Reauthorize the Creating Hope Act to help kids with rare diseases (The Washington Post)

LETTER TO THE EDITOR

Regarding Theresa Vargas’s Sept. 20 Metro column, “New hope for kids with rare tumors”:

It takes enormous courage for a child to live with a rare disease, and tremendous strength to be a parent of a child with a disease that has no cure. Families of children with cancer, genetic conditions and other rare diseases wake up each morning hoping for a scientific breakthrough. Especially now, when science is under attack, any new treatment should be celebrated.

The inspirational story of 10-year-old Autumn Schierling’s fight with neurofibromatosis and the successful development of selumetinib for her condition offer a timely example of how a law can help children with rare diseases. The Creating Hope Act, passed in 2012, encourages companies to develop drugs for children such as Autumn by awarding a priority review voucher when a new drug for a rare pediatric disease is approved. Selumetinib and 23 drugs for cancer, Duchenne muscular dystrophy, cystic fibrosis and other rare diseases have been approved and earned vouchers under the Creating Hope Act. The industry’s interest in creating drugs for rare pediatric diseases, almost nonexistent eight years ago, has been growing exponentially — more than 65 drugs for rare diseases that affect children are in the pipeline now.

But the Creating Hope Act is expiring at the end of September. Permanent reauthorization of this law is needed so more children can wake up with hope.

Elena Gerasimov, Washington

The writer is programs director for Kids v Cancer.

The Washington Post, September 25, 2020