Harnessing Real-World Evidence for the Development of Novel Cancer Therapies

Greshock, Joel; Lewi, Martine; Hartog, Bert; Tendler, Craig

doi:10.1016/j.trecan.2020.08.006

Cited by 9 publications

(8 citation statements)

References 13 publications

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“…Nonetheless, challenges in patient consent and data sharing may influence access to real-world patient-level health outcomes data for research, but guidance is available to incorporate such evidence into HTA for oncology and precision medicine. 61,62 These technical issues are consistent with previous research commenting on the complexities in the design of economic evaluations of precision medicine (albeit not hematological malignancy specific). 14,[63][64][65][66][67][68][69][70] This review has recognized that the limited number of health economic studies in recent years is concerning, but there is another layer of complexity to the conduct of health economic research for HTA purposes.…”

Section: Discussionsupporting

confidence: 84%

“…This is a possible future direction for this field of clinical molecular biomarkers in blood cancers. Nonetheless, challenges in patient consent and data sharing may influence access to real‐world patient‐level health outcomes data for research, but guidance is available to incorporate such evidence into HTA for oncology and precision medicine 61,62 14,63‐70 …”

Section: Discussionmentioning

confidence: 99%

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Health economic evidence for the use of molecular biomarker tests in hematological malignancies: A systematic review

Degeling

Thompson

et al. 2022

European J of Haematology

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Objectives Molecular biomarker tests can inform the clinical management of genomic heterogeneous hematological malignancies, yet their availability in routine care largely depends on the supporting health economic evidence. This study aims to systematically review the economic evidence for recent molecular biomarker tests in hematological malignancies. Methods We conducted a systematic search in five electronic databases for studies published between January 2010 and October 2020. Publications were independently screened by two reviewers. Clinical study characteristics, economic methodology, and results were extracted, and reporting quality was assessed. Results Fourteen studies were identified, of which half (n = 7; 50%) were full economic evaluations examining both health and economic outcomes. Studies were predominantly conducted in a first‐line treatment setting (n = 7; 50%) and adopted a non‐lifetime time horizon to measure health outcomes and costs (n = 7; 50%). Five studies reported that companion diagnostics for associated therapies were likely cost‐effective for acute myeloid leukemia, chronic myeloid leukemia, diffuse large B‐cell lymphoma, and multiple myeloma. Four studies suggested molecular biomarker tests for treatment monitoring in chronic myeloid leukemia were likely cost‐saving. Conclusions Although there is initial confirmation of the promising health economic results, the present research for molecular biomarker tests in hematological malignancies is sparse with many applications of technological advances yet to be evaluated.

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Section: Discussionsupporting

confidence: 84%

Section: Discussionmentioning

confidence: 99%

Health economic evidence for the use of molecular biomarker tests in hematological malignancies: A systematic review

Degeling

Thompson

et al. 2022

European J of Haematology

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“…Furthermore, high-quality real-world evidence (RWE) could be leveraged to enable drug approvals in oncology (34,35), linking it to the value proposition of drugs (36)(37)(38). Regulatory bodies, such as the US Food and Drug Administration, have recently shown a willingness to expedite access to new cancer medicines by using RWE (39).…”

Section: Discussionmentioning

confidence: 99%

Correlation Between Early Endpoints and Overall Survival in Non-Small-Cell Lung Cancer: A Trial-Level Meta-Analysis

et al. 2021

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Early endpoints, such as progression-free survival (PFS), are increasingly used as surrogates for overall survival (OS) to accelerate approval of novel oncology agents. Compiling trial-level data from randomized controlled trials (RCTs) could help to develop a predictive framework to ascertain correlation trends between treatment effects for early and late endpoints. Through trial-level correlation and random-effects meta-regression analysis, we assessed the relationship between hazard ratio (HR) OS and (1) HR PFS and (2) odds ratio (OR) PFS at 4 and 6 months, stratified according to the mechanism of action of the investigational product. Using multiple source databases, we compiled a data set including 81 phase II–IV RCTs (35 drugs and 156 observations) of patients with non-small-cell lung cancer. Low-to-moderate correlations were generally observed between treatment effects for early endpoints (based on PFS) and HR OS across trials of agents with different mechanisms of action. Moderate correlations were seen between treatment effects for HR PFS and HR OS across all trials, and in the programmed cell death-1/programmed cell death ligand-1 and epidermal growth factor receptor trial subsets. Although these results constitute an important step, caution is advised, as there are some limitations to our evaluation, and an additional patient-level analysis would be needed to establish true surrogacy.

