We understand that navigating the CMS public comment portal can be challenging and may not fully meet accessibility standards. To assist you, we have created a comprehensive guide designed to make the process smoother. This guide will help you access the portal and provide constructive suggestions for addressing each question you may encounter. You are free to use this guide as you wish—whether that means responding on your own or adapting the ideas that resonate with you. There is no pressure to answer every question; please contribute in a way that feels most comfortable for you. As always, please do not give out any personal or identifying information.
Remember, there are no wrong answers. The important thing is that you participate, answer the questions you feel comfortable with, and take action!
Here are some highlights from the Help 4 HD and HDYO talk on February 7, 2025, that you may want to keep in mind. You will also find a template for the questions below. You can download this template for easy copying and pasting where needed, if you choose.
Please go to: Public Submission Form for Information about Selected Drugs and Their Therapeutic Alternatives to enter your email for access into the public submission portal.
Once you enter in your email you will receive an email with a direct personal link for you to enter into the portal.
When you go to the link you will be asked a couple questions to get you to the right place for you to start answering questions in narrative form. Please note: Do not give out any personal or identifying information.
Below you will find our template to help guide you or give you some ideas for each question. Some of these questions are confusing and very redundant. This is not your imagination; this is not a very clear portal, and they made it very hard for patients and caregivers to navigate.
CMS Questionnaire Template
Things to consider:
- You do not have to be prescribed Austedo to participate in this feedback. It’s asking about experiences unrelated to the drug being considered.
- It is ok if you don’t answer every question. Do not feel pressured to answer anything that you are unsure of or do not have any experience with.
Getting Started:
There are loose guidelines for how they define patient versus caregiver. With Huntington’s disease, there are many different ways you or loved ones may identify within the community. Ultimately, it’s up to you. Here are some possibilities:
Patient:
- Individuals who personally are experiencing symptoms from HD
- Individuals who are gene positive and are in the presymptomatic/prodromal stage of HD
- Individuals At-Risk of HD
- Caregivers who are answering on behalf of a loved one with HD
Caregiver:
- Anyone who at one point provided care or support for someone with Huntington’s disease
Here is the template we created above typed out for this blog. To see the template, you can download the template above.
Q36a: Have you or someone you provide care for ever taken [the selected drug]?
Caregiver & Patient
· Yes
Q36a1: [If YES] For which condition(s) (including FDA-approved indication(s) or off-label use as defined in the instructions) was [the selected drug] taken?
Caregiver & Patient
· Austedo for a treatment for the symptom of chorea related to Huntington’s disease
Q36a2: [If YES] When were you or someone you provide care for given a diagnosis related to this condition or conditions? You may write an approximate date, or if you never received a diagnosis write “N/A.”
Caregiver
· My loved one was diagnosed in (add year).
Patient
· I have been clinically diagnosed with Huntington’s Disease since (month/year). I received the clinical diagnosis from a medical provider after meeting all criteria.
· I have gone through genetic testing and have tested positive for the HD gene (month/year).
· I’m untested for Huntington’s disease but carry a risk (50/50 or 25%) of inheriting the gene.
Q36a3: [If NO] What condition(s) (including FDA-approved indication(s) or off-label use as defined in the instructions) treated by [the selected drug] would you like to provide input on?
Caregiver
· Austedo for a treatment for the symptom of chorea related to Huntington’s disease
Patient
· I would like to provide input on living with chorea associated with Huntington’s disease treated by Austedo.
Q36a4: [If NO] What is your experience with this condition or conditions?
Caregiver
· I am a caregiver caring for someone with Huntington’s disease
Patient
· As a person living with Huntington’s Disease, I experience symptoms of chorea.
· I have experienced symptoms of chorea since (month/year).
· Chorea causes involuntary movements that affect my whole body.
· I have not developed symptoms, yet, but I understand how chorea can impact daily life which may happen in my future.
Q37: Information on Your Condition(s) or Condition(s) of Someone You Care For
Caregiver
· My loved one is facing the harsh reality of living with Huntington’s disease, a neurodegenerative condition that is both genetic and terminal.
· This illness leads to severe involuntary movements, cognitive decline, and various psychiatric issues.
