News

New LAR Consent - Sept (2021)

** Important message **

Below is the updated consent for Legally Authorized Representatives (LAR) participants - such as caregivers, parents, spouses. If the participant you are a LAR for is already part of the registry, you do not need to re-consent. No action on your part is required we just need to notify you of this newly available consent form. This information was emailed to current participants. If you are a current LAR for a participant and you did not receive this email, please verify that the address we have within your profile is up-to-date. Also, please contact Jennifer DiRaimo, PDSA's Research Program Manager at jdiraimo@pdsa.org if you did not receive an email with the updated consent included. 

 

Consent to Participate in the ITP Natural History Study Registry and to Allow Data to be Shared for Future Research 

Consent for a Person with a Legally Authorized Representative

 

Title: ITP Natural History Study Registry 

Principal Investigator: Caroline Kruse

Phone: 440/746-9003

Email: pdsa@pdsa.org

Sponsor: Platelet Disorder Support Association (PDSA)

 

This document is intended to give you the information you need to make an informed and voluntary decision whether or not to provide the personal and medical information of the individual in your charge to the ITP Natural History Study Registry. As the guardian or legally authorized representative for the Study Participant, we encourage you to discuss the registry with the Study Participant to the extent compatible with their understanding. Much of the information in this form is required by the regulations designed to protect research participants, and the headings and structure of the document were chosen to be sure that all the required information was included. While this information is meant to answer most of the questions we anticipate, it may not answer all of your questions. If you have questions about anything you read or other questions about the registry that are not answered here, please contact the Principal Investigator at the phone or email listed above.

Definitions

“Study Participant” refers to the person with immune thrombocytopenia (ITP).  Registry information will be collected on patients who have ITP (Primary and Secondary).  “You” refers to the person reading this form and providing the information; in this case a family member or guardian who is legally responsible for the healthcare of the Study Participant. “We” refers to the organization, the Platelet Disorder Support Association (PDSA).

Study Aims

The data collected in this Registry will be used by researchers to study ITP Natural History Study Registry with the following goals: 

  1. To describe the people who have ITP and to better understand the variability and stages of ITP. To do this, we will ask you about the Study Participant’s diagnosis, treatment, medical history, social and economic environment, and treatment outcomes.    
  2. To understand how ITP changes over a person’s lifetime and to learn about clinical practice patterns and variations over the course of treatment. 
  3. To help to develop best practices, management guidelines and recommendations so that clinicians can know how to give the best care to improve the quality of life and outcomes of people with ITP.
  4. To identify people with ITP who might be willing to take part in other research studies or clinical trials. You will be able to choose whether you want to hear about these other studies on behalf of the Study Participant.

Research Data Sharing 

A Registry collects and stores medical and personal information on patients, including their family history and other information that could be important for research. If you participate in the Registry on behalf of the Study Participant, you will be invited to provide their personal and medical information by answering online surveys and uploading medical information. We will store the information you provide so that it can be used for future research projects. 

You always have access to the data you enter in the registry, via the participant dashboard. You can also view charts and graphs derived from the combined data contributed to the registry by other Study Participants.  This data from the Registry community will not reveal participant identities.

How You Provide Your Data 

We will ask you to enter the Study Participant’s information using online surveys on a secure internet site. The specific information we ask for might include: 

  • name, 
  • date of birth, 
  • diagnosis, 
  • treatment, 
  • genetic mutations, 
  • lab results, 
  • other past and current medical information, 
  • school or job information. 

You will be asked to update the registry information at least once per year, and you may be asked to update some surveys more frequently. We may contact you to: 

  • remind you to update surveys, 
  • finish incomplete surveys, 
  • complete outstanding surveys, 
  • complete new surveys.

The Registry Staff may also ask you to upload new test results or other medical information and may contact you to clarify the data you have entered. You can update information in the Registry whenever there is a change to the Study Participant’s health, their treatment, or if they develop new symptoms. 

We may contact you by telephone, email, through study-wide announcements or reminders automatically generated by the Registry, and by prompts on the organization’s website and social media channels. You can view and revise your contact preferences at any time by logging into your registry account and changing your choices under edit/profile/contact preferences. Things you can decide you will allow or not allow us to contact you about include:

  • reminders to update surveys,
  • clinical trials the Study Participant might be eligible for,
  • donating specimens or DNA for future research.

 

How We Use Your Data

The goal of the Registry is to let researchers, clinicians and industry partners use medical and other ITP-related information to learn about ITP while protecting the privacy of Study Participants.  

