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Case study

Validating a PRO Measurement Strategy for FDA Approval

Fatigue is a symptom associated with many rare diseases, such as multiple sclerosis, lupus, Addison’s disease, Churg-Strauss syndrome and many others. For one company’s new rare disease therapy, the FDA recommended use of a patient-reported outcome (PRO) instrument to measure impact on fatigue and patient function. With evidence from analog diseases and use of the SF-12 Health Survey, QualityMetric helped organize a formal PRO evidence dossier for FDA review. In a free case study, learn more about the process of developing a validation approach for existing instruments and how they can be used in combination to secure new label claims for rare disease therapies.

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  • The validation of existing fatigue measurement instruments in new rare diseases
  • The value of using multiple PRO instruments to support label claims for therapies
  • The use of the SF-12v2 to understand the link between fatigue and physical function

The SF-12 Health Survey measures domains beyond fatigue, such as physical function and mental well-being. Responses to the SF-12 Health Survey could be translated into outcomes that payers and providers value, namely improvements in physical and mental health wellness. This case study discusses the reliability, validity and responsiveness of health surveys and their value in supporting formal evidence dossiers for FDA review.