Petition updateFDA Accelerated Approval of Genervon's GM604 for Use In ALSWe are running out of time. Please send a tweet to senators who can help get GM6 passed right now!
Nicholas GrilloSan Francisco, CA, United States
3 Feb 2015 — http://answerals.org/ A decision from the FDA on GM6 could come any day. Please click here to send a tweet to the senators who can help get it approved: http://ctt.ec/j230_ Or, to email the two top senators on the U.S. H.E.L.P. Committee: https://www.change.org/p/janet-woodcock-m-d-fda-accelerated-approval-of-genervon-s-gm604-for-use-in-als/u/9425621 Yesterday the ALSA posted an official consensus statement with several other organizations declaring they will not support GM604 advancement to the FDA’s Accelerated Approval Program because of the small numbers of subjects and short duration of the trial. Here's the rebuttal to that out-dated and unscientific way of thinking: "Clinical trials with small sample sizes can provide very useful results in the context of drug efficacy. Not all clinical trials can consist of hundreds of patients due to incidence rates of the disease or recruitment issues. These small clinical trials should not be viewed as meaningless simply due to the sample sizes. In fact, these small clinical trials can be very helpful when determining the meaningfulness of the effect size seen in the efficacy measurements. "Clinical trials that contain small sample sizes put the active treatment at a distinct disadvantage. These small clinical trials will be under-powered to detect significant differences between the active and placebo groups in almost all, if not all, efficacy endpoints. The statistical hypothesis test starts from assumption that there is no difference between the active treatment and placebo. A p-value of less than 0.05 is typically the threshold to reject that hypothesis and claim that the active treatment is superior in terms that specific efficacy endpoint. However, to achieve that threshold with small samples sizes, there must be complete or near complete separation of the result between the placebo and active treatment groups. For example, in the change from screening in FVC, 3 of the 4 changes from screening in the placebo group were less than the smallest change from screening in the GM604 group, leading to a p-value of 0.0476. There is obvious separation between the treatment groups in change from screening in FVC. This result should not be mitigated because of the small sample sizes. In fact, this result should be considered quite strong given the lack of power to detect that difference. "If there was only one significant result, one could argue that it was spurious and even what you would expect with a Type I error rate of 0.05, regardless of the size of the clinical trial. However, when several endpoints show statistical significance and others are trending that way and have p-values near the 0.05 threshold in the presence of small sample sizes, there is an obvious signal that the active treatment is working. The active treatment is demonstrating an efficacious response that should be heralded, not as a random result found due to the small sample sizes, but as a powerful result found in spite of the small sample sizes." Paul J. Lupinacci, PhD Director of Biostatistics CRO of Genervon It is important to remember that the ALSA, like many well-funded, older, connected disease organizations, takes a traditionalist approach to the development, approval and availability of new medicines. They take the money from a lot of corporate sources, including quite a few drug companies and other conservative companies and with the money always comes some control of the message and positions the organization can take. They become conservative and careful in their positions, and eventually stray from a part of the original mission. Instead of being a patient advocacy organization that represents real patients, they become companies that must always consider the effect of doing or saying anything potentially controversial against the effect it will have on their donations (i.e., their source of revenue that pays the bills and allows them to exist as an organization with employees, salaries, health benefits, offices, travel and fund-raising budgets, etc.). Most large, well-funded disease groups are primarily fund-raising companies that spend the raised funds on mainstream, non-controversial things. I assure you, if anyone at the ALSA or their loved one had ALS, they’d be doing anything possible to try GM604 given the statistically significant data (which was provided to the ALS Association on January 28, 2015 and never even mentioned in yesterday's consensus statement). The ALSA is what it is. They do some good things. The individual local chapters have and do make a substantial difference in our everyday lives, but the job of National to initiate changes and advocate for tangible medical advancements that benefit the prognosis for ALS patients is nowhere to be found. Remember their list of action items from the meeting they held with MDA 2 years ago? That ended up being just words about change. Nothing on that list has taken place: http://www.alsa.org/news/archive/alsa-and-mda-urge-fda.html Is all of this ugly? You bet it is. What can we do about it? Just go around it. No one has more credibility about the patient perception of potential benefits versus risks (including the risk of the disease) than someone who has the disease. Now, if you don’t agree with ALSA, you shouldn’t support them, and you have to make it clear to the FDA that the ALSA does not speak for us. We speak for ourselves. “I have ALS and the ALSA does not represent me!” Start using this tag line on all your posts and use this hashtag as well #alsadoesnotrepme What we are trying to do by getting AAP for GM604 is happening amid a larger backdrop in which pressure is mounting to change the old system into something much better. Our best bet is to target the FDA via the Senate H.E.L.P. Committee. We must insist that they convince the FDA to be flexible so GM604 can reach patients who need it now while also collecting additional information from patient experiences and ongoing non-randomized trials that can lead to full approval of the drug. It’s a no-brainer, right? We get access to GM604 while scientists get their data collected over a longer period of time. Starting right now, we have to be our own advocates, and our audience right now is Congress. It is urgent that we tell the Senators on the H.E.L.P. Committee that they have to make a change to the way ALS patients are able to access new drugs and treatments that could change the course of ALS progression and prognosis. Tell these senators that a change has to happen now starting with Genervon's GM604. Here’s the link to contact those senators: https://www.change.org/p/janet-woodcock-m-d-fda-accelerated-approval-of-genervon-s-gm604-for-use-in-als/u/9425621 Warm regards, NIck Grillo and Jehad Majed
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