Rare Candace Interview
Candace Lerman is a powerful voice for rare disease patients. After being diagnosed becoming ill with a rare blood disease of unknown origins in 2014, Candace lost her job. So she went to law school to learn how to fight for people like her.
Listen to her incredible story and be sure to visit her blog, as she fights to get the treatments that could help her.
Leonard: Hi, this is Leonard Kish with Real World Evidence Today where we talk about real world evidence and how it relates to drug development and patients and improving patient care and I'm really pleased to be here today with Candace Lerman who's an attorney consultant and author of the rare disease blog rarecandace.com. You're a JD with a certificate in health compliance. You've worked closely with members of Congress and have really taken a lead with this unfortunate condition that you have in finding ways to get new drugs on the market to treat it; so thanks for joining us today Candace.
Candace: Thank you for having me.
Leonard: So let's just start a little bit with your story. You have a rare condition known as ITP.
Candace: Yes. My B-cells are attacking my platelets and it can cause deadly internal bleeding.
Leonard: So tell us a little bit about how that came about or when this first happened and what's happened up until now?
Candace: So I started and what I now know were the first symptoms of ITP presented around January of 2014. I was at work and I slipped out of a van and kind of bumped my back of my legs on a metal bar in the seat and I had a set of bruises on the back of my legs that were very symmetrical and a little unusual. My family kind of point that out to me at the time but I didn't think much of it. Then over the next few months I had very strange symptoms. I had the petechiae which are sort of little broken blood vessels about the size of a pinprick show up in my legs in March 2014. I thought perhaps I had developed a rash. I had used a new shaving cream that morning and so I saw a dermatologist who just told me I needed to wear compression socks at work because I was on my feet too much. I didn't think anything of it. Come April of 2014 my family became very concerned because I started having giant bruising or what they would call clinically as hematomas and I am very fair skinned so they were very bright, very dark dark purple and red and painful. So I ultimately decided to go see my gynecologist thinking that maybe I had anemia or something was wrong with my thyroid not realizing how serious my condition was and she took a regular blood test to check my platelet levels and it came back to 7000 platelets, so I was in big trouble.
Leonard: So that was kind of the beginning but when did you really get a firm diagnosis of ITP?
Candace: So I ended up after being hospitalized for six days seeing a researcher and clinician at the University of Miami by a doctor who has spent decades researching ITP and it took about six months but I was eventually confirmed with chronic ITP as a diagnosis and we went through a process of putting me on prednisone trying to taper me off of it. I went through an extensive amount of blood tests to confirm that there was no other underlying cause of thrombocytopenia and I even saw an infectious disease doctor at the University of Miami just to make sure there wasn't something else that we were missing.
Leonard: So in 2014 this all started and then you decided because of the rare disease and how they're not well-represented rare diseases you decided to go to law school put forward about that and what drove that decision?
Candace: Yes. So I was very fortunate to have amazing medical care and a team of people behind me who wanted to give me sort of a new normal living with ITP and we had repurposed the drug Rituxan which ultimately put me in remission. It shut down my B-cells in my immune system that were targeting my platelets and ultimately having returned to a normal platelet level and since I lost my corporate job I was struggling with sort of where do I go from here. My life as I know had changed and I wanted to do something to fix the system and the problems that I encountered after I was diagnosed. So (talked to) my doctor who didn't think medical school was a great idea, since I'm immunocompromised and being exposed to different things would possibly trigger my immune system to do something terrible to me. So I looked at going to law school. It seemed to me that a lot of the problems stemming in medicine and drug development are caused by lawyers, so I decided to become one.
Leonard: Yeah well certainly the system in general and the way things are done and lawyers are part of that. So what is it that you're driving as an attorney now that you want to see change in the system?
Candace: One of my big things as an attorney is dealing with the regulatory process everything from patient groups and pharmaceutical companies and life science to life science industry in general, working with them on legislation on Capitol Hill and health data and how health data and patient data can change medicine to increase better outcomes for care. But also (to) change the regulatory environment and make it more friendly for drug development.
Leonard: So you've benefited I suppose from Rituxan and are there things around that specific drug or other drugs that you want to see changed for rare diseases or what is the issue with rare diseases and what regulatory is doing that you want to change?
Candace: I think one of the biggest challenges for rare diseases is that when you have a small patient population, it's extremely difficult to get the life sciences industry interested in researching and developing therapies. Since my story went viral in early 2015 when I wrote an article about using Rituxan I had so many patients come up to me with a variety of different rare diseases and tell me they are repurposing story that perhaps they themselves or in working with a physician found a drug that labeled for another indication that was changing their life and giving them a chance to find their new normal just as I have. So I'm trying very hard to figure out different innovative ways to engage with all stakeholders between patients and patient communities to the life sciences industry, health data groups, and the government just find friendlier pathways so when we do find a drug that works off label for a rare disease we can get it on label quicker and faster and also to eliminate the burden on the patient and patient community to try to fundraise and raise awareness for some of these rare diseases. I have friends that have children that are repurposing drugs that are keeping them alive and they've living past their life expectancy two to three times over. But there may be only 60 or 70 patients identified in the world and it's unfair to expect these parents with these children and these small communities to be able to operate in the same fashion as some of the larger disease or more common diseases do.
Leonard: So you mentioned with ITP there's maybe 50 to 80,000 patients in the US that have this disease, are there off-label drugs that you want to make it easier for them to know about. Or off-label drugs but you also mentioned citizen science and that you want them to be more of a resource to pharma, so how do you see some of this playing out or can you give some examples of how this might work?
