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.
[i] 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).
[ii]
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. ..."
As health care reform debates continue in the Senate, MDA remains committed to advocating for principles of coverage
set out in conjunction with leading national patient organizations that
establish key elements necessary for adequate health care coverage.
The principles require that meaningful access to care be affordable,
accessible, adequate and understandable. In addition to these elements,
maintaining a robust Medicaid program is essential
to ensuring our community has access to the care it needs. In May the
U.S. House of Representatives passed its version of health care reform,
and now that the U.S. Senate is working on its bill, we continue to work
with the Senate and encourage all members of Congress to engage in a
bipartisan effort to find solutions to the challenges with access to
care and with maintaining the critical protections set out in the
Medicaid program. Thank you to all advocates who reached out to your
members of Congress earlier this spring to advocate for the principles
of coverage and Medicaid protections. As the Senate considers its bill
this week, we urge you to again ask your senators to keep the priciples
of coverage in mind when moving their bill forward, and to highlight the
importance of Medicaid coverage for the neuromuscular disease
community.
