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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
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
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
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
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
7 Alone we are rare. Together we are strong.
NORD-Trio Partnership
9 Alone we are rare. Together we are strong.
Operational Quality Measures
Clinical Quality Measures
Access
Accountability
Awareness
Advocacy
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
10 Alone we are rare. Together we are strong. 10
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
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
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
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Trio HealthTrio Health Copyright 2016 Trio Health 16
Patient Journey: Time to Fill, Dispensing, Non-Start, Discontinuation
§ 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:
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Hepatitis C Publication Strategy
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
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Federal And State
NORD
Disease Based KOLs
SSC
. Actionable
Data Unique to each Disease.
KPI’s
Awareness: Global Publication Strategy for Physicians, Payers and MFRs
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BOSTON SAN FRAN
WASH DC SAN DIEGO
HAWAII PHILADELPHIA SOUTH KOREA
JAPAN SAN FRAN WASH DC AUSTRIA SPAIN
Trio Health
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.
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Physician Performance
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
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SVR12 Rates by Fibrosis
42/44 79/81 76/80 31/32 52/53 143/147 194/203 144/151 0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
8 weeks 12 weeks
95% 98% 98% 97% 95% 96% 97% 95%
F2 F3 F1 F0
Affordability: 30%-40% of the Hep C Patients can be treated with 8 weeks which is a 33% Price Reduction
24 Trio Health 24
Trio Health
If the patient wins, clinical and financial success will be achieved by all patient
stakeholders.
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