COVID-19 Vaccine Incentives vs Clinical Trial Reimbursement

This week, former congressman John Delaney (D-Maryland) proposed that $1500 stimulus checks be issued to people who get the COVID-19 vaccine. While I have been yelling from roof tops about patients who cannot get vaccines for medical reasons (and those reasons include ICU visits and/or death), this hit another point that has been equally as aggravating for many years. As an attorney, I am always questioning why biopharma companies refuse to cover the out of pocket expenses for patients participating in clinical trials. These expenses include things like gas, tolls, parking fees, childcare, and meals.

I have been told by people in legal/compliance that this is due to those reimbursements being seen as a form of “payment” that could influence a patient’s decision to participate in a trial. Of course, that sounds pretty ridiculous to most patients, we chat with our friends who have participated in trials and hear expenses being covered. The lack of consistency is not only annoying, it highlights a major problem with trial design on the industry side. Perhaps it can be attributed to a lack of meaningful patient engagement early in the drug development process, or a budgeting oversight. Either way, it is totally unacceptable and truly hurts creating inclusive trials.

So back to the stimulus check tied to the COVID-19 vaccine, it is maddening to see this idea being celebrated while patients participating in clinical trials are expected to foot the bill. I strongly believe it is time to have a conversation with patients, industry, and regulators about clearly defining reimbursement for trial participants, especially now that we have expanded our focus on diversity and inclusion in clinical trials. Far too often I hear stories of families struggling to afford childcare, balance schedules, manage school assignments, and taking care of pets while receiving an experimental therapy. Despite Sponsors and CROs collaborating with patient groups more often, we are still far off from truly addressing the needs of patients and families participating in clinical research. How is making a stimulus check contingent on getting a vaccine better than reimbursing costs associated with participating in clinical trials? Why would a stimulus check not be viewed in this manner as a “influencer” for participation, but covering the parking fee at a trial site is forbidden?

As we continue to see COVID-19 shift healthcare, it is important that patients push to see the same advancements, innovation, and sense of urgency occur within their communities. This story struck me as one area where we really need to be doing more to help patients and families participate in clinical trials. Financial burdens prevent participation, can cause patients to drop out of trials, and slow down enrollment. Time is money for industry, and time is of the essence for patients.

Let’s cover the costs associated with participation in clinical trials and stop pointing fingers for not doing so, patients know better.

The Harmful Embrace of “One Size Fit All” in Drug Development

In light of the news coming out from NurOwn this morning, it’s clear that the rare disease community needs a wake up call. 

NurOwn has shown great results in ALS patients with less-advanced disease. It’s important to note that disease progression for any condition varies from patient to patient. In addition, patients with more advanced ALS were also included in the trial. So the varying disease states absolutely play a role in how the data appears at the end of the trial. But instead of looking at the overall picture, why are we not looking closely at the group that had a “statistically significant” response to NurOwn? 

The rare disease community needs to stop thinking there is a “one size fits all” approach to drug development. We always complain about Pharma silos, and how it’s so hard to work with industry. Well, it’s hard to work with patient groups and the rare disease community, too. We create silos, letting our rare brothers and sisters fight alone. 


I can tell you from personal experience that advocacy groups who think this way end up hurting patients. A decision made in the mid-2000s on Rituxan still hurts myself and other patients like me. I do not have guaranteed access to my chemotherapy drug that stops my immune system from killing my platelets. It is an “off-label” therapy, which means my doctor, the hospital that administers it, and my insurance company all have to agree to dispense it.

All of this could have been prevented by conducting a clinical trial and working on identifying the ITP patients who would likely respond (something I did myself in 2014 when I made the decision to use the therapy). However, that did not happen. Why? Since not ALL Immune Thrombocytopenia patients responded, no one felt it was a worthy endeavor. It left patients like me to struggle and fight based on an arbitrary assessment from industry and a patient group that was suppose to represent every ITP patient. They didn’t represent all of us, people like me were left out. We are hurt by this mistaken belief that drugs to treat our rare diseases are only worthy under a “one size fits all” approach.

The “data” and “charts” looking at Rituxan use in ITP patients wouldn’t look good if you looked at EVERYONE who was given the drug. But if you looked at the patients who fit certain criteria, you would see an AMAZING response with a certain group of ITP patients. That’s where we are with ALS right now, and it’s where we will be in the months/years to come as more orphan drugs go through the FDA. You can’t just read the headlines of an article about clinical trial data to get the total picture. Unfortunately, this happens in disease groups and people quickly dismiss promising therapies. It has to stop, the rare disease community is better than this.

I sincerely hope the ALS community pushes for approval of NurOwn and applies pressure to the FDA.

I look forward to lending my voice to this fight, because all rare diseases need to support each other.

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.

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?