Public–Private Partnership: Targeting Real-World Data for Hepatitis C Direct-Acting Antivirals

Mishra, Poonam; Florian, Jeffry; Peter, Joy; Vainorius, Monika; Fried, Michael; Nelson, David R.; Birnkrant, Debra

doi:10.1053/j.gastro.2017.07.025

Cited by 19 publications

(19 citation statements)

References 20 publications

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“…As regulatory authorities, such as the FDA, approve new medications, this data set can serve to monitor for drug‐related adverse events and the impact of new interventions on liver, autoimmune, and neoplastic outcomes. TARGET‐PBC is based on the model used for hepatitis C virus (HCV)‐TARGET, a cooperative academic consortium that guides safe and effective use of direct‐acting antivirals approved for the treatment of chronic HCV infection and which has been extremely successful providing real‐world safety and effectiveness data for newer therapies . While there are only two drugs currently approved for PBC (UDCA and OCA), several other promising therapies are currently under investigation, and TARGET‐PBC will capture these new therapies as they enter the market.…”

Section: Discussionmentioning

confidence: 99%

A real‐world observational cohort of patients with primary biliary cholangitis: TARGET‐primary biliary cholangitis study design and rationale

Levy

Bowlus

Carey

et al. 2018

Hepatology Communications

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Primary biliary cholangitis (PBC) is a rare chronic cholestatic liver disease that may progress to biliary cirrhosis if left untreated. The first‐line therapy for PBC is ursodeoxycholic acid (UDCA). Unfortunately, 1 of 3 patients does not respond to UDCA. These patients are at risk for developing clinical events, including cirrhosis, complications of portal hypertension, hepatocellular carcinoma, liver transplant, or death. Recently, the U.S. Food and Drug Administration approved obeticholic acid to be used in certain patients with PBC. Off‐label therapies are also used, and several other therapies are currently under evaluation. Real‐world effectiveness of newly approved and off‐label therapies remains unknown. TARGET‐PBC is a 5‐year, longitudinal, observational study of patients with PBC that will evaluate the effectiveness of clinical practice interventions and provide practical information unobtainable in registration trials. Enrollment will take place at both academic and community sites. In addition to consenting to medical records review, participants will be asked to provide an annual blood sample and complete patient reported outcome surveys at predetermined intervals. Any available liver biopsies will be digitally preserved. Conclusion: Key study outcomes will be the evaluation of the safety and effectiveness of PBC interventions and the assessment of disease progression under real‐world conditions. (Hepatology Communications 2018;2:484‐491)

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Section: Discussionmentioning

confidence: 99%

A real‐world observational cohort of patients with primary biliary cholangitis: TARGET‐primary biliary cholangitis study design and rationale

Levy

Bowlus

Carey

et al. 2018

Hepatology Communications

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“…The HCV-TARGET infrastructure and real-world data collection methods have been leveraged to implement a unique approach to conduct investigative new drug clinical trials using combinations of approved drugs and to fulfill post-marketing commitment studies, as well as to execute investigative new drug protocols for Large Population Expanded Access Programs and late-phase 3b trials related to HCV therapy. 13 Study management was provided by the University of Florida (led by Dr David Nelson), data analyses by the University of North Carolina (led by Dr Michael Fried), with study medications and funding support provided by AbbVie to HCV-TARGET. The study protocol was approved by the Institutional Review Boards of all participating centers and all patients provided written informed consent before enrollment.…”

Section: Methodsmentioning

confidence: 99%

“…Data from source documents were extracted by trained personnel at the Clinical Coordinating Center as described previously. 13 SVR12 was defined as plasma HCV RNA below the lower limit of quantification (LLOQ) 12 weeks post treatment. Virologic failure was categorized as on-treatment failure (breakthrough) or relapse.…”

Section: Assessment Of Efficacy Safety and Virologic Resistancementioning

confidence: 99%

Efficacy of Glecaprevir and Pibrentasvir in Patients With Genotype 1 Hepatitis C Virus Infection With Treatment Failure After NS5A Inhibitor Plus Sofosbuvir Therapy

et al. 2019

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“…FDA also participated in the establishment of some international databases with the first one being the HCV-TARGET for which FDA has been a partner since its launch in 2011. It is a cooperative academic consortium designed to inform ongoing changes in the treatment and research of hepatitis C (21). HCV-TARGET established a common research database to evaluate the use of newly approved HCV drugs in a real clinical practice setting.…”

Section: Network and Databasesmentioning

confidence: 99%

Integrating Real-World Evidence in the Regulatory Decision-Making Process: A Systematic Analysis of Experiences in the US, EU, and China Using a Logic Model

Chen

Lai

et al. 2021

Front. Med.

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Real world evidence (RWE) and real-world data (RWD) are drawing ever-increasing attention in the pharmaceutical industry and drug regulatory authorities (DRAs) all over the world due to their paramount role in supporting drug development and regulatory decision making. However, there is little systematic documentary analysis about how RWE was integrated for the use by the DRAs in evaluating new treatment approaches and monitoring post-market safety. This study aimed to analyze and discuss the integration of RWE into regulatory decision-making process from the perspective of DRAs. Different development strategies to develop and adopt RWE by the DRAs in the US, Europe, and China were reviewed and compared, and the challenges encountered were discussed. It was found that different strategies on development of RWE were applied by FDA, EMA, and NMPA. The extent to which RWE was adopted in China was relatively limited compared to that in the US and EU, which was highly related to the national pharmaceutical environment and development stages. A better understanding of the overall goals, inputs, activities, outputs, and outcomes in developing RWE will help inform actions to harness RWD and leverage RWE for better health care decisions.

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Public–Private Partnership: Targeting Real-World Data for Hepatitis C Direct-Acting Antivirals

Cited by 19 publications

References 20 publications

A real‐world observational cohort of patients with primary biliary cholangitis: TARGET‐primary biliary cholangitis study design and rationale

A real‐world observational cohort of patients with primary biliary cholangitis: TARGET‐primary biliary cholangitis study design and rationale

Efficacy of Glecaprevir and Pibrentasvir in Patients With Genotype 1 Hepatitis C Virus Infection With Treatment Failure After NS5A Inhibitor Plus Sofosbuvir Therapy

Integrating Real-World Evidence in the Regulatory Decision-Making Process: A Systematic Analysis of Experiences in the US, EU, and China Using a Logic Model

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