Thalassemia Patients and Friends

Discussion Forums => Announcements => Topic started by: Andy on July 10, 2017, 09:43:40 AM



Title: Bluebird Bio Need People from the US for a Patient & Caregiver Advisory Board.
Post by: Andy on July 10, 2017, 09:43:40 AM
Request from bluebird bio (US patients only) for Patient & Caregiver Advisory Board on September 26th in Chicago, Illinois.
... at bluebird, we are embarking on an effort to better understand the medical needs and wants of those living with transfusion dependent beta-thalassemia. In order to do this, bluebird bio seeks to hold a Patient & Caregiver Advisory Board on September 26th in Chicago, Illinois.
Why Now?
Believe it or not, 2015 marked five years since a small gene therapy company called Genetix became bluebird bio and 2016 marked three years since the first patient with transfusion dependent beta-thalassemia had undergone infusion with bluebird bio’s LentiGlobin drug product in HGB-205.
As we continue with our LentiGlobin development program, we want to learn more from patients and caregiver to ensure we are focused on what they find most important so that we can better serve the thalassemia community.
Our Ask
bluebird bio is soliciting nominations for participation on the transfusion dependent beta-thalassemia Patient & Caregiver Advisory Board. This Advisory Board will convene for a single day and provide advice and recommendations to bluebird bio on topics pertaining to unmet medical needs concerning the treatment of transfusion dependent beta-thalassemia and perceptions of gene therapy. Furthermore, agency guidance and policies, patient preference, benefit-risk determinations, quality of life or health status issues and communication preferences are among the topics that may be discussed at the Advisory Board.
If you know of anyone who may be interested, please ask candidates to self-nominate by contacting our partner agency - Snow Companies (www.snow-companies.com):
Linda Lynch
757-903-9915
linda@mypatientstory.com
Tonia Chadwell
757-634-7519
tonia@mypatientstory.com
Specifically, we seek transfusion dependent beta-thalassemia patients and caregivers living in the United States. Additionally, nominees must be 18 years or older and need to be able to speak and understand English. Furthermore, nominees should not work for or sit on a board of an advocacy organization.
Nominations received by July 15th, 2017, will be given first consideration for participation on the Advisory Board. Nominations received after July 15th will be considered for nomination to the Advisory Board as later vacancies occur. Please note that nominees will go through a screening process; therefore, there is a chance that not everyone screened will be able to participate.
Other Information
During the screening process Snow companies will ask potential participants to provide detailed information concerning such matters related to financial holdings, employment, and research grants and/or contracts to permit evaluation of possible sources of conflicts of interest.
Participants will be required to sign a consulting and confidentiality agreements for participation in the Advisory Board.
An honorarium will be provided as compensation for participants contributions to Advisory Board and for time away from family and/or work. Additionally, travel and meals will be covered.
Thank you
We appreciate your help in making interested individuals aware of this unique opportunity. If you have any questions, please don’t hesitate to reach out to me with any questions. I am more than happy to discuss the nomination process and the Patient & Caregiver Advisory Board with you further if you desire.
Thank you in advance for your assistance!


Title: Re: Bluebird Bio Need People from the US for a Patient & Caregiver Advisory Board.
Post by: Pratik on July 11, 2017, 12:18:46 AM
Great news! Andy, does this treatment require chemotherapy?

And what about acceleron pharma? I think that's just an oral drug and won't require chemo?


Title: Re: Bluebird Bio Need People from the US for a Patient & Caregiver Advisory Board.
Post by: Andy on July 11, 2017, 04:46:34 PM
Gene therapy does require chemo preparation.

Luspatercept does not require any preparation.


Title: Re: Bluebird Bio Need People from the US for a Patient & Caregiver Advisory Board.
Post by: Pratik on July 13, 2017, 05:34:02 AM
Gene therapy does require chemo preparation.

Luspatercept does not require any preparation.
Nice to hear that!