NORD Issues Statement on President Trump’s Address to Congress

The process for expediting certain drugs has improved dramatically over the last decade. Yet, we need to keep the pressure on our representatives - lest they forget. There is still a lot of work to be done in finding a treatment for Kennedy's Disease.

NORD Issues Statement on President Trump’s Address to Congress

Posted by Jennifer Huron

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. ..."

To read the rest of the statement follow this link: NORD Statement