WEBVTT - Right to Try, Right to Fail 0:00:00.120 --> 0:00:03.920 I know it's probably too late for me. I made 0:00:03.920 --> 0:00:08.200 my piece with that I need to know before I 0:00:08.280 --> 0:00:14.560 die that my children find themselves in this unenviable position. 0:00:15.240 --> 0:00:20.959 This nation that I probably served will respect their liberties 0:00:21.400 --> 0:00:24.919 and the right to make their own decision about their 0:00:24.960 --> 0:00:35.400 medical treatments. Thank you for having me. Matt Billina is 0:00:35.400 --> 0:00:37.680 a father of three boys and a former U. S 0:00:37.800 --> 0:00:42.800 Naval aviator in he testified at a congressional hearing to 0:00:42.880 --> 0:00:46.440 advocate for dying patients who want access to experimental drugs. 0:00:47.280 --> 0:00:49.440 At that point, his speech was slightly slurred by his 0:00:49.520 --> 0:00:54.080 Luke Garrig's disease. It's a fatal condition with no treatment, 0:00:54.760 --> 0:00:58.279 it destroys the nerve cells and the muscles they control. Slowly, 0:00:58.560 --> 0:01:03.800 every part of the body stops working. Letting patients like 0:01:03.920 --> 0:01:08.120 Matt who have no other options use these experimental medicines 0:01:08.680 --> 0:01:12.720 seems like it should be easy, but it's not that straightforward, 0:01:13.800 --> 0:01:17.120 and the critics, the people who oppose making them widely available, 0:01:17.600 --> 0:01:23.760 aren't always who you would expect. Welcome to Prognosis, a 0:01:23.800 --> 0:01:27.600 podcast about innovation in medicine and science. I'm your host, 0:01:27.680 --> 0:01:30.600 Michelle fay Cortes, and today we're going to explore what 0:01:30.640 --> 0:01:33.240 people do when they're desperate for a chance to try 0:01:33.240 --> 0:01:40.920 an unapproved drug. Do you want to say something? Yeah? Good? 0:01:41.600 --> 0:01:48.880 Do you want to say yes? All right? Laura McGlenn 0:01:48.880 --> 0:01:52.360 is another American who wants access, not for herself but 0:01:52.480 --> 0:01:55.440 for her son, Jordan's. You just heard him in a 0:01:55.480 --> 0:01:58.520 clip from a few years ago, asking lawmakers to make 0:01:58.560 --> 0:02:02.760 it easier he has to shend Muscular dystrophee, a genetic 0:02:02.800 --> 0:02:05.840 disease that erodes the growing muscles of young boys and 0:02:05.880 --> 0:02:09.519 eventually hits the heart and lungs. The average life expectancy 0:02:09.720 --> 0:02:13.880 is twenty six. Jordan was diagnosed when he was almost four, 0:02:14.560 --> 0:02:17.400 and at that time he was diagnosed at our local 0:02:17.440 --> 0:02:20.239 hospital in Indianapolis, which is a pretty big, well known 0:02:20.320 --> 0:02:24.760 children's hospital. But we left there pretty much with no hope, 0:02:24.800 --> 0:02:27.960 and the doctor told us basically, this was a fatal disease, 0:02:28.040 --> 0:02:32.440 no cure, probably nothing even coming up through the pipeline 0:02:32.480 --> 0:02:36.600 that would happen in Jordan's lifetime. It turns out the 0:02:36.639 --> 0:02:40.520 doctors who are wrong. Fast forward now, I mean Jordan's 0:02:41.440 --> 0:02:44.560 is nine and a half and I think, um, there 0:02:44.560 --> 0:02:49.720 are more than forty drugs actually in the investigational stages 0:02:49.919 --> 0:02:53.120 for his disease. So a lot's happened in such a 0:02:53.200 --> 0:03:02.480 short time with the science. First, a little background. The 0:03:02.520 --> 0:03:05.400 easiest way to get access to an experimental drug is 0:03:05.440 --> 0:03:09.240 in a clinical trial, but many people can't participate in trials. 