Lots for u. S. News and report about a year ago. At the time only five states had passed right to try laos. Now it it has climbed to 24 states. Pretty quickly, and one year one of the senators, who supports this bill said it was the first piece of legislator he had come across those both prolife and prochoice. Lets just talk about what right to trial laws are. What right to try laws are is all about when your life hangs in the balance, when you have a terminal illness it is without giving you the right to try to fight to save your life by using experimental medicines while the understudy with the fda but before they receive that these final green light. The reason he calls that prolife and prochoice is because you have a choice as to whether or not you want to try some of these investigational medicines. It is prolife because instead of right to die laws we have not only the option to hasten your death but actually fight to live and prolong your life. In your book you talk about the Goldwater Institute got involved in this cause. You immediately dive into how it affected you and your personal life when he heard about this issue. Im going to read a passage quickly from your book. You write about your uncle kenny. He said he died when i was about four years old form of cancer for which there are now multiple treatments with very high curates. He is my fathers only brother and i remember my dad saying when i was growing up that uncle kenny had died just months before new treatment was approved. At that moment he hit me, if kenny had been allowed to try the treatment earlier, my father might still have his brother and my cousins still have their father. As we mentioned earlier this legislation has passed quickly and unanimously do you think having a story like that and a story you tell here, theres a lot of legislators who can immediately identify their own family or friends lives and since is where this has happened. Weve all had people in our lives like my father, michael kenny. Weve all known people have suffered from terminal illnesses. Many of us have known people where cures came onto market the best treatment came on shortly after they passed. This really hits home for a lot of people. One legislator told me, this is a twominute decision, the first one is for copy. This is just something that is common sense to people, of course you should have the right to fight for life. Even if something hasnt been tested and 50 nickel trials, if its safe for human Consumption Habits along that the moment process and the only alternative is that youre going to die, you should have the right to try those investigational treatments as long as you know the risk you are taking. At the beginning of your book you read about a mom with two sons both with muscular dystrophy which is a fatal disease that attacks the muscles. One of the children was able to access experimental drugs but the other was not. Can you tell us that story and go into detail about what happened . And the right to try our talk about jen who is the mom and i first read about jen story in the Burlington Free press in vermont. Both of her, two of her sons two of her sons has muscular dystrophy which will basically cripple the boys and most boys who have this will not live past their 20th birthday. One of her sons was able to get into a Clinical Trial and he was doing extraordinarily well. He he went from being in a wheelchair parttime to being able to play soccer and be on the playground at school. Really incredible progress, never before seen with boys with this disease. His his brother did not make it into the Clinical Trial. He did not qualify so he had to watch as his brother went on to the soccer field and he was deteriorating. He got to the point where he could not a cup of water to his lips. This is a situation today in the United States. If youre lucky enough to get into a Clinical Trial many patients will find improvements. The challenges it is about a 15 year process for that particular treatment to make it to market. In the the meantime, if you dont get into the Clinical Trial, you die. So the right to try is about saying if its good enough for people in Clinical Trial, its its good enough for all of the people who need it at this time. They should be able to make that choice was long as they know their risks and some things that are not quite proven. When he first got involved with this cause with the Goldwater Institute you decided to go after it at a state level targeting state lawmakers. Why did you you do that instead of going after congress. The fda is a federal agency. It would be wonderful to see washington reform this and they have the power to do it, in fact the fda already has rules and regulations that enable it to help more people than it chooses to do. We know that its been 25 years since the fda has made any major reforms to really affect large numbers of people with these terminal diseases. So we decided to go to the state legislatures of course, as you mentioned in the beginning of the program 24 states have adopted this law in just over a year. It has moved with unanimous support on a voting margin of 99 to 1. It is a left right and everyone in between type of issue. And going after this issue, why did you focus on people who are terminally ill . What about someone who is losing their site what about someone with multiples laroche sclerosis. We designed this law so it will prevail in the u. S. Supreme court, should the fda try to challenge. We know previous cases thats been important to the courts is a safety question. Focusing on people with terminal illnesses where their lives really to hang in the balance, where they they really have no other option, we not talking about rushing medicines for teenagers with pimples. Her cough medicine. This is for boys with dmd, people with lou gehrigs disease who may have two years to live when they get their diagnosis. As jen mcnary said to me once, by the time the fda finally approved this particular