Committee on Energy and Commerce

Fred Upton

And They’re Off! #Ride4Gabe Journey Begins for #CuresNow

2016/07/29

WASHINGTON, DC – Fresh off the heels of their Wednesday briefing on Capitol Hill, the folks behind Hope for Gabe kicked off Ride4Gabe 2016 today in Maine. As 11-year-old Duchenne patient and all-star rare disease advocate Gabe Griffin said this week, “We must pass #CuresNow!” Learn more about Gabe’s story here. [[{"fid":"1328","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"242","width":"351","class":"media-element file-full"}}]] Ride4Gabe 2016 commenced this morning at 6 a.m. in Houlton, Maine, and will stretch more 2,200 miles to Gabe’s home state of Alabama. The goal is to raise awareness for Duchenne and the #CuresNow effort. The cycling team will hit 9 states in 11 days and will be accompanied by a documentary crew as they head for Mobile, AL. The cyclists expect to ride a total of 200 miles today to Lewiston, Maine. [[{"fid":"1329","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"231","width":"413","class":"media-element file-full"}}]] If You Have 36 Seconds, WATCH THIS: Gabe’s father Scott narrates short video highlighting Gabe’s visit to the U.S. Capitol this week. Follow Ride4Gabe 2016 Mile by Mile www.ride4gabe.com - Live tracker HERE www.facebook.com/ride4gabe/ @Ride4Gabe Hashtags: #Ride4Gabe, #CuresNow, #EndDuchenne, #MakeDuchenneHistory Follow Cyclist Michael Staley: @mcstaley The House Energy and Commerce Committee will be following the journey on the #Path2Cures closely, and we hope you will too. To learn more about 21st Century Cures, click here. ###   Read More

Leading Duchenne Advocates Rally Support for #CuresNow

2016/07/28

[[{"fid":"1327","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"144","width":"468","class":"media-element file-full"}}]] WASHINGTON, DC – On Wednesday, leading advocates to #EndDuchenne rallied support for the 21st Century Cures Act. The leaders of Parent Project Muscular Dystrophy (PPMD), the Foundation to Eradicate Duchenne, and Hope for Gabe addressed a packed house in the U.S. Capitol on the importance for #CuresNow. Also in the crowd was the Ride4Gabe 2016 crew, who is making their way to Maine to commence on Friday the 2,200 mile bike ride to Mobile, Alabama, to raise awareness for Duchenne and the Cures effort. The cycling team will hit 9 states in 11 days and will be accompanied by a documentary crew as they head for Mobile, AL. 11-year-old Duchenne patient and all-star rare disease advocate Gabe Griffin stole the show yesterday. Gabe told the crowd he wanted to play football for Alabama, Ohio State, or Michigan and be either a policeman or in the army when he grows up. Gabe exclaimed to the crowd, “We must pass #CuresNow!” Learn more about Gabe’s story here. [[{"fid":"1325","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"251","width":"468","class":"media-element file-full"}}]] “We are united in our effort to get 21st Century Cures across the finish line – and the standing room only crowd clearly shows we’ve got the wind at our backs in this effort,” said Joel Wood, President of the Foundation to Eradicate Duchenne. “For my son James, and for kids like Gabe, it’s time for #CuresNow, and it’s time to eradicate Duchenne.” “The Duchenne community stands together in sending a clear message that patients are counting on Congress to pass the 21st Century Cures Act. We are energized as all eyes are on September for #CuresNow. Accelerating the discovery, development, and delivery of treatments will make a tremendous difference for Duchenne families and provide great hope,” said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy. PPMD is currently rallying behind the Ride4Gabe team by activating PPMD’s grassroots network to connect riders with local Duchenne advocates in each state to raise Duchenne awareness and spread the message that “it is time for #CuresNow.” “For kids like Gabe, Duchenne is an everyday reality,” said his father, Scott Griffin. “We owe it to them to make Cures their new reality. We are so thankful for the work that is being done to win this fight, and we’re ready, now more than ever, to see it through.” Follow Ride4Gabe on Facebook here, and click here to view photos from Wednesday’s event. To learn more about 21st Century Cures, click here. ### Read More

