{"id":638,"date":"2020-05-12T17:42:41","date_gmt":"2020-05-12T17:42:41","guid":{"rendered":"https:\/\/lab.research.sickkids.ca\/enrich\/?page_id=638"},"modified":"2026-02-06T20:14:37","modified_gmt":"2026-02-06T20:14:37","slug":"inform-rare","status":"publish","type":"page","link":"https:\/\/lab.research.sickkids.ca\/enrich\/research-projects\/inform-rare\/","title":{"rendered":"SPOR INFORM RARE"},"content":{"rendered":"

\"INFORM<\/strong><\/span><\/p>\n

 <\/p>\n

Strategy for Patient-Oriented Research (SPOR) INFORM RARE: An innovative registry-based trials platform to improve clinical care, outcomes, and health policy for children with treatable rare diseases.<\/strong><\/span><\/p>\n

New treatments for children with rare genetic diseases are appearing at a rapid pace. Many of these treatments have been found to improve outcomes in highly selected groups of patients under very specific conditions.<\/span><\/p>\n

In order to know how best to recommend, use, and pay for these treatments, patients and their families, health care providers, and policymakers need more evidence about how the treatments work in all patients and settings. We also need to understand what additional supportive interventions are needed to get the best value from new rare disease drugs in order to improve functioning, quality of life, and other patient centered outcomes.<\/span><\/p>\n

In\u00a0 SPOR INFORM RARE<\/strong>, a group of patients, family members, health care providers, policymakers, methodologists, and ethicists will work together to develop innovative registry-based randomized trials in order to rapidly develop comparative evidence about the effectiveness of treatments for rare genetic diseases in children. These high-quality trials will make use of comprehensive patient registries to enroll patients and measure outcomes. SPOR INFORM RARE<\/strong> will design and implement three inaugural trials to address priority questions for three rare diseases:<\/span><\/p>\n