MFN Drug Pricing and Global Access Challenges: Why Chargeable Expanded Access is More Critical Than Ever Before

As global pricing pressures like the U.S. MFN policy reshape launch sequencing and reference pricing risk, biopharma must rethink early access policy. Expanded access can facilitate a strategic, sustainable solution to address gaps in global access to medicines, while pressures resulting from policies like the U.S. MFN reshape launch sequencing and market access strategies. When fully integrated into product development and lifecycle planning, chargeable EA can support in a way that can mitigate risks like MFN exposure, enable earlier patient access, enhance evidence generation strategies, and create viable access pathways where full commercialization is delayed or infeasible.

Navigating MFN policy and global nuances to develop sustainable pre-approval access strategies

In today’s rapidly evolving pharmaceutical landscape, global policies such as the U.S. Most Favored Nation (MFN) drug pricing model are reshaping timelines, feasibility, and strategies for global market access.  The MFN policy, which links U.S. drug prices to the lowest rates paid in other OECD nations, has heightened global reference pricing risk potentially leading to delayed launches, restricted geographic rollout, or revised market access strategies. This ultimately may reshape how and when biopharma companies bring a new medicine to market.

While global policies are challenging commercial feasibility and timelines, the demand for lifesaving and life-altering treatments is growing. This is where pre-approval or expanded access, particularly chargeable models, becomes more important than ever, both to bridge the gap between pre-approval availability and commercialization while also facilitating access in markets where traditional commercialization may not be feasible. While charging for unlicensed medicines can only be done where permitted and in accordance with local laws and regulations, this approach enables earlier and broader global patient access in a manner which does not jeopardize resources needed for ongoing development activities.

As a result of these dynamics, providing clarity on pre-approval access options can be part of a deliberate, early component of global market access planning. By leveraging chargeable expanded access into broader market access strategies, biopharma can address global unmet needs sustainably while mitigating MFN-related risks.

How global delays, emerging market challenges, and MFN pricing are reshaping medicine access strategies

Delays in access to medicines for serious and life-threatening disease remain a persistent challenge.  Even within Europe, the time between central marketing authorization and national availability can stretch several years, largely due to pricing and reimbursement hurdles.¹ MFN promises to exacerbate this; by tying U.S. Medicare rates to the lowest prices in other developed countries, MFN puts pressure on biopharma companies to avoid low international benchmarks that could slash their American revenues. To sidestep this, companies may hold off on launching new drugs in lower-price European markets or push for higher list prices, further dragging out reimbursement discussions and widening access gaps across the region. This ripple effect from MFN policies could mean even longer waits for patients in Europe, turning existing hurdles into bigger roadblocks for timely access to medicines.

Emerging markets present a different but equally complex challenge. These regions are home to 85% of the global population and offer substantial growth potential, but due to regulatory complexity, budget challenges, and infrastructure limitations have faced median delays of approximately 7-8 years from the first global launch of new medicines.^{2, 3, 4}   Furthermore, the expansion of clinical trials into emerging markets, driven by cost-efficiencies and diverse patient populations, highlights a critical tension.  While these regions are contributing significantly to trial data, historical patterns show that trial participation has not reliably translated into timely commercial access. ⁵

That said, emerging markets sit outside of the MFN reference basket, which means that biopharma can pursue flexible pricing models without triggering U.S. price reductions. This ensures companies can roll out chargeable expanded access pathways to advance patient access, without the need to provide the drug for free, and at the same time, collect real-world data that can strengthen the push for long-term market entry and support impactful health policy change in these regions.