COMBATING OPIOIDS: Upton Highlights Sweeping New Law in the South Bend Tribune

2016/07/28

House Energy and Commerce Committee Chairman Fred Upton (R-MI) this week highlighted the sweeping new law to fight the nation’s opioid epidemic in the South Bend Tribune. The legislation includes over a dozen bills passed by the Energy and Commerce Committee. Full committee Chairman Fred Upton (R-MI) chaired the House and Senate conference meeting that finalized the comprehensive opioid package that passed the House by a vote of 407 to 5 and passed 92 to 2 in the Senate. [[{"fid":"1323","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"82","width":"230","class":"media-element file-full"}}]] July 27, 2016 Viewpoint: Addressing the opioid crisis By Rep. Fred Upton The simple truth is that we’re in the middle of an opioid abuse epidemic. Nearly every 12 minutes, someone in America dies of a drug overdose and the Centers for Disease Control reports that more than a quarter billion opioid prescriptions were written in 2012 alone. Abuse of opioids has impacted every community. In my home state of Michigan, we are enduring 10 times as many deaths today as there were 15 years ago. Indiana was ranked 16th nationally for drug overdose deaths in 2013. Our efforts to address this opioid crisis date back to 2015. We started with bipartisan hearings and round tables — listening to experts, stakeholders, individuals in recovery, and families as to what we in Congress could do to stem the tide of this epidemic and help those in real need. Folks on the front lines traveled to Washington to testify, including Cass County Prosecutor Victor Fitz. What we heard was eye-opening. One area of agreement was that these dangerous drugs must not get into the hands our kids. Nearly 800,000 children between the ages of 12 and 17 tried opioids for the first time within the past year. We also agreed that our legislation should not unjustly penalize patients who legally use opioids to alleviate chronic pain. Recently, the House of Representatives advanced a conference report for S. 524, the Comprehensive Addiction and Recovery Act, by a vote of 407-5. I was proud to chair the conference committee that worked to get the details of this package right — ironing out the differences between the House and Senate packages in an effort to achieve consensus. What our package will do is attack this public health crisis from all sides, from zeroing in on treatment for addiction and overdoses, to reforming prescribing practices and the delivery of medicines, and working with our law enforcement. We also made a targeted funding authorization of $181 million to help on a wide array of opioid abuse efforts. Because of our common-sense approach, our package achieved the support of more than 250 of the nation’s leading addiction advocacy groups. Then, last week, the U.S. Senate advanced our package by a vote of 92-2. Last week, President Obama signed the comprehensive package into law. Lives are at stake, and this critical legislation will help make a real difference. Read the column online HERE. ###   Read More

COMBATING OPIOIDS: “Help is on the Way.”

2016/07/26

Building upon the House Energy and Commerce Committee’s bipartisan #RecordofSuccess, President Obama late last week signed sweeping legislation to fight the nation’s opioid epidemic. The legislation includes over a dozen bills passed by the Energy and Commerce Committee. Full committee Chairman Fred Upton (R-MI) chaired the House and Senate conference meeting that finalized the comprehensive opioid package. Following the bill’s signing, Upton commented, “For folks suffering in Michigan and across America who are desperate for relief, help is on the way.” The opioid epidemic has no boundaries - Michigan is enduring 10 times as many deaths today as there were 15 years ago, and the numbers continue to surge. [[{"fid":"1317","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"169","width":"145","class":"media-element file-full"}}]] July 25, 2016 Bipartisan Legislation Seeks to Fight Opioid Epidemic [[{"fid":"1318","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"228","width":"419","class":"media-element file-full"}}]] Watch the report HERE [[{"fid":"1319","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"134","width":"234","class":"media-element file-full"}}]]   July 25, 2016 Upton Praises Signing of Opioid Legislation Legislation designed to reduce the number of deaths caused by opioid abuse has been signed into law by President Obama. The Comprehensive Addiction and Recovery Act is designed to strengthen treatment options, treat infants born to addiction, and increase state prescription monitoring programs. The plan was championed by Congressman Fred Upton in the House, with Upton saying deaths as a result of opioids have become a national health crisis. The package was approved by both the Senate and House by a wide margin with bipartisan support. Congressman Upton says it’s a “thoughtful solution that attacks the opioid epidemic from all sides — one that zeros in on treatment for addiction and overdoses, reforms prescribing practices, and works with law enforcement.” Read the story online HERE.   ### Read More