0:03:09.720 --> 0:03:12.919 They may be too sick or too young for them. 0:03:12.960 --> 0:03:17.200 There's something called compassionate use. It's an official program regulated 0:03:17.200 --> 0:03:20.840 by the Food and Drug Administration, but that's also difficult 0:03:20.840 --> 0:03:24.120 to navigate. There's paperwork, and you need the f d 0:03:24.120 --> 0:03:27.120 A s and the drug company's blessing, and it's possible 0:03:27.120 --> 0:03:29.480 that drug company may not give it. There's too much 0:03:29.560 --> 0:03:35.440 risk enter right to try. This is the law that 0:03:35.560 --> 0:03:38.960 Laura McGlenn and Matt Bolina fought for. Its main difference 0:03:39.440 --> 0:03:42.440 it cuts out the f d A. Instead, the doctor 0:03:42.520 --> 0:03:45.800 directly asked the company for a drug. The patients take 0:03:45.840 --> 0:03:49.160 on all the risk and make their own decisions, but 0:03:49.240 --> 0:03:51.120 they do still need to get the drug company to 0:03:51.160 --> 0:03:55.080 go along with it. I'm not gonna lie. I mean, 0:03:55.080 --> 0:03:58.920 when I started this journey, all I could think about 0:03:59.080 --> 0:04:04.400 was my one, my Jordan's. Jordan got lucky, he was 0:04:04.440 --> 0:04:07.600 accepted into a clinical trial, but Laura didn't give up 0:04:07.600 --> 0:04:10.120 her fight for right to try. She did it for 0:04:10.160 --> 0:04:13.480 the other boys, and because the disende community is so close, 0:04:14.000 --> 0:04:16.600 she's seeing firsthand what happens to those who can't get 0:04:16.600 --> 0:04:21.279 into trials. We want our boys to access experimental treatments 0:04:21.279 --> 0:04:25.920 through a clinical trial. Unfortunately, for example Jordan's, he's one 0:04:25.960 --> 0:04:28.839 of sixteen boys in North America that are able to 0:04:28.880 --> 0:04:32.279 try the drug that he's trying. While I watch many 0:04:32.360 --> 0:04:35.200 of my friends who have sons who are amendable to 0:04:35.240 --> 0:04:38.360 the same drug, who can't access the drug because they 0:04:38.360 --> 0:04:41.960 don't qualify for the trial, and so their sons are basically, 0:04:42.200 --> 0:04:45.680 you know, um, continuing to progress through this disease, which 0:04:45.720 --> 0:04:48.599 is a muscle wasting disease, and they're losing muscle function 0:04:48.680 --> 0:04:54.480 and it's it's truly heartbreaking. Mike Smith, a New York attorney, 0:04:54.520 --> 0:04:57.720 also wants better access to medicines. So now, I grew 0:04:57.800 --> 0:05:01.120 up on Long Island, New York, and the town called Sayaucet, 0:05:01.720 --> 0:05:05.239 Um basically had a up until I was fifteen, frankly 0:05:05.279 --> 0:05:10.640 had a perfectly normal and uneventful childhood. That's sort of 0:05:10.720 --> 0:05:13.440 changed on Black Monday, the day of the stock market 0:05:14.160 --> 0:05:19.599 crash on October seven. The day started with Mike scoring 0:05:19.600 --> 0:05:22.040 a goal in a soccer game during his gym class. 0:05:22.800 --> 0:05:26.279 The opposing goalie didn't take it well. He punched Mike 0:05:26.279 --> 0:05:28.599 in the chest and kicked him in the lower right side. 0:05:29.279 --> 0:05:32.240 He immediately fell ill. It was the start of a 0:05:32.279 --> 0:05:36.080 long and harrowing health odyssey. It took five years just 0:05:36.160 --> 0:05:40.240 to get a diagnosis, and we were starting to identify 0:05:40.279 --> 0:05:44.400 that I had a disease or disorder called chronic intestinal 0:05:44.480 --> 0:05:48.120 pseudo obstruction. It is an extreme spastic disorder of the 0:05:48.160 --> 0:05:51.000 digestive track. I'd be sitting in a high school classroom, 0:05:51.040 --> 0:05:54.120 for example, and get a sensation as if my stomach 0:05:54.160 --> 0:05:56.279 was being tied into a shoel. I snot, you know, 0:05:56.320 --> 0:05:58.800 And ever since, I really have never been able to 0:05:58.839 --> 0:06:02.240 take eating or even drink king for granted. Essentially, his 