treatment, we will have lost an entire generation of boys to this disease. That is unacceptable. We move this to the states because we knew we could get the action that patients need. The fda is responsible for overseeing whether drugs are safe and effective. I reached out to them about this issue, they point to the fact that they have trials available for which people can enroll to try these different treatments. They also have something called compassionate use in which you can apply for access, if you are terminally ill and outside of these Clinical Trials. Can you give me more detail about this and explain why passionate use is not enough to ensure that people can get access to lifesaving treatment westmark. Thats right, the fda fda does have a few programs, clinical treatment programs and it is supposed to help patients in situations like this. The fact is, less than 1 of patients with terminal illness will ever get access to these treatments through the Compassionate Use Program. The program was never designed to help hundreds of thousands of people let alone the million or so who will be given terminal diagnoses this year. Under the fda rules, compassion is an exception to the rule. What we are fighting for is to make compassion the rule. How does a hope rests us work for compassionate use and how how long does the application . The fact that 99 of of the people never make it through tells you if its working or not. Cancer patients a must have full try to get in and only 3 will even get in. We know Patients Want access to treatments and we know they are not getting them. The reason there not getting them are many. One is some people dont know about the program. Another one is the process is so incredibly timeconsuming that most physicians and doctors simply cannot navigate the system or have the time. By the fdas own admission, just the application paperwork requires over 100 hours of a physicians time. Its not something he can give to his assistant or intern to fill out. So doctors who do this basically clear their desk for two and half weeks just to do paperwork for one patient. You can see how if you have hundreds of patients, which, which positions do, this is not a good solution. Of course their tremendous delays, and the right to try to tell a heartbreaking story about a girl named andrea who is in her thirties, she had Ovarian Cancer and she fought it hard for seven years, she had she had exhausted all of the conventional treatments. Her dr. Was with the Anderson Center and said there is something that can help you, we we have to go to the compassionate use process. Said he had never seen a process used in more than ten years. Its sort of this mythical unicorn but he was an incredible physician, he went to the process, he did all of the paperwork and it still took the fda 24 days to turn around their decision. They say they turn it around on the spot, the fact is, they dont. By the time she got the treatment it was too late, even though it had started to reduce the tumors and their beginning to see shrinkage, she had had we can so much that she passed on. In fact, in texas her friends and family fought hard for the right to try law, it was too late for andrea but we are very grateful that will be in time for other patients needed. The fda also told me that they are working on shortening the forms, as you said it took 24 days i believe for the turnaround in that case. They say they have up to 30 days, d days, do you think shortening the form will help this a little bit along as we wait for more right to try lost a pass . I mean its fine but is really windowdressing. From a position of principal, you should not have to beg the federal government for permission to try to save your life. The person in the situation who has your best interest at heart, knows your case inside out, the first person is you, the second person is your physician. Frankly, there is no role for the fda in administering compassionate use. Its its really not what the agency was designed to do. Nor is it something the agency truly desires to do. When i was conducting interviews for this book, we interviewed the head of the center for new Drug Development at the fda. We asked the theoretical question of, would you, in theory like to see tens of thousands of patients accessing compassionate use, given the chance to try some of these promising cuttingedge treatment . She sighed and she said, well it would be another burden on the health care system. That attitude is quite prevalent throughout the bureaucracy. Now not saying everyone there has that attitude, theres incredible people fighting for patients there. As an agencys job job is not to treat patients. Its job is to approve medicine and products for the u. S. Market. It is for physicians to decide whether something is needed to save your life. So, we would like to see the fda not even have a role in this particular aspect of compassionate use. It really should be between the physicians and patients, in fact that is how the system works in europe. You talk about how you have approach state legislators in particular to pass these law, in most cases they have passed almost unanimously. And if you cases where they have not voted in favor of these laws, what have been some of the reasons they have given her some things you have heard where they may have hesitation about passing the laws . Is passed on a 9099one margin. People are concerned about safety, i think thats dense from a misunderstanding about what the right to try is. This is about equity. This is extending to all patients with terminal illness, the same treatments that are already available in clinical studies. And the fda has said its already okay to test for scientific purposes. The safety issue really stems from misunderstanding. We have had some hospice organizations oppose this because if you are fighting for your life at the end, often hospice cannot treat you or the insurance for hospice will not cover you. So their concern is that