· As the disease progresses, my loved one will lose the ability to work, drive, care for themselves and others, walk, dress, bathe, manage medications, communicate, eat, and swallow.
· Huntington’s disease strips away not only their independence but also their quality of life, forcing them to rely entirely on caregivers.
· Currently, there are no therapies or treatments available to slow or halt the progression of Huntington’s disease.
· Families like mine are left to manage symptoms, striving to maintain quality of life and comfort for our loved ones for as long as we can.
· This reality demands our full attention and unwavering commitment to support those affected by this devastating disease.
· Chorea movements due to Huntington’s disease cause my loved one to struggle with getting dressed, feeding themselves, speaking, swallowing, and many other daily activities.
· Chorea also causes them to get injuries from falls and also from their movements that are uncontrolled.
· Chorea causes them to become more dependent on me.
· Chorea causes them to lose independence.
Patient
· I am facing the harsh reality of living with Huntington’s disease, a neurodegenerative condition that is both genetic and terminal.
· This illness leads to severe involuntary movements, cognitive decline, and various psychiatric issues.
· As the disease progresses, you continue to lose your ability to manage all aspects of independent functioning until you are entirely reliant on caregivers.
· Currently, there are no therapies or treatments available to slow or halt the progression of Huntington’s disease.
· Families like mine are left to manage symptoms, striving to maintain quality of life and comfort as long as we can.
Q37a: How do the condition(s) you listed in Q36a1 impact your daily life and well-being or the daily life and well-being of someone you provide care for?
Caregiver
· Huntington’s disease has a profound impact on daily life and well-being.
· Living in constant fear, knowing that my loved one will eventually lose all their abilities and become entirely dependent on me, is a heavy burden.
· Financial uncertainty looms over us, as I know my loved one will lose their ability to work, and I will too when full-time caregiving becomes necessary.
· With every new symptom or loss of ability, we grieve what was once normal, and just when we seem to adjust to this new reality, another symptom emerges, shifting everything again.
· We are trapped in a cycle of uncertainty, never knowing when the next symptom will strike or how we will maintain the benefits we have.
· The next generation watches, acutely aware that they may inherit the same fate as their parent, which creates generational trauma that cannot be ignored.
· We lead lives filled with uncertainty, never knowing when the next symptom will strike or how we will maintain the benefits we have.
· The struggle to secure new benefits is a time-consuming battle that adds to our stress.
· Our priority is to preserve as much quality of life as possible for our loved ones before they pass, or at the very least, ensure their comfort throughout their journey with this devastating disease.
· It is a fight that is exhausting and often feels unnecessary, like getting benefits for our loved ones that they qualify for and should have immediate access to.
· HD has taken so much from us without a therapy in sight.
· This disease has taken so much from us physically, financially, emotionally, and spiritually, generation after generation.
· The changes that this disease has created are devastating.
· Being surrounded by HD every day has caused my own health to be hard to manage.
· We are living in a world with little knowledge and awareness of the disease. Isolation and loneliness can consume people impacted by HD.
List of some symptoms and impact due to the disease:
· Cognitive decline: thinking through problem-solving, managing household chores, managing finances, knowing what is safe for them or their children, multitasking, knowing what is best for them, delayed responses, lack of flexibility or getting stuck on a thought, lack of awareness of behaviors, trouble learning new information, difficulties retaining old information.
· Movements/chorea: injuries that include falls, eventual loss of speech, swallowing issues that can lead to choking and aspiration, difficulties with fine motor skills that make getting dressed difficult or impossible, spilling of food, drinks, etc.
· Psychiatric components: depression and anxiety, unstable moods, anger/aggression, hypersexuality, hallucinations, perseveration, OCD tendencies, apathy.
· Other things to take into consideration: sleep disturbance, social withdrawal, suicide ideation and action, loss of energy and fatigue, and Gastrologic and Urological issues, homelessness and displacement.
Patient
· Huntington’s Disease is a neuro-degenerative condition affecting multiple parts of the brain over time.
· Symptoms include severe involuntary movements (chorea), cognitive decline, and psychiatric issues.
· Chorea impacts Instrumental Activities of Daily Living (IADLs) such as cooking, cleaning, medication management, and navigating environments without assistive devices or caregivers.
