List of Comments
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PLEASE NOTE: The Public Comments included here are not an endorsement of the views or information by National Alzheimer's Project Act, its Advisory Council members, the Administration or the federal agencies involved in this project.
R. Paulsen 5-12-2023
I’m Chief Operating Officer of UsAgainstAlzheimer’s. I have no personal disclosures. My organization, as a nonprofit, receives support from thousands of donors, including pharmaceutical companies.
Thank you for the discussion today and for hearing from us.
Thanks, also, to the Advisory Council for the sense of urgency you bring to this fight against Alzheimer’s. As Dr. Bateman noted earlier, the first goal stated in the National Plan to Address Alzheimer’s Disease is time-bound: to prevent and effectively treat Alzheimer’s by 2025.
That urgency and that deadline were not arbitrary and were not set just because we live in a fast-paced world. The urgency is because every day, about 2,000 Americans progress to moderate Alzheimer’s disease. That stage is generally marked by a clear loss of independence and often much more, and it’s out of reach of these new disease-slowing therapies. Every day matters.
We heard quite a bit today about all of the complexity in moving from bench to bedside. Getting to effective prevention and treatment by 2025 is not just the job of the NIA and the scientists they fund, as we heard today. We all have a part to play:
- CDC needs to disseminate the science on prevention, and design and deploy public-health strategies that work.
- FDA needs to review and apply the speed and flexibility that is appropriate to therapies, diagnostics, and devices that address such a serious, unmet need.
- Health systems need to be creative in addressing bottlenecks in the system, especially around access to neurologists. If we just put people in line without figuring out how to open up the pipeline safely, that 2,000 people per day rate of progression will only go down a tiny bit.
- And CMS needs to provide the same coverage, with the same urgency, for Alzheimer’s diagnostics and therapeutics that they do for every other disease. We need to acknowledge that CMS made a decision last year without evidence in front of it that is now slowing down access to therapies that experts in the field have concluded are the first effective disease-modifying-therapies for Alzheimer’s ever.
We all need to do our part and do it with urgency, and that includes nonprofit patient-advocacy organizations. It’s time to move from capturing evidence in clinical trials to capturing evidence from the real world, and both the Alzheimer’s Association and UsAgainstAlzheimer’s have committed to different ways of capturing that. The Association’s Alz-Net registry will be an important way for patients in many practices to contribute information, and our AD Evidence Accelerator will generate significant evidence from broad, passive data collection. And that real-world data will help all of us understand how things work in broad clinical practice in diverse populations.
We all have a part to play. We cannot let up. We cannot let the quest for the drug perfect trial or the perfect non-pharmacological intervention be the enemy of goodness.
2,000 Americans progressed to moderate Alzheimer’s today. The next 2,000 and the 2,000 after that need us all to play our parts and to act with urgency.
M./C. Peterson 5-9-2023
We are the parents of three daughters. Mia was our middle child. She had Down syndrome and lived a full, independent, vital life.
From a young age Mia was blessed with articulation skills and a personality that made others want to listen to her. She wanted to help people with their struggles. She wanted to be a teacher. She wanted to be a friend. Mia was all of these. Her advocacy work both independently and with the National Down Syndrome Society took her to communities across the country. She was impactful. When Mia was 41 years old, she was diagnosed with Alzheimer’s Disease. 41. She progressed from mildly forgetful to confused. We took her to a clinic that specialized in disabilities and voiced our concerns. After a thorough evaluation we were told Mia did not have Alzheimer’s. Later that year our fears were confirmed when a neuropsychologist examined her. She said not only did Mia have Alzheimer’s; she had “fallen off the cliff.”
Mia’s social skills had masked it. We were shocked! Mike and I quickly moved 80 miles to Des Moines to provide support, eventually moving into a house the three of us could share. Despite the understanding and support from her employers, we could tell other employees were covering for her confusion at work. We encouraged Mia to retire. The three of us embarked on Mia’s Retirement Tour, traveling from one end of the country to the other visiting the people who loved Mia while she could still have conversations with them. We tried to do everything right. We followed a Mediterranean diet, exercised regularly, but failed in attempts to practice meditation. Mia and I became involved in a research project to explore the Down syndrome/Alzheimer’s Disease connection with the Linda Crnic Institute for Down Syndrome in Denver. As the disease progressed we acquired things--a stair lift, a custom wheel chair, a wheel chair ramp, and a specialized van. When the pandemic hit, Mike and I became Mia’s sole caregivers. Our independent daughter became totally dependent on us for all aspects of daily living. Mia developed a seizure disorder that required frequent tele-medicine (remember COVID) appointments with her neurologist and her memory care physician. We prepared mashed sweet potatoes and fresh fruit and vegetable smoothies with protein powder. We crushed her pills to make them easier to swallow.
