scholarly journals Building a National Reassessment Process for Oncology Drugs: Lessons Learned by the Canadian Real-World Evidence for Value of Cancer Drugs (CanREValue) Collaboration through a Simulated Reassessment Exercise

2021 ◽  
Vol 28 (6) ◽  
pp. 4645-4654
Author(s):  
Wei Dai ◽  
Erica Craig ◽  
Brent Fraser ◽  
Alex Chambers ◽  
Helen Mai ◽  
...  
Keyword(s):  
Real World ◽  
A Priori ◽  
Lessons Learned ◽  
Cancer Drug ◽  
Cancer Drugs ◽  
Real World Data ◽  
Study Protocols ◽  
Real World Evidence ◽  
Patient Representatives ◽  
Future Work

The CanREValue Collaboration established the Reassessment & Uptake Working Group to develop a preliminary process to reassess funded cancer drugs in Canada. A simulated exercise was conducted to evaluate the proposed reassessment process using a real-world case. We invited 32 attendees including representatives from Health Canada and Health Technology Assessment (HTA) agencies, along with payers, clinicians, academics, and patient representatives. A case was developed using a real-world study on a publicly funded cancer drug. In facilitated group sessions, participants were asked to deliberate upon the evidence presented in the case to issue reassessment recommendations. Several themes were identified through the deliberation discussions. While the generalizability of real-world evidence (RWE) is perceived as a strength, trust in the RWE depends largely on the source of the real-world data. The attendees suggested several improvements to the proposed reassessment process including evidence requirement for reassessment, recommendation categories, and a priori study protocols. This exercise generated important insights on the evidence required for conducting reassessment and considerations for improvements of the proposed reassessment process. Building upon lessons from this exercise, future work would continue to refine the reassessment process as part of the overall CanREValue framework.

scholarly journals Developing a framework to incorporate real-world evidence in cancer drug funding decisions: the Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration

BMJ Open ◽  
2020 ◽  
Vol 10 (1) ◽  
pp. e032884 ◽  
Author(s):  
Kelvin Chan ◽  
Seungree Nam ◽  
Bill Evans ◽  
Claire de Oliveira ◽  
Alexandra Chambers ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Healthcare System ◽  
Real World ◽  
Decision Makers ◽  
New Drugs ◽  
Cancer Drug ◽  
Cancer Drugs ◽  
Value For Money ◽  
Real World Evidence ◽  
Clinical Benefits

BackgroundOncology therapy is becoming increasingly more expensive and challenging the affordability and sustainability of drug programmes around the world. When new drugs are evaluated, health technology assessment organisations rely on clinical trials to inform funding decisions. However, clinical trials are not able to assess overall survival and generalises evidence in a real-world setting. As a result, policy makers have little information on whether drug funding decisions based on clinical trials ultimately yield the outcomes and value for money that might be expected.ObjectiveThe Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration, consisting of researchers, recommendation-makers, decision makers, payers, patients and caregivers, are developing and testing a framework for Canadian provinces to generate and use real-world evidence (RWE) for cancer drug funding in a consistent and integrated manner.StrategyThe CanREValue collaboration has established five formal working groups (WGs) to focus on specific processes in the generation and use of RWE for cancer drug funding decisions in Canada. The different RWE WGs are: (1) Planning and Drug Selection; (2) Methods; (3) Data; (4) Reassessment and Uptake; (5) Engagement. These WGs are acting collaboratively to develop a framework for RWE evaluation, validate the framework through the multiprovince RWE projects and help to integrate the final RWE framework into the Canadian healthcare system.OutcomesThe framework will enable the reassessment of cancer drugs, refinement of funding recommendations and use of novel funding mechanisms by decision-makers/payers across Canada to ensure the healthcare system is providing clinical benefits and value for money.

scholarly journals Real-world evidence use in assessments of cancer drugs by NICE

2020 ◽  
Vol 36 (4) ◽  
pp. 388-394 ◽  
Author(s):  
Ash Bullement ◽  
Tanja Podkonjak ◽  
Mark J. Robinson ◽  
Eugene Benson ◽  
Ross Selby ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Real World ◽  
Best Practice ◽  
Cost Effectiveness Analysis ◽  
Cancer Drug ◽  
Cancer Drugs ◽  
Best Practice Guidelines ◽  
Effectiveness Analysis ◽  
Real World Evidence ◽  
Related Quality