· Transportation becomes difficult due to inability to operate a vehicle or safely use public transportation.
· Chorea also affects Activities of Daily Living (ADLs) such as bathing, dressing, grooming, toileting, and transferring, increasing the risk of falls and injuries.
· Dysphagia (difficulty swallowing) caused by chorea increases the risk of choking and aspiration pneumonia.
· Cognitive decline negatively impacts daily living and well-being, affecting employment, financial management, and independent living.
· Executive functioning issues lead to problems with planning, multitasking, inhibition, and can cause behavioral and psychiatric issues.
· Individuals with Huntington’s Disease often require increasing assistance as the disease progresses, relying on family caregivers or governmental support such as Medicaid, Medicare, SSI, SSDI, and Long-Term Care.
· Access to treatments for chorea can positively impact the symptomatic individual and their caregivers by slowing the loss of independence.
· Maintaining as much independence as possible is crucial for both the individual and their family, although there is currently no cure for Huntington’s Disease.
· For families, accessing medications can significantly help, especially if the primary caregiver also works, allowing them to continue their employment while providing care.
Q37b: How has the condition(s) you listed in Q36 changed or progressed over time?
Caregiver
· Huntington’s Disease is a progressive disease, with symptoms and severity increasing over time.
· The condition has evolved from complete independence to an inability to manage finances, prepare food, drive, or communicate efficiently.
· Progression includes a significant level of apathy, leading to withdrawal from family events and conversations.
· Symptoms and abilities deteriorate step by step, leading to a gradual loss of interests and capabilities.
Q37c: What is important to you or those you provide care for in managing the condition(s) you listed in Q36?
Caregiver
· With no cure, therapy, or treatment, we hold tight to what we have.
· If we can treat one symptom, it is extremely beneficial to us.
· For us, chorea has created a lot of issues in day-to-day life, including public impact, swallowing, injuries, etc.
· We need to be able to manage this symptom for their quality of life.
· Managing symptoms of Huntington’s Disease is all that we can do.
· We need to manage each individual symptom with medications and things like physical and occupational therapy.
· Managing the impact of chorea helps our days be less difficult and stressful.
Patient
· Access to quality healthcare and medication management is imperative.
· There is no cure or treatment to delay Huntington’s Disease.
· Only three medications are approved by the FDA to treat the symptoms of chorea.
· All other symptoms are managed through various medications with a trial-and-error approach, often requiring continuous changes to the regime.
· With additional restrictions on drug formularies, treatment options become even further limited.
Q37d: What challenges do you, or someone you care for, face in managing this condition(s)?
Caregiver
· Everyone with HD is different. Chorea has been stressful for us because everything spills, things break, my loved one doesn’t fully recognize their chorea, and it causes them to do things that physically they shouldn’t be doing. This stresses me out. They bang into walls, windows, doorways. They have broken so many pieces of furniture including toilets from aggressive movements. Our challenges consist of keeping my loved one as safe as possible, while continuing to support their independence.
· Managing Huntington’s disease is complicated and forever evolving. The challenges are different today than they were when they first tested. Cognitive challenges started, and chorea followed, this combination of symptoms makes it hard to have a daily plan because each day is a struggle in different ways. Some days they can help while showering, other days they refuse a shower and sometimes they can’t help at all. This is one example of our struggle.
· Our challenge is that I cannot stay home 24/7 with my loved one because I have to work. Due to chorea, I often come home to messes, broken items, and sometimes even injuries. This is scary and we don’t have a solution. Another challenge we face is that my children are at risk for this disease and we will need to work through all of this again.
· Being a young caregiver can be extremely challenging. I often have been forced to become an adult earlier that I have expected helping with healthcare needs, financial hardships and management, living in uncertainty of my own risk, giving up opportunities like school or work to care for my loved one, etc. We struggle with helping my loved one understand that they need additional help, and they often refuse because they are not always aware of their symptoms.
Patient
· Several challenges exist.
· One major challenge is finding providers who offer specialty care for Huntington’s Disease within a reasonable traveling distance.
· Huntington’s Disease does not have a cure, and only three drugs are FDA approved to manage chorea.
· Medication management is crucial but difficult to maintain due to the variety of symptoms presenting risks.