The Registry is overseen by the Registry Advisory Board, a committee that may include scientists, doctors and patients. The Advisory Board reviews aggregated registry data (data that has been combined and from which things that might identify specific participants have been removed). The Advisory Board also reviews ongoing research studies that use the Registry and evaluates new proposals for research that want to use the Registry’s data. Lastly, the Advisory Board reviews any reported problems or breaches of confidentiality to make sure they are appropriately reported and dealt with.

The Advisory Board reviews three different kinds of research that use the Registry. First, the Principal Investigator and their colleagues who work with PDSA can see all the data in the Registry and use it for research the Advisory Board approves. The Principal Investigator will only be allowed to do research for which you have given your permission (by signing this form or another one in the future) and which has been reviewed by an Institutional Review Board (or IRB - an independent group that reviews research proposals to make sure they properly protect participants).

Second, outside researchers may apply to the Advisory Board to see aggregated data. In most cases, such researchers will describe what they want to see, and the data will be assembled by Registry Staff, ensuring that no identifying information is shared. In rare cases, the Advisory Board may allow outside researchers to see individual data, but only after anything that can identify an individual participant has been removed. In these cases, the outside researchers will have to agree not to try and identify individuals from the data they receive.

Third, outside researchers may be looking for individuals to participate in their own research study, not directly related to the Registry, and may ask the Advisory Board to use the Registry for this purpose. In this case, the Registry will not give outside researchers your contact information but will instead contact you with details of the planned study and tell you how to contact the researchers if you and the Study Participant are interested. You can also decide that you are not interested in receiving information on outside studies.

This Registry is maintained by the National Organization for Rare Disorders, Inc. (NORD®), a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. To further that mission, everyone who is a Study Participant in this Registry is enrolled in NORD’s Natural History Study Program (NHS). Information that specifically identifies participants are removed and their information is combined with information from other rare diseases for cross disease analysis, cross disease research, and to facilitate advocacy. NORD may also share information from the NHS that doesn’t identify individuals with other databases that further rare disease research and the interests of the rare disease community, such as the Rare Disease Cures Accelerator – Data and Analytics Platform (RDCA-DAP) of the Critical Path Institute.

Risks and Inconveniences  

Putting the Study Participant’s data in the registry does not put you or them at any risk of physical harm but, like any information you provide electronically, there is a risk that their privacy could be compromised if the data is inappropriately disclosed or misused.

The registry is designed to make the chance of this happening very small. The Advisory Board reviews ongoing and new research to help prevent misuse (see How We Use Your Data, above). And we have technical and administrative protections in place to reduce the risk of inappropriate disclosure (see Privacy Rights and Confidentiality, below).

The registry surveys may ask questions about the impact of ITP on the Study Participant's daily life, their economic status, mood, and other topics that you may find unpleasant or disquieting.

While these are the risks we can foresee, it is possible that other risks may arise in the future.

Benefits 

Participation in this Registry is voluntary and unlikely to lead to direct medical or financial benefits for you or the Study Participant personally, but we hope the results from the research we make possible will help people with ITP in the future. 

Cost and Payment

It should not cost you or the Study Participant anything to participate in the Registry, other than the costs of internet access, and you will not be paid for the information you provide.   

The information will only be used for research, but it is possible that the research could lead to the development of a commercial medical product. Should this happen, neither you nor the Study Participant should not expect to be paid. 

Privacy Rights

The information we collect in the Registry is personal, and where that information relates to health and medical records, it is considered Protected Health Information (PHI) and protected by a law called the Health Insurance Portability and Accessibility Act (HIPAA). This law only protects information when it is held by certain recognized parties, like doctors, hospitals, insurance companies, and some researchers. All reasonable efforts will be made to protect the Study Participant’s PHI, which may be shared with others to support this research, but it is possible that their information could be used or disclosed in a way that leaves it no longer protected by law. We have taken steps to make that unlikely, described below.

Confidentiality 

All identifiable information that is obtained in connection with this Registry will remain confidential. This Registry will share detailed medical and other information with researchers. We will protect your privacy and that of the Study Participant by removing names, addresses, and other identifying information before it is shared. It is important that the information you provide can all be kept together, as belonging to a single person, when it is used for research. To make this possible we will identify the Study Participant’s information with an arbitrary code, which will be shared, but researchers will not be able to link the code back to the Study Participant. This process is called “pseudonymizing” and the coded information is considered “pseudonymized.”

The Registry itself will maintain a database that links these arbitrary codes to the information that identifies Participants. We need to do this so that we can associate new information you give us with the pseudonymized information that we already have and to maintain the integrity of the database. Only trained and authorized people who work for the Registry will be able to see the information in this linking database.