0:06:02.360 --> 0:06:06.200 digestive tract stopped working. For several years, he couldn't need 0:06:06.240 --> 0:06:10.600 at all. He relied on artificial nutrition, and then thankfully 0:06:11.000 --> 0:06:14.560 new drugs came along. He could eat again. But the 0:06:14.560 --> 0:06:17.200 two treatments were pulled from the market in the US 0:06:17.240 --> 0:06:21.080 because of potential side effects, but not in Europe. Mike 0:06:21.200 --> 0:06:25.880 was back to square one. Now here's the thing even 0:06:25.960 --> 0:06:28.320 under right to try he might not get the drugs 0:06:28.320 --> 0:06:32.120 that he knows work for him, because technically it's only 0:06:32.160 --> 0:06:35.919 for people with the terminal diagnosis and Mike's condition. Isn't 0:06:35.920 --> 0:06:39.719 that simple? On the span of my condition at this point? 0:06:40.440 --> 0:06:44.840 Is it chronic and debilitating? Yes? Absolutely? Can we say 0:06:44.880 --> 0:06:48.560 fairly on a day to day basis? Is it life threatening? 0:06:48.880 --> 0:06:51.240 I'm not a doctor. It's not for me to determine 0:06:51.240 --> 0:06:54.400 whether or not it's life threatening. But who should make 0:06:54.440 --> 0:07:00.280 that call? Should patients like Mike have access? Over the 0:07:00.320 --> 0:07:03.039 past five years, more than forty states passed right to 0:07:03.080 --> 0:07:08.640 Try laws. Those efforts culminated in the federal legislation. It 0:07:08.720 --> 0:07:13.040 was a rare bipartisan achievement, pushed by Vice President Mike Pence. 0:07:13.520 --> 0:07:16.360 Right to Try one support in both Houses of Congress. 0:07:16.880 --> 0:07:19.640 It was touted by President Donald Trump in his first 0:07:19.720 --> 0:07:22.560 State of the Union address. He signed it into law 0:07:22.640 --> 0:07:25.880 in May. But this is such a great name. For 0:07:25.920 --> 0:07:28.600 the first day I heard it, It's so perfect right 0:07:28.720 --> 0:07:32.160 to try, and a lot of that trying is going 0:07:32.200 --> 0:07:35.360 to be successful. I really believe that. I really believe it. 0:07:35.880 --> 0:07:40.080 Ibraheim del Pasand is also a believer. More importantly, he's 0:07:40.120 --> 0:07:43.760 the medical director at Houston's Excel Diagnostics. He's also an 0:07:43.800 --> 0:07:48.120 expert in nuclear medicine. That means he uses radioactive materials 0:07:48.320 --> 0:07:53.760 to diagnose and treat patients. Hey, Michelle, Hi, here we go, 0:07:53.880 --> 0:07:58.360 we're working now. Okay, very good. Doctor del Pasand is 0:07:58.400 --> 0:08:01.320 the doctor who is most publicly braced right to try. 0:08:01.520 --> 0:08:04.560 He felt he had no other option. There was a 0:08:04.640 --> 0:08:07.320 drug he wanted to give his patients, a form of 0:08:07.400 --> 0:08:11.040 radiation for neuroendocrine cancer that had been used in Europe 0:08:11.080 --> 0:08:15.440 for years, but it wasn't approved in the US. Steve Jobs, 0:08:15.560 --> 0:08:18.480 the late founder of Apple, went to Switzerland to get it. 0:08:19.000 --> 0:08:22.600 But that's not something that most Americans can do. How 0:08:22.680 --> 0:08:26.600 sick were these patients? I mean majority of these patients 0:08:26.680 --> 0:08:30.440 that I treated, they had a stage four cancer, which 0:08:30.480 --> 0:08:34.680 means that the cancer has already spread throughout the body 0:08:35.320 --> 0:08:39.720 and involved different organs such as livers such as lymph 0:08:39.720 --> 0:08:43.640 notes such as bone, lungs. A majority of these patients 0:08:43.679 --> 0:08:48.320 they were expecting to leave between three to twelve months. 