
Do you remember the day when we could trust the Food and Drug Administration as being an unbiased drug review agency? Ever since the FDA started collecting user fees in 1992, it has appeared that their reviews have been a bit biased toward the drug manufacturers. The quality of the reviews got even worse when they implemented the “Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review” processes was implemented in 1997. Now drugs that meet the accelerated designation can be approved within 60 days with as little as 10 patients in the clinical trial. The drug manufacturers don’t have to demonstrate significant condition improvement, they leave that for the aftermarket studies. But who pays for the aftermarket studies, PATIENTS AND EMPLOYERS! Families of patients with orphan conditions are willing to pay whatever it takes to give their loved one’s relief, and in the case of employee benefits, employers are stuck paying the bill for drugs that cost a million dollars or more per year that have not been proven to be effective. For the life of me, I don’t understand why drug manufacturers are not asked to foot the bill for drugs that go through accelerated approval.
Now, the FDA has done the right thing. They pressed the pause button on the experimental ALS drug NurOwn. The panel of Food and Drug Administration experts voted 17-1 that drugmaker Brainstorm's stem cell-based treatment has not been shown to be effective for patients with the fatal, muscle-wasting disease known as ALS, or amyotrophic lateral sclerosis. Brainstorm's single 200-patient study failed to show that NurOwn extended life, slowed disease or improved patient mobility. The FDA agreed to convene the panel of outside advisers after ALS patients and advocates submitted a 30,000-signature petition seeking a public meeting. Approving this drug would have given ALS patients a sense of false hope that this drug would improve their condition. That is simply not fair.
Now there has been an unusual turn of events, a Senate committee is considering a bill that would allow provisional approvals of the drug and other investigational treatments for rare, incurable diseases. The FDA already has an accelerated approval process, but apparently it is not fast enough for some of those in Congress. I don’t want to sound insensitive, but we need to make sure that drugs that are released into the larger US population are safe AND effective before we start requiring employers to pay for these medications. Until the drug has been proven effective, and compared head to head with other drugs on the market, the cost of these medications should be born by the drug manufacturers as part of their clinical trial expenses.
We need to Expect More from our clinical trial process.
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