Chronic glucocorticoid exposure, even at low doses, is now seen by regulators and clinicians as an unacceptable safety burden. Sponsors that prioritize steroid-toxicity from the outset position themselves for label differentiation and stronger value propositions.

If you are planning to include a steroid-sparing claim in your Target Product Profile (TPP), you need to define it early, measure it rigorously, and align your evidence plan to meet regulatory and payer expectations.

Validated tools, trusted trial results

The Steritas Glucocorticoid Toxicity Index (GTI), developed by an international expert panel, provides clinician-reported scores that quantify cumulative and reversible steroid harm.

To integrate steroid-sparing effectively in a drug development project, sponsors must define it explicitly in the TPP, select validated measures to quantify it, and ensure their development program is powered to detect meaningful differences in steroid-toxicity scores. Establishing the steroid-sparing endpoint during early planning helps align protocol design, endpoint selection, and regulatory dialogue.

Trials designed to demonstrate that a drug is steroid-sparing increasingly include mandatory taper protocols to test whether investigational therapies can maintain disease control without steroid support. Endpoints such as the percentage of patients who reach zero prednisone by month six, or changes in GTI scores, are becoming more common.

Sponsors are planning ahead for events like disease flares that need a rescue course of steroids by clearly outlining how these situations will be handled in their trial design and using toxicity scores to measure their impact on study results.

Regulatory and competitive differentiation

The FDA and EMA recognize steroid-sparing as a clinically meaningful benefit, especially when supported by validated instruments. Early engagement with regulatory agencies or scientific experts helps sponsors socialize these goals and secure alignment.

Recent approvals, including avacopan for ANCA vasculitis, show that demonstrating reduced steroid exposure and toxicity can strengthen a product's benefit-risk profile in support of label claims.

In crowded autoimmune indications, a new therapy with non-inferior efficacy, improved safety, and lower steroid burden can stand out.

Incorporating a steroid-sparing target in the TPP and supporting it with credible data enables sponsors to provide a compelling narrative to both prescribers and regulators.

Quantifying steroid-induced harms, such as fractures, diabetes, or psychiatric events, also creates tangible health economic benefits. HEOR teams can incorporate validated toxicity scores into budget impact models and payer submissions, reinforcing a therapy’s value proposition by highlighting its potential to reduce downstream healthcare costs.

Validated tools like the GTI streamline data analysis through digital platforms that integrate with trial databases. Sponsors, such as Argenx, have partnered with Steritas to deploy the full STOX^® Suite across their immunology programs. CROs are increasingly embedding steroid-toxicity assessments into trial protocols, recognizing their importance for both clinical outcomes and regulatory strategy.

Final word

Steroid-sparing is now a strategic objective in the development of autoimmune drugs. Companies that plan for it early, measure it reliably, and present clear evidence will be better positioned for regulatory success, competitive advantage, and market access.