Rare Community Profiles
Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.
Alira Health and the MGFA Partner to Develop a Myasthenia Gravis Registry
An estimated ~0.002% of people living within the United States have myasthenia gravis, a rare autoimmune neuromuscular disease. Similar to many other rare conditions, myasthenia gravis tends to be misunderstood and under-researched; more funding and research are needed to better understand the disease itself, its long-term impacts, and potential treatment options.
In late January 2023, Alira Health and the Myasthenia Gravis Foundation of America (MGFA) announced that they would be partnering to develop and manage a registry of patients with myasthenia gravis (MG). Patients may submit their data, track their symptoms, coordinate with doctors, and even connect with the MG community via Health Storylines, a digital tool from Alira Health that will assist with registry management and data analysis.
In an interview with Patient Worthy, Alira’s Renee Willmon, Senior Director of Evidence & Outcomes, shared insights into the partnership, what Health Storylines is, and how patient-generated data can change the medical field.
About Renee Willmon
Renee is the Registry Lead on the Alira Health side of the partnership with MGFA, and works with both teams to ensure that MG patients’ experiences are well-represented in the registry and that other stakeholders can access and use the data to improve the development of new treatments, and access to care for MG.
When Renee Willmon first pursued her graduate studies, she focused on anthropology—and forensic anthropology in particular. After completing her graduate studies at the University of Toronto, University of Western Ontario, and Ecole Pratique des Hautes Etudes, she pivoted towards digital health. She shares:
“I was really interested in the vision of empowering patients to take a more active role in shared decision-making, the health data they’re generating, and how they can use technology to democratize clinical research. Patient-donated data can benefit research and enhance care, regardless of condition.”
In 2016, Renee joined Self Care Catalysts, a Toronto-based digital health company that was acquired by Alira Health in 2022. Now, Renee is the Senior Director of Evidence & Outcomes. She works with the Real World Evidence team to address how to use patient experience data —what matters to them in their own words—to empower patients to contribute their experience in a research setting. Says Renee:
“It has been an amazing experience working with the Real World Evidence team. The team not only holds a ton of expertise across the life cycle of drug development, but highly values relationships with patients and advocacy groups. Elevating the patient’s role in research and drug development helps advance our mission to humanize healthcare. Tools like Health Storylines also help us to infuse the patient voice in everything from patient support programs and treatment commercialization to streamlining the clinical trial experience.”
Partnering with the MGFA
Alira Health partnered with the Myasthenia Gravis Foundation of America (MGFA) to assist the MGFA in re-launching the Global MG Patient Registry. Renee shares:
“MGFA is a wonderful organization. They went through a very thoughtful 18-month process identifying what they wanted out of the “new” registry that defined five distinct stakeholder groups for the registry to serve. The MGFA selected Self Care Catalysts as a partner to help them leverage existing data from a 10-year period and help patients understand how their contribution is valuable so that the registry can continue to grow. This year, the registry re-launched in January. We helped migrate the data into the Health Storylines database and invited patients who previously contributed to the registry to fill out a questionnaire every six months. We have also offered tools in the Health Storylines app so that patients can contribute their data in real time as relevant events occur. Any new patients may also participate and can easily use the app to manage their daily myasthenia gravis experience.”
So what exactly is Health Storylines? Health Storylines itself is disease agnostic, meaning anybody with any condition can use the platform. It offers tools such as reminders and appointment calendars. Renee explains that Health Storylines was designed to be a central place for people to manage their health while personalizing the platform to their needs. She further states:
“There are also versions tailored for specific conditions such as myasthenia gravis. If patients download Health Storylines through the registry landing page, they have immediate access to the questionnaires, tools, and educational materials. The more tailored version offers full functionality plus specific insights from advocacy groups. Different versions or configurations can also be used for patients to participate in clinical trials, enabling them to fill out assessments from home. Health research is often biased towards including only patients that live near a research site or center of excellence, which can alter representation. Since anybody can use the app, it helps to expand representation and make research more inclusive.”
Health Storylines also allows users to journal for mental health. Says Renee:
“Mental health is health. It affects lived experience and is often measured in patient-reported outcomes. But by definition, these outcomes are designed to be applicable to a broad group and lack specificity. Our journaling tool provides fields where patients can add their own words about what they’re going through. In patients with a rare form of cancer, for example, we heard about how anxiety about the treatment they received through an injection was altering quality-of-life—but that wasn’t captured in other ways of looking at the data. So we want to capture elements of the experience that aren’t considered from a more traditional standpoint so that the patient experience is fully captured.”