0:08:48.360 --> 0:08:52.679 Mix Meanwhile, there were positive reports out of Europe. American 0:08:52.720 --> 0:08:56.520 patients were reading them and they wanted the drug. There's 0:08:56.559 --> 0:09:00.160 a twist here. The treatment isn't a pill, It a 0:09:00.280 --> 0:09:03.839 radioactive isotope that has to be carefully built in a lab, 0:09:04.679 --> 0:09:07.360 and Dr del Pasand just happens to be also the 0:09:07.400 --> 0:09:10.240 CEO of a company who has the equipment to make it. 0:09:11.120 --> 0:09:13.600 So he got permission from the U S regulators and 0:09:13.640 --> 0:09:16.080 the company that owned the rights to the therapy and 0:09:16.240 --> 0:09:19.800 ramped up production. In that way, I was able to 0:09:19.920 --> 0:09:24.960 actually provide this necessary treatment to my patients and they 0:09:24.960 --> 0:09:28.600 were able to get this treatment in US and in 0:09:28.679 --> 0:09:33.439 Houston versus UH instead of going across the ocean to Europe. 0:09:34.000 --> 0:09:37.319 Dr del Pasand initially had FDA approval to give the 0:09:37.400 --> 0:09:41.880 drug to his patients, but in April, the agency told 0:09:41.920 --> 0:09:45.040 him he was interfering with efforts to get the treatment approved. 0:09:45.800 --> 0:09:49.360 So at that point, basically I noted that, you know, 0:09:49.640 --> 0:09:53.600 I have to stop treating patients because it's going to 0:09:53.640 --> 0:09:58.040 be illegal for me to continue treatment. Fortunately, for his 0:09:58.120 --> 0:10:00.600 cancer patients, a Right to Try law ALWA was passed 0:10:00.640 --> 0:10:04.320 at the state level in Texas two months later, and 0:10:04.400 --> 0:10:07.400 as a result of this Right to tryl I was 0:10:07.440 --> 0:10:12.640 able to UH to treat hundred seventy eight patients until 0:10:12.679 --> 0:10:16.680 the drug became approved and commercially available in February of 0:10:16.880 --> 0:10:20.120 two thousand and eighteen a study of the drug used 0:10:20.120 --> 0:10:22.880 by Dr del pasand showed that patients lived for an 0:10:22.880 --> 0:10:26.720 average of thirty months without their cancer progressing. That compared 0:10:26.720 --> 0:10:29.480 to less than nine months for those given standard care. 0:10:30.400 --> 0:10:33.200 The doctor got letters from his patients thanking him for 0:10:33.240 --> 0:10:39.520 his efforts. I asked him to read me excerpts from one. 0:10:40.240 --> 0:10:45.000 Here it says that when I was diagnosed, my daughter 0:10:45.160 --> 0:10:49.120 was just graduating high school and my son just finished 0:10:49.160 --> 0:10:52.439 his first year in college. I have seen both of 0:10:52.480 --> 0:10:56.960 them graduate college and settled into their chosen professions. I 0:10:57.080 --> 0:11:00.439 also have seen each one fall in love with wonderful people. 0:11:01.120 --> 0:11:03.960 I own a business, and my family did not have 0:11:04.160 --> 0:11:07.040 to sell it. All this from the hard work of 0:11:07.200 --> 0:11:11.079 people devorded to helping with the serious challenges in someone's 0:11:11.120 --> 0:11:14.640 life and being assisted by the common sense right to 0:11:14.760 --> 0:11:22.320 try law. It's it's just amazing. I think this is 0:11:22.360 --> 0:11:25.720 the best pay day for when when you read something 0:11:25.800 --> 0:11:30.560 from a patients, from a human being. Uh, it's just 0:11:31.559 --> 0:11:39.200 it's just chriceless. Right to try has now been the 0:11:39.280 --> 0:11:42.280 law of the land on a national level for about 0:11:42.360 --> 0:11:46.120 six months. I have to be honest, I tried really 0:11:46.160 --> 0:11:49.559 hard to find patients, any patients who have gotten an 0:11:49.559 --> 0:11:53.640 experimental medicine under the federal law. No luck so far. 0:11:54.800 --> 0:11:58.200 Some patients and families were very candid with me, saying 0:11:58.200 --> 0:12:00.040 they would never tell if they were getting treat and 0:12:00.160 --> 