SP0055 Licensing New Drugs in SLE: The EMA Draft Guideline

The European Medicines Agency has released the draft Guideline on clinical investigation of medicinal products for the treatment of systemic lupus erythematosus, cutaneous lupus and lupus nephritis. A request for input and comments to this draft guideline by September 2013 is made. Guidelines are intended to provide a basis for practical harmonisation of the manner in which the EU Member States and the European Medicines Agency interpret and apply the detailed requirements for the demonstration of quality, safety and efficacy contained in the Community directives. Guidelines direct drug developers in their clinical programme such that: - O The requirements of and the options for how to demonstrate quality safety and clinically relevant efficacy are clear - O Provide an EU agreed position on the requirements This presentation will give an overview of the process of guideline development and specifically address the main issues required for the demonstration of efficacy in SLE, cutaneous lupus and lupus nephritis. It is highlighted that in order to demonstrate a reduction disease activity, patients need to have a clinically important and sufficient level of disease activity prior to treatment in order to demonstrate a significant change. The use of composite scores such as BILAG, ECLAM, LAI, SLEDAI and SLAM, ideally a combination of these, is considered appropriate and may be complemented with global patient assessments of the disease with visual analogue scales and health related quality of life. The efficacy aims can be considered as - reduction in disease activity (induction) - reduction in flares (maintenance) - prevention of long-term damage. Efficacy should be demonstrated preferably through validated composite indexes in which the effect seen in an objective measure of reduction in global disease activity is not offset by worsening of the subject’s condition overall or worsening in any specific organ system. Additional guidance on cutaneous disease and lupus nephritis as well as endpoints for juvenile SLE are provided. While many external comments received on the original concept paper for SLE released by the EMA have been incorporated, there are limitations in the guidance specifically in relation to certain complications of SLE such as CNS disease. The absence of specific guidance on some features of SLE reflects a lack of validated diagnostic and assessment criteria which could serve as reliable efficacy endpoints. The draft guideline is published on the EMA website for external comments. Once the consultation period ends all external comments received will be reviewed and the draft guideline will be amended as required before release of the final guideline. Disclosure of Interest None Declared