Orphan Drugs-The Reinvention of Specialty Pharmacy?
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1 Orphan Drugs-The Reinvention of Specialty Pharmacy? Gordon J. Vanscoy, PharmD, MBA, CACP; Chairman & CEO Jonathan Ogurchak, PharmD, CSP; Vice President, Business Operations Doug Gebhard, PharmD, MBA; Vice President, Quality & Education
2 Agenda Review the impact and status of legislation Orphan Drug Act Pediatric Rare Disease Voucher Program Delineate trends in FDA approvals & pipeline Discuss economic implications on healthcare Compare contemporary channel strategies Reinvention of Specialty Pharmacy?
3 Sources: 3, 6 Impact and Status of Legislation Orphan Drug Act Established in 1983 Aimed at stimulation of development of drug for rare diseases Established incentives for manufacturers Successful for development in areas such as acute myeloid leukemia, cystic fibrosis, and pulmonary arterial hypertension Requests for orphan designation have doubled since 2010
4 Sources: 3, 4, 6 Impact and Status of Legislation Pediatric Rare Disease Voucher Program Created in 2012 Vouchers are incentives meant to catalyze the development of new therapies for under-served disease states Awards priority review to sponsors of rare pediatric disease product applications that meet certain criteria Sponsors who receive an approval for a drug for a rare pediatric disease may qualify for a voucher 21 st Century Cures Act extended program to September 30, 2020
5 Sources: 5, 8 Trends in FDA Approvals & Pipeline Of the novel drugs approved: ~50% of total approvals were approved for orphan/rare indications in 2015 ~40% in 2016 Examples of orphan/ rare approvals: Exondys 51 Duchenne muscular dystrophy Kanuma lysosomal acid lipase deficiency Ocaliva primary biliary cholangitis Orkambi cystic fibrosis Spinraza spinal muscular atrophy Strensiq infantile- and juvenile-onset hypophosphatasia Unituxin pediatric neuroblastoma Xuriden hereditary orotic aciduria
6 Sources: 1, 2, 7 Trends in FDA Approvals & Pipeline Availability Approvals Impacted Population Areas of Focus Future Trends
7 Sources: 2, 7 Economic Implications of Orphan Drugs on Healthcare Orphan Expenditures Partial Orphan Drugs Drug Spend Incentive Healthcare Spend
8 Orphan Drug Channel Strategies Open Channel Limited Exclusive 8
9 Orphan Drug Channel Selection Specialty Pharmacy Characteristics: Stability & Reputation Accreditation(s) State Licensures Size: Goldilocks Principle Customizability & Flexibility Software / Information Systems Contracting / Payer Networks / Patient Assistance Programs Feedback / Satisfaction: Patients, Partners, Providers Full Operational Redundancy
10 Bringing Special Back to Specialty Competency Awareness Core Patient Centricity Performance & Outcomes Clinical Application of Technology Intelligent Data Analytics
11 References 1. Ault, A. "FDA Drowning in Orphan Drug Applications." Medscape, 19 Oct Web. 19 Dec < 2. Divino, V., M. Dekoven, M. Kleinrock, R. L. Wade, and S. Kaura. "Orphan Drug Expenditures In The United States: A Historical And Prospective Analysis, " Health Affairs 35.9 (2016): Web. 3. Gaffney, Alexander. "Pediatric Priority Review Voucher Program Set to End After FDA Approves New Drug RAPS." Pediatric Priority Review Voucher Program Set to End After FDA Approves New Drug RAPS. Regulatory Affairs Professionals Society, 18 Mar Web. 19 Dec < Focus/News/2015/03/18/21750/Pediatric-Priority-Review-Voucher-Program-Set-to-End-After-FDA-Approves-New- Drug/>. 4. Gingery, Derrick. "Sponsors May Race For Rare Pediatric Voucher Designations As Deadline Nears." Pink Sheet. N.p., 22 Dec Web. 23 Dec < Sponsors-May-Race- For-Rare-Pediatric-Voucher-Designations-As-Deadline-Nears>. 5. "Novel Drugs Summary 2015." Novel Drugs Summary U.S. Food & Drug Administration, Jan Web. 21 Dec < 6. "Orphan Drug Act - Relevant Excerpts." Orphan Drug Act - Relevant Excerpts. U.S. Food & Drug Administration, Aug Web. 19 Dec < signation/ucm htm>. 7. Orphan Drugs in the United States of America OrphaNet Jan Web. 4 Jan 2017 < GS_USA> 8. 9 Novel Orphan Drugs Approved in 2016" Rare Disease Report, Jan Web. 4 Jan
12 Thank You Questions?
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