FDA approves PRO labelling claims in rare disease
We are delighted to report that on February 18 2014, Chelsea Therapeutics announced that the US Food and Drug Administration (FDA) granted accelerated approval of NortheraTM (Droxidopa) for the treatment of symptomatic neurogenic hypotension (NOH). Not only is this the first new treatment for NOH in nearly 20 years, but it is the FIRST and ONLY FDA approved therapy to demonstrate symptomatic benefit to patients with NOH, namely orthostatic dizziness, light headedness, or feeling like you might black out. Please see NortheraTM full prescribing information at www.chelseatherapeutics.com.
Adelphi Values worked with Chelsea Therapeutics to evaluate the extent to which an existing patient-reported outcome (PRO) questionnaire, the Orthostatic Hypotension Questionnaire (OHQ), was developed in ways consistent with FDA standards. Once gaps were identified, a set of research activities were conducted including a literature review, interviews with therapeutic area experts, and qualitative concept elicitation and cognitive debriefing interviews with NOH patients. Once content validity was established in the questionnaire, Adelphi Values evaluated the psychometric performance and interpretability of scores produced by the OHQ. The collected information, along with the development history of the tool, was compiled by Adelphi Values into an evidence dossier to document that the OHQ was a well-defined and reliable measure, capable of substantiating treatment benefit claims for patients with symptomatic NOH. Finally, Adelphi Values supported Chelsea Therapeutics in preparing for and attending an FDA Advisory Committee to answer measurement questions and provide explanation of the information within the dossier. Ultimately, the OHQ generated the data to support the primary endpoint and the resulting indication statement for the Northera™ label.
“This is a significant achievement for Chelsea Therapeutics, and I am delighted that we have been able to support them in providing evidence that legitimizes and substantiates the patient perspective” said Alan Shields Vice President Endpoint Development & Outcomes Assessment at Adelphi Values. “We recognise that a patient centric approach is increasingly important in the evolving healthcare market, especially within rare diseases”
According to a recent news release, Judy Biedenharn, Co-President of the Multiple Systems Atrophy (MSA) Coalition said “This approval is a significant step forward for people with MSA forced to cope with the debilitating effects of NOH,” and Joseph G. Oliveto, President and Chief Executive Officer of Chelsea Therapeutics said “The approval of NortheraTM is an extraordinary achievement, one for which I thank patients and their families, investigators and the medical community, our partner Dainippon Sumitomo, and our employees,”
Established in 1993, Adelphi Values has been a global leader in patient-centred and healthcare value research for over 20 years. Our in-depth understanding of approaches for the selection, development, validation and implementation of PRO instruments is grounded in a solid understanding of regulatory expectations and standards as well as experience in commercialisation and market access.