Real-world data: how can we use it for health technology assessment decision-making?

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Randomised clinical trials (RCTs) are the gold standard of evidence where treatments and therapies are concerned. However, RCTs are often insufficient for HTA purposes. There is a growing interest in using real-world data (RWD) to help to fill evidence gaps when appraising new drugs. In June 2022, the National Institute for Health and Care Excellence (NICE) published a real-world evidence framework to improve the use of RWD in their guidance. My PhD thesis examined the use of RWD in a diverse way, to provide an in-depth understanding of this issue. The findings of my research can bring significant insights for decision-makers and have positive impacts on HTA decisions and the broader society and economy. During this fellowship, I will continue my upward career trajectory in health economics, specifically within the HTA context.Objective 1: Disseminate and translate my research findingsI will achieve academic impact through dissemination and translation to raise awareness of the use of RWD in NICE appraisals. This includes three critical activities: publication, soft dissemination, and translation. I will publish two further papers on the use of RWD in health economics journals. Another intended dissemination activity is soft dissemination. I will attend relevant conferences and promote the impact of my research with non-academic bodies. As a knowledge exchange activity, I will host a stakeholder workshop to raise awareness of the use of RWD in decision-making. I will also have advisory meetings with NICE appraisal committee members, independent academic groups, and NICE professionals. Having advisory meetings with NICE is timely as, in its guidance, NICE has recently moved toward advocating greater use of RWD.Objective 2: Foster research networks to develop impact opportunitiesCollaboration with other researchers is critical for developing my career. Essential collaborators are researchers in the Global Health Economics Centre (GHECO) and Inequalities in Cancer Outcomes Network (ICON) groups at the LSHTM; I will learn new skills and create future research opportunities. Especially, through collaboration with ICON, I will be able to access RWD as a collaborator to learn more about their RWD dataset and what sort of analysis can be undertaken. This will present a good opportunity to look at a real database and understand how RWD can provide information on variables in economic models to support HTA decision-making. This collaboration will lay the foundation for planning the next stages of my academic career.Objective 3: Training for personal and professional developmentI will concentrate on training and development for developing my skills and knowledge to underpin my future career. I will undertake various training activities during this fellowship: short courses, in-house training, mentorship. I will undertake short training courses, including advanced statistics courses and specialised classes focusing on challenges associated with critical areas of evidence generation and HTA methodology. Also, I will participate in development opportunities at my host institution, LSHTM, to equip myself with various skills, such as leadership, communication, budget management, and grant writing skills. Most importantly, I will receive mentorship from my mentor and the senior academics at LSHTM to guide my career development.Objective 4: Develop advanced funding proposalsI aim to develop funding proposals to answer relevant questions such as "How can trial evidence be calibrated using RWD for HTA decision-making?" I have identified relevant future funding opportunities, including ESRC new investigator grant and NIHR advanced fellowship. I will continue with my collaborations built from this fellowship during the next stage of my academic career, identifying the right partners to maximise the impact of my research, and drawing on advice from my collaborators and LSHTM's Strategic Research Office.