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

随机临床试验（RCT）是治疗和疗法相关证据的黄金标准。然而，RCT通常不足以达到HTA的目的。在评估新药时，人们越来越有兴趣使用真实世界数据（RWD）来帮助填补证据空白。2022年6月，英国国家健康与护理卓越研究所（NICE）发布了一个真实世界证据框架，以改善RWD在其指导中的使用。我的博士论文以不同的方式研究了RWD的使用，以深入了解这个问题。我的研究结果可以为决策者带来重要的见解，并对卫生技术评估决策以及更广泛的社会和经济产生积极影响。在此期间，我将继续我在卫生经济学，特别是在卫生技术评估的背景下，我的职业生涯轨迹上升。目标1：传播和翻译我的研究成果，我将通过传播和翻译，以提高在NICE评估中使用RWD的意识，实现学术影响。这包括三个关键活动：出版，软传播和翻译。我将在卫生经济学期刊上发表另外两篇关于RWD使用的论文。另一种有意的传播活动是软传播。我将参加相关会议，并与非学术团体推广我的研究的影响。作为一项知识交流活动，我将主办一个利益攸关方研讨会，以提高人们对在决策中使用RWD的认识。我还将与NICE评估委员会成员、独立学术团体和NICE专业人士举行咨询会议。与NICE举行咨询会议是及时的，因为在其指导下，NICE最近开始倡导更多地使用RWD。目标2：培养研究网络，以发展影响机会与其他研究人员的合作对发展我的职业生涯至关重要。重要的合作者是在全球卫生经济学中心（GHECO）和癌症结果网络（ICON）在LSHTM组不平等的研究人员;我将学习新的技能，创造未来的研究机会。特别是，通过与ICON的合作，我将能够作为合作者访问RWD，以了解更多关于他们的RWD数据集以及可以进行什么样的分析。这将是一个很好的机会，可以看到一个真实的数据库，并了解RWD如何提供经济模型中变量的信息，以支持卫生技术评估决策。目标三：个人及专业发展的培训我将专注于培训和发展，以发展我的技能和知识，为我未来的职业生涯奠定基础。在此期间，我将参加各种培训活动：短期课程，内部培训，指导。我将参加短期培训课程，包括高级统计课程和专门课程，重点是与证据生成和卫生技术评估方法的关键领域相关的挑战。此外，我将参加我的主办机构，LSHTM的发展机会，以装备自己的各种技能，如领导，沟通，预算管理和赠款写作技巧。最重要的是，我将得到导师和LSHTM资深学者的指导，以指导我的职业发展。目标4：制定先进的资助建议我的目标是制定资助建议，以回答相关的问题，如“如何使用RWD校准试验证据用于HTA决策？“我已经确定了相关的未来资助机会，包括ESRC新的研究员赠款和NIHR高级奖学金。我将继续与我的合作建立从这个奖学金在我的学术生涯的下一阶段，确定合适的合作伙伴，以最大限度地提高我的研究的影响，并借鉴我的合作者和LSHTM的战略研究办公室的建议。

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.