Welcome to our latest Research Roundup. In this series, we highlight the latest research conducted through PicnicHealth Research.
PicnicHealth is simplifying observational research by combining a direct-to-patient approach, industry-leading AI and technology, and world-class clinical expertise to capture the patient experience in an unprecedented level of detail.
Background: Classic congenital adrenal hyperplasia (CAH) is a rare genetic disorder that affects patients’ adrenal glands and disrupts androgen production. The only available treatments are life-long use of glucocorticoids (GC) and mineralocorticoids. Glucocorticoid use at supraphysiologic doses can have substantial adverse side effects with long-term use. The CAHtalog™ registry, developed by PicnicHealth in partnership with the CARES Foundation, contains over five years of data abstracted from the structured and unstructured medical records of patients with classic CAH, collected across all their U.S. sites of care. This study aimed to characterize the effects of high GC doses on pediatric and adult patients with classic CAH.
Methods: This analysis included 69 patients (45 pediatric patients, 34 adult patients, with 10 in both groups) with classic CAH from the CAHtalog™ registry. The study assessed growth related measurements, GC-related comorbidities, and androstenedione control by GC treatment dosing regimens over time.
Results: The study showed that patients with higher GC doses generally had higher proportions of GC-related comorbidities. Pediatric patients with high GC dosing were more often overweight/obese and had accelerated growth followed by deceleration during adolescence, which could affect future adult height. GC dose and/or androstenedione levels changed extensively over time in a longitudinal patient journey analysis, demonstrating the challenges of long-term management of classic CAH.
How PicnicHealth simplified this observational study
PicnicHealth’s platform played a crucial role in streamlining data collection for this study. By enabling patients to enroll virtually and access their complete medical history through a user-friendly portal, PicnicHealth simplified the process of gathering comprehensive, longitudinal data, with a mean observation period of 10 years for pediatric patients and 16.8 years for adult patients. This approach allowed for the efficient extraction of both structured and unstructured data, providing a detailed picture of each patient's medical journey and enhancing the accuracy of the research findings.
Background: Progressive supranuclear palsy (PSP) is a rare neurodegenerative disease characterized by rapid progression and a variety of debilitating symptoms, including balance issues, gait impairment, and cognitive dysfunction. PSP often remains undiagnosed for years, complicating patient management and care. This study aimed to provide a comprehensive understanding of PSP disease progression, management, and healthcare resource utilization.
Methods: The study retrospectively analyzed medical records, collected from over 194 healthcare centers, from 72 patients with PSP. Approximately eight years of data was collected from each patient. Researchers focused on key clinical outcomes such as the onset of PSP symptoms, the use of healthcare resources, and the progression of disability over time.
Results: The findings revealed that most patients experienced significant motor and cognitive impairments before diagnosis, with falls and gait problems being common early symptoms. The study also highlighted a high reliance on healthcare resources post-diagnosis, including the use of assistive devices, medications, and supportive care. Despite the use of these resources, the disease's rapid progression continued to severely impact patients' quality of life.
How PicnicHealth simplified this observational study
PicnicHealth* enabled the comprehensive collection and abstraction of medical records from multiple healthcare centers. By partnering directly with PSP patient communities for recruitment, the study was able to build a recontactable cohort of patients, facilitating long-term research and follow-up. This patient-centric approach not only streamlined the research process but also made participation more accessible for individuals with PSP, who often face mobility challenges. This study provided valuable insights into diagnosis and management of PSP, which could be used to improve patient’s experiences before and after a PSP diagnosis.
*This study was originally conducted by AllStripes, a platform dedicated to generating evidence about the rare disease community, which was acquired by PicnicHealth in 2023.
Background: Hemophilia B is a rare bleeding disorder characterized by spontaneous bleeding episodes, particularly into joints and muscles. These episodes can cause long-term joint damage and significantly impact patients' quality of life. The current standard of care for hemophilia B is prophylactic treatment. This study assessed the real-world burden of hemophilia B in the United States over a five-year period, as traditional data sources often lack comprehensive details on disease severity, bleeding events, and treatment outcomes.
