Raising the Patient Voice in Alzheimer's Research

When it comes to new treatments for Alzheimer’s disease, no one has a clearer idea of what’s needed than Alzheimer’s patients and caregivers. But to date, the patient and caregiver perspective has largely been absent from the drug development and approval process. Now, Lundbeck is working to change that.

I am very proud that Lundbeck is a founding sponsor of AD PACE, a groundbreaking new public-private collaboration led by UsAgainstAlzheimers that aims to raise the patient and caregiver voice in Alzheimer’s drug development. Through AD PACE, patients, caregivers, leading pharmaceutical companies, academics and government agencies are joining forces to incorporate the patient and caregiver perspective into drug development, clinical trial design, regulatory approval, policymaking and care.

All of us who are working toward Alzheimer’s treatments need to understand the daily challenges of Alzheimer’s patients and caregivers, and we must consider and respect which of those challenges are the most important to them. If the measures we use in our clinical trials assess items of little importance to our patients, then we risk developing a drug that misses the mark. For example, if a drug helps a patient who is highly sociable remember a few more items on a grocery list, but doesn’t help him or her continue to feel connected to those around them, the drug hasn’t met their needs.

AD PACE will ensure that the outcomes prioritized by researchers and regulators mirror those considered most meaningful by people impacted by Alzheimer’s disease. Because if we don’t get that right, we fail the very people we are working so hard to aid.

What matters most?

As the only global pharmaceutical company solely focused on the brain, Lundbeck is committed to improving the lives of people impacted by Alzheimer’s and we have a very strong connection with our patient communities. We recognize that the Alzheimer’s patient and their caregivers are powerful sources for both the efficacy and value of drug treatment.  Until now, however, it has been difficult to meaningfully act on their insights. 

Although the Food and Drug Administration has made patient-focused drug development a priority, there has not been a validated process for incorporating Alzheimer’s patient and caregiver feedback into the clinical trial review process. Much of the feedback provided by Alzheimer’s patients was considered either anecdotal or unreliable, due to the cognitive impact of the disease.

AD PACE aims to change this by developing a scientifically rigorous understanding of what matters most to Alzheimer’s patients and caregivers. Already, more than 6,000 people have joined the AD PACE A-LIST, a growing online community of Alzheimer’s patients, caregivers, and people at risk for developing dementia and Alzheimer’s. These A-Listers will participate in surveys and offer insights on what it is like to experience Alzheimer’s disease.

Also underway is the AD PACE What Matters Most study, an 18-month, two-part study focused on identifying and quantifying treatment-related needs, preferences and priorities. In the first phase of this study, AD PACE will work to understand which treatment-related outcomes matter to individuals with Alzheimer’s; in the second phase, researchers will analyze the data to understand how much each of those outcomes matter to patients and caregivers so that target outcomes can be prioritized appropriately.

Because the FDA is an advising member of AD PACE, there is strong potential these insights could inform the agency’s guidance on Alzheimer’s drug development and lead to a more patient-centric drug development process.

Listen up

This week in Chicago, Alzheimer’s researchers from around the world are gathering for the annual Alzheimer’s Association International Conference (AAIC). It is the first major gathering of Alzheimer’s researchers since the launch of AD PACE. Throughout the week, the scientists, clinicians and advocates in attendance will hear from leading researchers and Alzheimer’s experts. With the many speeches, presentations and conversations at this high-profile event, I hope we all take a moment to listen to the most important voices among us: Alzheimer’s patients and caregivers, for they need and deserve our most-focused attention and commitment.


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