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

Autor: Ugne Sabaliauskaite, Anne Vinther Morant, Anders Blaedel Lassen, Johan Hellsten, Katrine Schultz-Knudsen
Rok vydání: 2020
Předmět:
Drug
medicine.medical_specialty
Patient Empowerment
media_common.quotation_subject
Regulatory decision-making
Pharmacy
03 medical and health sciences
0302 clinical medicine
Patient experience
Drug approval
Humans
Medicine
Pharmacology (medical)
Patient Reported Outcome Measures
030212 general & internal medicine
Product (category theory)
Label claim
Drug Approval
Pharmacology
Toxicology and Pharmaceutics (miscellaneous)
Original Research
media_common
Biological Products
Patient-reported outcomes
United States Food and Drug Administration
business.industry
Patient experience data
030503 health policy & services
Public Health
Environmental and Occupational Health
Patient preference
United States
Pharmaceutical Preparations
Drug development
Patient-focused drug development
Family medicine
0305 other medical science
business
FDA
Zdroj: Therapeutic Innovation & Regulatory Science
ISSN: 2168-4804
2168-4790
Popis: 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 decision-making and a clear path to PED-based label claims could incentivize sponsors and enable patient empowerment in treatment decisions.
Databáze: OpenAIRE
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