Evaluation of precision medicine assessment reports of the Belgian healthcare payer to inform reimbursement decisions

2020 ◽  
Vol 36 (4) ◽  
pp. 410-417
Author(s):  
Laurenz Govaerts ◽  
Anouk Waeytens ◽  
Walter Van Dyck ◽  
Steven Simoens ◽  
Isabelle Huys
Keyword(s):  
Precision Medicine ◽  
Clinical Utility ◽  
Assessment Tool ◽  
Evaluation Criteria ◽  
Pharmaceutical Product ◽  
Assessment Procedure ◽  
The Novel ◽  
Companion Diagnostics ◽  
Assessment Practice ◽  
Assessment Reports

IntroductionPrecision medicines rely on companion diagnostics to identify patient subgroups eligible for receiving the pharmaceutical product. Until recently, the Belgian public health payer, RIZIV-INAMI, assessed precision medicines and companion diagnostics separately for reimbursement decisions. As both components are considered co-dependent technologies, their assessment should be conducted jointly from a health technology assessment (HTA) perspective. As of July 2019, a novel procedure was implemented accommodating for this joint assessment practice. The aim of this research was to formulate recommendations to improve the assessment in the novel procedure.MethodsThis study evaluated the precision medicine assessment reports of RIZIV-INAMI of the last 5 years under the former assessment procedure. The HTA framework for co-dependent technologies developed by Merlin et al. for the Australian healthcare system was used as a reference standard in this evaluation. Criteria were scored as either present or not present.ResultsThirteen assessment reports were evaluated. Varying scores between reports were obtained for the domain establishing the co-dependent relationship between diagnostic and pharmaceutical. Domains evaluating the clinical utility of the biomarker and the cost-effectiveness performed poorly, whereas the budget impact and the transfer of trial data to the local setting performed well.RecommendationsBased on these results we recommend three amendments for the novel procedure. (i) The implementation of the linked evidence approach when direct evidence of clinical utility is not present, (ii) incorporation of a bias assessment tool, and (iii) further specify guidelines for submission and assessment to decrease the variability of reported evidence between assessment reports.

Avenues to Precision Oncology

2020 ◽  
pp. 36-45
Author(s):  
Alexander Meisel
Keyword(s):  
Precision Medicine ◽  
Trial Design ◽  
Ad Hoc ◽  
Molecular Targets ◽  
Clinical Trial Design ◽  
Predictive Biomarkers ◽  
Precision Oncology ◽  
Companion Diagnostics ◽  
Bona Fide ◽  
The One

Until recently, the clinical management of cancer heavily relied on anatomical and histopathological criteria, with ad hoc guidelines directing the therapeutic choices in specific indications. In the last years, the development and therapeutic implementation of novel anticancer therapies significantly improved the clinical outcome of cancer patients. Nonetheless, such cutting-edge approaches revealed the limitation of the one-size-fits-all paradigm. The newly discovered molecular targets can be exploited either as bona fide targets for subsequent drug development, or as tools to precision medicine, in the form of prognostic and/or predictive biomarkers. This article provides an overview of some of the most recent advances in precision medicine in oncology, with a focus on novel tissue-agnostic anticancer therapies. The definition and implementation of biomarkers and companion diagnostics in clinical trials and clinical practice are also discussed, as well as the changing landscape in clinical trial design.

scholarly journals L-Carnitine and Acylcarnitines: Mitochondrial Biomarkers for Precision Medicine

Metabolites ◽  
2021 ◽  
Vol 11 (1) ◽  
pp. 51
Author(s):  
Marc R. McCann ◽  
Mery Vet George De la Rosa ◽  
Gus R. Rosania ◽  
Kathleen A. Stringer
Keyword(s):  
Precision Medicine ◽  
Inborn Errors Of Metabolism ◽  
Clinical Utility ◽  
Biomarker Discovery ◽  
Genetic Disorders ◽  
Acid Oxidation ◽  
Inborn Errors ◽  
Therapeutic Decisions ◽  
Literature Evidence ◽  
Clinical Biomarker

