Patients are enrolled prospectively at participating sites and treated per local standard of care. HCV-TARGET does not define treatment regimens, dosing, or duration or safety management practices. Source data is the original medical record. All original clinic notes, telephone notes, safety and efficacy labs/evaluations collected during treatment and in the post treatment observation period are submitted to a central data repository. Pertinent data from submitted records is abstracted by specially trained staff at the HCV-TARGET clinical coordinating center. Some data is mapped directly from electronic medical records into the database using the REDCap Eletronic Data Importer (RED-I) software.

• Demographics (gender, age, race, ethnicity, BMI, type of insurance)
• Baseline factors (HCV viral load, HCV genotype, cirrhosis determination, IL-28b genotypes, prior HCV treatment, medical history, co-morbid conditions)
• HCV treatment regimen and dose adjustments
• Adverse Events
• Concomitant Medications
• Labs to monitor safety and efficacy
• Long-term post treatment FU (co-morbid conditions, complications related to liver disease, mortality)

Regimens Time Table

Last Updated 04-2018

Listed below are HCV-TARGET Study Group presentations at the 2013 annual meeting of the American Association for the Study of Liver Diseases. For abstracts, visit http://www.aasld.org/livermeeting.
Contact: Joy A. Peter, Project Manager, Joy.Peter@medicine.ufl.edu

WASHINGTON — As doctors prepare to manage an influx of new hepatitis C patients and treatment options, a collaboration among academia, industry and the U.S. Food and Drug Administration is poised to deliver real-world data that can help doctors and patients optimize their treatment experience.

A research consortium known as the Hepatitis C Therapeutic Registry and Research Network, or HCV-TARGET, has joined forces with the FDA to share national data on how newly approved therapies for hepatitis C are used and managed in routine practice. HCV-TARGET is led jointly by investigators at the University of Florida and the University of North Carolina at Chapel Hill and is sponsored in part by multiple pharmaceutical companies.

The new partnership’s goal is to establish research collaborations using the HCV-TARGET database to better inform patients and clinicians about hepatitis C therapies.

“This collaboration will not only strengthen our ongoing efforts to monitor the safety and effectiveness of existing hepatitis C treatment regimens,” said Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, “it will also provide opportunities for FDA scientists to apply their research expertise in studying existing data held by HCV-TARGET to identify areas for improvement in clinical trial design that may help improve the future of HCV drug development programs.”

Hepatitis C is a viral liver disease that can lead to liver damage, cirrhosis, liver failure or liver cancer. It is transmitted through contact with infected blood. Because a person with chronic hepatitis C can live symptom-free for decades, many people do not know they are infected.

Two factors increase the significance of this collaboration:

• More patients to be screened and treated — Within the last year, both the Centers for Disease Control and Prevention and the U.S. Preventive Services Task Force recommended all baby boomers be tested for hepatitis C. The CDC estimates baby boomers represent three-quarters of the more than 3 million Americans believed to be infected with hepatitis C. Among those at highest risk are individuals who received blood transfusions before 1992, when screening donated blood for the virus began.
• New treatments on the horizon — The first all-oral hepatitis C treatment is moving through the final stages of FDA approval. If approved, it would be the first of a new generation of hepatitis C drugs that will improve what for many has been a grueling treatment regimen that can take up to 48 weeks and requires injections of interferon, a drug that can be difficult to tolerate. The introduction of new drugs will bring new questions about managing side effects, drug combinations and other clinical considerations.

“Leading liver doctors across the country have joined HCV-TARGET to study and navigate rapidly evolving treatment paradigms for hepatitis C. We see a healthier future for patients battling this virus and formed HCV-TARGET to help guide the way,” said Dr. David R. Nelson, co-principal investigator, director of the UF Clinical and Translational Science Institute and a professor of medicine at UF Health, which serves as the clinical coordinating center for HCV-TARGET.

Following close to 2,500 patients in North America who have agreed to participate in its study to date, HCV-TARGET includes populations underrepresented in clinical trials such as patients with cirrhosis, patients age 65 and older and African-Americans. The initial focus of the network’s observational study has been treatment with boceprevir and telaprevir, drugs newly approved by the FDA when HCV-TARGET launched in 2011. HCV-TARGET will expand its study this year to include the entire spectrum of antiviral hepatitis C therapeutics.

“Real-world data about how drugs perform outside of restricted clinical trials are extremely important. HCV-TARGET allows us to capture this information using novel approaches to ensure the integrity and quality of the data. Through our partnership with the FDA, we hope this information can be used to help doctors and their patients more readily determine the most beneficial treatment options across a broad spectrum of patients,” said Dr. Michael W. Fried, co-principal investigator and professor of medicine at the UNC School of Medicine, which serves as the HCV-TARGET data coordinating center.

HCV-TARGET and the FDA signed in May a memorandum of understanding to promote scientific research in the area of hepatitis C drug development. In mid-July, HCV-TARGET held meetings with representatives from the FDA Center for Drug Evaluation and Research’s division of antiviral products and offices of computational science, clinical pharmacology and biostatistics. Attendees agreed one of the first priorities should be to align how data elements of common interest are defined so the clinical trial data collected by the FDA can be reasonably compared to the real-world observational data collected by HCV-TARGET, a critical step in developing research collaborations and pilot projects. In addition, the agreement allows an FDA representative to join HCV-TARGET’s advisory council.

HCV-TARGET includes 103 academic and community sites in 31 states, Puerto Rico, Canada and Europe. HCV-TARGET currently receives ongoing industry support from Merck, Genentech, Kadmon and Vertex. Fried receives research grant support from and serves as ad hoc consultant to Genentech, Vertex, Merck, Gilead, Bristol Myers Squibb, Janssen Pharmaceuticals and Abbott. Nelson receives grant support from Genentech, Kadmon, Merck, Vertex, Gilead, Boehringer Ingelheim and Abbott/Abbvie; and payment for the development of educational presentations from Clinical Care Options, Rush University Medical Center, Practice Point Communications and the Chronic Liver Disease Foundation.

