Journal article

Rare disease registries: a call to action

Paul Lacaze, Nicole Millis, Megan Fookes, Yvonne Zurynski, Adam Jaffe, Matthew Bellgard, Ingrid Winship, John McNeil, Alan H Bittles

Internal Medicine Journal | WILEY | Published : 2017

Abstract

When registries collect accurate clinical data over time, they can act as fundamental support structures for patients and their families and powerful cost-effective instruments to support clinical trials and translational research to improve quality of care, quality of life and survival. Registries are critical for rare diseases (RD) with low prevalence and propensity for variation in treatment and outcomes. Rare Voices Australia is leading a call for action to the research and clinical community to prioritise RD data collection and develop an integrated RD Registry strategy for Australia. Financial, operational and governance challenges exist for establishing and maintaining RD registries. ..

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