PRecision Oncology Evidence Development in Cancer Treatment - Clinical: PREDiCTc

In Canada, adoption of new systemic therapies for cancer treatment is a multi-step process. After completion of clinical trials, pharmaceutical companies submit a request for a new drug approval to Health Canada. Therapies undergo rigorous review at Health Canada to confirm safety and efficacy before a Notice of Compliance (NOC) or NOC conditional (NOC/c) is issued that allows sale of the new medication in Canada. An additional layer of evaluation, a health technology assessment (HTA), is undertaken through the pan Canadian Oncology Drug Review (pCODR). A positive recommendation from pCODR is required for national price negotiations through the pan Canadian Pharmaceutical Alliance (pCPA). Once this step is completed, provincial pharmacies establish local contracts to permit local implementation of the new therapy.

As our understanding of pathogenesis evolves and next generation sequencing becomes more affordable, rare molecular entities amenable to treatment with targeted or selected therapies, like immunotherapy, are being increasingly identified. These molecular aberrations can occur with frequencies of 1-2% making it unfeasible to conduct large randomized trials to establish benefit. Determination of the added value of these new therapies in terms of outcomes and quality of life is challenging in the absence of comparators in trials resulting in increased uncertainty in terms of outcomes, quality of life (QOL) and cost-effectiveness. To address the uncertainty, pharmaceutical companies and the pCODR economic guidance panel create economic models with sensitivity analyses to try to estimate the cost-effectiveness of therapy. These models however, are dependent on the nature and quality of available data.

This study proposes to use real world data to generate evidence to assist in evaluate new therapies in rare molecular entities. The key components include regular interval radiographic imaging and collection of patient reported outcomes using standardized QOL questionnaires. The aim is to provide high quality real world evidence for assessment and economic modelling to reduce uncertainty and facilitate HTA decision-making.

• Response rate as defined by RECIST 1.1 [Срок оценки: Baseline until progression up to 5 years]
• Response rate as defined by physician assessed response [Срок оценки: Baseline until progression up to 5 years]
• Progression free survival (PFS) from initiation of targeted therapy [Срок оценки: Baseline until progression up to 5 years]
• Quality of life (QoL) assessments using the EQ-5D every 8 weeks from initiation of targeted therapy [Срок оценки: Baseline until death up to 5 years]
• Quality adjusted survival from initiation of targeted therapy [Срок оценки: Baseline until death up to 5 years]