Patient Input Is A Must — Even Under Time And Money Constraints

A conversation with Stacie J. Bell, Ph.D., chief clinical research officer of Lupus Therapeutics, the clinical affiliate of the Lupus Research Alliance

Patient advocacy can take many forms. For decades, community fundraisers and awareness walks have gained and retained traction in the public sphere. Now, their existence has given rise to advocacy that directly engages patients with drug developers, with the intention of better understanding the patient experience and developing drugs that meet patients’ needs.

While such engagement is logical, it’s not always commonplace. If it were, patient advocacy groups wouldn’t still be sitting on conference panels or penning editorials reminding, imploring even, pharmaceutical companies to work with them.

To explore the capabilities of patient advocacy groups and their positive impacts on drug development, Clinical Leader heard from Stacie J. Bell, Ph.D., chief clinical research officer of Lupus Therapeutics, the clinical affiliate of the Lupus Research Alliance.

How are patient advocacy groups suited to helping pharma companies develop drugs and design trials that better meet patients’ needs?

When patient advocacy or nonprofit organizations initially started, they took many forms. They may have been the local community walk, a donation to the local food bank, or a patient living with a condition that gave that condition a voice and a face for greater awareness.

Over the years, these advocacy groups have become a key stakeholder in public and governmental advocacy, speaking to that lived experience in a way that might change governmental and hospital policies. A structured approach has been developed such that those individuals can have formal input, whether it is partnering with biopharmaceutical companies to review their clinical trial materials or their protocols or having a one-to-one direct connection to share their experience with development teams trying to bring new therapies forward that address those unmet needs.

The Lupus Research Alliance and the experience we have within helps ensure that the research is funded, progressed, and advanced. Lupus Research Alliance has a clinical affiliate, Lupus Therapeutics, that can have direct input on those clinical trials by engaging the patients directly and linking them to the drug developers, investigators, and research teams conducting the clinical trials.

I'm curious about the structure of Lupus Research Alliance and Lupus Therapeutics. How do they work together?

Back in 2016, when the Lupus Research Alliance was formed from a merger of two prior organizations, it was very quickly recognized that one of the biggest challenges in lupus drug development is the number of clinical trial failures. And many of those failures were a result of having incorrect participants, the clinical trial design, and, in some cases, the timing of assessment. So, the Lupus Research Alliance formed a clinical trial network called the Lupus Clinical Investigators Network (LuCIN).

It's a unique model, and the Lupus Research Alliance provides not only financial support but infrastructure and training support for clinical trial centers to help them run the best clinical trials, accelerate timelines, and ensure representative participation.

After the LuCIN network was formed by the Lupus Research Alliance, it became very clear that it would be beneficial to have an organization overseeing all these activities within the network and a way for the organization as a whole to also partner with the biopharmaceutical industry.

So, in 2018, Lupus Therapeutics was formed as the fully owned clinical affiliate of the Lupus Research Alliance, really a parent-child type of format. We have some shared capabilities, but we function independently, and we each have our own board of directors. We have our own contractual arrangements with our biopharmaceutical partners, so we can keep that clinical expertise compliantly and effectively moving these treatment options forward.

Within Lupus Therapeutics, we are specifically trained in clinical trial development as well as patient engagement. We truly weave all those capabilities together, pulling that thread through for the overall vision and mission of the Lupus Research Alliance on the clinical part of the research continuum.

What does patient advocacy look like in practice? Can you give an example of when a group has helped change a protocol or impacted a trial in other ways?

Say that you have Company A, and they've got a new potential lupus therapy that they think has promise for the lupus community. They will reach out to Lupus Therapeutics and say, “We've done some initial work in clinical trials, but we'd like to move into a Phase 2 or a proof-of-concept trial. We would really like to have a better understanding of the unmet needs in the community, hear from patients about this. We've got a draft protocol right now. What could you do to partner with us and assist us?” First and foremost, we have protocol review committees both on the key opinion leader side and the patient side, a group trained specifically to review protocols, synopses, and informed consent forms.

They will sit down together, go through documents, proposed outcome measures, and proposed procedures to ensure that it can be recruited for and executed without being too burdensome on the patients. We also have patient visits where patients meet virtually or in person with the company to tell them why it's important that they are including these types of things in the clinical trial or in their development plan, or in how they are thinking about how they’re taking their program forward.

