New Drug and Biologics Approvals in 2019: A Systematic Analysis of Patient Experience Data in FDA Drug Approval Packages and Product Labels

Schultz-Knudsen, Katrine; Sabaliauskaite, Ugne; Hellsten, Johan; Lassen, Anders B.; Morant, Anne Vinther

doi:10.1007/s43441-020-00244-x

Cited by 2 publications

(1 citation statement)

References 7 publications

(14 reference statements)

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“…Similarly, at NICE in England, patient advocates and expert patients are able to both submit written responses and provide testimony in the deliberative committee meeting, this too is made publicly available in the HTA reports [ 62 ]. In the US since 2017, the FDA have required incorporation of a table (the “PXD Table”) within the assessment report, tabulating the patient experience data included in the submission [ 67 ]. These examples indicate that while there is increasing acknowledgment of patient input, this is currently not consistently or systematically reported, and the impact of the contribution is rarely articulated.…”

Section: Resultsmentioning

confidence: 99%

Patient Engagement and Patient Experience Data in Regulatory Review and Health Technology Assessment: A Global Landscape Review

Bertelsen,

Dewulf,

Ferrè

et al. 2023

Ther Innov Regul Sci

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Background Working with patients through meaningful patient engagement (PE) and incorporating patient experience data (PXD) is increasingly important in medicines and medical device development. However, PE in the planning, organization, generation, and interpretation of PXD within regulatory and health technology assessment (HTA) decision-making processes remains challenging. We conducted a global review of the PE and PXD landscape to identify evolving resources by geography to support and highlight the potential of integration of PE and PXD in regulatory assessment and HTA. Methods A review of literature/public information was conducted (August 2021–January 2023), led by a multistakeholder group comprising those with lived or professional experience of PE and PXD, to identify relevant regulatory and HTA initiatives and resources reviewed and categorized by geography and focus area. Results Overall, 53 relevant initiatives/resources were identified (global, 14; North America, 11; Europe, 11; Asia, nine; UK, six; Latin America, one; Africa, one). Most focused either on PE (49%) or PXD (28%); few (11%) mentioned both PE and PXD (as largely separate activities) or demonstrated an integration of PE and PXD (11%). Conclusions Our analysis demonstrates increasing interest in PE, PXD, and guidance on their use individually in decision-making. However, more work is needed to offer guidance on maximizing the value of patient input into decisions by combining both PE and PXD into regulatory and HTA processes; the necessity of integrating PE in the design and interpretation of PXD programs should be highlighted. A co-created framework to achieve this integration is part of a future project.

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Section: Resultsmentioning

confidence: 99%