theyre probably going to die anyway even if they try this, we want to make sure they get that quality treatment. We consider those logistical issues, they should not be hurdled from trying to save your life. Those. Those things can be worked out. In europe where they have widespread compassionate use, Insurance Companies do help cover these treatments. A lot of these things have been thought through before. The few voices of opposition in the United States i feel come from a misunderstanding of what the right to try is, or quest for absolute perfection in a system with terminal patients, they dont have the luxury of waiting for statistical perfection. I know you are approaching this from a state measure, wondering if you have heard anything from president ial candidates or if youve been able to meet with any of them to discuss right to try laws . We have not taken us to the president ial candidates, frankly, frankly we been so busy with the states moving us along that working at the federal level is an afterthought. It takes so long to get anything as far as reform through congress that we really felt like time was of the essence. We need to move quickly through the states. Once the issue was brought to our attention, in fact how this issue became an issue to the Goldwater Institute was that the Cancer Treatment centers for america learned about some of our other work, they came to us and said we have Cancer Patients that we want to treat, we know for some of them the most effective treatments out there are pending approval at the fda or are in europe or other places of the world, is there anything you can do, can you think of any way to help us able to improve our patients access to these treatments. We are being stymied by these rules that make you wait 15 years for a drug to come to market. Is there anything you can do . Time is of the absence so we have really focused on the state legislators were people are closer to their constituents and in every state where this is passed we have people who are testifying with all sorts of diseases. Lou gehrigs, cancer, some muscular dystrophy types and some really trying illnesses. Policymakers feel that they see those folks and as we mentioned earlier almost all of us have had an experience like this in our immediate families, certainly in our friendship groups. No matter what your politics are, your heart goes out to people, we would all like to see people access treatments if they can possibly be available. You just talked about the availability of some drugs in europe, a chapter in your book you share stories about patients who travel across the world to access drugs that are still being developed and are showing a lot of promise, can you tell her viewers about those stories . Sure many of the drugs of europe have been the standard of care there for ten or 20 years. They been fully developed and for whatever reason have not gone through the fda process. One story, boy name diego was 11, he lived in arizona. He was diagnosed with osteosarcoma, type of bone cancer in his leg. He was an athlete and he noticed the pain, they went in and got a diagnosis. The dr. Said that, the best available treatment for you is not available in the United States. Youre going to to need to go overseas. So he and his family moved to england for almost one year in order to get the treatment that saved his life. That drug is called knee pack. It has been the standard of care in europe for some time and another two dozen countries, when they tried to get that same drug approved in the United States, even after it was the standard of care and all of the test showing it had come out all over the world, the fda demanded that the company test on 900 people in its Clinical Trial. Well 900 patients, thats more patients than will be diagnosed with this disease in a year for this particular type. So sometimes the fda, theyre just not willing to look at avatar evidence, they come they come up with things that are insurmountable barriers of trying to get these things approved. So here you have something that is standard of care, it is lifesaving, lifesaving, the only people in the United States can access it are people who can afford to go abroad. Think we all know patients who can travel abroad for treatments are few and far between. Some of the criticisms of right to try save these oneoff cases get in the way of collecting data. Some part pharmaceutical Companies Say that whatever adverse effects occur and people have to be reported in the fda. As you mention Clinical Trials are very controlled in terms of who gets chosen to appear in these Clinical Trials. What are some of your responses to concerns like that . All of these things have been worked out in europe. I would say we do not have to recreate the wheel. We can take the lessons that have been learned and apply them here. One of the things is positions and patients, when they get these investigation treatments they want to be contributing to science. They want their information recorded. People have talked about open ended Clinical Trials where you let more people in with more conditions, is more reflective of the real world. Theres talk in Clinical Trials where theyre looking for athletes with tumors, and other words they want to focus on just that tumor treatment but the reality is, most patients have multiple conditions going on, and, and their excluded from Clinical Trials. With Something Like the right to try when have a bigger sample of people and you record that information, you can end up with better scientific information that will better serve patients in the long run. Those are some of the things we are talking about. With companies, to the the second point you raise, Many Companies are concerned that if you treat patients who are to the end, which by definition under the right to try laws these are folks that have terminal conditions, they have exhausted all of the standard treatment, so they are near the end unless they can get something that wil