CIHR – Team Grant: Pediatric Rare Disease Clinical Network – EOI

Well-being, Health and Biomedical Discovery

Deadlines

Academic Unit: Inquire within unit

Memorial Deadline: RIS review not required for EOI

External Deadline: Tuesday 27th, June 2023

Description

Please note: To be eligible for application an EOI must be completed.

RIS will be offering support with application development for this opportunity. Prospective applicants are encouraged to contact Dr. Jennifer Stevens (v5js@mun.ca) or Dr. Amanda Crompton (ajcrompt@mun.ca) early during the development process to discuss the services available to them.

Rare Diseases (RDs) constitute an under-recognized global health priority. In fact, notwithstanding their name, RDs are far from rare. The population prevalence of RDs is reported at 3.5-6.0% and affect as many as 446 million people globally^Footnote1. By extrapolating data generated in studies conducted in other countries to Canada, we can estimate that most pediatric hospital beds are occupied by rare diseases patients, that a minimum of 5% to 10% of all hospitalization costs in Canada are tied to rare diseases^Footnote2, that 563,000 to 2,331,000 Canadians are living with rare diseases^Footnote1 and that, on an annual basis, 14,000 Canadian kids under 15 die from a rare disease^Footnote3. Taken collectively, these numbers would make “rare disease land” the world’s third most populous country.

Using current clinical diagnostic pathways, a RD takes an average of five years to diagnose, with 2-3 misdiagnoses prior to the correct diagnosis. This diagnostic odyssey, along with social isolation, an absence of care coordination and attendant socio-economic and mental health burdens are too often the norm for those living with RDs. Compounding the situation is the fact that more than two thirds of RDs have a pediatric onset^Footnote1,^Footnote2,^Footnote4

There is an increased awareness and recognition of the profound morbidity and mortality caused by RDs ^Footnote5. In response to the burden posed by RDs, as well as the fundamental inequities faced by those affected, the UN High Commissioner for Human Rights^Footnote6 has designated RDs as a priority for the conceptualization and implementation of Universal Health Coverage. There are medical, economic and human rights imperatives to address these issues.

Purpose

This investment will catalyze the creation of a Pediatric Rare Disease Clinical Network to streamline rare diseases clinical research across Canada and to facilitate the collaboration among researchers across pillars, people with lived and living experience (e.g., patients, family, caregivers), health care providers, industry, policy and regulatory bodies. This Network will maximize synergies to advance discovery across the rare disease continuum from prevention to diagnosis through to treatment and survivorship to improve health outcomes for children and adolescents affected by rare diseases. The aim is to support national and international clinical trials to rapidly advance discoveries to support rare diseases patients and their families across Canada. It will maximize the impact of the Government of Canada’s efforts deployed in the regulatory modernization for health products, improve Canada’s standing globally, and attract industry dollars for future investments and sustainability.

Important Dates

More details can be found on ResearchNet.

Funding Sources

Canadian Institute of Health Research (CIHR)