Starlink Medicines in Jurassic Healthcare Systems—Are We Ready?

We Have Mastered Approvals. We Must Master Access

The biotech and regulatory communities have made remarkable progress. Dozens of cell and gene therapies have been developed and are now approved across the United States, Europe, and several emerging markets. The manufacturing complexities of viral vectors, gene editing platforms, and autologous therapies are being steadily overcome.

But the work remains unfinished. Regulatory approval is only the first milestone—not the final one. For the 90% of the world that still lacks meaningful access to these genomic innovations, hope remains a distant promise.

In Brazil, the potential for scaling gene therapies in resource-constrained environments has been demonstrated, alongside the fragility of sustaining such progress when commercial models are misaligned with local health system realities. ¹ The Ministry of Health implemented a risk-sharing agreement with Novartis to enable access to Zolgensma (onasemnogene abeparvovec), a gene therapy for spinal muscular atrophy, within the public health system (Sistema Único de Saúde, SUS). Under this arrangement, payments are contingent upon treatment effectiveness over a 4-year period, representing an innovative precedent for the integration of high-cost therapies into national healthcare frameworks.^2,3

In Europe, the heterogeneity of over 27 national healthcare systems—with their distinct pricing mechanisms, budgetary limitations, and evidentiary requirements—has necessitated the development of flexible access pathways. Initiatives such as “day-one access” agreements ⁴ and risk-sharing frameworks incorporating ongoing real-world evidence collection illustrate an emerging ethos of partnership between developers and health authorities to accelerate the adoption of genomic medicines.^5–7

In Africa and Asia, access to gene therapies continues to be impeded by procurement challenges, infrastructure deficits, and legacy delivery models optimized for chronic care rather than curative interventions. Nevertheless, recent experience in Egypt demonstrates that such barriers are surmountable.^8,9 Through coordinated efforts between public and private stakeholders, access to Zolgensma was achieved for patients without prior therapeutic options, reinforcing Nelson Mandela’s assertion that “it always seems impossible until it is done.” These examples highlight the importance of co-creation, adaptability, and sustained investment in health system readiness to democratize access to transformative genomic technologies.

These lived experiences raise a fundamental question: are our systems, our incentives, and our social contracts ready for the transformative potential of these therapies?

What Do We Value—and Who Decides?

We must confront a deeper societal reckoning: what does society really value in health innovation? And how are we choosing to reward it?

Traditional models of pricing and reimbursement were built for chronic, recurring treatments. A gene therapy that cures a disease with a single dose not only disrupts the biology of disease, it disrupts the economics of medicine. Many health systems and payers struggle to reconcile the high upfront cost of these therapies with their long-term value. The result is a policy paradox: society lauds innovation but balks at paying for it.

This misalignment raises critical questions. Should a one-time curative treatment be priced based on the value it delivers over a lifetime? Should payment be tied to performance and patient outcomes? Should public-private partnerships take greater risks to catalyze uptake in lower-income geographies?

We need a new language—and new frameworks—for talking about value. Traditional models such as cost-effectiveness analysis and quality-adjusted life years have long dominated pricing and reimbursement decisions. However, these frameworks often may undervalue one-time curative therapies by focusing narrowly on short-term costs rather than capturing lifetime health gains, societal productivity, or the alleviation of caregiver burden. Budget impact models further compound the problem by emphasizing immediate fiscal pressures over long-term system savings. A real societal debate is urgently needed—not just among technocrats and policymakers, but also among patients, ethicists, economists, and civil society—about how we recognize, reward, and share the value of transformative innovation. We must collectively reimagine how value is co-created, distributed, and sustained in an era where medicine can alter the natural history of disease.

Beyond Price: Reimagining Systems

Pricing is only one barrier. Even if every gene therapy were free tomorrow, most of the world would still struggle with access and uptake. Why? Because health systems weren’t built for the complexity of delivering genomic medicine. Many of these therapies require specialized infrastructure, including cryogenic supply chains, trained personnel, genetic diagnostic capacity, and longitudinal follow-up. Without investment in delivery systems, reimbursement reform, data infrastructure, and patient navigation, scientific breakthroughs will remain siloed in wealthy nations and elite academic centers.

Access must be redefined—not as charity or an afterthought but as an integral component of innovation. The responsibility for this lies not only with industry but with a broader ecosystem, including academic institutions, regulatory bodies, funders, and governments. It is essential to recognize that the actors within the genomic medicines space must work in concert, from early research to regulatory approval and through to the delivery of treatments to patients. However, the issue extends beyond the scientific and commercial sectors—it is also about global governance through the collective political will of nations, international organizations, and the private sector to ensure that science and health systems are aligned with the societal needs they seek to address. A global social compact must be established between these diverse stakeholders, creating a shared responsibility for equitable access, while fostering collaboration across borders to scale up these transformative therapies. This compact would not only aim to democratize access but also ensure that the governance structures in place can support the broader moral, political, and economic imperatives of these transformative advancements.

What if, for instance, the same urgency and capital industry devoted to regulatory acceleration were applied to health system readiness? What if access outcomes were included in how we measure scientific success? What if the architecture of global R&D incorporated outcome-based pricing, technology transfers, diagnostics, such as newborn screening, and health workforce training from day one?

Toward a Global Social Compact for Genomic Equity

As the Global Observatory 2025 International Summit rightly emphasizes, emerging biotechnologies sit at the intersection of values, ethics, and global justice. The promise of genome editing and regenerative medicine cannot be realized through molecules alone. It requires a rethinking of what it means to be human and what we owe one another as a global community.

We must craft a new social compact between biotechnology, healthcare, and society—one that acknowledges the asymmetrical distribution of scientific benefits and burdens across countries, communities, and generations. Such a compact would address the following priorities:

Embed equity in innovation. Funding mechanisms, clinical trials, and product design must reflect the needs of historically marginalized populations.

Conclusion: From Miracle to Momentum

The age of genomic medicine has arrived. But miracles alone are not enough. We must now build the structures—moral, economic, and logistical—that turn scientific miracles into everyday realities for people everywhere.

This is not only a question of science or policy. It is a question of who we want to be. Will we be the generation that cured the incurable for the few? Or will we build systems bold enough to deliver those cures to the many? Genomic satellites are in orbit. The question is, can our systems launch to meet them.