We also have patients who serve as peer mentors, either through peer-to-peer education before they would sign up for a trial or during the trial. That is called our Patient Advocates for Lupus Studies program, or PALS. As part of the public-private partnership, Lupus Accelerating Breakthroughs Consortium (Lupus ABC) founded and overseen by the Lupus Research Alliance, there’s also the Lupus Voices Council, where patients can work directly with the FDA. That voice has a constant feedback loop so that patient communication is constantly present.

I'm curious about the impact of PALS in patient recruitment, retention, and engagement throughout a trial. What role does that play?

A huge role, actually. The PALS program itself was formally evaluated, and we were able to show in the pilot setting that there was a positive impact on trust and desire or willingness to participate in clinical research.

We've evolved the PALS program into a centralized model so we can have greater reach to more sites and more patient communities, depending on or independent of their geographic location. In addition, educational programs have proved to help patient understanding through pre- and post-surveys. But the biggest thing is just talking to those participants who went through some of these programs and seeing their desire to be a greater part of clinical research, not only for their benefit but to help others living with their condition.

There is other public data available on patient advocacy organizations having peer-to-peer support like this, and in all cases, it's shown positive impacts on individuals’ willingness to participate in clinical research.

For a pharmaceutical company that wants to engage with a patient advocacy group that maybe hasn't done a great job of it before, but really knows the importance of it, what are some of your recommendations for getting started?

The earlier the better is what I'll say. If you're starting a development program, even before it goes into the clinic, that lived experience and understanding of the patient's journey is imperative to success.

For any pharmaceutical company, learn your therapeutic area, and get to know the patient advocacy organizations that are present and what their areas of focus are. Some patient advocacy organizations focus more on bringing the patient's voice forward. Or there may be organizations like the Lupus Research Alliance, that are one-stop shops where they have research funding, education, trial involvement, opportunities for patients to interface directly with developers, and more. A company should understand its therapeutic landscape and get to know what patient advocacy organizations are present both in the United States and beyond.

There are easy ways to do that. All the patient advocacy organizations have websites. From that, I would encourage a company to reach out to the organization. Almost all of the patient advocacy organizations have an information request contact. Reach out with interest. A company can share limited details if they want to keep things confidential, but they're still able to engage with the patient advocacy organization and understand their capabilities.

With Lupus Therapeutics, it doesn't matter if it's a small company reaching out to us or a very large pharma company. We can work with them and find ways to support their programs in advancement, and most importantly, support our patient community. If it's a very small company, we're going to augment a lot of the capabilities they don't have. With big pharma, they have a lot of capabilities and financial resources, but they're not uniquely positioned to really reach the community in such a trusted way. The earlier and the more those conversations and interactions happen, the more successful both can be in supporting the community.

How would you finish this sentence? I love it when a pharmaceutical company asks or requests _____.

How we can keep the patient at the center no matter what. Even from the standpoint of the Lupus Research Alliance, we have the expertise through our key opinion leaders and within the organization to support the scientific and clinical aspects of the program. But if the biopharmaceutical company is asking at the beginning how to keep the patients front and center at the heart of everything they do, everything else will follow.

Conversely, “I really wish pharmaceutical companies would ______.” What's a shortcoming that you noticed?

I understand the pressures that they're facing. Their drug development is very expensive, and so time is money for them. Sometimes, companies, especially smaller companies, may be financially strapped or have tight time commitments. They want to move forward as fast as they can, sometimes discounting the role that a patient advocacy organization could have or thinking that implementing the patient's voice may slow things down or be cost-prohibitive.

Companies assuming that they're not going to have the time or money to take those very critical steps, I would ask them to very carefully reconsider because those things can still happen and not impede their development programs.

About The Author:

An accomplished research scientist and organizational leader, Stacie Bell, Ph.D., came to Lupus Therapeutics with more than 25 years of experience in discovery research, clinical development, and patient engagement in the biopharmaceutical and nonprofit arenas. Prior to joining Lupus Therapeutics, she formed and served as CEO and president of the Global Nutrition Project and was previously the chief scientific and medical officer, leading the research and medical affairs department at the National Psoriasis Foundation. Bell began her career as a graduate research associate in the Department of Biochemistry at Vanderbilt University School of Medicine in Nashville, TN, where she earned her Ph.D. in biochemistry. She graduated summa cum laude with a BS in chemistry from the University of North Dakota.