A Dolon Institute publication
The pricing and access environment for rare disease and oncology medicines is entering an increasingly complex period. Rising pressure on drug prices, changing government priorities, major shifts in healthcare spending and the implementation of new assessment frameworks are all reshaping the conditions in which innovative medicines need to demonstrate value and secure patient access. Against this backdrop, manufacturers will need to anticipate change earlier and take a more integrated approach to pricing and access strategy.
2025 themes for pricing and access success for rare disease and oncology medicines, from the Dolon Institute, identifies six themes that the Value & Pricing Access (VPA) team expects to have a significant impact on pricing and access success during 2025.
The first is rising pricing tension. Pharmaceutical R&D is becoming more expensive and time-consuming, while success rates and revenues have declined. At the same time, inflation, demographic pressures and constrained healthcare budgets are driving policymakers towards greater intervention on medicine pricing. The result is an increasingly challenging environment in which companies must protect the value of successful innovations while demonstrating that pricing is sustainable for healthcare systems.
The second theme is protectionist policies, as governments seek to balance pharmaceutical cost containment with the desire to attract domestic investment, clinical research and manufacturing. This creates a more fragmented global environment in which pricing and access strategies need to account for increasingly country-specific political and industrial priorities.
The paper also considers the GLP-1 wave and its potential implications beyond obesity. With spending on GLP-1 therapies expected to grow substantially, competition for healthcare budgets could increase across therapeutic areas. Rare disease medicines may be particularly vulnerable if policymakers prioritise treatments that benefit larger patient populations, creating a need to reinforce the case for continued investment in areas of high unmet need.
For oncology, the challenge is increasingly one of crowding and differentiation. Rapid innovation has created highly competitive treatment landscapes, with multiple modalities, biomarker-driven approaches and combination therapies competing within the same indications. Successful access strategies will therefore need to consider positioning, differentiation, combination pricing and the broader franchise across indications.
The fifth theme is Joint Clinical Assessment (JCA) implementation. While EU-level processes are becoming clearer, significant uncertainty remains around how Member States will use JCA outputs, how PICOs will be consolidated and whether evidence requirements will accommodate rare diseases where uncertainty is inherently difficult to resolve.
Finally, AI and efficiency is highlighted as an emerging opportunity for the access function. Beyond current applications such as systematic literature reviews, data extraction and visualisation, the paper points towards predictive analytics, automated market access planning and more efficient development of value communication materials.
Together, these themes highlight the need for more forward-looking, evidence-led and strategically integrated pricing and access approaches.