#RecordOfSuccess: Commonsense Policy to Export Liquid American Freedom Already Getting the Job Done

2016/07/26

As Energy and Commerce Committee Chairman Fred Upton (R-MI) says, the proof is in the pudding. And the benefits of lifting the 40-year-old ban on crude oil exports are already unmistakable. The legislative effort that says #Yes2Energy was designed to lower prices at the pump, support job growth, and strengthen America’s national security and geopolitical influence. A recent report by the Conservative Reform Network points to record results. The first tankers loaded with liquid American freedom arrived in Europe in January, and already this year, American oil exports are on track to reach a 90-year high. Lifting the ban was a top priority for the Energy and Commerce Committee. Last year, the House passed H.R. 702, authored by Chairman Emeritus Joe Barton (R-TX), legislation to explicitly lift the export ban, and H.R. 8, the North American Energy Security and Infrastructure Act, which contained provisions lifting the ban. The third time was the charm as the committee was finally successful in getting the effort across the finish line when language to lift the ban was included in the year-end budget deal that was signed into law late last year. [[{"fid":"1315","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"164","width":"265","class":"media-element file-full"}}]] Policy Matters: U.S. Crude Oil Exports Set Records Last fall many conservatives made repeal of the 40-year-old crude oil export ban a policy priority. A number of conservative groups made the case for repeal, including the Heritage Foundation, Americans for Prosperity, and Freedom Works. Twenty conservative and free market organizations urged members of Congress to “support legislation that fully lifts the ban on crude oil exports.” Here at the Conservative Reform Network, we outlined how repealing the crude export ban could help encourage America’s energy revolution, create thousands of high-paying jobs, and lower gasoline prices for Americans. As part of the end-of-the-year omnibus spending bill, Speaker Ryan, Leader McConnell, and other leading Republicans successfully negotiated with the White House to include a full repeal of the ban. This was a significant achievement given that two months earlier President Obama had threatened to veto a bill repealing the ban on crude oil exports. … [[{"fid":"1316","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"367","width":"585","class":"media-element file-full"}}]] Barrels of U.S. crude oil exports per day, in thousands Read the full article HERE.   Read More

