Mark Thornton, a former medical officer at the FDA and current president of the Sarcoma Foundation of America, uses the recent Provenge approval to illustrate why the FDA needs to reverse its position on the cancer drug Mepact.
Back in 2007 the FDA decided that both Provenge and Mepact should go back to the clinic for a new study. Dendreon was able to afford that because it was pursuing a mega-blockbuster approval for prostate cancer. But the developers of Mepact were essentially asked to go back to the drawing board and recruit virtually every sarcoma patient in the U.S. in order to get the data it needed to qualify for an approval.
"Imagine if a clinical trial for a new asthma or diabetes drug required every single patient in the country to be included not once but twice and you will begin to appreciate the profound predicament those of us in the rare-disease community face when it comes to the FDA's irrational standards," writes Thornton in an op-ed piece in the Wall Street Journal. He goes on to encourage the new FDA commissioner to develop the kind of creative regulatory pathway that Europe has in place for new drugs that treat rare diseases. And he called on the FDA to make a rapid u-turn and approve Mepact immediately.
IDM Pharma had developed Mepact. Japan's Takeda bought IDM a year ago and handed control of the Mepact program over to its U.S. cancer subsidiary, Millennium: The Takeda Oncology Company.
- here's the op-ed piece from the Wall Street Journal