FDA Accelerated Approval of Genervon's GM604 for Use In ALS
My name is Nick and I am 54-years old. In October 2011, I was diagnosed with ALS. Today, I am in a wheelchair and cannot walk or stand. My arms grow weaker almost every day and my breathing is starting to be affected. I used to be very active but now I can only leave my home when I have someone with me who's strong enough to lift me into my wheelchair once I'm done scooting down to the bottom of the stairs on my butt. It can feel like I'm a 100-year old man who can't do things for himself anymore. At 54, I should still be a vibrant part of my community, but this disease is kicking me where it hurts. Through the enthusiasm generated by the Ice Bucket Challenge, many people now know that ALS is a degenerative neurological disease that currently affects over 30,000 Americans, and as it stands the disease is always fatal. Most people with ALS are given only 2-5 years to live. When I was diagnosed, I was devastated - it took me months to accept it. Although I am now resolved, I have not lost hope. Currently, there is only one drug available to treat ALS (Riluzole) and it is only able to extend life expectancy by a couple months. That's why the recent news about a small company named Genervon Biopharmaceuticals and their trial drug "GM604" has been so exciting for the Global ALS Community. While most trials have a hard time even finding a positive trend, the GM604 trial data supports the view that this drug could very well be is a game changer in the battle against ALS. There are multiple, statistically significant data. Not only in clinical data and results alone, but also correlated biomarkers data and results as well. (Please see "Files" section on the campaign website: https://sites.google.com/site/aap4gm6/) Genervon met with the FDA in February 2015 and made a potentially life-changing request on behalf of the ALS community; they asked the FDA to promote GM604 to the Accelerated Approval Program with Post-Marketing Phase 4 Requirements, so all ALS patients can have legal access to GM604 now. Under the FDA's Accelerated Approval Program, the treatments would be covered by health insurance. Although Genervon knows that this request might complicate their relationship with the FDA, they were willing to take a stand and do everything they can to help the ALS community because it is the right thing to do. If the FDA does not grant Accelerated Approval, it will likely be 3 more years before patients are able to access this drug -- meaning that most people currently living with ALS will not live to see it reach market. While acknowledging the paramount importance of public safety in the FDA’s drug approval process, I believe that ALS presents a compelling case for an exception to the usual process. I am asking you to help me appeal to the FDA through their sense of compassion for those of us with this dreadful illness. People with ALS don't have time to wait for another clinical trial to be completed. By signing this petition, you'll be helping patients like me request that the FDA expidite the way potentially life-saving treatments are made accessible to people with ALS -- starting right now with GM604. Doing so could very likely mean a change in the course of ALS progression not only for myself, but hundreds of thousands of other patients worldwide. After you sign, please follow this link to tweet and/or email the FDA: https://www.change.org/p/lisa-murkowski-fda-accelerated-approval-of-genervon-s-gm604-for-use-in-als/u/9475826 Thank you, Nick Like us on Facebook: https://www.facebook.com/gm604forals Please go to the Genervon website for a true understanding of the facts and the science behind GM604: http://www.genervon.com/genervon/about_pressreleases.php
Make Election Day a Federal Holiday. Americans Deserve Time and Equal Opportunity to Vote.
We the people believe that voting is the heart and soul of democracy. Nearly 60% of voting-eligible Americans did not vote in the last midterm elections, citing work or school-related conflicts as the primary cause. Americans deserve time and equal opportunity to vote. Blue Point Brewing Company, along with other forward-thinking companies, has committed to giving our employees the day off to vote. But we need implement this change on a bigger scale. Join us in creating the change and protecting democracy. Convince Congress to establish Election Day as a Federal holiday. Blue Point Brewing Company is committed to this initiative and will be making Voters' Day Off beer cans available for supporters to sign and further inspire change.
