drsclafani wrote:Please continue the discussion about how to design trials from your perspectives. I have already incorporated some of your ideas into my plans for the study to follow the safety study. I will share your thoughts when our physician colleagues discuss a multicenter trial
but for the moment, you guys should take the lead for a while.
Dr. Sclafani asked for suggestions on how a trial might be designed. Here's my two cents:
KISS: Keep It Simple Scalfani.
Drug companies approach MS trials from two different categories: 1. Disease Modifying Drugs and 2. Symptomatic Management Drugs.
Symptom management trials are the easiest. You simply test for the efficacy of a drug or treatment based on a single MS symptom. The FDA recently approved Ampyra, a drug that it says helps people with the symptoms of multiple sclerosis walk more rapidly.
The National MS Society reports that 35 to 43 percent of those who took the drug in Acorda's two phase III clinical trials had improved walking ability. The increase in function was 10 to 30 percent of baseline compared with those taking a placebo.
Amphyra Use and Side Effects?
Ampyra is intended to be taken as a pill twice a day. Side effects experienced by study participants included back pain, dizziness, insomnia, fatigue, nausea, balance disorder, urinary tract infection, falls and headache, according to the MS society.
Acorda Pharmaceuticals has announced a wholesale price of $1,056 for a 30-day supply (60 pills), or $12,850 annually. That pencils out to $17.60 a dose, or $35.20 for each day’s two doses.
Is Ampyra Worth the Cost?
It will be up to individuals and their physicians to determine whether Ampyra’s benefits justify its steep price. David Phillips, in a commentary on Bnet, is not sure that they do. He suggests that regulators may have downplayed safety issues and ignored the relatively light efficiency of the drug because of pressure from the MS society which, he points out, funded early stages of Ampyra research.
43% of patients taking this drug had function increase from 10-30% over baseline.
Can we find an MS symptom that Liberation will improve better than this in a trial?