Adelphi Values demonstrates PROs and ClinROs as ‘fit for purpose’ and FDA approves first-in-class aesthetic product
Adelphi Values is pleased to report that on 29 April 2015 the US FDA granted approval of KYBELLA™ indicated for improvement in the appearance of moderate to severe convexity or fullness associated with submental fat in adults. Developed by Kythera Biopharmaceuticals®, KYBELLA™ is the FIRST and ONLY approved non-surgical treatment for reduction of submental fullness (i.e. chin fat), a common yet under-treated aesthetic condition. In the pivotal Phase III studies, 68.2% of patients responded to KYBELLA™ based on a composite of “fit for purpose” clinician-reported and patient-reported outcomes (ClinRO and PRO). Patients also reported improvement in the emotional impact of submental fat when asked how happy, bothered, self-conscious, embarrassed, old, and overweight they felt following treatment in relation to the amount of their submental fat.
“This is an important achievement for Kythera, and I am delighted that we have been able to support their strong commitment to developing first-in-class aesthetic products in ways that are inventive, forward looking, and methodologically rigorous,” said Alan Shields, Vice President at Adelphi Values US. “We recognize that a patient-centric, appearance-based, and scientifically defensible measurement strategy is increasingly important to regulators and in the evolving aesthetics healthcare market.”
Adelphi Values supported Kythera to confirm that the clinical outcome assessment (COA) tools used in their Phase 2 and Phase 3 studies, including the Clinician-Reported Submental Fat Rating Scale (CR-SMFRS), the Patient-Reported Submental Fat Rating Scale (PR-SMFRS), and the Patient-Reported Submental Fat Impact Scale (PR-SMFIS), produced reliable, valid, and interpretable scores. The development history of these tools, and the results of analyses of their measurement properties, was compiled by Adelphi Values in a measurement dossier that was included by Kythera in the KYBELLA™ New Drug Application (NDA). This dossier provided critical evidence that helped the FDA Dermatologic and Ophthalmic Drugs Advisory Committee vote unanimously to recommend approval of the treatment in March 2015 and, ultimately, the division’s decision to grant full approval in April 2015. Alan Shields worked with his colleagues at PRO Consulting and Health Research Associates, Inc. to support Kythera in the original development of the CR-SMFRS, PR-SMFRS, and PR-SMFIS.