Ride4Gabe 2016 to Commence July 29 - 2,200 Miles from Maine to Mobile

2016/07/26

Remember Gabe Griffin? The 11-year-old Duchenne patient and all-star advocate was on Capitol Hill in March barnstorming for 21st Century Cures. The inspiration for the #Hope4Gabe effort continues to work hard raising awareness for a disease that needs more attention, Duchenne muscular dystrophy. Weld for Birmingham reports on Ride4Gabe 2016, three cyclists who will commence riding July 29 for 11 days, 2,200 miles, across nine states to eradicate Duchenne. Weld highlights,  “One of the goals of Ride4Gabe is to urge people to ask their congressmen to pass a law called The 21st Century Cures Act, which was passed by the House of Representatives but hasn’t been approved by the Senate.” As Ride4Gabe 2016 reports this week, “the team consisting of 3 cyclists, 7 support crew, 3 film crew, 2 SUVs, and 1 motorhome will start the journey from Houlton, Maine to Mobile, Alabama.” [[{"fid":"1314","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"268","width":"603","class":"media-element file-full"}}]] Credit: Hope for Gabe Foundation One of the three cyclists riding for Gabe is Michael Staley, a one-time chief of staff for former Rep. Spencer Bachus (R-AL). Staley commented, “We believe that Gabe could be a part of the first generation of Duchenne patients to have a treatment that is approved to fundamentally change his life… We believe it is very reasonable that within his lifetime a drug will be available for him that will make his life a whole lot better. It is very sad because we are racing against time.” Weld for Birmingham also provided an update on Gabe’s condition, reporting, “While Gabe waits for a drug that could help him, his muscles keep deteriorating. He can’t climb up stairs anymore, and he is starting to have trouble getting up off the floor. He gets around his school in a motorized scooter.” GABE GRIFFIN CARAVAN TO STOP IN DC WEDNESDAY TO RALLY SUPPORT WITH DUCHENNE ADVOCATES: The caravan, including Gabe and a documentary crew, is set to drive from Alabama today with a stop scheduled tomorrow at the U.S. Capitol to join other Duchenne advocates to build upon the momentum for 21st Century Cures. Parent Project Muscular Dystrophy is rallying behind the Ride4Gabe team by activating PPMD’s grassroots network to connect riders with local Duchenne advocates in each state to raise Duchenne awareness and spread the message that “it is time for #CuresNow.” Pat Furlong, Founding President and CEO of PPMD, commented, “We are activating our powerful PPMD community so the Ride4Gabe team can join hands with as many Duchenne families as possible to send a clear message that patients are counting on Congress to pass the 21st Century Cures Act.” The cyclists plan to commence their journey from Maine this Friday, and average 200 miles each day as they make their way to Mobile, Alabama. Follow Ride4Gabe on Facebook here. To learn more about 21st Century Cures, click here.  [[{"fid":"1312","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"96","width":"183","class":"media-element file-full"}}]] Riding for Gabe [[{"fid":"1313","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"229","width":"351","class":"media-element file-full"}}]] Three Cyclists Plan a Cross-Country Trip to Raise Money for a Cure for Local Boy and Others Suffering from Duchenne Muscular Dystrophy Michael Staley wakes up at 3:30 a.m., rides a bike for four hours, then puts on a suit and goes to his consultant job at a Regions Bank building in Birmingham as if it was just any other day. A little later the same day, Gabe Griffin tries his new motorized scooter in the school hallways. He is going to start middle school this year and he is excited. Both of them, each in his own way, are getting ready for the Ride4Gabe, where Staley will be doing a 2,200 mile cross-country tour with two other cyclists to raise funds and awareness for Duchenne Muscular Dystrophy. With his red hair, blue eyes and freckles, 11-year-old Gabe has become a symbol for this fight. He is the face and namesake of Hope for Gabe, a foundation that fights so that kids like Gabe who have Duchenne will have a cure in time. Duchenne is a fatal genetic disorder that results in muscle degeneration. At the moment it  doesn’t have an effective treatment. According to Parent Project Muscular Dystrophy, it is the most common fatal genetic disorder diagnosed in childhood. It affects approximately one in every 3,500 male births, with about 20,000 new cases every year worldwide. The loss of