Here’s an example of how drug companies work the drug development system:
Part 1: A Successful Phase III Study for a New Myelofibrosis Drug
This week, results of the second phase III trial of a drug called INC424 (co-developed by Novartis and Incyte) were announced. The drug is being tested for the treatment of myelofibrosis (MF), and was found to significantly reduce the spleen size of patients with this disease, compared with the current best therapy. Spleen size is a measure for clinical improvement for myelofibrosis. Novartis predicts that sales of INC424 will exceed $500 million annually once its approved. The two clinical trials of INC424—COMFORT-I and COMFORT-II—will serve as the basis for a new drug application, which Novartis plans to submit to the FDA soon.
(Phase I/II clinical trial results are here)
That’s just for MF, a disease where patients are in need of improved treatments (from what I understand, this disease is not always serious, but a large portion of mortality is due to treatment-related problems, when treatment is needed). But MF is also an orphan disease, that strange siren of pharmaceuticals. A representative from Novartis told Reuters that sales for INC424 (also called ruxolitinib) would likely exceed $1 billion annually if the drug is subsequently approved for polycythemia vera, a rare blood disorder.
Part 2: A Successful Phase III Study for a New Rheumatoid Arthritis Drug
INC424 belongs to the same class of drugs as tofacitinib, an agent being developed by Pfizer for rheumatoid arthritis. Tofacitinib is considered one of the most promising drug’s in Pfizer’s pipeline. These drugs are JAK (janus kinase) inhibitors, and they interfere with the molecular mechanisms that lead to inflammation. JAK inhibitors could radically alter the treatment of rheumatoid arthritis, which means tofacitinib could be a blockbuster drug.
And all signs point to that being the case. Also just recently, Pfizer announced successful results of the second phase III clinical trials tofacitinib for rheumatoid arthritis.
So, two drugs in the same class, with promising clinical trial results announced in the same week, on track for the treatment of two different diseases.
Part 3: What’s Going On?
On the one hand: great! Two diseases in need of improved treatments are going to get them. Rheumatoid arthritis is a painful disease with harsh and debilitating treatment options. On the other hand, it’s a great example of the inner workings of drug development. Tofacitinib (until recently known as tasocitinib) will corner the rheumatoid arthritis market (more than one million people suffer from RA in the United States), while INC424 will rake in its cash from two orphan diseases.
And as Novartis knows so well, treating rare diseases—those occurring in 200,000 people or fewer—can be extremely profitable. Novartis is the company behind Gleevec, the antileukemia drug that was developed under the Orphan Drug Act, which gives companies financial incentives to create new treatments for rare diseases. This drug saves lives daily. It turned out to be hugely profitable because patients have to take it daily.
Did Novartis’s prior experience with an orphan drug lead it to focus its JAK inhibitor on the narrow market for myelofibrosis (and, soon, polycythemia vera)? Or was Pfizer simply the first to get its JAK inhibitor into a study for rheumatoid arthritis, restricting Novartis to other diseases? Novartis’s drug entered phase III first, but if it was further along than Pfizer’s, why didn’t Novartis start a rheumatoid arthritis study?
And does it matter? If people suffering from rheumatoid arthritis, myelofibrosis, and polycythemia vera (which, though rarely, can lead to leukemia) are in need of new treatments, then isn’t having both compounds succeed good news, regardless of how they got there? Still, shedding light on the business strategies seems like a good way to understand pricing and marketing practices. Common versus rare: both constitute business decisions that guide marketing and the cost of a drug. For example, analysts are quoted by Reuters as saying that they believe myelofibrosis is underdiagnosed. Does that mean we’ll be seeing an uptick in MF diagnoses soon, or at least a new “ask your doctor” commercial?
I hope to do more research into the simultaneous development of these agents (and there are more JAK inhibitors to come). But in the meantime, this small dip into the water.
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