Bile Duct Cancer
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Medical genetics company Invitae announced on Thursday that it has inked a collaboration with pharma heavyweight AstraZeneca to leverage Invitae’s Ciitizen genomic and health history data in a retrospective and prospective study of patients diagnosed with the rare bile duct cancer cholangiocarcinoma. The patient-consented data for the study will come from an existing relationship between Invitae and patient advocacy group the Cholangiocarcinoma Foundation (CCF).

“CCF partnered with Invitae to use the Ciitizen data platform in its earliest days because we saw a need for our patient community to gain control of their own medical records and drive research with their own health data,” said Stacie Lindsey, CEO, CCF. “This is a groundbreaking example of how patient-initiated research and the quality of data Invitae’s Ciitizen platform produces from medical records can inform real-world questions that will help develop, improve and expand therapies. Our patient community wants their health data to support research that improves treatment options in this aggressive and rare cancer.”

This new research initiative is part of a growing trend for rare cancer and rare disease research in general that sees patient advocacy organizations seeking out data platform partners and organizations that have the technical capability of housing consented patient health and genomic data. Collecting these data for rare diseases in a single place helps facilitate research by pharma and biotech companies, which in the past may have shied away from rare disease studies due to the difficulty of finding a critical mass of patients.

For AstraZeneca, the access to the CFF data doesn’t represent a new disease area of focus, rather a deeper dive into the mechanisms of cholangiocarcinoma. On September 5, the company received approval from the FDA for its immunotherapy tremelimumab (Imfinzi) in combination with GemCis chemotherapy from its Phase III TOPAZ-1 clinical trial. This made it the only first line treatment for locally advanced or metastatic bile duct cancers, including cholangiocarcinoma and gallbladder cancer.

“We developed the first immunotherapy-based treatment option to be approved that meaningfully extends long-term survival and has a manageable safety profile for people with cholangiocarcinoma, but we’re not stopping there,” noted Camille Hertzka, vice president and head of U.S. oncology medical affairs, AstraZeneca. “We’re committed to harnessing the power of CCF and Invitae’s Ciitizen data set to more deeply understand treatment patterns and patient outcomes and continue improving care for this community of patients.”

The intent now is to use the CFF patient data in an ongoing longitudinal study of cholangiocarcinoma patients in the TOPAZ-1 regime as well as other treatment plans. These patients will be followed for the next three years to better understand the long-term efficacy of their treatments in the hopes of identifying new drugs that can produce even better outcomes. Data collected will include tolerability of the regimen; autoimmune comorbidities and their impact on outcomes; sequencing of treatments; and observation of real-world usage of Abraxane added to TOPAZ-1 backbone.

“We are thankful to the Cholangiocarcinoma Foundation and its patients for trusting us with their health data and to AstraZeneca for seeing the potential of this data to inform their scientific endeavors,” said Farid Vij, general manager of patient network and data at Invitae. “With this investment from AstraZeneca, our Cholangiocarcinoma Foundation patients are joining the research data ecosystem and seeing their data used to advance cures for this community, something our patients have always believed in. This reflects our core ethos, that data belongs to the patient and is more powerful when shared, and also demonstrates that we can generate data at a level of quality that can inform key insights and decisions for both AstraZeneca and cholangiocarcinoma patients.”

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