2018 International RARE Patient Advocacy Summit Preparing for the Road Ahead

Tuesday October 3, 2018 – Friday October 5, 2108 Agenda

Tuesday October 3, 2018 11:30 am – 5:00 pm CNF/GG Peer Support Group Program (pre-registration required) 3:00 pm – 7:00 pm General Registration Opens 3:30 pm – 5:30 pm Freshman Orientation (1 st time attendees only/pre-reg required) 5:30 pm – 6:00 pm Meet your Mentor 6:00 pm – 8:00 pm Foundation Alliance, Mentor, & Freshman Attendees Welcome

Reception Wednesday October 4, 2018 - Day One 6:00 am – 7:00 am Sunrise Yoga 6:30 am – 8:00 am Networking Breakfast / Partnering System Open 8:15 am – 9:15 am Welcome & Keynote, MSAB Presents

● Rachel Callendar - Rising Above: Using Empowering Language in Health Care

9:30 am – 10:30 am MSAB Presents ● MSAB Presents: Patients Driving Advances: From Research to

Therapy 10:30 am – 11:15 am Networking / Partnering System Open * Session titles in italics are subject to title change or change 11:30 am – 12:30 pm Track Sessions

Day One TRACK 1 -

TRACK 2 -

TRACK 3 –

TRACK 4

11:30 – 12:30

Living with a Life Altering Condition(Searching for a Diagnosis -> Adapting to the new norm -> Flourishing)

Architecting Your Disease

(Organizing -> Growing -> Sustaining and Adapting)

Patients as Partners and Drivers(Discovering -> Targeting -> Building on Advances)

Science and Technology Innovation

(New approaches for discovery -> Building on past modalities -> New approaches for therapeutics)

Looking for Answers in Uncertainty: Managing when there is no Diagnosis or Prognosis

Non-Profit 101: Launch and Governance of a Non-Profit

Data, Data, Data - Sharing and Owning Data to Game Change Rare Disease Research

RARE Research Resources: Animal Models and 3D Engineered Tissues

12:30 pm – 1:45 pm Networking Lunch / Partnering System Open 2:00 pm – 3:00 pm Track Sessions

TRACK 1

TRACK 2

TRACK 3

TRACK 4

2:00 – 3:00 l

3:15 pm – 4:00 pm Track Sessions

TRACK 1 TRACK 2

TRACK 3

TRACK 4

3:15 – 4:00

Grab and Go Break 4:00 pm – 4:15 pm 4:15 pm – 5:00 pm Track Sessions

TRACK 1 TRACK 2

TRACK 3

TRACK 4

4:15 – 5:00

5:15 pm – 5:30 pm Game Changing the World of Rare Disease through Collaboration: Global

Advocacy Code of Values 5:30 pm – 6:30 pm Stories of Success 6:45pm – 9:30pm Day 1 Reception

Understanding the Emotional Health of RARE Disease Patients

Capacity Building Strategies: Managing Volunteers

Building Valuable Registries and Natural History Studies

RARE Research Resources: CRISPR System and iPS Cells

Understanding the Emotional Health of RARE Disease Families

Sustaining an Organi-zation: Managing Long-term Growth and Development

The Role of a Chief Science Officer: The Secret Sauce for Forward Movement in Research and Scientific Collaborations

RARE Research Resources:Biomarkers

Update from the Community: Caregiver Survey

Update from the Community: Patient Focused Drug Development Meetings

Update from the Community: Legislative Update

Update from the Community: Quality of Life Markers

Thursday October 5, 2018 - Day Two 6:00 am – 7:00 am Sunrise Yoga 6:30 am – 8:00 am Networking Breakfast 8:15 am – 9:30 am Welcome, Main Session

Rare Disease Updates from the Global Stage: ● International Rare Disease Research Consortium ● Shire Global Commission ● RARE X Moonshot project ● Global Alliance For Genomics and Health

9:30 am – 4:00 pm ENTREPRENEURIAL READINESS BOOTCAMP – Invitation only, not on

general agenda * Session titles in italics are subject to title change or change 9:45 am – 10:45 am Track Sessions

Day 2

TRACK 1

TRACK 2

TRACK 3

TRACK 4

9:45 – 10:45

10:45 am – 11:30 pm Networking Break / Partnering System Open 11:30 am – 12:30 pm Track Sessions

TRACK 1 TRACK 2

TRACK 3

TRACK 4

11:30 – 12:30

Living with a Life-Altering Condition

(Diagnosis -> Adapting to the new norm -> Flourishing)(Diagnosis -> Adapting to the new norm -> Flourishing)

Architecting Your Disease Community

(Organizing -> Growing -> Sustaining and Adapting)

