Hindsight in 2020

Coming off a decade of amazing innovation in technology, new drugs/devices, and patient advocacy, this next decade has positioned itself to make drastic improvements for everything healthcare. Who will make the effort to maximize on this progress and elevate it to new levels?

Patient Advocacy and The Patient Voice

In the last ten years, we have seen an explosion of patient advocacy initiatives making major waves in everything from drug development to legislation. Life science companies are paying attention, and interested in engaging at an increasing rate. The big question is, how do these companies engage with patients and use what they learn from them to better their practices?

It is much easier said than done. I’ve spent a lot of time in focus groups, interviews, advisory boards, and meetings, listening and leading conversations around various topics. Everything from forming registries, recruiting patients to clinical trials, post-approval marketing and general outreach. Each project comes with unique challenges, especially for teams looking to really engage with disease communities.

Finding the right patients, extracting the right information to transform it into actionable items, and establishing plans for continued engagement are the biggest challenges moving forward in this new decade. I have watched some fantastic patient advocacy projects peak, and then fizzle out with no follow up.

Why invest time, money, and hard work into a project to only let it end and not continue with the momentum you created?

Whether you’re developing treatments, building our next groundbreaking tech platform, providing services directly to patients, or operating a non-profit group that is advancing science for a better quality of life, don’t let the propulsion of your hard work die off.

Unlocking The Patient Perspective To Maximize Results

How do you incorporate the patient voice into your project?

How do you locate the right patients to gather insight?

The answers are not so simple. Each project will require varying levels of expertise from patients and may require a lot of engagement. One of the problems I often come across is the failure to compile patient insights and extract the most valuable input. It seems to be a challenge for companies to separate the substance from the noise. That’s a time consuming and expensive mistake.

It is impractical to expect your teams to be able to work on their projects while simultaneously managing the complexities of gathering, developing, and implementing the patient voice. At some point along the way, something will be lost, which is why it is critical to incorporate team members to assist in these areas. It’s the easiest way to implement patient perspectives into projects, while helping your team remain efficient.

Let’s Take on 2020 Together

Is your organization looking to increase the strength of their patient engagement and further incorporate the patient voice into projects? If so, let’s work together to make that happen.

2020 is the decade of the patient, put your organization at the forefront of the movement.

ScriptMed 2019

I had the opportunity to attend the 12th annual ScriptMed conference in Miami Beach this week. It was a fantastic and dynamic group of professionals from insurance, pharmaceutical, technology, and specialty pharmacy companies. Also there, Kristen Hartman from WEGO Health, who hosted a panel of patients discussing Online Communities, Specialty Pharmacies and the Empowered Patient.

We heard about various topics like the challenges and advancements in medical training, trends in specialty pharmacy, improving patient care through digital technology, and the future of disruption in healthcare. No matter who was on stage and what the topic covered, every speaker came back to the overall goal: helping patients.

This was a unique opportunity as a patient advocate, because I had a chance to interact with and listen to so many key players in healthcare that traditionally do not interact with patients directly. This was especially true of the speakers from insurance companies. Often, many patients and advocacy groups gripe about not being able to interact with insurance companies outside of fighting for drug approvals, so this was a special treat. I had a chance to hear about their considerations and pain points in covering expensive orphan drugs and gene therapies.

I quickly realized that patient advocates and patient groups desperately need to engage with specialty pharmacies and insurance companies when it comes to expensive therapies. There seems to be a lot of collaboration between these groups and patients are at the center of the discussion. However, patients are not presenting their input, making everything a lot more complicated. One alarming point brought up was how companies are figuring out ways to terminate employees who cost too much for health insurance coverage. On the other side, some of these self-insured groups are asking insurance companies for analysis of Quality of Life and Productivity data. It is hard to imagine an insurance company or specialty pharmacy being able to collect and assess that kind of data, so patient groups have to step up to the plate.

I made a point to participate in the Q&A after two sessions, providing some patient perspective into the conversation. Unlike some conferences where you can hear a collective sigh when a patient speaks, the speakers and audience were engaged and eager to have a discussion! Although this conference is not geared towards patients, it is critical that we listen and participate. Especially because so many different industries are asking for our help in improving their services.

It is easy to get caught up in the mentality of “us versus them.” Especially as we hear more and more about the struggle to get new and expensive gene therapies covered by insurance. There also seems to be a lack of understanding from the industry side on how to engage with patient and parent advocates who are well connected, well informed, and can help push the needle forward.

