The Future of Clinical Trial Design in Oncology

Spreafico, Anna; Hansen, Aaron Richard; Razak, Albiruni R. Abdul; Bedard, Philippe L.; Siu, Lillian L.

doi:10.1158/2159-8290.cd-20-1301

Cited by 51 publications

(44 citation statements)

References 70 publications

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“…Demand will increase for innovative trial designs to determine these technologies' effectiveness or efficacy and safety. The demanded innovation will require that oncology trials be pragmatic to reflect routine care settings, but also adaptive to identify treatment effect heterogeneity for varied genetic subtypes to realize the promise the of personalized medicine (molecular targeted agents and immunotherapies) paradigm in oncology [63,109,110]. It will be particularly impactful for future oncology trials to fully incorporate continuous and meaningful patient and stakeholder engagement in a manner that can be scientifically evaluated to help address the barriers to trial participation to improve diversity and participation in trials [5,38,50].…”

Section: Discussionmentioning

confidence: 99%

“…With the growing discovery of genetic diversities in cancer types due to advances in diagnostic technology, more adaptive oncology trials are needed to discover and develop effective medicines for diverse oncology patients. The next generation of adaptive oncology trials require timely access to funding and underscores the need for meaningful patient engagement in these trials [20].…”

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confidence: 99%

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Pragmatic Patient Engagement in Designing Pragmatic Oncology Clinical Trials

et al. 2021

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Oncology trials are the cornerstone of effective and safe therapeutic discoveries. However, there is increasing demand for pragmatism and patient engagement in the design, implementation and dissemination of oncology trials. Many researchers are uncertain about making trials more practical and even less knowledgeable about how to meaningfully engage patients without compromising scientific rigor to meet regulatory requirements. The present work provides practical guidance for addressing both pragmaticism and meaningful patient engagement. Applying evidence-based approaches like PRECIS-2-tool and the 10-Step Engagement Framework offer practical guidance to make future trials in oncology truly pragmatic and patient-centered. Consequently, such patient-centered trials have improved participation, faster recruitment and greater retention, and uptake of innovative technologies in community-based care.

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Section: Discussionmentioning

confidence: 99%

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confidence: 99%

Pragmatic Patient Engagement in Designing Pragmatic Oncology Clinical Trials

et al. 2021

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“…Therefore, there is heightened need for alternative sources of evidence in such populations, for which real-world evidence may provide an opportunity. Data sharing and collaboration through multi-center registries and trial networks are potential avenues to integrate supportive real-world evidence to clinical trial data ( 44 ). Particularly given the relative rarity of these subpopulations, pooled analyses may represent efficient methods of generating high quality prospective data.…”

Section: Alternative Sources Of Data Such As Real-world Evidencementioning

confidence: 99%

Clinical Trial Eligibility Criteria and Recently Approved Cancer Therapies for Patients With Brain Metastases

et al. 2022

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Brain metastases cause significant morbidity and mortality in patients with advanced cancer. In the era of precision oncology and immunotherapy, there are rapidly evolving systemic treatment options. These novel therapies may have variable intracranial efficacy, and patients with brain metastases remain a population of special interest. Typically, only patients with stable, asymptomatic and/or treated brain metastases are enrolled in clinical trials, or may be excluded altogether, particularly in the setting of leptomeningeal carcinomatosis. Consequently, this leads to significant concerns on the external validity of clinical trial evidence to real-world clinical practice. Here we describe the current trends in cancer clinical trial eligibility for patients with brain metastases in both early and late phase trials, with a focus on targeted and immunotherapies. We evaluate recent newly FDA approved therapies and the clinical trial evidence base leading to approval. This includes analysis of inclusion and exclusion criteria, requirements for baseline screening for brain metastases, surveillance cerebral imaging and incorporation of trial endpoints for patients with brain metastases. Finally, the use of alternative sources of data such as real-world evidence with registries and collaborative studies will be discussed.

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“…general practitioners, local hospitals) in data acquisition, remote delivery of trial medication, and at-home infusion of medicine. 22 Besides increasing willingness of patients to participate in clinical trials, increasing the scope of clinical trials to a continental or global level will facilitate inclusion rates as well. As an additional benefit, results of clinical trialswhich are based on results from a highly selected patient populationmay become more generalizable, and will predict real-world outcome more accurately.…”

Section: Excludementioning

confidence: 99%

The wider perspective: twenty years of clinical trials in myelodysplastic syndromes

et al. 2021

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Summary Most patients with myelodysplastic syndromes (MDS) require therapeutic intervention. However, there are few approved treatments for MDS. To explore reasons, we searched clinicaltrials.gov and clinicaltrialsregister.eu for MDS trials from 2000 to 2020. We assessed which agents were under investigation and analysed clinical trial characteristics and continuation rates from phase I to II to III to approval. As such, we identified 384 unique agents in 426 phase I, 430 phase II and 48 phase III trials. Success rates for phase III trials and agents were low, and MDS trials took markedly longer to complete than the average clinical trial. Although success rates were higher when MDS‐specific phase I trials were conducted, 52% of the agents had not been evaluated in a phase I trial for MDS. MDS trials often failed to include quality of life, an especially important outcome for older MDS patients. Our work identifies factors potentially contributing to the paucity of available agents for MDS. We suggest a framework to improve clinical research in MDS that might ultimately augment the number of available agents.

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The Future of Clinical Trial Design in Oncology

Cited by 51 publications

References 70 publications

Pragmatic Patient Engagement in Designing Pragmatic Oncology Clinical Trials

Pragmatic Patient Engagement in Designing Pragmatic Oncology Clinical Trials

Clinical Trial Eligibility Criteria and Recently Approved Cancer Therapies for Patients With Brain Metastases

The wider perspective: twenty years of clinical trials in myelodysplastic syndromes

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