When the results of research conducted with data from the Registry are published or reported, no information that could identify you or the Study Participant will be disclosed unless you have explicitly given your consent for such disclosure. You are not giving that consent if you sign this form.

Representatives from NORD and from North Star Review Board, the IRB that oversees this Registry, may inspect study records during auditing procedures to be sure that the Registry is being protected according to regulations and policies. Additionally, NORD staff may access this registry in cases where technical support has been requested. However, these organizations and the individuals acting on their behalf are required to keep all information confidential.

Voluntary Participation, Withdrawal and Alternatives to Participating

It is up to you whether or not you want to participate in this Registry on behalf of the Study Participant. If you choose not to, neither you nor they will be penalized in any way, neither of you will lose any benefits you might otherwise be entitled to, and the Study Participant’s health care will not be affected. If you decide not to participate on behalf of the Study Participant, or choose to withdraw them, that decision won’t harm their relationship with their doctors, even if they are involved with the Registry or with PDSA.

There may be other Registries that serve patients with ITP, and you are free to provide data to any such registry instead of, or in addition to, working with us, or not to share the Study Participant’s data at all. There may be other research opportunities, including clinical trials, for patients with ITP, and you may choose to have the Study Participant participate in that research whether or not they participate in this Registry.

If you do choose to share information with us, you can change your mind at any time. If, after joining the Registry, you decide you no longer want to participate, the Registry researchers may still use data that was submitted before you changed your mind in order to complete research that has already started, however, the data you shared with us will no longer be available for future research, and will not be shared further by PDSA, even if it has been pseudonymized. It may not be possible for us to recover or destroy pseudonymized data that has already been shared with other researchers, who may still use the information to complete the research they have started. Similarly, pseudonymized data that has already been shared with NORD’s NHS or The Critical Path Institute’s RDCA-DAP cannot be removed from those databases.

If you choose to participate in the Registry on behalf of the Study Participant, that permission will not expire. If you change your mind and want to withdraw from the Registry, you can do so through the website or by contacting PDSA research staff at research@pdsa.org or Jennifer DiRaimo, Research Program Manager at jdiraimo@pdsa.org.  

Participation of Minors and Adults Unable to Consent 

Survey information will be collected on patients who are diagnosed with ITP. Patients who are legal adults who understand the consent form and legally provide their own consent (and thus do not have a legal guardian) are eligible to join the Natural History Study Registry on their own. Otherwise, the legally authorized representative or family member of the patient (minor or adult needing assistance) must sign the consent form for the patient to join. When a minor Study Participant becomes a legal adult (and if they are able,) consent will be obtained directly from them for continued participation.

If You Live Outside the United States

The Registry is maintained on servers that are physically present in the United States. For persons living outside the United States who choose to share information about themselves and about a person for whom they serve as a Legally Authorized Representative, the same protections for privacy and confidentiality are offered as in the United States; in addition, as explained below, residents of the European Union and Switzerland have additional particular rights related to personal information. By signing this consent, you acknowledge that you are disclosing information that would otherwise be private. Privacy laws in your country may have different protections than those provided in the United States. 

For persons who are residents of the European Union and Switzerland, transfer of your and the Participant’s personal information outside of the European Union and/or Switzerland, if any, will be undertaken in compliance with the General Data Protection Regulation under an appropriate transfer mechanism provided for by the General Data Protection Regulation, including the use of standard data protection clauses adopted by the European Commission. Please be aware that, under the General Data Protection Regulation, the European Commission is permitted to issue a decision that the data protection laws of a third country are adequate to the protection of personal information and that, to date, the European Commission has not done so with respect to the United States.  

For persons who are residents of the European Union and Switzerland, processing of personal information will also be undertaken in such a manner as to ensure the rights of data subjects provided for by the General Data Protection Regulation.  Specifically, Registry participants who are residents of the European Union and Switzerland are entitled to:

  • Request to obtain access to and rectification or erasure of personal data;
  • To receive personal data in a portable, readily accessible format; 
  • To restrict or withdraw permission for the processing of personal information;
  • To lodge a complaint with an appropriate supervisory authority.

Please note that the rights to erase personal data or restrict or withdraw permission for the processing of personal information are subject to limitations provided for by Article 17 of the General Data Protection Regulation, namely, that such rights may be limited as necessary to protect the public interest in the area of public health or for archiving purposes in the public and scientific interest.

