Characterization of the Clinical Evidence Supporting Repository Corticotropin Injection for FDA-Approved Indications:...

Area of Science:

• Pharmacology and Therapeutics
• Evidence-Based Medicine
• Clinical Trial Analysis

Background:

• Repository corticotropin injection, approved in 1952, is an expensive medication for inflammatory conditions.
• The clinical evidence supporting its use is limited, potentially due to pre-modern FDA review standards.

Purpose of the Study:

• To systematically evaluate the clinical evidence supporting repository corticotropin for its FDA-approved indications.
• To characterize the quality and findings of studies evaluating corticotropin's efficacy.

Main Methods:

• Conducted a comprehensive literature search across multiple databases (MEDLINE, CINAHL, CENTRAL) up to May 2021.
• Included randomized clinical trials (RCTs), nonrandomized trials, and cohort studies, extracting data for qualitative synthesis.
• Searched for terms including HP Acthar, ACTH gel, repository corticotropin, and specific disease indications.

Main Results:

• Analyzed 41 studies involving 2235 participants across various indications like infantile spasms, multiple sclerosis (MS), and nephrotic syndrome.
• Evidence was strongest for infantile spasms and MS, with MS studies showing corticotropin superior to placebo but not corticosteroids.
• For infantile spasms, only one small RCT found corticotropin superior to corticosteroids; other conditions had limited or no-control studies.

Conclusions:

• Few randomized controlled trials support the clinical benefit of repository corticotropin for most FDA-approved indications.
• Most RCTs indicate corticotropin is not superior to corticosteroids for treating relapses of MS or infantile spasms.
• The review highlights a need for more robust evidence to support corticotropin's use in many inflammatory conditions.