Alstrom Syndrome International was recently selected for the highly competitive Eugene Washington PCORI Engagement Funding Award. We will use the opportunity of the ASI Family Conference in May 2016 to begin involving individuals with Alstrom Syndrome, family members, and other stakeholders meaningfully in sharing and discussing what is important to YOU!
The healthcare research process has traditionally included only scientists and other research-related professionals. ASI strongly believes in the importance of the engagement of nontraditional stakeholders— patients, caregivers, clinicians, and other stakeholders. From topic selection through design and conduct of research to dissemination of results—we can influence research to be more patient-centered, open, and useful, and ultimately lead to greater use of research results by patients and the broader healthcare community.
We will begin the process now with the goal of eventually bringing to the ASI Family Conference topics that YOU indicate are important for your life. You will be hearing more about this as the conference draws near, beginning with a short survey about potential topics of discussion. More information about The Eugene Washington PCORI Engagement Funding Award can be found at http://bit.ly/1idIgtt
We have invited a small group of people with Alstrom Syndrome, Parents, Clinicians, and Research Scientists to form a Stakeholders Steering Committee to help guide the process from beginning to end. However, we need EVERYONE’S involvement and opinions! This engagement process will definitely continue after the conference, as we don’t expect that everyone who has some input to give will be there, and we want to give opportunities for all to participate. We also want people to have a chance to think about and digest what we come up with, and to give further input by whatever style best suits them. As you well know, it is normal and natural for the Alström community to always be engaged. It’s just that now we intend it more systematically and with larger goals in mind. Rather than simply imagining what’s important, or having “experts” decide next steps in isolation, we—the SSC with PCOR’s support—are creating an opportunity to break out of this “top-down” decision-making model and foster a shared responsibility for health between everyone who is involved with Alström Syndrome: affected persons, parents, care-givers, clinicians and researchers alike. “
Don’t hold back – this is a great opportunity to shape the direction of Alstrom Research, Education and Support! We are looking forward to getting started!
PLEASE PARTICIPATE IN THIS IMPORTANT PROJECT!
LINK TO ARC SURVEY
Alstrom Research Connect FAQs
1. How do I participate if I have low vision and use a screen reader?
You can enlist the assistance of a family member or friend to go through the process of setting up your account and your privacy settings. You can also contact Alstrom Syndrome International (email@example.com ) and we will set up the account for you and you can then access the survey and control your privacy settings using your screen reader software.
2. Why do I have to give an email address to get a code to proceed with the Registry?
3. What is the security level of the servers and how is the data encrypted? Who can access to my data and how?
The Genetic Alliance BioTrust Ethics Board and various Institutional Review Boards (discussed below) that have reviewed the PEER system have helped to establish a number of categories for access options that individuals may wish to consider. Depending on how individual users employ these tools, they may Grant (Allow), Deny (Decline to allow), or set a control called “Ask Me” that requires that the individual participant be consulted further, and provided with greater details about the intended use and the party seeking such access before deciding whether to allow or deny access. By using the controls, it is possible for individuals to restrict access to their information to a single researcher; just to researchers approved by the Medical Advisory Board of ASI; to researchers approved by the advisory board of other support organizations; to IRB-approved researchers working on a study that addresses one or more of the conditions affecting the individual; or to all researchers with IRB approval.
4. Can individuals or users can conduct their own studies using the data?
Yes, subject to the individuals’ right to determine who can access and use their respective information, this is possible. It is also consistent with the philosophy of the Patient-Centered Outcomes Research Institute (PCORI) initiative, of which this initiative is a part, as well as of Genetic Alliance, who is the Principal Investigator thereunder. Our collective hope is that we will be able to advance science by giving patients greater control over who can access and use their information that we will help make it possible for anyone who has a worthwhile idea that may assist the community, and who is willing to abide by responsible practices. This is also something on which we are working with UCSF (one of two academic medical center partners under the PCORI project initiative), and with whom we plan to test various UCSF tools they think may be useful in letting patients have a greater voice in devising studies of potential relevance.
5. Is there an IRB approval and if so by what board?
Yes. Actually there are several. The overall PEER Platform was reviewed and approved by Western Institutional Review Board (WIRB) in June 2014. The WIRB approval is explicitly referenced in a Prospective Participant Informational Notice that accompanies the confirmation email that is sent to anyone expressing possible interest in the registry. In addition to WIRB, the Genetic Alliance IRB has reviewed the ARC questionnaire instrument and materials, as well as other details. The BioTrust Ethics Committee of the Genetic Alliance is also actively overseeing this work, and has been instrumental in creating the PEER platform and its approach for empowering individuals to set granular and dynamic access levels.
6. How does one delete their account from the Registry?
One simply needs to click on the “Remove” button associated with a health profile. Alternatively, since the access controls are dynamic in nature, another option that is available to all users at all times is the ability to reduce who is permitted to access information in the registry and/or for what purpose such access may be used.