Getting Answers to Your Questions about the Registry

We have used some technical terms in this form and talked about issues in research and data sharing with which you may not have been familiar. Take as long as you need or want to consider what was presented here and whether you want to share the Study Participant’s personal and medical information with the Registry. If you have any questions or want anything explained further, please contact the PDSA research staff at research@pdsa.org or Jennifer DiRaimo, Research Program Manager at jdiraimo@pdsa.org.  It is our responsibility to answer your questions.

An Institutional Review Board (IRB) has reviewed this Registry to ensure that it meets ethical and regulatory standards for protecting your rights.  An IRB is an independent group that reviews research proposals to make sure they properly protect participants.  For questions about those protections and your rights as a Study Participant in this Registry, or to discuss other study-related concerns or complaints with someone who is not part of this Registry team, please contact North Star Review Board at 877-673-8439 (toll-free) or info@northstarreviewboard.org. You may want to contact the IRB if:  

  • You have questions, concerns, or complaints that are not being answered by the research team. 
  • You are not getting answers from the research team. 
  • You cannot reach the research team. 
  • You want to talk to someone else about the research. 
  • You have questions about your rights as a research subject. 

Please do not sign this form unless you have had all your questions answered. 

Authorization

The following statements are intended to ensure that you have had the time and opportunity to consider whether you want to participate in this Registry on behalf of the Study Participant, have had your questions answered, and agree to participate in the study as described. You will be asked to acknowledge that you have:

  • Read the consent form and you and the Participant have no further questions about the Registry and your participation
  • That you wish to provide personal data to the registry for the purposes of the Study
  • And that you wish to provide the Participant’s pseudonymized data for future research
  • That you have explained the study to the Participant to the extent they are able to understand

This is a web-based form and by answering Yes to all of the following statements, you are giving your consent to participate in the ITP Natural History Study Registry on behalf of the study participant, just as if you had signed your name to a paper document. After signing, a copy of the consent form will be emailed to you. If you cannot comfortably answer “Yes” to these three statements or you have further questions, please do not check the consent boxes in the following section.

  1. I have read (or someone has read to me) this Consent and Authorization Form to provide the Study Participant’s personal and medical data to be shared for the purpose of research. All my questions about the Registry have been answered to my satisfaction and I understand the purpose of the Registry and the risks of participation. 


Yes ___   No ___ 

  1. I wish to provide the Study Participant’s research data to the ITP Natural History Study Registry for the purposes described above under Study Aims (page 1).


Yes ___   No ___ 

  1. I wish to provide the Study Participant’s research data that has been pseudonymized to the ITP Natural History Study Registry for future research within recognized ethical standards for scientific research, as described under How We Use Your Data (page 3).


Yes ___    No ___ 

  1. I have explained the study to the Study Participant to the extent they are able to understand, and the Study Participant has given their assent to participate in this study.


Yes ___    No ___

 

New Adult Consent - Sept (2021)

** Important message **

Below is the updated consent for adult participants. If you are an adult participant who is already part of the registry, you do not need to re-consent. No action on your part is required we just need to notify you of this newly available consent form. This information was emailed to current participants. If you are a current adult participant and you did not receive this email, please verify that the address we have within your profile is up-to-date. Also, please contact Jennifer DiRaimo, PDSA's Research Program Manager at jdiraimo@pdsa.org if you did not receive an email with the updated consent included. 

 

 

Consent to Participate in the ITP Natural History Study Registry and to Allow Your Data to be Shared for Future Research 

Adult Consent 

Title: ITP Natural History Study Registry 

Principal Investigator: Caroline Kruse, President & CEO (PDSA)

Phone: 440/746-9003

Email: pdsa@pdsa.org

Sponsor: Platelet Disorder Support Association (PDSA)

This document is intended to give you the information you need to make an informed and voluntary decision whether or not to provide your personal and medical information to the ITP Natural History Study Registry. Much of the information in this form is required by the regulations designed to protect research participants, and the headings and structure of the document were chosen to be sure that all the required information was included. While this information is meant to answer most of the questions we anticipate, it may not answer all of your questions. If you have questions about anything you read, or other questions about the registry that are not answered here, please contact the Principal Investigator at the phone or email listed above.

Definitions

“Study Participant” refers to the person with Immune Thrombocytopenia (ITP).  Registry information will be collected on patients who have ITP (Primary and Secondary).  “You” refers to the person reading this form and providing the information. “We” refers to the organization, the Platelet Disorder Support Association (PDSA). 