Hospice came into the picture when we needed more frequent advice on daily care. They provided a nurse and a bath aide. At the nurse’s urging we acquired a hospital bed to put in our den and stopped taking her upstairs to her bedroom. Mike began sleeping on a mattress on the floor by Mia. It was a struggle coming up with activities to engage her when Mia could barely communicate. We showed her pictures of family and friends and familiar activities from her amazing life. We showed her pictures in cookbooks and talked about foods we might prepare. We played a game of tossing a balloon around the house. Every smile we got was treasured. Her sister, her brother-in-law, and nephew made COVID era visits standing on the porch and talking to Mia through a slightly opened window. Her sister from Portland made several masked visits. Her boyfriend from California called frequently. Hospice services expanded to a music therapist and social worker. We were doing pretty well, but we panicked when Mia stopped eating, even more so when she stopped drinking. The hospice nurse came daily, her sister several times per day. The last day of her life, June 8, 2021, Mia struggled to breathe. With her family surrounding her and her Oregon family on FaceTime, Mia passed away. She was 47 years old. It was gut-wrenching and sad, but it was also a relief.
Through it all, Mia knew us. Perhaps she continued to know us because COVID’s isolation allowed us to spend every minute of every day caring for and engaging with her. A vivid memory Carol has is of sitting with Mia on chairs at the dining room table. Mia had mostly lost the ability to talk, but clearly said to Carol, “I’m lucky.” Over the course of the next couple minutes, she said it four times. Carol wanted to tell Mia she was anything but lucky, but in her heart, Carol knew Mia was trying to express gratitude. It was so very Mia.
This is ONE story. In recent years we have become aware of how frequently this story repeats itself. Four people with Down syndrome we know intimately have been diagnosed with Alzheimer’s disease and passed away before the age of 50. All of them were amazing people, living healthy, impactful lives before they were diagnosed with Alzheimer’s Disease.
Because 80-90% of persons with Down syndrome will be diagnosed with Alzheimer’s disease, the work NAPA does affects our community, the Down syndrome community, in profound ways. Because there is a known significant connection between Trisomy 21 and Alzheimer’s Disease, we propose professionals from the Down syndrome community should be part of NAPA Council subcommittees, so we can more readily help each other.
It is very common that people caring for Alzheimer’s patients in their homes are children caring for their parents. With Down syndrome the opposite is the case. As shared earlier, we were able to provide for Mia’s needs in our home. We were old enough to be retired, but young and strong enough for the very physical work of caregiving. That is not always the situation. Many parents caring for their children with Down syndrome and Alzheimer’s are significantly older care providers. The caregiving experience is more likely to be detrimental to the health of more senior caregivers, unlike what we experienced as the relatively young care providers for Mia. We have a request. Please recognize the full-time nature of older parents caring for their adult children. Provide these caregivers support and respite services.
We have additional concerns:
People with Down syndrome make up the largest group of early onset Alzheimer’s disease patients. Therefore, it is important for research and treatment studies to include persons with Down syndrome. It is important for clinical drug trials to include persons with Down syndrome. It is important to include professionals with knowledge of Down syndrome in all aspects of the Alzheimer’s disease conversation.
As for our story, while we were aware people with Down syndrome could develop Alzheimer’s as adults, it wasn’t really on our radar. Mia was so vital and so healthy--and so young.
We appreciate the opportunity to be heard. Thank you!
C. DeMatteis 5-9-2023
Since the first National Plan to Address Alzheimer’s disease was published in 2012, the ambitious goal to “prevent and effectively treat Alzheimer’s disease and related dementias by 2025” has been a major focus. In 2012, the main roadblock was the science. In fact, some researchers criticized the goal of 2025 as too ambitious given that we were "just beginning to understand the disease."