Biopharma companies also face increasing pressure to improve their Environmental, Social and Governance (ESG) performance and Access to Medicines Index (ATMI) rankings. Strong equitable access initiatives not only enhance investor appeal and corporate reputation but also align with broader commitments to reduce global health disparities. ^{6, 7}

These global trends highlight the critical need for biopharma to adopt innovative solutions which integrate expanded access strategies with their broader market-access plans, positioning them as viable alternatives to traditional distribution or out-licensing arrangements to optimize global reach and economic viability, while navigating pricing harmonization risks. ^{8, 9}

Why chargeable expanded access is becoming essential to bridge access gaps

Under traditional expanded access pathways, biopharma companies provided medicines free of charge. While well-intentioned, this approach places significant resource burdens on companies and ultimately hinders the fullest utilization and impact of expanded access programs.

In parallel, awareness and demand for expanded access have increased in recent years. In the United States, the 21^st Century Cures Act introduced greater transparency requirements and public awareness about expanded access, making program availability and procedures more visible to patients and physicians.¹⁰  At the same time, research has shown the demand for expanded access is positively associated with clinical trial activity, meaning that as clinical trial activity expands globally so too does expanded access. ¹¹ In a world of growing global needs and constrained commercialization, the free-of-charge model can impose unsustainable resource burdens on sponsors leading to a decision to limit availability for free of charge product under expanded access requests.

Chargeable expanded access models can address this by integrating sustainability in the approach.  Early revenue streams help offset manufacturing, distribution, and development costs, while structured programs facilitate market entry through importation and distribution networks while working towards a full commercial infrastructure.  Alongside real-world data collection from diverse patient populations and healthcare settings, expanded access can generate valuable insights that refine clinical development, regulatory strategies, and future market access plans.

Local healthcare systems may as a side effect, also benefit. Expanded access requires a dedicated infrastructure and consequently incentivizes advanced investments in specialized facilities, trained personnel, and treatment protocols. Physicians gain hands-on experience with novel therapies earlier than otherwise possible, which may support improved patient outcomes and allow for evidence generation on the impact of treatment on the local disease burden. This practical exposure strengthens the healthcare ecosystem and supports long-term advancements.

Recent policy developments in several emerging markets reflect growing interest in more sustainable access frameworks. For example, Malaysia’s National Policy for Rare Diseases (launched in February 2026) underscores a shift towards sustainable access models, aiming to provide equitable and timely access to life-saving treatments in the country^{. 12} This national framework creates structured opportunities for chargeable models to support national goals.

What are country-specific approaches and variations to chargeable expanded access models?

There is no one-size-fits-all model for chargeable expanded access. The approach and feasibility vary across countries and requires careful navigation of regulatory processes, importation requirements, and reimbursement mechanisms. Developing a strategy and defining the scope of a chargeable expanded access strategy therefore demands that companies understand the feasibility, requirements, and associated risks on an individual country basis.

In some jurisdictions, very well-defined and integrated reimbursement pathways exist.  France’s expanded access pathways, Autorisation d’accès précoce (AAP) and Accès Compassionnel (AAC), provide full reimbursement through the national social security system. Biopharma is reimbursed at a freely set price subject to rebate and claw back mechanisms.¹³ Another common example is Italy’s Law 648/1996 which enables reimbursement by the national health system for products included on AIFA’s national “Lista 648” following favorable opinion by AIFA’s Technical-Scientific Committee (CTS).¹⁴

Other countries are also adopting integrated models, recognizing the urgency to address local unmet medical needs while offering sustainable options for biopharma. Most recently Belgium introduced “Early and Equitable Fast Access System” (EEFA), effective January 2026. This nationally funded system provides temporary reimbursement for promising therapies provided through compassionate use or medical needs pathways, with the payor engaged from the outset in an end-to-end process.

These models reflect a broader trend toward integrating payors early in the expanded access decision-making process and structurally incorporating real-world data collection. In many respects, they resemble managed entry agreements—conditional arrangements between manufacturers and payors that manage clinical or financial uncertainties through mechanisms like risk-sharing, performance-based pricing, data collection protocols, volume caps, and payback clauses. Overall, this signifies a fully integrated approach that enhances HTA decisions, mitigates government spending risks, and enables informed country-specific pricing strategies, while advancing patient access.

