AACR 2020: real-world evidence highlights

In this article, we summarize our news and program highlights from the American Association for Cancer Research (AACR) Virtual Annual Meeting I (27–28 April 2020) that covered real-world data and evidence in relation to oncology.

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The subject of real-world evidence has gained increasing interest in the field of oncology as researchers and clinicians alike look for alternative data sources beyond clinical trial data alone to help them address clinical and policy-relevant issues; at the American Association for Cancer Research (AACR) Virtual Annual Meeting I (27–28 April 2020) the topic of real-world data and evidence drew much attention.

If you missed the chance to attend this exciting meeting, we’ve collated the meeting’s real-world evidence highlights below, including the use of an international cancer registry to advance research and precision medicine in oncology, and what real-world evidence has revealed about the management of COVID-19 in individuals with cancer.

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How is an international cancer registry advancing research and precision medicine in oncology?

Our first real-world evidence highlight from the meeting was the virtual mini symposium entitled ‘Advancing cancer research through an international cancer registry: AACR Project GENIE use cases’, which was held on Monday 27 April at 4:00pm.

During this session, six case studies were presented that provided an update on the status of the AACR Genomics Evidence Neoplasia Information Exchange (GENIE) project, highlighting how use of this international real-world data registry could help both inform healthcare decision making for rare cancers and achieve precision medicine in oncology.

A major challenge to realizing the promise of precision medicine in oncology is that no single institution is able to sequence and treat sufficient numbers of patients to improve clinical decision making independently; the AACR GENIE project was setup to help remedy this.

The project involves combining existing and ongoing clinical genotyping data – which are routinely collected for cancer patients – into a single registry that is shared between 19 of the world’s leading cancer centers. This real-world dataset allows researchers and clinicians to select clinical outcomes; key applications of this registry include drug and biomarker discovery, evaluation of clinical trial feasibility and much more.

Indeed, in one study presented during this session, researchers from Novellusdx (Jerusalem, Israel) detailed their profiling of the effects of 151 different BRAF mutations identified in the AACR Project GENIE dataset on the activity of the MAPK signaling cascade – hyperactivation of which is commonly observed in many tumors – and their responses to various BRAF inhibitors used as cancer treatments.

Researchers observed that whilst vemurafenib – a US FDA-approved BRAF inhibitor – efficiently inhibited BRAF V600 mutations, other, less-common BRAF mutations were less responsive to this drug. By contrast, second-generation experimental BARF inhibitors were effective against both V600 and non-V600 mutations [1].

These data can serve as a basis for rational drug design as well as help inform more accurate treatment options for individuals with BRAF-mutated cancers.

In their abstract, the authors concluded:

Using this large-scale approach to characterize BRAF mutations, we were able to functionally annotate the largest number of BRAF mutations to date."

In another session presentation, Valsamo Anagnostou (Sidney Kimmel Cancer Center at Johns Hopkins University, MA, USA) described her team’s use of the Project GENIE registry to estimate cancer-specific prevalence of RAS and non-RAS somatic mutations, utilizing a Bayesian hierarchical model [2].

Read about all six studies presented here>>

COVID-19 and cancer: what clinicians need to know

Our second program highlight was the virtual plenary session on COVID-19 and cancer, which took place on Tuesday 28 April at 9:00am.

A number of early and published studies suggest that individuals with cancer may be more vulnerable to COVID-19. In this virtual plenary session, several real-world studies were presented that relate to the practical management of cancer patients with COVID-19 and how treatment strategies may need to be adapted to better care for them.

In one study, researchers analyzed data on 28 cancer patients with COVID-19 in three Wuhan hospitals from 13 January–26 February 2020, as well as 124 individuals receiving immune checkpoint inhibitors, and their family members, to help identify risk factors that could indicate poor outcomes for patients [3].

Read more about this study on our sister site, BioTechniques®, now>>

Another critical study presented during this session concerned the first ever results obtained from the Thoracic cancERs international coVid 19 cOLlaboraTion (TERAVOLT) registry.

