Maximize Your ROI Working With Patients

No question the hot topic in healthcare is utilizing patient insights in various projects. Whether you’re building a tech platform or developing a new therapy, collaborating with patients is a must. The problem is, where do you find the right patients to work with? How do you locate the correct insights that add value to your company? While it may seem you just need to consult with your target audience, the answer is not that simple.

Modern Problems Require Modern Solutions

Acquiring patient insights should be treated with the same level of care and involvement as any other project requiring the input of subject matter experts. That being said, finding the right subject matter expert is critical.

From a lawyer’s perspective, you wouldn’t bring a plumber in to testify as an expert witness in a medical malpractice lawsuit. So why would you bring in just any patient to evaluate your project?

I’ve been involved with enough projects that I could author a book on horror stories involving patient insights gone wrong. Unfortunately, NDAs prevent me from sharing these stories in great detail. So let’s dive in to some of the major mistakes I’ve seen over the last few years, and how your organization can avoid them.

Patients don’t understand the goal(s): Focus groups, advisory boards and interviews should have distinct endpoints for patients. This means outlining the topics of discussion and what your organization hopes to learn. I have sat in far too many advisory board meetings where the patients are confused by what the meeting organizer is asking. In the end, everyone is frustrated, time and money have been wasted with very little actionable information gathered. A simple “table of contents” for the meeting goes a long way.
Meetings with no breaks: No one wants to sit around talking about their frustrations with healthcare for four hours without a break. Has that happened? More times than I can count. Never ignore the power of a break. It keeps everyone engaged and focused. Side note: providing snacks is a huge bonus but be sure to clear any dietary restrictions with patients ahead of time.
No compensation: You wouldn’t dare not pay your consultants for their services, so why would you treat patients differently? I am shocked at certain companies who put together patient advisory boards and refuse to pay them. Meanwhile, employees who work with these “volunteer” advisory committees are posting about their company expense accounts on Instagram (you know who you are). Not only is that poor form, it makes it seem like you don’t value patient input. When you do this, you miss out on the most valuable feedback, the kind that can help you scale projects and receive great ROI. Like the old saying goes, you get what you pay for.
Bringing in the wrong patients: Patients who get into arguments about which pharmaceutical company they hate more, talk over others, dominate the conversation, or just like to hear themselves talk. I could write blogs every day for a year just on this. It is critical to have a vetting process before selecting patients for certain projects. One on one interviews don’t require much vetting, but putting together an in person meeting or advisory committee where groups of patients will be working together warrants more attention. As a patient who also participates in these projects, it is a major headache. One rogue person can throw off the entire meeting and impede on the amount of information you collect. It is critical that you throughly screen potential participants and pay attention for any signs that there may be issues with some of the behaviors listed above.
Confusing professional patients with “Patient Professionals“: Perhaps they work in healthcare, own a business, work in marketing, or practice law (shout out to my fellow lawyers out there battling chronic conditions). These are the patients who are balancing their “new normal” living with a health condition while participating in society. They are a gold mine of knowledge, experience, and fantastic analytical skills. These are the people I reach out to when I am putting together a team on behalf of a client. “Patient Professionals” not only share their perspectives living with and managing a condition, they can also contribute their expertise gathered from their career field. For certain initiatives, companies need to seek out these superstars and work with them.

The overall message here is that depending on your project and your goals, you have to find the right patients to work with. Too often I see companies rush to put something together, and they fail to get the feedback they need to move forward. Don’t make these mistakes, and you are on your way to obtaining great ROI working with patients.

Sinkholes, Not Silos

At every conference, someone is talking about Pharma’s “silo” problem. Information, ideas, projects, all trapped within a small area just waiting for someone to come along and break them out. Patients hear about this all the time, and are often asked for input on how to help break down those silos.

The problem is not silos, it’s sinkholes.

For patients like myself, the quest for answers to manage and overcome our conditions goes well beyond just Pharma. It starts at cell line research, moves to translational models, then finally arrives to those Pharma silos. Every step in this process is a deep abyss, void of any guide to climb out of each hole to reach the next.

For patients and caregivers pursuing treatments, these sinkholes not only appear bottomless, they’re a behemoth. Understanding the complexities of each one, exploring and tackling all the issues before moving on, while avoiding costly mistakes along the way is overwhelming (to put it mildly). However, this is the road we patients and caregivers must take to find treatments and cures for our diseases. If tackling the silos in Pharma is the focus, most of us will die well before we ever get to human trials for drug candidates.

For patient organizations driving drug development, building bridges over these sinkholes to connect the dots is critical. Time is money, and rare disease groups struggle to reach fundraising goals. Not every disease has a celebrity parent or patient who can attract big donations to sustain programming. Few conditions are “large” enough in the rare community to have groups that can divide and conquer. So any setback can be fatal, and with a race against time to find life-saving treatments, no one has time to sit around discuss how to build a bridge.