Now, we do have treatments that delay progression and we have a rich pipeline of research that promises more--making that ambitious goal a reality within our reach. BUT only if people have access.
The major problem is not a lack of science needed to unlock the mind to treatment, the problem is unlocking the minds of people at CMS who are blocking people’s access to the first ever FDA-approved treatments for Alzheimer’s that actually interrupt disease progression.
The National Plan did not anticipate the erection of barriers to access by Medicare. Treatments decades in the making, evaluated and approved as safe and effective by the FDA, that delay the progression of illness are being celebrated widely among physician experts--including many here today--as well as ordinary people dealing daily with this cruel, fatal illness. Yet, Medicare’s actions to restrict coverage and erect access barriers are a significant blow to meeting the 2025 goal and related goals to eliminate disparities.
As you move forward, we urge you to encourage the Biden Administration to reconsider its decision to require coverage with evidence development that blocks access to these treatments and delays progress. In the course of today’s meeting, 2,000 more people will progress beyond the reach of these new treatments. The National Plan is not concerned with our ability to prevent or effectively treat Alzheimer’s disease and eliminate disparities, but on our actually doing those things.
Thank you for all you are doing to make this possible.
S. Keller 5-8-2023
Hi, I appreciate the opportunity in being able to speak to the NAPA council today about the assessment of cognitive functioning in the neuroatypical population for signs of cognitive decline.
I am a neurologist and co-president of the NTG, the National Task Group on Intellectual Disabilities and Dementia Practices, and past president of the American Academy of Developmental Medicine and Dentistry as well as a leader in the American Academy of Neurology’s Section on Adults with IDD.
Being able to assess, diagnosis and ultimately care and support adults with dementia as we all know is a huge growing issue and concern and need. The advent of new anti-amyloid therapies is exciting and the future is brighter for those who may develop Alzheimer’s disease. The annual wellness visit has recommendations that a cognitive assessment be performed to help with this assessment of possible decline.
There are however, millions of Americans who are considered to be neuroatypical, as they have inherent comprehension, oral communication difficulties, motor task performance impediments, or recognition of visuals. Because of these various difficulties the often used standard office based assessment tools such as the MMSE and MOCA, are not able to adequately and accurately determine the true presence or not of cognitive decline. Being that there are certain populations that have an extraordinary high prevalence of early onset AD such as those with Down syndrome, it is imperative that an accurate assessment be caried out as soon as possible to help determine whether MCI and dementia has begun.
Due to these concerns, an expert panel was created under the auspices of the NTG and LuMind IDSC, I was one of the panelists who was involved.
The panel’s report titled Examining Adults with Neuroatypical Conditions for MCI/Dementia During Cognitive Impairment Assessments--Report of the Neuroatypical Conditions Expert Consultative Panel, was published in 2022. www.the-ntg.org.
Some of the conditions that were included in the report included:
- Acquired/Traumatic Brain Injury
- Autism Spectrum Disorder
- Cerebral Palsy
- Down Syndrome
- Intellectual Disability
- Intellectual Disability with Dual Diagnosis with a Mental Health Condition
- Serious Mental Illness
- Significant Vision/Hearing Impairments
The panel found that many of those neuroatypical conditions posed significant barriers for early detection and assessment. This mostly happened when the primary conditions masked changes in cognitive functioning or in areas where clinicians weren’t knowledgeable enough of assessment nuances among adults with such conditions. The panel also found that adults with neuroatypical conditions (NAC’s) faced a variety of barriers to being accurately examined and having determinations made about whether they had a new or additional cognitive impairment. Further, the panel recognized that most clinicians have trouble discriminating between current and pre-existing behavior and function, especially in conditions with long-term cognitive deficits.
Expert Panel recommendations included: (1) broadening federal guidance to include adaptations of assessment practices to accommodate NACs; (2) enhancing education for clinicians about NACs and how to detect and diagnose MCI or dementia; and (3) expanding research to produce more evidence-based information on assessing NACs for later life adult cognitive diseases/disorders and for planning subsequent post-diagnostic care.
