Europe – Guideline on clinical investigation of medicinal products for the treatment of gout

Executive summary
The main aim of the guideline is to address general guidance on the development of medicinal products for the treatment of gout. This guideline should be read in conjunction with other EMA and ICH guidelines, which may apply to these conditions and patient populations.
Gout is a common disorder, which is caused by hyperuricaemia and the formation of monosodium urate (MSU) crystal deposits. Crystals accumulate preferentially in joints, tendons and the surrounding soft tissues, and may induce an inflammatory reaction. Gout may manifest as intermittent acute gouty arthritis (referred to as “flares” in this document), with symptom-free periods between attacks. During prolonged hyperuricaemia, tophi, i.e. nodular masses of MSU crystals that form within soft tissue, may occur. Tophi can be symptomless, or can trigger an inflammatory reaction. In severe cases, chronic tophaceous gouty arthropathy characterised by inflamed tophi at multiple joints and bone erosions may occur, where patients are not symptom-free in between flares. Renal damage and kidney stone formation may develop in gout.

Standard of care treatment of gout consists of urate-lowering therapy (ULT). Acute flares are treated with analgesics and anti-inflammatory drugs. In addition, prophylactic treatment with NSAIDs or other drugs is frequently given at the start of ULT, as a sudden drop in uric acid levels induced by ULT may precipitate an acute attack of gout…