Study Aims

The data collected in this Registry will be used by researchers to study ITP with the following goals: 

  1. To describe the people who have ITP and to better understand the variability and stages of ITP. To do this, we will ask you about your diagnosis, treatment, medical history, social and economic environment, and treatment outcomes.    
  2. To understand how ITP changes over a person’s lifetime and to learn about clinical practice patterns and variations over the course of treatment. 
  3. To help to develop best practices, management guidelines and recommendations so that clinicians can know how to give the best care to improve the quality of life and outcomes of people with ITP.
  4. To identify people with ITP who might be willing to take part in other research studies or clinical trials. You will be able to choose whether you want to hear about these other studies.

Research Data Sharing 

A Registry collects and stores medical and personal information on patients, including their family history and other information that could be important for research. If you participate in the Registry, you will be invited to provide personal and medical information by answering online surveys and uploading medical information. We will store the information you provide so that it can be used for future research projects. 

You always have access to the data you enter in the registry, via the participant dashboard. You can also view charts and graphs derived from the combined data contributed to the registry by other Study Participants.  This data from the Registry community will not reveal participant identities. 

How You Provide Your Data 

We will ask you to enter your information using online surveys on a secure internet site. The specific information we ask for might include: 

  • name, 
  • date of birth, 
  • diagnosis, 
  • treatment, 
  • genetic mutations, 
  • lab results, 
  • other past and current medical information, 
  • school or job information. 

You will be asked to update your registry information at least once per year, and you may be asked to update some surveys more frequently. We may contact you to: 

  • remind you to update surveys, 
  • finish incomplete surveys, 
  • complete outstanding surveys, 
  • complete new surveys.

The Registry Staff may also ask you to upload new test results or other medical information and may contact you to clarify the data you have entered. You can update your information in the Registry whenever there is a change to your health, your treatment, or if you develop new symptoms. 

We may contact you by telephone, email, through study-wide announcements or reminders automatically generated by the Registry, and by prompts on the organization’s website and social media channels. You can view and revise your contact preferences at any time by logging into your registry account and changing your choices under edit/profile/contact preferences. Things you can decide you will allow or not allow us to contact you about include:

  • reminders to update your surveys,
  • clinical trials you might be eligible for,
  • donating specimens or DNA for future research.

 

How We Use Your Data

The goal of the Registry is to let researchers, clinicians and industry partners use medical and other ITP-related information to learn about ITP while protecting the privacy of Study Participants.  

The Registry is overseen by the Registry Advisory Board, a committee that may include scientists, doctors, and patients. The Advisory Board reviews aggregated registry data (data that has been combined and from which things that might identify specific participants have been removed). The Advisory Board also reviews ongoing research studies that use the Registry and evaluates new proposals for research that want to use the Registry’s data. Lastly, the Advisory Board reviews any reported problems or breaches of confidentiality to make sure they are appropriately reported and dealt with.

The Advisory Board reviews three different kinds of research that use the Registry. First, the Principal Investigator and their colleagues who work with PDSA can see all the data in the Registry and use it for research the Advisory Board approves. The Principal Investigator will only be allowed to do research for which you have given your permission (by signing this form or another one in the future) and which has been reviewed by an Institutional Review Board (or IRB - an independent group that reviews research proposals to make sure they properly protect participants).

Second, outside researchers may apply to the Advisory Board to see aggregated data. In most cases, such researchers will describe what they want to see, and the data will be assembled by Registry Staff, ensuring that no identifying information is shared. In rare cases, the Advisory Board may allow outside researchers to see individual data, but only after anything that can identify an individual participant has been removed. In these cases, the outside researchers will have to agree not to try and identify individuals from the data they receive.

Third, outside researchers may be looking for individuals to participate in their own research study, not directly related to the Registry, and may ask the Advisory Board to use the Registry for this purpose. In this case, the Registry will not give outside researchers your contact information but will instead contact you with details of the planned study and tell you how to contact the researchers if you are interested. You can also decide that you are not interested in receiving information on outside studies.

This Registry is maintained by the National Organization for Rare Disorders, Inc. (NORD®), a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. To further that mission, everyone who participates in this Registry is enrolled in NORD’s Natural History Study Program (NHS). Information that specifically identifies participants is removed and their information combined with information from other rare diseases for cross disease analysis, cross disease research, and to facilitate advocacy. NORD may also share information from the NHS that doesn’t identify individuals with other databases that further rare disease research and the interests of the rare disease community, such as the Rare Disease Cures Accelerator – Data and Analytics Platform (RDCA-DAP) of the Critical Path Institute.

Risks and Inconveniences  

Putting your data in the registry does not put you at any risk of physical harm but, like any information you provide electronically, there is a risk that your privacy could be compromised if your data is inappropriately disclosed or misused.

