Clinical trials framework proposed to bring cancer treatments to sub-Saharan Africa

In the journal PLOS Medicine, Satish Gopal, MD, MPH, cancer program director for UNC-Project Malawi, outlined a framework to design studies and bring cancer therapies to sub-Saharan Africa in the absence of clinical trial data specific to the region’s population and health care infrastructure.

Clinical trials framework proposed to bring cancer treatments to sub-Saharan Africa click to enlarge Satish Gopal, MD, MPH, is cancer program director for UNC-Project Malawi.

Physicians who seek to bring cancer treatments to sub-Saharan Africa that are available in other parts of the world are often hindered by a lack of clinical trial data showing how these treatments might help or harm patients in the region, according to a University of North Carolina Lineberger Comprehensive Cancer Center physician-researcher.

In the journal PLOS Medicine, Satish Gopal, MD, MPH, outlined a framework to design studies and bring cancer therapies to sub-Saharan Africa in the absence of clinical trial data specific to the region’s population and health care infrastructure.

Gopal is cancer program director for UNC-Project Malawi, a care, research and training collaboration between UNC-Chapel Hill and the Malawi Ministry of Health in Malawi, a country in southern Africa of more than 18 million people.

UNC Lineberger researchers are working to open clinical trials in Malawi to test treatments already available in the United States, as well as to study possible novel agents. In addition, cooperative clinical trial groups sponsored by the National Cancer Institute have started working in the region as well, with UNC-Project Malawi being one of the regional centers recruited to participate in their efforts.

“The concept of evidence-based medicine is to take studies that have been done in populations similar to yours, and use the results of those studies to inform practice and health care guidelines,” Gopal said. “The problem in sub-Saharan Africa, where more than one billion people live, is that we have little evidence from this part of the world specifically. Often, you hear two diametrically opposed ways of dealing with this problem.”

Gopal said one approach is to use data from clinical trials done elsewhere to develop identical treatment strategies for sub-Saharan Africa. However, Gopal said that approach does not account for issues specific to the population in the region, such as differences in tumor biology, the burden of infectious diseases, and differences in health care infrastructure. For example, Gopal said there are no radiation oncology services in Malawi. In addition, there are major deficiencies in supportive care for patients that limit the intensity of chemotherapy that can be safely administered. Alternatively, an opposing strategy is to repeat all trials in sub-Saharan Africa to guide care in the local context, but Gopal said this is simply not feasible.

“There has to be a sensible middle ground,” Gopal said.

Gopal argued trials may be required for some treatments, while for others, new data may not be required if the drugs have been shown to be particularly well-tolerated and efficacious. For example, he said the favorable therapeutic ratio for the drug imatinib for chronic myelogenous leukemia led to the launch of the Novartis Glivec International Patient Assistance Program, one of the most “far-reaching global oncology pharmaceutical access program ever attempted,” without first requiring new clinical data in low-resource settings.

“How can trials be designed in a way that both produces new knowledge relevant for the region, while also resulting in the highest likelihood that patients in Malawi get an effective treatment for their cancer? These questions are central to contemporary cancer clinical trial design, and are being asked even here in the United States.” Gopal said. “We need to have a nuanced discussion about what evidence is required, and where the evidence is sufficient to just advocate for implementation. These are issues which the global cancer community needs to tackle with some specificity in this part of the world.”