How should the rare community react to this week’s news from bluebird bio?

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.

• To enact change, we all need to amplify our collective voice towards Governments, regulators, and payor bodies. Innovation clearly still outpaces process, with technicalities and mechanisms requiring urgent attention to truly recognize transformative value and subsequent cost effectiveness
• We need to further collaborate to get to the heart of the burden of living with a rare condition over many years. We need to involve patients, their families and other care givers to fully understand the societal impact of rare diseases and the possibilities offered by a transformative therapy
• We need to create further supportive platforms that allow the voice of the patient community to be truly heard as part of the decision-making process

This led me to consider how we, as an industry, can change the dynamic:

• Encouraging regulators and payors to change assessment processes is a collective challenge, but industry needs to shout loudest. We need more consolidated efforts, through bodies such as ARM, EUCOPE, and ABPI, to make the case for change
• We need greater cross-industry collaboration on a ‘vision towards affordability’. Are there ways that we can further remove duplication of efforts through the deployment of programs to meet specific gaps, rather than replicate them by individual companies?
• Patient advocates still feedback regarding transparency and communication. If patients are truly at the center of our thinking, how can we further work to create appropriate information-sharing channels, with urgency and sensitivity, that help to sustain trust and collaboration through challenging times?

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

Email: GavinJones@openhealthgroup.com