FDA 敦促从遗传学试验中收集更多的长期安全性治疗(上)
WASHINGTON (Reuters Health) - Expert advisors to the US Food and Drug Administration (FDA) on Wednesday recommended that the agency require sponsors of genetic trials to track patients for at least 15 years, regardless of whether or not the therapy was delivered using a virus vector with the ability to replicate.
At present, the FDA requires such long-term patient follow-up for only those therapies using retroviral vectors, or viruses with the ability to replicate and spread between different organs. The committee recommendation was meant to address the growing use of other RNA and DNA-based delivery mechanisms, for which there is no FDA policy.
In making the recommendation, the Biological Response Modifiers Advisory Committee said that its primary concern was that all RNA and DNA-based virus vectors could have long-term side effects.
Historically, DNA and RNA-based viruses have been shown to cause or be associated with malignancies such as leukemia, as well as liver failure, central nervous system disorders and autoimmune conditions such as multiple sclerosis. These conditions have also been shown to take months or even years to occur after the initial infection.
"The public, Congress expect it of us," said Dr. Daniel R. Salomon, the committee chair and an associate professor of Medicine at the Scripps Research Institute in La Jolla, California, while summing up the committee recommendation. "It will help move the field forward."
Currently, an estimated 4,000 Americans are participating in about 100 genetic trials, according to figures from the National Institutes of Health.
But in making the recommendation, some investigators fear the committee may have advised the agency to set too high a bar for sponsors in the field. While supporting the basic need for long-term follow-up, their concern is that these additional requirements would discourage patient participation and create a financial barrier that would be difficult for even the deep-pocketed sponsors of the experimental therapies to overcome.
"It's a tough problem," Dr. Jolly Douglas, CEO of BioMedica Inc., a sponsor of experimental genetic therapies, told Reuters Health. Even in conventional clinical trials taking place over a span of several years, patients are often lost over time due to a move or other uncontrollable event, Douglas explained., 百拇医药
At present, the FDA requires such long-term patient follow-up for only those therapies using retroviral vectors, or viruses with the ability to replicate and spread between different organs. The committee recommendation was meant to address the growing use of other RNA and DNA-based delivery mechanisms, for which there is no FDA policy.
In making the recommendation, the Biological Response Modifiers Advisory Committee said that its primary concern was that all RNA and DNA-based virus vectors could have long-term side effects.
Historically, DNA and RNA-based viruses have been shown to cause or be associated with malignancies such as leukemia, as well as liver failure, central nervous system disorders and autoimmune conditions such as multiple sclerosis. These conditions have also been shown to take months or even years to occur after the initial infection.
"The public, Congress expect it of us," said Dr. Daniel R. Salomon, the committee chair and an associate professor of Medicine at the Scripps Research Institute in La Jolla, California, while summing up the committee recommendation. "It will help move the field forward."
Currently, an estimated 4,000 Americans are participating in about 100 genetic trials, according to figures from the National Institutes of Health.
But in making the recommendation, some investigators fear the committee may have advised the agency to set too high a bar for sponsors in the field. While supporting the basic need for long-term follow-up, their concern is that these additional requirements would discourage patient participation and create a financial barrier that would be difficult for even the deep-pocketed sponsors of the experimental therapies to overcome.
"It's a tough problem," Dr. Jolly Douglas, CEO of BioMedica Inc., a sponsor of experimental genetic therapies, told Reuters Health. Even in conventional clinical trials taking place over a span of several years, patients are often lost over time due to a move or other uncontrollable event, Douglas explained., 百拇医药