The whole patient
Primary care physicians will not be expected to be experts in gene therapy or solely in charge of patient safety. They will have support from industry and subspecialists leading the development of these treatments, experts agreed.
But generalists should expect to be drawn into multidisciplinary care teams, be the sounding boards for patients making decisions about gene therapy, help arrange insurance coverage, and be the recipients of late-night phone calls about side effects.
All that, while never losing sight of the child’s holistic health. In children so sick, specialists, subspecialists, and even parents tend to focus only on the rare disease. The team can “get distracted from good normal routine care,” Dr. Nelson said. But these children aren’t exempt from check-ups, vaccine regimens, or the other diseases of childhood.
“In a world where we mitigate that core disease,” he said, “we need a partner in the general pediatrics community” investing in their long-term health.
A version of this article first appeared on Medscape.com.