0:12:03.800 under Right to Try. There's too much risk of a backlash, 0:12:03.960 --> 0:12:07.200 and if you're getting the drug, there's absolutely no benefit 0:12:07.240 --> 0:12:11.760 to going public. Eventually we will find out if anyone's 0:12:11.760 --> 0:12:14.600 getting treatment under Right to Try. The companies have to 0:12:14.640 --> 0:12:17.280 send the f d A a report once a year 0:12:17.800 --> 0:12:21.120 telling the agency about any medicines they provided under the law. 0:12:21.840 --> 0:12:25.920 We still don't know exactly when that will be. Not 0:12:26.120 --> 0:12:29.280 everyone agrees that dying patients should have unfettered access to 0:12:29.400 --> 0:12:35.240 unapproved drugs. Experimental medicines are precious and unpredictable things. You 0:12:35.320 --> 0:12:38.959 have to be careful with both. When the medicines do work, 0:12:39.320 --> 0:12:42.520 when they really are going to save lives, proving their 0:12:42.559 --> 0:12:46.880 efficacy and scientific studies has to be the priority. Controlled 0:12:46.880 --> 0:12:49.440 clinical studies are also the best way to protect patients 0:12:49.440 --> 0:12:53.760 from harm even death when they don't work. I was 0:12:53.800 --> 0:12:56.960 surprised about who's opposed to Right to Try. The laws 0:12:57.000 --> 0:13:02.240 backers believe that cancer patients will really benefit. Otis Browley 0:13:02.320 --> 0:13:05.679 is the chief medical officer of the American Cancer Society. 0:13:06.240 --> 0:13:08.840 He spoke to me from his office in Atlanta, where 0:13:08.840 --> 0:13:12.320 a fire alarm went off while we were talking. I 0:13:12.400 --> 0:13:16.720 have come to realize that there truly is something worse 0:13:16.760 --> 0:13:23.080 than dying from cancer. I've seen people suffer getting drugs 0:13:23.120 --> 0:13:27.040 for a treatment and then not have benefit. I have seen, 0:13:27.600 --> 0:13:32.880 even in standard practice, people who were tortured because of treatment. 0:13:33.000 --> 0:13:36.360 I just don't want to see that um ever again. 0:13:36.760 --> 0:13:39.560 Dr Browley is concerned that the right to try law 0:13:39.600 --> 0:13:43.360 will exploit people who have no other options. One of 0:13:43.360 --> 0:13:46.520 the problems with people who are in this situation I've 0:13:46.559 --> 0:13:50.120 talked to many of them is sometimes I fear that 0:13:50.160 --> 0:13:53.640 they see too much hope in the drug and don't 0:13:53.720 --> 0:13:57.240 really appreciate what we're talking about in terms of harms. 0:13:57.840 --> 0:14:01.160 Keep in mind, I'm somebody who wants these people to 0:14:01.240 --> 0:14:05.000 be able to have access to these drugs if they 0:14:05.000 --> 0:14:09.280 are well informed and understand. My problem is, I know 0:14:09.400 --> 0:14:12.840 a large number of people are not well informed and understand. 0:14:13.480 --> 0:14:16.839 Dr Browley's point was that sometimes hope can be misleading. 0:14:17.440 --> 0:14:20.120 Most medicines don't make it through. Is that correct. It 0:14:20.280 --> 0:14:24.440 is absolutely correct. Most of the medicines that we have 0:14:25.080 --> 0:14:29.000 theoretic belief that it's going to be beneficial for cancer, 0:14:29.600 --> 0:14:34.160 so much belief that we actually start clinical trials in 0:14:34.280 --> 0:14:38.000 those drugs to figure out if they work and figure 0:14:38.040 --> 0:14:42.720 out if they improve outcome. We find that most of 0:14:42.760 --> 0:14:47.040 those drugs actually fail. So most people who are going 0:14:47.080 --> 0:14:50.000 to be getting these drugs through Right to Try UM 0:14:50.920 --> 0:14:53.440 are going to get drugs that aren't going to work, 0:14:54.240 --> 0:14:59.160 but some undoubtedly will work, and that creates another problem. 