Patient advocacy groups ‘critical’ in rheumatology drug development, advancing transparency
The world of drug development and commercialization relies heavily on industry stakeholders, as well as the investigators and researchers who carry out the trials and evaluate experimental therapies.
However, behind each clinical trial are patients fitting narrow definitions, who are recruited to do their part and ensure medicine continues to progress. And patient advocacy groups — like CreakyJoints and the Lupus Research Alliance — are uniquely positioned to act as a liaison between those patients and physicians and researchers.
As such, advocacy groups play a critical role in the development of new therapies for critical diseases, including lupus.
According to Maria Dall’Era, MD, a professor of medicine in the division of rheumatology at the University of California, San Francisco, and a member of the research committee at the Lupus Accelerating Breakthroughs Consortium, patient voices are critically important in drug development and access. By elevating patient voices and taking them into account on an equal level with industry and academic stakeholders, it may be possible to design more inclusive and comprehensive clinical trials, resulting in life-changing therapies across a spectrum of disease states.
“I believe that patient advocacy groups are critical in rheumatology,” Dall’Era told Healio. “Everything that we do should put patients at the center.”
Elevating patient voices
Some patient advocacy groups are positioned in a way that provides access to patients as well as researchers and industry stakeholders. Elevating those patients’ voices and giving them a seat at the table is just one of the first necessary steps to advancing therapies with potential to help those with debilitating diseases, Dall’Era said.
“Patient advocacy groups are critical to making sure patients have a voice in all of these elements,” she added.
According to Dall’Era, when patients are given a space to express their challenges and needs, it becomes possible for physicians to connect with and respond to the issues they may be having.
“It is very important for patients to be able to express their opinions about what they are feeling with this disease,” she said.
Additionally, although clinical markers and manifestations are important for measuring the progress of a disease within patients, the lived experiences of those patients can be just as valuable.
“As a patient advocacy and research organization, CreakyJoints and our ArthritisPower Research Registry have always amplified the lived experience of those with chronic disease,” Shilpa Venkatachalam, PhD, MPH, director of patient-centered research operations and ethical oversight at CreakyJoints, told Healio. “We know well from many studies and interactions with our patient community that patients evaluate their treatment success based on their functional and practical abilities, as well as through their own assessment of quality of life, often more than disease markers.”
Meanwhile, patient advocates — and often patients themselves — can also play a critical role in helping to recruit individuals to trials and getting them involved in research.
“It is important that patients have a say in the design of these trials so that they can make trials more conducive for people who lead busy lives and still want to participate in research,” Dall’Era said. “I believe that by bringing patients to the table with academic investigators, with industry, with the FDA, and with other stakeholders, we can come to a consensus about how best to design clinical trials that are meaningful for lupus patients. By doing this, we will be in a better position to study and develop targeted therapies that will improve long-term outcomes and decrease mortality.”
In several autoimmune and rheumatic diseases, including lupus, there are critical disparities based on gender and sex. For example, approximately 31% to 43% of patients with lupus are Black. However, Black patients account for just 14% of all lupus clinical trial patients.
Advocating for diverse patient representation can help ensure that all impacted populations are included in clinical trials, Dall’Era said.
In the interest of advancing patient participation and representation, the Lupus Research Alliance and the FDA recently announced that they were partnering to create the Lupus Accelerating Breakthroughs Consortium (Lupus ABC).
A blueprint for future advocacy
Announced in March, Lupus ABC aims to connect patients with various industry stakeholders and elevate the voices of those with lupus to create more representative trials and advance therapies for the disease.
“The Lupus ABC emerged from three key initiatives, or influences,” Teodora Staeva, PhD, the chief science officer of the LRA and the Lupus ABC coordinating lead to the FDA, said during a webinar announcing the initiative. “One is the urgent need to address the longstanding challenges in lupus drug development; two is the requirement to integrate the patient perspective in this process; and three is the lupus community’s desire and commitment to deliver safer and more effective treatment options for individuals with lupus.”
Lupus ABC is still in its infancy — its first meeting with the FDA occurred in April — but the hope behind connecting government agencies and patient advocacy groups is a fierce one.
