Amy Judge DeLong is manager of Federal Government Relations at Boston Children’s Hospital.
In the midst of a seismic shift in Presidential administrations and anticipation of the incoming Congress, a landmark medical research bill with several provisions important to children cleared the lame duck session of Congress. The 21st Century Cures Act (Cures) is the end result of nearly three years of bipartisan Congressional activity. Last week, it was signed into law.
Cures includes scores of provisions aimed at strengthening National Institutes of Health funding for medical research and accelerating review efforts at the Food and Drug Administration. The law cleared Congress with overwhelming majorities, an example of bipartisanship that may be challenged in the months ahead.
While Cures enjoyed tremendous support from Republicans and Democrats, there were some detractors in each party. Republican critics claimed it was inappropriate spending. Democrats detractors cited the bill’s lack of mandatory funding, meaning Congress will have to decide each year whether and how much to fund Cures.
The Cures Act and children
Together with our peer institutions, Boston Children’s Hospital took a close look to see what was in Cures for children. Our conclusion was that the positives, particularly provisions focused on child health research, far outweigh the negatives. We worked with legislators to encourage its passage into law.
Cures authorizes $4.8 billion in new funding over the next ten years for a set of priorities at the NIH, including many championed by the outgoing Obama administration. It authorizes $1.4 billion for the Precision Medicine Initiative, which aims to tailor treatments to people based on their genes and lifestyles; $1.6 billion for a new brain research initiative, which seeks to advance scientists’ understanding of the human brain; and $1.8 billion for the Vice President’s Cancer “Moonshot” initiative, which aims to propel cancer research forward. We expect all of these research initiatives will benefit our patients.
Another provision Boston Children’s strongly supports is aimed at driving forward the NIH’s implementation of the National Pediatric Research Network Act (NPRNA). The NPRNA became law in November 2013, but it has yet to be meaningfully implemented at the NIH. We were pleased that Cures included language specifically directing the NIH to implement the NPRNA. We expect this will speed the development of a new network of consortia focused on pediatric research. Boston Children’s and our peers look forward to continuing to engage Congress and NIH leadership on this important issue.
Rare disease provisions in the Cures Act
Two further provisions address the dire need for therapeutic development for rare pediatric diseases. Rare disease research is key to medical discoveries for everyone, while helping children and their families face profound medical challenges. The Act calls on the NIH to support a global pediatric clinical trials network. It also extends an incentive known as the Pediatric Rare Disease Priority Review Voucher to encourage companies to develop therapies for these populations.
Additionally, Cures contains language that would develop policies and recommendations supporting early career scientists, vital for advancing medical discovery. It also gives greater attention at the FDA to the view of the patient in therapy development, a topic known as Patient Focused Drug Development (PFDD).
Opioid, mental health provisions
Beyond medical research and innovation policy, Congress included an opioid response in the final Cures package. This was of interest locally, as Massachusetts faces a growing epidemic of opioid addiction and overdose deaths. The law authorizes $1 billion over two years in grants to support state response efforts, giving priority to states with a higher incidence of abuse. The funds can be used to train healthcare practitioners and advance best practices in prescriptions, referrals to treatment and overdose prevention.
Cures also includes a significant package of provisions to strengthen federal support for mental health care. These include several policies of particular interest to Boston Children’s. For example, the final Cures package calls for the creation of mental health access grants to promote the integration of behavioral health into pediatric primary care.
Finally, just before adjourning on December 9, Congress passed a separate funding measure that included full funding of Cures for the first year: a robust $872 million, including the $500 million for states to curb opioid abuse.
Boston Children’s is very pleased by the commitment Congress showed in passing Cures. We look forward to working with the executive branch and with Congress to ensure future funding and to implement many of these policies in the months ahead.