“2nd World Congress on Rare Diseases and Orphan Drugs” Management of Rare Diseases: An Integrated Approach to Break Down Barriers and Facilitate Patient Access to Healthcare “2nd World Congress on Rare Diseases and Orphan Drugs” Date: 29th and 30th June, 2017

“I am Dr. Gayathri Balasubramanian, a part of focus scientific research center, the scientific arm of phamax, which is a healthcare market access company. The views presented here are from secondary desk research and various initiatives undertaken by phamax at a global level.” Disclosure

Rare diseases – A snapshot 350 M 80% 50% 35%

Now a global issue Developed Countries Developing Countries More than 6,000 rare diseases Rare disease affects < 1/500,000 More than 7,000 rare diseases Affects ~30 M European Union citizens Neonatal morbidity of < 1/10,000 Rare disease affects < 0.1% of the population ~ 70 M Indians are affected

Unaffordability Untreated cases Underdiagnoses Undefined process Unavailing research Unawareness Unavailing research Undefined process Underdiagnoses Untreated cases Unaffordability Unawareness

Stakeholders’ Network Journey so far Initiatives Plans and Policies Data/Registry Access Programs Cost of Care Centers of Excellence Stakeholders’ Network Comprehensive - Care

Recommendations Process Collaboration Awareness Regulations Guidelines Policy Makers/Regulatory Bodies Healthcare Providers Process Collaboration Awareness Regulations Guidelines Policies Continued education Training Clinical guidelines Centers of excellence Comprehensive care Industry Partners/Researchers Patients/Patient Organization Continued research Drug development Mixed-methods research Awareness Advocacy Pathway establishment

Integrated Approach Drivers and Barriers Technology Novel Research Stakeholders

Implementation Engage Develop Build Generate Collaborate Identify Disseminate Facilitate

Lessons learnt from our experience Tackling hepatitis in Africa – A Case study REACH Problem 01 Objective 02 Epidemiology Awareness Collaboration Health access Research Continued Approach 03 Outcomes 04

Global network of ~2,000 stakeholders of health; both international and local experts Network Composition: Physicians: 68% Policy: 23% Patient bodies: 9%

Thank you