Abstract

In the rapidly evolving landscape of healthcare, the introduction of new technologies—from novel therapeutics to innovative medical devices—continues to promise meaningful advances in patient care, outcomes, and overall health. However, new innovations often present high costs and may require significant changes in medical practice to adopt effectively, especially some of the most potentially transformative therapies. Additionally, for these new technologies, data on longer-term outcomes that matter to patients and how they are being integrated with other key components of a patient-centered care model, such as diagnostics, patient engagement supports, and complementary therapies, are important.

Clinical trials serve as the basis for approving such technologies and are themselves evolving into being more patient centric and community based. Use of data in the “real world,” including from electronic health records (EHRs), provides a basis for further understanding the impact that these technologies have on the lives of patients and their caregivers across diverse care systems, as well as the coverage, payment, and other reforms needed to ensure patients have access to treatments that deliver value. Payment and regulatory agencies across the world are deploying new strategies for generating ongoing evidence post approval from the US Food and Drug Administration, for example, coverage with evidence development and now Transitional Coverage for Emerging Technologies in the United States, although stakeholders have significant disagreements about the impact of these tools on patient access, care affordability, and biomedical innovation. The Centers for Medicare & Medicaid Services, similar to many other global payers, is also seeking to use qualitative evidence related to value in its implementation of price negotiations under the Inflation Reduction Act. The Centers for Medicare & Medicaid Services has highlighted its desire to encourage and reward more and better real-world evidence development relevant to Medicare beneficiaries through this process, but whether and how this will happen is not yet clear and will also likely lead to further debates about how to develop and use evidence on value.