The Committee on Energy and Commerce released a discussion document yesterday, marking the launch of the legislative phase of the bipartisan 21st Century Cures initiative (#Cures2015). Children's Cause is carefully reviewing and analyzing the draft from the perspective of children with cancer and their families. Of the numerous proposals outlined, here are some areas of particular interest:
- Patient-Focused Drug Development: This provision would require the FDA to establish a framework for incorporating patient experience data into the regulatory decision-making process. We are particularly interested in the risk-benefit assessment process as it relates to testing new drugs in children with cancer. (Related: White House launches Precision Medicine Initiative)
- Compassionate Use: Led by Texas Representatives Michael McCaul and Michael Burgess, MD, this section would require drug companies to make transparent how they handle compassionate use and expanded access requests from individual patients. This issue gained particular attention in the news media and childhood cancer community last year, led by the #SaveJosh viral campaign.
- Cures Acceleration Network: This provision would grant additional funding and flexibility to the National Center for Advancing Translational Science (NCATS) and its Cures Acceleration Network funding mechanism. Children's Cause has been a long-time advocate of the Cures Acceleration Network and its potential to fund new therapies for childhood cancers.
There are many other provisions we'll be monitoring and weighing in on as the draft legislation evolves into a final bill, including issues relating to pediatric clinical trials, incentives for unmet medical needs for rare diseases (based on the MODDERN Cures Act), programs to foster emerging scientific talent, changes to Medicaid and CHIP, and more.
According to the Committee's newest white paper, the outlined draft provisions are "neither perfect nor complete," with more feedback and fine-tuning required. We'll be communicating with Committee staff and submitting comments during this stage of the process. As we engage around this ongoing legislative effort, we are optimistic that the final bill will contain thoughtful provisions that align with our mission of achieving access to less toxic and more effective pediatric cancer therapies and expanding resources for research and specialized care.