost-Sharing Requirements, 45 CFR 156.130 (2012). Available at: http:// www.ecfr.gov/cgi-bin/text-idx?type=simple,c=ecfr;cc=ecfr;idno=45;region=DI V1;q1=155;rgn=div5;sid=7fda22484d8dcdd44f869475d74823d6;view=text;n ode=45%3A1.0.1.2.71#se45.1.156_1130. Accessed August 28, 2014.
ConnectiCare. Standard silver point-of-service. HD 3000 silver plan. Summary of benefits and coverage. 2014. Available at: http://www.con- necticare.com/globalfiles/sbc/POS-OA-30-45-3000HD-CAL-HIXS-F.pdf. Accessed August 17, 2014.
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NY State of Health. Standard silver plan. Schedule of benefits, pp 6-10. Available at: http://www.healthbenefitexchange.ny.gov/sites/default/files/ Standard%20Products.pdf. Accessed August 17, 2014.