Patient-reported outcomes assessment to support accelerated access to advanced cell and gene therapies: PROmics


"Cell or gene therapies show great potential in treating patients with conditions that cannot be cured with current treatments. These include arthritis, liver disease, several types of cancer, and diabetic ulcers. It is important to assess the impact of these new therapies on patient quality of life and symptoms, both at the point of receiving therapy and over the longer-term once out of hospital.

The project will develop an electronic capture system to assess patient symptoms and quality of life when they receive these new therapies. Patients will be able to report how they feel on the treatment by using electronic devices. Any worrying symptoms will be flagged to the clinical team to help promote patient safety. These data will also be used to assess the effectiveness of treatment and be used as an evidence base for regulators and policy makers to support uptake in the NHS. Patients have been directly involved in development of the project and will provide direct input into the system development to ensure that it meets their needs.

The Datatrial Nucleus database platform being rolled out across the Advanced Therapy Treatment Centres in the UK will collect data from multiple sources and will be important in allowing assessments of cost-effectiveness of these therapies.

The project lead is the Midlands & Wales Advanced Therapy Treatment Centre (MW-ATTC) - a national centre of excellence in partnership with the Centre for Patient Reported Outcomes Research at The University of Birmingham, health technology companies Dignio and Datatrial. The project draws on expertise from stakeholders in the ATTC network, London's network of Advanced Therapy Centres (ATCs), the pharmaceutical industry including ATMP developers and producers such as Chiesi and Autolus, the Cell and Gene Therapy Catapult, UK regulators and payers."


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