The lack of education in this area is concerning, and patient groups need to engage with their base about who to quickly bring advocates up to speed on these issues. We also need to work on ways to incorporate the patient perspective into these conversations and projects in the future.

A huge thank you goes out to the team at Inovalon for having myself and other patients at the conference!

Karen Silverblatt of Inovalon with Patient Advocate and Nutritionist Diane Melano.

Trust: Another Buzzword

It seems like every year there is a fixation on a buzzword surrounding the rocky relationship between healthcare companies and patients. From my experience, 2018 was the year for “patient engagement”. Every conference, meeting, advisory board, and conversation focused on this idea. If you spoke to 100 people about what exactly “patient engagement” means, you would get 100 different answers. 

What does that mean? Patients were not being “engaged” consistently. We heard the phrase repeatedly, but oftentimes those using it completely missed the mark. 

So what is the 2019 buzzword? Trust. 

Healthcare companies must have the trust of their patients in order to succeed, but building it goes well beyond just talking about it. When I think about trust, I remember the scene in Mean Girls where they are doing trust falls in the gym. Some companies come off as Janis Ian and are celebrated. Others take an approach like Regina George, where patients will let you hit the ground because they can see how disingenuous you are. 

Which brings up the question: how do you create trust with patients and patient communities?

Instead of focusing on the buzzword, consider what actions build trust. Going back to my Mean Girls reference (forgive me), a trust fall requires that people are there to catch you. How can you expect to establish a relationship where someone will be willing to extend their arms, if they don’t know who you are?

I see a lot of healthcare companies get this wrong. Just existing is not enough. Having social media, putting out press releases, or handing out swag at conferences will not build the relationships necessary to have patients work with you. Additionally, building trust is a two way street. No one is going to catch you falling if they can’t rely on you to do the same. That’s where you become a Regina George, and quite frankly wearing pink on Wednesdays doesn’t always work. 

Building true connections by getting to know patients and patient communities is the key to creating a mutually beneficial relationship. Whether you are a hospital, pharma/biotech, physician practice or health data/IT company, everyone has a responsibility to engage on a higher level, more than just making an appearance or talking about the latest buzzword. 

In my experience helping companies build “trust” with their patient consumers, I have found that everyone wants to be engaged, they just don’t know where to start. It is like walking into a party where you only know the host, there is no dog to pet in the corner, so you are forced to walk up to groups of people and introduce yourself. Once you start, every party becomes easier, the communication is more streamlined and you start to make friends. The greatest hurdle is making that initial introduction, especially for companies where various departments need to partner with patients. 

When creating plans to build “trust” with patients, whether it be for research, marketing campaigns, advertising your healthcare app, or inviting people to take part in sharing their data, you have to build relationships beyond repeating buzzwords. Employees responsible for creating programs, products, and devices for patients should be involved with their target audience. This is especially true for clinical trial recruitment, where many patients cite a lack of trust with pharmaceutical companies as a main reason why they would not participate.

Think about it this way: if you were to do a trust fall, wouldn’t you want to know who was catching you?

More Than a Patient

The man who saved my life, twice. Dr. Ahn.

We often talk about great doctors, those men and women who treat us with dignity and respect. They listen to us, help us, work with us, and care. Those doctors are special, and when they retire or move away, it is difficult to replace them.

When I received the letter informing me that Dr. Ahn was retiring effective February 2018, I was sad but certain that another physician at the University of Miami would take good care of me. Would they be Dr. Ahn? No, no one could ever be him. I certainly never imagined I would be treated horribly and struggling to get Rituxan for a second time.

Which brings me to writing this blog. Today I had my one month post-Rituxan checkup, and my platelets are holding strong at 236,000. Besides feeling relieved and victorious, I also became very emotional. It dawned on me that none of this would have been possible without Dr. Ahn. Had I been diagnosed after he retired, who knows where I would be (or if I would even be alive). He gave me the tools to fight this disease, fight for myself, and fight for a better world for patients like me.

I thought back to the scared 27 year old in his exam room, unsure of what the future holds. I felt like my life was spiraling out of control. I questioned if life was worth living, if my existence was a burden on everyone around me. I was beaten down and demoralized by my diagnosis, yet this man saw something in me that I could not see in myself. He saw the real me, the fire and determination that while stifled at the time, was under the surface. It just needed to be cultivated within my “new normal”.

I didn’t realize it, but Dr. Ahn took me under his wing and started teaching me about my disease, how the human body operates, and what he had learned over decades of studying platelet disorders. He sent me home to research treatments not because he didn’t want to treat me, but he wanted me to find the answer myself and come back to him. He was building up my confidence and repairing my broken spirit. I was being taught to think like a researcher, to put myself in Dr. Ahn’s shoes. Little did I know, five years later I would be using what he taught me to save my life again. Only this time, without him there to coach me.

