FDAnews Drug Daily Bulletin

EMA Releases Lupus Trials Guideline

April 3, 2015

For the first time, the European Medicines Agency has produced a guideline on clinical trial design for drugmakers developing targeted products to treat lupus — an effort by the EMA to encourage more companies to target the underserved disease.

The guideline lays out how trials should be designed to show either complete remission or major or partial clinical response specific to lupus. Most lupus treatments, such as corticosteroids or antimalarials, are not specific to the disease, according to the Lupus Foundation of America. Only GlaxoSmithKline’s Benlysta (belimumab) targets lupus specifically.

Under the new guideline, to demonstrate complete remission, patients must show no lingering lupus symptoms and require no ongoing drug treatment, the EMA says. Major or partial clinical responses should be measured by disease scales, including the Systemic Lupus Erythematosus Disease Activity Index that assigns points commensurate with onset and severity of symptoms.

To demonstrate short-term efficacy requires four-to-eight week trials, but establishing longer-term or chronic disease outcomes may require trials lasting up to a year.

In addition to establishing clinical efficacy, sponsors should also include patient-centric response measures, such as health-related quality of life, in their trials as well, the guideline says. That could be in the form of tools such as the Lupus Quality of Life checklist, or it could be in measuring levels of one specific serious symptom, such as fatigue.

The EMA prefers double-blind, parallel group, randomized superiority trials evaluating the study drug against an active comparator. If a sponsor chooses to use a placebo as a comparator, then patients on placebo must be given current standard of care, the EMA says.

Studies specifically targeting forms of lupus that primarily affect the kidneys should use outcomes such as improvement in protein or other sediments in patient urine or improvements in creatinine levels in the blood that specifically measure kidney symptoms, and patients should be followed for three to six months to track partial responses, and for at least a year to document a complete response. Preventing kidney flare-ups or long-term organ damage could also be primary endpoints in these cases, the EMA says.

The guideline, approved by the Committee for Medicinal Products for Human Use, will take effect Sept. 1. Read it at www.fdanews.com/03-27-15-EMAlupusguideline.pdf. — Lena Freund