Your experience could provide key insights for PK Deficiency research

PicnicHealth works directly with people like you to help collect and manage your medical records. That means going out and collecting all of your medical records from your doctors and keeping them up-to-date over time. PicnicHealth will then transform those records into a timeline that makes it easier to find what you’re looking for. When you see your doctor again, PicnicHealth will update your timeline with new records. You can access your PicnicHealth timeline from anywhere and even share it with your doctors if you would like to with a few clicks.

When you sign up for PicnicHealth, they will create a timeline of your medical records. Your PicnicHealth timeline provides a single source of information for you and your doctors. It is a tool to help you coordinate your care and understand your health. You can share your timeline with any of your doctors with a few clicks. The doctor will receive a secure link and get access to your medical history if you choose to share it with them.

PicnicHealth and Agios take privacy very seriously. Your medical records are kept safe using the highest encryption standard available: 256-bit SSL. This is the same technology that banks use to keep account information safe.

The Pyruvate Kinase (PK) Deficiency Real World Observational Study is a study sponsored by Agios Pharmaceuticals to help researchers learn more about pyruvate kinase deficiency.

Adults and children diagnosed with PK deficiency who live and receive medical care in the United States can participate in the study.

Unfortunately, anyone who is part of an ongoing mitapivat clinical trial will not be able to participate in the study.

We also want to learn more about the impact that PK Deficiency has on caregivers. If you are a family member providing care or assistance for someone living with PK deficiency, whether they are an adult or a child, you are eligible to sign-up for this study as a caregiver. Caregivers may only sign up if a patient signs up.

Medical records document what’s actually happening when doctors see patients, so they can be really helpful for understanding how care is delivered. They also contain a lot of information about a patient’s condition, like PK Deficiency, health status, and care. Putting all of this information together for a diverse group of patients will allow the scientific community to see patterns that no one doctor can see on his or her own individual patient, enabling the community to better care for individuals living with PK Deficiency.

PicnicHealth will collect medical records from all the doctors, hospitals, and clinics that you tell us about. PicnicHealth does not collect dental or optometry records.

As part of this study, data will be extracted from your medical records in a manner that ensures that your medical and personal information is kept confidential. To do this, PicnicHealth will assign you a unique patient identification number that will be used on any of your medical data that is used for this study, or in any other study. Data will not include your name, picture, address, or any other personally identifying information.

Please review the PicnicHealth Privacy Pledge and Agios Privacy Notice for more information.

PicnicHealth will never share your records without your explicit consent.

By signing up for the PK Deficiency Real World Observational Study and the PicnicHealth application, you're agreeing to share your de-identified medical records collected by PicnicHealth in accordance with the consent provided in relation to the PK Deficiency Real World Observational Study.

PicnicHealth will de-identify your records and will share their de-identified information with qualified researchers to better understand your disease.

Note that only the information collected immediately following sign up will be used for research purposes.

You can also choose to share the collected medical records using the share function at the top of your PicnicHealth timeline. You will be the only person who can access your medical records on PicnicHealth. No one — including your doctor or insurance company — will be able to see your medical records unless you choose to share them.

Yes. You can view this sample timeline.

You may choose to delete your medical records from the PicnicHealth service at any time and for any reason. Just email PicnicHealth at [email protected].

Everyone who wishes to join the study will use the same sign-up link found onthe PicnicHealth study webpage. During the sign-up process, you will be asked if you are signing up for yourself, or someone else. Select “someone else” and you will be prompted to provide information about your child during the sign-up process.

That's ok. Enter as many doctors as you remember. The hospital or clinic name is usually enough and the PicnicHealth team will follow up with you if there’s any confusion. If PicnicHealth finds doctors mentioned in your records who you did not include on your doctor list, you will get an email asking for your permission to request records from that new doctor.

Every parent/guardian will fill out a survey, called a proxy-report, on behalf of their child if the child is 17 years old or younger.

If your child is 8 years of age or older, they will fill out their own survey, called a self-report.

Every parent/guardian will also be able to fill out a caregiver-specific survey in addition to the proxy-report survey.

Completion of each version of the survey (self-report, proxy-report, caregiver- specific) is compensated at $30 each.

PicnicHealth will update your medical records regularly (approximately 4 times a year). To keep your records most up-to-date, you can always tell PicnicHealth when you have seen the doctor. Just click the “Request Records” button at the top of the timeline, and PicnicHealth will retrieve new records for you.