The registry is designed to make the chance of this happening very small. The Advisory Board reviews ongoing and new research to help prevent misuse (see How We Use Your Data, above). And we have technical and administrative protections in place to reduce the risk of inappropriate disclosure (see Privacy Rights and Confidentiality, below).

The registry surveys may ask questions about the impact of ITP on your daily life, your economic status, mood, and other topics that you may find unpleasant or disquieting.

While these are the risks we can foresee, it is possible that other risks may arise in the future.

Benefits 

Participation in this Registry is voluntary and is unlikely to lead to direct medical or financial benefits for you, but we hope the results from research we make possible will help people with ITP in the future. 

Cost and Payment

It should not cost you anything to participate in the Registry, other than the costs of internet access, and you will not be paid for the information you provide.   

Your information will only be used for research, but it is possible that the research could lead to the development of a commercial medical product. Should this happen, you should not expect to be paid. 

Privacy Rights

The information we collect in the Registry is personal, and where that information relates to your health and medical records, it is considered Protected Health Information (PHI) and protected by a law called the Health Insurance Portability and Accessibility Act (HIPAA). This law only protects information when it is held by certain recognized parties, like doctors, hospitals, insurance companies, and some researchers. All reasonable efforts will be made to protect your PHI, which may be shared with others to support this research, but it is possible that your information could be used or disclosed in a way that leaves it no longer protected by law. We have taken steps to make that unlikely, described below.

Confidentiality 

All identifiable information that is obtained in connection with this Registry will remain confidential. This Registry will share detailed medical and other information with researchers. We will protect your privacy by removing your name, address, and other identifying information before it is shared. It is important that the information you provide can all be kept together, as belonging to a single person, when it is used for research. To make this possible we will identify your information with an arbitrary code, which will be shared, but researchers will not be able to link the code back to you. This process is called “pseudonymizing” and the coded information is considered “pseudonymized.”

The Registry itself will maintain a database that links these arbitrary codes to the information that identifies Participants. We need to do this so that we can associate new information you give us with the pseudonymized information that we already have and to maintain the integrity of the database. Only trained and authorized people who work for the Registry will be able to see the information in this linking database.

When the results of research conducted with data from the Registry are published or reported, no information that could identify you will be disclosed unless you have explicitly given your consent for such disclosure. You are not giving that consent if you sign this form.

Representatives from NORD and from North Star Review Board, the IRB that oversees this Registry, may inspect study records during auditing procedures to be sure that the Registry is being protected according to regulations and policies. Additionally, NORD staff may access this registry in cases where technical support has been requested. However, these organizations and the individuals acting on their behalf are required to keep all information confidential.

Voluntary Participation, Withdrawal and Alternatives to Participating

It is up to you whether or not you want to participate in this Registry. If you choose not to, you won’t be penalized in any way, you won’t lose any benefits you might otherwise be entitled to, and your health care will not be affected. If you decide not to participate, or choose to withdraw, that decision won’t harm your relationship with your doctors, even if they are involved with the Registry or with PDSA.

There may be other Registries that serve patients with ITP, and you are free to provide data to any such registry instead of, or in addition to, working with us, or not to share your data at all. There may be other research opportunities, including clinical trials, for patients with ITP, and you may choose to participate in that research whether or not you participate in this Registry.

If you do choose to share your information with us, you can change your mind at any time. If, after joining the Registry, you decide you no longer want to participate, the Registry researchers may still use data that was submitted before you changed your mind in order to complete research that has already started, however, the data you shared with us will no longer be available for future research, and will not be shared further by PDSA even if it has been pseudonymized. It may not be possible for us to recover or destroy pseudonymized data that has already been shared with other researchers, who may still use your information to complete the research they have started. Similarly, pseudonymized data that has already been shared with NORD’s NHS or The Critical Path Institute’s RDCA-DAP cannot be removed from those databases.

If you choose to participate in the Registry, that permission will not expire. If you change your mind and want to withdraw from the Registry, you can do so through the website or by PDSA research staff at research@pdsa.org or Jennifer DiRaimo, Research Program Manager at jdiraimo@pdsa.org.  

If You Live Outside the United States

The Registry is maintained on servers that are physically present in the United States. For persons living outside the United States who choose to share information about themselves, the same protections for privacy and confidentiality are offered as in the United States; in addition, as explained below, residents of the European Union and Switzerland have additional particular rights related to personal information. By signing this consent, you acknowledge that you are disclosing information that would otherwise be private. Privacy laws in your country may have different protections than those provided in the United States. 

