The use of digital tools means that longitudinal real-world evidence studies can be considered as adaptive registries, with a powerful range of both pre- and post-approval use cases.

Pre-approval use cases include granular understanding of burden of disease, healthcare resource utilization, and patient journey and treatment patterns for audiences such as regulators and health technology assessment.

Once a sponsor’s medicine is approved, they have safety reporting obligations to the FDA, EMA, and MHRA (UK), among other local regulatory authorities. These obligations are non-trivial and include the need to identify adverse events that are actual, suspected, unexpected, etc., and determine whether they are serious or not. Moreover, the sponsor is obliged to be proactive in ensuring that safety information can be reported wherever data on their product are being collected.

These obligations, however, do not mean that pre-approval RWE studies need to stop. Rather, if safety reporting is built in at the time of approval, the study can run uninterrupted and with greater confidence on the part of the sponsor.

This brings with it the opportunity to fully exploit post-approval use cases and continue to build invaluable longitudinal data. Post-approval use cases include:

  • Meeting regulatory requirements (e.g., PASS or REMS requirements, depending on the regulator)
  • Generating data for HTA/pricing and reimbursement (e.g., conditional approvals, coverage with evidence development, performance-based risk sharing, value-based contracting, managed-entry agreements, and so on)
  • Generating publications and further strengthening links with stakeholders such as key opinion leaders and patient advocacy groups, particularly as they may have an array of safety concerns very separate to those of regulators or payers.

This could also include payer-led arrangements – which can vary by country – where safety information and more may be required to be collected in a patient registry, meaning that an ongoing registry built for interoperability could be immediately plugged into such a reporting system.

Our proprietary Vitaccess Real™ platform includes a safety reporting module, offering adverse event (side effects) reporting, which can be flexibly deployed to meet a sponsor’s reporting obligations – addressing, for instance:

  • What events are reported
  • Whether severity or seriousness is measured, and how
  • Whether there is adjudication by a healthcare professional, or it is entirely patient-reported
  • Whether the events need to be reported to regulatory authorities
  • Whether the treatments/healthcare responses, and their outcomes, need to be reported
  • Whether treatment cessation, dose adjustment, etc., have to be reported
  • How these data need to be reported, to whom, and within what timeframe.

In this regard, our platform is designed to be almost endlessly customizable. The aim is to deploy a registry that continues to create and deliver value as far into a product’s lifecycle as possible – well beyond simply approval.

To find out how we can help you to develop a powerful digital real-world study that meets your safety reporting obligations, get in touch here.

By Mark Larkin & Casey Quinn



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