Media Contacts:

• Claire Baralt, (352-273-8211), cbaralt@ufl.edu
• Michelle Maclay, (919-843-5365), maclay@med.unc.edu

AMSTERDAM –– Two antiviral drugs used to treat hepatitis C appear to work as well in the real world as they did during clinical trials, an international research consortium has observed. The consortium also released data that may help inform how doctors and patients manage treatment-related adverse events.

The international effort, known as HCV-TARGET, follows how newly approved therapies for hepatitis C are used and managed in routine practice. It is led jointly by the University of Florida and the University of North Carolina at Chapel Hill.

The ongoing research suggests that the safety and efficacy of the antiviral drugs telaprevir and boceprevir are similar for North American patients taking the treatments in real-world settings to what was observed in clinical trials.

The evaluation of data available from November 2011 through April 2013, presented at the 48th Annual Meeting of the European Association for the Study of the Liver, found that anemia was the most relevant adverse event affecting clinical care. Approximately two-thirds of anemic patients were managed with drug dose reductions, which minimized the need for expensive growth factors and blood transfusions.

The analysis also reveals that patients with cirrhosis were at increased risk for treatment-related complications, including severe anemia and significant deterioration of the liver, which often resulted in stopping therapy early. One of the consortium’s priorities for future analyses will be to investigate indicators that may predict adverse outcomes in cirrhotic patients and guide safer use of these drug regimens.

“We plan to track up to 5,000 patients internationally over five years to continue to assess the benefits and risks of new treatments in real-world settings,” said Dr. Michael W. Fried, co-principal investigator and professor of medicine at UNC, which serves as the HCV-TARGET data coordinating center. “HCV-TARGET serves as a model for cross-cutting collaborative studies that can rapidly advance knowledge in an important illness of public health concern.”

“This is a long-term study, and we plan to release similar interim analyses each spring and fall to provide clinicians with up-to-date knowledge that can inform how we manage therapy for patients with hepatitis C,” said Dr. David R. Nelson, co-principal investigator, director of the UF Clinical and Translational Science Institute and professor of medicine at UF, which serves as the clinical coordinating center for HCV-TARGET.

Hepatitis C is a viral liver disease transmitted through contact with infected blood. Chronic hepatitis C can lead to serious liver problems including liver damage, cirrhosis, liver failure or liver cancer. Because a person with chronic hepatitis C can live symptom-free for many years, many people do not know they are infected.

Globally, the World Health Organization estimates about 150 million individuals are chronically infected with hepatitis C, and more than 350,000 people die each year from hepatitis C-related liver diseases. In the U.S., the Centers for Disease Control and Prevention estimates 3.2 million people are chronically infected with hepatitis C, although a 2011 review article in Liver International suggests the estimate is likely higher – at least 5.2 million people – if U.S. populations not surveyed by the CDC are included, such as the homeless and incarcerated.

HCV-TARGET is an international research consortium created to inform the ongoing transformation of hepatitis C treatment and research. The HCV-TARGET model is rooted in the infrastructure and collaborative network developed through the National Institutes of Health’s Clinical and Translational Science Award (CTSA) program, which is led by the National Center for Advancing Translational Sciences. In addition to UNC and UF, HCV-TARGET includes 23 other CTSA-supported institutions among its 103 academic and community sites in 31 states, Puerto Rico, Canada and Europe. HCV-TARGET also partners with multiple industry sponsors, regulatory agencies and the patient advocacy community.

In 2011, HCV-TARGET established a nationwide registry to observe patients in the United States undergoing hepatitis C treatment over time and to coordinate real-world monitoring on a national scale for new therapies as they enter the market. For patients who agree to be in the study, the project is capturing demographic, clinical, adverse event and virological data. To date approximately 1,900 patients have agreed to participate, including patients with cirrhosis and other populations underrepresented in clinical trials.

“The data coming out of the HCV-TARGET consortium will help inform physicians and patients as they weigh important decisions regarding therapy, decisions that can greatly impact quality of life,” said Dr. Donald M. Jensen, professor of medicine at the University of Chicago and a member of the HCV-TARGET Steering Committee.

HCV-TARGET’s participating patients are treated according to local standards of care. In addition, participants can allow HCV-TARGET to collect their whole blood for DNA and serum and store it at a central biorepository for future research.

The network’s initial study has followed a broad population of adult patients in North America treated with telaprevir or boceprevir, which were newly approved by the U.S. Food and Drug Administration when HCV-TARGET launched. In 2013, HCV-TARGET is expanding its study to include European sites and patients treated with any direct-acting antiviral agent approved by the FDA.

HCV-TARGET receives ongoing industry support from Merck, Genentech, Kadmon and Vertex. Dr. Fried receives research grant support from and serves as ad hoc consultant to Genentech, Vertex, Merck, Gilead, Bristol Myers Squibb and Abbott. Dr. Nelson receives grant support from Genentech, Kadmon, Merck, Vertex Pharmaceuticals, Gilead, Boehringer Ingelheim and Abbott/Abbvie; and payment for the development of educational presentations from Clinical Care Options, Rush University Medical Center, Practice Point Communications and Chronic Liver Disease Foundation.

Related Resources

• April 2013 Interim Analysis Summary

Media Contacts:

• Claire Baralt, (352-273-8211), cbaralt@ufl.edu
• Michelle Maclay, (919-843-5365), maclay@med.unc.edu