FDA’s draft guidances on real-world data do not provide strict requirements for industry stakeholders to abide by, but they offer patient advocacy organizations with a clearer understanding of how to model their registries to have a more substantial impact for their patient communities.
Having a full view of a patient’s health story through real-world data (RWD) and evidence (RWE) is the key to improving health outcomes and advancing treatment options. Yet, many historical challenges have slowed consistent adoption, such as data standards, data holders disregarding and blocking the sharing of information, and a lack of awareness among a broader population. In response, the 21st Century Cures Act in 2016 prompted government agencies such as the Center for Medicare and Medicaid Services (CMS), the Office of the National Coordinator for Health IT (ONC), and the Federal Drug Administration (FDA) to publish regulations and guidance to address these issues.
Since then, industry stakeholders have increased their understanding of the importance of electronic health data, including patient registries, in the clinical care and research process. In tandem, patient registries have been exploring how to become strong industry partners by providing the data needed to improve the treatments available to their patient communities. The recent draft guidances released by the FDA in late 2021 and early 2022 offer broad recommendations for using real-world evidence in drug development.
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While these draft guidances do not provide strict requirements for industry stakeholders to abide by, they offer patient advocacy organizations with a clearer understanding of how to model their registries to have a more substantial impact for their patient communities.
What is in this draft guidance?
To successfully utilize registry data in drug submissions, the FDA released this draft guidance to provide recommendations on evaluating the relevance and reliability of a potential registry partner. Also included are several considerations for linking registry data to other sources to provide supplemental information, including EHRs, digital health technologies, and other registries.
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How does this impact patient advocacy organizations?
This draft guidance is the exemplary roadmap for how patient advocacy organizations should model their registries to build industry partnerships and maintain relevance. The key features that the FDA recommends include relevance of the registry data for a given study, the reliability of the registry data information and quality, and the ability of the registry data to be linked with other systems. Patient advocacy organizations should implement these recommendations now to ensure success long term.
What is in this draft guidance?
In this draft guidance, the FDA outlined the best practices for the use of RWD for investigational new indication of drug (IND) applications. Research for INDs aim to gain approval for new uses of existing drugs (i.e., a drug for epilepsy being approved for alternative use to treat migraines). Best practices outlined in this guidance include clear documentation on all the data analyses performed during the study design phase when submitting the application to the FDA.
How does this impact patient advocacy organizations?
Through this draft guidance, patient advocacy organizations will be able to play a major role in advancing IND studies as a trusted data source. Historically, some of the biggest treatment advancements for many disease groups have come from utilizing drugs that are already established. By supporting the IND process, patient advocacy organizations have new opportunities to achieve their core mission of finding better treatments for their patient communities. Getting involved with IND studies is also a way for patient advocacy organizations with strong registry data to strengthen business sustainability.
Guidance: Data Standards for Drug and Biological Product Submissions Containing Real-World Data
What is in this draft guidance?
The FDA suggests that all RWD, including EHR and registries, that is submitted for review and approvals should be in a standard electronic format. This draft guidance outlines the necessary standards that industry stakeholders must comply with when submitting any study data that includes RWD. While the FDA acknowledges that there is a wide range of approaches to data transformation, they will require detailed documentation and the rationale behind the specific approach used.
How does this impact patient advocacy organizations?
The purpose of this draft guidance is to promote the ease of interoperability (i.e., the sharing of data) by creating compatibility standard. The ability to bring together insights from multiple sources helps provide valuable insights to patients, clinicians, and researchers, about the manifestation of different diseases. This is important to patient advocacy organizations because it empowers their patient communities to make more informed decisions about their health and increases the likelihood of successful treatment development.
What is in this draft guidance?
The FDA goal with this draft guidance is to provide recommendations for appropriate study designs using RWD sources prior to submission to the agency for approval. By outlining study design considerations, the FDA is aiming to ensure the safety and efficacy of drugs submitted for approval, including data source relevance and validity of data sources.
How does this impact patient advocacy organizations?
The fundamental aim of this draft guidance is ensuring drugs endorsed by the FDA improve health outcomes while also reducing the risk of potentially life-threatening side effects. By encouraging researchers to submit EHRs and registry data directly to the FDA, it will reduce provider burden and help with site recruitment and retention. Improved site recruitment and retention is important to patient advocacy organizations because it is vital to successful treatment advancements for their patient communities.
Overall takeaways for patient advocacy organizations
These guidances provide patient advocacy organizations with the opportunity to increase their influence in advancing drug developments that will keep patients at the center of the conversation. Because these draft guidances are recommendations, not requirements, the FDA is sending a signal to stakeholders that they are open to continuing a dialogue on key priorities.
When it comes to next steps for organizations, creating a systematic strategy that incorporates all data handling best practices that the FDA lays out in these draft guidances, will be critical. This includes cross-checking registry data against the ONC’s USCDI requirements for interoperability and providing clear documentation about the relevance and reliability of the data for a stakeholder’s specific goals. Reducing the burden on stakeholders and establishing trust in the value of an organization’s registry will be the key to sustainability and advancing treatment options for their patient communities.
Photo: Getty Images, Sarah Silbiger
Jamie Skipper
Jamie Skipper, BSN, PhD, joined IQVIA in 2021 to leads assessments of the technical and operational requirements of clinical data registries for not-for-profit clients. She serves as a senior expert for client-centric design of innovative solutions and for client registry program strategic planning. She is also a senior expert on thought leadership around IQVIA’s agile registry and integrated health platform solutions.Prior to IQVIA, Jamie served as a federal official at HHS where she directed research funding at NIH, helped craft FDA guidance around the use of EHRs in research, and created the ONC PCOR program to create policies and technical requirements for the use of EHI in research
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