7. Who are the financial sponsors of the registry?
The ARC registry has been built under a grant from PCORI. ASI is one of nine disease advocacy groups participating in this project, along with UCSF and UC Davis, and Private Access, a technology vendor. Sharon Terry, the CEO of Genetic Alliance serves as the Principal Investigator.
Secure online registration for the 8th Family Conference and Scientific Symposium in Plymouth MA (May 12 – 16, 2016) is now OPEN!
Register you and your family today!
At last, the Alstrom Research Connect and Family Exchange has launched! Find out more about this patient-centered registry that will enable researchers as well as other families to learn more about the syndrome as they work towards a cure. You control your information! YOU can drive research forward! Sign up today!!
To enter the ARC Registry, click HERE
For additional information about ARC, click HERE
Dear Alström Family,
Many thanks for the compliments following our posts about the Precision Medicine Initiative. A few members need to understand that the work is already underway and that ASI has been energetically involved for about a year, so this is real, not a fantasy. More importantly, the actual payoff will begin shortly when you have to dig deep for the benefit of all and complete yet another survey for ASI, this one the all-important Alström Syndrome Registry (ASR) about which we have written several times. In the future we will refer to it as ARC (Alstrom Research Connect & Family Exchange). You, not we, will be the true heroes in this adventure!
It has occurred to us that you could help the effort and help yourselves by starting to collect and to recollect your [or your child(ren)’s] experience with the syndrome. Take some notes as you review. What happened when? How old were you? What medications were prescribed for which problem? What dosages were used? For how long? Did it work? Think about how dual-sensory deficits and different health issues have affected all the different areas of your life – home and family, school, community, work? What solutions helped or hurt?
The survey is very comprehensive, and the most cool, wonderful, and very important thing about it is that you’ll be able to see not only how other unidentified respondents answered the same question but also see percentages relative to all those who have answered that same question. You can come back over time and compare your personal experience again and again as the number of those participating gets bigger and bigger. And, remember that your privacy is rigorously protected!
We all know that Alström Syndrome is rare and that there is a lot of variability and that treating physicians are often working in the dark. Think about going on line and being able to show the doctor right then and there what others with the syndrome have experienced. Also, we’re working on mobile apps and appropriate technology that will be accessible, so think about being able to whip out your phone and say, “Siri, please tell Dr. Knows Little how many Alström patients suffered from glue ear at age 5.” Siri can respond, “out of 832 patients registered, 76% had glue ear at age 5. Would you like more information on this topic?” When we’re done, we’ve been told that 90 % of the entire survey will be computable and viewable, even (wow!) as charts and graphs. What an amazing gift!
Also, don’t be worried that this may be just too difficult for you! There will be demos and you can stop (AND “SAVE”) and pick-up where you left off. We have “Guides” in place already to reassure you, and we are developing a team of “Faciltators” who can help you complete whatever you find challenging, even work through the whole process with you, if needed.
So, please prepare yourself to move quickly once we are well and truly launched. Do not get discouraged by the size of the task, and remember that you will be personally contributing to one of the landmark developments in Alström Land as well as throughout the global community of those who suffer from rare disorders. Get ready and stay tuned! We are very excited!
All the best and thank you in advance to each and all!
ASI Executive Director
Barack Obama, President of the United States, has announced a new initiative called the Precision Medicine Initiative. What he was really talking about was making medicine relevant and appropriate for specific individuals with specific clinical needs and specific treatments through the use of cutting edge medical technology. Because of its partnership with Genetic Alliance and other advocacy organizations, Alstrom Syndrome International is a part of this initiative which was introduced in the recent ASI Newsletter. Sharon Terry, leader of the project, CENA –- was present at the White House event for the announcement yesterday, January 30, with dozens of other patients and advocates. In fact, a young woman, a survivor of and advocate for a rare liver cancer, introduced the President. This was a day of recognition for all of you who suffer from Alstrom Syndrome and for all those who suffer from rare conditions. The work will create a large national registry, of which ASI is a part. We have a jumpstart on everyone. The President and NIH are looking to our CENA coalition to lead the way.
Robin and I were actually in Washington to meet with Sharon Terry when we were advised that she would not be available – she was at the White House! We still met with the Genetic Alliance senior staff, and feel incredibly fortunate and thrilled that ASI is part of this project.
We expect our Alstrom Syndrome Registry to be available in March. Let’s all do our part!
After that exciting morning, we also met with several of the expert physicians who were part of the recent NIH Alstrom Syndrome Study at the NIH Clinical Center. We enjoyed knowing that we were walking in the footsteps of those of you who have participated in this study.
You will be happy to know that an amazing amount was learned for all of you that will advance Alstrom Syndrome research well into the future, and the staff remembers each and every one of you and send their best regards. There are aspects of the study that we definitely hope to pursue to add to our understanding of Alstrom Syndrome!
All in all an exciting time for all of us.