With individual rare diseases affecting such a relatively small proportion of patients globally, capturing effective data to aid with research into treatments is incredibly important.
Relying on data from clinical trials alone is no longer sufficient to ensure safety, efficacy, and discovery of biomarkers for new drug development. Increasingly regulators and industry are incorporating data from outside of these trials into their decision making.
Real-world evidence (RWE) supporting these decisions now includes patient-reported outcome measures (PROMs), patient reported experience measures (PREMs), disease specific data, and natural history data.
Real-World Data Sources Explained:
Patient reported outcome measures (PROMs) assess the quality of care and treatment from the patient’s perspective to calculate benefits from treatment using pre- and post-treatment surveys, over time. The key emphasis is on quality of life.
Patient reported experience measures (PREMs) measure the patients’ perceptions of their experience whilst receiving care.
Natural history is the course a disease takes in individual patients from its pathological onset (‘inception’) until its eventual resolution.
The Democratization of Data
The healthcare ecosystem is filled with incoherent and often incomplete data, which is scattered, siloed, and littered with critical gaps, making it difficult and time-consuming to translate into RWE.
Real-world evidence combined into ‘one voice,’ such as that from Rare Central™, provides many opportunities for rare disease communities, advocacy groups, and other stakeholders.
The evidence can be examined and democratized for real-world use in disease-specific natural history studies, studies of treatment effectiveness, quality of life measurement, epidemiology, burden of disease, and drug development.
For patients and advocacy groups, one of the main benefits of a registry rich in real-world evidence is the ability to accelerate research of treatments and possible cures, as well as improving diagnosis times, increasing awareness of rare diseases, understanding disease progression to tailor support and, in turn, generate interest and support for additional research and funding.
Alan Thomas, founder of Ataxia & Me, a charity based in Wales that advocates for the voices of people with ataxia and other related diseases, is passionate about “talking with the patient, not at the patient.”
Alan recently spoke with us on the subject of real-world evidence and why it’s an essential step to understanding disease.
“I find that a lot of charities focus on the research element of the work, which is good, but I feel like it needed more patient input, so I founded a charity to make sure the patient’s views are put across.”
Alan feels there is an opportunity to greatly improve research by understanding the impact of a disease on a person’s life, and using real-world evidence and patient registries can be a valuable tool in capturing these patient experiences.
Supporting Decentralized Trials
Now more than ever, it is vital for patients to be able to participate in trials without the need to travel to a medical facility unnecessarily. Rare Central™ allows for data to be collected from patients at home, as well as in a hospital, which allows for more patients to participate, meaning the data is diverse and representative of the real world.
To achieve this, the registry provider must have the appropriate partnerships. Our partnership with the wearables company Aparito, for example, provides an end-to-end solution to collect data fit for regulatory purposes and to accelerate research in rare diseases.
Here at Pulse Infoframe, we have expertise in both health and technology, meaning the data sets generated from our system meet the requirements of all stakeholders, regardless of where and how evidence was gathered.
Bringing Stakeholders Together
We are ideally positioned to be the meeting point for information from a range of sources, such as key opinion leaders (KOLs), physicians, patients and patient groups, and sponsors.
We bring together all stakeholders involved in trials to gather information which is valuable and enhances trials, while keeping patient and advocacy groups involved throughout the development process.
The benefit of this approach for patient groups is that it cuts down the contact points required for creating such a database and allows for a targeted approach to data gathering, meaning only useable information is collected, saving time in getting the system live, but also avoiding unnecessary stress for patient contributors.
In a recent announcement by the National Kidney Foundation on their partnership with Geisinger on the upcoming NKF Patient Network, they spoke about the benefits of having a longitudinal database that includes data on a wide range of variables, including demographics and medical history, diet, lifestyle, socioeconomic status, kidney disease and its complications, blood and urine test results, and medications. These variables will provide new insights into kidney disease at all levels, for professionals in the field and the people with the disease.
“We are excited to provide an opportunity for patients living with kidney disease to participate in this project,” said Alex Chang, M.D., co-director of the Geisinger Kidney Health Research Institute. “The ability to capture patient reported data not normally collected in clinical practice or in research studies is very important. By linking this data with routinely collected EHR data, we believe this database could provide novel insights on how we can improve the lives of patients with kidney disease.”
Knowing Where to Turn
Considering establishing a disease registry can be a daunting prospect, particularly for patient advocacy groups for rare diseases with a small, albeit passionate, following. However, we have tools in place which allow you to start small and easily scale up, all while benefiting from input from leading physicians, KOLs, and researchers in the field.
Advocacy groups for patients with rare diseases can particularly benefit from having their own registry to drive forward research and speed up the process of finding treatments and even possible cures for patients. We can come with you on that journey.
For more information on Rare Central and how we can work with you and your organization, please visit www.pulseinfoframe.com or email Nina Liu at firstname.lastname@example.org.