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MASTER KEY Project: Powering Clinical Development for Rare Cancers Through a Platform Trial.

ABSTRACT: For rare cancers, challenges in establishing standard therapies are greater than those for major cancers, and effective methods are needed. MASTER KEY Project is a multicenter study based in Japan, with two main parts: prospective registry study and multiple clinical trials. Advanced rare cancers, cancers of unknown primary origin, and those with rare tissue subtypes of common cancers are targeted. The registry study accumulates highly reliable consecutive data that can be used for future drug development. The multiple trials are conducted simultaneously, targeting either a specific biomarker or a rare tumor type of interest. The first interim data set from the registry part presented here shows the prevalence of genetic abnormalities, response rates, survival rates, and clinical trial enrollment rates. From May 2017 to April 2019, 560 patients (mean age = 53) were enrolled in the project. Frequent cancer types included soft tissue sarcomas, neuroendocrine tumors, and central nervous system tumors. Among the 528 patients with assessable data, 69% (364/528) had next-generation sequencing tests, with 48% (176/364) harboring an "actionable" alteration. Seventy-one (13%) patients have been enrolled in one of the clinical trials, with an accrual rate of 3.94 patients/month. A descriptive analysis of biomarker-directed or non-biomarker-directed treatment survival was performed. This project is expected to accelerate development of treatments for rare cancers and show that comprehensive platform trials are an advantageous strategy.

SUBMITTER: Okuma HS

PROVIDER: S-EPMC7484913 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature

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MASTER KEY Project: Powering Clinical Development for Rare Cancers Through a Platform Trial.

Okuma Hitomi S HS Yonemori Kan K Narita Shoko N SN Sukigara Tamie T Hirakawa Akihiro A Shimizu Toshio T Shibata Taro T Kawai Akira A Yamamoto Noboru N Nakamura Kenichi K Nishida Toshiro T Fujiwara Yasuhiro Y

Clinical pharmacology and therapeutics 20200407 3

For rare cancers, challenges in establishing standard therapies are greater than those for major cancers, and effective methods are needed. MASTER KEY Project is a multicenter study based in Japan, with two main parts: prospective registry study and multiple clinical trials. Advanced rare cancers, cancers of unknown primary origin, and those with rare tissue subtypes of common cancers are targeted. The registry study accumulates highly reliable consecutive data that can be used for future drug d ...[more]

PMID: 32112563

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Project description:BackgroundMonitoring and evaluation of prostate cancer (PCa) screening is key to ensure that the programme achieves the desired objectives. Utilizing a set of prioritized, feasible and harmonized key performance indicators (KPIs) is crucial for this purpose. We describe the methodology used to identify the PCa screening KPIs and the outcome of this process within the scope of the EU-funded PRostate cancer Awareness and Initiative for Screening in the EU (PRAISE-U) project. Feasibility of implementing these KPIs will be evaluated in the five pilots set up at multiple sites in the EU (Spain-2 sites, Poland, Ireland, Lithuania).MethodsThe indicators were developed following a structured methodology involving the following steps: (i) Development of a specific conceptual framework for PCa screening is adapted from the existing risk-based algorithms and modified to guide the selection and mapping of indicators (from identification of population eligible for screening to the decision for treatment or follow-up), (ii) Scoping review of literature (coverage from 1971 to June 2023) to identify existing performance indicators for PCa screening to adapt to the new framework with redefining the indicators, where necessary, (iii) Survey among experts (October-November 2023) to select the indicators fulfilling pre-determined criteria such as accuracy of definition and calculation, importance, and feasibility, and (iv) Deliberations among experts to list the finalized set of indicators, held in December 2023.FindingsA total of 63 KPIs were selected for review using the step-wise methodology as described earlier. Following the review, survey, and deliberations, 21 KPIs were finalized to be piloted in the PRAISE-U project. The resulting 21 KPIs cover the different phases of the screening programme, including invitation, screening test, risk stratification, diagnosis, and also on treatment, harms, and impact. Each KPI has been defined with agreed numerator and denominator.InterpretationContinuous monitoring of PCa screening programmes using the KPIs will serve as a powerful tool for optimizing service delivery, programme improvement, comparison per screening site, and ultimately contributing to a better benefit to harm ratio. The KPIs will be implemented in five pilot sites identified to be included in the PRAISE-U project aiming to identify an evidence-based scalable model for risk adapted PCa screening for Europe.FundingThis project has received funding from the EU4Health program under grant agreement 101101217, co-funded by the European Union.

Dataset Information

MASTER KEY Project: Powering Clinical Development for Rare Cancers Through a Platform Trial.

Publications

MASTER KEY Project: Powering Clinical Development for Rare Cancers Through a Platform Trial.

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