New Drug and Biologics Approvals in 2019: A Systematic Analysis of Patient Experience Data in FDA Drug Approval Packages
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ORIGINAL RESEARCH
New Drug and Biologics Approvals in 2019: A Systematic Analysis of Patient Experience Data in FDA Drug Approval Packages and Product Labels Katrine Schultz‑Knudsen, MSc1 · Ugne Sabaliauskaite, MSc2 · Johan Hellsten, PhD, MSc3 · Anders Blaedel Lassen, MA, MSc, LLM3 · Anne Vinther Morant, PhD, MSc4 Received: 8 September 2020 / Accepted: 10 November 2020 © The Author(s) 2020
Abstract Background The FDA Patient-Focused Drug Development Initiative was launched to ensure the incorporation of the patient voice into drug development and evaluation. Since 2017, the FDA must publish a statement outlining patient experience data (PED) considered in the approval of new drugs. This study investigated the presence and role of PED in drug approval and translation into product label claims. Methods PED reported in approval packages of the 48 drugs approved by FDA’s Center for Drug Evaluation and Research in 2019 was identified and categorized. PED in the form of clinical outcome assessments (COAs) was characterized by endpoint positioning and outcome. The product labels were analyzed for PED-related claims. Results PED was reported as relevant for 39 of 48 (81.3%) drugs approved in 2019. COAs were the predominant PED type; other PED was identified for only 9 (18.8%) drugs, and none included qualitative or patient preference studies. COAs were the only type of PED for which associated claims were identified in the product labels. 27 out of 48 (56.3%) labels contained one or more efficacy claims based on COAs; of these, patient-reported outcomes were the most prevalent with claims identified in 19 labels (39.6%). Conclusion There are ample opportunities for incorporating PED beyond COAs to inform drug development and facilitate availability of medicines tailored to patient needs. A higher level of transparency on the role of PED in regulatory decisionmaking and a clear path to PED-based label claims could incentivize sponsors and enable patient empowerment in treatment decisions. Keywords Patient experience data · FDA · Patient-focused drug development · Label claim · Regulatory decision-making · Patient-reported outcomes
Introduction In recent years, patient contributions to the regulatory ecosystem have evolved from what was primarily a process scope to also play a central role in evidence generation. * Anne Vinther Morant [email protected] 1
Regulatory Science & Strategy, H. Lundbeck A/S, Valby, Denmark
2
Regulatory Dossier Management, H. Lundbeck A/S, Valby, Denmark
3
Patient Insights, H. Lundbeck A/S, Valby, Denmark
4
Anne Morant Consulting, Frederiksberg, Denmark
Leading regulatory agencies have embarked on different initiatives aimed at strengthening the patient relevance in drug development and regulatory decision-making. Examples include the US Food and Drug Administration’s (FDA) Patient-Focused Drug Development (PFDD) program [1]; the European Medicines Agency’s Regulatory Science Strategy to 2025 [2]; and Japan’s Pharmaceutical and Medical Devices Agency’s ‘Patients First’ in
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