muscle mass causes patients to be in a wheelchair in their early teenage years and to die in their 20s or 30s as a result of cardiac or respiratory failure. Gabe was three years old when he was diagnosed with Duchenne. “We kind of knew something wasn’t right,” his father Scott Griffin explained. “He has a twin sister and he wasn’t developing as fast as she was. Our next-door neighbor, who is an occupational therapist, was babysitting him one day and noticed how he got up from the floor. He was doing this thing called the Gower’s maneuver, which people with Duchenne do.” Gabe’s parents hadn’t heard of Duchenne until that moment. When they looked it up on the internet, what they found left them shattered. “We couldn’t believe it,” Griffin recalled. “These kids typically are on a wheelchair between the ages of 10 and 12, and potentially lose their life between 17 and 22 years old. It’s 100 percent fatal.” Their worst fears were confirmed by their doctor. Griffin clearly remembers the day they got the phone call, because it changed their lives forever. “I knew the moment she said hello. I knew he had it,” he said. … One of the goals of Ride4Gabe is to urge people to ask their congressmen to pass a law called The 21st Century Cures Act, which was passed by the House of Representatives but hasn’t been approved by the Senate. According to Staley, that law would help Gabe and people with other genetic conditions. They are especially interested in two provisions that would allow the following: first, that the FDA would rely on data from previously approved drugs for future approvals (for instance, evidence to approve a drug could be based on case histories instead of new clinical trials), and second, that the patients’ perspectives would be included in the drug approval process. Those who love Gabe are optimistic, including Staley. “We believe that Gabe could be a part of the first generation of Duchenne patients to have a treatment that is approved to fundamentally change his life,” Staley said. “We believe it is very reasonable that within his lifetime a drug will be available for him that will make his life a whole lot better. It is very sad because we are racing against time. While Gabe waits for a drug that could help him, his muscles keep deteriorating. He can’t climb up stairs anymore, and he is starting to have trouble getting up off the floor. He gets around his school in a motorized scooter. These things aside, he lives a normal life. However, the emotional side of the illness is more difficult, his father says. “There’s so many things he can’t do, and he just wants to be a normal little boy. He watches his brothers play all these sports and he wants to know why he can’t do it. “And I don’t have a good answer for him. I can say that he has Duchenne but I don’t think he gets the whole picture yet. I think he is smarter than I give him credit for, and he has probably Googled some things, but I don’t think he knows… “I dread the day… Every parent should dread the day that they have to tell their child that they’re going to die.” Staley has been a huge support for the family. “I can’t put it into words,” Griffin said. “He is more than a friend. He is family.” … This year’s Ride4Gabe will cover about the same distance as the Tour de France, 2,200 miles, from Maine to Mobile. Staley and his two fellow cyclists, Wes Bates and Payne Griffin, will be riding 200 miles per day for 11 straight days. They will travel with a documentary film crew and Gabe and his father themselves. The plan is to meet all the political figures they can while en route, as well as other families with Duchenne. The ride will arrive in Birmingham on Sunday, August 7, and cyclists are welcome to join as long as they can keep up the pace. … Like Staley, Gabe’s dad has decided that, instead of burying his head in the sand, he will go out, talk about it and try to make a difference, no matter how hard it gets.   “As a parent, you should never, ever, ever, have to bury your child,” Griffin said. “And I pray that day doesn’t come. As a dad, my job is to make everything better. When the kid falls down and hurts his knee, it is my job to put a Band-Aid on it. Just to… make it better. And I can’t make this better. I can fight, and I will, but I can’t personally put a Band-Aid on this. “But by the same token, I am a parent, and it is my job to jump in front of a moving train for my child. Duchenne is that moving train and I am going to jump in front of it every chance I get to try to save him.” Read the full story online HERE. Read More

