Evidence-Based Approach to Assuring Value for Orphan Drugs: Pam Gavin (NORD)
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Canadian Expert Patients in Health Technology Conference Nov 7 – 8, 2016: Day 2
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Evidence-Based Approach to Assuring Value for Orphan Drugs: Pam Gavin (NORD)
- 1. Evidence-Based Approach to Assuring Value for Orphan Drugs CEPHT Conference, Toronto November 8, 2016 Pamela Gavin Chief Operating Officer The National Organization for Rare Disorders
- 2. 33 YEARS AGO… • Only 10 RD treatments were developed by industry from 1973-1983 • Patient organizations formed an ad hoc committee • Waxman subcommittee hearings • Jack Klugman & Quincy ME episodes Alone we are rare. Together we are strong.® 2
- 3. 1983 ORPHAN DRUG ACT • Affecting less than 200,000 people nationwide • 7 years of marketing exclusivity • 50% tax credit on costs related to orphan drug clinical research • Federal grants for orphan product development Alone we are rare. Together we are strong.® 3
- 4. ODA Current Day Progress Alone we are rare. Together we are strong.® 4
- 5. That’s 1 in 10 people 30 Million Americans Are Impacted By Rare Disease 5
- 6. ⅔ of those impacted are children under the age of 5 95% are without any FDA approved treatment 100% are without a cure On average, it takes a patient 5-7 years to receive an accurate diagnosis Alone we are rare. Together we are strong. 6
- 7. Changing Landscape A Clear and Strong Movement Towards Patient- Centeredness: • 2010 Patient-Centered Outcomes Research Institute • 2012 FDA’s Patient Focused Drug Development • 2015 Precision Medicine 7Alone we are rare. Together we are strong.
- 8. How Can We Ensure Adequate Access is Provided?
- 9. NORD-Trio Partnership 9Alone we are rare. Together we are strong. Operational Quality Measures Clinical Quality Measures Access Accountability Awareness Advocacy
- 10. Program Objectives • Trio Health Patient Centric Technology Platform • Measures Stakeholder Performance • Scientific Steering Committee Defined Quality Measures • Publication and Awareness Advocating for Patients and High Performance • Business and Financial Model Based on Performance • Cost Effective Drug Distribution • Align all Patient Stakeholders Without Direct Control • Actionable Data Derived from a High Performance Network 10Alone we are rare. Together we are strong. 10
- 11. Trio Health 11 MD writes Rx Prior to Therapy During Therapy Out of Therapy Patient Starts Rx Patient completes Rx SPP sends Rx • Non-starts • Time to fill • Transfers between specialty pharmacies • Payer insights (appeals) • Earlier view of product demand • Patient consents • Discontinuations • Gaps between dispenses • Actual therapy start date • Outcomes Standard secondary data Patient Journey
- 12. Trio Health 12 Script Received Transfer PA Submitted PA Approved PA Denied Appeal Submitted Transfer Non-Start Appeal Denied Transfer Appeal Approved Non-Start Non-Start Non-Start Financial Assistance PRIOR TO THERAPY DURING THERAPY Dispense Fill 1 Dispense Fill 2 Dispense Fill 3 Transfer Complete OUT OF THERAPY Discontinue Patient Status Tracking
- 13. Trio Health 1. Physician Portal: • Value add service to drive referrals to high performing pharmacies • Analytical tools for each practice • National insight to empower physicians with payers 2. Specialty Pharmacy Portal: • Input portal to supplement nightly files • Analytical tools to track clinical programs • Analytical tools to measure revenue (lost, pending, actual) • Measure physician and payer burden 3. MFR Commercial Team: • Disease based insight throughout patient journey • Performance based metrics for each stakeholder: • (Payer, Drug, Physician and Pharmacy) • Patient advocacy and access to care Trio Platform Deliverables 13
- 14. Trio HealthTrio Health 14 Physician Portal: Comprehensive Data Source (Clinical + Pharmacy)
- 15. Trio Health Patient Case Management and Follow Up 15
- 16. Trio HealthTrio Health Copyright 2016 Trio Health 16 Patient Journey: Time to Fill, Dispensing, Non-Start, Discontinuation
- 17. Trio HealthTrio Health Copyright 2016 Trio Health 17 Compare Patient Cohorts
- 18. Hepatitis C Case Study
- 19. § Stakeholder Performance § Access to Care Efficacy Side Effects Duration Convenience Cost Old <2014 New >2014 50-55% Significant 24-48 weeks Injections Moderate 90-95% Minimal 8-12 weeks Oral Expensive § Affordability § Safety Current Issues Confronting Hep C Patients: 19 Hepatitis C Publication Strategy
- 20. Trio Health HCV Scientific Steering Committee ü Published hundreds of peer-review articles and manuscripts ü Participated in majority of pivotal HCV clinical trials ü Serve on national and international advisory boards for all companies with HCV programs 20
- 21. Federal And State NORD Disease Based KOLs SSC . Actionable DataUnique to each Disease. KPI’s Awareness: Global Publication Strategy for Physicians, Payers and MFRs 21 BOSTON SAN FRAN WASH DC SAN DIEGO HAWAII PHILADELPHIA SOUTH KOREA JAPANSAN FRAN WASH DCAUSTRIA SPAIN Trio Health
- 22. Trio Health LDV-SOF +/-RBV VKP +/-RBV SMV+SOF +/-RBV Total Outside Approved FDA Labeling 85% (115/135) 83% (5/6) 63% (5/8) 84% (125/149) Inside Approved FDA Labeling 95% (1391/1462) 93% (38/41) 82% (27/33) 95% (1456/1536) Total 94% (1506/1597) 91% (43/47) 78% (32/41) 94% (1581/1685) SVR12 Rates Inside Approved FDA Labeling vs. Outside Approved FDA Labeling *Patients prescribed outside approved FDA labeling: GT1a on VKP without RBV, tx failure cirrhotic patients on 12 weeks of VKP+/-RBV, LDV-SOF without RBV, or SMV+SOF+/-RBV Only six treatment options available. 9% of the patients were prescribed the wrong regimen that yielded a 11% decline in cure rates. Outside the label impacted overall performance by 100 basis points. 22 Physician Performance
- 23. Trio Health Total Scripts = 2,111 Harvoni Olysio + Sovaldi Viekira Pak Non-Starts 17% (n=369/2111) Harvoni 16% (297/1867) Olysio + Sovaldi 38% (26/68) Viekira Pak 26% (46/176) Pending >150 Days 5% (n=97/2111) Starts 78% (n=1645/2111) Harvoni 4% (69/1867) Olysio + Sovaldi 4% (3/68) Viekira Pak 14% (25/176) Harvoni 80% (1501/1867) Olysio + Sovaldi 57% (39/68) Viekira Pak 60% (105/176) Access to Care: Non-Start & Pending Patients 23
- 24. SVR12 Rates by Fibrosis 42/44 79/81 76/80 31/3252/53 143/147 194/203 144/151 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% 8 weeks 12 weeks 95% 98% 98% 97% 95% 96% 97% 95% F2 F3F1F0 Affordability: 30%-40% of the Hep C Patients can be treated with 8 weeks which is a 33% Price Reduction 24 Trio Health 24
- 25. Trio Health If the patient wins, clinical and financial success will be achieved by all patient stakeholders. 25
- 26. Thank You.