In the case that an adult with PK deficiency (aged 18 years or older) is unable to sign up or complete the survey themselves, the caregiver/guardian of an adult patient with PoA for the adult patient may sign up and complete a survey on the patient’s behalf. During the sign-up process, the caregiver will be asked if they are signing up for themselves, or someone else. They should select “someone else” and will be prompted to provide information about the adult with PK deficiency during the sign-up process.

In addition to filling out an adult patient survey on behalf of the adult with PK deficiency, every caregiver/guardian of an adult with PK deficiency will also be able to fill out a caregiver-specific survey.

Completion of each version of the survey (adult patient survey, caregiver- specific) is compensated at $30 each.

In the first month, some records might be missing because PicnicHealth is still collecting them for you. After the first month, if you suspect that records are still missing, you can let us know about those records by clicking "Request Records" at the top of your timeline. If you suspect that any information on your Timeline is incorrect, please contact us at [email protected].

Whether you are a parent/guardian of a minor with PK deficiency, or provide care for an adult with PK deficiency, you can participate in this study by answering questions about your experience caring for individuals with PK deficiency.

During study sign-up you will have the choice to “sign-up for someone else”. You should select this option and then fill out the information as prompted during sign-up.

If you are a parent or guardian, fill out your information and your child’s information as prompted during sign-up. You will then be asked to consent for your child’s participation in the study, as well as for your participation in answering surveys as a caregiver.

If you are a caregiver/guardian that is signing up another adult for the study (aged 18 years or older), you will need to provide a power of attorney (POA) document to legally be able to consent for the adult you provide care for. You will be prompted to provide this information during the study sign-up. As a caregiver to an adult with PK deficiency, you will also be asked to consent to your participation in answering surveys as a caregiver.

PicnicHealth has partnered with Agios Pharmaceuticals and another third-party, Adelphi Real World, to help researchers collect and manage medical records in order to learn more about how people living with PK deficiency are affected by their disease.

When you sign up for the study, PicnicHealth will organize your medical records on a secure online platform that you will be able to access. To protect your privacy, information that directly identifies you will be removed, and a unique code will be assigned to your medical records (de-identified) before sharing with others for the purpose of this study. The information gathered from patient medical records is helpful for researchers to understand real-world care patterns and short- and long-term outcomes.

At the top of your timeline, click on "Doctors" to add new doctors or medical facilities. PicnicHealth will then collect your records from your new doctors. If you have any questions about how to use the timeline, please feel free to contact us at [email protected].

Agios is the research sponsor of the PK Deficiency Real World Observational Study.

Agios has engaged PicnicHealth, a digital health company, to help collect & manage study participants’ medical records for the purpose of this study.

Adelphi was engaged to help develop the patient and caregiver surveys

It takes about 3 to 4 weeks to retrieve most of your historical medical records. The timing mainly depends on how quickly your doctors respond to PicnicHealth's requests for your records.

To participate you will need to sign up for PicnicHealth, list information about your healthcare providers in an onboarding survey, complete a medical record collection consent form, and an informed consent form. This process should take approximately 20 minutes in total

After you sign up, you will be asked to complete a survey assessing health- related quality of life, the indirect cost and daily life impact on patients and caregivers associated with PK deficiency. We estimate each survey will take another approximately 20 minutes

In most states, doctors are required to keep records for at least 7 years, but many will keep them for much longer. So how far back we can go really depends on the record-keeping practices of your doctors.

No, it will not cost you anything. You can access your medical records collectedfor this study through PicnicHealth free of charge for the duration of the PK Deficiency Real World Observational Study.

Compensation will be provided through a Visa gift card for your participation in this study. Participants will be compensated for signing up to join the study and for completing each survey.

You will be compensated $30 for joining the study and $30 for completing each survey. In the case that an adult patient is unable to complete the survey themselves, their caregiver may complete the patient survey on their behalf.

You will receive your first gift card once the PK deficiency diagnosis is verified (around 4 - 6 weeks after study sign-up) which will reflect your total compensation based on survey completion.

Please note that the timeframe may vary depending on how responsive your doctor’s office is when requesting records.

Agios, PicnicHealth, and Adelphi spoke with people living with PK deficiency, caregivers, and doctors that specialize in treating PK Deficiency to identify meaningful questions about the impact of PK deficiency that have not yet been answered by research.