Arora Meena, Kilcoyne Adrian, Bolodeoku John, El Baghdady Assem
Onconova Therapeutics Inc, Newtown, Pennsylvania, USA
Celularity Inc, Florham Park, New Jersey, USA.
BMJ Open. 2024 Dec 26;14(12):e086058. doi: 10.1136/bmjopen-2024-086058.
To understand the competitive position of the UK in comparison to Europe and the USA for haematological cancer clinical research.
Using commercially available databases, clinical trial numbers, their effectiveness and publication outputs were evaluated in two analyses: a macrodevelopment and a research activity and performance analysis.
The following databases were used for this analysis: Organisation for Economic Co-operation and Development, Thomson Reuters Incidence and Prevalence, the Cortellis Clinical Trial Intelligence, the Clarivate Cortellis Innography Patent Intelligence, Thomson-Reuters Cortellis Regulatory Intelligence, Thomson Reuters Web of Science and data from the Centre for Medicine Research (CMR).
European countries with comparable geography, healthcare standards and economies, as well as the USA, the largest country where research is conducted. All haematological oncology clinical trials from phase 1 to phase 3 were included.
All data were retrieved in September 2017 and macroeconomic data were reviewed in 2022; haematological cancer data were restricted to leukaemias generally as a surrogate reference for haematological oncology indications; research output publication data were evaluated using specific MeSH/keyword search terms between 2010 and 2017. Key metrics explored included healthcare expenditure per capita, study experience across countries, comparative capability of each country for clinical trial implementation, clinical trials' performance and impact of research as measured by impact factor and citation metrics of publications.
Revenue for clinical studies is lower in the UK than European comparators. All studied countries had comparable leukaemia prevalence rates, but the UK spent least per capita on healthcare versus France, Germany, Spain and the USA. The number of clinical studies in the UK showed a decline compared with other European countries. Clinical trial implementation was lowest in the UK (n=380) versus Germany (n=665), France (n=643), Spain (n=632), Italy (n=538) and the USA (n=3254). Registered versus active clinical studies suggested the USA had the highest number underway (n=824), with the UK ranked fourth of five European countries (Germany=239, Italy=232, France=211, UK=177 and Spain=141). However, the UK had the highest completion rate of phase 3 studies it did initiate (n=154, 87%) and performance was comparable with Germany (n=188, 78.7%) and France (n=151, 71.6%). When analysed by phase, the UK was the second highest European performing country (n=121) for phase 2 study completion compared with Germany (n=182) both less than the USA (n=345). The UK completed the most phase 1 studies compared with other European countries, only second to the USA (n=31 vs n=126). However, the UK clinical trial performance metrics were negatively impacted for the UK compared with other European countries with respect to clinical trial application (CTA) process, timelines, ethics committee approvals, median time to start up and rate of non-enrolling sites. The UK was slower to initiate studies (median 186 days) vs Germany (92 days), France (141 days), Italy (122 days) and only marginally faster than Spain (195 days). While median enrolment rates were comparable across all countries, the UK had the highest proportion of sites that failed to enrol any patients (despite regulatory timings being comparable to Germany (90 days) and France (95 days)). However, publication of data following clinical trials in the UK was robust and of the highest quality compared with other countries, judged by journal placement and publication citations. The UK published high-quality, diverse research with citation rates (11.8) from clinical studies which was higher than every other country, including the USA who published fivefold more publications per year.
While research in the UK remains among the highest quality and value globally, the UK may be losing its position globally as an attractive destination for executing clinical trials. This may be a trend that is recognised by the UK Government, but it is vital to reverse the trend of clinical trial decline and to improve the economic outlook for the UK and patient early access to innovative cancer medicines.
了解英国在血液癌症临床研究方面与欧洲和美国相比的竞争地位。
利用商业可用数据库，在两项分析中评估临床试验数量、其有效性和发表成果：宏观发展分析以及研究活动与绩效分析。
本分析使用了以下数据库：经济合作与发展组织、汤森路透发病率与患病率数据库、科睿唯安临床试验情报数据库、科睿唯安专利情报数据库、汤森路透监管情报数据库、汤森路透科学网以及医学研究中心（CMR）的数据。
地理、医疗保健标准和经济状况可比的欧洲国家，以及开展研究的最大国家美国。纳入所有1期至3期的血液肿瘤学临床试验。
所有数据于2017年9月检索，宏观经济数据于2022年进行审查；血液癌症数据一般限于白血病，作为血液肿瘤学适应症的替代参考；研究产出发表数据使用2010年至2017年期间的特定医学主题词/关键词搜索词进行评估。探索的关键指标包括人均医疗保健支出、各国的研究经验、各国实施临床试验的相对能力、临床试验的绩效以及通过出版物的影响因子和引用指标衡量的研究影响力。
英国临床研究的收入低于欧洲可比国家。所有研究国家的白血病患病率相当，但与法国、德国、西班牙和美国相比，英国人均医疗保健支出最少。与其他欧洲国家相比，英国的临床研究数量呈下降趋势。英国的临床试验实施数量最低（n = 380），而德国为（n = 665），法国为（n = 643），西班牙为（n = 632），意大利为（n = 538），美国为（n = 3254）。注册与正在进行的临床研究表明，美国正在进行的临床试验数量最多（n = 824），英国在五个欧洲国家中排名第四（德国 = 239，意大利 = 232，法国 = 211，英国 = 177，西班牙 = 141）。然而，英国启动的3期研究完成率最高（n = 154，87%），绩效与德国（n = 188，78.7%）和法国（n = 151，71.6%）相当。按阶段分析时，与德国（n = 182）相比，英国在2期研究完成方面是欧洲表现第二好的国家（n = 121），两者均低于美国（n = 345）。与其他欧洲国家相比，英国完成的1期研究最多，仅次于美国（n = 31对n = 126）。然而，与其他欧洲国家相比，英国的临床试验绩效指标在临床试验申请（CTA）流程、时间线、伦理委员会批准、启动的中位时间和未招募患者的站点比例方面受到负面影响。英国启动研究的速度较慢（中位时间186天），而德国为（92天），法国为（141天），意大利为（122天），仅略快于西班牙（195天）。虽然所有国家的中位入组率相当，但英国未招募任何患者的站点比例最高（尽管监管时间与德国（90天）和法国（95天）相当）。然而，从期刊发表位置和发表引用判断，英国临床试验后数据的发表情况良好且质量最高。英国发表了高质量、多样化的研究，临床研究的引用率（11.8）高于其他所有国家，包括每年发表数量是英国五倍的美国。
虽然英国的研究在全球范围内仍保持最高质量和价值，但英国作为开展临床试验的有吸引力目的地，可能正在失去其全球地位。这可能是英国政府已经认识到的一种趋势，但扭转临床试验下降趋势并改善英国的经济前景以及患者尽早获得创新癌症药物至关重要。
