Development of successful new treatments for fibrolamellar will require a deep understanding of the causes and progression of the disease. Since FLC patient populations are so small and dispersed, information about their treatments and outcomes is incomplete. Systematic data gathering efforts, such as registries and natural history studies, can play a major role in closing critical gaps in our knowledge of the disease.
Increasing the number of participants involved in these data studies increases the accuracy and power of the results. Every patient’s information is important, so please participate if you can!
What is a patient registry?
Patient registries are systems for collecting, storing and analyzing a uniform set of information about patients for research or clinical purposes. Most patient registries collect a defined set of demographic, epidemiological, quality of life, treatment, and clinical data. That data can be collected through patient-reported surveys or extracted directly from medical records. Since most registries are broad in scope, registry studies can help researchers collect disease information, consider different research hypotheses, identify patient outcomes and study best practices in treatment. Data that a registry shares with researchers is “de-identified” to preserve the privacy of the participants.
What is a natural history study?
Some use the terms registry and natural history study interchangeably. However, unlike registries which store a broad range of data that can be used to answer many different research questions, natural history studies are designed with a very specific goal – to track a disease’s course over time by identifying factors such as demographics, environmental conditions, and genetic variables that correlate to the disease and its outcomes. These studies can produce greater understanding of how a disease like FLC develops and how it can be treated most effectively.
Why are registries and natural history studies important for fibrolamellar research?
Registries and natural history studies can help accelerate FLC research in many ways. Critically, they provide researchers a set of consistent, comparable data from a large group of patients to study. Analyzing such data can help FLC researchers build a more comprehensive understanding of the disease, advance research hypotheses, determine patient outcomes, and identify potential best practices in treatment. The information can help answer questions such as:
- Is the disease more common in particular demographic groups?
- What treatments are being administered? How is that changing over time?
- How do patient outcomes vary? Why?
- How does the FLC develop and progress? Are there different disease subtypes?
Registries and natural history studies can also reduce the time and cost of individual research efforts, especially if they offer open access to relevant data. In addition, the “real world evidence” obtained from a natural history study could potentially provide a baseline to drive the approval of new drugs or therapies that demonstrate better results. This could be extremely important for extremely rare diseases like FLC where the inclusion of a placebo control arm in clinical trials may never be feasible.
The Fibrolamellar Cancer Foundation does not directly fund or manage any registry or natural history studies for FLC. We are thrilled that several other organizations are systematically gathering patient data and are making that data accessible to all FLC researchers. FCF collaborates with all these research data providers and strongly encourages FLC patients to participate in as many of these initiatives as possible.
Current fibrolamellar data initiatives include:
The Fibrolamellar Registry is a fibrolamellar-specific patient registry that gathers survey information, medical histories, and test results for patients with FLC, and stores them in a highly secure database. The Fibrolamellar Registry is the largest and most long-standing data initiative for FLC, having launched in October 2016. It currently contains information on over 200 current and former FLC patients from 35 U.S. States and 21 countries.
A number of fibrolamellar patients, caregivers, clinicians and researchers, including a team from Rockefeller University, worked together to build the Fibrolamellar Registry. At the request of epidemiologists, statisticians and pathologists, the platform was recently migrated to REDCap, a browser-based, an electronic data capture system that is widely used in academic research. The Fibrolamellar Registry is a 501(c)(3) non-profit organization, and its survey has been approved as a scientific study by the Genetic Alliance Institutional Review Board.
Participation process:
The Fibrolamellar Registry collects information about patients both living and deceased. Key steps for enrollees include:
- Signing up at fibroregistry.org, then activating an account and providing profile and contact information.
- Completing the Fibrolamellar Registry survey, estimated to require approximately 1 hour. The survey includes questions about patients’ demographics, medical history, habits, family background, FLC diagnosis and treatments.
- Uploading blood test and imaging reports.
- Providing periodic updates, as necessary, to keep the Registry information up to date.
Program benefits:
According to the Fibrolamellar Registry, their program offers several benefits:
- They are a well-established registry with a rich set of data that goes beyond the standard information contained in electronic medical records. In addition to information about treatments and their outcomes, it asks about personal narratives, opinions about clinical trials, viral and environmental exposures, fertility issues, usage of complementary medicines, and more. The Fibrolamellar Registry also contains uploads of patients’ blood test results and imaging reports.
- Their non-profit structure and management by patients and caregivers help to maintain patient trust and ensure that patient data will not be sold to a third party for profit.
- They have a simple and straightforward mechanism to allow any researcher to apply to use their data. In the first half of 2022, three research papers using data from the Registry have been submitted for publication and two more are in preparation.
