A Date-Rape Drug for Fibromyalgia: How Could This Possibly Go Wrong?
Clearly, the FDA advisory committee looking at Jazz Pharmaceuticals (JAZZ)'s application for Rekinla -- better known as the street drug GHB -- has its work cut out for it.
For executives interested in risk management, Jazz's attempt to persuade the feds that making GHB (pictured) available by prescription for fibromyalgia will be an interesting case study: Is there any way in which an inherently risky product with a spectacular history of consumer abuse can be made safe?
You don't have to look very far for clinical doubts as to whether fibromyalgia is actually a disease. Here's the Mayo Clinic's definition:
You hurt all over, and you frequently feel exhausted. Even after numerous tests, your doctor can't find anything specifically wrong with you. If this sounds familiar, you may have fibromyalgia.
Fibromyalgia symptoms often begin after a physical or emotional trauma, but in many cases there appears to be no triggering event.You can read more here. The main concern is that there is no way to clinically diagnose fibromyalgia other than by agreeing with a patients' description of her symptoms; and there's no identified clinical cause of the condition. It seems to be helped by drugs as powerful as Pfizer (PFE)'s Lyrica and practices as feeble as Tai Chi. Here's what Robert Shmerling, clinical chief of rheumatology at Beth Israel Deaconess Medical Center in Boston, says about the disease:
Fibromyalgia is so common, and we have such a difficult time treating it effectively. It's defined by what the patient tells you," he added. "It's hard for some patients' families and their doctors to get their head around what it is and whether it's real."There's one person on the FDA panel for whom the question of whether fibromyalgia is real could be a hotbutton issue: Public Citizen's Sidney Wolfe. Earlier this year Wolfe chastised the FDA for approving Savella, a different drug for fibromyalgia from Cypress Bioscience and Forest Labs (FRX):
There has never been an advisory committee for any of the three FDA-approved drugs for fibromyalgia (Lyrica, Cymbalta, and Savella); thus, none of the issues relating to the definition of the disease, endpoint measurements, risk/benefit ratio, length of studies, and adverse reactions have been discussed by experts in the field.Next comes the tricky issue of how you stop the drug being abused. Here's the first sentence of the FDA's briefing document:
... Jazz Pharmaceuticals, Inc.['s] Sodium oxybate, also known as gamma-hydroxybutyrate (GHB), is a Schedule III central nervous system depressant with hypnotic properties that has well recognized abuse potential.Ouch! The drug is already on the market under the name Xyrem to treat narcolepsy. Only about 150,000 people in the U.S. have that condition. Jazz's new proposal for fibromyalgia would extend the drug to a patient base of as many as 5 million.
The briefing document makes a good case that the actual use of GHB as a date rape drug is relatively low. Only two cases of sexual assault have been linked to Xyrem:
Although some cases of drug facilitated sexual assault involving GHB have been documented in the scientific literature, no cases of sexual assault have been reported to the [drug facilitated sexual assault] committee over the last seven years (2002-2009).Jazz is proposing a laborious system under which patients could only obtain the drug through the mail from 15 specialty pharmacies. The greater concern is abuse of the drug by addicts or recreational users. In a single year Jazz received 237 reports of improper use of Xyrem by patients, the FDA found.
The FDA's tentative conclusion is that so few patients are treated with Xyrem that it can't say whether Jazz's anti-abuse program successfully curtailed abuse or whether there are simply not enough patients for abuse to show up.
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Image from wikimedia, CC.