MICYRN is a Canadian non-profit organization established in 2011 to reduce barriers and duplication of effort in multi-jurisdictional pediatric research, particularly for rare diseases. Rare diseases affect over 2.7 million Canadians but each affects fewer than 2000 individuals. MICYRN works to improve diagnosis of rare diseases and develop new therapies through national research networks and collaborating in international research initiatives. MICYRN also supports patient registries and clinical trials infrastructure to study rare diseases and determine drug safety and effectiveness in children through its research informatics platform and partnerships with health organizations and industry.

1. Safe and Effective Medicine for Children
Daring to be Rare, BIOTECanada Panel
2015 CADTH Symposium
Saskatoon, April 12-14

2. MICYRN
• Federal non-profit society (2011)
• Joins 20 maternal – child health research institutions and disease
networks
• Collaboration on research infrastructure (ethics review, data
management…) to reduce duplication of effort and harmonize processes
• Prime focus to reduce barriers to multi-jurisdictional research (national,
international)
• Focus on rare diseases

3. Why a focus on Rare Diseases?
• Affect < 1/2000 individuals, 1/12 = 2.7 M Canadians
• Around 7,000 disorders
• 80% genetic origin
• 75% present in childhood

4. Global Objectives 2020:
 diagnosis of most rare diseases
 200 new therapies
New therapies

5. MICYRN & Rare Diseases
1. Diagnosis:
- Advisory to Orpha-net
- Rare Diseases Models & Mechanisms Network
- Patient Registries/Harmonization
2. Therapies:
- National plan for pediatric clinical trials research infrastructure
(KIDSCAN)
- Collaboration in Industry-instigated PCTRI and EU Research
Infrastructure 2020 Roadmap initiatives

6. www.orpha.net

7. RD Models and Mechanisms Network
GeneX
$25K
$2.3 M Catalyst Grant

8. RDMM

9. Understanding Pathogenesis: Patient Registries
Health Canada
Industry
Investigator trials
MOH-HTA
Patients
Foundations
Care providers
Numbers: incidence, prevalence
Natural history
Patient outcome measures
Surrogate measures pre-debility
Stratification of disease
Post authorization monitoring
beyond safety
Adaptive authorization
MICYRN Clinical Research Informatics platform supports Registries;
International harmonization projects between rare disease registries

10. Clinical trials: methodological Challenges
- Small populations, original methods
- Rare disease, recruitment at multiple sites
- Identifying age –appropriate clinical outcomes
- Age –appropriate measurements
- QOL questionnaires and validation
- Age –appropriate interventions
- Doses , routes…..
- Blood draws in pediatric patients/ invasive tests
- When “2 table spoons” are already too much
- “Will it hurt?”
- Inclusion in multiple trials
- Complex regulation, multiple REB, contracts,..

11. • Children take medications, many of which have not been proven
safe and effective for their use.
• Children respond to medications differently from adults; thus,
medicines must be studied in children and formulated for
children.
• Studying medicines in children is always possible and in their
best interests.
• In the United States and the European Union, pediatric
medicines research is encouraged, required and monitored in
ways that offer lessons for Canada.
• Pediatric medicines research is a Canadian strength, but it
requires reinforcement and sustained capacity and infrastructure
to realize its full potential.
Council of Canadian Academies report, Sept 2014

13. KIDSCAN: a Proposal to develop a Canadian
Coordinating and Advisory Network and
Infrastructure to Ensure Best Therapies for Children

14. • A national infrastructure coordinating and enabling pediatric clinical trials.
• New clinical trial methodologies, developed with patients and other
stakeholders, will provide regulators and reimbursors with the best
evidence to determine drug authorization and access.
• Coordinated development of registries of patient populations with sharing of
harmonized phenotype and genotype data both nationally and
internationally, will capture the natural history of disorders, establish
markers of disease and disability, and determine the impact of
interventions.