If I had to describe what fighting ITP is like, I would liken it to walking a tightrope. Under Dr. Ahn, I had a safety net. My fear of falling was minimal because he was there to protect me. This time, it was like showing up and discovering you have a rope and a stick. No safety net and no one to help if you fall. Despite this, I stuck to what I knew- everything Dr. Ahn taught me.

Here I am in remission, again.

While Dr. Ahn treated my ITP, he also assisted me in rebuilding my life with a rare disease. I often joke that he created a monster, but truly I would not be where I am today if not for him. His lessons and mentorship helped me fight ITP in his absence. He gave me the confidence to walk the tightrope without a safety net.

I’m forever grateful that he believed in me, and saw my potential at the lowest point in my life. Every day is a gift, thanks to Dr. Ahn.

The Doctor Didn’t Listen

When I found out my platelets were below normal this year, I went through a variety of emotions, but absent was fear. I was in remission for four years and three months, surely I could do it again. After all, I was in the same hospital system seeing a colleague of my favorite doctor, I thought for sure it would be smooth sailing.

I was wrong.

I had to push for weekly CBCs, not being taken seriously about how quickly my platelets could crash. Two weeks later, I dropped from 129,000 to 53,000. I was put on Prednisone, the drug that destroyed my body and left lasting damage. The doctor assured me it was only a temporary treatment. I thought once ITP showed him what it was capable of, he would take what I say seriously.

Again, I was wrong.

My platelets started to rise, and stayed stable on high doses, but that drug was killing me. I kept asking my doctor about Rituxan, my life saver. The one drug that Dr. Ahn and I knew would put ITP into remission. It dangled in front of me like a carrot. He again refused to prescribe me Rituxan, he was perfectly fine leaving me on the drug that was destroying my body. The doctor kept saying, “Rituxan is not a benign treatment.”

I was in self-preservation mode at this point, getting worse as the weeks went by. I found my new physician, who knew Dr. Ahn and his research. I opened up to him about my desire to use Rituxan and he didn’t string me along. Instead, we created a plan based on how I wanted to fight ITP.

Rituxan worked, again.

I spent four weeks watching as the infusions started fighting back. I also was slowly feeling like myself, regaining my identity beyond just being “sick”.

After all of this, my emotions are still a bit raw. However, I am reminded that Dr. Ahn gave me the tools to continue to fight this disease well after he retired. It wasn’t just preparation for a single victory against ITP, but a lifetime of fighting back.

So here I am, in remission.

The doctor didn’t listen, so I found one who did.

Remission Accomplished

Left: Bloodwork two weeks after IVIg. Right: Bloodwork one week after my final Rituxan infusion.

My second attempt at repurposing Rituxan was a success. The last two weeks were busy and I was tired, so I decided not to blog.

It was incredible to see my platelets return to normal half way through the infusions. I continued with the IV steroids as part of my pre-medication routine, so I did not have any reactions. However, I had to ask for it every time so I will be requesting that this becomes a standard part of infusion prep when we start a trial. While they generally just give Benadryl and Tylenol to cancer patients using Rituxan because they need to gauge their reaction, this is unnecessary for ITP.

On the left is my bloodwork taken before my third infusion, on the right was my CBC before my final infusion. Huge difference in one week!

Overall, I am relieved that I am back in remission again. However, this was quite the physical and emotional rollercoaster. I am still tired, and likely will be for the next few weeks. I am trying to take a short nap every day to keep my energy up. I have noticed that the brain fog is completely gone, and I am very “clear headed” now. The purpura is gone, but I do have some darker spots where they once were. I am hoping they will go away in a few more weeks. My taste buds are still a little off, but not nearly as bad as before.

I’m still pretty burnt out emotionally, this was a hard fight and it took a lot out of me having to constantly advocate for every little thing. I certainly don’t want to go through this again in a few years when ITP comes back, so I am pushing to get Rituxan on label.

I’ll see the doctor again next month, and it will be interesting to see what my platelets are at. From there, we will decide how often I see him. I am looking forward to a break! I will be seeing a new Rheumatologist to address my Fibromyalgia and Sjögren’s Syndrome. However, I have noticed less joint pain since I finished my infusions. Perhaps some of that was B-cell related as well.

Infusion #2- It’s working!

