1. “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

2. “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

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

4. 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

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

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

7. 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

8. Integrated Approach Drivers and Barriers Technology Novel Research
Stakeholders

9. Implementation Engage Develop Build Generate Collaborate Identify
Disseminate
Facilitate

10. 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

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

12. Thank you