So why sinkholes? Simple, the hard work conducted in various stages of drug development end up entrenched within groups and companies. As time goes on, it becomes difficult to relocate that knowledge and product across a bridge. The burden falls on patients and caregivers to make this happen, but they usually fall short of understanding the complexities of each of these sinkholes, and have limited time to invest in mastering them while managing the complexities of living with a rare disease. The problems get deeper and deeper as time goes on, so calling it a silo does not do it justice.

I tell my non-profit clients the best way to overcome these obstacles is to create educational programming for disease communities, and for my industry side- fund educational programing through disease-specific non-profits. Each group has varying levels of understanding on the drug development process, so there is no “one size fits all” system that can work across the board. Individually tailoring these initiatives pays dividends, but you have to do them right. Patient and caregiver communities are a driving force in drug development, so it is wise to utilize their advocacy throughout clinical development.

My prediction is over the next decade we will see a shift in how patient groups work with industry to defeat the sinkhole problems that plague their communities. We will also see industry become more focused on building lasting relationships to make this happen, which in turn will help with clinical trial design, recruitment, and retention.

Finally Found My New Normal

My mother, Judge Diaz and I after being officially sworn in.

I apologize for yet another lengthy absence from my blog. I’ve been diverting all my energy into a few big projects lately. Most notably studying for the Florida Bar Exam. If you know anything about the test, you have probably heard just how stressful it is! My last blog post was right after the exam. I needed a break from blogging.

I’m so excited to announce that I passed the bar exam and was sworn in on April 16th! It was a crazy morning, I barely slept the night before, Sjögren’s was causing a nasty flare, and I was nervous. When I saw I passed, I cried! It was great to be surrounded by friends, my professors and my family during the ceremony.

Since then, I have been busy establishing my new normal. I had the chance to chat with Marco Rubio and thank him for co-sponsoring the OPEN Act. He is enthusiastic about getting it passed, the next time I am in DC, we are going to make sure everyone is on the same page strategically to get it done.

As for my work- I continue to consult with pharmaceutical and biotech companies on various projects, ranging from patient advocacy plans to legislative strategy. I am excited to be using my legal education to continue the pursuit of making the world a better place for rare disease patients.

I look forward to rebuilding my life after all of these great things, and continuing the push forward. Thank you for being a part of my journey.

The Struggle of Being Disabled in Law School

Discrimination and bullying of disabled students is rampant in colleges and universities. Now that I have a full year of law school under my belt since being diagnosed with a rare disease and three other chronic conditions, I connect with those who have had similar experiences. I refuse to be silent about “administrators” who torment students with invisible disabilities because they are ignorant. It is obvious that they disregard the Americans With Disabilities Act and university policies with their outrageous and targeted discriminatory behavior. It is an epidemic for the largest minority in the United States. Continue reading →

Industry: Friend, Not Foe

There has been a great deal of criticism of pharmaceutical and biotechnology companies in recent months. Most of the anger surrounds drug pricing, and many in the rare disease community have participated. While affordable healthcare is a priority and we need access, an even more alarming need is actually treatments for orphan disorders. Many rare disease patients are struggling day to day, without anything to help them manage their symptoms and conditions. Drugs for small populations cost money, the FDA is particularly critical of rare disease drugs making investments risky and the cost of treatments sky rocket. Continue reading →

I’m Not Sorry, I Hate My Body

It is no secret that since I became sick and was stuck on prednisone for months that I began to hate my body. After all, I’m up about 30 pounds from where I was nearly two years ago and I lost a lot of muscle that I had worked so hard to gain. I never acknowledged to myself how much I hate my body until I was out for a walk tonight. My hatred isn’t just for the weight gain or loss of muscle. It comes from a feeling of betrayal: my body tried to kill me. My immune system malfunctioned and for some miraculous reason, I survived months of physically challenging work in heels without internally bleeding to death.

Continue reading →

Rare Disease Week 2016

I am headed up to Washington, DC on Sunday for Rare Disease Week. I am excited to be able to meet with a lot of different groups to work on legislation for the 30 million of us fighting these disorders.

Last year was about me, I was focused on what I went through. This year is about YOU, the patient, the caregiver, the healthcare provider, the researcher. Now we have to work on unity between everyone involved in the rare disease space. We need to see to it that the 21st Century Cures bill is signed into law as soon as possible.

I expect this trip to be quite different, as I have found myself at peace with what life has thrown at me.

If you’re going to be in DC and at the events, I would love to meet you. Tweet me @RareCandace and let me know what you’re up to.

