"I'm optimistic we will see similar products in a few years' time to treat other forms of cancer", the professor said.
Although the price of the therapy is high by any standard, Cooper stated that Novartis is "committed to doing everything we can to ensure that children and young adults who can benefit from [tisagenlecleucel] have access to the therapy and we are working with payers to ensure they fully understand the value of [it] and provide coverage for patients". Other CAR trials at Penn are exploring the technique for prostate cancer, melanoma, and triple-negative breast cancer.
What sets the drug CAR-T apart from others, is that it is known as a "living drug".
This involves having to travel to a limited number of approved centres across the US and then waiting for the individually tailored treatment to be sent back. His team plans to wait for a review on the drug class from the Institute for Clinical and Economic Review, due in March.
"It must be remembered that the current chemotherapies and bone marrow transplants are also very toxic, with major side effects".
Soliris from Alexion runs at $542,000 each year to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Asked if he thought Novartis's pricing was reasonable, Craig Devoe, acting chief of medical oncology and hematology at Northwell Health Cancer Institute in NY, told AFP: "It's not". Most ALL patients respond to initial therapy, but Kymriah will be an invaluable back-up option for relapsed and refractory patients who now have poor prognosis. If a patient receives the treatment, but doesn't show any response to Kymriah in the first month, Norvartis will not charge them, CNN reported. "Obviously this will change the lives of many patients and their families and the field". But because the pay-for-performance deal is with a government payer, this arrangement will mark a step forward for the model as it can be publicly tracked, he added.
The FDA said it has granted 76 applications for trials involving experimental CAR-T therapies. For now, only about 600 people a year will be eligible for Kymriah. Other revenue would come with additional indications, however. To some, that price tag is unjustifiable.
"Of course, we have to talk about pricing from a national standpoint", he said.
For now, Kymriah is only available to a small subsection of cancer patients.
The one-time treatment isn't cheap: Novartis said on Wednesday that the price would be $475,000, which is actually lower price than many expected. Novartis CEO Joseph Jimenez, however, says developing the treatment cost more than $1 billion.
But some patient advocates criticized the cost nevertheless.
Despite the big promises of the therapy, side effects are still a concern, especially given the accelerated approval of the therapy hasn't allowed for long-term patient follow-up. Kite Pharma's CAR-T drug Axi-Cel is now under priority review by the FDA with results to come out in November. The company gained notoriety during its launch of superpricey hep C meds Sovaldi and Harvoni back in 2014 and 2015.