Hedge Fund Manager, Value, Event Driven, Special Situations
Contributor Since 2011
Heretofore, the opinions of MS patients and their physicians have been overlooked in the conversation over Lemtrada. What do the regulators want? What do the corporations want? Ultimately, who cares what either want? This is a question about a choice between a patient and her doctor. And both patients and their doctors are starting to speak up. One recent petition started by a patient currently has
45 66 signatures including a noteworthy number of physicians. I am sure that additional signatures would be welcome. Meanwhile, what do petitioners say about Lemtrada?
Patients with more severe MS definitely need effective treatments. The FDA needs to approve this drug, even if not as first line.
- Dr. Mitchell Isaac
This medication should be used for a specific group of MS patients, and they and their physicians should be allowed to make the decision. A strong REMS should be in place.
- Dr. Brian Steingo
While the FDA did not agree with the trial methodology used for Lemtrada, the evidence of efficacy was overwhelmingly clear. MS patients deserve to have access to more treatment options for their condition. Finally, this drug is already approved in Europe, Australia and Canada based on the same data provided to the FDA, which should be taken into account.
- Tyler Robinson
Bottom line - the patients need more options and while the FDA didn't like the trials, the evidence of efficacy was overwhelmingly clear.
- Nicole Romagossa
Please approve Lemtrada. Patients need more choices. Compared to what else is out there, Lemtrada seems to be one of the best.
- Eric Milner
This is of the utmost importance to me. Because this medication has not been approved by the FDA I have had to search for a neurologist that would give me the only medication that has worked for me. My chances are now 1 in 106 of getting a deadly disease called PML. Dont you think that is enough reason. The risks with this Lemtrada are not deadly and can be monitored for and even reversed. Let me choose the only medication that has the same efficacy as what is working and going to be discontinued (for me) due to my risks. I already know how life was for me before. I guess the FDA has forced my hand in taking the high stakes risk to become severely disabled or die. What would you want if this was you. I can take you back a couple of years and show you life in my shoes with quickly progressing Multiple Sclerois, not a nice journey. Now I can work full time, play with my kids, drive, dance and that is how I want to live. Please approve Lemtrada.
- Colleen Rutherford
I have had very active MS for 20 years and was moving out of a Relapsing Remitting course of MS (RRMS) into a Secondary Progressive (SPMS) phase until I found a doctor who would treat me with alemtuzumzb off-label (my MS was too old to qulify into a study). I received my first treatment in 2010 and second in 2011. I have been exzcerbtion free since 2010!!!!
My MRI scans improved the first two years but in 2013 saw some new activity so opted to join an alemtuzumab study and received another round (3 days) of infusions. I developed Graves disease which was diagnosed in the summer of 2013 (prior to my third round of infusions). This is not a big deal. I have no vision problems, no ophthalmopathy, and treatment is a small pill (methamiazole), once daily. I feel great! My EDSS (disability) score has improved. I can walk, dance, ride a bike, and exercise just like I used to do (well almost, I still have MS fatigue and MS heat sensitivity). I want ALL MS patients to have this medication as an option when they are making therapy decisions. No other medication shuts down disease progression AND improves disability beside alemtuzumab. Please bring this drug to the US market.
- Karen Mumley
Another petitioner testified that,
The FDA, by demanding a placebo controlled trial for Lemtrada, is going against the Declaration of Helinski that declares such placebo controlled trials are unethical when an active comparator is available. Genzyme took the high road, therefore, by going against the advice of the FDA in using an active comparator. Why not make this double dummy then? Genzyme answered this at the Ad Com hearing; namely infusion reactions would unmask the drugs. So why put people thru 2 years of fake shots (3 times per week) for the sake of a sham masking? Would you want to stick in needle in your thigh over 300 times, knowing it was a fake? Isn't better to make the raters of the MRI's blinded?
The Stanford Professor of Biostatics said it best in his testimony at the Advisory Committee; simply, it is hard to have bias when looking at MRI's for lesions on the brain.
Another petition has
96 183 signatories (and growing each time I click on the link). This is a grassroots effort to organize patients who are pleading for their right to have more choices.