Safe and Effective Medicine for Children

Daring to be Rare, BIOTECanada Panel

2015 CADTH Symposium

Saskatoon, April 12-14

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

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

Global Objectives 2020: diagnosis of most rare diseases 200 new therapies

New therapies

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

www.orpha.net

*

RD Models and Mechanisms Network

GeneX

$25K

$2.3 M Catalyst Grant

RDMM

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

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,..

• 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

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

• 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.