For persons who are residents of the European Union and Switzerland, transfers of your personal information outside of the European Union and/or Switzerland, if any, will be undertaken in compliance with the General Data Protection Regulation under an appropriate transfer mechanism provided for by the General Data Protection Regulation, including the use of standard data protection clauses adopted by the European Commission. Please be aware that, under the General Data Protection Regulation, the European Commission is permitted to issue a decision that the data protection laws of a third country are adequate to the protection of personal information and that, to date, the European Commission has not done so with respect to the United States.

For persons who are residents of the European Union and Switzerland, processing of personal information will also be undertaken in such a manner as to ensure the rights of data subjects provided for by the General Data Protection Regulation.  Specifically, Registry participants who are residents of the European Union and Switzerland are entitled to:

  • Request to obtain access to and rectification or erasure of personal data;
  • To receive personal data in a portable, readily accessible format; 
  • To restrict or withdraw permission for the processing of personal information;
  • To lodge a complaint with an appropriate supervisory authority.

Please note that the rights to erase personal data or restrict or withdraw permission for the processing of personal information are subject to limitations provided for by Article 17 of the General Data Protection Regulation, namely, that such rights may be limited as necessary to protect the public interest in the area of public health or for archiving purposes in the public and scientific interest.  

Getting Answers to Your Questions about the Registry

We have used some technical terms in this form and talked about issues in research and data sharing with which you may not have been familiar. Take as long as you need or want to consider what was presented here and whether you want to share your personal and medical information with the Registry. If you have any questions or want anything explained further, please contact PDSA research staff at research@pdsa.org or Jennifer DiRaimo, Research Program Manager at jdiraimo@pdsa.org.  It is our responsibility to answer your questions.

An Institutional Review Board (IRB) has reviewed this Registry to ensure that it meets ethical and regulatory standards for protecting your rights.  An IRB is an independent group that reviews research proposals to make sure they properly protect participants.  For questions about those protections and your rights as a Study Participant in this Registry, or to discuss other study-related concerns or complaints with someone who is not part of this Registry team, please contact North Star Review Board at 877-673-8439 (toll-free) or info@northstarreviewboard.org. You may want to contact the IRB if:  

  • You have questions, concerns, or complaints that are not being answered by the research team. 
  • You are not getting answers from the research team. 
  • You cannot reach the research team. 
  • You want to talk to someone else about the research. 
  • You have questions about your rights as a research subject. 

Please do not sign this form unless you have had all your questions answered. 

Authorization

The following statements are intended to ensure that you have had the time and opportunity to consider whether you want to participate in this Registry, have had your questions answered, and agree to participate in the study as described. You will be asked to acknowledge that you have:

  1. Read the consent form and have no further questions about the Registry and your participation
  2. That you wish to provide personal data to the registry for the purposes of the Study
  3. And that you wish to provide your pseudonymized data for future research

This is a web-based form and by answering Yes to all of the following statements, you are giving your consent to participate in the ITP Natural History Study Registry, just as if you had signed your name to a paper document. After signing, a copy of the consent form will be emailed to you. If you cannot comfortably answer “Yes” to these three statements or you have further questions, please do not check the consent boxes in the following section. 

  1. I have read (or someone has read to me) this Consent and Authorization Form to provide my personal and medical data to be shared for the purpose of research. All my questions about the Registry have been answered to my satisfaction and I understand the purpose of the Registry and the risks of participation. 
     
    Yes ___   No ___ 
  2. I wish to provide my research data to the ITP Natural History Study Registry for the purposes described above under Study Aims (page 1).

    Yes ___   No ___ 
  3. I wish to provide my research data that has been pseudonymized to the ITP Natural History Study Registry for future research within recognized ethical standards for scientific research, as described under How We Use Your Data (page 3).  

           Yes ___    No ___ 

 

Frequently Asked Questions

1. What is a Patient Registry?

A patient registry is a collection of standardized information about a group of patients who share a condition and is used for a variety of purposes such as conducting natural history studies and supporting disease specific clinical trial recruitment.

2. What is a Natural History Study?

A natural history study is a study designed to track the course of a disease over time and includes people who have a specific medical condition or disease and those who are at risk of developing such. This method of research explores the disease in a comprehensive way and identifies demographic, genetic, environmental, and other variables that correlate with the disease and its outcomes. Natural history studies have many potential uses such as patient care best practice developments and clinical trial recruitment.

3. What is a Research Study Sponsor?

The National Health Service defines a study sponsor as, “… the individual, company, institution or organization, which takes on ultimate responsibility for the initiation, management […] of and/or financing […] for that research.”1 The Study Sponsor ensures that the study is conducted in a reputable manner and upholds regulations as they apply to the study.

