The Rise of Multi-Indication Therapies: Limitations of Existing Frameworks and Pathways for Future Innovation
Mechanism-based medicine, single-indication systems
Advances in disease biology have made it possible for a single therapy to treat many conditions that share an underlying pathway, a shift that is reshaping oncology, immunology, metabolic disease, and rare disease. Yet the ecosystem that develops, evaluates, prices, and reimburses these therapies has not kept pace. Pricing rules, budget caps, and health technology assessment (HTA) processes were designed around a single drug for a single disease. The result is uncertainty for manufacturers, inefficiency for payers and regulators, and delays for patients awaiting treatment.
Inside this white paper, you’ll discover:
- Why single-indication frameworks fall short
How uniform pricing, external reference pricing, and product-level budget caps quietly penalize each new indication, and why that discourages the discovery of high-value uses.
- The true scale of the multi-indication shift
A grounded look at oncology, immunology, and metabolic disorders, including the platforms and mechanisms driving indication expansion across global markets.
- How global health technology assessment and policy heterogeneity reshape access
A country-by-country view of pricing models, uniform, blended-average, and separate-list mechanisms, plus the downstream effects of the U.S. Inflation Reduction Act on evidence generation.
- Pathways for future innovation
Practical reforms, including joint clinical assessment, modular dossiers, carried-forward safety evidence, and value-aligned pricing, that align value recognition with sustained innovation and timely patient access.
Ready to dive deeper?
Explore a landscape in motion. Download the full white paper for detailed frameworks, cross-market comparisons, and strategic guidance to help you position multi-indication therapies for value recognition and access.
FAQs
A multi-indication (MI) therapy delivers benefit across multiple disease states, therapeutic areas, lines of therapy, or patient subgroups, typically because it targets a common biological pathway rather than a single disease. It reflects a broader shift from developing treatments for individual diseases to targeting mechanisms shared across multiple conditions.
Most systems apply a single price across all indications as they were built for single-indication products. Under uniform pricing, external reference pricing, and product-level budget caps, the value of new high-value indications often isn’t recognized, which can discourage manufacturers from pursuing them, delaying patient access.
Because MI therapies accumulate higher total spending across pooled indications, they are more likely to appear on Medicare price-negotiation lists. Analyses suggest that the resulting economics can reduce incentives to develop additional indications, potentially affecting downstream innovation.
Launched in Europe in January 2025, the Joint Clinical Assessment provides member states with a shared clinical assessment to support national reimbursement and access decisions, reducing the need for duplicate evaluations. It does not assess cost-effectiveness or set value, so national pricing negotiations still occur, but it signals a move toward less redundant, more aligned HTA.
Post-marketing real-world evidence can validate safety across broader populations, support indirect comparisons, strengthen cost-effectiveness models, and reduce payer uncertainty. Planned proactively, it can justify streamlined evidentiary requirements for later indications, focusing resources on what is genuinely new.
Author details
Rachel Gamburg
Senior Associate,
RWE & HEOR
Axtria Inc.
Divya Tamminina
Senior Associate,
RWE & HEOR
Axtria Inc.
Ruihong Wang
Senior Associate,
RWE & HEOR
Axtria Inc.
Coby Martin
Senior Manager,
RWE & HEOR
Axtria Inc.
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