Biogen's recent FDA approval for an Alzheimer's disease (AD) drug is making headlines globally, and not necessarily for all the right reasons. While welcome news to patients and caregivers, the decision has stirred controversy amongst dementia experts. Experts claim that the clinical evidence to approve the drug was insufficient. In this blog, we break down the key issues here and highlight how this could present business opportunities for your organization.
1. What does the drug do?
Biogen's antibody drug, aducanumab (brand name: Aduhelm), works by binding to and removing sticky deposits of protein called amyloid beta (Aβ) plaques from the brains of patients with early-stage Alzheimer's disease (AD). While it is still an area of debate, evidence suggests that these plaques disrupt the calcium ion balance in neurons by inserting themselves into the membranes and allowing unregulated flow between the neurons and their surroundings. Simply put, these Aβ plaques disrupt important molecular balances in brain cells that are important for normal brain function. However, both Biogen and the FDA are careful to mention the drug is not a cure but rather a treatment that buys time and keeps more serious aspects of AD at bay.
2. If the evidence is not clear, why did the FDA approve the drug?
Alzheimer's disease is the sixth leading cause of death in the U.S., and with no drugs coming to market in almost 20 years, there has been strong pent-up demand and a huge business opportunity. The only other drug to receive approval recently is one from China, whose implications we discuss later below.
Almost every major pharmaceutical company has looked into developing a drug for Alzheimer's disease, without clear winners. Given the lack of medications to manage AD, the FDA had to carefully weigh the health risks and benefits and decided that it would rather make the drug available to patients than make them wait. The FDA made it clear that this is part of an accelerated approval pathway, and Biogen must still conduct confirmatory trials post-approval.
3. What does this mean for me?
Amid this controversy is an opportunity to support Biogen and other companies looking to develop drugs for AD and/or even other diseases where there has not been an efficacious drug for years. Traditional clinical trials only tell you part of the story, where tracking the efficacy of a drug is concerned. This is becoming more and more evident and being recognized by both pharmaceutical companies and regulators. This is why recent clinical trials incorporate an element of monitoring for digital biomarkers, both for side effects and for digital endpoints, to provide a broader, more comprehensive overview a patient's overall progress through a treatment. In addition, digital therapeutics (DTx) can also be used alongside pharmaceutical treatments, not just to improve medical adherence but also to educate and empower patients and their caregivers with actionable information to better manage their condition.
4. What should I do next?
If you have been unsure about the value proposition of new and emerging digital health technologies like digital biomarkers and DTx that track treatment progression, here is a prime example of a gap that sorely needs complementary solutions to track the treatment. This is especially true considering that, for wider adoption, Biogen will look to payers for reimbursement, and payers will demand unambiguous efficacy data.
Right now, patients who can afford the out-of-pocket cost can get access to the drug, but this will be a very niche segment, as Biogen estimates that the drug will cost around $56,000 per patient per year, which puts it out of reach for many without insurance coverage. Estimates suggest that Biogen can expect anywhere from $10 billion to $50 billion in sales annually. Biogen will not want to risk the FDA pulling the plug on Aduhelm if it cannot prove that the drug continues to benefit patients post-approval.
Over in Asia, China approved its first AD drug and is seeking to run trials in the U.S., presenting a potential threat to Biogen's market share. Biogen will want to cement its market leadership sooner rather than later and will be on the lookout for partners that can add value in supporting its confirmatory trials post-approval.
Here are a couple of ways a potential partner could work with Biogen:
- Complementing Biogen's drug: Developers like Dthera have developed digital therapeutics (using reminiscence therapy), receiving FDA Breakthrough Device designation recognizing their potential to complement AD treatment. Neeuro takes a gamification approach by training the brain with cognitive tasks, adding a layer of treatment beyond a pharmacological effect.
- Monitoring patients using Biogen's drug: Neuroglee Therapeutics is not just a DTx developer – its technology also enables monitoring tools that can help track drug treatment. Winterlight Labs uses voice-based biomarkers for early detection of AD but could potentially be explored for tracking AD progression as well.
While we discussed the immediate opportunity with Biogen for AD here, you should also be performing a larger strategic assessment of health conditions in their key markets that still do not have medications. It is unlikely that big pharma players are not targeting some of these conditions, but it may be struggling to get clarity from clinical trials on whether its drug candidates are efficacious. This is where digital biomarkers, DTx, remote monitoring, wearables, and other health IoT tools may provide further insights into a drug when used in conjunction with traditional biomarkers and clinical endpoints.