The photo above shows my last two CBCs. I was at 94,000 platelets when I had my first Rituxan infusion on 7/1, and before my second infusion this past Monday (7/8), I went up to 100,000! I was killing off around 12,000 platelets a day before treatment, so this was extremely impressive.

I am feeling pretty good despite the mild headaches, constipation, fatigue, and lack of appetite. Those things really don’t bother me when I know my B-cells are being suppressed!

My next infusion is Monday, and I’ll have another CBC before treatment. I am curious to see where my platelets will be at the half-way mark. I’ve noticed my purpura fading, I haven’t developed any new bruises and I have no brain fog. So far, a night and day difference.

Some other good news: my nurse Jen was able to tap my wrist for the infusion, which saved me from having to hold my arm straight for a few hours. I also was administered 100mg of IV steroids in addition to Benadryl and Tylenol as pre-meds. I infused pretty quickly, from 10 am to 1:15pm, with no reaction. The only thing I don’t like about my wrist is the burning in my arm after Benadryl. It hurts for about 15 minutes and then subsides. I also have to be careful that I don’t let anything touch the area for 24 hours after, because it is tender. I usually wear my Apple Watch on my left wrist, but have to keep it off while the area heals.

That’s the update for now, I am happy with how things are progressing and ready for infusion three. #PlateletsUp

Infusion One in the Books

This is my “I’m finally getting Rituxan!” smile.

I’m home from my first Rituxan infusion, only three left to go!

While I’m smiling, I need to be brutally honest. Today was hard, the smile is put on. I’m tired, stressed, and I had another massive emotional rollercoaster caused by our broken healthcare system. I almost didn’t have my infusion today.

It was my first time taking an infusion at the elbow. While I had to focus on keeping my arm straight, it hurt a lot less and didn’t burn!

First, upon my arrival to the infusion center my authorization was not in the system. Instead of calling upstairs to get the information, they made me go upstairs to find the employee responsible. Why it couldn’t have been handled by a phone call I’ll never know. Just another part of the process where the systems and staff don’t seem to have an open line of communication. Insurance issues are handled in an entirely separate department and no one knows how to navigate it outside of a few people. Thankfully, the problem was fixed with a phone call down to the infusion center from the authorization staff.

When signing all the paperwork to do Rituxan last Tuesday, I had to take a pregnancy test to process the orders. It took my doctor a good ten minutes of navigating the EHR system to determine which of the tests was needed for Rituxan. At no point was a Hepatitis B panel shown, but apparently I needed that too. Unfortunately when I went to infuse today, my treatment was almost cancelled because I didn’t have the panel done. It wasn’t discovered until the Pharmacy was prepping my infusion. My amazing nurse got my doctor on the phone (he’s out on vacation) to override the testing requirement so we could get the prescription processing. She ended up drawing blood and everything came back fine.

At the time this all went down, I finally broke down and cried. This disease has been a constant source of unrelenting stress since March. I honestly never anticipated having this much trouble when ITP came back. Pair that with not sleeping and feeling generally miserable because of crashing platelets, I couldn’t hold in my frustration any longer.

While I tend to handle my disease with sarcasm, humor, and witty banter, I am still human with raw emotions. The system is not designed for rare disease patients, especially ones who are using off label therapies. We are forced to micromanage care, and anytime you look away, you risk someone else dropping the ball. Most of the time, those people are not the ones directly caring for you (like doctors or nurses), so it makes advocating for yourself much more difficult.

I absolutely hate having to be assertive, direct, and at times downright demanding to get what I need to battle ITP. I want to find creative solutions to the problems all of us rare disease patients face. Every time I’ve shared these bumps in the road, patients tell me their horror stories. I know we are not alone, but it seems like the system temporarily corrects itself for one of us, then goes right back to operating under broken processes. At the end of the day, this helps no one, because the cycle continues.

I hope that after all my infusions are finished and I am back in remission, I can work with payers, hospital systems, EHR companies, legislators and rare disease groups to fix a lot of these issues. There’s no sense in dismissing them once my treatments are over, because I will eventually be back in the system when I come out of remission again. I also want to turn negatives into positives. I want my challenges, problems, and pain points to help inspire change.

Now on to some interesting things about today’s infusion:

I had a reaction an hour in, complete with itchy eyes, throat and ears. I became flushed while my nose got stuffy. They had to stop the infusion and administer more Benadryl plus IV steroids. I had pre-meds of Benadryl and Tylenol, but no steroids. That’s likely what caused my allergic reaction (it was similar to what I experience with cats). For the next three infusions, I am going to ask for the steroids as pre-meds too.

