I've created a breakdown of the potential patient population to get an idea of Korlym's valuation. I would like feedback on the clinical review and whether the drug pricing model is sane.
Please do not criticize me personally. I am not perfect. I am not paid by anyone to write this. I am interested in hearing the other side of the discussion, so if you're going to dispute my model, please rebut my data with facts and links I can verify.
"Korlym faces a 12 FEB 2012 PDUFA date. Here, we analyze Korlym’s market potential by examining current Cushing syndrome treatments."
Last FDA says PDUFA date is the 17th. No A/C.
Your analysis did not include possible use for psychotic depression.
Hi Joe, I'm an MD PhD candidate right now in grad school too. Nice site, I disagree with some of your picks but I really appreciate the time you take to keep ur site updated especially as a grad student.
I dont understand why in your article you say "mifepristone only works 80% of the time" when both the other 2 drugs work less than 75% of the time... You outright state that mifepristone works BETTER than Ketoconazole and Metyrapone but word it such that it reads that Mife is not as good as these two drugs...
The PPS of CORT is high and I agree that the upside on approval would not be super but still 30% is 30%...
I think there is a problem with the assumptions though. The most important is the incidence of Cushing's. While the rate is commonly cited at 1 in 200,000 it is actually much higher when you included subclinical cases. In studies of obese type II diabetics shows a rate in that patient population of 2-5%. Globally, 150 million people have diabetes. In patients with simple obesity, 9.33% were found to have Cushing's Syndrome. PMID 20126340
There were 300 medical journal articles published in 2011 and 2,400 published in the last ten years. Why would there be this much research into a "rare" disease? Because it is not rare. There are political and cost reasons for maintaining the myth that "Cushing's is rare". Much of this published data advocating a few per million is fabricated by those who have a financial interest.
Diagnosis of Cushing's has to be near 100% certain before referring a patient to surgery. The total cost to diagnose and refer a patient to surgery is closer to $50,000-$100,000 to reach the first surgical outcome. Many patients need numerous labs, MRIs, Inferior petrosal sinus sampling and so forth in order to confirm and diagnose. Surgery is not benign.
As you write, the surgical outcome is problematic, with surgeon self-reported cure rates way higher than actual by the five or ten year point. There are only a handful of surgeons competent in this surgery, because if part of an endocrine tumor is left behind, the disease isn't cured.
Often the responsible neuroendocrine tumor is not in the pituitary, though it may be less than 1 mm in size, making it impossible to find. There aren't satisfactory techniques to locate these and even small carcinoids may not be found because NP-59 scintigraphy isn't available anymore. (U of Michigan no longer makes the secret sauce) These patents and surgical failures are good candidates for pharmaceutical treatment.
There is no reason to believe the medication would sell for the same cost when sold in packs of three for pharmaceutical abortion as it does through the current managed distribution channel. That pricing reflects it's use in a specific treatment plan. It is not a difficult medication to manufacture, compared with for instance recombinant human growth hormone which retails in the range of $12,000 per year. I believe this reflects the upper-most potential price point for the korlym but I believe the volumes will be way way higher.
You can't really directly compare this medication with the other medications. Ketoconazole will work for some patients, but it's safety is limited for some by liver toxicity.
The larger market is the use for treatment resistant depression. Mifepristone will find wide application in this if the safety experience is satisfactory.
Interesting points to consider regarding an underdiagnosis of Cushings, thanks for the link to the article. My concern is that by expanding the indication, now you are prescribing a tightly-controlled abortofacient with significant side effects of its own to a much larger group. That definitely changes the risk-benefit ratio.