More Data, Speedier Product Development?
Marjorie Shapiro, PhD, chief of the FDA’s Laboratory of Molecular and Developmental Immunology, stressed that the better the data on the nature of the product, the less additional data will likely be needed, possibly making for a speedier development.
“The more good data we have from those types of studies may reduce the need for other types of studies, additional clinical studies, and perhaps some nonclinical studies,” she said. “But there are different pathways to get to biosimilarity.”
Dr. Nikolov said that it’s possible to extrapolate clinical and nonclinical data from one indication to another, “provided there is a sufficient scientific justification.” That justification should address mechanism of action in each condition of use, pharmacokinetics and biodistribution of the product in different patient populations, and differences in expected toxicities for each condition of use and each patient population.
An FDA panel was asked how a trial could be conducted to compare a new product’s risk to an existing product, when one group of patients would be getting the established product and the other wouldn’t. Badrul Chowdhury, MD, PhD, director of the DPARP, said it’s important to remember that the premise of such a trial would be that the products are biosimilar. “One needs to conclude that, first of all, there’s biosimilarity and there’s equipoise—that we do know what the benefit or the harm is,” he said. “And then one does a study.”
The panel was also asked whether a drug sponsor could conduct a pharmacokinetic study, if called for, while doing a clinical study at the same time to resolve remaining questions. Dr. Christl said that, “there’s really no one-size-fits-all approach,” and that it depends on the risk that the sponsor is willing to absorb. But, she added, “we hope that folks will come in with some analytical characterization data before they do any clinical studies,” so that the regulators can offer suggestions on appropriate ways to design clinical studies.
In response to another question, Sarah Yim, MD, associate director of DPARP, said that biosimilars would trigger the Pediatric Research Equity Act. Dr. Christl said that when regulators review the product, it might be possible that they would consider the new product so similar to the reference product that pediatric studies wouldn’t be necessary. But there also might be enough differences that additional data, such as a pharmacokinetic study, would be needed. “We’re trying not to have to re-prove safety in every population,” she said, “if we can avoid it.”
