Representatives of the European Liver Patients Association and the European Association for the Study of the Liver (EASL) urged European governments to implement compassionate access programs that would allow hepatitis C-infected patients with cirrhosis access to new hepatitis C drugs. Compassionate access programs, which “provide medicines to people with an urgent unmet need for treatment, prior to marketing approval,” may apply to individual patients, cohorts of eligible patients within a study that collects safety and effectiveness data, or a large number of patients who get access without collection of data.
Liver patients and doctors disagreed on who should decide about the new drugs’ risk for patients. EASL spokesperson Dr. Daniele Prati was in favor of compassionate access for hepatitis C-infected patients who would progress to decompensated cirrhosis, but only if the benefits outweigh the risks. Ivan Gardini, vice president of the European Liver Patients Association, argued that individual patients should have the right to make an informed decision to take a new drug, even before evidence about safety and efficacy is available.
Although some European countries lack a legal mechanism to allow compassionate access, the European Union gives the European Medicines Agency’s Committee for Human Medicinal Products the power to establish eligibility criteria for compassionate access programs. Each country `must approve the program. Philip Josephson, a representative of the European Medicines Agency, recommended a hepatitis C program that would combine compassionate access with a study that collects data about safety and efficacy. Other experts urged that compassionate access programs operate from specialty centers with the capacity to monitor patients and manage any adverse events. Potential issues include unexpected serious toxicities and drug-drug interactions.
People co-infected with HIV and hepatitis C comprised the population most likely to need new drugs because liver disease progresses more quickly in this group.