Without an ability to assess these neuroatypicals, they are being left out and unfairly denied the ability of receiving a accurate and timely diagnosis as well as the necessary care, supports and therapeutics that they deserve. The clock is sadly tickly very rapidly for many and time is not on our side, so urgency is needed to address these inequities in care.
H. Hillerstrom 5-4-2023
I am the father of a lovely 9-year-old son Oskar who has Down syndrome. Professionally, I previously co-founded and was an executive at an Alzheimer’s biotech company and I am presently the President & CEO of LuMind IDSC Foundation. LuMind IDSC is a leading national non-profit organization accelerating Down syndrome research to increase availability of therapeutic, diagnostic, and medical care options and empowering families through education, connections, and support. LuMind IDSC is closely connected to 300,000 families in our online community.
Today, I want to speak to the Council about Down syndrome and Alzheimer’s disease. Adults with Down syndrome (DS) are genetically predisposed towards the early onset amyloid deposition in the brain and face a dramatically increased lifetime risk for Alzheimer’s disease (AD), called Down syndrome associated AD (DS-AD). All individuals with DS, with rare exceptions, show cerebral amyloid accumulation by the age of 40, and onset for dementia symptoms at a mean age of 53.8. The risk of developing DS-AD is more than 95% by age 68, and it is the leading cause of death of adults over the age of 35 in this population, accounting for approximately 79% of deaths.
Given this, we are very excited that several anti-amyloid immunotherapies were recently approved by the FDA and more drugs with promising data might follow suit. We believe these disease modifying therapies represent great advances for the field of Alzheimer’s. However, it is vitally important for our high-risk population to have equitable and unfettered access to newly authorized antiamyloid Alzheimer's therapeutics as they become available.
A first issue is that the payers’ current eligibility criteria for access to newly authorized Alzheimer's therapeutics in the general population inadvertently exclude people with DS-AD. The criteria for patients with Late Onset Alzheimer’s Disease (LOAD) focus on age, exclusion of non-Alzheimer’s causes for dementia, demonstrated cognitive impairment due to mild cognitive impairment or mild Alzheimer's disease, and biomarker indicators of amyloid plaques presence.
Instruments recommended to identify dementia in the LOAD population may not sufficiently be sensitive to accurately detect early stages of the disease in individuals with Down syndrome. Some of the other criteria are also problematic as they create a barrier for inclusion and need to be adapted for adults with Down syndrome. To address this, we have therefore brought together a panel of 20 Down syndrome and Alzheimer’s experts to generate an equivalency consensus statement designed to serve as a guide for determining eligibility for adults with Down syndrome by offering alternative measures, criteria, and processes. We expect to be able to share the final statement with the Council in about a month.
A second issue is that to date, estimates suggest that no adults with Down syndrome were included amongst the 6,000 or more participants in clinical trials of Aduhelm, Leqembi, and donanemab, and a delay of up to 14 years in access to these three promising therapeutics for early Alzheimer’s disease is the result. This delayed access means missed opportunities for treatment mitigation for almost a whole generation of older age adults with DS. It is critical that safety trials with these therapies are conducted as soon as possible in adults with Down syndrome so that we can reduce the wait time for accessing these medications.
On behalf of the Down syndrome community, I ask the Council for its advocacy in the following areas:
- That barriers for accessing the latest Alzheimer’s therapies are removed at the national and state levels and that accommodations are made in instruments and procedure recommendations so that people with Down syndrome have equity in access to these new medications,
- That NAPA Plan updates provide specific supports for the Down syndrome community to have access to these life-lengthening medications, and
- That the Council adopts actions to advocate for and support the conduct of needed safety studies with anti-amyloid immunotherapies in adults with Down syndrome.
For my son Oskar, and, more importantly, for the thousands of adults with Down syndrome that are turning 30, 40 or 50 years old every year, please don’t disregard our population and do everything in your power to ensure speedy and equitable treatment access to them.
L. Norins 4-11-2023
As is required by all reputable medical journals, physicians and scientists attending or submitting papers or comments to the Advisory Committee must append a footnote disclosing any possible conflicts of interest they have through having received any monies whatsoever from drug companies sponsoring any drug. If there are no possible conflicts whatsoever, the submitter merely notes: "No conflicts to report". If this requirement is not already in force at Advisory Committee meetings, I urge it be immediately implemented to preserve the credibility of the Committee.