#RecordOfSuccess: Sweeping Bipartisan Opioid Package Now Law

2016/07/23

WASHINGTON, DC – Building upon the House Energy and Commerce Committee’s bipartisan #RecordofSuccess, President Obama today signed sweeping legislation to fight the nation’s opioid epidemic. The legislation includes over a dozen bills passed by the Energy and Commerce Committee. Full committee Chairman Fred Upton (R-MI) chaired the House and Senate conference meeting that finalized the comprehensive opioid package. Earlier this month the House voted 407 to 5 and the Senate voted 92 to 2 in support of the consensus bill. “In the face of a surging crisis, we came together and got the job done,” said Chairman Upton. “We have a thoughtful solution that attacks the opioid epidemic from all sides – one that zeros in on treatment for addiction and overdoses, reforms prescribing practices, and works with law enforcement. For folks suffering in Michigan and across America who are desperate for relief, help is on the way.”  For more information on the conference report, click here. ###  Read More

GOTTLIEB: “Regulators made the barriers higher, not lower”

2016/07/22

Momentum continues to build in the effort to deliver #CuresNow. The 21st Century Cures Act delivers a one-two punch of increased resources and sensible regulatory reforms to accelerate the discovery, development and delivery of safe and effective cures and treatments. Dr. Scott Gottlieb, a health care policy expert and former FDA official, writes on the importance of pursuing regulatory reforms at the FDA in Massachusetts’ South Coast Today. He writes, “The issue of whether drugs for rare and deadly diseases should face a more efficient path to patients is a constant tension. New legislation now before Congress would implement a series of measures to make the development and regulatory review of drugs for ultra rare conditions like these enzyme diseases more efficient.”
 He talks about how despite promising progress and advancement over a decades by researchers in treating a rare disease affecting children, “regulators made the barriers higher, not lower.” He continued, “As we improve our scientific understanding of rare diseases, and our ability to target their molecular origin, we’ll have more opportunities to treat and even cure the rarest disorders — but only if policymakers allow us to realize these openings.” To learn more about H.R. 6, the 21st Century Cures Act, click here. [[{"fid":"1311","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"53","width":"468","class":"media-element file-full"}}]] July 22, 2016 Act prods bureaucratic FDA into taking actions
 By Scott Gottlieb
 There’s a group of rare diseases in which babies are born missing one of 11 different enzymes our bodies use to break down fatty sugars in our blood. Absent these enzymes, fat molecules accumulate in critical organs like the heart and liver, often claiming the lives of those afflicted while they are still children. When the first drug to target one of these diseases came along in 1991 — a medicine called Ceredase, which was used to treat Gaucher’s disease — regulators approved the new enzyme replacement on the basis of a small and short trial. The drug was tested on just 13 children and showed it could effectively, and importantly, shrink livers. Regulators at the Food and Drug Administration figured that if the drug was reducing the size of diseased livers, it was also clearing away accumulated fat molecules. With the rarity of these diseases, performing larger trials is hard, if not impossible. Fast-forward a decade and scientists had created similar enzyme replacements for several of the other diseases in this same family of disorders. Yet each time a new and distinct drug was created for one of these slightly different deficiencies, regulators made the barriers higher, not lower, even though we were learning a whole lot more about how enzyme drugs worked to treat these conditions. So when a drug came along in 2006 for another enzyme disease called Hunter’s syndrome, regulators required the largest trial ever for one of these conditions.   It enrolled about 100 children and required the kids to be randomly assigned to get the experimental drug, or an inert placebo, for 52 weekly infusions. The FDA no longer trusted its ability to measure such a drug’s benefit by evaluating its impact on a “surrogate” marker for benefit, like the ability to shrink swollen organs. This time, the FDA required scientists to test the children’s ability to walk and breath. Needless to say, the drug proved highly effective. As for the kids who were obligated to the placebo arm, many were permanently disabled at the trial’s end. The issue of whether drugs for rare and deadly diseases should face a more efficient path to patients is a constant tension.
 New legislation now before Congress would implement a series of measures to make the development and regulatory review of drugs for ultra rare conditions like these enzyme diseases more efficient.
 Among the measures offered by the 21st Century Cures Act is a path for incorporating wider use of “surrogate” measures. These are interim endpoints that can be used to more quickly gauge a medicine’s benefit, such as measuring its ability to shrink a patient’s liver rather than having to wait for kids to accrue enough disability to see if a drug can help them walk or breath better. The legislation also nudges FDA to make wider use of “adaptive” trials that let regulators get earlier answers about a drug’s benefits while testing it on smaller groups of patients. Some think the FDA is risk-averse primarily out of concerns of drug safety. But when it comes to medicines that target rare diseases, it’s rarely a question of safety that hangs up the FDA’s decision to approve a new drug. Instead, it’s almost always a question of drug efficacy. Getting more statistical certainty means enrolling more patients, for longer periods of time, in order to goose the mathematical precision of the results. But in dealing with rare diseases, longer and larger clinical trials come at a big human cost. As we improve our scientific understanding of rare diseases, and our ability to target their molecular origin, we’ll have more opportunities to treat and even cure the rarest disorders — but only if policymakers allow us to realize these openings. Read the column online HERE. ### Read More

MOVING THE NEEDLE: Upton Welcomes NHTSA Release of Documentation Related to Vehicle Safety Systems

2016/07/22

WASHINGTON, DC – House Energy and Commerce Committee Chairman Fred Upton (R-MI) today applauded the National Highway Traffic Safety Administration’s (NHTSA) decision to release documentation for the Vehicle-to-Vehicle Public Key Infrastructure (V2V PKI) initiative proposed by the agency. The documentation was published in the Federal Register on Thursday following the committee’s urging that the information be made public. “We applaud NHTSA’s decision to release this documentation – the information will go a long way in improving roadway infrastructure and highway safety,” said Upton. “It’s important that we give the experts and the public the opportunity to study the documentation and provide feedback to improve the V2V PKI and boost overall confidence in the system. This is a win for consumers, and we are glad we helped move the needle toward greater transparency.” To read the documentation released by NHTSA, click here. ###  Read More

