The FDA signaled that it supported a new accelerated pathway for approval of new drugs for serious diseases that use biomarkers and show high response in early trials.
U.S. may speed approval of 'breakthrough' drugs
When the Critical Path was launched, the goal was to align the approval of drugs with the science of drug development. That process was derailed after the Vioxx panic. It is now back on track thanks to a bi-partisan effort on Capitol Hill, support from the Obama administration and the persistence of Janet Woodcock and Peggy Hamburg at the FDA. This is a great day for innovation and patients, at least in principle.
There will be those who see this reform as a sop to evil, profit making drug companies... Such as Merrill Goozner: "Pfizer last week won Food and Drug Administration “accelerated approval” for Xalkori (crizotinib) for a rare form of metastatic lung cancer that strikes about 3 percent of people — almost always non-smokers — who come down with the disease. That’s about 10,000 patients per year in the U.S., and perhaps a similar number abroad. Multiply that times the $115,200 a year the company plans to charge for Xalkori, and they’ve got themselves a billion dollar blockbuster. Through this period where there will be no concrete proof of efficacy, Pfizer will get to charge an outlandishly high price for the drug. Is there a way to put pressure on the company to complete the trials in a more timely fashion, so that the paying public knows that it is truly getting value for its health care dollar and not subjecting patients to some pretty rough side effects (nausea, diarrhea, etc.) for no benefit?"
Goozner is either ignorant or willfully misleading. By definition accelerated approval will be reserved for treatments that show significant benefits in specific groups of patients through well-characterized biomarkers and clinical endpoints. That will cut the time required for approval and reduce costs. (If Goozner can figure out a way to reduce the cost of producing new biologics for small groups of people, he should start his own biotech company.) The price of drugs will be relative to their value. Or should be.
The question is, as accelerated approval is adopted will CER just add back delay and access. Let's hope the courageous impatience of the FDA, the Obama administration, Congress and the public is shared by the CER crowd and PCORI.
U.S. may speed approval of 'breakthrough' drugs
When the Critical Path was launched, the goal was to align the approval of drugs with the science of drug development. That process was derailed after the Vioxx panic. It is now back on track thanks to a bi-partisan effort on Capitol Hill, support from the Obama administration and the persistence of Janet Woodcock and Peggy Hamburg at the FDA. This is a great day for innovation and patients, at least in principle.
There will be those who see this reform as a sop to evil, profit making drug companies... Such as Merrill Goozner: "Pfizer last week won Food and Drug Administration “accelerated approval” for Xalkori (crizotinib) for a rare form of metastatic lung cancer that strikes about 3 percent of people — almost always non-smokers — who come down with the disease. That’s about 10,000 patients per year in the U.S., and perhaps a similar number abroad. Multiply that times the $115,200 a year the company plans to charge for Xalkori, and they’ve got themselves a billion dollar blockbuster. Through this period where there will be no concrete proof of efficacy, Pfizer will get to charge an outlandishly high price for the drug. Is there a way to put pressure on the company to complete the trials in a more timely fashion, so that the paying public knows that it is truly getting value for its health care dollar and not subjecting patients to some pretty rough side effects (nausea, diarrhea, etc.) for no benefit?"
Goozner is either ignorant or willfully misleading. By definition accelerated approval will be reserved for treatments that show significant benefits in specific groups of patients through well-characterized biomarkers and clinical endpoints. That will cut the time required for approval and reduce costs. (If Goozner can figure out a way to reduce the cost of producing new biologics for small groups of people, he should start his own biotech company.) The price of drugs will be relative to their value. Or should be.
The question is, as accelerated approval is adopted will CER just add back delay and access. Let's hope the courageous impatience of the FDA, the Obama administration, Congress and the public is shared by the CER crowd and PCORI.
