Patient-reported outcome claims in European and United States orphan drug approvals

Abstract

Purpose: We aimed to evaluate the rate of usage and the kind of patient-reported outcome (PRO) claims in orphan drug approvals from the European Medicines Agency (EMA) dated between 1/1/2012 and 31/12/2016 and to compare them to those from the US Food and Drug Administration (FDA).Methods: Orphan drug approval documentation was obtained from the EMA website. PRO-related language was extracted from the Summaries of Product Characteristics (SmPCs). Data were compared to a previously published analysis of the FDA approvals from the same time period.Results: Out of 60 approvals that met the inclusion criteria, 12 products approved by the EMA for 13 (21.7%) orphan indications contained PRO language in the Clinical Studies section of the SmPC. Twelve SmPCs contained PRO instruments based on symptoms, five of which also concerned patient functioning. Eight approvals included PRO claims related to quality of life (QoL) most commonly in cancer treatment.Conclusion: The rate of PRO claims was lower for orphan drugs specifically than for all drug approvals by the EMA. However, in accordance with previous findings, the EMA appeared more inclined to grant PRO claims including health-related QoL than the FDA.

Gene Therapy in Hemophilia: A Transformational Patient Experience

“…While decision-making bodies have historically marginalized PRO tools and the patient experience data they generate, these are now included in regulatory guidelines to support labeling claims. 47,48 Furthermore, PROs can provide comprehensive data and important nuances to inform the risk-benefit profile of a new product, particularly in rare diseases such as hemophilia where trial populations may be smaller than ideal 49 ; they could also provide more accessible and relevant data for PWH to enable shared decision-making. 50 However, studies are needed to develop GT-specific PRO tools in PWH or adapt existing tools for PWH receiving GT.…”

Patient-reported outcomes in Gaucher’s disease: a systematic review

“…In recent years, some countries and regions, such as the European Union and United States, have made increasing efforts to incorporate the patient voice into drug development. Between 2012 and 2016, orphan drug approvals by the European Medicines Agency and U.S. Food and Drug Administration were mainly focused on rare drugs, with 21.7% and 9.0% of all approved orphan drugs applying for PROs, respectively [ 57 , 58 ]. Therefore, the use of PROs should be expanded to include studies of reported outcomes in patients with GD in order to better help patients in making decisions about disease management and health technology assessment.…”

Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU

“…Finally, a study looking into establishing the use of PROs in the approval of orphan medicines, revealed that, in the European context, PRO use in orphan medicines was lower when compared with all medicines’ approval. Nonetheless, when compared to the results from a similarly conducted study in the United States of America, the authors state that FDA approvals included significantly less PROMs in their processes in comparison with the EMA approvals, during the study period ( 60 ). This could be due to a relatively longer experience within the European context following the publication in 2005 of the HRQoL guidance, which suggests that regulatory guidance can stimulate the use of PROs ( 60 , 61 ).…”