The US House of Representatives Energy and Commerce Committee has released a new 21st Century Cures discussion draft. This is a revised and expanded version of the first draft that was released in January.
The 21st Century Cures initiative was created to investigate how the government can help “accelerate the discovery, development, and delivery of promising new treatments and cures for patients.”
This initiative is led by Rep. Fred Upton, who also chairs the Energy and Commerce Committee. At a hearing to discuss the release of this latest draft, Chairman Upton cited the SMA community—including sisters Brooke and Brielle Kennedy, from his home state of Michigan, who have SMA type II—as part of the inspiration for his support of 21st Century Cures. Chairman Upton was one of the congressional guests at our 2014 Hope on the Hill event.
“We started this journey because all of us know patients and families who are desperate for hope. We’ve also seen and read about the incredible advances made in science and technology,” said Chairman Upton. “But it has become increasingly clear in recent years that our regulatory policies have not kept pace with innovation and there is much more we can be doing to provide that hope to folks.”
This latest version includes provisions on risk/benefit analysis and on expanded access, two topics of particular interest to the SMA community. The risk/benefit provision would require the FDA to establish a structured framework for using “patient experience data” in making drug approval decisions. This means that patients would have greater input in determining what would be a meaningful benefit of treatment, and what would be an acceptable level of risk.
The expanded access provision would require transparency from companies in making expanded access determinations, and would require the FDA to create guidelines on how data from expanded access should be interpreted. Expanded access, also called “compassionate use,” occurs when individuals who are not part of a clinical trial are given access to an experimental drug or treatment. Generally, expanded access can only be offered if:
- The drug is being actively studied, or all studies have been completed.
- There is evidence that the drug may be effective.
- The drug does not expose patients to unreasonable risks given the severity of the disease.
In addition, granting expanded access to a single patient or group of patients might delay approval of the drug for the wider patient community. Families, medical professionals, researchers, and regulatory groups will continue to work together to navigate this difficult topic. However, 21st Century Cures would create the framework for that navigation.
Members of the Energy and Commerce Committee will continue to meet with patients, innovators, researchers, providers, consumers, and regulators to refine this document.
Later this year, the discussion document will be converted into formal legislation to be voted on by the full House of Representatives and then by the Senate. Sign up for our email list in the box below to receive ongoing updates on our engagement with Congress, the FDA, and other regulatory bodies, and to be notified on critical next steps for our community once this legislation is presented.