Biomarker discovery and implementation are at the forefront of the precision medicine movement. Modern advances in the field of metabolomics afford the opportunity to readily identify new metabolite biomarkers across a wide array of disciplines. Many of the metabolites are derived from or directly reflective of mitochondrial metabolism. L-carnitine and acylcarnitines are established mitochondrial biomarkers used to screen neonates for a series of genetic disorders affecting fatty acid oxidation, known as the inborn errors of metabolism. However, L-carnitine and acylcarnitines are not routinely measured beyond this screening, despite the growing evidence that shows their clinical utility outside of these disorders. Measurements of the carnitine pool have been used to identify the disease and prognosticate mortality among disorders such as diabetes, sepsis, cancer, and heart failure, as well as identify subjects experiencing adverse drug reactions from various medications like valproic acid, clofazimine, zidovudine, cisplatin, propofol, and cyclosporine. The aim of this review is to collect and interpret the literature evidence supporting the clinical biomarker application of L-carnitine and acylcarnitines. Further study of these metabolites could ultimately provide mechanistic insights that guide therapeutic decisions and elucidate new pharmacologic targets.

Companion Diagnostics and Precision Medicine for Colorectal Cancer

2019 ◽  
pp. 189-204
Author(s):  
Seung Eun Yu ◽  
Ji Yeon Baek ◽  
Sang Myung Woo ◽  
Kyun Heo ◽  
Byong Chul Yoo
Keyword(s):  
Colorectal Cancer ◽  
Precision Medicine ◽  
Companion Diagnostics

Student Pharmacist Perceptions of the Utility of a Medication Therapy Management-Based, Medication-Related, Falls Risk-Assessment Tool

2020 ◽  
Vol 35 (10) ◽  
pp. 439-445
Author(s):  
Collin M. Clark ◽  
Scott V. Monte ◽  
Mary Hejna ◽  
Robert G. Wahler
Keyword(s):  
Risk Assessment ◽  
Clinical Utility ◽  
Outcome Measure ◽  
Assessment Tool ◽  
Medication Therapy Management ◽  
Risk Assessment Tool ◽  
Patient Acceptance ◽  
Main Outcome Measure ◽  
Therapy Management ◽  
Medication Therapy

OBJECTIVE: To evaluate student pharmacists' perceptions of the Medication Fall Risk-Assessment Tool (MFRAT), a novel medication therapy management (MTM)-based clinical decision support tool.<br/> DESIGN: A cross-sectional study<br/> SETTING: One school of pharmacy<br/> PARTICIPANTS: Participants were eligible if they had used the MFRAT as part of a wellness clinic or as part of required coursework prior to April 2016.<br/> INTERVENTIONS: An online, author developed survey was distributed to assess previous MFRAT use, perceptions of the tool on a 5-point Likert-type scale, and qualitative feedback related in the areas of pharmacy workflow, clinical utility, and perceived patient acceptance.<br/> MAIN OUTCOME MEASURE(S): Positive agreement to survey items served as the main outcome measure. A chi-square test was used to test for a difference in positive response between naive and experienced MFRAT users. Cronbach's alpha was calculated to assess internal consistency of items in each domain.<br/> RESULTS: Of the 127 potential participants surveyed, 110 completed the survey for an overall response rate of 87%. Greater than 85% of participants found the MFRAT's report clinically useful. Eighty-seven percent of respondents agreed or strongly agreed that the information provided by the tool could easily be understood by patients. The most significant limitation to the use if the MFRAT in existing workflow was time required to enter a patient's medication profile.<br/> CONCLUSION: The MFRAT was viewed positively in terms of clinical utility and perceived patient acceptance. Improvements will need to be made to make using the tool more time efficient in pharmacy MTM workflow.

Student Pharmacist Perceptions of the Utility of a Medication Therapy Management-Based, Medication-Related, Falls Risk-Assessment Tool

2020 ◽  
Vol 35 (10) ◽  
pp. 439-445
Author(s):  
Collin M. Clark ◽  
Scott V. Monte ◽  
Mary Hejna ◽  
Robert G. Wahler
Keyword(s):  
Risk Assessment ◽  
Clinical Utility ◽  
Outcome Measure ◽  
Assessment Tool ◽  
Medication Therapy Management ◽  
Risk Assessment Tool ◽  
Patient Acceptance ◽  
Main Outcome Measure ◽  
Therapy Management ◽  
Medication Therapy