ObjectiveTo establish how real-world evidence (RWE) has been used to inform single technology appraisals (STAs) of cancer drugs conducted by the National Institute for Health and Care Excellence (NICE).MethodsSTAs published by NICE from April 2011 to October 2018 that evaluated cancer treatments were reviewed. Information regarding the use of RWE to directly inform the company-submitted cost-effectiveness analysis was extracted and categorized by topic. Summary statistics were used to describe emergent themes, and a narrative summary was provided for key case studies.ResultsMaterials for a total of 113 relevant STAs were identified and analyzed, of which nearly all (96 percent) included some form of RWE within the company-submitted cost-effectiveness analysis. The most common categories of RWE use concerned the health-related quality of life of patients (71 percent), costs (46 percent), and medical resource utilization (40 percent). While sources of RWE were routinely criticized as part of the appraisal process, we identified only two cases where the use of RWE was overtly rejected; hence, in the majority of cases, RWE was accepted in cancer drug submissions to NICE.DiscussionRWE has been used extensively in cancer submissions to NICE. Key criticisms of RWE in submissions to NICE are seldom regarding the use of RWE in general; instead, these are typically concerned with specific data sources and the applicability of these to the decision problem. Within an appropriate context, RWE constitutes an extremely valuable source of information to inform decision making; yet the development of best practice guidelines may improve current reporting standards.

scholarly journals Real-world evidence: perspectives on challenges, value, and alignment of regulatory and national health technology assessment data collection requirements

2021 ◽  
Vol 37 ◽  
Author(s):  
Hannah Sievers ◽  
Angelika Joos ◽  
Mickaël Hiligsmann
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Health Technology ◽  
Practical Experience ◽  
Added Value ◽  
Real World Data ◽  
Conflicting Demands ◽  
Real World Evidence ◽  
Semistructured Interviews

Abstract Objective This study aims to assess stakeholder perceptions on the challenges and value of real-world evidence (RWE) post approval, the differences in regulatory and health technology assessment (HTA) real-world data (RWD) collection requirements under the German regulation for more safety in drug supply (GSAV), and future alignment opportunities to create a complementary framework for postapproval RWE requirements. Methods Eleven semistructured interviews were conducted purposively with pharmaceutical industry experts, regulatory authorities, health technology assessment bodies (HTAbs), and academia. The interview questions focused on the role of RWE post approval, the added value and challenges of RWE, the most important requirements for RWD collection, experience with registries as a source of RWD, perceptions on the GSAV law, RWE requirements in other countries, and the differences between regulatory and HTA requirements and alignment opportunities. The interviews were recorded, transcribed, and translated for coding in Nvivo to summarize the findings. Results All experts agree that RWE could close evidence gaps by showing the actual value of medicines in patients under real-world conditions. However, experts acknowledged certain challenges such as: (i) heterogeneous perspectives and differences in outcome measures for RWE generation and (ii) missing practical experience with RWD collected through mandatory registries within the German benefit assessment due to an unclear implementation of the GSAV. Conclusions This study revealed that all stakeholder groups recognize the added value of RWE but experience conflicting demands for RWD collection. Harmonizing requirements can be achieved through common postlicensing evidence generation (PLEG) plans and joint scientific advice to address uncertainties regarding evidence needs and to optimize drug development.

scholarly journals Generating real-world evidence from unstructured clinical notes to examine clinical utility of genetic tests: use case in BRCAness

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Yiqing Zhao ◽  
Saravut J. Weroha ◽  
Ellen L. Goode ◽  
Hongfang Liu ◽  
Chen Wang
Keyword(s):  
Targeted Therapy ◽  
Data Quality ◽  
Real World ◽  
Genetic Information ◽  
Genetic Data ◽  
Real World Data ◽  
Rule Based ◽  
Clinical Notes ◽  
Real World Evidence ◽  
F Measure

Abstract Background Next-generation sequencing provides comprehensive information about individuals’ genetic makeup and is commonplace in oncology clinical practice. However, the utility of genetic information in the clinical decision-making process has not been examined extensively from a real-world, data-driven perspective. Through mining real-world data (RWD) from clinical notes, we could extract patients’ genetic information and further associate treatment decisions with genetic information. Methods We proposed a real-world evidence (RWE) study framework that incorporates context-based natural language processing (NLP) methods and data quality examination before final association analysis. The framework was demonstrated in a Foundation-tested women cancer cohort (N = 196). Upon retrieval of patients’ genetic information using NLP system, we assessed the completeness of genetic data captured in unstructured clinical notes according to a genetic data-model. We examined the distribution of different topics regarding BRCA1/2 throughout patients’ treatment process, and then analyzed the association between BRCA1/2 mutation status and the discussion/prescription of targeted therapy. Results We identified seven topics in the clinical context of genetic mentions including: Information, Evaluation, Insurance, Order, Negative, Positive, and Variants of unknown significance. Our rule-based system achieved a precision of 0.87, recall of 0.93 and F-measure of 0.91. Our machine learning system achieved a precision of 0.901, recall of 0.899 and F-measure of 0.9 for four-topic classification and a precision of 0.833, recall of 0.823 and F-measure of 0.82 for seven-topic classification. We found in result-containing sentences, the capture of BRCA1/2 mutation information was 75%, but detailed variant information (e.g. variant types) is largely missing. Using cleaned RWD, significant associations were found between BRCA1/2 positive mutation and targeted therapies. Conclusions In conclusion, we demonstrated a framework to generate RWE using RWD from different clinical sources. Rule-based NLP system achieved the best performance for resolving contextual variability when extracting RWD from unstructured clinical notes. Data quality issues such as incompleteness and discrepancies exist thus manual data cleaning is needed before further analysis can be performed. Finally, we were able to use cleaned RWD to evaluate the real-world utility of genetic information to initiate a prescription of targeted therapy.

