PATIENT GROUPS & CLINICAL TRIALS CASE STUDY

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1 February 1, 2017 PATIENT GROUPS & CLINICAL TRIALS CASE STUDY Jeffrey W. Sherman, M.D., FACP Past President Inaugural Fellow Chief Medical Officer Executive Vice President

2 Disclaimer The views and opinions expressed in this presentation are those of the individual presenter and do not necessarily reflect the views of their employer, professional association affiliation, or Clinical Trials Transformation Initiative. 2

3 Why Adopt CTTI Recommendations? Expedite development of potential products for unmet medical needs Over 7000 rare diseases of which only 5% have treatments. Patient groups have the knowledge of the disease to expedite clinical trials Best to start engagement as early as possible Facilitate planning and implementation of development activities Continue engagement throughout the R&D continuum Greatly increases chances for success (new treatment) Saves time, money, and resources - ROE 3

4 Patient Groups & Clinical Trials Questions to be Answered How did you operationalize engagement? What were challenges? How did you manage the challenges? What were specific successes? How are you applying your learning from this experience to other engagement opportunities? Were there things that didn t work well that you won t try to replicate, or modify a future approach? Key Consideration Patients Come First 4

5 Drug Information Association - Fostering Innovation to Improve Health and Well-Being Worldwide Bringing health care product development professionals together in a trusted, global, neutral environment to share insights and drive action in health care product development and life cycle management Academia Government Regulators Industry Patient Groups Payers Over a 50-year history of driving collaboration worldwide as a non-profit professional association 5

6 Horizon Pharma Focused Commitment to Community Four Pillars Therapeutic Areas STEM/Education Innovation Children s Healthcare Company Confidential Not for Distribution

7 7

8 Pharmaceutical Interferon Products Interferon (IFNs) Proteins made and released by host cells in an immune response to the presence of pathogens such as viruses, bacteria, parasites, or tumor cells. Treatment targets appear to not overlap Hepatitis C (soon to be historical use as non-interferon agents continue to emerge) Interferon alpha 2a - Roferon A Pegylated Interferon alpha 2a - Pegasys Interferon alpha 2b - Intron A Pegylated Interferon alpha 2b - PegIntron Multiple Sclerosis Interferon beta 1a - Rebif (liquid); Avonex (lyophilized) Interferon beta 1b - Betaseron Chronic Granulomatous Disease (CGD); Severe Malignant Osteopetrosis (SMO) Interferon gamma 1b ACTIMMUNE 8

9 9

10 ACTIMMUNE (Interferon gamma-1b) Orphan Diseases Modulating Genetic Deficiencies Chronic Granulomatous Disease (CGD) Severe Malignant Osteopetrosis (SMO) Friedreich s ataxia (FA) 10

11 11

12 Why FARA? Preclinical Models of Disease 12

13 Why FARA? Clinical Trial Infrastructure 13

14 FARA and Horizon Embracing Best Practices in Effective Engagement 14

15 ACTIMMUNE Phase 3 FA Clinical Study FARA and Horizon Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy, Safety, and Pharmacokinetic Study of ACTIMMUNE (interferon γ-1b) in Children and Young Adults with Friedreich s Ataxia Short title: Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich s Ataxia Study 15 Clinicaltrials.gov: NCT

16 Patient Groups & Clinical Trials Questions Answered Patients Come First How did you operationalize engagement? Follow best practices Make engagement a priority What were challenges? Minimize challenges by building trust upfront for a long-term relationship How did you manage the challenges? Have frequent interactions particularly early on What were specific successes? Completed enrollment in a Phase 3 trial on time and with no screen failures How are you applying your learning from this experience to other engagement opportunities? Apply the same success factors as above Were there things that didn t work well that you won t try to replicate, or modify a future approach? Important again to build trust upfront 16

17 Industry & Patient Group Interactions: Participate, Listen, and Learn RideAtaxia Illinois RideAtaxia Atlanta NORD Rare Impact Awards

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