So the FDA Missed Review Deadlines on New Drugs Like Nplate—Isn’t It a Good Thing to be Cautious?

News about Nplate seems to focus on the fact that the FDA missed the review deadline by about a month.

Oy vey. The drug industry in general is apparently pretty crabby about the idea that the FDA may be getting a little bit more cautious about approving new drugs after the Vioxx debacle. Heck, when you spend an average of 15 years developing a drug, you don’t want it sitting on the shelf for an extra month or two—regardless of any potential for deadly side effects.

Amgen’s platelet-stimulating drug may well have sat there for a reason.

Of course the FDA had questions. 14 of the 204 patients studied apparently had adverse reactions, eight patients developed a resistance, and there’s an increased risk of clotting or of serious bleeding when the patient stops taking the drug. The FDA was also concerned about abnormal marrow growth and malignant tumors.

“Serious adverse reactions associated with Nplate in clinical studies were bone marrow reticulin deposition and worsening thrombocytopenia after Nplate discontinuation. Additional risks include Bone Marrow Fibrosis, Thrombotic/Thromboembolic Complications, Lack or Loss of Response to Nplate, and Hematological Malignancies and Progression of Malignancy in Patients with a Pre-existing Hematological Malignancy or Myelodysplastic Syndrome (MDS),” reads Amgen’s disclaimer.

So the FDA had to figure out whether Nplate should be approved, and if so, under what—if any—conditions. Which turned out to include an ongoing long-term safety study.

Everything has consequences. Anyone with a platelet disorder that hasn’t responded to other treatments may well decide that the risks are worth it, and Amgen is to be applauded for developing a last-ditch drug. But patients deserve to be informed about what those risks are, and the drug industry should back off on criticism of the FDA’s requests for more safety data or a lengthy pause for thought.

What's your opinion? If you take Nplate, are you having any side-effects, and if so, are they what you expected? Do you think the FDA took too long to approve Nplate?