NRBDO Access to Innovative and Curative Therapies Virtual Conference: Nov 5 – 7, 2020

The NRBDO is a pan-Canadian coalition of patient organizations representing people with rare blood disorders and/or people with a chronic condition who are recipients of blood or blood products or their alternatives. Together we work to educate and advocate for Canadians affected by rare blood disorders.

Many innovative and curative therapies are coming to market for rare blood disorders, but will they be available to Canadian patients?

What can patient groups do to ensure access to innovative and curative therapies?

These questions will be explored over two days of interactive and engaging sessions. 

Your fellow conference participants include patients and patient organization leaders, healthcare providers, industry professionals, and policymakers.

Conference RSVP link

The agenda is available below and can also be downloaded as a PDF.


November 5: Innovative Therapies (12pm-3pm ET)
12:00 – Welcome, Introductions and Land Acknowledgement
1. Erin Harder, Chair, NRBDO
2. Norm Berberich, National Director, External Affairs and Patient Advocacy, Takeda Canada
12:10 – Keynote: A New Era of Personalized Healthcare
Michael Duong, Director of Personalized Healthcare, Roche Canada 
12:30 – Session 1: Introduction to novel therapy types: Gene therapies, RNAis, Monoclonal antibodies
Research Perspectives: 
1. Dr. Kevin Kuo, UHN – Gene Therapy 
2. Dr. David Lillicrap – RNAis and Monoclonal Antibodies 
Patient Perspectives: 
1. Biba Tinga, Sickle Cell Disease Association of Canada (SCDAC)
2. Leanne Owen, Canadian Hemophilia Society  (CHS)
3. Erin Morrison, Aplastic Anemia & Myelodysplasia Association of Canada (AAMAC)
Discussion: How might these novel therapies impact the burden of illness and associated direct and indirect costs? 
1:30 – Break (Lobby Chat Room available)
1:50 – Session 2: Groundbreaking trials, domestic and international 
Clinical/Regulatory Perspectives: 
1. Dr. Celia Lourenco, Director General, Biologic and Radiopharmaceutical Drugs Directorate, Health Canada 
2. Dr. Luis Murguia Favela, Alberta Children’s Hospital 
Patient Perspectives: 
1. Haley and Ian Cowie, CIPO 
2. Andrea Fernandez de Soto Abdul Rahim and Kamil Guziak, CIPO
3. Ali Oonwala, Thalassemia Foundation of Canada
Discussion: What is the role of clinical trials in access to innovative therapies? How do they allow us to gain access, and what happens to our access once the trial ends? Are Canadian Centres of Excellence sufficiently represented in clinical trials for groundbreaking therapies? If not, why not?
2:50 – Day Wrap Up 
3:00 – Adjourn for the day
November 6: Innovative Access (12pm-3pm ET)
12:00 – Welcome back and recap 
Erin Harder, Chair, NRBDO
12:10 – Keynote: Accessing innovative and curative therapies
Fred Horne, Public Advisor, 3Sixty Public Affairs  
12:30 – Session 3: What needs to happen for innovative and curative therapies to be accessible to Canadian patients? 
Reimbursement Perspectives: 
1. Brent Fraser, Vice President, Pharmaceutical Reviews, CADTH 
2. Stephanie Stypa, Senior Manager, Market Access, Takeda Canada
3. Wayne Critchley, former Executive Director of the PMPRB
Patient Perspectives: 
1. Yhulan Walters, Answering TTP
2. David Page, National Director of  Health Policy, Canadian Hemophilia Society
Discussion: Does the patient perspective currently receive the weight it deserves in new product reviews? How can HTA bodies and patient organizations work together to ensure more genuine engagement?
1:30 – Break (Chat Room available)
1:50 – Session 4: What needs to happen for innovative and curative therapies to be accessible to Canadian patients?
Supply and Distribution Perspectives: 
1. Sylvain Grenier, Director, Plasma Protein Products Formulary Program, Canadian Blood Services
2. Rima Khalil, Héma-Québec?
Patient Perspectives:    
1. Angela Diano, Executive Director, Alpha-1 
2. Donna Hartlen, Executive Director, GBS/CIDP 
3. Whitney Goulstone, Executive Director, CIPO
Discussion: What role can patient groups play in realizing these changes discussed today?
2:50 – Day Wrap Up 
3:00 – Conference Adjourned