- Bluebird bio Inc. said Tuesday it expects to file three gene therapies for U.S. approval by 2019, setting down a marker for when the biotech hopes to transition into life as a commercial company.
- First up is bluebird's LentiGlobin candidate, which the company plans to submit for European approval in transfusion-dependent beta thalassemia this year. Filings for Lenti-D in cerebral adrenoleukodystrophy (CALD) and for the CAR-T candidate bb2121 in multiple myeloma are slated for 2019.
- While those dates are still some time away, bluebird CEO Nick Leschly isn't waiting to start addressing the difficult questions around price and value. "If you have a life-long value on a patient, you can't expect a payer to pay that all up front at one time," said Leschly. "How do we address that?"
This year bluebird bio secured a coveted slot presenting in the Grand Ballroom of the Westin St. Francis, where the J.P. Morgan Healthcare Conference is held each year.
Leschly used that stage and top billing to take an unorthodox tack to pitching a room full of high-powered investors and analysts. The CEO chose not to fill his presentation slides with colorful waterfall plots of patient responses to treatment or detailed financial models.
Eschewing a data-heavy approach, Leschly instead featured personal patient stories across each of the company's many therapeutic areas. Such a focus formed a bridge to broaching one of the most challenging topics for companies making strides in cell and gene therapy: what to charge.
Leschly laid out several of the key hurdles that impede easy solutions for how to appropriately pay for gene therapy, flagging Medicaid Best Price regulations and a mismatch between upfront payment and long-term value as particularly thorny roadblocks.
"If you want to get creative there [in pricing], you have got to take down some barriers," Leschly explained. "We want to recognize value over time. We want to share risk and we need the participants in the system to play ball with us on that."
A particular issue in the U.S. is the frequency at which individuals change insurance providers. Insurers which pay for a patient to receive a (potentially) near-curative gene therapy will want to ensure they are the beneficiary of that long-term value.
"If people are moving payers, how do we make sure — just like a preexisting condition moves with you — maybe a financial preexisting condition moves with you," Leschly mused. "But that's okay. Now you have allowed a payer to be willing to invest in these procedures because they are not worried about this amortization problem."
Bluebird has no illusions that some of these problems go beyond its ability as a public company to address. Many are embedded in the foundation of a healthcare system set up to pay for recurring treatments given for chronic disease — not a one-time lump sum for a cure.
Spark Therapeutics Inc. recently tested these waters, announcing it would charge $425,000 per eye for its groundbreaking gene therapy Luxturna (voretigene neparvovec). While the price may not have broken the $1 million mark, Luxturna is now easily one of the most expensive drugs available. At the same time, Spark unveiled arrangements with a regional insurer and the major pharmacy benefit manager Express Scripts to better align payment with value and to streamline billing.
That bluebird is thinking about these problems now is a positive sign. Such forethought should help the company down the road if all goes according to plan. But delivering a pricing strategy that pleases investors as much as it wins over payers remains a high bar to clear.