Cem Zorlular
CEO
Er-Kim

When we think about access inequity, we often have the immediate image of a low-income nation unable to afford essential medicines. While this may be partly true, it’s not a problem exclusive to this demographic. IVQIA’s EFPIA Patients W.A.I.T. Indicator 2024 Survey highlights that many European countries also face significant challenges in making innovative medicines available. The issue is bigger than we realize, but it’s not without a solution.

Access inequity is, in general, a death by a thousand paper cuts. It’s rarely only about unaffordability, but rather a multitude of smaller, interconnected operational and bureaucratic challenges that collectively hinder patients from receiving life-saving treatments they desperately need.

For pharmaceutical executives, access inequity is a fundamental business issue. When only a fraction of the target population can access a product, the remaining portion represents a missed commercial opportunity. Understanding and addressing the hurdles of drug access is not merely a humanitarian gesture; it’s a critical strategic necessity to realize the value of R&D investment, enhance corporate reputation, and secure long-term global growth.

Nuances of access inequity beyond the price tag

The landscape of access inequity is highly fragmented and different across various therapeutic areas. Essential medicines, rare disease medicines, oncology drugs, and chronic disease therapies each present unique access challenges with distinct market dynamics and regulatory pathways. For instance, while over 60% of global pharmaceutical revenue traditionally comes from the US, in the highly specialized area of rare disease, this figure drops to just 35%, even as the price differential between the US and Europe can be significant. This figure highlights that the issue extends beyond simple purchasing power.

In my experience, to effectively address and capitalize on these complex issues, the focus should be on specific segments and incrementally resolving issues, rather than attempting a broad, uncoordinated “boil the ocean” approach. We need to rethink our assumptions about affordability, as our current perspective might be a significant blind spot for growth.

Realizing the full value of R&D investment

Contrary to conventional wisdom, there are surprisingly high levels of affordability in lower-income countries for high-unmet-need medicines, particularly in rare diseases. While hard data is scarce, anecdotes suggest that these nations, despite limited overall healthcare budgets, can allocate significant resources and have the capacity to pay top dollar for certain rare disease treatments. This isn’t an issue of limited budgets, but one of different priorities. For many countries, spending focuses on what they perceive as a worthwhile investment, which may be substantially different to what we see in the US. This is why some nations will invest heavily for rare disease treatments while simultaneously allocating zero funds for blockbuster chronic treatments that are commonplace in the US.

While these countries' healthcare systems are not perfect, we often underestimate their willingness to spend significant funds on what they consider a priority. They may have limited resources, but they have a surprising capacity to pay a high price for specific, high-unmet-need medicines, particularly for rare diseases.

Rare Disease Funding: A Global Reality

While we often think of access to medicine as a problem for low-income countries, these issues exist everywhere, including in developed nations. The reason isn’t a single, major policy failure, but a series of a "thousand paper cuts"—a collection of small, systemic hurdles that make it impossible for small-to-midsize biotech companies to operate effectively. However, many countries are finding innovative ways to fund expensive rare disease treatments, demonstrating that a lack of funding isn't always the issue.

• Uzbekistan and Egypt: These governments have made high-cost therapies like Zolgensma for Spinal Muscular Atrophy (SMA) a national priority through dedicated budget allocations and presidential initiatives, ensuring free access for patients.
• Argentina: Argentina used an outcomes-based deal for Zolgensma, paying a discounted price only if the therapy works.
• Brazil and Thailand: Brazil is manufacturing its own CAR-T cell therapy to cut costs for cancer patients, while Thailand has included expensive enzyme replacement therapy for Gaucher disease in its universal health coverage.
• India: India launched National Policy for Rare Diseases (NPRD) that provides a one-time grant for rare disease treatments, marking a first step towards public funding for orphan drugs, even with its limitations.

Pharmaceutical companies are generally flexible in terms of pricing structures, provided such arrangements do not negatively impact broader market dynamics. The net price of rare disease drugs can sometimes be higher in lower-income countries than in more developed markets. This is not due to exploitation, but to the absence of the sophisticated negotiation infrastructure found in larger, established markets. While managed access programs are common in the West, their lack of presence in these regions necessitates a proactive and transparent approach to pricing.

The thousand paper cuts theory and operational complexity

Innovation, especially in rare disease treatments, comes from small, agile biotechs with limited global commercial infrastructure. This brings us back to the “thousand paper cuts” theory of access inequity. Beyond pricing, there is an overwhelming operational complexity involved in access. Adding to this is the intricate process of actually supplying these treatments across diverse regulatory and logistical landscapes. This is where the issues truly pile up:

• Export and Import Permit Procedures: This is often a very difficult process. Even affluent countries with funds to pay top dollar for medications face administrative complexity and delays in simply importing products. This highlights a critical need for small to midsize biotechs to be able to generate the necessary documents to comply with the requirements.
• Compliance: Navigating diverse regulatory landscapes adds immense time, cost, and bureaucratic burden, delaying patient access. It is necessary to understand national regulatory frameworks and country-specific submissions and interpretations in order to avoid delays in patient accessibility to life-altering treatments.
• Quality Control & Supply Chain Integrity: Maintaining the integrity and quality of products across borders presents ongoing challenges for less developed logistics networks. Upholding stringent GDP standards throughout the supply chain, from manufacturing to patient delivery, is critical for retaining product quality and ensuring complete traceability.
• Payments & Financial Logistics: International financial transactions, currency controls, and varied payment systems can be a challenge with inefficiencies and delays, affecting the consistent supply of treatments. As such, a different commercial and financing approach is needed – one that focuses on cash and risk mitigation as opposed to EBITDA.

Individually, these hurdles may seem minor, but their combined effect can transform what should be a straightforward process into a logistical nightmare. For pharmaceutical executives, addressing these “paper cuts” with a strategic approach and innovative operational models is not just about fulfilling a humanitarian mission; it is about transforming untapped patient populations into viable markets, driving sustainable growth, and strengthening a competitive advantage in a globalized industry.