What Are The Keys To Making Rare Disease Drug Development Sustainable?

Rare disease drug development presents challenges that extend beyond the scientific. With small patient populations, high clinical and manufacturing costs, and complex regulatory pathways, sustainability is an ever-present concern for developers, regulators, and patients alike. In this roundtable discussion, thought leaders from across the biopharma ecosystem share perspectives on how to balance innovation with commercial viability in this unique space.

Contributors highlight the fragility of rare disease programs, pointing to supply chain vulnerabilities, trial recruitment hurdles, and the pressure of delivering advanced therapies such as gene and cell therapies at scale. They emphasize the importance of collaboration among industry, regulators, academia, and patient groups, as well as the role of adaptive trial designs, data harmonization, and platform technologies in de-risking development. Discussions extend to the evolving regulatory environment, where flexibility and innovative review models are critical, and to the payer landscape, where affordability and access remain central to long-term sustainability.

The dialogue also explores manufacturing agility, integrated partnerships, and digital infrastructure as enablers of more resilient and efficient development pipelines. By weaving together diverse perspectives, this article provides a multifaceted look at what it will take to create a sustainable path forward in rare disease drug development.