- The Food and Drug Administration has unveiled its MyStudies app, a tool aimed at collecting patient-generated health data (PGHD) and other information to inform regulatory decision-making and clinical trial development.
As the healthcare industry continues to embrace patient-centered care and consult patient preferences for and experiences with care, researchers are looking to integrate the patient into research opportunities. Specifically, those in charge of clinical trials are looking to create trials that glean information important to the patient and with patient interests in mind.
Additionally, understanding how a patient interacts with a medical product can help FDA make regulatory decisions about the tool, according to FDA Commissioner Scott Gottlieb.
“There are a lot of new ways that we can use real world evidence to help inform regulatory decisions around medical products as the collection of this data gets more widespread and reliable,” Gottlieb said in a statement. “Better capture of real world data, collected from a variety of sources, has the potential to make our new drug development process more efficient, improve safety and help lower the cost of product development.”
This type of data can also help incorporate the patient voice in decisions about which tools to adopt into a care management routine.
“If done right, it can also help make sure doctors and patients are better informed about the clinical use of new products, enabling them to make more effective, efficient medical choices,” Gottlieb explained. “This will ultimately help us achieve better outcomes, and safer and more efficient use of expensive technology.”
The MyStudies app, piloted in partnership with Kaiser Permanente, is geared toward meeting those patient-centric needs. The tool collects patient information from the patient portal, EHR, claims and billing documents, and product and disease registries. Patients can also offer PGHD from their own medical record-keeping, mHealth apps, wearable tools, or remote patient monitoring devices.
FDA has released the open source codes to health IT developers and regulatory policymakers. Organizations can now adapt the code to meet the specific needs of their clinical trials, patient populations, and healthcare developments. This will allow stakeholders to understand patient experiences with healthcare and technology and to also design clinical trials.
In using the open source code from FDA, stakeholders can ensure they meet FDA standards for data integrity, the agency said.
For example, a data collection app for an individual clinical trial can now integrate the MyStudies app functions. This will allow stakeholders to collect patient-reported outcomes, symptoms, and other PGHD, FDA explained.
“This digital platform enables developers to adapt our technology to advance new ways to access and use data collected directly from patients—with the necessary controls in place to ensure patient privacy,” Gottlieb said. “Our hope is that the collection of more real world data directly from patients, using a secure app, will lead to more efficient product development and assist with safety monitoring.”
FDA’s efforts to develop MyStudies were led by David Martin, MD, associate director for real world evidence in the Office of Medical Policy at the FDA’s Center for Drug Evaluation and Research. Martin and his team leverage a grant from the HHS Patient Centered Outcomes Research Trust Fund.
Coding was developed in partnership with Harvard Pilgrim Health Care Institute, LabKey, and Boston Technology Corporation.
The MyStudies app developments come on top of other FDA efforts to put patients at the center of medical device development. As the healthcare industry continues to integrate the patient voice as an integral part of care, FDA aims to make medications and medical devices align with patient needs.
“Our work demands that we must continue to reflect on how we can make the science of drug development and review more modern and more patient-centered, so that approved products impact the metrics that real-world patients and families value most,” FDA Commissioner Scott Gottlieb wrote in a June statement.
“Done well, the result is more and more of our review of new medical products benefits from a better understanding of the patient’s experience, providing our reviewers with the critically-important context of a disease, and helping them to understand what’s most important to patients related to treatment benefits, risks and disease burden,” Gottlieb continued.
FDA has gone to lengths to follow through on that promise. In March 2018, the agency partnered with the National Organization for Rare Diseases (NORD) to hold listening sessions with patients with rare diseases to better understand patient experiences with treatment and allow FDA to create patient-centered research opportunities.
Just over one year ago, FDA also held its first Patient Engagement Advisory Committee (PEAC) meeting. The PEAC meeting sought to better understand patient, family caregiver, and patient advocate experiences with care that could impact FDA regulatory actions.
“The FDA’s work requires us to establish objective, consistent criteria on which we base our decisions,” Gottlieb wrote in a statement prior to the event. “But ultimately, the criteria we use to judge benefit and risk turn on the parameters that matter to patients. Involving the end-user – the patient – in identifying health priorities and outcomes desired from health interventions is critically important.”