What is the POWERHD Data Collection Program (DCP)?

The POWERHD Data Collection Program (DCP) collects and stores de-identified health-related information (data) about patients with rare diseases/disorders. With your permission, your de-identified data will be shared with researchers and patient organizations associated with Huntington’s disease world-wide.

What will be asked of patients participating in the POWERHD Data Collection Program?

The first step is setting up an account, followed by answering a set of questions (survey) regarding the patient’s health history, and treatment. Over time additional surveys will be added and you will be notified of future surveys relevant to you.

Why is this important to Huntington’s Disease?

Participation in this DCP may…

• Inform researchers how Huntington’s disease progresses over time 

• Enable better data to use in clinical trials

• Reduce the time it takes to study new medicines in clinical trials

• Speed up the time to get therapeutics to patients

• Enable the use of data as a placebo (instead of actual patients) in a clinical trial

Who can participate?

Upon account creation, you will be asked to identify as a Patient Participant, Caregiver Participant or A Person Who Has Lost a Loved One. These are parameters set to help the platform analyze and compare data from many different rare diseases; however, we know those terms may not be encompassing of how you identify being impacted by Huntington’s disease. Below are descriptions on how you may consider participating.

• Patient:
  • Have been clinically diagnosed with symptoms related to Huntington’s disease
  • Gene-positive and believe your symptoms have begun but haven’t been formally diagnosed
  • Gene-positive without symptoms
  • At-risk and untested but have a biological parent or sibling who is either diagnosed with HD or gene positive for HD
    

• Caregiver:

  • Primary or secondary caregiver who has access to and knowledge of their loved one’s experience and medical information with HD.

• Lost a Loved One:

  • Any individual 18 & older who has lost a loved one to HD and who has medical information about their loved one’s experience with HD.

What if I identify as a part of multiple relationships to HD as previously listed? 

The POWERHD study values and enables community members who have multiple relationships to HD. Due to the family nature of this condition, this scenario is very common. For example, someone might be at risk of developing HD (and therefore be a “patient” in the study) but also be a caregiver of someone affected with HD. Each individual who participates in POWERHD only needs to register once, but then can choose multiple roles.

Who is a Research Partner?

A research partner is someone who can provide information about a patient’s journey with HD. Information given by the research partner can provide critical observations about the way that HD impacts the patient’s physical functioning, emotional health, and activities of daily living. A research partner can be:

• A Family Member

• A Friend or Roomate

• A person you interact with regularly (3-4 times a week) 

• A person that is able to observe your health and behaviors

NOTE: Participants can NOT choose Research partner as one of their roles. A “patient” (whether that person does or does not have symptoms) must choose to invite someone to be their research partner. Our study team will help register the research partner after they have been invited. 

Can I have a RARE-X “caregiver” role and a research Research Partner role? 

Yes. But not for the same person…

A “Caregiver” on RARE-X is someone who is entering data on behalf of a patient who is not cognitively able to consent for themselves. 

• If you are a caregiver and you are enrolling a patient who cannot consent for themselves, you will enroll as a caregiver on RARE-X and then add your patient. You will not be able to also be their Research Partner in this case, because you will already be answering surveys on behalf of the patient in your caregiver role. 
• If the patient can consent for themselves, they will enroll as a patient and then invite you as a Research Partner. You will not have a “Caregiver” role in RARE-X if this is the case. 
• If you need to help the patient enroll physically (by reading to them, typing for them, etc) but they can consent for themselves and are answering the survey questions on their own (online or out loud to you) please help to enroll them as a patient.
• You might be the caregiver for one person with HD (example: your spouse, who can no longer cognitively consent and answer their own questions) and also be designated as the research partner for someone else (example: your 20 year-old child who is at risk, and lives with you). In this case, you can enroll as the caregiver for your spouse, and your child can invite you to be their research partner.  

How is the POWERHD data different from the data collected with Enroll-HD?

Enroll-HD collects some of the same data that the POWERHD Study collects, such as demographics, genetic data, and current medications. In addition, both studies measure the impact HD has on an individual’s quality of life and every day activities. Differences between the studies include:

• In POWERHD, all of the data is reported or provided by individuals impacted by HD directly. The POWERHD platform has more patient-reported and caregiver-reported research surveys, which make it easy for families to participate from home.  These research surveys provide a way to collect the patient and family experience in a quantifiable way, which can be tracked over time.
• In the Enroll-HD study, physicians and psychologists measure and rate patient’s physical and cognitive capabilities. 

POWERHD is not intended to replace Enroll-HD. These are complementary studies, and we intend to link data from these studies in the future. 

If you participate in Enroll-HD and POWERHD, will my data be connected to my Enroll-HD data?

We plan to implement a way to connect the data from these two studies in the near future. When that happens, we will ask participants in the POWERHD study for their permission. Data will only be connected with participant permission. Linkage between studies will happen using the “HDID”, a unique participant identifying number currently generated by CHDI and the HD Centers of Excellence. 

How will I be notified of New Surveys for RARE-X?

RARE-X has automatic notifications that will send you an email once new surveys are available on your dashboard. You will also receive automatic notifications when it is time for you to retake a survey. All emails will come from RARE-X.  Note that some surveys were initially developed for one specific disease community, but have since been used across many other conditions. The names of some of the surveys assigned to you might seem like they do not apply to you or your family, but they do! Surveys are selected and recommended by expert groups based on their content. Individuals in the HD community might receive a survey that was initially developed for Parkinson’s Disease or Spinal Muscular Atrophy, but the content is useful for HD as well. 

How will data be kept confidential?

Your privacy is very important. Name and other personal identifiers will be removed from the data and replaced with a unique code number before data is shared with researchers. Data is encrypted (scrambled) when stored for added security.

Where is the data stored?

The data is securely stored on the RARE-X data platform and made available to researchers and patient organizations.

Who owns the data?

Patients/family members who contribute their data to the Data Collection Program own and manage their data, including who has access to it and how it’s shared. 

What choices are there for data sharing?

You will be able to choose to share de-identified data with specific types of researchers (ie. all researchers, just biomedical researchers, researchers from Biopharma, etc.)

You may choose to share identified data (name and email) with patient organizations so they may connect with you. 

How long will the data be stored?

There is no set time limit on how long this Program will store data for future research.

Will patients/guardians be contacted for clinical trials?

It is your choice if you would like to be contacted for possible clinical trials. 

Can answers be updated over time?

Yes

Do I have to answer all the questions at once or can I leave and finish later?

You can always leave and return to complete surveys later.

Will my data be sold?

No

Can patients quit this Program?

Yes, patients can stop taking part in this Program at any time for any reason. RARE-X has a process in place to remove the patients data if they chose so. If you would like to stop participating in the POWERHD Program, please email rarexsupport@globalgenes.org.

Is this program a United States only initiative or is it global?

This program is a GLOBAL data collection program. Over time additional translations will be rolled out.  

What is the best web browser for accessing the Data Collection Program ?

The best web browsers to use for the Data Collection Program are Google Chrome or Apple Safari. Do not use Microsoft Edge or Internet Explorer.

What if I have questions or need to troubleshoot?

If you have questions about the POWERHD Program or consent please contact the research staff by sending an email to rarexsupport@globalgenes.org.

Additional Helpful Definitions 

• DCP: Data Collection Platform 

• Data: Data delivered to researchers or Patient Advocacy Groups is de-identified ALWAYS – identifying information removed

• Researcher: All types of researchers, from academia to industry