0:14:59.320 --> 0:15:02.400 Companies are focused on developing medicines for big groups of 0:15:02.440 --> 0:15:05.840 people who suffer from a disease. In the early days, 0:15:06.120 --> 0:15:09.960 they can't make big batches just enough for studies, and 0:15:10.000 --> 0:15:13.239 the drugs can be hard to make. It's a particular 0:15:13.280 --> 0:15:16.480 problem at small biotech companies that are often the engine 0:15:16.520 --> 0:15:19.960 of innovation. They work on tighter budgets and are more 0:15:20.080 --> 0:15:25.320 vulnerable to mishaps. Typically, patients are very sick, and giving 0:15:25.320 --> 0:15:27.880 away treatment to a single patient under Right to Try 0:15:28.040 --> 0:15:31.560 could jeopardize the entire research effort if something goes wrong. 0:15:32.160 --> 0:15:35.000 If someone is taking one of these drugs and you're 0:15:35.040 --> 0:15:39.120 not in an established clinical trial where people can learn 0:15:39.160 --> 0:15:44.200 about that experience on the drug, then you're actually slowing 0:15:44.280 --> 0:15:48.120 down the development of the drug for everybody else. It 0:15:48.200 --> 0:15:52.240 really is selfish. It's not a theoretical risk. Some companies 0:15:52.280 --> 0:15:55.120 have trouble in enrolling early stage studies, known as phase 0:15:55.160 --> 0:15:58.360 one and phase two. Allowing some patients to have the 0:15:58.400 --> 0:16:02.000 experimental drug without participating in a trial could make the 0:16:02.040 --> 0:16:06.640 process harder. I really want people to go into those 0:16:06.680 --> 0:16:10.960 phase one and phase two clinical trials and really try 0:16:11.000 --> 0:16:15.800 to help us to move cancer medicine forward as expeditiously 0:16:16.040 --> 0:16:22.480 and as efficiently as possible. Dr Browley isn't against patients 0:16:22.480 --> 0:16:25.800 getting fast access to promising new drugs. He just wants 0:16:25.840 --> 0:16:28.840 to capture all the information possible about how they work, 0:16:29.360 --> 0:16:32.680 preferably in a monitored setting, and he wants to protect 0:16:32.680 --> 0:16:39.120 the patients who are taking experimental drugs from unintended harm. 0:16:39.280 --> 0:16:42.920 Remember before Right to Try, terminally ill patients could get 0:16:42.960 --> 0:16:46.600 experimental drugs under the FDA's Compassionate Use Program, which is 0:16:46.600 --> 0:16:50.440 also known as Expanded Access. In recent years, it's approved 0:16:50.440 --> 0:16:55.520 more than of applications. F d A Commissioner Scott god Leaves, 0:16:55.560 --> 0:16:59.560 a fan of Expanded access Right to Try, not so much. 0:17:00.120 --> 0:17:02.480 He declined to talk to us for this podcast, but 0:17:02.560 --> 0:17:07.200 he did testify before Congress about the topic in right now, 0:17:07.240 --> 0:17:11.760 our expanded access program is generally available for patients facing 0:17:12.520 --> 0:17:16.200 life threatening conditions and terminal illness. The agency has gotten 0:17:16.240 --> 0:17:19.920 even more vigilant at trying to move these things through 0:17:19.960 --> 0:17:22.919 the agency in a in a efficient fashion and approve 0:17:23.040 --> 0:17:26.480 these The FDA also has access to information that no 0:17:26.560 --> 0:17:30.639 single doctor or patient has. It knows if complications cropped 0:17:30.680 --> 0:17:33.840 up with earlier use of the drug or with similar medicines. 0:17:34.600 --> 0:17:38.920 Such side effects aren't always immediately disclosed. The FDA's guidance 0:17:38.960 --> 0:17:47.439 can help make sure experimental drugs are used safely. Drug 0:17:47.480 --> 0:17:51.040 makers have the last word on their medicines, and it's 0:17:51.080 --> 0:17:54.320 tempting to point the finger at pharmaceutical companies that say 0:17:54.359 --> 0:17:58.360 no to a desperate patient, But it's more complicated than that. 0:17:59.359 --> 0:18:02.600 Drug maker have been grappling with these requests for years. 