Science, Not Statistics

I’m alive today because I relied on scientific research to find a treatment for my rare disease. Without the drug I used off-label, I wouldn’t be alive today. My internal bleeding was becoming progressively worse, and it was only a matter of time before I faced a situation that could have had a terrible outcome.

So why am I writing about science? Because today the rare disease community, our families and friends need to unite to send a message to the FDA. Next Friday is an important meeting for the Duchenne Muscular Dystrophy (DMD) community as Sarepta’s Eteplirsen is up for review. This drug has the potential to save many lives.

I have seen first hand via social media just how powerful Eteplirsen is. With DMD, boys are not able to walk once they reach their teenage years. Rare family members have gifted us with videos and photos of their sons who are participating in the Eteplirsen clinical trials walking, playing, and just being normal kids. They’re not losing abilities like the children who aren’t receiving the drug. While it is heartwarming to see improvements, I am constantly reminded that other families are waiting while their children are losing strength. Time is of the essence, Duchenne Muscular Dystrophy is not waiting for FDA approval of Eteplirsen. Just about every week my timeline lights up in remembrance of another son lost. How many more of our boys have to die before the FDA approves Eteplirsen?

There are no safety issues with Eteplirsen, I want to highlight that. The FDA has taken issue with statistical data. Let me explain to you why this enrages me, and why I see this as an attack on our entire rare family, all 30 million of us.

In October 2014, there was no statistical data to tell me that Rituxan would put my ITP in remission. In fact, if I just analyzed raw data, I had less than a 50% chance of it working. Why? Because Rituxan was being used on an entire ITP population, not those who had platelet destruction issues which the CD-20 inhibitor would address. Instead, I used patient data- meaning I analyzed what those who had successful Rituxan treatments had in common. I found the common links on our blood work, how we responded to other treatments, what our doctors thought, even how we bled. I researched the science behind how Rituxan works, what exactly it does to the body and how that relates to the destruction of my platelets. I went in to my doctor and I asked for Rituxan. I knew it was my only hope, I knew my life depended on it. And I was certain that with my research, I had an excellent chance of success.

Rituxan saved my life, but it was NOT a miracle, it was science.

Eteplirsen is to Duchenne Muscular Dystrophy what Rituxan is to Immune Thrombocytopenia. It isn’t about statistics because these treatments aren’t meant for everyone and you cannot apply mass-market measures to orphan drugs for a complete analysis. You cannot expect small patient populations to produce statistic data sets that rival those of drug trials for diseases like diabetes or COPD.

Success for orphan drugs lies in science. It is in the photos and videos of 15 year old Billy (above with Pluto), who still has the ability to walk. This wouldn’t be possible without Eteplirsen.

Billy is living proof that Eteplirsen works. He is the walking scientific evidence of a safe and effective treatment for Duchenne Muscular Dystrophy.

So friends, I ask that you take a few seconds to fill out this short form that will request members of Congress to sign a letter urging the FDA to apply the FDASIA tools to Duchenne which would allow for an accelerated approval of Eteplirsen. This historic moment will set the stage for future orphan drug development, so your participation is critical.

Parent Project Muscular Dystrophy has a short form you fill out with your name and address, and they will send the letter to your representative for you.

Let’s end Duchenne.

Tis The Season for Action!

Kona wanted to make a special holiday appearance in honor of our call to action for the Senate over the 21st Century Cures initiative. I sincerely hope that when I arrive in DC in a few weeks, progress will have been made. There is no time to waste when 30 million Americans depend on Congress to take action.

My blog is a road map of my rare disease journey, starting last year when I decided to take a chance and use Rituxan off label. It is not approved for ITP patients. Why? Because we don’t have enough information and research on ITP to conduct a comprehensive clinical trial. One of my goals for 2016 is to change that. Through my research, I have been able to isolate a population of ITP patients who can and will benefit from use of the drug. Simply put, this drug is a life saver and the people who achieve remission from it enjoy a few years of a fairly normal life. There is absolutely no reason why this should be denied to people like myself.

Tomorrow is December 18th, which happens to be the one year anniversary of my remission. I truly believe this gift was bestowed upon me to give me a chance to fight for others. I have lost a few friends in the past year to ITP, it doesn’t get any easier and it makes me realize just how fortunate I am.

I’ve spent the last 365 building my new normal, now it is time to change medicine.

Chronic Illness Gift Guide 2015

Since being diagnosed with fibromyalgia right before my birthday, conserving spoons has become a priority. About this time last year, I wrote a gift guide for prednisone. Now I want to compile a list of gift ideas for people who suffer from chronic illnesses and fatigue. These gifts will help out that spoonie in your life! Continue reading →

Rare Candace

An unconventional disruption of rare disease.

Tag Archives: Rare Disease