Implementing the Kidney Health Initiative Surrogate Efficacy Endpoint in Patients With IgA Nephropathy (the PROTECT Trial)

<p>There has been little progress in the development</p><p>and regulatory approval of novel therapies for</p><p>glomerular diseases. There are several reasons for this</p><p>dilemma, including safety and efficacy of tested therapies,</p><p>the slowly progressive nature of glomerular</p><p>diseases, challenges with clinical trial design, and the</p><p>traditional endpoints required by regulatory agencies</p><p>for drug labeling. This is compounded by the fact that</p><p>most primary glomerular diseases are recognized</p><p>internationally as rare diseases. The time required and</p><p>feasibility to conduct large-scale phase 3 clinical trials</p><p>to evaluate whether a new therapy improves kidney</p><p>survival and decreases the development of end-stage</p><p>kidney disease (ESKD) is prohibitive, particularly</p><p>when that disease is rare. Even using doubling of</p><p>serum creatinine concentration, an accepted surrogate</p><p>endpoint of ESKD, requires expensive trials with</p><p>lengthy follow-up.</p>