A Dolon Institute Publication
Scientific advances are creating new opportunities to develop medicines that target shared biological pathways across multiple autoimmune diseases. For patients with underserved conditions, these multi-indication medicines could address significant unmet need and expand access to effective treatment options. However, bringing these medicines to patients presents complex challenges across development, health technology assessment (HTA), pricing and reimbursement (P&R), and ultimately patient access.
This paper explores whether current European approaches to managing multi-indication medicines are fit for purpose and considers alternative approaches that could better balance patient access, payer affordability and manufacturer incentives to invest in additional indications.
Why is this an important issue?
Multi-indication medicines are particularly challenging in autoimmune diseases because different conditions can overlap within the same patient, while measures of treatment value vary considerably between indications. This makes it difficult to attribute medicine use to a specific indication and complicates the implementation of indication-based pricing and outcomes-based agreements. At the same time, conventional pricing approaches can result in price erosion as new indications are added, potentially reducing incentives to develop and launch medicines for smaller, underserved patient populations.
The research combined a targeted literature review, European case studies, interviews with payer and market access experts across the UK, France, Italy and Spain, and insights from an ISPOR Europe 2024 stakeholder panel involving approximately 800 participants. The findings were also assessed against a theoretical pricing framework designed to explore how pricing mechanisms can align manufacturer incentives with overall patient benefit.
What does the research show?
The analysis highlights limitations in both existing and theoretical approaches to multi-indication pricing. While pure indication-based pricing (IBP) offers the clearest theoretical alignment between price and value, practical implementation can be difficult because it requires sophisticated infrastructure to track utilisation and reconcile payments by indication. Where IBP has been implemented, lengthy negotiations can also delay access to subsequent indications.
The paper therefore highlights multiyear multi-indication (MYMI) agreements as a potentially more pragmatic approach. Rather than negotiating each indication independently, MYMI agreements establish a framework covering multiple indications and years, enabling predefined processes for assessing and reimbursing future indications.
Evidence from Belgium demonstrates the potential impact: MYMI agreements for PD-(L)1 medicines accelerated access to new indications by more than 550 days. Such agreements could also reduce administrative burden, improve budget and price predictability, and support broader and faster launches.
What does this mean for stakeholders?
For patients, more pragmatic approaches could accelerate access to innovative treatments across underserved autoimmune diseases.
For payers, MYMI agreements offer the potential for greater financial predictability and streamlined assessment while retaining mechanisms to reassess value as evidence develops.
For manufacturers, they could provide greater certainty around the commercial environment for subsequent indications and strengthen incentives to invest in developing treatments for smaller patient populations.
However, MYMI agreements are not without challenges. They require stakeholders to manage uncertainty around future indications, clinical benefit, treatment alternatives and timing. The paper therefore calls for greater multistakeholder collaboration to establish practical frameworks and determine where these agreements can work effectively across different country contexts.
The research points towards a need to move beyond one-size-fits-all pricing approaches and explore flexible, collaborative models that can support sustainable access to the full potential of multi-indication medicines.
Read the article here.
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