Multi-indication medicines highlight both the promise of scientific innovation and the need for thoughtful approaches to patient access.
Multi-indication medicines have the potential to transform care by addressing multiple conditions with a single therapy, often leveraging well-understood mechanisms of action and safety profiles. This approach can expand patient access to safe and effective treatment options.
While already common in oncology, multi-indication medicines continue to face significant challenges across the development and access pathway, from regulatory reviews to payer assessments and clinical adoption. As their use expands into other therapeutic areas, such as immunology and inflammation, these challenges are likely to become even more complex.
Timely and coordinated solutions will be essential to unlock the full potential of multi-indication medicines beyond oncology. A proactive, multi-stakeholder response is required to ensure these innovations can deliver maximum value for patients, health systems and society.
We have therefore been working on a project to address this topic*.
Completed work
Activities to date include:
- Literature review and expert interviews to answer the following research questions:
- What is the key value of multiple indication products recognised in the literature?
- What are the current access policies and regulations for multiple indication products in EU4 + UK?
- What are the key P&R challenges for multiple indication products currently experienced in EU4 + UK in practice, within the existing policy systems?
- What are the recommended best practices / potential solutions?
- Wider engagement through a panel and stakeholder engagement with ~800 participants at ISPOR Europe 2024
Deliverables to date
ISPOR Europe panel report – Pricing and reimbursement of multiple indication medicines: Can a balance be found between different stakeholder perspectives to optimise value and access for patients, while ensuring sustainable and affordable innovation?
March 2025
This ISPOR Europe 2024 panel report explores pricing and reimbursement (P&R) of multiple medicines in Europe. Work has culminated from the panellists and audience of ~800 participants, where they discussed key challenges and forward-looking solutions for multi-indication products…
Paper – Supporting patients with underserved autoimmune diseases: challenges and opportunities for innovative medicines
September 2025
Effectively addressing patient and public health needs – the role of policy. Over the past several decades, public health systems around the world have contended with a series of challenges that reflect the evolving landscape of healthcare needs…
Engagement with Dutch stakeholders on Netherlands specific challenges and solutions
September 2025
Pricing and reimbursing multi-indication therapies: how do we get it right? That was the central question at this week’s hollandbio Market Access Expert meeting, organized by argenx with support from Dolon…
Publication coming soon
December 2025
Ongoing work*
We are working with a team of health economist experts to explore one possible solution, the multi-year, multi-indication agreement, to understand if it provides added benefit for better managing multi-indication medicines.
One solution option, the multi-year multi-indication (“MYMI”) agreement, has been recognised as having potential, but requires deeper investigation to understand its implementation feasibility and benefits over existing approaches
MYMI can be viewed as a holistic framework to enable an agreement between manufacturers and payers across multiple indications and years, creating a comprehensive approach to value assessment, pricing and reimbursement for a product’s approved and future indications
MYMI can be viewed as a holistic framework to enable an agreement between manufacturers and payers across multiple indications and years, creating a comprehensive approach to value assessment, pricing and reimbursement for a product’s approved and future indications- We will soon be uploading a questionnaire on the topic for any interested stakeholders to complete.
- We will be discussing the topic during a panel session at the World Orphan Drug Congress in Amsterdam in October, we hope to see you there.
- Panel: Expanding patient access to multi-indication medicines in Europe through innovative agreements
- 15:00, 28 October 2025
- World Orphan Drug Congress Europe 2025 | Agenda
Other resources
Related work from collaborating partners coming soon.
Invitation to participate
We are working to collaborate and understand from all stakeholders’ perspectives what feasible solutions for multi-indication medicines could look like. As this work has evolved, we recognise the need to create a wider consortium that can contribute to and help drive the conversation on important topic.
If you would like to be kept updated, collaborate or express interest as an industry sponsor, please visit:
bit.ly/multi-indication
*Disclaimer: work and deliverables to date have been funded by argenx