The nonprofit, Huntington’s Disease Society of America (HDSA) continues to regularly update information on research and advocacy. Consider signing up for the HDSA newsletter. It contains a wealth of information. http://hdsa.org.
Perhaps you are wondering about Right-to-Try legislation for Huntington’s disease patients who are end stage. HDSA provides a summary of the topic.
On its face, the idea behind Right-to-Try legislation is to allow families access to medications and treatments still in the clinical trial phase when an individual has no other treatment options left and is facing a terminal illness. Right-to-Try legislation focuses on cutting red tape in the Food and Drug Administration (FDA) to allow easier access to these experimental treatments. But, in reality, the FDA isn’t the issue. Access to investigational therapies is already permitted through the Food and Drug Administration’s expanded access program. FDA currently approves 99.7% of all expanded access requests submitted by physicians and companies for patients with immediately life-threatening illnesses who cannot participate in clinical trials. Removing FDA from the process of obtaining investigational drugs, as Right-to-Try proposes, increases the risk of patient harm and creates confusion. When access to an experimental drug or treatment is denied, it’s almost always because the request has been denied by the pharmaceutical company, not the FDA.”
At this time all Expanded Access and Right-To-Try requests to Roche will be denied. This was discussed in the recent HD Community-wide letter that was released by Roche in June. Specifically, they said “We understand that families may wish to seek access to investigational medicines as soon as possible. However, access to RG6042 can only be through clinical trial participation at this time. Because the benefits and risks of RG6042 are not fully understood, we are not able to grant pre-approval, compassionate use or “right-to-try” requests. The company Roche/Genentech
100% of the proceeds from the nonfiction book, Watching Their Dance, is being donated to the nonprofit, HDSA. https://theresecrutchermarin.com/purchase-book/