Global health priorities are defined as health issues that require urgent, collaborative international action. In 2025, the World Health Assembly recognized that rare diseases should fall under this category and officially recognized them as a global health priority.
In part one of this interview series on barriers to rare disease treatment access, experts discussed challenges in drug development, including difficulties conducting clinical trials, designing adaptable drugs to meet patient requirements, and contending with funding constraints.
Despite their individual rarity, these conditions have far-reaching consequences for individuals, their loved ones, and the wider healthcare system. The majority of rare diseases affect children and are severe, progressive, and life-limiting in nature. Due to condition-related decline, 30% of children living with rare diseases die before their fifth birthday.
Despite this, people living with rare diseases (PLWRD) lack treatment options, with approved therapies available for only 5% of rare diseases.
To learn more about the importance of addressing the unmet needs of PLWRD, Technology Networks asked an array of experts the same question:
“Across all disease states, patients face inequitable access to treatment. Why does this occur for PLWRD, and why is it particularly important to address?”
In part three of this series, experts will discuss opportunities to advance therapy development and improve treatment access for PLWRD.
“In my view, the key driver of inequitable access, particularly in the European Union, is what happens after the Marketing Authorization Approval (MA). Obtaining MA is only the first step. Pricing and reimbursement decisions happen at a national level; each country has its own Health Technology Assessments (HTA), willingness-to-pay threshold, and budget constraints.
Patients in one country may have access to a life-altering drug within months, while others gain access within years, or never get access. This is especially critical in rare diseases, where there is a high unmet medical need.
I continuously live this paradox: working hard to get one centralized MA approval, followed by 27 different national decisions that inevitably create inequality.
There is hope that the new EU Pharmaceutical Legislation Package will support rapid patient access across nations.”
“For PLWRD, inequity begins long before a treatment reaches the market. It starts in the research phase, when patient-derived cell models don’t exist or don’t represent the true diversity of affected populations. Entire communities are left out of scientific processes before a therapy is ever developed.
We consistently hear from the rare disease foundations we work with that the research tools simply haven’t kept pace with the need. Changing this requires intentional effort to build models that put real patient biology at the center of discovery — not as an afterthought, but as the foundation.”
“This is simply because of the unit economics of disease therapies. At scale, therapies for diseases affecting larger populations make more money. However, there are some areas of rare diseases where the disease burden is high, and the potential treatment is curative.
These areas of rare disease are now seeing a resurgence in novel therapeutics, and high morbidity in young patients is one reason why this area is so important to address.”
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