Guest Column | February 21, 2019

How An Advocacy Group Elevates The Patient Voice In Research And Clinical Trials

By Stevan W. Gibson, president and CEO, Lupus Foundation of America


In medical product development, the harsh reality is that failure is the overwhelming norm. Over the past several decades the lupus community has seen promising treatments face this reality. There has been only one treatment designed specifically to treat lupus approved by the FDA in the last 50 years: belimumab.

But this is not a reflection of the advocates, patient organizations, healthcare professionals, researchers, or biopharmaceutical companies that work tirelessly each day to solve this disease. The drug development process and landscape are complicated, and the incredible work of all these stakeholders has created a new reality where there is more investment in lupus drug development than ever before, bringing renewed hope that these investments will expand our knowledge of the disease and how it’s treated.

A key reason for this renewed hope is the patient voice, a factor that is even more critical in lupus because the disease impacts each person differently. Over the last few years, we have seen stakeholders across the healthcare community increasingly work to meaningfully incorporate the patient perspective into drug development. Patient engagement is only valuable if it’s meaningful; for us that means looking to a leader in this area, the National Health Council (NHC). The NHC defines patient engagement as “Direct relationships and partnerships that are bidirectional, reciprocal, and continuous. Communications are open, honest, and clear. Engagement goals, participants, methods, desired impacts, and actual impacts are clearly outlined and transparent.”

Making The Patient Voice Heard On The Hill

In recent years, we’ve seen the federal government get more involved in creating initiatives to elevate the patient voice. In 2012, the FDA launched its patient-focused drug development (PFDD) initiative. The PFDD initiative is grounded in a simple belief: that the people living with a condition are experts and must be involved in the development of therapies. The FDA called for PFDD meetings to more systematically gather patients’ perspectives on their condition and therapies available to them. As the Lupus Foundation of America knows well, people living with lupus are the experts and have a unique understanding of the disease and what outcomes are most important in developing a treatment. Nonprofit organizations like ours, the biopharmaceutical industry, and other stakeholders are embracing this effort to elevate the patient voice.

While we are excited to see the patient voice incorporated more and more into drug development, it’s still a growing initiative with many layers and complexities. One of these layers our foundation finds very important is advocacy. Advocacy plays an important role in making the patient voice heard on Capitol Hill in order to establish federal funding for research and to advance policies that will break down barriers and bring more effective treatments to market. On March 4 and 5, 2019, more than 300 lupus advocates will make the trip to Washington, D.C., for the 2019 National Lupus Advocacy Summit, and more than 5,000 are expected to participate online, to raise their voices on Capitol Hill in support of policies to improve the lives of people affected by lupus.

Our advocacy efforts, and those of other organizations for chronic illnesses and diseases, have made an impact. One example of this is our advocacy, along with other organizations, for the historic legislation in the 21st Century Cures Act and the Prescription Drug User Fee Act (PDUFA) VI. Both became law, and some of the most important provisions within each elevated the patient voice by expanding the aforementioned FDA PFDD initiative.

In addition to advocating for PFDD, our team partnered to host an externally-led PFDD meeting along with the Lupus and Allied Diseases Association and the Lupus Research Alliance. At this meeting, members of the lupus community shared with the FDA their perspectives on living with the disease. Prior to the meeting, the organizations used a survey (available in both English and Spanish) to solicit input from people with lupus about how the disease affects their lives. More than 2,000 people responded to the survey and provided their perspective on the burdens of living with the disease, issues with currently available treatments, attitudes toward participating in clinical trials, and more. There were 300 people in attendance at the meeting, and hundreds of others joined online to watch the live webcast. In addition to people living with lupus, those participating included more than 20 representatives from the National Institutes of Health (NIH) and the FDA, including key officials across different FDA centers and divisions, and 60 representatives from more than 15 pharmaceutical companies. The subsequent report from the meeting was shared with the FDA, industry, and other key stakeholders throughout the lupus community.

The PFDD meeting focused on lupus was a huge success, but we know that these efforts must continue in order to ensure the voice of people living with lupus is central to medical product development. Since 2012, the FDA has held or participated in 34 PFDD meetings focused on a variety of diseases and conditions. These meetings have been important as the PFDD program evolves and the idea of meaningfully engaging patients continues forward.

Our work doesn’t stop here; we are continuing to put patients first and make sure their voices are considered at every step of the drug development and treatment process. In fact, the foundation has launched several programs that further elevate the patient voice in drug development and the regulatory process. These programs aim to:

  1. Improve legislative and regulatory policies to enhance the patient perspective
    • One part of the FDA PFDD initiative is for the agency to produce a series of guidance documents to help clarify how patient data can be collected and used to improve regulatory decision-making and drug development.
    • People living with lupus will have different perspectives/views from people living with other chronic conditions and that is why it is so critical that the FDA fully understands the experiences of people living with lupus and their family caregivers. The foundation is working with a variety of stakeholders, including people living with lupus, to provide the lupus communities’ perspective as the FDA develops the guidance documents.
  2. Improve data collection
    • Two years ago, the Lupus Foundation of America launched a patient registry and is currently working to add a component to the registry that will collect information aligned with the FDA’s PFDD initiative. When complete, people living with lupus across the country and the world will have the ability to contribute their real-world experiences to help researchers, drug developers, and regulators to better understand the disease and the patient perspective. This vital source of data is another way the foundation is accelerating the discovery, development, and delivery of new treatments.  

Progress In The Pharmaceutical Industry

In addition to working with government to elevate the patient voice, we also find it critical to work with the pharmaceutical industry to do the same throughout the drug development pipeline.

At the Lupus Foundation of America (LFA), we are committed to increasing funding for research and accelerating the development of new treatments while ensuring a focus on putting the patient first. To that end, we have launched a number of programs helping to set the standard in elevating the patient voice in research and clinical trials. One such initiative is the Rapid Evaluation of Activity in Lupus, LFA REAL. This is a first-of-its-kind, easy-to-use tool to monitor lupus disease activity. The system fills an unmet need in lupus clinical care and clinical trials, as it not only incorporates the clinical perspective but also highlights patient concerns and priorities. The program is currently being utilized in a global Phase 3 clinical trial by Janssen Pharmaceuticals, Inc. Our team also co-authored a groundbreaking white paper that proposed actionable solutions to address critical issues that have stymied lupus clinical trials. This white paper was recently presented to top decision makers at the FDA.

Today, there is so much promise that new treatments will be found, and we believe that the chance of this happening will greatly increase as the voices of people living with lupus and other diseases are elevated. The Lupus Foundation of America has helped to usher in an era that empowers people living with lupus and their families to meaningfully contribute to the research and development of new treatments. We hope these efforts will aid other patient advocacy organizations, pharmaceutical companies, and regulators looking to elevate the patient voice in more innovative ways.

About The Author:

Stevan W. Gibson is president and CEO of the Lupus Foundation of America, overseeing the senior management of the organization, focusing on the foundation’s growth and strategic framework, and carrying out its mission through research, advocacy, care services, and development. Gibson is committed to accelerating the research pathway toward better treatments and an end for lupus while also raising the profile of lupus on the nation’s health agenda through government advocacy and awareness initiatives. Prior to joining the foundation, Gibson spent nearly 20 years with The ALS Association (ALSA). He also has experience consulting with global corporations and serving on national and governmental organizations.