解锁新技能：巧用流病数据制定产品组合战略，就是这么机智！
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免疫疗法一直被业内认为是未来肿瘤领域新一轮销售增长点，特别是将药物研发与新型疗法相结合的时候， 整个市场格局可以用“热火朝天”四个字来形容。就比如说，最近超级火爆的CAR-T领域，仅前7大相关项目在2022年就将高达45亿美金的产值（EvalutatePharma预估医药，图1）。
图1： 2022年全球排名前7的CAR-T项目
在所有49个CAR-T在研项目当中，最火热的两大项目－诺华的CTL019以及Kite的KTE-C19瞄准的都是白血病和淋巴瘤这样的“缝隙市场（Niche market）。该疾病拥有着巨大的未满足的药物“需求”，尤其是对于儿童，复发性及难治性的人群。根据EvaluatePharma的流行病学分析模块（Epi Analyzer），预计到2018年，欧洲五国和美国中分别有48,460例淋巴细胞性白血病，10,820例和急性淋巴细胞性白血病及3120例 前B淋巴母细胞性淋巴瘤的新增病例（如图2所示）。
图2：2018年欧洲五国和美国急性淋巴细胞性白血病（ALL）发病率
图3: 2018年难治性及复发性，急性B细胞淋巴瘤发病率（欧洲五国与美国）
考虑到极其高昂的研发投入，虽说有孤儿药方面的政策优势，但把市场瞄准在如此“小众的”的人群，从商业角度上来看，还是很不划算呀！如何制定价格才能在商业上变成有利可图呢？ 深度挖掘预估医药中的一些数据，可以看到一种关联性，即“每药物每年每病人的花费”与“每年治疗人数”存在一定的比例关系（见图4）。因此，瞄准如此小得市场的CAR-T疗法很有可能面临更高的花费。
图4: 2016年美国孤儿药排名前20的药物销量：每病人花费与治疗人数
此外，相关临床试验虽然在儿童中显示出更好的疗效，但从市场的角度，儿童患者显然比成年人群体更小（更不划算了）。然而，“无药可医”的状态，意味着加快评审的可能性以及在价格博弈中的优势地位。此外，许多公司近期正考虑将其适应症扩展到弥漫性大B细胞淋巴瘤（DLBCL）领域，并不仅仅是出于对其安全性的考虑，还有其在商业上的野心。
最后，作为流行病出身的小编，再次给各位“商家”与“看官”安利流行病学数据（库）对于医药市场的意义。关注人群流行病学分布与动态，战略性选择对应适应症及最终定价的考量，以获得更多靶向人群或许是应对之策。
如您对EvaluatePharma 新增的流行病学版块感兴趣，想一探究竟或线上演示，欢迎联系我们evaluate@lingmed.net 或拨打电话：86 21 5386 3003
Unlocking the value of epidemiology data for improved portfolio decision-making: Success in the CAR-T space
It has been well documented that immunotherapy is a key growth area in the oncology market.
Figure1: Total Worldwide Market Value: Top 7 CAR-T Products in 2022
Despite the large number of companies operating in this space, there are still opportunities for drug development, particularly when married with the advancement of new therapeutic technologies and combination therapies. One of the newest and most technologically advanced therapeutic categories to emerge in this space has been chimeric antigen receptor expressing T-cells (CAR-T) – in essence a ‘living drug’ that utilises the process of adoptive cell transfer to prime a patient’s T cells to recognise and kill cancer cells. Evaluate forecasts have valued the top 10 products in this market to be worth a combined $4.5bn by 2022, with a pipeline of 49 unique products in development.
Figure2: Incidence of Acute Lymphocytic Leukaemia in 2018, EU5+US
The major CAR-T therapeutics, such as Novartis’ CTL019 and Kite Pharma’s KTE-C19 (axicabtagene ciloleucel), are predominantly targeting niche patient populations in leukaemias and lymphomas with high unmet need. However, some recent trials have been somewhat marred by safety concerns highlighting the need to refine the treatment approach and reduce toxicity. Understanding these patient populations can also feed into lifecycle management strategies as companies seek expanded labels treating others cancers such as diffuse large B-cell lymphoma.
In the case of CTL019, acute lymphocytic leukaemia (ALL) is the lead indication. ALL has high unmet need, particularly amongst the paediatric population and patients who relapse or are refractory to treatment and it is this group currently being addressed in the Phase1/2 pivotal trials ELIANA and KITE’s ZUMA-4 (KTE-C19). (CTL019 was recently filed on the back of strong efficacy data from the ELIANA trial.) When analysing the incidence of ALL in the EU5 and the USA using Epi Analyzer, segmentation by the overall lymphocytic leukaemia population followed by ALL reveals 2018 population forecasts of 48,460 and 10,820 respectively. A further analysis shows the 2018 incidence forecasts of ALL B-precursor to be 3,120 in the EU5 and USA combined. In all cuts, the USA is the largest market.
Figure3: Incidence of Acute Lymphocytic Leukaemia B-cell Relapsed and Refractory Patients in 2018, EU5 + US
A deeper assessment of the populations targeted in the ELIANA and ZUMA-4 trials reveals incidence to be approximately 753 patients across the EU5 + USA in 2018.
Figure4: Top 20 USA Orphan Drug in 2016 by Sales: Cost per Patient vs No. Patient Treated
Notwithstanding the high costs, it might be reasonable to assume for cell therapies such as CAR-T, these data indicate that the initial treatment-eligible population will be very small. This logically implies that a high price point will be required to make development commercially viable.
Data from Evaluate reveal that there is a correlation between the cost per patient per drug per year and the number of patients treated per year in the USA 2016 (R2=73%*), also supporting the notion that CAR-T will be likely have a high price point.
The greater efficacy in ALL trials is for the paediatric population which in turn is significantly lower in number than the adult population. However, the fact that there is little or no treatment for relapsed and refractory paediatric patients means a successful treatment choice here could warrant expedited approval and a higher price point in payer negotiations.
Funding for the development of these novel therapies and the eventual pricing for CAR-T are major challenges for manufacturers in the space. Equally challenging is the development of an appropriate pricing model that will factor in the increasing number of treatment-eligible patients reachable through further clinical development and indication expansion. A deep understanding of the dynamics of the patient population is a must to drive the right strategic decisions.
If you want to subscribe to the newly launched Epi moduel or request a demo, please contact evaluate@lingmed.net or call 86 21 5386 3003.
灵麦医药是以下两家情报数据库在中国的独家合作伙伴：
Biotechgate/博谷 - 全球创新药和器械产品收购和引进数据库
EvaluatePharma/预估医药 - 全球医药研发、销售、市场预估等竞争情报数据库
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