Evaluating the Value for Money of Precision Medicine from Early Cycle to Market Access: A Comprehensive Review of Approaches and Challenges

Chen, Wenjia; Wang, Yi; Zemlyanska, Yaroslava; Butani, Dimple; Wong, Chong Boon Nigel; Virabhak, Suchin; Matchar, David B.; Teerawattananon, Yot

doi:10.1016/j.jval.2023.05.001

Cited by 5 publications

(4 citation statements)

References 40 publications

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“…Not all patients or healthcare systems may have the same level of access to the latest precision medicine advancements, potentially exacerbating existing healthcare inequalities. Ensuring that the benefits of precision medicine are accessible to all patients, regardless of socioeconomic status or geographic location, is a crucial ethical consideration. , Moreover, issues related to informed consent and privacy are of paramount concern. Patients often undergo extensive molecular profiling to guide treatment decisions, and this generates a wealth of genetic and medical data.…”

Section: Challenges and Considerations In Precision Medicine Implemen...mentioning

confidence: 99%

Advances in Precision Medicine Approaches for Colorectal Cancer: From Molecular Profiling to Targeted Therapies

Kiran,

Yashaswini,

Maheshwari

et al. 2024

ACS Pharmacol. Transl. Sci.

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Precision medicine is transforming colorectal cancer treatment through the integration of advanced technologies and biomarkers, enhancing personalized and effective disease management. Identification of key driver mutations and molecular profiling have deepened our comprehension of the genetic alterations in colorectal cancer, facilitating targeted therapy and immunotherapy selection. Biomarkers such as microsatellite instability (MSI) and DNA mismatch repair deficiency (dMMR) guide treatment decisions, opening avenues for immunotherapy. Emerging technologies such as liquid biopsies, artificial intelligence, and machine learning promise to revolutionize early detection, monitoring, and treatment selection in precision medicine. Despite these advancements, ethical and regulatory challenges, including equitable access and data privacy, emphasize the importance of responsible implementation. The dynamic nature of colorectal cancer, with its tumor heterogeneity and clonal evolution, underscores the necessity for adaptive and personalized treatment strategies. The future of precision medicine in colorectal cancer lies in its potential to enhance patient care, clinical outcomes, and our understanding of this intricate disease, marked by ongoing evolution in the field. The current reviews focus on providing in-depth knowledge on the various and diverse approaches utilized for precision medicine against colorectal cancer, at both molecular and biochemical levels.

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Section: Challenges and Considerations In Precision Medicine Implemen...mentioning

confidence: 99%

Advances in Precision Medicine Approaches for Colorectal Cancer: From Molecular Profiling to Targeted Therapies

Kiran,

Yashaswini,

Maheshwari

et al. 2024

ACS Pharmacol. Transl. Sci.

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“…Notably, the majority of them (68%) are GTs, with 88% receiving an orphan designation and 58% approved under the PRIME scheme. Nevertheless, the EU does not hold the forefront in GT development, as China and the United States have a higher number of market-approved GTs [42,43].…”

Section: Market Authorization and Reimbursement Challengesmentioning

confidence: 99%

“…While some are categorized as life-saving medications, often intended for children, the majority of these products are formulated to address rare cancers in adults [2,51]. In those cases, the scarcity of survival data poses a significant challenge in determining the appropriate time frame for benefit, particularly given the heightened sensitivity of cost-effectiveness analysis to the applied discount rates [30,42,50].…”

Section: Innovative Reimbursement Modelsmentioning

confidence: 99%

Navigating Gene Therapy Access: The Case of Bulgaria in the Context of the EU Regulatory Landscape

Kostadinov,

Marinova,

Dimitrov

et al. 2024

Healthcare

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Gene therapies (GTs) have recently emerged as revolutionary personalized therapeutic options. Despite their promising potential, challenges such as uncertainty regarding long-term health benefits and safety, along with extreme price tags, pose significant obstacles to patient access. Within the EU, the European Medicines Agency plays a pivotal role with regards to GT market authorization. However, national authorities are responsible for pricing and reimbursement, which results in fragment patient access within the EU. This study aimed to provide an overview of the complex landscape of post-market authorization accessibility for GT products in Bulgaria, comparing it with neighboring EU countries. We applied a mixed-methods approach, including desk research, public data requests, and list price comparisons. As of 1 April 2023, 14 GTs had a valid market authorization at the EU level. In Bulgaria, Kymriah® was the only GT included in the Positive Drug List (PDL), with an official list price of EUR 335,636.94. Similar results were found in Romania, whereas five GTs were included in Greece’s PDL. Additionally, Zolgensma® was found accessible in Bulgaria through an alternative individual access scheme at an estimated price of EUR 1,945,000.00. In conclusion, this study emphasized targeted policy interventions to address health inequalities and to ensure timely access to GTs within the EU.

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“…By integrating CDx, there could be a significant reduction in developmental costs, offering a pathway to enhance accessibility within constrained cancer health systems. Chen W. et al [46] argued that this development in PM demands a reassessment of existing guidelines or the creation of new reference frameworks to effectively navigate the evolving healthcare landscape. The integration of PM introduces complexities beyond mere cost reduction, urging a comprehensive review of decision-making frameworks.…”

Section: (B) Ethical Considerationsmentioning

confidence: 99%

The Buzz Surrounding Precision Medicine: The Imperative of Incorporating It into Evidence-Based Medical Practice

Muharremi,

Meçani,

Muka

2023

JPM

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Precision medicine (PM), through the integration of omics and environmental data, aims to provide a more precise prevention, diagnosis, and treatment of disease. Currently, PM is one of the emerging approaches in modern healthcare and public health, with wide implications for health care delivery, public health policy making formulation, and entrepreneurial endeavors. In spite of its growing popularity and the buzz surrounding it, PM is still in its nascent phase, facing considerable challenges that need to be addressed and resolved for it to attain the acclaim for which it strives. In this article, we discuss some of the current methodological pitfalls of PM, including the use of big data, and provide a perspective on how these challenges can be overcome by bringing PM closer to evidence-based medicine (EBM). Furthermore, to maximize the potential of PM, we present real-world illustrations of how EBM principles can be integrated into a PM approach.

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Evaluating the Value for Money of Precision Medicine from Early Cycle to Market Access: A Comprehensive Review of Approaches and Challenges

Cited by 5 publications

References 40 publications

Advances in Precision Medicine Approaches for Colorectal Cancer: From Molecular Profiling to Targeted Therapies

Advances in Precision Medicine Approaches for Colorectal Cancer: From Molecular Profiling to Targeted Therapies

Navigating Gene Therapy Access: The Case of Bulgaria in the Context of the EU Regulatory Landscape

The Buzz Surrounding Precision Medicine: The Imperative of Incorporating It into Evidence-Based Medical Practice

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