My platelets went from 169,000 to 94,000 in 6 days. I am destroying them rapidly, so I am anxious to see how quickly Rituxan will work.

Food already tastes a little bland, but I’m hoping that will help me lose the 10lbs I gained from steroids!

Now that infusion one is finished, I am anxiously awaiting next Monday. Keeping my fingers crossed that the rest of the process is smooth from here on out. I appreciate the compassion shown by my nurse today. She went above and beyond to advocate for me when I felt defeated. She made it happen, and for that I am eternally grateful.

#PlateletsUp

My N-of-1 Odyssey

On Monday 7/1, I’ll be repurposing Rituxan for Immune Thrombocytopenia for a second time. The goal is to put me back into remission for an extended period of time. When I used Rituxan at the end of 2014, I had a remission period over four years. I’d like to think we will be able to achieve similar results this time around.

Rituxan is a chimeric monoclonal antibody targeted against the pan-B-cell marker CD20. In plain English, it targets B-cells by going after CD20 which is expressed on the surface. Ultimately, after multiple blood tests and positive results from immunosuppression by steroids, we were able to figure out my B-cells were responsible for destroying my platelets. By shutting them down using Rituxan, I am able to maintain a normal platelet count.

The first test I received is Anti-Nuclear Antibody (ANA) which came back positive and indicates autoimmune activity. We also tested for Lupus, Rheumatoid Arthritis, and Sjögren’s Syndrome among others. I ended up also testing positive for SS-B antibodies, further confirming autoimmune activity. Another great marker: I respond very well to immune suppression with Prednisone. All of these factors collectively helped my doctor come to the conclusion that I had chronic Immune Thrombocytopenia and I was destroying platelets via my B-cells. I never had a bone marrow biopsy because when I am on high doses of Prednisone, my platelet count returns to normal. There is nothing to indicate that I have any issues with platelet production in my bone marrow.

Because of this, N-Plate and Promacta would not be the right treatment for me. I could stimulate platelet production, but my B-cells would destroy those platelets as well. Ultimately, the idea is to stop the process of platelet destruction. That’s how I came to the decision to use Rituxan in 2014, and why I advocated so hard to use it again this time.

So now I begin the process of making myself an N-of-1 example of how Rituxan can help ITP patients with B-cell led destruction of platelets. We will collect a CBC before each of the four infusions to track progress. I am currently on no medication for my platelets after I finished my Dexamethasone pulse last Monday (one week before my first infusion).

Besides chasing remission for a second time, I had an inordinate amount of trouble getting insurance to approve this treatment. We must see to it that Rituxan become an on-label therapy for Immune Thrombocytopenia, especially since patients with B-cell led destruction of their platelets do not have an FDA-approved treatment.

Acknowledging the clinical diversity of ITP is a must, especially with multiple drugs on the market and more in development. There is no one size fits all treatment for ITP, and what works for me may not work for another patient. Gathering treatment data and comparing it with ANA tests will be a great way to dissect some potential remission patterns in patients.

Calluses From Living Rare

Last week, I was forced to reflect on the last five years of my life. The diagnosis, the treatments, the risks, and reward of remission for over four years. The heartbreaks that came from losing people (and my dog) to things I could not save them from, no matter how hard I tried. It was the first time I told my story from start to finish. It was also the beginning of my self realization that I am a completely different person from who I was on April 30th, 2014, the day before my D-day (diagnosis day). 

Before ITP, I was a tough chick with a no nonsense attitude. But I had no calluses. That came to an abrupt halt on May 1st, 2014. Baptism by fire, taking on a dysfunctional body in a broken system that does little to help patients like me. There was no soft introduction into the world of rare diseases, I was simply dropped into the pit and expected to sink or swim.

I was forced to become my own advocate, to research, absorb, digest, and become the expert on ITP. My view of physicians changed from putting my implicit trust in them, to questioning everything. I no longer wore rose colored glasses when it came to my views on healthcare. I was being raked over hot coals, and that’s how I developed my calluses. 

There is a certain level of pressure to be gentle, warm, and kind as a patient. I’d like to think I have retained those qualities over the last five years, but they’re not as apparent now. It’s hidden underneath a hardened exterior, one I had no control over developing because I needed it for survival. It builds through every bump in the road, side effects from treatments, disappointing lab results, and just the overall way life can seem to come down on you during difficult times.

To every rare disease patient who takes time to reflect on their patient odyssey, don’t beat yourself up over a hardened exterior. While we do our best to navigate a system that constantly throws us curveballs, a roughed up exterior is part of the package. Wear your calluses with pride, you earned them like your zebra stripes.