November 23, 2022
By Dean A. Regier, Samantha Pollard, Melanie McPhail, Tania Bubela, Timothy P. Hanna, Cheryl Ho, Howard J. Lim, Kelvin Chan, Stuart J. Peacock & Deirdre Weymann
Healthcare decision-makers use evidence of effectiveness and value to understand which treatments to offer patients. When treating patients with cancer, new therapies may use patients’ DNA to guide whether that treatment will be effective.
Treating cancer patients based on their DNA is called precision oncology. Evidence to support decision-making for precision oncology is hard to generate, partly because of the rarity of disease. When diseases are rare, it is difficult to enroll enough patients in clinical trials to understand causal estimates of safety and effectiveness.
Evidence can also be low quality because randomized controlled trials are uncommon in precision oncology. Without randomized controlled trials, we only see the outcomes of treated patients; this means decision-makers are often uncertain if precision oncology works compared to other treatments.
This uncertainty prevents timely patient access to new therapies.
Funded by Genome BC and Genome Canada through the GiSIRT initiative, the Canadian Network for Learning Healthcare Systems and Cost-effective ‘Omics Innovation (CLEO) developed a framework to generate better evidence for precision oncology. CLEO based the approach on a concept called ‘learning healthcare’, where data is systematically collected as part of routine patient care. The CLEO Network’s primary aim is to support learning healthcare for precision oncology in Canada. Learning healthcare systems collect data, create evidence and use that evidence to inform treatment decisions.
In their article published in the journal npj Precision Oncology, CLEO investigators describe how a life cycle health technology assessment (LC-HTA) framework allows learning healthcare systems to provide patients with new treatments while they monitor how well those treatments work in the real world. The LC-HTA framework helps decision-makers to understand whether to continue collecting evidence, fund the new treatment, or stop collecting evidence. The goal is to achieve timely patient access to precision oncology that improves population health and helps us understand the value of precision oncology.
The CLEO Network is continuing to build on the framework and consider implementation requirements. The validity of the recommended methods for measuring effectiveness and value are being tested in precision oncology case studies. Case studies include only-in-research precision oncology initiatives, such as the Ontario-wide Cancer Targeted Nucleic Acid Evaluation (OCTANE – NCT02906943) and the BC Personalized OncoGenomics program (POG – NCT02155621). Evaluations of publicly available precision oncology technologies are also underway. In BC, investigators are examining the real-world impacts of using multi-gene panels to inform cancer treatments as part of standard care.
CLEO is partnering with BC’s provincial cancer control system and pharmaceutical companies to pilot the entire proposed framework and develop learning healthcare. Through the PRecision Oncology Evidence Development in Cancer Treatment program (PREDiCT), BC Cancer is providing patients with promising new therapies that are challenging to evaluate. They are expanding the data collected in everyday patient care, monitoring real-world outcomes and using that evidence to inform cancer care for future patients in BC.
CLEO’s evidence-generation framework provides a roadmap to achieve learning healthcare for precision medicine. To make this framework fit for purpose, researchers need to engage with healthcare systems and all stakeholders, including patients, the pharmaceutical industry and Canadian regulatory and reimbursement agencies.
Recognizing this need, CLEO is working with healthcare system partners and stakeholders. They are sharing knowledge to precision oncology networks, like the Terry Fox Research Institute Marathon of Hope Cancer Centres Network (TFRI MOHCCN). They are also working with drug regulators in Canada to understand legal and policy levers and barriers for learning healthcare. Similar engagement efforts will be needed in other jurisdictions implementing the framework.
Visit the website to find out more about the CLEO Network or email them at firstname.lastname@example.org. Together, let’s co-create learning healthcare systems for precision oncology.