关于报告
A record number of new oncology drugs has been approved in recent years, bringing new treatment options to patients. However, despite robust levels of pipeline activity, oncology remains a challenging area for research and development. This report examines the productivity and output of the oncology pipeline, new treatment mechanisms, and which patients will likely benefit from new therapies. Our research brings into focus the amount spent on oncology medicines globally, clinical trial activity, complexity and success, and the outlook through 2023. The report also addresses shifts in therapy use with the emergence of immuno-therapies, Next-Generation Biotherapeutics and biosimilars.
报告摘要
A record 15 new oncology therapeutic drugs were launched in 2018 for 17 indications. Over half of the new therapies are delivered as an oral formulation, have an orphan indication or include a predictive biomarker on their label. Recently introduced therapies are also being used more broadly across varied tumor populations and in earlier lines of therapy. The use of immuno-oncology therapies has doubled in the United States since 2017 and treatment with novel CDK 4/6 inhibitors for HER-2 negative breast cancer has dramatically increased in the United States and Europe.
后期发育中的药物管道仅在2018年就扩大了19％，自2013年以来为63％。在管道和临床开发的所有阶段中，最激烈的活动集中在近450个免疫疗法上，具有60多种不同的作用机制。98个下一代生物治疗剂（定义为细胞，基因和核苷酸疗法）也在临床研究中，并利用18种不同的方法。
尽管管道活动水平很高，但肿瘤学仍然是研发最具挑战性的领域之一，面临着大量失败和长期发展时期的巨大风险。2018年，2018年相结合的综合成功率从2017年的11.7％下降到8.0％，肿瘤学试验的临床试验持续时间仍然高于其他疾病领域。在过去的五年中，I期肿瘤学试验的临床试验复杂性也急剧增加。自2010年以来，肿瘤学试验的总体生产率（相对于试验工作（复杂性和持续时间））的总体生产率已提高了22％，但仍远低于其他治疗领域的试验。
用于治疗癌症患者的所有药物的支出在2018年达到了近1500亿美元，这一年度增长了12.9％，并标志着连续第五年的双位增长，这完全由治疗药物驱动，该药物的增长15.9％。新药物的平均每年成本继续趋势上升，尽管中位成本在2018年下降了13,000美元，至149,000美元，而每种产品的成本范围为90,000美元，超过30万美元。中国领导了药房的支出和增长市场，在2018年增长了24％，达到了90亿美元的总支出，即使中国的支持性护理治疗下降了10％。在接下来的五年中，预计将以复合年增长率将治疗性支出增长11-14％，使总市场达到200-23.33亿美元。
关键发现
在过去的五年中，有57种新推出的肿瘤学治疗剂获得了89种适应症的批准，一些药物治疗了多种肿瘤类型
- A record number of new oncology drugs was launched in 2018, bringing new treatment options to patients and continuing the transformation of treatment patterns occurring from the introduction of immunotherapies less than five years ago.
- Almost one-third of the approved indications over the past five years have been for hematologic cancers while lung cancer leads the solid tumors with 12 indications, followed by breast cancer and melanoma.
- In 2018, larotrectinib became the second tissue-agnostic oncology therapy to be approved, following the first approval of pembrolizumab in 2017. This reflects the beginning of a paradigm shift occurring in oncology to treat tumors based on genetic profile rather than site-of-origin in the body.
后期肿瘤学管道包括2018年的849个分子，自2008年以来增长了77％，由于目标疗法数量增加
- The number of late-stage pipeline therapies grew from 711 in 2017 to 849 in 2018 – an expansion of 19% – due to the growing number of targeted therapies in the oncology pipeline.
- 91% of the late-stage oncology pipeline in 2018 were targeted small molecule and biologic therapies, rather than non-specific therapies like cytotoxic agents.
- 由于探索了一系列机制，所涉及的公司数量以及研究的发展速度，因此管道中越来越多的药物尤其值得注意。
- In late-stage oncology R&D, 711 companies are active, working on a total of 849 products, with the majority (88%) from emerging biopharma companies.
Almost 100 Next-Generation Biotherapeutics are now in late-stage development
- 自2013年以来，肿瘤学的开发中定义为细胞，基因和核苷酸疗法的下一代生物治疗剂的数量已增加了一倍以上，并且从2017年到2018年增长了32％，随着疾病治疗的新途径和治疗途径不断增长。。
- Nearly 450 immuno-oncology therapies are currently in development across all phases, with the Phase III and pre-registration pipeline containing nine mechanisms and the early- stage pipeline containing 62 mechanisms.
- PD1/PD-L1 checkpoint inhibitors remain the most successful immuno-oncology therapies, and improvements in formulation (e.g., oral) or immunotherapy combinations with targeted therapies (e.g., TKIs) or Next-Generation Biotherapeutics may lead to therapy breakthroughs.
Oncology clinical trials have a high risk of failure with a composite success rate of 8% in 2018, slightly lower than the average since 2010
- 自2013年以来的肿瘤产品以来，综合成功率衡量了进入I阶段的可能性，该产品将进入市场，而2015年和2017年的肿瘤学产品差异大致相同，而差异在7-8％之间。2010 - 2018年的总体平均水平为10.6％。
- 在所有试验阶段，2018年的肿瘤学试验的平均持续时间为3。2年，而其他所有治疗领域的平均持续时间为1。8年，差异超过40％。
- 肿瘤学的临床试验复杂性从2014年到2018年增长了11％，从2010年到2018年增长了22％，并受到端点和资格标准数量的增加而驱动的，并且被国家数量和所包括的站点数量减少所抵消。
建模当前临床发展趋势对未来生产率的影响，预筛查患者和生物标志物测试的可用性分别提高生产率，分别提高到2023年，分别提高了104％和71％。
- Overall productivity of oncology trials – measured as success rates relative to trial complexity and duration – has improved by 22% since 2010 but remains far lower than trials for other therapy areas.
- 预计肿瘤学发展中最有影响力的趋势是预先筛选的患者的可用性，以评估试验的资格并提高试验的速度和效率。
- 无论是由于药物发现还是通过其他研究，生物标志物仍在继续发现，并且测试的范围和可用性将大大增强药物开发的所有方面。
到2023年，肿瘤学支出将达到近2400亿美元，增长9-12％
- 在治疗药物驱动的所有药物治疗中，所有用于治疗癌症患者的药物的支出均在2018年达到12.9％，因为在治疗药物的驱动下，在2018年支付支持性护理药物的支出下降了1.5％。
- 新肿瘤药物的平均每年成本继续趋势，尽管中位成本在2018年下降了13,000美元，至149,000美元。
- 在过去的五年中，中国的肿瘤学支出增加了一倍以上，主要来自现有品牌药物的使用，而新推出的药品和人均支出的使用量很少，每人4.50美元，而美国在美国的$ 173。。
- Growth in spending on oncology therapeutics through 2023 is forecast at double-digit levels in the United States, pharmerging markets and rest-of-world, will reach the high single-digit growth in the EU5, and 5–8% in Japan.