Systemic underfunding of rare disease research enables corporate monopolies on life-saving treatments like Arikayce

Indigenous and traditional medicine systems have long addressed chronic lung diseases through holistic, low-cost interventions, yet these are excluded from mainstream narratives about rare disease treatments. The focus on Arikayce reflects a biomedical paradigm that prioritizes corporate-controlled pharmaceuticals over community-based care, erasing centuries of empirical knowledge. The marginalization of these systems is a form of epistemic violence, reinforcing the dominance of Western medical frameworks in global health governance.

The Orphan Drug Act (1983) was designed to incentivize rare disease research but has been exploited by corporations to secure monopolies, as seen with Arikayce’s accelerated approval pathway. This mirrors historical patterns where regulatory loopholes enable profit extraction from public-funded research, such as the Bayh-Dole Act (1980) and its unintended consequences for drug pricing. The current system perpetuates a cycle where public investment in basic science is privatized, leaving patients and taxpayers bearing the costs of corporate monopolies.

Cross-culturally, the emphasis on pharmaceutical solutions for rare diseases reflects a Western biomedical model that prioritizes individual treatment over community health. In contrast, many African and Asian traditional systems integrate spiritual, dietary, and communal approaches to chronic illnesses, reducing reliance on high-cost drugs. For example, the use of *Tinospora cordifolia* in Ayurveda for respiratory conditions offers a lower-cost alternative, though it lacks the regulatory and financial backing of Western pharmaceuticals. These systems are systematically devalued in global health policy.

Arikayce’s efficacy is supported by clinical trials, but the accelerated approval pathway relies on surrogate endpoints (e.g., sputum conversion) rather than long-term patient outcomes, raising questions about its real-world effectiveness. The drug’s high cost ($120,000/year) is justified by the rarity of the disease, yet this pricing model is not grounded in scientific evidence of cost-effectiveness. Publicly funded research (e.g., NIH studies on *Mycobacterium avium* complex) underpins Arikayce’s development, yet this contribution is obscured in corporate narratives.

Artistic and spiritual traditions often frame illness as a manifestation of imbalance, whether in the body, community, or environment, offering a counter-narrative to the mechanistic view of rare diseases. For instance, the Navajo concept of *Hózhǫ́* (harmony) emphasizes holistic healing, while African spiritual practices incorporate herbal remedies into rituals for respiratory ailments. These perspectives are rarely integrated into biomedical approaches, which treat diseases as isolated, technical problems requiring pharmaceutical intervention.

If current trends continue, the rare disease treatment market will remain dominated by a handful of corporations, with prices continuing to rise as monopolies tighten. Scenario modeling suggests that without structural reforms (e.g., mandatory price negotiations, open-source drug development), access to treatments like Arikayce will remain inequitable, particularly in low-resource settings. Alternative models, such as the WHO’s Global Observatory on Health R&D, could prioritize public funding for rare diseases, but these face resistance from pharmaceutical lobbies.

Marginalized communities, including low-income patients, Indigenous groups, and people in Global South countries, are disproportionately excluded from rare disease treatments due to cost and lack of infrastructure. The focus on Arikayce’s benefits ignores the fact that most rare disease patients worldwide lack access to basic diagnostics, let alone advanced therapies. Additionally, the narrative centers corporate and investor perspectives, sidelining patient advocacy groups that advocate for systemic reforms over individual drug approvals.