DAY 1: DELIVERING RARE ALLIANCE CANADA

Objectives:

Mobilize all stakeholders around common vision for Rare Alliance Canada Develop consensus on a “best-fit” model for Canada’s rare disease network Create a guiding framework to map current resources, define gaps, identify opportunities and challenges, and prioritize needs Articulate next steps; gain commitment to action; define success factors, and outcome measures

What is Rare Alliance Canada?

We are patient and family driven

We bring together all stakeholders, all disciplines, all activities, all services We work toward rare disease sites linked from coast to coast We envision centres of excellence empowering GPs, therapists, teachers, and support workers to effective serve rare disease patients and families in their own community We will enhance Canada’s reputation as a leader in rare diseases

DAY 2: DELIVERING THERAPIES FOR LIFE

Objectives:

Share vision of possibilities for health with innovative therapies of today and tomorrow

Challenge all stakeholders to identify and “go beyond” barriers to providing access to old and new therapies for life

Develop consensus on a “best-fit” framework for “life-long” access to innovative therapies

Articulate next steps; gain commitment to action; define success factors, and outcome measures

What are Principles of Canada’s Therapies for Life Program?

Principles: patient-driven, comprehensive, responsive, responsible access to therapies for unmet needs

Innovative: Canada is favourable environment for R&D, clinical trials, timely access, patient support

Patient-driven: Patients at all levels from policy, program development, criteria for specific drug access, patient participation in registration, monitoring, and data collection, reassessment of drug place in therapy

Priced Right: Canadian pricing and financing (non-excessive for payers, incentive for further and affordable access for patients)

DELIVERING INNOVATION: A MOON SHOT FOR RARE DISORDERS -

AND BEYOND

“Mission Possible: Delivering Innovation to All Patients”

November 6 -7, 2017

Hyatt Regency Toronto

CONFERENCE AGENDA DAY 1: DELIVERING RARE ALLIANCE CANADA

8:00 am Registration and Breakfast: Regency Ballroom

8:30 am Conference Opening Durhane Wong-Rieger, Canadian Organization for Rare Disorders Invited Address Neeta Sarta, Director, Provincial Programs Branch, Ministry of Health and Long Term Care Keynote Address Eric Tse, Shire Canada Patient Matters Peter Dorfman, CLL PAG; Gail Wright, Canadian PBC Society; Patti Bryant, CORD/Dravet Canada

9:00 am Session 1

Chair: Durhane Wong-Rieger, CORD

Approaches to Developing Rare Disease Networks Panel: Overview of origin and vision (why and so what) (5 min)

• Vancouver Vision: Opportunities and Challenges in Canadian Landscape - Durhane Wong-Rieger, CORD

• Children’s National Rare Disease Institute - Marshall Summer, Children’s National Health System; Pam Gavin, NORD

• Ontario Rare Disease Framework - Ronald Cohn, The Hospital for Sick Children • European Reference Networks - Louise Clément, Health Standards

Organization Panel discussion on strengths, risks, and outcome

• What is the intended impact? • What will it take to make it work? • What are indicators working or not working?

Audience discussion on relevance for Canada • What are the relative merits of each model, and how what do these suggest

for “Rare Alliance Canada?” • What would a “best of” look like for Canada?

10:30 am Networking Break

10:45 am Session 2

Chairs: Alex MacKenzie, CHEO; Etienne Richer, CIHR

Rare Gems in Canada’s Research Landscape

Panel 1: Research and Clinical Expertise (30 min)

• CIHR RD Emerging Team Grants and Contributions to Canadian Excellence, Paul Lasko, CIHR Institute of Genetics

• Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay - Bernard Brais, McGill University

• The FACTs Project: Fabry Disease Clinical Research and Therapeutics - Tony Rupar, Western University

• Developing Effective Policies for Managing Technologies for Rare Diseases – Devidas Menon, University of Alberta

• Canadian Framework for Evaluation and Decision-Making for EDRD - Larry Lynd, University of British Columbia

Panel 2: Research Improving Care and Treatment (30 min) • Diagnosis to treatments - Kym Boycott, CHEO • Scleroderma Patient-centered Intervention Network - Brett Thombs, Jewish

General Hospital • Achieving the "triple aim" for inborn errors of Metabolism – Pranesh

Chakraborty, University of Ottawa Discussion: Contributions to Rare Alliance Canada Network (30 min)

12:15 pm Networking Lunch

1:15 pm Session 3

Chair: Durhane Wong-Rieger, CORD

Rare Disease Research and Clinical Centres of Excellence/Networks Panel 1: Disease Specific Networks

• Cystic Fibrosis - John Wallenburg, Cystic Fibrosis Canada • Red Blood Cell Clinic – Richard Ward, University Health Network • Dravet Syndrome - David Dyment, CHEO • Ehlers-Danlos Clinic - Roberto Mendozo-Londono, Toronto General Hospital • Gaucher’s Disease Network - Dominick Amato, Mt. Sinai Hospital

Panel 2: Rare Disease Clusters • Neuromuscular - Alex MacKenzie, CHEO • Metabolic networks - Andreas Schulze, The Hospital for Sick Children • Rare Cancers - Jim Whitlock, The Hospital for Sick Children

2:45 pm Networking Break

3:00 pm Session 4

Chair: Alex MacKenzie, CHEO

Designing Rare Alliance Canada

Panel 3: Multiple Disease Networks • MYCRN - Stephen Barbazuk, BC Children’s Hospital • Genomics - Monica Justice - The Hospital for Sick Children • Community-based RD Networks - Shikha Mittoo, Mount Sinai Hospital