OBJECTIVE: To evaluate student pharmacists' perceptions of the Medication Fall Risk-Assessment Tool (MFRAT), a novel medication therapy management (MTM)-based clinical decision support tool.<br/> DESIGN: A cross-sectional study<br/> SETTING: One school of pharmacy<br/> PARTICIPANTS: Participants were eligible if they had used the MFRAT as part of a wellness clinic or as part of required coursework prior to April 2016.<br/> INTERVENTIONS: An online, author developed survey was distributed to assess previous MFRAT use, perceptions of the tool on a 5-point Likert-type scale, and qualitative feedback related in the areas of pharmacy workflow, clinical utility, and perceived patient acceptance.<br/> MAIN OUTCOME MEASURE(S): Positive agreement to survey items served as the main outcome measure. A chi-square test was used to test for a difference in positive response between naive and experienced MFRAT users. Cronbach's alpha was calculated to assess internal consistency of items in each domain.<br/> RESULTS: Of the 127 potential participants surveyed, 110 completed the survey for an overall response rate of 87%. Greater than 85% of participants found the MFRAT's report clinically useful. Eighty-seven percent of respondents agreed or strongly agreed that the information provided by the tool could easily be understood by patients. The most significant limitation to the use if the MFRAT in existing workflow was time required to enter a patient's medication profile.<br/> CONCLUSION: The MFRAT was viewed positively in terms of clinical utility and perceived patient acceptance. Improvements will need to be made to make using the tool more time efficient in pharmacy MTM workflow.

Habituation of Visual Fixation Responses: An Assessment Tool to Measure Visual Sensory-Perceptual Cognitive Processes in Nonverbal Profoundly Handicapped Children in the Classroom

AAESPH Review ◽  
1979 ◽  
Vol 4 (2) ◽  
pp. 136-147 ◽  
Author(s):  
Harvey N. Switzky ◽  
Janet Woolsey-Hill ◽  
Therese Quoss
Keyword(s):  
Assessment Tool ◽  
Visual Stimuli ◽  
Visual Fixation ◽  
Special Education Teacher ◽  
Fixation Time ◽  
The Novel ◽  
Black And White ◽  
Educational Implications ◽  
Processing Information ◽  
Novel Target

Twelve profoundly retarded nonverbal, nonambulatory children were repeatedly exposed to one of two visual stimuli-a 2 times 2 or a 12 times 12 black-and-white checkerboard target-until a set criterion of habituation was demonstrated, as measured by a decrement in visual fixation time. When the habituation criterion was reached, the children were shown alternative presentations of the same and a novel target. Results showed an increase in visual fixation to the novel target. A control condition was instituted also; so that when the habituation criterion was reached, the children were shown only presentations of the same target. Results showed no increase in visual fixation to the same targets. Together these results suggest that profoundly retarded children do show habituation and dishabituation to visual stimuli, and are actively storing and processing information about their perceptual world. The educational implications of the habituation paradigm for the special education teacher in the classroom are discussed.

scholarly journals Development of Mobile Application for Sustainable Creative Tourism Assessment using Confirmatory Factor Analysis Approach

2019 ◽  
Vol 13 (06) ◽  
pp. 27
Author(s):  
Chitralada Suphachaimongkol ◽  
Chavalit Ratanatamskul ◽  
Siriwan Silapacharanan ◽  
Patcha Utiswannakul
Keyword(s):  
Factor Analysis ◽  
Confirmatory Factor Analysis ◽  
Mobile Application ◽  
Assessment Tool ◽  
Model Development ◽  
Development Stage ◽  
The Novel ◽  
Confirmatory Factor ◽  
Tourism Enterprises ◽  
Delphi Techniques

<span>Creative tourism has received increasing attention. At present, the concept of creative tourism is still in development stage since there are few or no indicators for assessing the potential of creative tourism to area-based sustainable goals. This document aims to introduce the process of mobile application for sustainable creative tourism (SCT) model development using confirmatory factor analysis (CFA) approach. The study was divided into 4 phases. The first phase involved establishing elements, indicators and criteria drawn from some previous studies and the opinions of experts’ judgement in this study. Secondly, indicators and criteria were selected by using Delphi technique. Thirdly, the indicators and criterias were confirmed by utilizing CFA, Finally, the prototype of SCT mobile application assessment tool is proposed for SCT in this study, based on CFA. Tools for the study included: 1) structured questionnaire from reviews of literature for interviewing the expert, 2) questionnaire for Delphi techniques among the experts for collecting an opinion about the selection of the indicators, and 3) a questionnaire to assess the possibility of data collection concerning the components and indicators of SCT. 300 samples from creative tourism enterprises from all around Thailand were collected and 253 were responded. Data analysis was conducted by using descriptive statistics and CFA. The study found that SCT model consists of 8 components and 36 indicators. The finding from our research shows that it is eligible to establish the novel SCT assessment indicators. The result of SCT application model is appropriate to develop mobile application for SCT assessment tool. </span>

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