In other countries, however, chargeable expanded access relies on less structured frameworks, including private insurance, hospital or regional budgets, specialized government funds, or even through litigation. The feasibility to gain reimbursement under these various frameworks in the context of expanded access often depend on a cost-benefit analyses that evaluates the product’s price, available clinical data, expected treatment duration, and the competitive landscape. Furthermore, these reimbursement mechanisms often require that the drug is licensed in at least one market, signifying confirmation by an esteemed regulatory agency of the demonstrated safety and effectiveness of the treatment.

Another possibility for chargeable expanded access, in certain settings, is out-of-pocket payment by patients or caregivers, although the viability of this approach is significantly impacted by the nature of the disease (acute or chronic) and value of the product. Moreover, while this model can enable access in regions lacking formal reimbursement pathways, it raises ethical considerations around equity and affordability potentially limiting availability to those with sufficient financial resources, underscoring the need for balanced strategies.

It is important to note that in certain countries expanded access regulations may explicitly prohibit charging, for example, Germany’s Arzneimittel-Härtefall-Verordnung (compassionate use program- CUP) pathway requires free drug provision.¹⁵ However, Germany’s importation pathway allows biopharma to charge for drugs approved elsewhere. This dynamic creates strategic choices for biopharma: establish a free CUP for early penetration and data generation opportunities or postpone the approach to take advantage of revenue opportunities in parallel with advancing patient access.

Given the diversity of regulatory and reimbursement environments, a hybrid approach that offers expanded access in markets where reimbursement is feasible, and free-of-charge elsewhere, can enable a cost-neutral, sustainable global strategy that biopharma may find appealing.

What are the key pillars for successful implementation of sustainable expanded access?

In the dynamic biopharma landscape, mastering sustainable expanded access programs requires more than regulatory compliance or operational capacity. It demands a coordinated strategy that aligns ethical considerations, market access planning, operational infrastructure, and financial sustainability.

As biopharma companies increasingly integrate expanded access into broader global access strategies, several operational pillars have emerged as critical for successful implementation. These pillars help ensure that programs remain compliant, scalable, and capable of delivering meaningful patient access while supporting long-term sustainability.

Implementing these key pillars helps biopharma companies:

• Turn expanded access models into a core part of their global market access plans.
• Improve global patient access and address global health disparities.
• Reduce reference pricing risks more effectively
• Generate a reliable supporting revenue stream in key regions, like emerging markets, which otherwise may not have been appreciated given limited commercial footprint.

Key takeaways: how biopharma can drive global impact through chargeable expanded access

As pricing policies such as MFN and broader international reference pricing dynamics continue to influence launch sequencing and commercialization strategies, biopharma companies are increasingly facing difficult trade-offs between pricing considerations and timely patient access. These pressures are forcing companies to rethink how innovative medicines reach patients during the period between clinical development and full commercial launch.

Chargeable expanded access represents a sustainable solution for biopharma to enable scalable global access for innovative medicines. Effective implementation demands strategic foresight: early stakeholder engagement, rigorous regulatory navigation, careful pricing, and robust risk management.  Systems for oversight, streamlined operations, insight generation, and physician support are essential to maximize impact.

Ultimately, when managed with intention, chargeable expanded access can serve as a cornerstone of sustainable global health equity.  It empowers the biopharma industry to innovate responsibility, delivering transformative therapies broadly and sustainably for patients who need it most, while driving systematic change within healthcare systems by promoting joint efforts between stakeholders to foster collaborative policy advancements and directing investments more effectively toward high-impact areas.

myTomorrows, a leading expert in chargeable expanded access, partners with companies of all sizes to develop tailored strategies that not only enable sustainable, patient-centered programs, but also help to de-risk product launches and create meaningful stakeholder engagement across clinical, commercial, and regulatory objectives. Through operational excellence and local, hands-on support for healthcare providers through a Medical Science Liaison network, we help implement scalable programs that make a lasting difference for patients and partners alike.

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