From the NEJM: The FDA's New “Breakthrough Therapy” Designation

['lizbeth]

I still don't understand why Perjeta is not approved for all HER2 positive Stage IV and Metastatic Breast Cancer patients, no matter if it is first line, second line, third line or 10th line. I would expect that it will also be effective for lower expressors of Her2.

I am with you Sarah, and Phil - the seriously ill should have more access to drugs, even ones in clinical trials.

I learned early on that the drug approval system accepts the deaths of patients as a necessary part of the process of drug approvals. They must keep the data pure, and the process controlled. However - if the point of the drug is to save lives why not save the lives of those in the most desperate need?

Perhaps more trained personnel could be helpful. The US has tried to spend their way out of the Great Recession. We are going to see the repercussions of this eventually. We cannot continue to indefinitely spend more than we earn. Adding personnel to an already strained budget would not be as effective as changing the mindset of the FDA and big pharma.

I don't know about this lottery system? Is that a European thing? The US system is usually begging for patients to join clinical trials. I think I read only 2-3% of cancer patients enroll in a trial.

I'm hoping that more and more treatments come from the Genome Project too. But there is other science that is being shunned, like the functional profiling. When we combine the treatments of genetic pathways with the personalizing of functional profiling of an individual's cancer - and the FDA allows the patient access to the treatments most effective for their cancer (not the highest percentage of a randomized group) - then real progress will be made.