4. What is a Principal Investigator?

The Principal Investigator is the research group leader or, the person with the primary responsibility for the design and conduct of the research project or study.

5. What is an Institutional Review Board (IRB)?

According to the Mayo Clinic an IRB is, “[a] specifically constituted review body established to protect the rights and welfare of human subjects recruited to participate in biomedical or behavioral/social science research.”2 An institutional review board is a group of people who are responsible for protecting the rights and welfare of people who participate in studies.

6. What is the purpose of the ITP Natural History Study Patient Registry?

One of the most important purposes of the ITP Natural History Study is to bring the ITP community together and collect data which could be used to create therapeutics and improve the quality of life for patients. Some other goals of the ITP Natural History Study are to:

  • Conduct a prospectively planned natural history study that will result in the most comprehensive understanding of ITP and its progression over time.
  • Characterize and describe the ITP population as a whole.
  • Assist the ITP community with the development of recommendations for standards of care.
  • Assist researchers studying the pathophysiology of ITP.
  • Assist researchers studying interventional outcomes.
  • Support the design of clinical trials for new treatments.

7. What types of data will be collected in the ITP Natural History Study Patient Registry?

The data will be collected is uniform and includes but is not limited to:

  • Socio-demographics
  • Medical and diagnostics
  • Treatment and disease progression
  • Management of care
  • Quality of life

8. How is the data collected?

Data is collected through a secure web-based system developed by the National Organization for Rare Disorders (NORD), an independent non-profit committed to the identification, treatment, and cure of all 7,000 rare diseases. Study participants respond to questions grouped within a series of surveys developed per study standards and in collaboration with disease specific experts.

9. Who is a study participant?

A study participant is the individual who takes part in a research study and whose information is collected for that research. Study participants may consent to enter and share their own personal data. 10. Who is a reporter/respondent? A reporter/respondent is an individual who completes the surveys on behalf of the patient/study participant, when they are unable to do so on their own behalf.

10. Who is a reporter/respondent?

A reporter/respondent is an individual who completes the surveys on behalf of the patient/study participant, when they are unable to do so on their own behalf.

11. What is a legally authorized representative (LAR)?

A legally authorized representative is an individual who, under law, has the ability to act on behalf of another person (such as a minor study participant). The LAR may be a parent, grandparent, caregiver who has the legal authority to grant consent on behalf of another who has been invited to participate in research. 

12. Can data be collected worldwide?

The patient registry uses an online platform which allows participants to contribute data from anywhere in the world.

13. Where is the data stored?

The data is stored on NORD’s registry platform system which adheres to industry standards regarding security protections.

14. Is the data safe?

Yes, the data is safe. The registry follows strict government guidelines to assure patient information is protected. The platform is served over HTTPS, which provides traffic encryptions so as to prevent eavesdropping and man-in-the-middle attacks. Communications between the registry platform application server and the database are also encrypted.

15. Who owns the data?

The identifiable and de-identifiable data are owned by the study sponsor, the Platelet Disorder Support Association (PDSA). PDSA decides how and with whom to share the data. A subset of the de-identified data collected across the NORD Registry Platform is available to NORD to support cross disease analysis and advocacy activities to members of the rare disease community as a whole. 

16. How is the Patient Registry maintained?

The registry is maintained by NORD who hosts the registry on its cloud-based Platform and provides oversight and ongoing support of the system. PDSA provides the day-to-day management of their patient registry, including the development and adherence to the study procedures.

17.What is PDSA – the Platelet Disorder Support Association?

The Platelet Disorder Support Association is a 501 © (3) organization dedicated to enhancing the lives of people with Immune Thrombocytopenia (ITP) and other platelet disorders through education, advocacy, research and support. As the leading ITP patient advocacy organization in North America, PDSA serves the worldwide ITP community of patients, practitioners, caregivers, advocates, and key disease stakeholders, promoting their needs and uniting them on a global level. We build awareness, educate the ITP community, and provide critical connections and resources that empower patients to take charge of their disease and encourage practitioners to exercise patient-centered medical care. Learn more about PDSA at www.pdsa.org.

18.Who is NORD – the National Organization for Rare Disorders?

NORD, a 501(c)(3) organization, is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD was founded by patients and families who marshaled grass-roots efforts to secure the passage of the Orphan Drug Act in 1983. Today, NORD represents the united voice of more than 250 rare disease-specific groups and thousands of patient advocates. Together, we are committed to the identification, treatment and cure of rare disorders through programs of advocacy, education, research and patient support services. Learn more about NORD at https://rarediseases.org/.