scholarly journals Disease Network Delineates the Disease Progression Profile of Cardiovascular Diseases

2020 ◽  
Author(s):  
Zefang Tang ◽  
Yiqin Yu ◽  
Kenney Ng ◽  
Daby Sow ◽  
Jianying Hu ◽  
...  
Keyword(s):  
Cardiovascular Diseases ◽  
Disease Progression ◽  
Real World ◽  
Disease Risk ◽  
Disease Network ◽  
Real World Data ◽  
Risk Models ◽  
Age Bias ◽  
Real World Evidence ◽  
Tremendous Amount

AbstractAs Electronic Health Records (EHR) data accumulated explosively in recent years, the tremendous amount of patient clinical data provided opportunities to discover real world evidence. In this study, a graphical disease network, named progressive cardiovascular disease network (progCDN), was built based on EHR data from 14.3 million patients 1 to delineate the progression profiles of cardiovascular diseases (CVD). The network depicted the dominant diseases in CVD development, such as the heart failure and coronary arteriosclerosis. Novel progression relationships were also discovered, such as the progression path from long QT syndrome to major depression. In addition, three age-group progCDNs identified a series of age-associated disease progression paths and important successor diseases with age bias. Furthermore, we extracted a list of salient features to build a series of disease risk models based on the progression pairs in the disease network. The progCDN network can be further used to validate or explore novel disease relationships in real world data. Features with sufficient abundance and high correlation can be widely applied to train disease risk models when using EHR data.

scholarly journals Development of an Innovative Real-World Evidence Registry for the Herpes Simplex Virus: Case Study

10.2196/16933 ◽  
2020 ◽  
Vol 3 (1) ◽  
pp. e16933 ◽  
Author(s):  
Michelle Helena van Velthoven ◽  
Ching Lam ◽  
Caroline de Cock ◽  
Terese Stenfors ◽  
Hassan Chaudhury ◽  
...  
Keyword(s):  
Herpes Simplex Virus ◽  
Herpes Simplex ◽  
Real World ◽  
Physical Symptoms ◽  
Use Cases ◽  
Real World Data ◽  
World Data ◽  
Real World Evidence ◽  
Simplex Virus

Background Infection with the herpes simplex virus (HSV) is common but not well understood. Furthermore, there remains a social stigma surrounding HSV that can have psychosocial implications for those infected. Despite many patients infected with HSV experiencing mild-to-severe physical symptoms, only one subeffective treatment is available. A registry collecting real-world data reported by individuals potentially infected with HSV could help patients to better understand and manage their condition. Objective This study aimed to report on the development of a registry to collect real-world data reported by people who might be infected with HSV. Methods A case study design was selected as it provides a systematic and in-depth approach to investigating the planning phase of the registry. The case study followed seven stages: plan, design, prepare, collect, analyze, create, and share. We carried out semistructured interviews with experts, which were thematically analyzed and used to build use cases for the proposed registry. These use cases will be used to generate detailed models of how a real-world evidence registry might be perceived and used by different users. Results The following key themes were identified in the interviews: (1) stigma and anonymity, (2) selection bias, (3) understanding treatment and outcome gaps, (4) lifestyle factors, (5) individualized versus population-level data, and (6) severe complications of HSV. We developed use cases for different types of users of the registry, including individuals with HSV, members of the public, researchers, and clinicians. Conclusions This case study revealed key considerations and insights for the development of an appropriate registry to collect real-world data reported by people who might be infected with HSV. Further development and testing of the registry with different users is required. The registry must also be evaluated for the feasibility and effectiveness of collecting data to support symptom management. This registry has the potential to contribute to the development of vaccines and treatments and provide insights into the impact of HSV on other conditions.