Methods: The study included 93 male patients with hemophilia B, identified through social media, provider partnerships, and patient advocacy groups. Retrospective unstructured and structured data were collected from 2015 to 2020 across all sites of care, focusing on patients' medical records, including annualized bleeding rates (ABR), comorbidities, and treatment regimens.
Results: The study found that prophylaxis use increased with disease severity, with 86% of patients with severe hemophilia B receiving prophylactic treatment compared to 40% of those with mild/moderate disease. Common comorbidities included arthropathy, synovitis, and mental and behavioral disorders. The mean ABR was higher in patients with severe disease compared to those with mild/moderate disease, reflecting the ongoing burden of illness despite prophylactic treatment.
How PicnicHealth simplified this observational study
PicnicHealth’s platform facilitated the collection of comprehensive medical records across multiple institutions, regardless of the provider system. By digitizing these records and integrating structured and unstructured data, PicnicHealth enabled the researchers to capture a detailed and accurate picture of each patient’s disease progression and treatment outcomes. While traditional methods of conducting research require high patient engagement to capture bleeding events, PicnicHealth’s approach of using narrative text extraction reduced the burden on patients and researchers, allowing for a more efficient and thorough analysis of the real-world impact of hemophilia B.
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See more posters and publications at research.picnichealth.com/publications, and explore how PicnicHealth can drive your observational study forward at research.picnichealth.com.
patients onboarded to platform
medical visits processed
facilities provided medical records
healthcare providers
research programs
published posters and manuscripts
partnerships with top 30 pharma
Discover how PicnicHealth data powered medical research in 2021
New methods to improve the identification of lupus nephritis patients in RWD using narrative text extraction were presented at the American College of Rheumatology Convergence virtual conference.
Ultragenyx and PicnicHealth presented preliminary data at the International Network for Fatty Acid Oxidation Research and Management (INFORM) LC-FAOD conference, showcasing the power of linking medical records to patient reported outcomes to better understand the complexities in LC-FAOD care.
We joined Roche-Genentech and other key researchers to introduce a longitudinal, patient-centric Hemophilia A real-world dataset that features abstraction of data points from medical records at scale and supplementation with patient-reported outcomes at the American Society of Hematology annual meeting.
Also at ASH 2021, Roche-Genentech and our research partners presented new findings looking specifically at mild and moderate hemophilia A patients found in PicnicHealth’s RWD cohort to better understand disease burden and healthcare resource utilization in this historically underrepresented patient group.
This year, experts from PicnicHealth joined podcasts, webisodes, virtual summits and much more to speak to the importance of patient-centric approaches when building complete, deep real-world datasets.
CMO Dan Drozd joined The Evidence Base to discuss how to build and measure data completeness
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BBC Storyworks and Roche featured Noga Leviner and her medical journey in “The power of partnerships”
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CEO and Co-founder Noga Leviner joined our partners Komodo Health to discuss patient-centric RWD in “For the Health of It”
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PicnicHealth experts joined Arsenal Advisors in 3 summits to highlight our research partnerships
(from 13 patients with LC-FAOD)
38% Female
15 providers / patient
7.5 years of data / patient
(from 13 patients with LC-FAOD)
patients onboarded across 19 conditions
medical visits processed
healthcare providers
Facilities provided medical records
Change Champions onboarded
Research programs
published posters and manuscripts
partnerships with top 30 pharma
At ASH 2022, Genentech, a member of the Roche group, and our research partners presented their study examining the epidemiology and bleeding phenotype in females with mild Hemophilia A (HA), a minority group that is poorly characterized, compared with a cohort of males with mild HA.
At EHA 2022, Novartis used PicnicHealth’s real-world data to characterize sickle cell disease and burden of illness in the United States.