Outside of journaling, Health Storylines supports mental health through a daily mood tool, where users can report their mood and any relevant notes; “Healthy Doses,” a set of memes where patients can select what they need a dose of—happiness, love, humor—and receive cards with quotes and images; and fields to celebrate improvements or wins.
So How Does This Help Patients?
Health Storylines confers patient benefits through bringing people joy, as well as measuring data in a way that the research community will take seriously. The journaling and more personalized elements also help patients share their thoughts in a non-judgmental way. In terms of data, this tool can be helpful when speaking with doctors: pulling up the app to discuss months-worth of symptoms, trends, and other information. Renee says:
“This helps lend additional credibility to what the patient was saying for the doctor by bringing it to them in a language that they understand. The patient is empowered, which improves collaboration and decision-making. We also allow patients to invite others to see the data, act as an accountability partner, read educational materials, or communicate with peers. As we were developing Health Storylines, we spoke to patients about reminders or alarms, binders, notes—all of these distributed tools. We wanted to create a seamless, central interface. You can even sync your data from your wearables and send it to the app!”
What is the MGFA Global MG Patient Registry?
As explained on the Registry website:
“A patient registry is a collection of information about a group of patients who share a condition or experience, and represents an important way for patients to share their experience and perspective about their disease with the medical community to improve care, treatment, and research. MG patients from around the world can privately, safely, and securely enter their data which is protected as part of a HIPAA-compliant secure database.”
The MGFA Global MG Patient Registry collects patient health data through standardized surveys twice per year, and is now expanded through the capabilities of Health Storylines to include data any time in addition to the questionnaires. By contributing data to the registry, patients can provide real-time, up-to-date data that helps inform future research. The registry data is anonymized and aggregated, so it cannot be attributed directly to patients. Renee says:
“The feedback from patients has shown how impactful a tool like Health Storylines can be in collecting registry data. Patients say they love the functionality of jotting things down as they occur. There can be a deep feeling of being misunderstood, or difficulty in relaying your experience without it feeling like a complaint or reflecting the bad parts. They can now offer insights into what is important to them, which can be explored further through systematic research. One of the motivating reasons that they remain engaged is the knowledge that their de-identified data is going to help others. Registries are not new, but patient-generated data in a registry is not as common. We really appreciate the MGFA for choosing and trusting us to take their registry forward in this new and modern way.”
Learn more about the registry, and how you can participate, here.
What is Myasthenia Gravis (MG)?
Myasthenia gravis is aptly named, deriving its name from the Greek and Latin words for “grave muscle weakness.” People with myasthenia gravis experience varying degrees of weakness and fatigue in voluntarily controlled muscles. Normally, nerve impulses release acetylcholine at the neuromuscular junction; this travels to a receptor site to contract the muscle. In MG, the immune system creates an antibody which attacks the neuromuscular junction, destroying receptor sites and preventing the muscles from reading signals from the brain. An estimated 20 in every 100,000 people has MG. Symptoms of MG often worsen with activity and improve with rest. Potential symptoms may include:
- Drooping eyelids
- Dysphagia (difficulty swallowing)
- Slurred speech
- Double or blurred vision
- Shortness of breath
- Fatigue or general malaise
- Difficulty making facial expressions
- Neck or limb weakness or fatigue
- Impaired speech
- Changes in gait
There is no cure for myasthenia gravis—but the prognosis is far from grave! Most individuals can manage their MG with steroids, plasmapheresis, immunosuppressants, cholinesterase inhibitors, and more. However, 10% of those with MG do develop life-threatening respiratory complications which may require further support.
About the Myasthenia Gravis Foundation of America
The Myasthenia Gravis Foundation of America (MGFA) is the largest, leading patient advocacy organization solely dedicated to finding a cure and improved treatments for the rare neuromuscular disease myasthenia gravis (MG) by funding critical research discoveries and providing patient-centric programs, guidance, events, and webinars to connect, educate, and assist members of the global MG Community. You can visit the MGFA at myasthenia.org.
About Alira Health
Alira Health is an international patient-centric and technology-enabled advisory firm whose mission is to humanize healthcare. The company works with healthcare and life sciences organizations looking for support across their entire solutions lifecycle. From development and transaction advisory to market access and medical care, Alira Health complements their clients’ expertise with a full spectrum of services including research and clinical development solutions, technology-powered consulting, and real-world evidence. Alira Health’s integrated and multidisciplinary team of over 800 scientists, strategists, economists, clinicians, and biostatisticians collaborate across our North American, European, and Asian offices and advise 80% of the top 50% of MedTech companies and 75% of the top 50% of Pharma companies.