Assessing the impact of bluebird bio’s exit from “untenable” European market:
WINDROSE’S TAKE ON BLUEBIRD BIO’S EXIT FROM EUROPEAN MARKET
Potential ripple effects for gene therapy access
Bluebird bio, one of the world’s earliest innovators in the gene therapy space, recently announced that they will move away from launching and operating in Europe as a company, driven primarily by misalignment around the value and prices of their rare disease portfolio.
On bluebird’s Q2 2021 Earnings Call, Andrew Obenshain, President of Severe Genetic Diseases at bluebird, stated that “[European] Governments and payers have not yet recognized and appropriately priced the innovative value of our transformative gene therapies,” calling the situation “untenable” for launch of new therapies.
The bold decision appears to be the culmination of a number of failed negotiations with European governments and payers. In April 2021, bluebird announced that they would be withdrawing Zynteglo, intended to treat beta thalassemia, from the German market due to failed pricing negotiations. April’s announcement also included a scaling down of staff and infrastructure in Europe, an apparent sign of things to come.
Obehshain’s most recent statement expands this disillusionment to the entirety of Europe. “The price levels that we were seeing were not sufficient for us to move forward in Europe,” he said, meaning that “as a small innovative company, we cannot afford to wait it out.”
It remains to be seen whether bluebird will pursue partnerships with companies possessing more prominent footholds in Europe, and how this news will impact their impending spin out of their oncology business unit.
Windrose’s take:
The withdrawal from Europe by bluebird could act as both a lesson and a warning for future gene therapy and rare disease developers. While it’s certainly disheartening news for patients specifically seeking access to bluebird’s therapies, the pipeline of cell, gene, and other rare disease therapies is too robust to ignore. Stakeholders in Europe and abroad will need to come together to make progress here, as there is too much at stake from a political, economic, and population health standpoint to allow full portfolio withdrawals to become a normal occurrence.
In the meantime, bluebird may also provide a clearer directional roadmap for future entrants. As a result, we believe a number of key questions must be considered as we forward:
How do other gene therapy manufacturers choose to (or not to) approach European payers? Will they be more hesitant to invest and/or launch in Europe given bluebird’s experience? Or will this create a new urgency to adapt industry pricing models and expectations?
How can gene therapy manufacturers proactively address payer uncertainty, given the realistic limitations around trial design (e.g., small patient populations, lack of clear comparator, use of nontraditional/surrogate endpoints) in this space?
How can gene therapy manufacturers support price expectations while remaining mindful of payer affordability/budget concerns? How can managed entry/annuity models be adapted to drive more uptake in European markers?
For products with complex delivery and safety protocols, how can manufacturers engage with payers and governments ahead of submission to ensure they are providing the most pertinent cost information to drive efficient decision making?
Will small and even midsize gene therapy manufacturers need to lean even more on larger companies with significant presence in Europe to help launch and market their products
How will European patients and physicians react to this news? Will they engage with payers to encourage progress toward accessing these medicines or stand in solidarity against perceived excessive pricing (or possibly both)?
Will European payers respond to the pressure, or dig in their heels to avoid setting unrealistic expectations and/or precedents?
Will local gene therapy development, academic “home brews”, and/or medical tourism outside of the EU proliferate as a result?
Does this create additional burden on the US to financially support these innovations? Could it create more urgency for the US to consider international price referencing as a way to control costs?
Article written by Colin Nash Senior Principal at Windrose Consulting Group.
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