Panel 4: Opportunities Created

• Learning from Canada’s Rare Disease Centres - Michael Gottlieb, Sanofi Genzyme

• RareConnect / PhenomeCentral / MatchMaker Exchange - Michael Brudno, University of Toronto; Orion Buske, The Hospital for Sick Children; Dorin Manase, The Hospital for Sick Children

Design Panel: Kym Boycott, CHEO; Louise Clément, HSO; Stephen Barbazuk, BC Children’s Hospital; Paul Lasko, CIHR; Ian Stedman, CORD; Michael Gottlieb, Sanofi Genzyme

• Essential features • Criteria, quality, standards, guidelines • Outreach, education, training

4:30 pm Plenary

Coming Together for Next Steps

DAY 2: INNOVATIVE APPROACHES: DELIVERING THERAPIES FOR LIFE

8:00 am Registration and Breakfast Regency Ballroom

8:30 am Session 1

How Patients Drive Access

• Parent and Patient Advocacy - Barb Kulyk and Jordan Janz, Cystinosis • Engaging with the System - Susi Vander Wyk, CURE SMA Canada • Driving the System - Pat Furlong, Parent Project Muscular Dystrophy

9:00 am Day 1 Recap

Introduction to Panel Debates

Durhane Wong-Rieger, CORD

9:15 am Session 2

Chair: Bill Dempster, 3Sixty Public Affairs

How Will Pharmacare Assure Timely Access to Innovative Therapies? Opening Addresses to Set the Stage

• What are Canadian pathways for access to innovative therapies, and how well are they working? Sandra Anderson, Innomar Strategies

• What are the BIG innovative therapies happening now and in the near future? What are expectations and challenges for next generation therapies? Chander Sehgal, Innovative Medicines Canada

• What are opportunities and challenges experienced by pharmaceutical companies bringing innovative therapies to Canada, including next generation cellular therapies? Helen Trifonopoulos, Novartis

• What are private insurers doing to assure timely and sustainable access to innovative therapies? Ara Kolandjian, Manulife

Debate Panel: How will a national Pharmacare program assure access to innovative therapies?

Introduction to CAR-T Cell Therapy: Jim Whitlock, The Hospital for Sick Children

Panelists: Cheryl Greenberg, University of Manitoba; Jim Whitlock, The Hospital for Sick Children; Lisa Machado, Canadian CML Network; Sandra Anderson, Innomar Strategies, Ara Kolandjian, Manulife, Helen Trifonopoulos, Novartis

1. Resolution: A single-payer model (pan-Canadian, national, federal) would improve access to innovative therapies.

2. Resolution: Canadians would have better access if we paid the lowest drug prices in the world.

3. Resolution: Canada should treat innovative therapies like an investment, not an expense.

10:45 am Networking Break

11:00 am Session 3

Chair: Wayne Critchley, Global Public Affairs/CORD

Does Canada Need an Orphan Drug Regulatory and Access Framework?

Opening Addresses to Set the Stage

• What are incentives for industry under Health Canada’s Orphan Drug Roadmap? Cathy Parker, Health Canada

• What are challenges for industry to bring rare disease drugs to Canada? Lisa Chartrand, Roche Canada

• In the absence of a panCanadian “Expensive Drugs for Rare Diseases Program, are RD drugs been fairly assessed and reviewed? Suzanne McGurn, Ontario Public Drug Program

Debate Panel: How will a “made-in-Canada” Orphan Drug Regulatory and Access Framework serve all Canadians?

Panelists: John Moore, CORD; Cathy Parker, Health Canada; Tania Stafinski, University of Alberta; Larry Lynd, UBC; Karen Voin, CLHIA; Peter Brenders, Sanofi Genzyme; Suzanne McGurn, Ontario PDP, pCPA

1. Resolution: Most drugs designated as orphan are accessible to Canadian patients without specific OD regulations.

2. Resolution: Canada would benefit from a nationally administered and funded program for rare disease drugs.

3. Resolution: Publicly funded drug programs are designed to serve population needs, not individual needs.

Case Study Application - Spinraza

12:30 pm Networking Lunch

1:30 pm Session 4

Chair: Bill Dempster, 3Sixty Public Affairs

Will Biosimilars Improve Access to Innovative Therapies?

Opening Addresses to Set the Stage

• Will CADTH’s proposed streamlined approach to review of biosimilars serve the needs of all stakeholders? Helen Mai, pCODR

• What is the current evidence base for biosimilars use, including switching? David Zante, Janssen Canada

• What is the current status of clinical use of biosimilars; globally and locally? Jennifer Chan, Merck Canada

• Biosimilar policy issues: A patient advocate’s perspective Andrew Spiegel, Global Colon Cancer Association

Debate Panel: Is the Canadian healthcare system ready for biosimilars?

Panelists: Allan Miranda, Janssen; Jennifer Chan, Merck Canada; Ned Pojskic, Greenshield; Karen Voin, CLHIA; Mina Mawani, Crohn’s and Colitis Canada, Helen Mai, pCODR

1. Resolution: With 10+ years of experience in Europe, switching to a biosimilars is no longer an issue.

2. Resolution: Preferential listing for biosimilars is good for the healthcare system and good for patients.

3. Resolution: Patient preference should be the deciding criterion on use of original biologic or biosimilar.

Case Study Application - Infliximab

3:00 pm Plenary

Concluding Arguments/Next Steps

The Canadian Organization for Rare Disorders acknowledges the contribution of all our Corporate Partners to improving the lives of patients and families with rare disorders. We are especially grateful to the following that have supported this conference.