After Novartis put a $2.125 million sticker on its spinal muscular atrophy gene therapy Zolgensma, all eyes were on how Bluebird Bio would price its own gene therapy for transfusion-dependent beta-thalassemia (TDT). And now, industry watchers have some answers.
Bluebird will price Zynteglo, formerly known as LentiGlobin, at €1.575 million ($1.77 million) in Europe, the company said during an investor presentation Friday. Just like Novartis did with Zolgensma, Bluebird is offering a five-year, results-driven installment payment plan. After the initial €315,000, patients only pay the same amount each year thereafter if they continue to respond to the therapy.
As for the U.S., where Bluebird expects to win a Zynteglo go-ahead in 2020, the company said it plans to price the drug within a “reasonably close” range, Reuters reported.
Earlier this month, the one-time drug was granted conditional approval by the European Commission to treat patients 12 years and older with TDT, a disease caused by mutations in the beta-globin gene that leads to reduced or absent hemoglobin. The drug’s effectiveness will be measured by whether a patient needs blood transfusions to maintain blood count.
At this year’s J.P. Morgan Healthcare Conference in January, Bluebird's CEO Nick Leschly laid out that payment model, which he said is “putting up to 80% of our price at risk based on success.”
Bluebird does have some new data that has bolstered confidence in Zynteglo’s long-term efficacy. In the phase 1/2 Northstar trial, eight of 10 treated patients who did not have a particular genotype were transfusion free for at least 10 months and up to 45 months. And in those who did have the genotype, three among eight achieved that target for a median duration of 16.4 months, Bluebird unveiled at the 24th European Hematology Association Congress on Friday.
“The maturing data from our clinical studies of LentiGlobin for TDT show that patients across genotypes are able to achieve and maintain transfusion independence with stable production of gene therapy-derived-hemoglobin [...] extending for years,” Bluebird chief medical officer David Davidson said in a statement.
The $1.77 million exceeded SVB Leerink analyst Mani Foroohar’s previous estimate of $900,000 in Europe or $1.2 million in the U.S. But it did come in below $2.1 million, what Bluebird said is the “intrinsic value” of the therapy.
“We understand that each country in the EU has a different approach to assessing value and determining reimbursement, and we are working with each country to provide them with all of the necessary information to conduct their country-specific assessment,” a Bluebird spokeswoman told FiercePharma.
Gene therapies often come with the promise of a cure with just one dose for diseases that would otherwise require lifelong care. But the current healthcare systems, with their “pay-as-you-go model,” are not built for these one-time treatments, Novartis CEO Vas Narasimhan noted in recent CNBC commentary.
“[W]e need new economic models to determine exactly how much value [a cure] represents” when compared with the cost and suffering saved from longtime chronic care, he wrote. He also called on Congress to clarify that value-based payment models, like the ones Novartis and Bluebird have proposed, will not trigger anti-kickback provisions.
Editor's Note: The story has been updated with additional information from Bluebird's presentation.