scholarly journals Generating Real-World Evidence from Unstructured Clinical Notes to Examine Clinical Utility of Genetic Tests: Use Case in BRCAness

2020 ◽  
Author(s):  
Yiqing ZHAO ◽  
Saravut J Weroha ◽  
Ellen Goode ◽  
Hongfang Liu ◽  
Chen Wang
Keyword(s):  
Targeted Therapy ◽  
Data Quality ◽  
Real World ◽  
Genetic Information ◽  
Genetic Data ◽  
Real World Data ◽  
Rule Based ◽  
Clinical Notes ◽  
Real World Evidence ◽  
F Measure

Abstract Background: Next-generation sequencing provides comprehensive information about individuals’ genetic makeup and is commonplace in oncology clinical practice. However, the utility of genetic information in clinical decision-making process has not been examined extensively from a real-world, data-driven perspective. Through mining real-world data (RWD) from clinical notes, we could extract patients’ genetic information and further associate treatment decisions with genetic information.Methods: We proposed a real-world evidence (RWE) study framework that incorporates context-based natural language processing (NLP) methods and data quality examination before final association analysis. The framework was demonstrated on a Foundation-tested women cancer cohort (N=196). Upon retrieval of patients’ genetic information using NLP system, we assessed completeness of genetic data captured in unstructured clinical notes according a genetic data-model. We examined the distribution of different topics regarding BRCA1/2 throughout patients’ treatment process, and then analyzed the association between BRCA1/2 mutation status and the discussion/prescription of targeted therapy. Results: We identified seven topics in clinical context of genetic mentions including: Information, Evaluation, Insurance, Order, Negative, Positive, and Variants of unknown significance (VUS). Our rule-based system achieved a precision of 0.87, recall of 0.93 and F-measure of 0.91. Our machine learning system achieved a precision of 0.901, recall of 0.899 and F-measure of 0.9 for four-topic classification and a precision of 0.833, recall of 0.823 and F-measure of 0.82 for seven-topic classification. We found in result-containing sentences, capture of BRCA1/2 mutation information was 75%, but detailed variant information (e.g. variant types) is largely missing. Using cleaned RWD, significant associations were found between BRCA1/2 positive mutation and targeted therapies.Conclusions: In conclusion, we demonstrated a framework to generate RWE using RWD from different clinical sources. Rule-based NLP system achieved the best performance for resolving contextual variability when extracting RWD from unstructured clinical notes. Data quality issue such as incompleteness and discrepancies exist thus manual data cleaning is needed before further analysis can be performed. Finally, we were able to use cleaned RWD to evaluate real-world utility of genetic information to initiate prescription of targeted therapy.

Information technology in the evaluation of RWD / RWE (Real-World Data / Real-World Evidence)

2021 ◽  
Author(s):  
OV Zhukova ◽  
AL Khokholov
Keyword(s):  
Health Care ◽  
Information Technology ◽  
Clinical Practice ◽  
Clinical Efficacy ◽  
Real World ◽  
Information Technologies ◽  
Real World Data ◽  
Real World Evidence ◽  
Software Modules ◽  
Real Clinical Practice

The features of evaluating data from real clinical practice are discussed. Approaches to material processing for decision support in medicine and health care are also given. The development of standardized methods of analysis with the possibility of obtaining a unified indicator for assessing data from routine clinical practice, combined with the development of information technology is the direction of development of the concept of result-oriented health care. The classification of information technologies used in medicine and public health is presented. The main characteristics and functioning features of the developed software modules for automated data evaluation of real clinical practice are presented: a program for the distribution of drugs on the levels of clinical efficacy, a program to assess the effectiveness of therapy for the specified period; a program to determine the interval of clinical efficacy of drugs.

scholarly journals The Expanding Role of Real-World Evidence Trials in Health Care Decision Making

2019 ◽  
Vol 14 (1) ◽  
pp. 174-179 ◽  
Author(s):  
David C. Klonoff
Keyword(s):  
Real World ◽  
Clinical Evidence ◽  
Real World Data ◽  
Medical Products ◽  
Advantages And Disadvantages ◽  
Randomized Controlled ◽  
Real World Evidence ◽  
Care Decision

Real-world evidence (RWE) is the clinical evidence about benefits or risks of medical products derived from analyzing real world data (RWD), which are data collected through routine clinical practice. This article discusses the advantages and disadvantages of RWE studies, how these studies differ from randomized controlled trials (RCTs), how to overcome barriers to current skepticism about RWE, how FDA is using RWE, how to improve the quality of RWE, and finally the future of RWE trials.

Sign in / Sign up

Export Citation Format

Share Document