Support for #CuresNow Continues to Sweep the Nation
Everywhere you look, support continues to build for 21st Century Cures.
Louisville’s Courier-Journal: “This bill could be a true game-changer for patients.”
Utica’s Observer-Dispatch: “One priority should be to enact the 21st Century Cures Act.”
IPPF: “In short, this bill could be a game-changer for patients with both rare and common diseases.”
Every community around us has a connection to disease. From the rarest diseases to those that are more common, there is a unifying thread that brings hope to families and their loved ones.
21st Century Cures will accelerate the critical cycle of discovering, developing, and delivering new cures and treatments. By boosting precious research dollars and streamlining approval of these treatments, the nonpartisan bill presents an opportunity for unprecedented medical developments.
From leaders in the research community to the nation’s leading cancer advocates, momentum continues to build for 21st Century Cures. From Kentucky, New York, and across the country, patient’s can’t wait another year for #CuresNow.
Innovative medicine must be priority
By Mark Dant, Louisville, KY
There’s been outcry over the cost of prescription drugs, but little public discussion about the need for innovation for patients who don’t yet have an effective therapy. One in 10 Americans suffer from a rare disease, but only 5 percent of rare diseases have a treatment approved by the FDA.
My son Ryan was diagnosed with MPS I when he was three, and we were told that he would not live through the decade. Now a senior at the University of Louisville, Ryan would have died many years ago had it not been for innovative science and industry investment that spawned a new drug that saved his life.
The 21st Century Cures Act would provide new funding for research at the National Institutes of Health as well as funding to enhance the review of new medicines by FDA. The legislation includes crucial incentives such as the OPEN ACT, which would encourage companies to repurpose existing medicines for rare diseases, potentially bringing hundreds of new treatments to patients more quickly than traditional drug development.
This bill could be a true game changer for patients. …
Click HERE to read the letter to the editor online.
EDITORIAL: Government must get priorities straight
One priority should be to enact the 21st Century Cures Act. The bill, which was approved in the House of Representatives in July 2015, would provide more funding for medical research, provide incentives for the development of rare disease treatments and expedite the federal Food and Drug Administration approval process for certain treatments. The Senate has yet to vote on similar legislation.
Linda and Kirk Hinman of Lee have, in very short time, become tireless advocates for the bill. Their grandson, Spencer, was diagnosed in August with Sanfilippo syndrome (also known as MPS III), a metabolic disorder that will, short of a medical breakthrough, kill him, most likely in his teens. Spencer is 2. More research into the rare disease could possibly save him. …
Yet the Senate can’t get its act together on appropriating funds that could save childrens’ lives?
Sen. Charles Schumer, D-N.Y., says he’s on board to approve the 21st Century Cures Act. Tell him and Sen. Kirsten Gillibrand to get this done. Now. …
Click HERE to read the full column online.
Rare Disease Patients Need 21st Century Cures Now
By Mark Yale, Interim Executive Director at IPPF
Although there has been a huge public outcry over the cost of prescription drugs, there has been little public discussion about the need for innovation for patients who don’t yet have an effective therapy. It is estimated that 1 in 10 Americans suffer from a rare disease, and only 5% of rare diseases have a treatment approved by the Food and Drug Administration (FDA). …
I was diagnosed in 2007 with cicatricial pemphigoid, a rare autoimmune blistering skin disease. Like others with a rare disease, I experienced delays in diagnosis and difficulty finding knowledgeable physicians. Eventually, I lost vision in one eye from the disease. The pain associated with my disease was severe, and the list of complications extensive. Although my disease can be treated, there are still no FDA approved treatments and no cure. No disease should be too rare for a cure.
The good news is that Congress can do something to help. The 21st Century Cures Act, which passed the House of Representatives with broad bipartisan support (a rarity in the current political environment), could provide billions in new funding for research at the National Institutes of Health, the nation’s premier biomedical research institution, along with needed funding to enhance the review of new medicines by FDA. …This could potentially bring hundreds of new treatments to patients more quickly than traditional drug development. In short, this bill could be a game-changer for patients with both rare and common diseases. …
Click HERE to read the full column online.