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“…Facing this statistical catastrophe through regular prevention-focused approaches and early intervention programs falls short of patients’ expectations, who demand therapeutic alternatives to be affordable, side-effect discriminated, and more efficient and effective. Among the existing cancer treatments are surgery, chemotherapy, radiation therapy, targeted therapy, immunotherapy, laser treatment, stem cell transplant, hyperthermia, and small molecule-based therapy, being those of immunosuppressive-type most commonly used [ 2 , 3 ]. However, although chemotherapy and ionizing radiation can destroy malignant growths at sufficient drug/radiation doses, they are noxious to the bone barrow and provoke immunostimulatory effects such as induction of heat-shock proteins [ 4 ].…”

Section: Introductionmentioning

confidence: 99%

Photodynamic Therapeutic Effect of Nanostructured Metal Sulfide Photosensitizers on Cancer Treatment

et al. 2022

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Photodynamic therapy (PDT) utilizes photosensitizers (PSs) to produce reactive oxygen species (ROSs) upon irradiation, which causes the shutdown of vessels and deprives the tumor of nutrients and oxygen, and in turn induces adverse effects on the immune system. However, significant efforts are needed to increase the efficiency in PDT in terms of light delivery to specific PSs for the clinical treatment of tumors located deep under the skin. Even though PDT offers a disease site-specific treatment modality, current efforts are directed to improve the solubility (in body fluids and injectable solvents), photostability, amphiphilicity (for tissue penetration), elimination, and systemic toxicity of traditional PSs based on porphyrin derivatives. Nanostructured materials show promising features to achieve most of such combined efforts. They can be artificially engineered to carry multiple theranostic agents onto targeted tumor sites. However, recent studies on photosensitive Cd-based nanostructures, mostly used in PDT, indicate that leeching of Cd2+ ions is stimulated when they are exposed to harsh biological conditions for continuous periods of time, thus making them acutely toxic and hindering their applications in in vivo settings. Since nanostructured materials are not completely immune to degradation, great strides have been made to seek new alternatives. In this review, we focus on the latest advances of Cd-free nanostructured metal transition sulfides (MTSs) as alternative PSs and study their high-energy transfer efficiency, rational designs, and potential applications in cancer-targeted PDT. Nanostructured MTSs are discussed in the context of their versatility to serve as phototherapy agents and superior properties, including their strong absorption in the NIR region, excellent photothermal conversion efficiency, controlled reactive oxygen species (ROS) production, versatile surface chemistry, high fluorescence, and structural and thermal stability. We discuss the latest advancements in correlating the self-aggregation of MTSs with their passive tumor cell targeting, highlighting their ability to efficiently produce ROSs, and mitigating their dark toxicity through polymeric functionalization. Treatment of deep-seated tumors by using these PSs upon preferential uptake by tumor tissues (due to the enhanced permeability and retention effect) is also reviewed. We finally summarize the main future perspectives of MTSs as next-generation PSs within the context of cancer theranostics. Graphical Abstract

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Harnessing Real-World Evidence for the Development of Novel Cancer Therapies

Cited by 9 publications

References 13 publications

Health economic evidence for the use of molecular biomarker tests in hematological malignancies: A systematic review

Health economic evidence for the use of molecular biomarker tests in hematological malignancies: A systematic review

Correlation Between Early Endpoints and Overall Survival in Non-Small-Cell Lung Cancer: A Trial-Level Meta-Analysis

Photodynamic Therapeutic Effect of Nanostructured Metal Sulfide Photosensitizers on Cancer Treatment

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