In the patient-reported outcomes (PROs) field, strict regulatory requirements must be met for qualitative research that contributes to labeling claims for medicinal products. These requirements not only emphasize the importance of reaching saturation but also of providing documentary evidence that saturation has been reached. This paper reviews qualitative literature for useful definitions of the concept and for practical approaches for assessing saturation. The paper considers approaches in light of the rigorous regulatory requirements for PRO research that are used to support labeling claims for medicinal products and the wider requirements for flexibility and creativity in qualitative research in general. This assessment is facilitated by the use of examples from our past qualitative PRO studies. Based on conclusions from this assessment, we offer preliminary recommendations for future qualitative PRO studies for assessing and documenting saturation.

IUSS is a validated patient reported measure of urgency severity for collecting event specific information in the context of a clinical trial.

LGS is a severe form of childhood epilepsy which is characterized by multiple seizures and cognitive impairment. Semi-structured interviews were conducted with 40 parents of children with LGS in the US, UK, and Italy. Parents were asked to report on their perceptions of the HRQL of their child and also to describe the impact on their own HRQL. Thematic analysis was conducted to develop themes relating to the impact on HRQL. The themes were organized into conceptual models of the impact of LGS on the HRQL of the parent and the child. The models demonstrate the complex relationships between the components of LGS and their impact on HRQL.

In multiple myeloma (MM), health-related quality of life (HRQoL) data is becoming increasingly important, owing to improved survival outcomes and the impact of treatment-related toxicity on HRQoL. Researchers are more frequently including HRQoL assessments in clinical trials, but analysis and reporting of this data has not been consistent. A systematic literature review assessed the effect of novel agents (thalidomide, bortezomib and lenalidomide) on HRQoL in MM patients, and evaluated the subsequent reporting of these HRQoL results. A relatively small body of literature addresses HRQoL data in MM patients treated with novel MM therapeutic agents: 9 manuscripts and 15 conference proceedings. The literature demonstrates the complementary value of HRQoL when assessing clinical response, progression, overall survival and toxicity. However, weaknesses and inconsistencies in analysis and presentation of HRQoL data were observed, often complicating interpretation of the impact of treatment on HRQoL in MM. Further evaluation of HRQoL in MM patients treated with novel agents is required in larger cohorts, and ideally in head-to-head comparative studies. Additionally, the development of standardised MM-specific best practice guidelines in HRQoL data collection and analysis is recommended. These would ensure that future data are more useful in guiding predictive models and clinical decisions.