Patient-reported outcomes for US oncology labeling
Oncology is an area that is notoriously hard to achieve patient-reported outcome label claims, but experts at Adelphi Values continue to evaluate ways to work with the FDA guidelines to ensure data are robust and stand up to the scrutiny of regulators. To demonstrate this, and in collaboration with Novartis, we have published an article in the journal Expert Review of Pharmacoeconomics & Outcomes Research, entitled, Patient-reported outcomes for US oncology labeling: review and discussion of score interpretation and analysis methods. This paper approaches the conceptual and practical challenges associated with interpreting the clinical meaning of scores produced by patient reported outcome (PRO) questionnaires used to inform efficacy decisions for regulatory approval.
According to Alan Shields, Vice President of Adelphi Values US and lead author of the manuscript, “the issue of ‘score interpretation’ poses a challenge to the industry and we wanted to provide an accessible and practical perspective for outcomes researchers. Though the paper has an oncology focus, we feel that the summary of the history of terms such as “Minimal Important Difference” or “MID”, the review of the FDA position on the topic along with a depiction of sponsor challenges and success, and the illustration of available data analytic techniques are relevant and applicable to all outcomes researchers independent of therapeutic area.”
Below is the full citation and here is the link to the abstract and article.
Shields AL, Coon C, Hao Y, Krohe M, Yaworsky, Mazar I, and Foley C (2015). Patient-reported outcomes for US oncology labeling: review and discussion of score interpretation and analysis methods. Expert Review of Pharmacoeconomics & Outcomes Research. 15(6), 951-959.