· Some medications may help with chorea but worsen psychiatric symptoms.
· No medications are FDA approved for the Cognitive Decline and Psychiatric Behaviors caused by Huntington’s Disease, making the treatment trial and error.
· Restrictions on medication formularies further limit providers, resulting in unmanaged symptoms.
· Unmanaged symptoms pose a greater risk for crisis situations, including medical and psychiatric hospitalizations, involvement with Law Enforcement, and disruptions in home placement due to safety risks.
· Even though I will eventually develop symptoms, because I currently don’t have chorea, I may not be able to visit a neurologist to help manage any underlying progression unseen by the human eye or in current measuring tools.
Q38: Information on the Current Medication to Treat Your Condition
Caregiver & Patient
· As of today, the FDA has approved only three medications for Huntington's disease.
· Primarily two of these treatments are widely used.
· The first drug, now available as a generic, is generally less effective for most patients compared to the other two approved drugs, which are not generic.
· All three medications are intended for symptom management.
· The drug addresses one symptom of HD but does not slow the progression of the disease.
Q38a: Are you, or someone you care for, currently taking medication(s) to manage the condition(s) you listed in Q37?
Caregiver
· Everyone with HD is different.
· Chorea has been stressful for us because everything spills, things break, my loved one doesn’t fully recognize their chorea and it causes them to do things that physically they shouldn’t be doing.
· This stresses me out.
· They bang into walls, windows, doorways.
· They have broken so many pieces of furniture including toilets from aggressive movements.
· Our challenges consist of keeping my loved one as safe as possible, while continuing to support their independence.
· Managing Huntington’s disease is complicated and forever evolving.
· The challenges are different today than they were when they first tested.
· Cognitive challenges started, and chorea followed, this combination of symptoms makes it hard to have a daily plan because each day is a struggle in different ways.
· Some days they can help while showering, other days they refuse a shower and sometimes they can’t help at all. This is one example of our struggle.
· Our challenge is that I cannot stay home 24/7 with my loved one because I have to work.
· Due to chorea, I often come home to messes, broken items, and sometimes even injuries.
· This is scary and we don’t have a solution.
· Another challenge we face is that my children are at risk for this disease and we will need to work through all of this again.
Q38a1: [If YES] What medication(s) are you, or someone you provide care for, currently taking to manage the condition(s) you listed in Q36?
Caregiver
· Here are SOME medications commonly used to treat symptoms of HD: Austedo, Ingrezza, Clonazepam, Zyprexa, Bupropion, Scopolamine, Triazolam, Temazepam, Paroxetine, Omeprazole, Valproate, Vyvanse, Depakote, Risperidone, Haldol, Valproic acid, Seroquel, Tegretol.
· Here is where you can list the medications your loved one takes.
Q38a2: [If YES] How did you or someone you care for decide to start taking the medication(s) currently used to manage the condition(s) you listed in Q36?
Caregiver
· When talking to our doctor, they recommended medications that would help manage their symptoms.
· We were told about Austedo during a family education day and brought information to our doctor to see if it would benefit my loved one’s chorea.
Q38a3: [If YES] What has been your experience, or the experience of someone you provide care for, with the medication(s) currently used to manage the condition(s) you listed in Q36?
Caregiver
· Our experience has been ever changing, they were on medications prior to testing, and we keep needing to add to the list.
· My loved one is on Austedo for movements, this is the only medication due to Huntington’s Disease that they are on and have been on this medication since it came out.
Q38: Information on the Current Medication to Treat Your Condition
Caregiver/Patient
· List current medications you or your loved one are on.
Q38a4: [If YES] How satisfied are you, or someone you care for, with the medication(s) you take now to manage your condition(s)?
Caregiver
· Relief with medications prescribed, but symptoms still present.
· Austedo has greatly improved quality of life by reducing spills, falls, and the need for physical assistance.
· Medication list changes frequently due to changing symptoms.
· Symptoms are managed decently, but better control is desired.
· New medications are often added to the list as symptoms change.
· Some medications have side effects, requiring new prescriptions to offset them.
Q39: Information on the Medication(s) Used in the Past to Treat Your Condition
Caregiver
· List medications you have used in the past.
