Discussion Forums > Working Towards a Cure
Bluebird Bio Releases Data on Current Gene Therapy Trials
JV:
Sharmin...you bring up an excellent point...
Andy Battaglia:
I have received further explanation about the RAC recommendation.
--- Quote ---"The RAC brought up challenges intrinsic to developing novel therapeutics in the pediatric population across therapeutic areas and modalities - not unique to gene therapy trials. Ultimately, evaluating the risks and benefits for this population are decisions that are really made on an individual basis, by individual families, investigators and sites.
And, as our CEO told Adam Feuerstein from The Street, “We respect RAC and the process. RAC took a reasonable position when it comes to dealing with kids. We disagree and believe the decision [to proceed with a gene therapy pediatric study] should be placed in the hands of the institutional review boards, the physicians of patients and their families.”
(My Question to Bluebird). Is Bluebird bound by RAC or can they proceed on their own?
"The outcome of the RAC review is a series of recommendations and advice, and it does not constitute a formal approval of a proposed protocol. The RAC review is specific to the U.S. and their comments are provided to the IRBs at U.S. trial sites, who individually decide on whether to follow the recommendations.
We do appreciate the recommendations from the RAC members and will take the RAC feedback on the timing of initiating HGB-208 under advisement. Of course, we will continue to work closely with the regulatory authorities and our clinical study sites to pursue appropriate accelerated regulatory approval pathways in the U.S. and the EU."
--- End quote ---
JV:
Thank you for the follow up Andy...doesn't sound like it will be a problem but further evaluation in children cant hurt either.
Andy Battaglia:
Bluebird feels that it is their decision to make and that their scientists are in the best position to know if trials with children should commence. The RAC has made a rather general recommendation covering all research using children in trials. I feel that specific therapies must stand on their own merits.
Canadian_Family:
Good News.
Andy,
Thanks for sharing Blue thoughts.
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