FDA, Academia and Industry Team Up to End Hepatitis C

As doctors prepare to manage an influx of new hepatitis C patients and treatment options, a collaboration between 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 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. The Hepatitis C Therapeutic Registry and Research Network, or HCV-TARGET, is led jointly by investigators at the University of North Carolina School of Medicine and University of Florida and is sponsored in part by multiple pharmaceutical companies.

The goal for the new partnership with the FDA 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 on-going 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. 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 professor of medicine at UF, which serves as the clinical coordinating center for HCV-TARGET.

"Real-world data about how drugs perform outside of restricted clinical trials is 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.

Other UNC faculty participating in HCV-TARGET are Paul Stewart, PhD, research professor, and Thomas Stewart, PhD student, both from the UNC Gilllings School of Global Public Health, and John Baron, MD, from the School of Medicine.

HCV-TARGET and the FDA signed 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 ensure FDA and HCV-TARGET data are comparable, 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.

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.

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. Dr. Fried receives research grant support from and serves as ad hoc consultant to Genentech, Vertex, Merck, Gilead, Bristol Myers Squibb, Janssen Pharmaceuticals, 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.