An Updated Review of Food and Drug Administration Warning and Untitled Letters for Clinical Outcome Assessment Claims Between 2013 and 2021.

Objectives: We aimed to ascertain the frequency and types of Food and Drug Administration (FDA)-identified clinical outcome assessment (COA) violations in US pharmaceutical promotional materials from 2013 to 2021 by updating the review by Symonds et al (covering the period 2006-2012 for patient-reported outcomes [PRO] measures), expanding to infringements across all types of COAs, and grouping by efficacy or overall patient experience.

Methods: Warning letters (WLs) and untitled letters (ULs) issued by the FDA's Office of Prescription Drug Promotion were reviewed for COA violations and classified by their type: (1) COA measure not fit for purpose, (2) issue with study design or interpretation of results, and (3) inadequate statistical analysis.

Results: From 2013 to 2021, the FDA issued 22 WLs and 65 ULs.

Clinical outcomes following bone marrow transplantation in patients with sickle cell disease: A cohort study of US Medicaid enrollees.

Objectives: Bone marrow transplantation (BMT) is currently the only curative therapy available for patients with sickle cell disease (SCD), but clinical outcomes in routine care are not well understood. We describe the rates of vaso-occlusive crises (VOCs), transplant complications, and mortality in SCD patients after BMT.

Methods: A cohort study of SCD patients who underwent BMT was designed using US Medicaid claims data (2000-2013).

Clinical outcomes and healthcare utilization in patients with sickle cell disease: a nationwide cohort study of Medicaid beneficiaries.

To add to the limited existing evidence on clinical outcomes and healthcare use in sickle cell disease (SCD) among beneficiaries of the US Medicaid program, we conducted a cohort study using nationwide Medicaid claims data (2000-2013). Patients were included based on HbSS SCD diagnosis and followed until Medicaid disenrollment, death, bone marrow transplant, or end of data availability to assess vasoocclusive crises (VOC), emergency room (ER) visits, hospitalizations, outpatient visits, and blood transfusions. Annualized event rates (with 95% confidence intervals [CI]) were reported.

Treatment patterns and medication adherence among patients diagnosed with multiple myeloma and treated with panobinostat.

Aim: To examine real-world treatment patterns in multiple myeloma (MM) patients treated with panobinostat.

Materials & Methods: Using a US claims database, MM patients treated with panobinostat during 02/01/2015-01/31/2017 were evaluated. Lines of therapy, combination regimens, dosing and duration were measured.

Patient-Reported Outcomes in Oncology Drug Labeling in the United States: A Framework for Navigating Early Challenges.

Background: Despite an increased use of patient-reported outcomes (PROs) in oncology clinical trials, integrating the patient perspective into drug approval decisions and documentation has been challenging.

Objectives: To review important regulatory and measurement terminology, and to provide oncology outcomes researchers and those involved with building oncology programs with tools to plan PRO data collection, particularly in relation to drug efficacy claims for drug labeling in the United States.

Discussion: When contemplating a PRO measurement strategy for oncology clinical trials, outcomes researchers are challenged in several ways.