Genentech leveraged the FlywheelMS cohort to describe COVID-19 vaccination patterns and COVID-19 outcomes among people with multiple sclerosis (pwMS) and in subgroups of pwMS, including by disease-modifying therapy (DMT) status.
We partnered with Alexion, AstraZeneca Rare Disease to help identify flares in Lupus Nephritis patients by combining structured and unstructured medical records with lab data.
Cohort Overview. Understand patient healthcare utilization throughout disease history with ability to probe for meaningful mentions and events.
Sickle cell (SC) is the most common inherited blood disorder in the United States. Red blood cells become rigid and shaped like crescent moons, preventing oxygen from getting to parts of the body. This can cause fatigue, severe pain, organ damage or stroke.
Addition of Narrative Text Abstraction to ICD-Based Abstraction Significantly ImprovesIdentification of Lupus Nephritis in Real-World Data
Join our early breast cancer registry to be counted and share your story with research.
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List the names of all the doctors, hospitals, and other facilities your loved one visits regularly, along with those they have visited in the past. Try to go back as far as you can, striving for at least the last 5-10 years, but do your best. Even if you can’t remember them all, having a strong baseline can help you quickly identify gaps in records.
It is important to make sure that you are fully empowered to make decisions on behalf of your loved one with Alzheimer’s. Your relationship status with the patient may not be enough to legally give you access to your loved one's medical information. It is a good idea to talk to an expert about securing special legal status, such as Power of Attorney (POA), a legal document that allows an individual to name someone as their decision maker should they no longer be able to make decisions on their own.
It’s important to have all of your loved one’s medical records together in one spot. This makes it much easier for you and your loved one’s physicians to accurately map the patient’s medical journey and more easily share information between doctors. Fortunately, tools exist to make record management and access simple. A free resource like PicnicHealth helps you collect and organize all of this information. PicnicHealth’s intuitive timeline allows you to pinpoint data across the medical history, eliminating your need for keeping heavy binders filled with paper records or keeping track of multiple software portal logins.
The better you understand your loved one's medical history, the better you can advocate on their behalf. Access and understanding of this information will help you to ask informed questions with physicians. Through regular communication backed by the data in the medical records, you can help your loved one’s care team develop a more successful care plan.
Learn more about PicnicHealth’s commitment to the Alzheimer’s community and the Alzheimer’s Association
Learn MoreWhen you’re juggling appointment times and insurance claims, putting a robust support system together might not strike you as the most urgent task. Investing the time to cultivate relationships with people can turn to in times of need will pay dividends. The next time you need a last-minute ride or just someone to listen, you won’t be on your own.
There are many condition-specific support groups and support groups for caregivers generally in person or online. In addition to the encouragement and empathy they provide, support groups can be a helpful source of tips, resources, and recommendations for navigating caregiving.
The backbone of effective caregiving is organization. Keep medical information, appointment schedules, and medication lists in order. Use a planner or a digital service like PicnicHealth to stay on top of your responsibilities. This attention to detail can prevent future complications and reduce day-to-day stress.
We’ve seen incredible breakthroughs in treatment over the past couple of years, powered by patients and their caregivers participating in research. Stay in the loop about the latest in medical advancements and available resources that could benefit your loved one. Whether it’s a new therapy option or a community service that aids independence, being informed can make a world of difference in the quality of care you provide.
It may seem self-centered to focus on self-care—but when you feel good, you can be a better caregiver. Whether it’s exercise, a mindfulness practice, a soak in the bath, or just time to rest when you need it, carve out those moments in the day when you can unwind, reset, and stay healthy mentally and physically. Think of it as building up your reserves of kindness, patience, and understanding—which can only benefit your loved one. No one can pour from an empty cup.
Easily manage all of your loved one's medical records and contribute to ongoing Alzheimer's research with PicnicHealth.
Learn More(from 13 patients with LC-FAOD)
38% Female
15 providers / patient
7.5 years of data / patient
(from 13 patients with LC-FAOD)
We hope you found this session informative! Sign up for PicnicHealth’s Alzheimer’s research program below.
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