Today I moderated this @theA4LI panel featuring several biotechs who are already using qualified state therapeutics access regimes such as Florida's Stem Cell Access law to bring treatment to patients, and others who are launching under Montana's Right to Try law shortly. We were also joined by @cole4nh and @KesselringSteve from the NH government, to discuss the future of their state's Right to Try programs. These state level pathways are critical to provide scalable patient access. Federal Right to Try and Expanded Access are important, but no single system is perfect, and inevitably patients fall through the cracks. Now, the states are there to step up and catch them when they do. If you're a biotech wanting to learn more about coming to Montana starting this July, my DMs are open!

My name is Stephen Martin. I am trying to get access to a nasal spray for this three year old boy, Brody DeVault. He has Creatine Transporter Deficiency. The creatine in his bloodstream does not make its way to his brain. Without treatment, he will live a life of intellectual disability, and probably die young from a seizure. There is a treatment which has finished Phase 1 clinical trials which may help him. I have spoken to the manufacturer, they will provide it to him under Montana's new law SB535. But only if we can get them official promises from HHS that they will not be punished for treating a patient under a state law. I have a meeting with the FDA on the 18th. Right now the single thing which most increases the likelihood of getting Brody treatment, is making sure that President Trump becomes aware of this before that meeting. I'm posting this in the hopes that I can bring this to the attention of people who can, in turn, bring this to the attention of President Trump. The meeting is in 6 days. Thank you for your attention to this matter!

Later this month I will join a number of patients and biotechs who will be meeting with @US_FDA to discuss the importance of the department codifying its stance towards state Right to Try, Right to Personalized Treatment, and stem cell laws. Since 2016 when @SenRonJohnson first asked HHS and FDA to clarify their enforcement stance towards state programs, and was rebuffed, there has never been an official stance on this issue clarified. Yet, in the 10 years since, HHS and FDA have never actually escalated to enforcement against a doctor or biotech using a state Right to Try program. In one high profile example, they actually granted a drug priority breakthrough status after it was used successfully on 75 patients under Texas Right to Try. To some extent they have clearly been exercising enforcement discretion, which is the right thing for them to do! It's not a good use of their resources to go after companies sending medicine to patients for use under a state law. But, this position being "unwritten" is actively blocking patients from receiving care. And it's time for their position on the matter, whatever it may be, to be made official. These programs represent a chance to catch patients who fall through the cracks in the Federal patient access system. And the fact more and more states continue to pass new laws even today demonstrates a clear mandate from the American people to allow the states to step up to the plate, and get their patients medicine.