Past research and projects
We are actively involved in a range of methodological research and other projects.
ADAPT-SMART
ADAPT-SMART aimed to design new collaborative approaches to the development of medicines, known as Medicines Adaptive Pathways to Patients (MAPPs).
We've played a leading role in this and other projects, and we now engage with pharmaceutical companies via our Office for Market Access and the Accelerated Access Collaborative.
DO->IT (Big Data for Better Outcomes)
The DO->ITproject was launched to provide a coordination platform for the BD4BO programme, exploiting synergies across the projects and maximising its impact on healthcare systems. It aimed to:
aggregate learnings and disseminate findings from the projects
develop minimum data privacy standards
engage with key stakeholders
recommend areas for future collaborative research.
The project delivered a toolkit for the identification, selection and measurements of outcomes (PDF) to support the other BD4BO projects.
Exploring the assessment and appraisal of regenerative medicines and cell therapy products
In April 2015, we launched an exploratory study to highlight key issues in the evaluation of regenerative medicines (PDF) and consider the suitability (or otherwise) of current methods. The study responds to recommendations from the Department of Health Regenerative Medicine Expert Group (RMEG).
NICE has worked in collaboration with the Centre for Reviews and Dissemination and Centre for Health Economics, University of York, who have produced an extensive independent report (PDF).
Visit the University of York website to view the full report.
The NICE Regenerative Medicines and Cell Therapy report (PDF) summarises key findings from the study, considers implications for NICE and makes a number of recommendations.
Extending the QALY (E-QALY)
The E-QALY project aimed to develop a broad measure of quality of life for use in economic evaluations across health and social care, for both patients and carers.
It explored the importance of non-health aspects (such as social and emotional wellbeing) in addition to physical and mental health so that important benefits, other than those related to health, are captured.
Flatiron Health
The partnership between NICE and Flatiron Health aimed to explore if and how real world evidence can inform the clinical and cost effectiveness of health technologies. An initial research project compared survival estimates from clinical trials to survival data observed in actual patient records to evaluate opportunities to reduce uncertainty in the estimation of long-term outcomes.
The collaboration tested how additional sources of evidence could complement trial and research data in NICE's technology appraisals, especially in helping to resolve uncertainty in the evidence.
View the Flatiron Health project.
GetReal Initiative
The GetReal Initiative was an Innovative Medicines Initiative (IMI) project which aimed to develop tangible solutions to key challenges associated with using real world data in drug development and subsequent regulatory and health technology assessment.
The project established a real world evidence Think Tank, which gathered international thought leaders to discuss, assess and give recommendations on the opportunities and barriers to the generation, use and acceptability of real world evidence.
This project followed on from successful completion of the IMI GetReal project on the use of real world evidence in effectiveness research.
View the GetReal Initiative project.
HARMONY: Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in Haematology
The HARMONY alliance aimed to use big data and big data analytics to deliver knowledge to improve the care of patients with several blood cancers.
A key output from this project was the HARMONY BigData platform. As of April 2023, this held datasets of approximately 156,000 patients. This platform harmonises and analysis these data sets, with the aim of accelerating the development of more effective treatments for people with blood cancer.
HARMONY PLUS
HARMONY PLUS, launched in October 2020, builds on the success of HARMONY in using big data and analytics to deliver knowledge that will improve the care of patients with several blood cancers.
The project expands the focus to additional blood diseases that were not covered by HARMONY and further deploys artificial intelligence techniques on an expanded big data platform.
View the HARMONY PLUS project.
Histology independent cancer drugs
The European Medicines Agency approved the first histology independent cancer drug in 2019. The clinical evidence on the effectiveness of these drugs makes it hard to assess:
the level of benefit to patients
if the drug provides value for money to health systems such as the NHS.
We initiated methods research through the National Institute for Health Research health technology assessment programme. This addressed the following questions:
Can we apply our existing technology appraisal approaches and are any changes required?
What evidence will be available at the point of initial marketing authorisation for these drugs?
What guidance is needed to establish the evidence and analyses required to inform our recommendations?
Read the pre-publication report and recommendations (Word) from researchers at the University of York and the University of Sheffield.
HTx: Next Generation Health Technology Assessment
The project aims to create and test a framework for ‘next generation’ health technology assessment to support patient-centred, societally oriented, real-time decision-making on access to and reimbursement for health technologies throughout Europe.
The framework will provide solutions for a broad range of challenges, such as:
personalised medicine
combination therapies
big data and artificial intelligence (AI).
IMI GetReal
GetReal aimed to develop a clearer understanding of how real-world evidence (RWE) can contribute to pharmaceutical research and development and healthcare decision-making.
As part of GetReal, we worked with the University of Manchester and the Association of British Pharmaceutical Industry (ABPI) to publish a report of recommendations for advancing the UK's capability for data science research in healthcare.
We also developed the RWE Navigator, an online platform to support better understanding of the potential of real world evidence in the development and assessment of new drugs.
IMPACT HTA
IMPACT HTA proposed new and improved methods, tools and guidance for decision-makers across 10 research areas in the context of health technology assessment and health system performance measurement. This contributed to the understanding of:
costs and health outcomes variations within and across countries
costs and health outcomes data integration from different sources.
Our role in the project included:
assessing the performance of a range of statistical methods used to analyse non-randomised studies
providing recommendations on which methods are likely to produce valid and unbiased estimates of relative effectiveness of interventions.
The methodological recommendations produced in this project will be useful for informing future updates of our methods guides in relation to the use of non-randomised studies, sometimes referred to as ‘real world studies’.
NEURONET: Efficiently Networking European Neurodegeneration Research
NEURONET was a coordination and support action project setup to provide support to a broad portfolio of IMI projects on neuro-degenerative diseases.
A key output from the project is an interactive Knowledge Base. This brings together key information about the 18 projects in the IMI neuro-degenerative diseases portfolio, including links to project publications, tools, and deliverable reports.
The Knowledge Base is an integral part of NEURONET's endeavour to boost collaboration across the research portfolio and enhance its visibility with related initiatives in Europe and worldwide.
NICE led work on the Regulatory and HTA Decision Tool, which provides a clickable overview of the processes and procedures for HTA and regulatory interactions at different stages of the development pipeline. This will help ensure that the outputs being developed by projects are relevant for regulatory and HTA settings, where applicable.
Patient Preferences
We worked in partnership with Myeloma UK to explore potential quantitative methods for capturing and using patient preferences within health technology assessment decision-making. This initial ‘proof of concept’ project was funded by Myeloma UK and focused on patients with multiple myeloma.
ROADMAP (Big Data for Better Outcomes)
The ROADMAP project worked on improving real world evidence in Alzheimer's disease.
The project launched the Data Cube in 2019, which provides an interactive overview of European data sources that capture outcomes relevant for Alzheimer's disease.
VALUE-Dx: The value of diagnostics to combat antimicrobial resistance by optimising antibiotic use
VALUE-Dx will facilitate and accelerate the rigorous assessment and implementation of diagnostic technologies into healthcare settings. It will do this by establishing the methods, processes and approaches needed to understand, evaluate, and assess the value of diagnostics in the context of optimising antibiotic use. VALUE-Dx has a particular focus on community-acquired acute respiratory tract infections (CA-ARTI).
The project will produce recommendations on how to improve existing pricing and funding policies to ensure timely accessibility of cost-effective diagnostics to reduce antimicrobial resistance.