0:18:03.520 --> 0:18:07.960 Johnson and Johnson's chief medical officer, Joanne wald Striker says 0:18:07.960 --> 0:18:10.879 it's one of the hardest and most heartbreaking parts of 0:18:10.920 --> 0:18:16.160 her job. Sometimes, before we know everything about our products 0:18:16.160 --> 0:18:19.439 in advance of getting approvals from the health authorities like 0:18:19.520 --> 0:18:21.840 the f d A, before they even get to see 0:18:21.880 --> 0:18:25.359 the data on the product, we sometimes get requests for 0:18:25.480 --> 0:18:30.160 our products from patients and families around the globe who 0:18:30.200 --> 0:18:34.560 have exhausted all other options for treatment. There's a long 0:18:34.680 --> 0:18:38.840 list of issues the company has to consider when evaluating requests. 0:18:39.720 --> 0:18:42.880 Is there enough medicine available, We'll giving it to one 0:18:42.920 --> 0:18:46.040 patient make it harder to enroll others in clinical trials. 0:18:47.080 --> 0:18:50.280 If J and JA gets more requests than the available supply, 0:18:50.800 --> 0:18:54.280 who gets the drug and who doesn't? And if things 0:18:54.320 --> 0:18:57.440 go wrong and if fragile patient dies, will that put 0:18:57.480 --> 0:19:00.840 the drugs entire development in jeopardy. We have to weigh 0:19:00.840 --> 0:19:04.320 the need of an individual person and family who's requesting 0:19:04.359 --> 0:19:09.520 access versus the needs of society, versus our our obligation 0:19:09.680 --> 0:19:12.840 to society into public health. And that is a real 0:19:13.119 --> 0:19:17.199 bioethic challenge that we face. And you can imagine if 0:19:17.280 --> 0:19:19.640 a person is enrolled in a clinical trial and they 0:19:19.680 --> 0:19:23.359 have the chance of getting either the drug or the placebo, 0:19:23.520 --> 0:19:26.119 it may not be fair to that person if we 0:19:26.240 --> 0:19:28.840 decide to give access to someone else outside of the 0:19:28.840 --> 0:19:32.320 clinical trial setting, just because they asked and they're not 0:19:32.359 --> 0:19:34.439 going into a study where they have a chance of 0:19:34.480 --> 0:19:38.760 getting either drug or placebo. Hanging over the entire issue 0:19:39.200 --> 0:19:43.119 is fairness. Some families and patients have the wherewithal to 0:19:43.240 --> 0:19:47.760 create powerful social media campaigns to shame companies or to 0:19:47.800 --> 0:19:50.600 try to force them to make an experimental medicine available, 0:19:51.240 --> 0:19:55.120 especially for children. But if there's one child who needs 0:19:55.119 --> 0:19:58.399 an experimental drug, there are likely more. We have to 0:19:58.440 --> 0:20:01.600 treat people in clinical trials early. But also we want 0:20:01.640 --> 0:20:06.040 to treat people in all different socioeconomic circumstances fairly. If 0:20:06.080 --> 0:20:09.240 we get a request of someone who's a public figure, 0:20:09.680 --> 0:20:12.760 someone who's in the government, someone who's very wealthy, we 0:20:12.800 --> 0:20:15.760 want to be sure we're treating those requests the same 0:20:15.800 --> 0:20:18.359 as people who have a need who may not be 0:20:18.400 --> 0:20:21.959 in those circumstances. We want to treat everyone fairly, and 0:20:22.000 --> 0:20:26.600 so that's why this is such a challenging issue for us. 0:20:26.600 --> 0:20:29.439 So the company decided to bring in outside experts to 0:20:29.520 --> 0:20:33.600 help deal with all these issues. In twenty fifteen, we 0:20:33.720 --> 0:20:36.840 approached the Division of Medical Ethics at the n y 0:20:36.960 --> 0:20:40.000 U School of Medicine to really help us think this through. 