Pill Instead of a Needle May Soon Be Option for RA
Studies Show a New Kind of Drug Works at Least as Well as a Current RA Biologic and Is Effective as a Stand-Alone Treatment
From Lab to Pharmacy
In May, a panel of advisors voted that despite the risks, the FDA should approve the drug. The agency was set to make its decision on the drug this month. But last week, the drug's manufacturer, Pfizer, announced that regulators had asked for more information and said they expected that the FDA's decision to be delayed.
Pfizer has not revealed how it will price the drug if approved, but it is expected to be expensive. The company said in a statement that it is committed to helping patients get access to the medication should it become commercially available.
If approved, tofacitinib is expected to be a blockbuster, generating sales of more than $1 billion a year, according to EvaluatePharma.
How much money it makes will depend on how doctors use it. Much of that will ride on the labeling the FDA might require. The agency could say that it should only be used in combination with methotrexate, for example. Or it could say that it should only be prescribed after other medications have failed.
"Will I use it in every patient? Probably not because I will still start with methotrexate," says researcher Roy Fleischmann, MD, a rheumatologist with the Metroplex Clinical Research Center in Dallas.
Fleischmann says it makes sense to start with methotrexate because it's a much less expensive drug. But he says for the roughly two-thirds of RA patients who don't respond to methotrexate or can't tolerate it because of side effects like mouth sores and hair loss, "I wouldn't hesitate to use this" as a stand-alone medication before trying a biologic.
Fleischmann has accepted grants and consulting fees from Pfizer. He was paid by the company to conduct the studies.
But other experts think that how the drug should be used is still an open question.
"Will this drug get added to methotrexate? Will [it be a] substitute for methotrexate? Will it be used before a biologic or after a biologic failure? That will depend on experience, on talking to other doctors, on seeing what the insurance companies allow us to do," says Richard Furie, MD, a rheumatologist who is chief of the division of rheumatology at North Shore Long Island Jewish Health System in New York.
Other experts agree.
"While this is a pretty exciting drug because this is a new biologic pathway -- it's not just a 'me too' drug -- it does have issues with respect to, frankly, our total lack of understanding about who might be best suited to take it," Curtis says.