Patients as Partners and Drivers

(Discovering -> Targeting -> Building on Advances)

Science and Technology Innovation

(Building on past modalities -> New approaches for discovery -> New approaches for therapeutics

Setting Up Your Environment to Adapt to the New Normal

Organizing and Mobilizing Your Community of Stakeholders Regionally and Globally

We Can’t Do It Alone: Structure and Governance for Research Collaborations

RARE Therapies: Pharmacological Chaperones and Cell Therapies

Modern Therapies for Emotional Health: Different Ways to Cope

Clinical Care: Building Standards of Care for Your Disease Community

Is the Research Plan Working? Evaluation and Accountability for Research Grants and Contracts

RARE Therapies: Gene Therapy and Oligonucleotides

12:30 pm – 1:45 pm Networking Lunch / Partnering System Open 2:00 pm – 3:00 pm Track Sessions

TRACK 1 TRACK 2

TRACK 3

TRACK 4

2:00 – 3:00

3:15 pm – 4:00 pm Closing Session - Data and Technological Innovation 4:00 pm – 4:30 pm Closing and Rising Star/Celeb Preview of Awards Ceremony 4:30 pm – 5:00 pm Break 5:00 pm – 6:00 pm Champion of Hope Awards Reception 6:15 pm – 7:45 pm Champion of Hope Awards Presentation 8:00 pm – 10:00 pm Dinner and After Party Friday October 5, 2018 7:00 am – 9:00 am Breakfast 8:00 am – 12:00 noon Networking / Partnering System Open 8:00 am – 12:00 noon Corporate Alliance/CAB Annual Meeting (with MSAB) 11:30 am – 4:00 pm Global Advocacy Leadership Symposium (pre-registration required)

Relax and Refocus Trying Emotional Health Therapies

Patient Advocacy In and Out of Industry: What You Need to Know About Working Together

The Nuts and Bolts of Collaborations: Be Prepared for the Paperwork

RARE Therapies: Monoclonal Antibodies and Enzyme

Entrepreneurial Readiness Bootcamp Agenda Thursday October 4, 2018 9:30 am- 4:00 pm Overview: This first-annual Bootcamp has been designed for Patient Advocates who have started funding rare disease research and are looking to better coordinate and build structure around their efforts. There is limited space for this hands on and in depth day of learning. Patients interested in learning more or in attending will have the opportunity to apply. Space is limited, and attendees will be required to participate for the full day. Morning session – Why start a company? When is the time right?

1. Rare Disease – Unique Opportunities and Challenges 1. Possible speakers: Emil K, Phil R, Jeremy L, John Crowley, Brad Margus

2. Science – how do you know when you’re ready 1. Both a CSO and a parent who started a company to hear both

perspectives: 2. Speaker suggestions: CSO from BioMarin or other biotech. Matt

Wilsey. Mike ? (MiaMed founder sold to Amicus) 3. Multi-year Plan – milestones and value creation

1. Walt to lead 4. Capital – raising money over time

1. Possible speaker: Neil K Afternoon session – Making it happen: Tactical Modules

1. Building the team 1. Virtual versus brick and mortar 2. In house versus externalizing 3. Consultants, founders, SAB 4. Possible speaker: Andrea D (TRV)

2. Partnering – pharma/biotech: How and who you partner with when 1. Yael to lead

3. Corporate and legal – incorporating, ownership, and IP 1. Representative a firm we have worked with (from workshop last year)

RARE Global Advocacy Leadership Symposium Friday Oct 5, 2018 Overview: Global Genes envisions a world where rare disease regions are strong, collaborative and vibrant, equipped to address the growing challenges and needs facing them. The Symposium will bring together global advocacy leaders to 1. Help identify and Address regional needs 2. Problem-solve solutions to address these challenges together 3. Propose a Global Collaborative Support Alliance that will commit to sharing best practices and mentoring each other, with the intentions of helping build strong rare disease communities in all regions around the globe. 11:30 - 12:00 Welcome & Introductions (boxed lunch provided) Level Setting - Why we are here 12:00 - 12:45 Presentation of Needs Assessment Results** 12:45 - 1:00 Break 1:00 - 1:45 Problem Solving Regional Challenges 1:45 - 2:30 Education, Coordination and Connections driving

Activism - A Case Study of Success, Genetic Disorders UK, Caroline Harding (to be invited)

2:30 - 2:45 Break 3:00 - 3:45 Regional Breakouts - with moderators; Stephen Groft (to be

invited), Barbara Wuebbels (to be invited), 3:45 - 4:00 Meeting Wrap Up & Next Steps ** Pre-Meeting: Needs Assessment sent in advance