What are Clinical Outcome Measures?

Clinical outcome measures or clinical outcome assessments are measures that describe or reflect how a patient feels, functions, or survives, as described by the FDA. The clinical outcome measures included on this website are performance outcome measures which assess how a participant functions.

What are normative reference data and why are they important?

Normative reference values or norms are data collected for outcome measures from a pre-determined ‘normal’ population. Normative reference values are an integral part of clinical practice and are one of the most powerful tools in healthcare to aid the clinical decision-making process. Normative reference values provide information that assist healthcare professionals to diagnose pathology, evaluate change in response to an intervention and accurately describe the natural history of clinical conditions. They are also vital to the development of new outcome measures with enhanced precision and responsiveness to be utilised in clinical trials. Individuals in a normative reference sample have met clearly defined criteria, for example that establish them as normal or healthy.

Knowledge of what is normal is essential in order to identify and subsequently manage abnormal presentations or pathologies.

Who developed the Clinical Outcome Measures on this website?

The CMTPedS, CMTInfS and CMTNS were developed by the Inherited Neuropathy Consortium (INC) to address the need for clinical outcome measures of disease severity across the lifespan for people with Charcot-Marie-Tooth Disease.

The 1000 norms calculator was developed by Professor Joshua Burns, Dr Marnee McKay and Dr Jennifer Baldwin at the University of Sydney who set out to develop a comprehensive normative reference data set of established clinical outcome measures from one thousand healthy Australians aged 3-101 years.

Who are the INC?

The Inherited Neuropathy Consortium (INC) is an integrated group of academic medical centers, patient support organizations, and clinical research resources dedicated to conducting clinical research in different forms of CMT and improving the care of patients. Funded by the National Institutes of Health (NIH), the INC is part of the Rare Diseases Clinical Research Network. For more information on the INC