Contact information:
Interested patients can sign up at http://fibroregistry.org or send an email to contact@fibroregistry.org for more information.
Researchers interested in accessing de-identified data from the Finrolamellar Registry can visit https://fibroregistry.org/faq-for-researchers/ for more information or send an email to contact@fibroregistry.org to request an application.
The Natural History Study of Rare Solid Tumors is being carried out by NCI’s MyPART (My Pediatric and Adult Rare Tumor Network), a group in the Center for Cancer Research at the National Cancer Institute that drives research across a range of pediatric, adolescent, and young adult rare tumors.
This study collects clinical information, family medical history, imaging scans, tissue samples, and patient-reported outcomes (PRO) data that will be analyzed to better understand how rare solid tumors like FLC develop and grow over time. This trial does not test any new treatments. Instead, it compiles information from patients, tumor samples and their medical records to better understand the “history” or progression of the cancer over time. By participating, patients are helping provide researchers with the data they need to discover trends that may lead to the development of new treatments.
MyPART and the Natural History Study of Rare Solid Tumors are part of NCI’s “Cancer Moonshot” portfolio. In 2016, President Obama launched the national “Moonshot” initiative to accelerate cancer research efforts and eliminate barriers to progress by broadening access to important data and facilitating collaboration among researchers, patients, advocacy organizations, and pharmaceutical companies. To support these efforts, Congress passed the 21st Century Cures Act in December 2016, authorizing $1.8 billion in funding over 7 years. Participation of FLC patients in this study creates a valuable opportunity to focus some of that sizeable “Moonshot” research effort on fibrolamellar carcinoma.
Participation process:
Potential study participants can enroll in the Natural History Study of Rare Solid Tumors from their homes. Steps in the process include:
- Patients contact the MyPART nursing staff to participate; After the patient signs the appropriate consent forms, the MyPART team then works with healthcare providers to collect all of the patient’s required medical records and available tumor tissue.
- Patients receive and mail back a saliva sample kit which will be used for DNA sequencing.
- Patients then fill out questionnaires about their medical history, their family’s medical history, and how having a rare tumor impacts their life.
- Patients may be asked to visit the NIH Clinical Center (at no cost to patients traveling from within the US), where the study team will collect a blood sample that will be analyzed for immune system proteins. When at the NIH Clinical Center, patients will have the opportunity to meet with experts about their rare tumor who can provide treatment and clinical trial recommendations. All care and consultation that a patient receives from the NCI clinical team will be free of charge to both the patient and their insurance company.
- Every year, patients are asked to provide an update if their condition or medical care has changed. Patients can stop participating in the study at any time.
Program benefits:
This NCI study offers value to both researchers and participating patients.
- Research benefits: Patients who take part in the study may help speed up the development of new treatments for fibrolamellar by helping researchers learn more about why and how fibrolamellar progresses. The analysis of the clinical and biospecimen data from this natural history study may allow researchers to gain new insights into fibrolamellar biology and develop treatments tailored to the disease. Additionally, approval of new treatments for FLC will require a demonstration that they change the natural history of the disease. If enough FLC patients this study, the baseline data may make it possible to meaningfully assess the effectiveness of new treatments in clinical trials.
- Patient benefits: In addition to aiding research:
- Participating patients can also receive treatment perspectives directly from the NCI clinical team through the study’s optional clinical component. Because the NCI clinical team reviews the medical records collected and the study includes some CLIA-grade testing suitable for diagnosis, the team can give participating patients, at their request, treatment recommendations, “second opinions”, and information about clinical trials they may be eligible for.
- Patients and their doctors will be notified by the NCI research team if any clinically actionable mutations that could suggest viable treatment options are discovered in the study’s genetic testing process. Study participants will also have the opportunity to discuss these results with a NIH Clinical Center genetic counselor.
- Importantly, there are no direct costs to patients or their insurance companies for participating in this study, including costs related to any care and consultation the patient receives from the NCI clinical team and approved travel to the NIH Clinical Center.
To learn more about this opportunity to contribute to research, click here to view additional details about the study and a series of frequently asked questions (FAQs).
Contact information:
To learn more or to enroll in the study, patients can:
- Visit www.cancer.gov/mypart, or www.clinicaltrials.gov/ct2/show/NCT03739827 to learn more about the study
- Email NCICCRRareTumorClinic@mail.nih.gov or call 1-888-624-1937 (NCI CCR Referral Coordinator) to ask specific questions to the MyPart team, or begin the enrollment process