“I think that it is going to be a blueprint, especially for the very challenging and rarer diseases,” Dall’Era said.
If Lupus ABC proves impactful, Dall’Era said she hopes that advocacy organizations concerned with other difficult disease states may follow its lead to integrate patient voices and blaze a path forward.
Meanwhile, CreakyJoints has similarly championed the role advocacy groups play as a source of trust and structure for platforming patient voices in the interest of advancing therapeutics.
“Patient advocacy groups can build and host infrastructure or capacity for engaging patients to document their patient-reported outcomes, or other patient-generated data, in a manner that is useful to research, but also to the patient themselves and their care team,” W. Benjamin Nowell, PhD, director of patient-centered research at the Global Healthy Living Foundation and CreakyJoints, told Healio. “In addition to being shaped by clinical input, critical end points should be informed by the symptoms and concerns that are most important to individuals living with a disease.”
Acting as liaison
Contacting researchers and academic physicians can be daunting for the average patient. Organizations like CreakyJoints give these patients a place to connect with each other, as well as receive trustworthy information from professionals, according to Nowell.
“Patients in this community are uniquely situated to educate peers, raise awareness about diseases, facilitate the conduct of research and disseminate evidence-based information,” he said. “By building a community of trust and by understanding the various needs of a population living with these diseases, patient advocacy groups become credible, safe spaces for both newly diagnosed and long-time patients who seek to access a community for support and reliable information.”
Specifically, CreakyJoints provides education and advocacy support to its thousands of patient members, and offers achievable ways for patients to participate in advocacy and research themselves.
“Our role is to make complex medical information understandable so that it can be used by patients as they navigate their disease,” Nowell said. “To help make information relevant to patients, it is important for rheumatologists and researchers, as well as other health system leaders, to support and integrate patient advocacy groups into their efforts.”
That said, although these groups can connect patients and researchers, there are clear boundaries for how much they should be involved in drug development, according to Venkatachalam.
“Patient advocacy groups should not be called upon to endorse specific brands or biomedical products but rather to provide unbiased and reliable information to equip their communities to make informed medical decisions,” Venkatachalam said. “The role of patient advocacy groups involves mobilizing patients and other stakeholders so that different kinds of data and different types of expertise can be better integrated.”
Beyond acting as a liaison and catalyst for connection, patient advocacy groups are also integral to improving transparency regarding the drug development process, trials and commercialization.
Increasing transparency
“We encourage health care providers to directly interact with our patient community to promote open and transparent dialogue, as well as mutual understanding,” Nowell said. “For example, we regularly interview rheumatologists for our webinars and articles, and invite them to participate in live CreakyChats on Twitter.”
The result of these interviews and conversations is a better understanding, among providers and patients alike, of the others’ perspective, according to Nowell.
“Patients can benefit from understanding how health care providers evaluate treatment options and make decisions based on research in order to manage disease,” he said. “Patient advocacy groups are in a unique position to facilitate productive and meaningful interactions among patients, clinicians, and other health care stakeholders.”
Although drugs are developed with narrow clinical trials designed to test their efficacy under certain conditions, these therapies are ultimately deployed for use in the real world, where the variables are not nearly as controlled.
According to Nowell, by having open conversations with patient advocacy groups and patients, and by involving patient input in therapy development, it may be possible to design trials that are more representative of the actual populations who will eventually be using these therapies, and their lives.
“More needs to be done to integrate vulnerable patients in all aspects of health care, from drug development to the provision of care for the uninsured,” he said. “Concerted efforts would include building trust with vulnerable populations, adapting studies to suit their needs, integrating translators who understand the cultural underpinnings of different communities and their relationship to the health care ecosystem, interacting with vulnerable populations with respect and empathy, and sharing results of study findings with these populations.”
Reference:
Lupus Research Alliance, FDA partner to counter challenges in lupus drug development. https://www.healio.com/news/rheumatology/20230329/lupus-research-alliance-fda-partner-to-counter-challenges-in-lupus-drug-development. March 29, 2023. Accessed April 6, 2023.
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