FDA 敦促从遗传学试验中收集更多的长期安全性治疗(下)
"It will take a lot of money to get done. It's a disincentive," he said.
Instead, Douglas and other sponsors of experimental genetic therapies want the FDA to make the determination of how much follow-up is needed and how that information is to be gathered on a case-by-case basis, which has been the agency's de-facto policy. They also want either the FDA or another regulatory agency to collect the required long-term follow-up information in a central database managed by the government rather than the sponsors.
But even then, questions remain about how would this information be gathered. Committee members suggested that the task could be accomplished by sending a yearly postcard to patients. But the problem with that approach is that it would fail to account for any patients that died due to a side effect, the committee noted.
Another proposed solution was asking the patients' primary physician to note any possible long-term side effects. But that solution could discourage patients' participation because they would be required to sign an informed consent, allowing the sponsor of their genetic therapy to question their doctor about their health for almost two decades, the committee observed.
The recommendations echoed suggestions made earlier this year by the same committee. At that April meeting, the panel suggested that the length of the follow-up period--which could potentially range from 5 years to about 20 years--should be tailored in part to the biological system the gene therapy manipulates and the risk that is likely to be associated with the treatment.
For example, they suggested that a gene therapy that carries a risk of cancer should be monitored in patients for as long as 20 years, because the associated gene mutagenicity could take that long to develop. On the other hand, a gene transfer product that could trigger an autoimmune response may need only a 5-year follow-up.
While the committee comments could lead to a broader general principle or policy, these details will need to be ironed out, Dr. Jay P. Siegel, director of the FDA's therapeutic research and review office, told Reuters Health.
"What we are hearing is that there is no one-size-fits-all solution," Siegel said., http://www.100md.com
Instead, Douglas and other sponsors of experimental genetic therapies want the FDA to make the determination of how much follow-up is needed and how that information is to be gathered on a case-by-case basis, which has been the agency's de-facto policy. They also want either the FDA or another regulatory agency to collect the required long-term follow-up information in a central database managed by the government rather than the sponsors.
But even then, questions remain about how would this information be gathered. Committee members suggested that the task could be accomplished by sending a yearly postcard to patients. But the problem with that approach is that it would fail to account for any patients that died due to a side effect, the committee noted.
Another proposed solution was asking the patients' primary physician to note any possible long-term side effects. But that solution could discourage patients' participation because they would be required to sign an informed consent, allowing the sponsor of their genetic therapy to question their doctor about their health for almost two decades, the committee observed.
The recommendations echoed suggestions made earlier this year by the same committee. At that April meeting, the panel suggested that the length of the follow-up period--which could potentially range from 5 years to about 20 years--should be tailored in part to the biological system the gene therapy manipulates and the risk that is likely to be associated with the treatment.
For example, they suggested that a gene therapy that carries a risk of cancer should be monitored in patients for as long as 20 years, because the associated gene mutagenicity could take that long to develop. On the other hand, a gene transfer product that could trigger an autoimmune response may need only a 5-year follow-up.
While the committee comments could lead to a broader general principle or policy, these details will need to be ironed out, Dr. Jay P. Siegel, director of the FDA's therapeutic research and review office, told Reuters Health.
"What we are hearing is that there is no one-size-fits-all solution," Siegel said., http://www.100md.com