“There’s Hope” - #CuresNow Critical for Brain Research

2016/07/21

The effort to deliver #CuresNow invests in 21st century science and technology to quicken the discovery and development of important research, and accelerate the development of safe and effective drugs for rare diseases. Boosting research is critical, especially when it comes to the brain and neuroscience. Hollis Cline, president of the Society for Neuroscience, and Hahn Professor of Neuroscience at The Scripps Research Institute, writes about the importance of supporting research in The San Diego Union Tribune. Cline writes, “With sustained support, future discoveries in neuroscience will transform our understanding of the brain — and help us race to solve long-term problems like autism, Parkinson’s disease, Alzheimer’s disease and mental illness.” Cline continues, “That leadership not only improves and saves millions of lives worldwide, but also generates significant domestic economic activity and spurs scientific innovation. … Public funding of long-term research is the proven path that will translate into treatments and cures for myriad neurological and psychological disorders that afflict millions of American families. There’s hope. New discoveries in physics, chemistry, biology and engineering are powering the latest revolution in brain research.” To learn more about H.R. 6, the 21st Century Cures Act, click here. [[{"fid":"1307","view_mode":"full","fields":{"format":"full","field_file_image_alt_text[und][0][value]":"","field_file_image_title_text[und][0][value]":""},"type":"media","link_text":null,"attributes":{"height":"107","width":"505","class":"media-element file-full"}}]] July 20, 2016 COMMENTARY: Brain research funding: U.S. must keep momentum going By Hollis Cline          It may not surprise you that the mysterious three-pound mass of tissue found in our heads is the least understood of all the human organs. At the same time, what the global brain research community is discovering about the brain is nothing short of astonishing, and we are poised to make discoveries that will help us one day better understand and treat hundreds of debilitating diseases. Building on strong support in Washington over the last two years, America has the power to keep this momentum going. In a time of great disagreement, Congress took strong bipartisan action at the end of 2015 to infuse the National Institutes of Health (NIH) with an additional $2 billion to support research, the largest increase in more than a decade. This funding boost halted a downward trend in research funding and is a crucial step toward making up for the enduring effects of years of chronic underfunding. Earlier this summer, a bipartisan group of policymakers again demonstrated sustained support for the importance of scientific discovery when the Senate Appropriations Committee approved a bill that would increase the FY17 NIH budget by $2 billion to $34 billion. Last week, the House Appropriations Committee passed its bill, though less robust, to expand funding for NIH in the next fiscal year. The public greatly needs investment in biomedical research to continue, and we urge both chambers to come together to ensure that investment in NIH advances. Investments made through agencies such as the NIH and National Science Foundation (NSF) drive knowledge and fuel innovation in the neuroscience community. Neuroscience, the study of the brain and nervous system, needs consistent and predictable public funding of basic research to unlock the brain’s complex secrets and advance medical progress that ultimately will lead to a better human condition. By its nature, basic science is curiosity-driven, has unpredictable outcomes and ultimately lays the foundation for every new medicine that appears on the shelves of our local pharmacies. NIH and NSF investments in basic neuroscience research have had — and continue to have — widespread impact on our nation, society and on all of us as individuals. The U.S. has long been a leader in biomedical research. That leadership not only improves and saves millions of lives worldwide, but also generates significant domestic economic activity and spurs scientific innovation. Neurological illnesses and mental disorders affect an estimated 1 billion people throughout the world and cost the U.S. more than $760 billion a year. More than 44 million people suffer from Alzheimer’s disease worldwide, and that number is expected to increase dramatically in coming decades. Public funding of long-term research is the proven path that will translate into treatments and cures for myriad neurological and psychological disorders that afflict millions of American families. There’s hope. New discoveries in physics, chemistry, biology and engineering are powering the latest revolution in brain research. … With sustained support, future discoveries in neuroscience will transform our understanding of the brain — and help us race to solve long-term problems like autism, Parkinson’s disease, Alzheimer’s disease and mental illness. Read the entire column online HERE. Read More

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