Supported by the European Society for Medical Oncology (ESMO; Ticino, Switzerland), the International Association for the Study of Lung Cancer (IASLC; CO, USA) and the European Thoracic Oncology Platform (ETOP; Bern Switzerland), 160 institutions, across 21 countries, have shown interest in TERAVOLT.

Abstract author, Marina Garassino (IRCSS National Cancer Institute Foundation, Milan, Italy), presented the initial findings, which relate to 200 patients with COVID-19 and thoracic cancers, in eight countries.

The results demonstrate that patients with more than one comorbidity were at increased risk of prolonged hospitalization and mortality due to COVID-19. There was also an increased risk of hospitalization if the patient had chronic obstructive pulmonary disease [4].

Read more about the TERAVOLT study on our sister site, Oncology Central, now>>

Do real-world data corroborate clinical trial data outcomes?

Our final real-world evidence highlight from the meeting was a study presented during the virtual plenary session on lung cancer targeted therapy, on Monday 27 April at 2:00pm.

In this new study, investigators from the US FDA, Syapse (CA, USA) and Advocate Aurora Health (WI, USA) compared pneumonitis – an acutely life-threatening adverse event that can be associated with administration of certain common anti-cancer drugs – incidence rates amongst more than 7500 individuals with advanced non-small-cell lung cancer receiving either immune checkpoint inhibitors or chemotherapies.

Investigators utilized both real-world and clinical trial data, to determine if the observed outcomes were comparable and reflective of real-world clinical settings.

Clinicians and regulators are interested in real-world safety profiles to develop new practices for managing risk and evaluating potentially beneficial therapies when comorbidities such as pneumonitis are present. This research provides encouraging new data that clinicians can use to better understand the post-approval behavior of drugs in the real world,"

– Michael Thompson, Medical Director of the Early Phase Cancer Research Program at Advocate Aurora Health [5]. 

Similar rates of treatment-associated pneumonitis were observed in both the clinical trial and real-world datasets; researchers observed greater incidences of treatment-associated pneumonitis in individuals who had past medical histories of pneumonitis compared with those who had never had the condition [6].

Further, the real-world data suggest that pneumonitis incidence is strongly related to radiotherapy provision, when administered in combination with either immune checkpoint inhibitors or chemotherapy; 73% of all pneumonitis diagnoses resulted from radiotherapy [6].

Read more about this news here>>

Want more news from AACR 2020? Check out the daily coverage from our sister site, Oncology Central, for the latest news, opinions and more>>


[1] Barbash Z, Haham D, Hafzadi L et al. A systematic analysis of BRAF mutations and their sensitivity to different BRAF inhibitors. Presented at the American Association for Cancer Research Virtual Annual Meeting I (27–28 April 2020). Abstract number 1092. 

[2] Scharpf R, Riely G, Awad M et al. Comprehensive pan-cancer analyses of RAS genomic diversity. Presented at the American Association for Cancer Research Virtual Annual Meeting I (27–28 April 2020). Abstract number 1095.

[3] Zhang L, Zhu F, Xie L et al. The experience of treating patients with cancer during the COVID-19 pandemic in China. Presented at the American Association for Cancer Research Virtual Annual Meeting I (27–28 April 2020). Session number VCTPL09.

[4] Garassino MC. TERAVOLT (Thoracic cancERs international coVid 19 cOLlaboraTion): First results of a global collaboration to address the impact of COVID-19 in patients with thoracic malignancies. Presented at the American Association for Cancer Research Virtual Annual Meeting I (27–28 April 2020). Session number VCTPL09.

[5] www.syapse.com/news/press-releases/aacr-2020-pneumonitis 

[6] Liu Q, Zhang C, Gong Y et alPneumonitis incidence in patients with non-small cell lung cancer treated with immunotherapy or chemotherapy in clinical trials and real-world data. Presented at the American Association for Cancer Research Virtual Annual Meeting I (27–28 April). Session number: VCTPL08.


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