Written by Gavin Jones, Global Advisor, Rare Disease on Friday 13th August 2021
Bluebird bio have announced that they are sadly exiting Europe as it believes that it is untenable to continue. I feel for the thalassemia, sickle cell, and leukodystrophy communities. As an advocate, patient, or parent, the journey will have undoubtedly started with caution, but will have evolved to optimism and excitement about a treatment option that has the potential to be transformative. Given this journey and the efforts of these communities to support the company in getting these therapies to patients, this decision will be very difficult to absorb. I also feel for the members of the European bluebird bio team that I have gotten to know; they always impressed me with their passion and expertise in trying to make things work.
So how should the rare community react to this news?
Firstly, I think we need to be concerned. A decision like this is monumental for a company like bluebird bio, given its mission and years of investment. We need to recognize that blocks to patient access were beyond borders, resulting in significant delays for patients. An innovative company the size of bluebird bio can only delay paying its bills for so long and, whilst you may not feel huge sympathy, I fear that without companies like this active across the world, progress in new treatment development will be diminished. I think we should be particularly concerned that research will become focused on safe options and many disease areas could once again be neglected.
I would ask that patient advocacy groups continue to collaborate with industry partners. We may not always talk your language, have a full understanding of your needs, and may have conflicting priorities but we are trying, and we can’t make progress without you! Together we are stronger and more capable of overcoming the challenges brought home this week and in bringing gene therapies to people most in need.
This led me to consider how we, as an industry, can change the dynamic:
We can’t lose faith or focus despite this significant setback in the developing story of gene therapy. We’re in a difficult chapter, and we need to turn the page carefully together if we are to reach a happier ending.
If you'd like to discuss this further, please reach out to Gavin Jones, Global Advisor, Rare Disease at OPEN Health