Candace: I think one of the big things that pharma is missing out on right now that they could capitalize on and would benefit patients greatly and improve medicine is engaging with the community if they are able to find or have found a drug that is possibly in their portfolio for another indication and is being repurposed. There seems to be a lack of communication in this respect and a lot of times these discussions are occurring openly on social media within Facebook groups or on Twitter even on Instagram under pictures sometimes patients are connecting and sharing innovative ways that medicine is working for them. I would love to see more clinical trials for off-label drugs so we can put them on label. We can confirm their safety and efficacy for rare disease populations and then we can go from there with that therapy and perhaps improve it. In the age of precision medicine and the idea that every patient is unique building a sort of information highway between all stakeholders to identify the key needs in different rare disease communities and then going back out and looking at what we already have on the market that could help them would transform medicine in a positive way for everyone.
Leonard: It looks like with so-called real world evidence usually today that means the EHRs, claims data we're starting to see some approvals. There was one for (I don’t remember the name of the drug) recently but for breast cancer that was just approved for men had initially been approved for women just based on some of that real world evidence (in EHR data), so in that instance a whole new clinical trial i(was not) needed are you seeing the situation or maybe openings where things could be approved without full clinical trials?
Candace: I think so. I think that there's a lot of drugs out there that are being repurposed right now in rare disease communities where the physicians are possibly collecting the data for themselves. Perhaps they see a cluster of patients and have found a pattern and are treating them and they're getting the data and then the data is just sitting there. But ultimately all of these patients are sort of like their own n of one trial where they're using the repurposed drug if they have success and then their quality of life is improved. So there is possibly a chance that we could take the data that's already out there through patient portals and claims data and maybe a hospital system EHR and develop a sort of compelling argument to the FDA that look we don't really need to figure and go back to square one and start a trial all over. We kind of have everything perhaps we're able to skip Phase 1, Phase 2 and go right to Phase 3 and I think that this is something where everybody sorts all the stakeholders need to sit down at the table and figure out, how would we regulate that? What type of conversation could we have surrounding if we're already repurposing a drug in a kidney disease community and its successful what are ways that we can eliminate the roadblocks to getting it on label so that the patient community can access it faster which will ultimately help health outcomes for those patients.
Leonard: I think with ITP in particular there's 50,000 to 80,000 patients out there large part of those are probably in community like you are because there just aren't that many experts that they can lean on because there just aren't that many experts in rare diseases. So patients and particularly the patient communities wind up becoming de facto experts on the disease and what works and what doesn't. So there's probably a lot of great info there, right?
Candace: Absolutely and a lot of times like just in the ITP community or rare diseases in general patients will tell you when they see a doctor, it's not uncommon for the doctor to tell you, “I know you have this disease but I've never heard of it before.” And I think sometimes when we look at rare diseases we think about the one specialist that they may see for that disease but health care as a whole is comprehensive. We all have primary care physicians. For women we're seeing OBGYN and men see the urologist and so our diseases aren't limited to whatever the category is they fall under. So like for ITP we see hematologist but it’s my gynecologist who has to understand ITP. My primary care physician does too as does my dentist. So when patient communities are able to engage with real world evidence from their own patients and the recognized maybe care patterns or treatment patterns surrounding a specific drug or perhaps a device or technique there's an opportunity there to get that information and validate it, package it, and have the patients be able to provide it to their physicians no matter what specialties are practicing in and it provides us a way in a sort of a roadmap to deal with these patients but it has to come from the patients because it's impossible to exact the medical community to be an expert in all 7000 rare diseases.
Leonard: It makes me wonder if we could get a continuing medical education CME credits for engaging with patient communities for physicians, right?
Candace: Yes. This is something I always advocate for especially when I work with non-profits who are looking to sort of create a better community and engage with researchers and clinicians who are interested in their disease so one of the first things I say is, “if you can get a CME together and also educate your local medical school and the students there.” I lecture a lot of times at medical schools and pharmacy schools about rare diseases and one of the things that always shocks all of them is when I tell them that one in 10 people has a rare disease and my pharmacy students are always the ones that tend to be the most shocked. If I say yes when every one of every 10 customers that comes to your counter when you're working has a rare disease you just may not realize it and what the drug you're dispensing to them may actually be being used off label to treat it. It's just they're not telling you.
Leonard: In aggregate rare diseases I guess are not all that rare but there is just amazing power in the population now in this day and age where everybody can come together so I think to sum it up what we really need is better ways of bringing patients together in communities that can lead to new insights around these rare diseases.
Candace: Absolutely. There is a mountain of data and potential data out there stemming from patients and patient communities that pharma could use to develop better treatments or to take something that's sitting on a shelf somewhere and provide it to a community that desperately needs help. But the key is engaging with patients and patient communities, building a solid relationship that is a sort of a give and take and then providing them with a way to give some sort of input whether it be their own patient data from lab results and reports or doctor's visits or something like using (Self Care Catalysts) Health Storylines and recording their daily activities or how they feel after they eat or how a medication is interacting with them and impacting their daily life. There's so much out there that's just untapped resource and I think if we do better to get the message out there and to engage with these communities in this way we're going to see just an explosion of orphan drug development.
Leonard: Let's hope so. Well thank you so much Candace it's been a really great pleasure talking with you today and good luck with everything and thanks so much for turning difficulty into a potential win for a lot of people.
Candace: Thank you for having me.