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

ODA Current Day Progress

Alone we are rare. Together we are strong.® 4

That’s 1 in 10 people

30 Million Americans Are Impacted By Rare Disease

5

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.

How Can We Ensure Adequate Access is Provided?

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

13

Trio HealthTrio Health 14

Physician Portal: Comprehensive Data Source (Clinical + Pharmacy)

Trio Health

Patient Case Management and Follow Up

15

Trio HealthTrio Health Copyright 2016 Trio Health 16

Patient Journey: Time to Fill, Dispensing, Non-Start, Discontinuation

Trio HealthTrio Health Copyright 2016 Trio Health 17

Compare Patient Cohorts

Hepatitis C Case Study

§  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

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

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

21

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.

22

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

23

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.

25

Thank You.