Part two of two parts; the first ran Thursday, June 10.
There’s still a great deal we don’t yet understand about aducanumab (brand name Aduhelm) or its longer-term effects. If early-stage Alzheimer’s disease is diagnosed in time for someone to begin taking the drug, are the potential adverse effects cumulative? Could long-term toxicity build up over time? How long might the drug stave off development of the plaque, and how long might it slow down the process of cognitive decline, if at all, and how will clinicians assess its benefit in patients? Continue reading
National Institute on Aging/National Institutes of HealthBeta-amyloid plaques and tau in the brain.
Part one of two parts; the second runs tomorrow, Friday, June 11.
You might think that the first new drug to treat Alzheimer’s in 18 years — and the first to treat underlying disease and not just symptoms — would be heralded by patients, families, and medical professionals alike. After all, the FDA’s approval on Monday of aducanumab (brand name Aduhelm) sounds like a tremendous breakthrough for the estimated 6 million Americans, and 50 million people globally, who suffer from the disease.
However, because of the supporting clinical data on its effectiveness, the drug has been controversial from the start. Drug maker Biogen actually halted its parallel Phase 3 studies, ENGAGE and EMERGE, because they failed to meet their primary endpoints. Those original endpoints were a change in the Clinical Dementia Rating-Sum of Boxes (CDR-SB), which is similar to a composite endpoint because it assesses improvement in multiple different domains. Continue reading
The history of inequity in medical studies is long and harrowing, and it continues today. But at least today, there is more awareness of the history and the present-day problems that persist. For example, the Endocrine Society recently released a scientific statement demanding more research into sex differences for the sake of public health.
The fact that males and females — not to mention individuals who do not identify as either binary category — do not respond the same way to different diseases, drugs and other interventions has been a relatively new development in the history of clinical trials. As recently as 1977, women of childbearing age were explicitly excluded by the FDA from phase 1 and 2 drug trials. In practice, that often extended to phase 3 trials and other types of studies for various reasons. Continue reading
As someone who was used to covering multiple medical conferences in person each year, 2020 was a big shift. I had to adapt to covering conferences virtually.
On the one hand, it was great: I got to sit in my home, eat my own (far less expensive) food, and watch many of the presentations on my own time instead of racing from one end of a convention center to another. Continue reading
In a teleconference announcing the company’s first-quarter 2021 earnings earlier this month, Pfizer laid out its timeline for when different versions of its COVID-19 vaccine may become available for various populations. The company also said it is working on two approaches for an mRNA-based flu vaccine (similar technology as the COVID-19 vaccine), with plans to run clinical trials in the third quarter.
By the end of May, the New York City company expects to submit a biologics license application (BLA) asking the FDA to fully approve the vaccine rather than continue its use under an emergency use authorization (EUA). Continue reading