0:20:40.480 --> 0:20:44.560 Instead of physicians like myself at Johnson and Johnson reviewing 0:20:44.600 --> 0:20:48.320 every request and then having individual teams try to juggle 0:20:48.400 --> 0:20:52.080 and make this difficult bioethical decision in terms of whether 0:20:52.080 --> 0:20:56.240 to provide the product. The requests go to this group 0:20:56.280 --> 0:21:00.480 called the Compassionate Access Group Compact. You know, I feel 0:21:00.800 --> 0:21:07.719 very strongly that people who have such important medical needs 0:21:07.800 --> 0:21:10.800 and who are in such urgent situations should have a 0:21:10.920 --> 0:21:13.760 level of control over what happens to them. And I 0:21:13.840 --> 0:21:16.280 sympathize and I understand that they want to be able 0:21:16.320 --> 0:21:20.800 to access experimental medicines. But on the other hand, at 0:21:20.800 --> 0:21:23.760 a time like that, when you're desperate to try anything, 0:21:24.359 --> 0:21:28.200 it's a time when expert input really can help make 0:21:28.200 --> 0:21:31.560 a difference. Of course, Jay and J is an outlier. 0:21:32.119 --> 0:21:34.760 It's the biggest health care company in the world with 0:21:34.880 --> 0:21:39.199 unmatched resources. Clearly, not every company can afford to do 0:21:39.320 --> 0:21:42.359 what it does, but it is a start, right to 0:21:42.400 --> 0:21:44.400 try us put the f d A and the industry 0:21:44.400 --> 0:21:47.240 on notice that the immediate needs of patients must be 0:21:47.320 --> 0:21:54.600 taken into account. Laura McGlynn, the woman we met at 0:21:54.640 --> 0:21:57.680 the beginning of this episode, is taking it day by day. 0:21:57.880 --> 0:22:00.240 Her son Jordan, is enrolled in a clinical try aisle, 0:22:00.720 --> 0:22:03.840 but she believes one day he'll need other medicines, and 0:22:03.880 --> 0:22:07.200 that's why she keeps fighting. I feel proud to know that, 0:22:07.400 --> 0:22:10.399 you know, my son played a part in helping create 0:22:10.440 --> 0:22:13.720 this additional pathway, so rather Jordan is able to access 0:22:13.800 --> 0:22:18.000 better treatments in the future through clinical trials, through um 0:22:18.320 --> 0:22:21.600 compassionate use, through right to try, through maybe you know 0:22:21.960 --> 0:22:25.080 an f D approved drug. I'm just glad to know 0:22:25.160 --> 0:22:29.080 that there are options available and to those who feel 0:22:29.119 --> 0:22:31.760 like right to try as a shortcut a form of 0:22:31.800 --> 0:22:35.120 cutting in line that could hurt someone else. I don't 0:22:35.200 --> 0:22:37.680 let my mind go there as far as thinking about 0:22:37.760 --> 0:22:40.720 what's fair or not fair if a life is saved, 0:22:40.800 --> 0:22:43.560 or if someone has a chance to try to save 0:22:43.560 --> 0:22:46.920 their own life and they want to, I don't care. 0:22:47.280 --> 0:22:50.080 It's one life at a time, and that's to me, 0:22:50.200 --> 0:23:03.399 that's what matters. And that's it for this week's prognosis. 0:23:03.640 --> 0:23:06.600 Thanks for listening. Do you have a story about healthcare 0:23:06.640 --> 0:23:09.240 in the US or around the world, We want to 0:23:09.280 --> 0:23:12.399 hear from you. You can email me m Cortes at 0:23:12.400 --> 0:23:15.720 Bloomberg dot net or find me on Twitter at bay Cortes. 0:23:16.440 --> 0:23:18.880 If you were a fan of this episode, please take 0:23:18.880 --> 0:23:21.200 a moment to rate and review us. It helps new 0:23:21.240 --> 0:23:25.840 listeners find the show. This episode was produced by Lindsay Cratterwell. 0:23:26.440 --> 0:23:29.800 Our story editor was Cecile Durraant. Thanks also to Drew 0:23:29.920 --> 0:23:34.160 Armstrong Francesca Leavi as head of Bloomberg Podcasts. We'll see 0:23:34.200 --> 0:23:34.760 you next week.