The healthcare field is increasingly proactive in its focus on engaging patients in ALL stages of medical care instead of solely as the end-user. This allows patients to not only benefit from medical advancements and care improvements, but to drive them based on real-life experiences, not just theory and lab science. The patient-centric approach is affecting everything from research to clinical care to basic health choices, truly a comprehensive “bench-to-bedside” effect.
The ability to engagement patients into the process of healthcare advancements comes in many different forms, including collecting data directly from the patients. These patient-reported outcomes and data now has positive advancements in acceptance of valid evidence. Such approaches include technological survey platforms, HIPAA compliant registries, and wearables, and as advances in technology continues to inspire major evolutions in the healthcare field, new challenges and new opportunities arise in ensuring a patient-centric approach.
While the processes for ensuring patient-centrism are facilitated by medical centers, physician’s academic investigators, industry and pharma, and other healthcare professionals, one of the more valuable major connections to patients comes in the form of patient advocacy and education, and engagement foundations. Examples of these may be non-disease specific, such as 23AndMe and Global Genes to very specific, such as foundations serving rare disease such as Rett syndrome, Lymphangioleiomyomatosis, and sarcoidosis. Foundations that exist to support the education, awareness, and research of such disease spaces are excellent resources for enabling high levels of real-world data via the patients directly.
These organizations are especially valuable in collaborating with investigators to conduct observational studies that serve to obtain real world data as it occurs. In contrast to a clinical trial, observational studies or non-interventional studies reflect actual patient behavior and what a patient’s experience may be in relation to anything from therapies taken, products used, demographics, burden of disease, etc. Such studies are increasingly important to assess the nature of the disease space and/ or the value of a drug or technology with evidence drawn from a realworld setting. This data is essential for multiple stakeholders including regulatory agencies, payers, health care management organizations, industry/pharma, healthcare professionals, and patients.
Real world data is essential, from a patient-centric perspective, for industry to truly impact patient outcomes. Data resourcing postapproval decision making can be obtained in a number of ways:
- Electronic health records (EHRs)
- Claims and billing activities
- Product registries
- Patient-related activities in out-patient or in-home use settings
- Health-monitoring devices
- Disease registries
Real world data can be divided into two types: Primary data are generally collected in interventional Phase IV studies, non-interventional prospective observational studies, medical patient registries, and health surveys. Secondary data are often obtained from clinical chart reviews, patient reported registries and/or insurance claims databases, and are used in such things as: retrospective database studies; prospective study design; hybrid studies; as well as for pre-approval and post-approval product review.
Of course, some of this information cannot be provided by any other than the medical provider. And while all this real-world data can be obtained from a variety of providers, under-valued or overlooked key. These foundations, usually non-profit, neutral-party stakeholders, strongly advocate for the uses of these non-interventional studies. This includes the Foundation for Sarcoidosis Research, which is an excellent example of collaboration to provide real world data for a variety of purposes, including both pre- and post-approval.
Non-interventional data collection rings so true for foundations such as ours because the voice of the patient – information accessed directly from the patients’ perspective and derived from real world patient experiences – can say as much as, if not more, than just clinical information. Foundations can facilitate collections of real world data to help support industry and other stakeholders in health care decisionmaking. By obtaining real world data from their patient databases and outreach venues, foundations can provide real world data more quickly and inexpensively to support the understanding of treatment effectiveness as well as to provide insight into patterns of care, longterm drug safety, product use, health care resource utilization, and disease epidemiology, all of which can support decision making.
Non-interventional methods of collecting patient data can be supported by Foundations quite readily as these foundations often have not only immediate access to patients but also, they secure the TRUST and ENGAGEMENT of patients to follow through on the collection methods. The “real world” quality of the information collected is usually extremely timely and relevant, and the collect methods can be tailored to meet the patient population as well as fulfilled specific needs of those looking to access the data. These Foundations can work in partnership with various stakeholders by collecting the data and sharing to support end goals.
On such specific disease foundation, the Foundation for Sarcoidosis Research (FSR), provides a strong example of how these non-profit organizations can provide real world data to collaborators. FSR provides a formalized program - SARConnect - developments methods of real world data collection to meet specific needs of various stakeholders including:
- Pharma
- Academia
- Healthcare providers
- CROs
- Biotechs
SARConnect provides real world data collection including PRO from registries, wearables, surveys, interviews, outreach etc. to support various projects such as studies, trials, product development, disease epidemiology, etc. One of FSR’s major SARConnect real world data collection projects is our Patient Report Outcomes (PRO) Patient Registry. The data serves many collaborators in a variety of ways including studies, trials, pre- and post-approval product development, disease epidemiology, etc. The data collected from this is shared with partners, including to pharma to provide to them insight into the disease space as they navigate and explore post-approval elements within product usage for therapies as well as for repurposing considerations and new drug therapies arenas.
The FSR Patient Registry (FSR SARC) is providing information to researchers and industry, first by collecting real world data from patients, and then sharing anonymous information with stakeholders. FSR-SARC is an IRB-approved collection of longitudinal patient data via our on-line state-of-the art patient registry tool, a HIPAA-compliant hosting facility that provides a secure environment to protect properly consented de-identified patient reported outcomes. This data repository allows for real-time de-identified data queries for stakeholder’s analysis use. Currently in English, the registry is global and will soon expand to other languages.
This Real-World Data is available for: industry, investigators, health care professionals, etc. for:
- expanding the knowledge base for sarcoidosis
- Industry analyzing markets/products
- clinicians treating patients
- epidemiologists analyzing disease data
- investigators seeking patients for new clinical trials
FSR collaborates with industry partners to help fill the knowledge gap between clinical trials and actual clinical practice. Real world data is critical for ensuring a true impact for patients. Successful product development and end-user satisfaction is crucial to industry and positive patient impact is the absolute core of foundations like FSR’ s mission, it is in the best interests for the collaborations to exist.
The following slides present just a snapshot of the data FSR is collecting via the PRO Patient Registry. The patient registry presents 78 questions written by their Scientific Advisory Board designed to capture critical PROs that have multiple impact on academic research as well as industry review. While the data is presented here in summarized graph format, it is share the raw data via specific queries in database format to allow for collaborators to utilize the raw data for their own analysis. FSR holds a Data Sharing Agreement set in place with each collaborator, and the organization has abstracts and publications coming out in 2018 regarding the overall data results.
Technology advances enable us to gather and store huge amounts of health-related data allows for health care providers, researchers, industry and other stakeholders so much more rapidly. And the development of sophisticated, new analytical capabilities, allows better ways to analyze these data and apply the results of analyses to drug development and medical product development and approval.
It is essential that stakeholders not undervalue or worse, not recognize, the key role medical research foundations can have in providing this real-world data. As collaborators, Foundations can provide essential data.