Q39a: Have you, or someone you care for, taken other medication(s) in the past to manage the condition(s) you listed in Q38?
Caregiver
· Yes or No
Q39b1: [If YES] What medication(s) have you, or someone you care for, taken in the past to manage the condition(s) you listed in Q36?
Caregiver
· List past medications you have used.
Q39b2: [If YES] How did you, or someone you care for, decide to start taking the medication(s) used in the past to manage the condition(s) you listed in Q36?
Caregiver
· We wanted to try the newer version of a medication that treats chorea because it was said to be improved and also had fewer side effects.
· We have found that medication formulas work differently for us than for others. Even though it is the same class of drug to treat a symptom in the same way, we experience different side effects from the same class of drug.
· We want the option to try and have access to all drugs that treat symptoms of HD, so if we have a side effect to one drug, we can switch fairly quickly without too much disruption in our lives.
· Fighting with insurances and being denied coverage is a terrible process. We are always trying to appeal, which is a stressor that caregivers or patients should not have to take on.
· It is an unnecessary burden on families that we constantly have to put up with, especially with payees.
Q39b3: [If YES] What was your experience, or the experience of someone you provide care for, with the medication(s) used in the past to manage the condition(s) you listed in Q36?
Caregiver/Patient
· Talk a little about your personal experience on any treatments you listed on question Q36.
Q39b4: [If YES] Why did you, or someone you provide care for, stop taking the medication(s) used in the past to manage the condition(s) you listed in Q36?
Caregiver/Patient
· Some reasons people stop taking medications include side effects, accessibility issues, high copayments, difficulty swallowing, changes in health status, reduced effectiveness over time, loss of income, and loss of insurance.
Q40: What other information about the condition(s) you have identified, or the medication(s) used to manage these condition(s) do you think CMS should consider while evaluating [the selected drug]?
Caregiver
· I am worried that having Austedo on this list will create an unhealthy environment for innovation and research and development, both things we need.
· HD is the only neurodegenerative, terminal disease on the list of diseases that are represented in your negotiation list. If this negotiation depletes further research development, even with Teva, we are stranded alone with no other options at this time. We don’t have any way to combat this disease really. Consider that when you put drugs up for negotiation.
· We already have limited research and development in our rare disease space. Our fear is that rare diseases coming onto these CMS lists for negation will further deplete efforts that go towards research development in HD.
· We are concerned that this new negotiation may lead to a reformulation of our current medication. This could result in my loved one experiencing more side effects, and there is a possibility that the treatment could become less effective or even completely ineffective.
· Forcing individuals to utilize a “generic drug” can have significant negative consequences, including adverse side effects and medication interactions.
·
Patient
· It is important to evaluate the totality of Huntington’s Disease and its overall impact.
· Huntington’s Disease is a rare, terminal neurodegenerative disease with multiple symptoms, including Chorea.
· People with Huntington’s Disease should have the ability to choose what best treats their symptoms, not insurance companies.
· Forcing individuals to utilize a “generic drug” can have significant negative consequences, including adverse side effects and medication interactions.
· Huntington’s Disease affects individuals nationwide, and much of the healthcare system is still unaware of the disease or how to appropriately treat patients in crisis.
· This burden will impact not only individuals and families but also multiple levels of government.
· This trend will continue until a cure or ongoing treatment is found due to the genetic expansion component, which introduces previously unaffected families to Huntington’s Disease in the first generation.
· This factor results in three to four generations of at-risk offspring before an individual even shows symptoms to receive a diagnosis.
Q41: Provide up to 10 visual representations, if any, such as tables, charts, and/or graphs that support the responses to Questions 36 through 40. Indicate which question each file corresponds to.
Caregiver/Patients
· Consider attaching pictures of injuries, falls, and other impacts to your or your loved ones life due to Huntington’s disease.
Q42: Demographic Questions
Caregiver/Patient
· Age (i.e., 18-24 years, 25-34 years, … , 100 years or older)
· Race/Ethnicity (i.e., American Indian or Alaska Native, Asian, … , Other not Listed)
· Regional Location (i.e., New England: CT, ME, MA, NH, RI, VT; ... ; Other)
· Medicare Beneficiary