Patient Group Letter on Gene Therapy
The APBDRF has joined with EveryLife Foundation, along with Parent Project Muscular Dystrophy, the National MPS Society, and the MLD Foundation, in offering support for existing Hill proposals that include provisions for best price/AMP exemptions. In recent months, there has been significant Hill activity to create legislative fixes to existing barriers in implementing innovative payment solutions for the emerging transformative and gene therapies. While many coalition groups have been active on these issues, they have not included strong patient community voices and thus the Hill has asked that the patient advocacy community specifically weigh in on the need for legislation to allow for outcomes-based agreements for innovative and gene therapy. This proposed legislative solution establishes a flexible framework that would enable biopharmaceutical companies and payers to develop customized approaches that ultimately foster patient access.
May 16th, 2017
Action ALERT: Urge your Legislator to Support the OPEN ACT!
Dear Rare Disease Advocate,
As you may already know, we are working to pass legislation called the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures & Treatments) that could help double the number of affordable treatments available to rare disease patients.
As rare disease advocates, we need to make sure Congress gets our message loud and clear.
This bipartisan bill is supported by over 220 patient organizations and could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases. We need your help to make this bill a law.
Please share this email with your networks and you can also link to our alert on Facebook or Twitter. We appreciate all that you do for rare patients!
Thank you all for taking action to advance this life-saving legislation.
The EveryLife Team
March 1st, 2017
NORD Issues Statement on President Trump’s Address to Congress
Washington, D.C., March 1, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement in response to President Trump’s first speech to Congress:
“Yesterday evening, President Trump recognized Rare Disease Day and the 30 million Americans living with a rare disease in his first address to Congress. We are grateful for his recognition of the day that raises awareness for all individuals with rare diseases and their families. We are elated that he is joining us in our efforts.
President Trump also recognized Megan Crowley, and her father John, as shining examples of the untiring, steadfast commitment individuals with rare diseases and their families bring to finding treatments and cures for their disease. The Crowleys are one of many superstar families that deserve recognition, and we are thrilled that their tireless work was recognized on such a large stage.
The President continued, stating, ‘…our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need. If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan.’
We agree that FDA review processes can be improved upon to expedite the development and review of orphan drugs. Yet we disagree with the President that restraints must be slashed, or that the approval process at the FDA is preventing advances from reaching those in need.
Between 2008 and 2013, 87 percent of the 113 rare disease treatments reviewed by the FDA received an expedited review, compared to 35 percent of treatments for common diseases. Seventy-eight percent of rare disease treatments were approved using one or more flexible development approaches (generally defined as an approach that does not include two adequate and well-controlled trials or uses novel endpoints).
For patients with immediately life-threatening illnesses who cannot participate in clinical trials, the FDA approves 99.5 percent of all expanded access requests submitted by physicians and companies.
We also believe that the current safety and effectiveness standards for drugs and biologics are crucial to ensuring individuals with rare diseases receive therapies that will positively impact their lives. Our patients deserve the same quality therapies as everyone else, and to weaken the standards will only threaten our population with unsafe, ineffective therapies.
The FDA already shows an incredible amount of flexibility in reviewing and regulating orphan drugs. But we can still improve the process.
First, we can adequately fund the FDA to allow them to hire and retain the experts needed to quickly and thoroughly review orphan drugs. Without experts on staff, the review of orphan therapies, and consequently the delivery of orphan therapies to the patient population, may be slowed. The Center for Drug Evaluation and Research (CDER) has hundreds of unfilled positions, and we support a paradigm in which the FDA can freely hire and retain the expert reviewers our patients deserve.
Second, we can reauthorize the critical user fee agreements that fund a substantial portion of FDA’s budget. These user fees must be reauthorized this year.
Finally, we can enact the critical reforms included in the Prescription Drug User Fee Act (PDUFA) and Medical Device User Fee Act (MDUFA) commitments. For orphan drug review, we can greater integrate the patient perspective into drug development review through the further use of patient preference information and patient-reported outcomes. We can also ensure the unique rare disease experience is reflected in orphan drug review through the integration of the FDA Rare Disease Program into orphan drug reviews. Both of these key reforms are under consideration in Congress to be included in the drug user fee act reauthorization.
We at NORD again wish to emphasize our appreciation of the attention President Trump has brought to the rare disease community. Moving forward it is our hope to partner with President Trump and his administration in supporting the needs of our community, such as with the passage of these key user fee agreements.”