scholarly journals Pragmatic pharmacoeconomic analyses by using post-market adverse drug reaction reports: an illustration using infliximab, adalimumab, and the Canada vigilance adverse reaction database

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Tuhin Maity ◽  
Christopher Longo
Keyword(s):  
Adverse Reaction ◽  
Real World ◽  
Adverse Reactions ◽  
Pharmaceutical Products ◽  
Controlled Trials ◽  
Market Data ◽  
The Real ◽  
Randomized Controlled ◽  
Post Market ◽  
The Cost

Abstract Background The prediction of the real-world cost of adverse drug reactions (ADRs) has historically relied on the data from randomized controlled trials (RCT). However, trial conditions do not always reflect the real-world applications of pharmaceutical products; hence, they may not accurately portray the actual risks of ADRs associated with them. The objective of this study is two-fold: (a) demonstrate whether and how post-market and RCT ADR data could lead to different conclusions for a set of drugs of interest, and (b) evaluate the potential economic impact of the post-market ADRs associated with those drugs. Methods We selected two TNF-α inhibitor biologics, infliximab and adalimumab, and used the Canada Vigilance Adverse Reaction (CVAR) online database as a source of post-market ADR data. Adverse reaction data from RCTs were obtained from ClinicalTrials.gov. Direct healthcare costs associated with adverse reactions were obtained from Canadian Institute for Health Information (CIHI) or Interactive Health Data Application, Alberta. We calculated post-market ADR rates and compared them with those found in the randomized controlled trials of these two drugs. Using the post-market data, we estimated the costs associated with serious ADRs from three perspectives: patient, health system, and societal. Results For both drugs, the post-market and RCT data exhibited significantly different adverse reaction rates for several different clinical outcomes. As a general trend, more serious adverse reactions, such as death, appeared to have a higher rate in post-market applications compared to the clinical trials. The estimated average annual economic burden of the severe adverse reaction outcomes ranged from $10 million to $20 million for infliximab and $6 million to $19 million for adalimumab. Conclusions The frequency and severity of post-market adverse reactions associated with pharmaceutical products may significantly differ from those detected in the clinical trials. Despite possible methodological differences, this is due to the fact that post-market data reflect the externalities of the real-world that are absent in RCTs. The economic burden of adverse reactions can be substantial, and the cost calculated using post-market data is better reflective of the cost of ADRs in the real-world.

scholarly journals Identification of Patient Perceptions That Can Affect the Uptake of Interventions Using Biometric Monitoring Devices: Systematic Review of Randomized Controlled Trials (Preprint)

2020 ◽  
Author(s):  
Alexander Perlmutter ◽  
Mehdi Benchoufi ◽  
Philippe Ravaud ◽  
Viet-Thi Tran
Keyword(s):  
Randomized Controlled Trials ◽  
Real World ◽  
Glucose Monitoring ◽  
Median Number ◽  
Patient Perceptions ◽  
Controlled Trials ◽  
Data Handling ◽  
The Real ◽  
Randomized Controlled ◽  
Monitoring Devices

BACKGROUND Biometric monitoring devices (BMDs) are wearable or environmental trackers and devices with embedded sensors that can remotely collect high-frequency objective data on patients’ physiological, biological, behavioral, and environmental contexts (for example, fitness trackers with accelerometer). The real-world effectiveness of interventions using biometric monitoring devices depends on patients’ perceptions of these interventions. OBJECTIVE We aimed to systematically review whether and how recent randomized controlled trials (RCTs) evaluating interventions using BMDs assessed patients’ perceptions toward the intervention. METHODS We systematically searched PubMed (MEDLINE) from January 1, 2017, to December 31, 2018, for RCTs evaluating interventions using BMDs. Two independent investigators extracted the following information: (1) whether the RCT collected information on patient perceptions toward the intervention using BMDs and (2) if so, what precisely was collected, based on items from questionnaires used and/or themes and subthemes identified from qualitative assessments. The two investigators then synthesized their findings in a schema of patient perceptions of interventions using BMDs. RESULTS A total of 58 RCTs including 10,071 participants were included in the review (the median number of randomized participants was 60, IQR 37-133). BMDs used in interventions were accelerometers/pedometers (n=35, 60%), electrochemical biosensors (eg, continuous glucose monitoring; n=18, 31%), or ecological momentary assessment devices (eg, carbon monoxide monitors for smoking cessation; n=5, 9%). Overall, 26 (45%) trials collected information on patient perceptions toward the intervention using BMDs and allowed the identification of 76 unique aspects of patient perceptions that could affect the uptake of these interventions (eg, relevance of the information provided, alarm burden, privacy and data handling, impact on health outcomes, independence, interference with daily life). Patient perceptions were unevenly collected in trials. For example, only 5% (n=3) of trials assessed how patients felt about privacy and data handling aspects of the intervention using BMDs. CONCLUSIONS Our review showed that less than half of RCTs evaluating interventions using BMDs assessed patients’ perceptions toward interventions using BMDs. Trials that did assess perceptions often only assessed a fraction of them. This limits the extrapolation of the results of these RCTs to the real world. We thus provide a comprehensive schema of aspects of patient perceptions that may affect the uptake of interventions using BMDs and which should be considered in future trials. CLINICALTRIAL PROSPERO CRD42018115522; https://tinyurl.com/y5h8fjgx

scholarly journals Identification of Patient Perceptions That Can Affect the Uptake of Interventions Using Biometric Monitoring Devices: Systematic Review of Randomized Controlled Trials