Stop Forcing Mail-Order Pharmacy as the Only Option of Coverage
Patients' lives depend on choice. **Since starting the petition, I have realized that there are many issues other than temperatures with forced mail-order pharmacy. Mail-order pharmacy is very loosely regulated. There are life-threatening delays, lack of face-to-face relationship with pharmacists for people with chronic conditions, and rapid closures of our independent pharmacies; although, a recent study showed people prefer independent pharmacy (2018, Gill). *************Our Story************* Our son, received a life-saving liver transplant at the age of 2 from a 3-year-old little girl. His life depends on the potency and effectiveness of chemotherapy/immune suppression medications every 12 hours to prevent his body's immune system from fighting off his transplanted liver. In the past mail-order delivered his liquid oral medications in nothing but a plastic envelope on a 102-degree day on a hot enclosed not temperature controlled UPS truck. Shortly after, he went into liver rejection which could have resulted in complete liver failure or death. I speculated that the medication could have been too weak after the delivery of medications in high heat. I vowed to never again risk his life with mail order pharmacy. Recently, we were mandated/forced to only use mail-order pharmacy in order to receive coverage for his life-saving medications. The package arrived in only a bag on a hot day without an ice pack. The hot non-temperature controlled enclosed delivery truck can reach temperatures up to 170 degrees. His labs elevated again afterward. My son wants to know, "Why would they do that?" I contacted the manufacturer, who performs the testing stated that both of my son's medications should be discarded and considered less potent once stored above 86 degrees as higher temperatures and freezing could both result in lower potency. Liquid medication is the most harmed by the mishandling of medications outside of the manufactures temperature storage guidelines. I contacted the mail-order pharmacy who refused to replace or take back the medication. They said the law & USP Pharmacopoeia allows them to ship up to 104 degrees, although the manufacturer states it is not proven safe at these temperatures. However, I have received communication from USP Pharmacopoeia who writes guidelines for storage, and they also said that the mail order pharmacy should follow the manufacturer's guidelines of 59-86 degrees for storage. Again, the trucks reach up to 170 degrees which is much hotter than 104. I contacted the FDA, who states that the mail order pharmacy should be using the manufacturer's guidelines that have been proven safe..However, since the mail-order pharmacies are regulated loosely by the State Board of Pharmacy, not the FDA there was nothing that the FDA could do. I made over 30 calls to the insurance company begging for them to please let us pick my son's medications up at the local pharmacy at which they are filled. My son's physician wrote a note/appeal as his transplant team has stated that they have tried to voice their concerns about this issue with their pediatric/child patients and no one is listening! The insurance company still denied the doctor's appeal for us to pick up my son's medications in the safest way. It was not until the Media became evolved that the insurance company budged. I felt helpless and have united with many other pharmacists, physicians, patients, mothers and fathers, and caregivers who feel the same way. Helpless. Mail-order of prescription drugs should be a choice, not the only option of coverage. Mail-order pharmacies may appear to save money, but when my son ended up in the hospital after taking medications that could have been compromised by having lower potency, the cost of the rejection was thousands of dollars. If his liver would have fully failed, the cost of his liver transplant for just 5 days (he was in the hospital for 5 weeks) was over $1,000,000. The lax regulation and oversight may save money on prescription drug plans but may come at an increased cost to the health plan itself. Also, keep in mind the endless waste of medications that automatically are sent regardless of whether or not patients need them. Also, people with chronic, complex conditions, should always have the option of face to face interaction with a pharmacist who knows their complex needs and medical history. The pharmacist and patient relationship is crucial to the successful outcome of the patient's overall health. Taking this away is harmful to patients and be more costly to our already stressed healthcare system. Another important fact. Mandatory mail-order programs are discriminatory. It is estimated that 40% of the homeless are disabled. How is mandatory mail order fair and working for them as they may not have an address and not even know where they will be from day to day? Only allowing mail-order pharmacy for coverage is unethical and irresponsible. I share stories on my social media sites every day of patients who are suffering from lack of choice. We need your help to make mandatory mail-order an option, not a mandate. YOUR URGENT SUPPORT WILL SAVE LIVES! THANK YOU!! WE APPRECIATE ALL OF THE SUPPORTERS, CAREGIVERS, & PATIENTS. Thank you, Loretta Boesing, Founder of Unite for Safe Medications You may make a tax-deductible contribution to support our advocacy here Email: firstname.lastname@example.org Facebook Page: Issues with Mail Order Pharmacy @justamomwholovesherson Twitter: @BoesingLoretta The greatest way that you can help is by sharing this petition & gathering 2-3 people in your community who are having issues and speak to your legislatures. Reference: Gill, Lisa L. “Consumers Still Prefer Independent Pharmacies, CR's Ratings Show.” Product Reviews and Ratings - Consumer Reports, 7 Dec. 2018, www.consumerreports.org/pharmacies/consumers-still-prefer-independent-pharmacies-consumer-reports-ratings-show/