Discussion Draft on Compassionate Drug Use for Disease Such as SMA Released

A 21st Century Cures discussion draft has been released by the U.S. House of Representatives Energy and Commerce Committee as a basis for discussing new methods of accelerating discovery, development and delivery of novel therapies and cures. The initiative was established to investigate different ways the government can support these advancements.

The newly revised and expanded document was announced by the head of the initiative, Rep. Fred Upton, who is also the chair of the Energy and Commerce Committee. Upton has stated that the spinal muscular atrophy (SMA) community was his inspiration for the work he has been developing, in particular the sisters Brooke and Brielle Kennedy, who live in his home state of Michigan and both suffer from SMA type II.

“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, according to a press release from Cure SMA. “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.”

The 21st Century Cures discussion draft includes recommendation on the risks and benefits to expand access to novel treatments, which is a matter of concern for SMA patients. In order to expand access to investigational drugs, it would be necessary a structural framework established by the U.S. Food and Drug Administration (FDA), enabling the inclusion of patient experience data in the process of drug approval.

If this was made possible, SMA patients could play a larger role in determining the benefits of a therapy, as well as the level of risk that is considered acceptable. In addition, transparency from healthcare companies is crucial for expanding access and compassionate use, which would enable other patients — in addition to the ones participating in clinical trials — to be treated with experimental drugs.

Currently, expanded access applies to drugs that are actively in study or that have been already studied, as well as drugs that have been shown effective and that do not offer unreasonable risks compared to the severity of the medical condition. Expanded access to investigational drugs may, however, delay its approval for the treatment of all patients.

The Energy and Commerce Committee has announced they would continue working in collaboration with patients, innovators, providers, consumers and regulators to reach a more accurate document that can help improve the treatment of chronic, debilitating or life-threatening diseases, such as SMA. The document is expected to be converted into a formal legislation later this year, which will be voted on by the House of Representatives and the Senate.