10.2196/18986 ◽  
2020 ◽  
Vol 22 (9) ◽  
pp. e18986
Author(s):  
Alexander Perlmutter ◽  
Mehdi Benchoufi ◽  
Philippe Ravaud ◽  
Viet-Thi Tran
Keyword(s):  
Randomized Controlled Trials ◽  
Real World ◽  
Glucose Monitoring ◽  
Median Number ◽  
Patient Perceptions ◽  
Controlled Trials ◽  
Data Handling ◽  
The Real ◽  
Randomized Controlled ◽  
Monitoring Devices

Background Biometric monitoring devices (BMDs) are wearable or environmental trackers and devices with embedded sensors that can remotely collect high-frequency objective data on patients’ physiological, biological, behavioral, and environmental contexts (for example, fitness trackers with accelerometer). The real-world effectiveness of interventions using biometric monitoring devices depends on patients’ perceptions of these interventions. Objective We aimed to systematically review whether and how recent randomized controlled trials (RCTs) evaluating interventions using BMDs assessed patients’ perceptions toward the intervention. Methods We systematically searched PubMed (MEDLINE) from January 1, 2017, to December 31, 2018, for RCTs evaluating interventions using BMDs. Two independent investigators extracted the following information: (1) whether the RCT collected information on patient perceptions toward the intervention using BMDs and (2) if so, what precisely was collected, based on items from questionnaires used and/or themes and subthemes identified from qualitative assessments. The two investigators then synthesized their findings in a schema of patient perceptions of interventions using BMDs. Results A total of 58 RCTs including 10,071 participants were included in the review (the median number of randomized participants was 60, IQR 37-133). BMDs used in interventions were accelerometers/pedometers (n=35, 60%), electrochemical biosensors (eg, continuous glucose monitoring; n=18, 31%), or ecological momentary assessment devices (eg, carbon monoxide monitors for smoking cessation; n=5, 9%). Overall, 26 (45%) trials collected information on patient perceptions toward the intervention using BMDs and allowed the identification of 76 unique aspects of patient perceptions that could affect the uptake of these interventions (eg, relevance of the information provided, alarm burden, privacy and data handling, impact on health outcomes, independence, interference with daily life). Patient perceptions were unevenly collected in trials. For example, only 5% (n=3) of trials assessed how patients felt about privacy and data handling aspects of the intervention using BMDs. Conclusions Our review showed that less than half of RCTs evaluating interventions using BMDs assessed patients’ perceptions toward interventions using BMDs. Trials that did assess perceptions often only assessed a fraction of them. This limits the extrapolation of the results of these RCTs to the real world. We thus provide a comprehensive schema of aspects of patient perceptions that may affect the uptake of interventions using BMDs and which should be considered in future trials. Trial Registration PROSPERO CRD42018115522; https://tinyurl.com/y5h8fjgx

The Cost-Effectiveness of Pre-school Peanut Oral Immunotherapy in the Real World Setting

2021 ◽  
Author(s):  
Marcus Shaker ◽  
Edmond S. Chan ◽  
Jennifer LP. Protudjer ◽  
Lianne Soller ◽  
Elissa M. Abrams ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Real World ◽  
Oral Immunotherapy ◽  
The Real ◽  
Real World Setting ◽  
The Cost

PP469 From Theory To Practice: Which Value Framework Is Applied For Onco-Hematology Therapies In Italy? A 5-year Retrospective Analysis

2020 ◽  
Vol 36 (S1) ◽  
pp. 37-37
Author(s):  
Americo Cicchetti ◽  
Rossella Di Bidino ◽  
Entela Xoxi ◽  
Irene Luccarini ◽  
Alessia Brigido
Keyword(s):  
Real World ◽  
Ad Hoc ◽  
Grey Literature ◽  
National Level ◽  
Pharmaceutical Products ◽  
European Medicines Agency ◽  
The Real ◽  
R Process ◽  
The One ◽  
The Impact

IntroductionDifferent value frameworks (VFs) have been proposed in order to translate available evidence on risk-benefit profiles of new treatments into Pricing & Reimbursement (P&R) decisions. However limited evidence is available on the impact of their implementation. It's relevant to distinguish among VFs proposed by scientific societies and providers, which usually are applicable to all treatments, and VFs elaborated by regulatory agencies and health technology assessment (HTA), which focused on specific therapeutic areas. Such heterogeneity in VFs has significant implications in terms of value dimension considered and criteria adopted to define or support a price decision.MethodsA literature research was conducted to identify already proposed or adopted VF for onco-hematology treatments. Both scientific and grey literature were investigated. Then, an ad hoc data collection was conducted for multiple myeloma; breast, prostate and urothelial cancer; and Non Small Cell Lung Cancer (NSCLC) therapies. Pharmaceutical products authorized by European Medicines Agency from January 2014 till December 2019 were identified. Primary sources of data were European Public Assessment Reports and P&R decision taken by the Italian Medicines Agency (AIFA) till September 2019.ResultsThe analysis allowed to define a taxonomy to distinguish categories of VF relevant to onco-hematological treatments. We identified the “real-world” VF that emerged given past P&R decisions taken at the Italian level. Data was collected both for clinical and economical outcomes/indicators, as well as decisions taken on innovativeness of therapies. Relevant differences emerge between the real world value framework and the one that should be applied given the normative framework of the Italian Health System.ConclusionsThe value framework that emerged from the analysis addressed issues of specific aspects of onco-hematological treatments which emerged during an ad hoc analysis conducted on treatment authorized in the last 5 years. The perspective adopted to elaborate the VF was the one of an HTA agency responsible for P&R decisions at a national level. Furthermore, comparing a real-world value framework with the one based on the general criteria defined by the national legislation, our analysis allowed identification of the most critical point of the current national P&R process in terms ofsustainability of current and future therapies as advance therapies and agnostic-tumor therapies.

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