RWE and HEOR

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:

  1. 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.

  2. 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.

  3. 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.

  4. 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.

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