Changing Lives with Digital Patient Registry and Analytics Platforms

Pictured: the TRRF project in action with the eResearch@QUT team. Image courtesy of QUT.

The Trial Ready Registry Framework (TRRF) project co-funded by MTPConnect Project Fund Program and Queensland University of Technology (QUT), set out to develop a single digital trial ready registry platform to better collect and analyse health data.

The pilot aims to create a new standard of digital analytics platform to explore the feasibility with three rare diseases - Motor Neurone Disease (MND), Cystic Fibrosis (CF) and Angelman Syndrome (AS).

According to the Asia-Pacific Economic Cooperation (APEC)[1], rare diseases affect approximately 6-8% of the global population, which is an estimated 200 million individuals within 21 APEC economies alone. It is vital that health data and patient registries are set up to raise awareness and understanding of the conditions, provide valuable information to health professionals and service providers and to fast-track recruitment of patients, undertake clinical studies more easily and evaluate treatments more rapidly.

The TRRF is backed by a consortium of Australian research institutes and organisations, led by QUT, and includes Murdoch University, Telethon Kids Institute, and patient organisations: MNDi Foundation and Foundation for Angelman Syndrome and Therapeutics Australia. The partnership is enabling these registries to input patient and clinical data, as well as allowing patients themselves and clinicians to record observations and symptoms. In the case of Angelman Syndrome, this is a global digital platform for this genetic condition that causes developmental disabilities and nerve-related symptoms.

Queensland University of Technology's Director of eResearch, Professor Matthew Bellgard, said the implementation of digital patient-centric trial-ready registries will see a major improvement in treatment of patients, operating seamlessly across health jurisdictions.

"The development of a combined 'registry and analytics' platform will enable empowerment for patients to manage their condition, clinicians to rapidly make decisions, and data to drive a better understanding," Prof Bellgard explained.

The TRRF has been designed to embed statistical analysis of the data on a continuous basis to highlight any improvements of symptoms throughout these rare disease populations. By using statistical analysis to do this work, changes to treatment can be determined, and this creates health improvements for individuals as well as improving care of the specific rare disease population as a whole.  

Chair of the Foundation for Angelman Syndrome Meagan Cross, whose daughter Molly was diagnosed with AS in 2008 just after her first birthday, said there are currently about 1,400 people from 64 countries on QUT's TRRF AS registry.

"We believe people will find the TRRF platform more user-friendly and we are now able to not only collect data but use it to help improve outcomes for people living with AS, and advance clinical trials and research towards a cure," Ms Cross added.

In February 2020, when the Novel Coronavirus (COVID-19) began to emerge in Australia, Professor Bellgard and his co-Chief investigators - Professors Nik Zeps and James McGree (QUT), Professor Tom Snelling and Tim Shaw (University of Sydney) and Professor Ann Nicholson (Monash University) - immediately began to repurpose TRRF as a core digital platform to support COVID-19 patients.

The platform is pivoting to include a real-time point of care decision support focus, re-branded as the Clinical Data and Analytics Platform (CDAP) and a new team was formed within weeks.

The CDAP team is now supported through a $4 million Digital Health CRC project with partners QLD Health, NSW Health, and Commonwealth Health.

Professor Bellgard said it's great to secure the support of partners around Australia to adapt the registry to respond to COVID-19.

"The design and functionality of the TRRF to securely enable electronic informed consent, as well as share, aggregate and analyse data across jurisdictions lends itself elegantly to COVID-19 as well as a number of other clinical care settings," Prof Bellgard added.

The team at QUT concludes its MTPConnect project in November 2020, at which time the consortium expects to release the results of the parallel pilots.

For more information, visit QUT.

[1]  APEC Rare Disease Action Plan Sets Precedent to Integrate Rare Disease Care by 2025