Translational Research: Precision Medicine, Personalized Medicine, Targeted Therapies: Marketing or Science?

Marquet, Pierre; Longeray, Pierre-Henry; Ameye, Véronique; Augé, Pascale; Cazeneuve, B; Chatelut, Étienne; Diaz, Isabelle; Diviné, Marine; Froguel, Philippe; Goni, Sylvia; Gueyffier, François; Hoog-Labouret, Natalie; Morin-Surroca, Michèle; Perche, Olivier; Perin-Dureau, Florent; Pigeon, Martine; Tisseau, A.; Verstuyft, Céline

doi:10.2515/therapie/2014231

Cited by 9 publications

(6 citation statements)

References 9 publications

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“…Challenges remain in describing the broader value of precision medicine to the patient at the collective level and shared decision-making at the individual level that incorporates patient individual preferences and personal tradeoffs in value and benefit/risk. 45 For regulatory approval there is a lack of aligned processes for the constituent components of precision medicine, which causes substantial uncertainty in the potential for return on investment. [46][47][48][49][50][51][52] The advent of tests using NGS is likely to compound regulatory approval of precision medicines.…”

Section: Patient-level Perspectivementioning

confidence: 99%

Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group

Faulkner

Holtorf²,

Walton

et al. 2020

Value in Health

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Precision medicine is a dynamic area embracing a diverse and increasing type of approaches that allow the targeting of new medicines, screening programs or preventive healthcare strategies, which include the use of biologic markers or complex tests driven by algorithms also potentially taking account of patient preferences. The International Society for Pharmacoeconomics and Outcome Research expanded its current work around precision medicine to (1) describe the evolving paradigm of precision medicine with examples of current and evolving applications, (2) describe key stakeholders perspectives on the value of precision medicine in their respective domains, and (3) define the core factors that should be considered in a value assessment framework for precision medicine. With the ultimate goal of improving health of well-defined patient groups, precision medicine will affect all stakeholders in the healthcare system at multiple levels spanning the individual perspective to the societal perspective. For an efficient, timely and practical precision medicine value assessment framework, it will be important to address these multiple perspectives through building consensus among the stakeholders for robust procedures and measures of value aspects, including performance of precision mechanism; aligned reimbursement processes of precision mechanism and subsequent treatment; transparent expectations for evidence requirements and study designs adequately matched to the intended use of the precision mechanism and to the smaller target patient populations; recognizing the potential range of value-generation such as ruling-in and ruling-out decisions.

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Section: Patient-level Perspectivementioning

confidence: 99%

Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group

Faulkner

Holtorf²,

Walton

et al. 2020

Value in Health

View full text Add to dashboard Cite

show abstract

“…Therefore, the development of biomarkers and associated therapies require rethinking the design of clinical trials to demonstrate the clinical utility of the biomarker. However, such studies might be difficult to implement if the biomarker identification arrives late in the development process (sometimes not before the phase III clinical trial results), and also due to the ethical issues raised of testing biomarker in negative patients, for a therapy that may not benefit them [ 38 ].…”

Section: Discussionmentioning

confidence: 99%

French Health Technology Assessment of Antineoplastic Drugs Indicated in the Treatment of Solid Tumours: Perspective for Future Trends

et al. 2016

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BackgroundFrance is one of the European countries that spend the most on oncology drugs. To keep pharmaceutical expenditure under control, Health Authorities highly scrutinize market access of costly medicines.ObjectiveTo assess current and future trends in French health technology assessment (HTA) of antineoplastic drugs indicated in the treatment of solid tumours.MethodsA review of the SMR and ASMR drivers of the Transparency Committee (CT) opinions issued for antineoplastic drugs indicated in the treatment of solid tumours and approved between 2009 and 2014 was performed to assess current trends in French health technology assessment (HTA), complemented by an expert board consultation to capture the critical issues on the future of antineoplastic drugs HTA.ResultsThirty-one drugs indicated for the treatment of solid tumours were identified (77 % targeted therapies). Initial CT assessments were available for 26 drugs. Four key items in the CT assessment were identified: 1) Clinical trial methodology; 2) Acceptance of progression-free survival (PFS) as a valuable endpoint; 3) Transferability of clinical trials in clinical practice; 4) Unpredictability of CT decisions. Experts raised the important development of personalised medicines in oncology and key challenges for oncology products to generate information expected from HTA perspective.ConclusionThe French system remains committed to its values and philosophy (access of all innovations for everybody) which are threatened by the increasing launch of innovative therapies and budget constraint. Both HTA decision framework evolution and revision of the current pricing process should be considered in France to cope with these new challenges.Electronic supplementary materialThe online version of this article (doi:10.1007/s11523-015-0411-8) contains supplementary material, which is available to authorized users.

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“…The expectedly high burden of neurological disease together with the substantial genomic heterogeneity of African populations [9,10] offers a unique opportunity to identify and understand other novel genes and molecular pathways, which contribute to the neurobiology of brain disorders. This may lead to new and better detection, prevention and treatment options in people of African ancestry and possibly by extension, other global populations particularly in the new paradigm of personalized precision medicine [11,12]. …”

Section: Introductionmentioning

confidence: 99%

Neurogenomics in Africa: Perspectives, progress, possibilities and priorities

Akinyemi

Owolabi

Oyeniyi

et al. 2016

Journal of the Neurological Sciences

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The understanding of the genetic basis of neurological disorders has grown rapidly in the last two decades. Despite the genomic heterogeneity within African populations, large-scale candidate gene or linkage and exome studies are lacking. However, current knowledge on neurogenetics in African populations is limited and geographically very uneven. Isolated reports indicate the existence of autosomal dominant or recessive conditions incorporating cerebrovascular, movement, neuromuscular, seizure and motor neuron disorders in Africans. In addition, few African families with neurodegenerative disorders associated with dementia have been characterized in North, West and South Africa. The current insurgency in genomic research triggered by among others the Human Health and Heredity (H3) Africa Initiative indicates that there are unique opportunities to advance our knowledge and understanding of the influence of genomic variation on the pattern, presentations and prognosis of neurological disorders in Africa. These have enormous potential to unmask novel genes and molecular pathways germane to the neurobiology of brain disorders. It would facilitate the development of novel diagnostics, preventative and targeted treatments in the new paradigm of precision medicine. Nevertheless, it is crucial to strike a balance between effective traditional public health strategies and personalized genome based care. The translational barriers can be overcome through robust stakeholder engagement and sustainable multilevel, multigenerational and multidisciplinary capacity building and infrastructural development for genomic medicine in Africa.

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Translational Research: Precision Medicine, Personalized Medicine, Targeted Therapies: Marketing or Science?

Cited by 9 publications

References 9 publications

Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group

Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group

French Health Technology Assessment of Antineoplastic Drugs Indicated in the Treatment of Solid Tumours: Perspective for Future Trends

Neurogenomics in Africa: Perspectives, progress, possibilities and priorities

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