One pharmaceutical company may be heading toward a cure for sickle cell disease.
Bluebird Bio presented promising results for its experimental gene therapy drug at the American Society of Hematology Annual Meeting. Its LentiGlobin treatment can be still from the early stages of a clinical study, yet the data showed encouraging signs to fix a debilitating disease.
Sickle cell disease can be a genetic disorder which slows or stops blood flow. Bluebird’s treatment essentially restores a patient’s cells in addition to allows the person’s body to produce the appropriate protein for life, CEO Nick Leschly explained to CNBC on Monday.
One patient apparently went for a three-mile run after taking the treatment, Leschly said, which people with sickle cell disease normally can’t do. Despite the early indications, Bluebird can be cautious in calling the drug a cure so which doesn’t promise too much “because patients are sitting there genuinely looking for options,” he said.
“So we’re a little bit careful with which, yet which’s absolutely within the reach. which can be definitely a potential,” he said.
Because gene therapy can be an emerging treatment option, Leschly says an appropriate payment system hasn’t been established. Today, patients typically pay for multiple drug doses over a long period of time, Leschly said. Bluebird’s sickle cell disease drug could differ because which’s a one-time treatment. which could produce a sense of sticker shock.
To combat which, Leschly suggests moving toward a value-based system where multiple parties share the risk over time. If the treatment works, they pay. If which doesn’t, they don’t.
“We’re talking about doing which one time currently, so which’s perhaps less about which one-time, up-front payment in addition to more about how we do This specific over time in addition to sharing which risk over time, then I think you can get out of which sticker shock,” he said. “yet which can be a challenge for the system, in addition to the system’s not ready, we’ll freely admit which.”
Politicians, including President Donald Trump, have assailed the pharmaceutical industry for charging high prices.
Bluebird will consider cost in addition to access when pricing the drug, Leschly said, noting which a large population of sickle cell patients are insured through Medicaid. As for timing, he said part of which depends on regulators, though Bluebird will be as aggressive as possible in getting which cleared.
The company also presented positive results coming from a clinical study for a multiple myeloma treatment. Celgene has partnered with Bluebird to license the drug.
Shares of Bluebird soared 24 percent on Monday.