For Aduhelm, the final stages of development included two randomised, controlled, clinical studies, known as phase 3. Clinical decline was slowed in one research, therefore that objective was fulfilled. The primary endpoint was not reached in the second study.
However, Aduhelm consistently and very substantially decreased the quantity of amyloid plaques in the brain in a dose- and time-dependent manner across all studies in which it was analysed. If amyloid plaque can be reduced, it is hoped that clinical decline can slow down as a result. Continue reading for more information Alzheimer’s Drug Poses a Dilemma For The F.D.A.
The FDA established the Accelerated Approval Program so that life-saving medications might be approved more quickly if they addressed an unmet medical need. Surrogate or intermediate clinical endpoints are used for approval (in this case reduction of amyloid plaque in the brain). A surrogate endpoint is a marker that is used to predict clinical benefit but is not a direct measure of clinical benefit itself, such as a laboratory value, a radiographic image, a physical sign, etc. A significant amount of time can be saved when using a surrogate endpoint to gain FDA approval.
After a drug has been approved, further research must be done to ensure it actually provides the intended clinical benefit. The term “phase 4 confirmatory trials” is used to describe these types of investigations. The FDA has regulatory mechanisms in place that might lead to the drug’s removal from the market if the confirmatory trial fails to validate the drug’s projected clinical benefit.
Impact of Alzheimer’s Disease
Having said that, we are well aware of the devastating effects of Alzheimer’s disease, which include the slow but steady decline of memory and other cognitive abilities over time. Patients in the latter stages of a disease are unable to communicate or react to their surroundings. Patients with Alzheimer’s disease have a median survival of four to eight years after diagnosis, however some have been known to live with the disease for as long as twenty years.
Over 6 million Americans currently suffer from Alzheimer’s disease, and that number is only likely to rise as the country’s population ages, highlighting the critical need for effective treatments. As of 2015, Alzheimer’s disease was the sixth greatest cause of mortality in America.
Although the clinical benefit data for Aduhelm are complicated, the FDA has assessed that there is significant evidence that Aduhelm decreases amyloid beta plaques in the brain and that the reduction of these plaques is fairly expected to predict important benefits to patients. The approval of Aduhelm by the FDA provides individuals with Alzheimer’s disease with a crucial new therapy option.
When Aduhelm finally makes it to stores and, later, to patients’ bedsides, the FDA will keep a close eye on it. To further ensure the drug’s clinical benefit, the FDA has mandated that Biogen execute a post-approval clinical investigation.
This drug can be taken off the market if it turns out to be ineffective. However, as more people participate in the clinical trial and receive Aduhelm, we can only hope to see more conclusive proof of the drug’s efficacy. As a government organisation, we will also keep pushing to encourage drug research for this devastating illness. Thanks for reading our article Alzheimer’s Drug Poses a Dilemma For The F.D.A.