The economic case for precision medicine

Gavan, Sean P; Thompson, Alexander; Payne, Katherine

doi:10.1080/23808993.2018.1421858

Cited by 68 publications

(52 citation statements)

References 89 publications

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“…Early consideration of the evidence required by decision makers can improve evidence collection and analysis for precision medicine technologies and services in very early stages of development [ 77 ]. Innovative approaches for evidence generation to facilitate this are currently being developed: new trial designs [ 55 ] and robust statistical methods for analysing observational data [ 78 ] will help fill evidence gaps and improve trial recruitment numbers.…”

Section: Discussionmentioning

confidence: 99%

The Future of Precision Medicine: Potential Impacts for Health Technology Assessment

et al. 2018

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ObjectivePrecision medicine allows healthcare interventions to be tailored to groups of patients based on their disease susceptibility, diagnostic or prognostic information, or treatment response. We analysed what developments are expected in precision medicine over the next decade and considered the implications for health technology assessment (HTA) agencies.MethodsWe performed a pragmatic literature search to account for the large size and wide scope of the precision medicine literature. We refined and enriched these results with a series of expert interviews up to 1 h in length, including representatives from HTA agencies, research councils and researchers designed to cover a wide spectrum of precision medicine applications and research.ResultsWe identified 31 relevant papers and interviewed 13 experts. We found that three types of precision medicine are expected to emerge in clinical practice: complex algorithms, digital health applications and ‘omics’-based tests. These are expected to impact upon each stage of the HTA process, from scoping and modelling through to decision-making and review. The complex and uncertain treatment pathways associated with patient stratification and fast-paced technological innovation are central to these effects.DiscussionInnovation in precision medicine promises substantial benefits but will change the way in which some health services are delivered and evaluated. The shelf life of guidance may decrease, structural uncertainty may increase and new equity considerations will emerge. As biomarker discovery accelerates and artificial intelligence-based technologies emerge, refinements to the methods and processes of evidence assessments will help to adapt and maintain the objective of investing in healthcare that is value for money.Electronic supplementary materialThe online version of this article (10.1007/s40273-018-0686-6) contains supplementary material, which is available to authorized users.

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Section: Discussionmentioning

confidence: 99%

The Future of Precision Medicine: Potential Impacts for Health Technology Assessment

et al. 2018

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“…Based on this profile there is convincing evidence that biomarkers, pharmacogenetic tests and specific targeted therapy significantly reduce visits to the emergency room and the requirement for hospitalized care. 31 , 32 In the same way, the improvement in quality of life has a transcendental effect on the therapeutic course of infrequent diseases with limited alternative therapies that have not been very effective up until now. This is particularly the case in developing nations, where access to targeted therapies is not widespread, possibly due to economic conditions, limitations on coverage, and physical accessibility.…”

Section: The Importance Of Precision Medicine In Countries With Limitmentioning

confidence: 99%

Precision medicine and its implementation in patients with NTRK fusion genes: perspective from developing countries

Zorrilla

Arrieta

Ruíz-Patiño

et al. 2020

Ther Adv Respir Dis

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Precision oncology is the field that places emphasis on the diagnosis and treatment of tumors that harbor specific genomic alterations susceptible to inhibition or modulation. Although most alterations are only present in a minority of patients, a substantial effect on survival can be observed in this subgroup. Mass genome sequencing has led to the identification of a specific driver in the translocations of the tropomyosin receptor kinase family (NTRK) in a subset of rare tumors both in children and in adults, and to the development and investigation of Larotrectinib. This medication was granted approval by the US Food and Drug Administration for NTRK-positive tumors, regardless of histology or age group, as such, larotrectinib was the first in its kind to be approved under the premise that molecular pattern is more important than histology in terms of therapeutic approach. It yielded significant results in disease control with good tolerability across a wide range of diseases including rare pediatric tumors, salivary gland tumors, gliomas, soft-tissue sarcomas, and thyroid carcinomas. In addition, and by taking different approaches in clinical trial design and conducting allocation based on biomarkers, the effects of target therapies can be isolated and quantified. Moreover, and considering developing nations and resource-limited settings, precision oncology could offer a tool to reduce cancer-related disability and hospital costs. In addition, developing nations also present patients with rare tumors that lack a chance of treatment, outside of clinical trials. This, in turn, offers the possibility for international collaboration, and contributes to employment, education, and health service provisions. The reviews of this paper are available via the supplemental material section.

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“…The application of precision medicine to cardiovascular diseases, as in many other diseases linked to high mortality risk in the population, holds the promise of improving health as well as revolutionizing prevention and treatment options, similar to what has occurred in the field of oncology. The clinical translation and application of precision medicine can be achieved by developing the personalized treatment of patients based on the premise that prevention is better than treating [16]. Furthermore, we also want to outline the difference between personalized medicine (PeM) and precision medicine (PM) into managing these complex chronic disorders by demonstrating the potential benefits of precision medicine novel technologies demonstrated to date in order to offer insights into routine clinical practice.…”

Section: Introductionmentioning

confidence: 99%

Precision and Personalized Medicine: How Genomic Approach Improves the Management of Cardiovascular and Neurodegenerative Disease

Strianese

Rizzo

Ciccarelli

et al. 2020

Genes

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Life expectancy has gradually grown over the last century. This has deeply affected healthcare costs, since the growth of an aging population is correlated to the increasing burden of chronic diseases. This represents the interesting challenge of how to manage patients with chronic diseases in order to improve health care budgets. Effective primary prevention could represent a promising route. To this end, precision, together with personalized medicine, are useful instruments in order to investigate pathological processes before the appearance of clinical symptoms and to guide physicians to choose a targeted therapy to manage the patient. Cardiovascular and neurodegenerative diseases represent suitable models for taking full advantage of precision medicine technologies applied to all stages of disease development. The availability of high technology incorporating artificial intelligence and advancement progress made in the field of biomedical research have been substantial to understand how genes, epigenetic modifications, aging, nutrition, drugs, microbiome and other environmental factors can impact health and chronic disorders. The aim of the present review is to address how precision and personalized medicine can bring greater clarity to the clinical and biological complexity of these types of disorders associated with high mortality, involving tremendous health care costs, by describing in detail the methods that can be applied. This might offer precious tools for preventive strategies and possible clues on the evolution of the disease and could help in predicting morbidity, mortality and detecting chronic disease indicators much earlier in the disease course. This, of course, will have a major effect on both improving the quality of care and quality of life of the patients and reducing time efforts and healthcare costs.

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The economic case for precision medicine

Cited by 68 publications

References 89 publications

The Future of Precision Medicine: Potential Impacts for Health Technology Assessment

The Future of Precision Medicine: Potential Impacts for Health Technology Assessment

Precision medicine and its implementation in patients with NTRK fusion genes: perspective from developing countries

Precision and Personalized Medicine: How Genomic Approach Improves the Management of Cardiovascular and Neurodegenerative Disease

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