politics
FDA approves leucovorin for rare disorder without trial data
The FDA yesterday approved the generic medicine leucovorin for cerebral folate deficiency, a rare brain disorder that resembles autism, while pulling back from previous statements made by top health officials that children with autism could benefit from taking the drug.
The agency granted the approval to GSK, which first introduced the branded version of leucovorin to the market in 1997, but has stopped manufacturing it. The approval will allow generic manufacturers to update their labels as well.
Notably, the FDA said that approval was based on “a systematic review of the published literature on the topic, including published case reports with patient-level information, as well as mechanistic data.” It was not based on any new clinical trial data.
A senior FDA official said the agency does this kind of review in cases of extremely rare diseases, where it’s not feasible to conduct a randomized, controlled trial, Endpoints reported.
That appears to be at odds with the agency’s recent approach toward several gene therapies. For example, the FDA hasn’t been satisfied with the data behind UniQure’s Huntington’s disease candidate, which was studied in comparison to an external control group based on a natural history study.
from 1440 News:
Paying to Skip the Waiting Room
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What’s concierge medicine?
Concierge medicine is a membership model of healthcare in which patients pay a recurring fee on top of their insurance, copay, and deductibles. These fees may average around $3,500 annually, and come with the promise of around-the-clock access, same-day and longer appointments, and more personalized care. Some practices offer clinician house calls. Older, medically complex patients and patients with challenging schedules make up the majority of concierge medicine subscribers.
The concept of concierge care—sometimes referred to as retainer medicine or VIP medicine—is often traced to the 1996 establishment of MD2 International, a medical office in Seattle, Washington. By 2005, it was charging $13,000 per year for an annual membership. Concierge medical practices, including direct primary care ones that require membership fees but also don’t take insurance, grew swiftly between 2018 and 2023—increasing an estimated 83%. Physician assistants and nurse practitioners are now seeing many of the patients in these concierge practices, a change from earlier concierge medicine models but consistent with a larger healthcare shift in which more nonphysicians are seeing patients.
Supporters say the concierge model offers comprehensive, improved care, and its smaller patient load combats physician burnout. Critics contend that concierge care widens healthcare inequity and worsens primary care physician shortages by making it harder for people to see primary care physicians outside of membership-based offerings as more doctors move to concierge practices.
Explore everything else we’ve found on Concierge Medicine.
Also, check out …
> Concierge medicine offerings are growing on college campuses. (Read)
> Over 20 years ago, the Government Accountability Office considered whether concierge medicine threatens the future of Medicare. (Read)
> By 2036, the US may face tens of thousands of primary care shortages. (Read)
> Doctors report wanting more time to plan, provide care for patients. (Read)
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